Compounds > interferon-gamma

Page last updated: 2024-12-08

interferon-gamma

Description Research Excerpts Clinical Trials Roles Classes Pathways Study Profile Bioassays Related Drugs Related Conditions Protein Interactions Research Growth

Description

aplysamine-1: from sponge Aplysia sp.; structure in first source [Medical Subject Headings (MeSH), National Library of Medicine, extracted Dec-2023]

Cross-References

ID Source	ID
PubMed CID	362025
CHEMBL ID	380697
MeSH ID	M0011490

Synonyms (13)

Synonym
nsc-625525
aplysamine-1
nsc625525
n-(3-(2,6-dibromo-4-(2-(dimethylamino)ethyl)phenoxy)propyl)-n,n-dimethylamine
3-(2,6-dibromo-4-(2-(dimethylamino)ethyl)phenoxy)-n,n-dimethyl-1-propanamine
3-[2,6-dibromo-4-(2-dimethylaminoethyl)phenoxy]-n,n-dimethyl-propan-1-amine
CHEMBL380697 ,
aplysamine
bdbm50177729
159026-30-9
3-(2,6-dibromo-4-(2-(dimethylamino)ethyl)phenoxy)-n,n-dimethylpropan-1-amine ,
3-[2,6-dibromo-4-[2-(dimethylamino)ethyl]phenoxy]-n,n-dimethylpropan-1-amine
AKOS040747833

[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Protein Targets (2)

Inhibition Measurements

Protein	Taxonomy	Measurement	Average	Min (ref.)	Avg (ref.)	Max (ref.)	Bioassay(s)
Histamine H3 receptor	Rattus norvegicus (Norway rat)	Ki	0.2490	0.0001	0.2963	8.5110	AID260990
Histamine H3 receptor	Homo sapiens (human)	Ki	0.0300	0.0001	0.3399	8.5110	AID1545099; AID260989; AID599203
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Biological Processes (5)

Process	via Protein(s)	Taxonomy
neurotransmitter secretion	Histamine H3 receptor	Homo sapiens (human)
G protein-coupled serotonin receptor signaling pathway	Histamine H3 receptor	Homo sapiens (human)
G protein-coupled receptor signaling pathway, coupled to cyclic nucleotide second messenger	Histamine H3 receptor	Homo sapiens (human)
chemical synaptic transmission	Histamine H3 receptor	Homo sapiens (human)
adenylate cyclase-inhibiting G protein-coupled acetylcholine receptor signaling pathway	Histamine H3 receptor	Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Molecular Functions (3)

Process	via Protein(s)	Taxonomy
histamine receptor activity	Histamine H3 receptor	Homo sapiens (human)
G protein-coupled acetylcholine receptor activity	Histamine H3 receptor	Homo sapiens (human)
G protein-coupled serotonin receptor activity	Histamine H3 receptor	Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Ceullar Components (4)

Process	via Protein(s)	Taxonomy
plasma membrane	Histamine H3 receptor	Homo sapiens (human)
presynapse	Histamine H3 receptor	Homo sapiens (human)
plasma membrane	Histamine H3 receptor	Homo sapiens (human)
synapse	Histamine H3 receptor	Homo sapiens (human)
dendrite	Histamine H3 receptor	Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Bioassays (9)

Assay ID	Title	Year	Journal	Article
AID260990	Displacement of N-[3H]methylhistamine from rat histamine H3 receptor in rat cortical hemispheres	2006	Bioorganic & medicinal chemistry letters, Feb-15, Volume: 16, Issue:4	Aplysamine-1 and related analogs as histamine H3 receptor antagonists.
AID669238	Antimicrobial activity against Staphylococcus aureus 01A1095 assessed as inhibition of cell viability at 350 uM after 18 hrs by spectrophotometry	2012	Journal of natural products, May-25, Volume: 75, Issue:5	Ianthelliformisamines A-C, antibacterial bromotyrosine-derived metabolites from the marine sponge Suberea ianthelliformis.
AID669234	Antimicrobial activity against Pseudomonas aeruginosa PAO1 assessed as inhibition of cell viability at 350 uM after 18 hrs by spectrophotometry	2012	Journal of natural products, May-25, Volume: 75, Issue:5	Ianthelliformisamines A-C, antibacterial bromotyrosine-derived metabolites from the marine sponge Suberea ianthelliformis.
AID365358	Antagonist activity at histamine H3 receptor in guinea pig ileum assessed as inhibition of (R)-alpha-methylhistamine-induced response	2008	Journal of medicinal chemistry, Sep-11, Volume: 51, Issue:17	The alkaloid conessine and analogues as potent histamine H3 receptor antagonists.
AID599205	Antagonist activity at human histamine H3 receptor expressed in SK-N-MC cells assessed as inhibition of forskolin stimulated cAMP accumulation after 6 hrs	2009	European journal of medicinal chemistry, Nov, Volume: 44, Issue:11	Heterocyclic replacement of the central phenyl core of diamine-based histamine H3 receptor antagonists.
AID260989	Displacement of N-[3H]methylhistamine from human histamine H3 receptor expressed in SK-N-MC cells	2006	Bioorganic & medicinal chemistry letters, Feb-15, Volume: 16, Issue:4	Aplysamine-1 and related analogs as histamine H3 receptor antagonists.
AID1151357	Induction of apolipoprotein E secretion from human CCF-STTG1 cells at 30 uM after 96 hrs by ELISA relative to control	2014	Journal of natural products, May-23, Volume: 77, Issue:5	Aplysinellamides A-C, bromotyrosine-derived metabolites from an Australian Aplysinella sp. marine sponge.
AID599203	Displacement of [3H]N-alpha-methylhistamine from human histamine H3 receptor expressed in SK-N-MC cells	2009	European journal of medicinal chemistry, Nov, Volume: 44, Issue:11	Heterocyclic replacement of the central phenyl core of diamine-based histamine H3 receptor antagonists.
AID1545099	Inhibition of histamine H3 receptor (unknown origin)	2019	Bioorganic & medicinal chemistry, 07-15, Volume: 27, Issue:14	Nature-inspired pyrrolo[2,3-d]pyrimidines targeting the histamine H
[information is prepared from bioassay data collected from National Library of Medicine (NLM), extracted Dec-2023]

Research

Studies (8)

Timeframe	Studies, This Drug (%)	All Drugs %
pre-1990	0 (0.00)	18.7374
1990's	0 (0.00)	18.2507
2000's	4 (50.00)	29.6817
2010's	4 (50.00)	24.3611
2020's	0 (0.00)	2.80
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Study Types

Publication Type	This drug (%)	All Drugs (%)
Trials	0 (0.00%)	5.53%
Reviews	0 (0.00%)	6.00%
Case Studies	0 (0.00%)	4.05%
Observational	0 (0.00%)	0.25%
Other	8 (100.00%)	84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Clinical Trials (111)

Trial Overview

Trial	Phase	Enrollment	Study Type	Start Date	Status
The Utility of Interferon-Gamma Release Assays in TB-HIV Co-infected Children [NCT00604617]		564 participants (Actual)	Observational	2009-01-26	Completed
Role of Interferon-gamma 1-b (IFN-γ) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous Disease [NCT03548818]		9 participants (Actual)	Observational	2018-05-16	Completed
Cellular Responses To Intradermal-Gamma (IFN-gamma) in Normal and Psoriatic Patients [NCT01317017]	Early Phase 1	2 participants (Actual)	Interventional	2010-07-31	Terminated(stopped due to Unable to isolate sufficient cells from the skin biopsy to perform study experiments.)
Safety and Efficacy of γIFN Treatment in Friedreich Ataxia [NCT03888664]	Phase 2	12 participants (Actual)	Interventional	2016-06-26	Completed
Open Controlled Randomized Study of the Efficacy and Safety of Ingaron (Interferon-gamma) in the Treatment of Anogenital Warts (Phase III) [NCT05156541]	Phase 3	30 participants (Actual)	Interventional	2009-05-18	Completed
Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia [NCT02797080]	Phase 3	38 participants (Actual)	Interventional	2016-06-28	Completed
Phase 1 Study of Intraperitoneal Infusion of Autologous Monocytes With Sylatron (Peginterferon Alfa-2b) and Actimmune (Interferon Gamma-1b) in Women With Recurrent or Refractory Ovarian Cancer, Fallopian Tube Cancer or Primary Peritoneal Cancer [NCT02948426]	Phase 1	18 participants (Actual)	Interventional	2017-02-08	Terminated(stopped due to Study was closed to accrual due to lack of drug supply.)
A Pilot Study of IFN-γ to Treat Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) That Has Relapsed After Allogeneic Hematopoietic Stem Cell Transplantation [NCT04628338]	Early Phase 1	8 participants (Actual)	Interventional	2021-03-08	Completed
Better Identification of Latent Tuberculosis Infection Among Israeli Young Adults by Comparison Skin Tests and Interferon Gamma Releasing Assays (IGRA) [NCT02073669]		115 participants (Actual)	Interventional	2014-03-31	Completed
Interferon-gamma as Adjunctive Therapy in Chronic Pulmonary Aspergillosis: a Randomised Feasibility Study [NCT05653193]	Phase 2	50 participants (Anticipated)	Interventional	2024-01-31	Not yet recruiting
Double-Blind, Randomized, Placebo-Controlled, Single Ascending and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Target Engagement of BMS-986184 in Healthy Subjects and to Evaluate the Safety, Efficacy, Pharmacokinetics, [NCT02864264]	Phase 1	7 participants (Actual)	Interventional	2016-09-14	Terminated(stopped due to Adverse change in the risk/benefit)
A Phase 1b, Randomized, Multi-center, Open-label Study of a Conditionally Replicative Adenovirus (DNX-2401) and Interferon Gamma (IFN-γ) for Recurrent Glioblastoma or Gliosarcoma (TARGET-I) [NCT02197169]	Phase 1	37 participants (Actual)	Interventional	2014-09-11	Completed
Prospective Randomized Open-label Comparative Study of the Use of Intranasal Form of Interferon Gamma Human Recombinant in Patients for the Prevention of Acute Respiratory Viral Infections, Including COVID-19 [NCT05054114]		630 participants (Actual)	Interventional	2020-12-21	Completed
Evaluation of a CD4/CD8+ Interferon Gamma Release Assay for Monitoring Anti-Tuberculosis Treatment [NCT05724212]		220 participants (Actual)	Observational	2017-01-27	Active, not recruiting
A Phase II Trial of MK-3475 (Pembrolizumab) and Interferon Gamma 1-b Combination Immunotherapy in Patients With Previously Treated Mycosis Fungoides and Sezary Syndrome (Treatment Group 1) and in Patients With Advanced Synovial Sarcoma (Treatment Group 2) [NCT03063632]	Phase 2	28 participants (Actual)	Interventional	2017-12-14	Completed
A Phase 1 Study to Evaluate Safety, Toxicity, and Potential Mechanisms of Interferon Gamma-primed Mesenchymal Stromal Cells (MSCs) for Moderate-to-severe Persistent Asthma [NCT05035862]	Phase 1	12 participants (Anticipated)	Interventional	2022-03-16	Recruiting
Assessment of the Immune Response to SARS-CoV-2/COVID-19 Vaccination in Patients With Sarcoidosis [NCT05089565]		101 participants (Actual)	Observational [Patient Registry]	2021-12-01	Completed
The Treatment of Uveitic Cystoid Macular Edema With Topical Interferon Gamma [NCT00943982]	Phase 1	5 participants (Actual)	Interventional	2009-07-17	Completed
Protocol Title: A Phase II Open-labeled Study to Determine the Safety and Preliminary Efficacy of Interferon-gamma 1b (IFN-γ 1b) in Patients With Chronic Hepatitis B Who Are HBV DNA Positive [NCT00753467]	Phase 2	30 participants (Anticipated)	Interventional	2008-09-30	Not yet recruiting
Phase I/II Study of Autologous CD8+ and CD4+ Transgenic T Cells Expressing High Affinity MCPyV-Specific TCRs Combined With Avelumab and Class I MHC-Upregulation in Patients With Metastatic MCC Refractory to PD-1 Axis Blockade [NCT03747484]	Phase 1/Phase 2	16 participants (Anticipated)	Interventional	2019-07-03	Recruiting
A Phase II Study of Chemoimmunotherapy for Patients With Potentially Platinum Sensitive Müllerian (Epithelial Ovarian, Peritoneal, or Fallopian Tube) Carcinomas [NCT00501644]	Phase 2	59 participants (Actual)	Interventional	2003-01-31	Completed
Randomized Phase II Trial of Dendritic Cell-Based Idiotype Vaccination With Adjuvant Cytokines for Plateau Phase and Post-Transplant Multiple Myeloma [NCT00616720]	Phase 2	15 participants (Anticipated)	Interventional	2001-08-31	Completed
Prospective Open Controlled Non-interventional Study of the Use of the Drug Ingaron (Interferon Gamma Human Recombinant, NPP Farmaklon LLC, Russia) in Volunteers for the Prevention of Coronavirus Infection COVID-19 [NCT05386446]		100 participants (Actual)	Observational	2020-04-23	Completed
An Open Controlled Study of the Efficacy and Safety of Ingaron (Interferon-gamma Human Recombinant) in the Treatment of Chronic Prostatitis [NCT05378646]	Phase 3	30 participants (Actual)	Interventional	2009-01-29	Completed
Human Recombinant Interferon Gamma-1b for the Prevention of Hospital-acquired Pneumonia in Critically Ill Patients: a Double-blind, International, Phase 2, Randomized, Placebo-controlled Trial - the PREV-HAP Study [NCT04793568]	Phase 2	109 participants (Actual)	Interventional	2021-03-29	Active, not recruiting
Effect of Interferon-gamma 1-b on Innate Immune Cells [NCT02609932]	Phase 1	20 participants (Actual)	Interventional	2016-07-31	Completed
Profile of Mother-caregivers of Children With Duchenne Muscular Dystrophy [NCT01921374]		60 participants (Actual)	Interventional	2013-08-31	Completed
High-Dose Chemotherapy Followed By Autologous Hematopoietic Stem Cell Support And One Of Two Regimens Aimed At Modifying Immune Reconstitution In Women With High Risk Stage 2 And Stage 3 Breast Cancer [NCT00008203]	Phase 3	0 participants	Interventional	1996-05-31	Completed
Evaluation of the Safety and Immunogenicity of Intratumoral Injection of Interferon Gamma During Vaccination in Patients With Subcutaneous or Cutaneous Metastases of Melanoma [NCT00977145]	Phase 1	11 participants (Actual)	Interventional	2009-11-30	Terminated(stopped due to Closed short of the enrollment goal, due to slow enrollment and adequate data to address endpoints)
New Immunomodulatory Therapy Strategies in Chronic Reactive Arthritis: Immunostimulation Plus Antibiotic Versus Immunosuppression Plus Antibiotic Versus Conventional Standardtherapy [NCT00244179]	Phase 2	40 participants	Interventional	2003-01-31	Recruiting
A Pilot Study of Aerosol Interferon-gamma for Treatment of Idiopathic Pulmonary Fibrosis [NCT00563212]	Phase 1	12 participants (Actual)	Interventional	2007-01-31	Completed
Role of A. Lumbricoides in the Development of Pulmonary Aspergillosis in Chronic Obstructive Pulmonary Disease Patients [NCT05783544]		200 participants (Anticipated)	Interventional	2015-05-01	Recruiting
A Phase IIa Clinical Trial to Test the Safety and Efficacy of Interferon Gamma Treatment in Elevating Frataxin Levels in Friedreich's Ataxia (FRDA) Patients [NCT02035020]	Phase 2	10 participants (Actual)	Interventional	2013-05-31	Completed
The Effects of Interferon-gamma on Sepsis-induced Immunoparalysis, a Randomised Double-blind Placebo-controlled Pilot (Phase IIIb) Study [NCT01649921]	Phase 3	4 participants (Actual)	Interventional	2012-11-30	Completed
Study of Systematic Tuberculosis Testing for Active, Sub-clinical and Latent Tuberculosis Infection in a United Kingdom Human Immunodeficiency Virus (HIV) Infected Cohort [NCT02712671]		300 participants (Anticipated)	Observational	2013-06-30	Active, not recruiting
Interferon-gamma With Interferon Alpha and Ribavirin for Hepatitis C Patients Who Are Non-responders to Interferon Alpha Plus Ribavirin [NCT00538811]	Phase 2/Phase 3	40 participants (Anticipated)	Interventional		Completed
A Phase II Trial of a MART-1/gp100/Tyrosinase Peptide-Pulsed Dendritic Cell Vaccine Treated With CD40 Ligand/Gamma Interferon With Subcutaneous IL-2 for Patients With Metastatic Melanoma [NCT00006113]	Phase 2	25 participants (Actual)	Interventional	1999-06-30	Terminated(stopped due to Lack of efficacy)
Combination Immunotherapy With Interferon-gamma and Nivolumab for Patients With Advanced Solid Tumors: A Phase 1 Study [NCT02614456]	Phase 1	26 participants (Actual)	Interventional	2015-12-11	Completed
Safety and Efficacy of Recombinant Interferon-Gamma 1b (rIFN-Gamma 1b) Given With Standard Therapy in Patients With Candidemia [NCT04979052]	Phase 2	200 participants (Anticipated)	Interventional	2022-03-31	Recruiting
A Randomized Phase III Trial of Hyperthermic Isolated Limb Perfusion With Melphalan, Tumor Necrosis Factor, and Interferon-Gamma in Patients With Locally Advanced Extremity Melanoma [NCT00001296]	Phase 3	122 participants	Interventional	1992-02-29	Completed
Phase I-II Study of Fluorouracil in Combination With Phenylbutyrate, Indomethacin and Recombinant Human Interferon-Gamma in Advanced Colorectal Cancer [NCT00002796]	Phase 1/Phase 2	46 participants (Actual)	Interventional	1997-05-31	Terminated(stopped due to Administratively complete.)
Randomized Trial of Autologous GVHD for Refractory Lymphoma [NCT00003414]	Phase 3	50 participants (Anticipated)	Interventional	1997-10-31	Completed
Intraperitoneal (IP) Autologous Therapeutic Tumor Vaccine AUT-OV-ALVAC-h.B7.1 Plus IP rIFN-gamma for Patients With Ovarian Cancer. A Pilot Study [NCT00004032]	Phase 1	12 participants (Actual)	Interventional	1997-10-31	Completed
Open-label Early Phase 2 Study With a Single Arm of Interferon Gamma-1b Treatment of Osteopetrosis [NCT02666768]	Phase 2	5 participants (Actual)	Interventional	2016-02-22	Completed
An Open-Label Study of the Safety of Subcutaneous Recombinant Interferon Gamma-1b in Patients With Idiopathic Pulmonary Fibrosis [NCT00076635]	Phase 3	91 participants (Actual)	Interventional	2003-11-30	Terminated(stopped due to program discontinued based on GIPF-007 results)
A Phase II/III Study of Immunomodulation After High Dose Myeloablative Therapy With Autologous Stem Cell Rescue for Refractory/Relapsed Hodgkin Disease [NCT00070187]	Phase 2/Phase 3	24 participants (Actual)	Interventional	2003-11-30	Completed
A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of the Safety and Efficacy of Interferon Gamma-1b in Patients With Idiopathic Pulmonary Fibrosis (The INSPIRE Trial) [NCT00075998]	Phase 3	826 participants (Actual)	Interventional	2003-12-31	Terminated(stopped due to test drug showed lack of benefit at interim analysis)
A Phase I Trial of Monoclonal Antibody HGS-ETR2 (Lexatumumab) With or Without Interferon Gamma in Patients With Refractory Pediatric Solid Tumors [NCT00428272]	Phase 1	30 participants (Actual)	Interventional	2006-12-04	Terminated
Pilot Study on Interferon Gamma in Association With Peg-Interferon Alpha 2a and Ribavirin Among Patients With a Chronic Hepatitis C and Non Responders to the Association of Peg-Interferon Alpha 2b or 2a and Ribavirin ANRS HC16 Gammatri [NCT00148863]	Phase 2	65 participants	Interventional	2004-06-30	Completed
An Extension Study to Protocol VIR-NCHR-01 to Assess the Antiretrovirological Properties of a Therapeutic HIV Vaccine Candidate Based on Recombinant Fowlpox Virus (rFPV) (ITV Extension Study) [NCT00332930]	Phase 1/Phase 2	35 participants	Interventional	2002-09-30	Completed
A Study of Adjuvant Cytokine Therapy in Pulmonary Mycobacterium Avium Complex and Other Pulmonary Nontuberculous Mycobacterial Infections [NCT00111397]	Phase 1	2 participants (Actual)	Interventional	2005-05-13	Completed
Phase II Clinical Trial of Intra-lesional Administration of TG1042 (Adenovirus-Interferon-gamma) in Patients With Relapsing Primary Cutaneous B-Cell Lymphomas. [NCT00394693]	Phase 2	13 participants (Actual)	Interventional	2006-11-30	Completed
Phase II Trial Of Combined Resection, Intraperitoneal Chemotherapy, And Whole Abdominal Radiation For Treatment Of Peritoneal Mesothelioma [NCT00024271]	Phase 2	0 participants	Interventional	2001-05-31	Active, not recruiting
Phase I/II Study of Recombinant Human Interferon-gamma (rIFN-gamma) in HIV-Infected Children [NCT00000761]	Phase 1	20 participants	Interventional		Completed
A Randomized Multicenter Phase II Trial of Recombinant Tumor Necrosis Factor and Recombinant Human Interferon-gamma in Patients With AIDS Related Complex [NCT00001004]	Phase 2	30 participants	Interventional		Completed
A Multicenter Study of the Safety and Anti-Fibrotic Efficacy of Interferon-Gamma 1b (Actimmune) in Patients With Severe Lever Fibrosis or Compensated Cirrhosis Due to Hepatitis C. [NCT00043303]	Phase 2	502 participants (Actual)	Interventional	2001-09-30	Completed
A Phase I/II Study of Interferon Gamma-1b by Subcutaneous Injection for the Treatment of Patients With Cystic Fibrosis [NCT00043342]	Phase 1/Phase 2	51 participants (Actual)	Interventional	2002-04-30	Completed
Interferon Gamma-1b in Combination With Chemotherapy (Carboplatin/Paclitaxel) for First Line Therapy of Advanced Ovarian or Primary Peritoneal Carcinoma. [NCT00047632]	Phase 3	847 participants (Actual)	Interventional	2001-10-31	Terminated(stopped due to futility)
A Randomized, Double-Blind, Three-Arm, Phase IIIb Study Comparing the Safety and Efficacy of Interferon Gamma-1b Alone, IFN-Gamma 1b With Azathioprine, and Azathioprine Alone in Patients With Idiopathic Pulmonary Fibrosis Receiving Prednisone [NCT00052039]	Phase 3	0 participants (Actual)	Interventional	2002-04-30	Terminated(stopped due to Study design changes were needed based on GIPF-001 results)
A Prospective, Randomized, Double-Blind, Multicenter Pilot Study Of The Safety And Efficacy Of Interferon Gamma- 1b (IFN-y 1b) Plus Voriconazole Versus Placebo Plus Voriconazole In The Treatment Of Invasive Aspergillosis And Other Filamentous Fungal Infec [NCT00059878]	Phase 2	0 participants	Interventional	2003-08-31	Completed
A Phase II, Double-Blind, Placebo-Controlled Study to Determine the Safety and Efficacy of a Humanized Anti-Interferon-γ Monoclonal Antibody (HuZAF) Administered to Patients With Moderate to Severe Crohn's Disease [NCT00072943]	Phase 2	175 participants	Interventional	2002-03-31	Completed
A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging, Phase II Study of the Safety and Antifungal Activity of Subcutaneous Recombinant Interferon-Gamma 1b (rIFN-Gamma 1b) in Conjunction With Standard Therapy in Patients With Acute Cryptococcal Men [NCT00012467]	Phase 2	60 participants	Interventional	2000-01-31	Completed
A Randomized, Double-Blinded, Placebo-Controlled, Phase II Study of the Safety and Efficacy of Inhaled Interferon Gamma-1b With Antimycobacterial in Previously Treated or Moderate to Severe Pulmonary Mycobacterium Avium Complex (MAC) Infection [NCT00021567]	Phase 2	20 participants	Interventional	2001-07-31	Completed
Pilot Studies of Gamma Interferon Therapy for Chronic Hepatitis C [NCT00028275]	Phase 2	20 participants	Interventional	2001-12-31	Completed
Phase I/II Study of the Safety of Subcutaneous Interferon Gamma-1b Combined With Rituximab in Patients With Low Grade/Follicular Non-Hodgkin's Lymphoma [NCT00057447]	Phase 1/Phase 2	24 participants (Actual)	Interventional	2003-03-31	Terminated(stopped due to administrative reasons)
Eine Phase II Studie über Interferon Gamma 1b Zur Behandlung Der steroidrefraktären Bronchiolitis Obliterans Nach Allogener SZT [NCT01639261]	Phase 2	10 participants (Anticipated)	Interventional	2012-07-31	Active, not recruiting
A Phase I/II Study of Interferon Gamma-1b by Inhalation for the Treatment of Patients With Cystic Fibrosis [NCT00043316]	Phase 1/Phase 2	66 participants (Actual)	Interventional	2001-02-28	Completed
A Randomized, Double-Blind, Placebo-Controlled, Phase II Study of Safety and Efficacy of Inhaled Interferon Gamma-1b With Antimycobacterials in Previously Treated or Mod-to-Sev Pulmonary Mycobacterium Avium Complex Infection [NCT00043355]	Phase 2	100 participants (Actual)	Interventional	2000-12-31	Terminated(stopped due to Futility)
A Randomized, Double-Blind, Placebo-Controlled, Phase III Study of the Safety and Efficacy of Subcutaneous Recombinant Interferon-Gamma 1b in Patients With Idiopathic Pulmonary Fibrosis [NCT00047645]	Phase 3	330 participants (Actual)	Interventional	2000-04-30	Completed
Post-Marketing Surveillance Study of Actimmune (Interferon Gamma-1b) in Patients With Severe Malignant Osteopetrosis [NCT00043329]		6 participants (Actual)	Observational	2002-01-31	Completed
An Open-Label Study of the Safety and Efficacy of Subcutaneous Recombinant Interferon Gamma-1b in Patients With Idiopathic Pulmonary Fibrosis. [NCT00052052]	Phase 2	210 participants (Actual)	Interventional	2002-09-30	Completed
A Phase II, Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Biology, and Clinical Effects of Interferon Gamma-1b Administered Subcutaneously to Patients With IPF Followed by an Open-Label Extension [NCT00047658]	Phase 2	32 participants (Actual)	Interventional	2001-11-30	Completed
Safety and Tolerability of Consensus Interferon-Alpha (CIFN) Plus Interferon Gamma-1b (IFN-γ 1b) With or Without Ribavirin (RBV) in the Treatment of Patients With Chronic Hepatitis C Who Are Non-Responders to PEG-IFN-a (2a or 2b) Plus RBV [NCT00084279]	Phase 2	81 participants (Actual)	Interventional	2004-04-30	Completed
Pilot Phase I/II Study of the Evaluation of Interferon Gamma-1b Administered Topically for Macular Edema/Intraretinal Schisis Cysts in Rod-Cone Dystrophy (RCD) and Enhanced S-Cone Syndrome (ESCS) [NCT02338973]	Phase 1/Phase 2	4 participants (Actual)	Interventional	2015-01-14	Terminated
Phase II Study of Gamma Interferon (IFN-γ) Added to Bolus + Infusional 5-Fluorouracil (5-FU) and Leucovorin (LV) +/- Bevacizumab (BV) in Metastatic Colorectal Carcinoma [NCT00786643]	Phase 2	48 participants (Actual)	Interventional	2006-02-28	Completed
Phase 2a Study of Interferon Gamma-1b for the Treatment of Autosomal Dominant Type 2 Osteopetrosis [NCT02584608]	Phase 2	12 participants (Actual)	Interventional	2016-01-01	Completed
An Open Prospective Observational Study Evaluating the Efficacy and Tolerability of Interferon Gamma (Ingaron) Injections in Patients With Drug-resistant Pulmonary Tuberculosis [NCT05359315]		84 participants (Anticipated)	Observational	2022-04-15	Recruiting
Interferon Gamma Administration in Leukocyte Adhesion Deficiency Type I [NCT00001905]	Phase 2	5 participants	Interventional	1999-04-30	Completed
A Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of Childhood [NCT00001317]	Phase 4	100 participants	Interventional	1992-05-31	Completed
Treatment of Multiply Drug Resistant Tuberculosis With Interferon Gamma: A Phase I/II Dose Escalation Trial [NCT00001407]	Phase 2	30 participants	Interventional	1994-05-31	Completed
Activation of Alveolar Macrophages by Aerosolized r-metHuIFN-Gamma (IFN-Gamma) in Patients With AIDS [NCT00002433]		12 participants	Interventional		Completed
A Trial of Active Intralymphatic Immunotherapy With Interferon-Treated Cells and Cyclophosphamide [NCT00002475]	Phase 2	40 participants (Anticipated)	Interventional	1991-04-30	Completed
RANDOMIZED PHASE II TRIAL OF AUTOLOGOUS TUMOR CELL VACCINE [NCT00002505]	Phase 2	0 participants	Interventional	1992-08-31	Completed
PHASE I/II STUDY OF IMMUNIZATION WITH MHC CLASS I MATCHED ALLOGENEIC HUMAN PROSTATIC CARCINOMA CELLS ENGINEERED TO SECRETE INTERLEUKIN-2 AND INTERFERON-GAMMA [NCT00002637]	Phase 1/Phase 2	25 participants (Anticipated)	Interventional	1995-01-31	Completed
Interferon γ-Primed Mesenchymal Stromal Cells as Prophylaxis for Acute Graft v Host Disease After Allogeneic Hematopoietic Cell Transplantation for Patients With Hematologic Malignancies and Myelodysplasia [NCT04328714]	Phase 1	45 participants (Anticipated)	Interventional	2021-12-02	Suspended(stopped due to Study is suspended pending renovation and reopening of the facility manufacturing the study product.)
Prospective Two-week Open-label Application Experimental Randomized Single-center Non-interventional Study of the Drug Ingaron in Patients With a New Coronavirus Infection COVID-19 [NCT05386459]		36 participants (Actual)	Observational	2020-04-21	Completed
Assessment of the Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease [NCT01147042]	Phase 4	2 participants (Actual)	Interventional	2010-05-18	Terminated(stopped due to Early termination due to only 2 subjects completing trial.)
Treatment of Non-Tuberculous Mycobacterial Infections With Interferon Gamma [NCT00001318]	Phase 2	60 participants	Interventional	1992-08-31	Completed
[NCT00004402]	Phase 3	30 participants	Interventional	1999-11-30	Completed
Pilot Study of Mesenchymal Stromal Cells in Patients With Xerostomia After Radiation Therapy for Head and Neck Cancer [NCT04489732]	Phase 1	6 participants (Actual)	Interventional	2022-02-18	Active, not recruiting
Feasibility of Assessing Drug Response to Precise Local Injection of Anti-cancer Drugs Using Presage's CIVO Device in Soft Tissue Sarcoma Patients Undergoing Surgery. [NCT03056599]	Phase 1	23 participants (Actual)	Interventional	2016-12-15	Completed
The Treatment of Macular Edema Secondary to Uveitis Using Topical Interferon Gamma [NCT01376362]	Phase 1/Phase 2	5 participants (Actual)	Interventional	2011-06-30	Completed
Randomized, Multicenter, Double-Blind, Placebo-Controlled, Efficacy, Safety, and Pharmacokinetic Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia [NCT02415127]	Phase 3	92 participants (Actual)	Interventional	2015-06-30	Completed
A Phase I Study To Determine the Safety of the Optimal Monocyte Activating Administration Schedule of Subcutaneous Human Recombinant Interferon-gamma in ZDV-Treated Patients With AIDS [NCT00001112]	Phase 1	5 participants	Interventional		Completed
Phase I Trial of Immunotherapy With Adenovirus-Interferon- Gamma (TG1041) in Patients With Malignant Melanoma [NCT00004016]	Phase 1	0 participants	Interventional	1999-04-30	Completed
CAMP-010: PHASE I/II STUDY OF IN VIVO PURGING FOLLOWED BY HIGH DOSE CHEMOTHERAPY, AUTOLOGOUS HEMATOPOIETIC STEM CELL INFUSION AND IMMUNOTHERAPY IN PATIENTS WITH CHRONIC MYELOID LEUKEMIA [NCT00002761]	Phase 1/Phase 2	0 participants (Actual)	Interventional	1996-02-29	Withdrawn(stopped due to PI left institution)
Open-label Pilot Study of Interferon Gamma-1b (Actimmune™) for the Treatment of Friedreich Ataxia (FRDA) [NCT01965327]	Phase 2	12 participants (Actual)	Interventional	2013-08-31	Completed
A Personalized Randomized Trial of Validation and Restoration of Immune Dysfunction in Severe Infections and Sepsis [NCT03332225]	Phase 2	36 participants (Actual)	Interventional	2017-12-15	Completed
Multicenter, Safety and Efficacy, Open-Label Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia [NCT02593773]	Phase 3	86 participants (Actual)	Interventional	2015-12-25	Completed
Pilot Phase I/II Study of the Treatment of Classic Central Serous Chorioretinopathy With Topical Interferon Gamma-1b [NCT01468337]	Phase 1/Phase 2	5 participants (Actual)	Interventional	2011-10-31	Completed
A Randomized, Double-Blind, Crossover Study to Determine the Effect of Actimmune® Dose Titration on the Severity and Incidence of Interferon Gamma-1b-Related Flu-Like Symptoms and the Pattern of Dropouts in Healthy Volunteers [NCT01929382]	Phase 1	40 participants (Anticipated)	Interventional	2013-07-31	Active, not recruiting
A Phase I-II Study of Interferon-gamma Plus Weekly Paclitaxel, Trastuzumab and Pertuzumab in Patients With HER-2 Positive Breast Cancer [NCT03112590]	Phase 1/Phase 2	51 participants (Actual)	Interventional	2017-06-23	Completed
A Pilot Study to Test Whether Systemic Interferon Gamma Increases Tumor Class I MHC Expression in Patients With Synovial Sarcoma and Myxoid/Round Cell Liposarcoma [NCT01957709]	Early Phase 1	8 participants (Actual)	Interventional	2013-09-25	Terminated(stopped due to Enough samples were collected for data analysis.)
Pilot Single-center Open Study of the Effect of Ingaron on the Efficacy and Resistance to Antibiotics in Antibacterial Therapy in Patients With Community-acquired Pneumonia [NCT05395702]		114 participants (Actual)	Interventional	2017-05-12	Completed
A Multicenter Clinical Trial of Recombinant Human Interferon Gamma (Ingaron) in Pulmonary Tuberculosis [NCT06118619]		350 participants (Anticipated)	Observational	2022-06-01	Recruiting
A Multicentre, Prospective, Randomized Open-label Pilot Study to Assess the Feasibility and Preliminary Efficacy of Interferon-gamma in Combination With Anidulafungin for the Treatment of Candidemia [NCT01270490]	Phase 3	20 participants (Anticipated)	Interventional	2011-01-31	Recruiting
Human Recombinant Interferon Gamma in the Treatment of Ventilator-acquired Pneumonia in ICU Patients [NCT05843786]	Phase 3	132 participants (Anticipated)	Interventional	2023-06-30	Recruiting
Antigen Specific Adoptive T Cell Therapy for Refractory Opportunistic Adenovirus Infection After a Hematopoietic Stem Cell Transplantation [NCT03378102]	Early Phase 1	20 participants (Anticipated)	Interventional	2019-01-04	Recruiting
A I//II Phase Study of the Efficacy and Safety of Interferon-Gamma by Subcutaneous Injection in the Complex Treatment of Patients Infected With HIV and Tuberculosis [NCT05065905]	Phase 1/Phase 2	78 participants (Actual)	Interventional	2006-01-19	Completed
Early Diagnosis of Active Tuberculosis Using Ultra Low-dose Chest CT to Predict Progression to Active Tuberculosis Among Contacts [NCT03220464]		116 participants (Anticipated)	Interventional	2017-06-20	Recruiting
Host Response to Tuberculosis and Acquired Immune Deficiency Syndrome [NCT00201123]	Phase 2	89 participants (Actual)	Interventional	2005-04-30	Completed
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

Trial	Outcome
NCT00201123 (3) [back to overview]	Bronchoalveolar Lavage (BAL) to Measure Flow of Cytometry and Cytokine Levels
NCT00201123 (3) [back to overview]	Sputum Conversion
NCT00201123 (3) [back to overview]	Chest Cavity Size
NCT00501644 (1) [back to overview]	Number of Patients With Response
NCT00786643 (3) [back to overview]	Best Response (BR)
NCT00786643 (3) [back to overview]	Time to Progression
NCT00786643 (3) [back to overview]	Early Response Rate (RR) (Stratum 1 Only)
NCT01376362 (17) [back to overview]	Change in Macular Volume in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week Two Compared to Baseline
NCT01376362 (17) [back to overview]	Change in Macular Volume in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week One Compared to Baseline
NCT01376362 (17) [back to overview]	Change in Macular Volume in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week Two Compared to Baseline
NCT01376362 (17) [back to overview]	Change in Macular Volume in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week One Compared to Baseline
NCT01376362 (17) [back to overview]	Change in Intraocular Pressure (IOP) in the Study Eye at Week Two Compared to Baseline
NCT01376362 (17) [back to overview]	Change in Intraocular Pressure (IOP) in the Study Eye at Week One Compared to Baseline
NCT01376362 (17) [back to overview]	Change in Intraocular Pressure (IOP) in the Fellow Eye at Week Two Compared to Baseline
NCT01376362 (17) [back to overview]	Change in Intraocular Pressure (IOP) in the Fellow Eye at Week One Compared to Baseline
NCT01376362 (17) [back to overview]	Change in Excess Central Macular Thickening in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week Two Compared to Baseline
NCT01376362 (17) [back to overview]	Change in Excess Central Macular Thickening in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week One Compared to Baseline
NCT01376362 (17) [back to overview]	Change in Excess Central Macular Thickening in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week One Compared to Baseline
NCT01376362 (17) [back to overview]	Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Study Eye at Week Two Compared to Baseline
NCT01376362 (17) [back to overview]	Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Study Eye at Week One Compared to Baseline
NCT01376362 (17) [back to overview]	Proportion of Participants With a Visual Loss of 15 or More Early Treatment Diabetic Retinopathy Study (ETDRS) Letters in the Study Eye
NCT01376362 (17) [back to overview]	Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week One Compared to Baseline
NCT01376362 (17) [back to overview]	Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week Two Compared to Baseline
NCT01376362 (17) [back to overview]	Change in Excess Central Macular Thickening in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week Two Compared to Baseline
NCT01468337 (9) [back to overview]	Total Number of Ocular Adverse Events Related to Investigational Product
NCT01468337 (9) [back to overview]	Changes in Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week 2 Compared to Baseline
NCT01468337 (9) [back to overview]	Changes in Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week 48 Compared to Baseline
NCT01468337 (9) [back to overview]	Changes in Best-corrected Visual Acuity (BCVA) in the Study Eye at Week 2 Compared to Baseline
NCT01468337 (9) [back to overview]	Changes in Best-corrected Visual Acuity (BCVA) in the Study Eye at Week 48 Compared to Baseline
NCT01468337 (9) [back to overview]	Number of Participants Who Withdrew From the Study
NCT01468337 (9) [back to overview]	Total Number of Non-ocular Adverse Events Related to the Investigational Product
NCT01468337 (9) [back to overview]	Total Number of Severe Non-ocular Adverse Events Related to the Investigational Product
NCT01468337 (9) [back to overview]	Total Number of Severe Ocular Adverse Events Related to the Investigational Product
NCT01957709 (3) [back to overview]	Change in Class I Major Histocompatibility Complex (MHC) Expression After Treatment With IFN Gamma
NCT01957709 (3) [back to overview]	Changes in Immune Response
NCT01957709 (3) [back to overview]	MHC Class II Expression
NCT01965327 (2) [back to overview]	Change in Total Friedreich Ataxia Rating Scale (FARS) Score
NCT01965327 (2) [back to overview]	Change in Whole Blood Frataxin Levels
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 3
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 5
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 52
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 52
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 8
NCT02338973 (32) [back to overview]	Change in Central Visual Field Sensitivity at Day 2 and Week 5 Compared to Baseline.
NCT02338973 (32) [back to overview]	Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 1
NCT02338973 (32) [back to overview]	Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 2
NCT02338973 (32) [back to overview]	Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 3
NCT02338973 (32) [back to overview]	Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 2
NCT02338973 (32) [back to overview]	Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 5
NCT02338973 (32) [back to overview]	Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 52
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 2
NCT02338973 (32) [back to overview]	Number and Severity of IP-related AEs
NCT02338973 (32) [back to overview]	Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 8
NCT02338973 (32) [back to overview]	Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 2
NCT02338973 (32) [back to overview]	Number of Participants Who Withdrew
NCT02338973 (32) [back to overview]	Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 1
NCT02338973 (32) [back to overview]	Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 2
NCT02338973 (32) [back to overview]	Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 3
NCT02338973 (32) [back to overview]	Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 5
NCT02338973 (32) [back to overview]	Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 52
NCT02338973 (32) [back to overview]	Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 8
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 1
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 2
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 3
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 2
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 5
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 52
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 8
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 1
NCT02338973 (32) [back to overview]	Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 2
NCT02415127 (4) [back to overview]	Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW)
NCT02415127 (4) [back to overview]	Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score
NCT02415127 (4) [back to overview]	Change From Baseline to Week 26 in Total Friedreich Ataxia Rating Scale Score (FARStot)
NCT02415127 (4) [back to overview]	Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score
NCT02584608 (2) [back to overview]	Change in Bone Turnover Markers Between After Completion of 6-12 Weeks of Treatment
NCT02584608 (2) [back to overview]	Changes in Bone Resorption Markers From Baseline to 14 Weeks.
NCT02593773 (2) [back to overview]	Number of Participants With Positive/Negative Neutralizing Antibody (NAb) and Anti-Drug Antibody (ADA) Tests
NCT02593773 (2) [back to overview]	Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs
NCT02666768 (4) [back to overview]	Percent Change From Baseline in Bone Mineral Density (BMD)
NCT02666768 (4) [back to overview]	Number of Participants With Treatment Related Adverse Events CTCAE v4.0 Grade 3 or Higher
NCT02666768 (4) [back to overview]	Change From Baseline in White Blood Cell Count (WBC)
NCT02666768 (4) [back to overview]	Change From Baseline in Pain
NCT02797080 (1) [back to overview]	Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs
NCT02948426 (7) [back to overview]	Overall Maximum Tolerated Dose of Actimmune (Interferon Gamma-1b)
NCT02948426 (7) [back to overview]	Number of Participants With Serious or Non-serious (Any) Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0)
NCT02948426 (7) [back to overview]	Number of Participants With a Grade 3 or Higher Dose-Limiting Toxicity (DLT)
NCT02948426 (7) [back to overview]	Number of Participants With a Response
NCT02948426 (7) [back to overview]	Time to Disease Progression
NCT02948426 (7) [back to overview]	Overall Maximum Tolerated Dose of Sylatron (Peginterferon Alpha-2b)
NCT02948426 (7) [back to overview]	Overall Maximum Tolerated Dose of Intraperitoneal Autologous Monocytes
NCT03063632 (7) [back to overview]	Time to Response (TTR)
NCT03063632 (7) [back to overview]	Rate of Overall Response Duration Beyond 12 Months (ORR12)
NCT03063632 (7) [back to overview]	Overall Response Rate (ORR)
NCT03063632 (7) [back to overview]	Progression-free Survival (PFS)
NCT03063632 (7) [back to overview]	Incidence of Adverse Events
NCT03063632 (7) [back to overview]	Event-free Survival (EFS)
NCT03063632 (7) [back to overview]	Duration of Response (DOR)
NCT03112590 (4) [back to overview]	Phase 1: Recommended Phase 2 Dose (RP2D)
NCT03112590 (4) [back to overview]	Phase 2: Pathologic Complete Response Rate (pCR)
NCT03112590 (4) [back to overview]	Phase 2: Progression Free Survival (PFS)/Number of Participants Who Progressed
NCT03112590 (4) [back to overview]	Phase 2: Clinical Response

Bronchoalveolar Lavage (BAL) to Measure Flow of Cytometry and Cytokine Levels

(NCT00201123)
Timeframe: 16 Weeks

Intervention	cells/mL (Median)
	Lymphocytes Week 0	Lymphocytes Week 16	Machrophages Week 0	Machrophages Week 16	Neutrophils Week 0	Neutrophils Week 16
DOTS	4	15	60	64	28	11
Nebulized rlFN-y	5	15	60	63	24	4
Subcutaneous rlFN-y	6	22	62	66	30	2

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Sputum Conversion

(NCT00201123)
Timeframe: Measured at 16 Weeks

Intervention	percentage of participants (Number)
DOTS Control Group	36
Aerosol Interferon Gamma for TB	60
Subcutaneous Interferon Gamma for TB	36

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Chest Cavity Size

(NCT00201123)
Timeframe: 16 Weeks

Intervention	millimeters (Mean)
	Baseline	16 Weeks
Aerosol Interferon Gamma for TB	39	29
DOTS Control Group	34	20
Subcutaneous Interferon Gamma for TB	34	18

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Number of Patients With Response

Per World Health Organization (WHO) Tumor Response: Complete Response (CR), Partial Response (PR) or Progressive Disease (PD). CR defined as disappearance of all target lesions, PR as > = 30% decrease in sum of longest dimensions of target lesions with reference baseline sum longest dimensions and if CA 125 levels declined by >50%, provided target lesion size did not increase by >20% on imaging, and PD as >20% increase in sum of longest dimensions of target lesions taking as references smallest sum of longest dimensions recorded since treatment started, or appearance of 1 or > new lesions. (NCT00501644)
Timeframe: Follow up CT scans after every 3 courses of treatment and following completion of all treatments.

Intervention	Participants (Number)
	Complete Response	Partial Response	Progressive Disease
Chemoimmunotherapy	9	21	24

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Best Response (BR)

BR is recorded from start of treatment until progressive disease (PD). Imaging was repeated by same technique after every 4 cycles of treatment. Response was evaluated per Response Evaluation Criteria In Solid Tumors (RECIST) guidelines version 1.0. Per RECIST v1.0 and CT scan: Complete Response (CR), disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of longest diameter of target lesions; Stable Disease (SD), neither sufficient shrinkage in sum of LD of target lesions to be PR nor increase of >=20%; PD, increase in existing lesions or new lesions. (NCT00786643)
Timeframe: After every 4 cycles of treatment (approximately every 56 days for up to about 280 days)

Intervention	Participants (Number)
	Complete response (CR)	Partial response (PR)	Stable disease (SD)	Progressive disease (PD)	Not evaluable (NE)
Stratum 1	0	6	7	6	1
Stratum 2	0	3	15	6	4

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Time to Progression

Patients were censored if they did not progress, stopped particiaption due to an adverse event, or withdrew consent following the start of study treatment. Response was evaluated by Response Evaluation Criteria In Solid Tumors (RECIST) guidelines version 1.0. Per RECIST v1.0, Progressive Disease (PD) is defined as a measurable increase in smallest diameter of any target or non-target lesion, or the appearance of new lesions, since baseline. (NCT00786643)
Timeframe: From date of study treatment start until date of first documented progression or date of death from any cause, whichever came first, assessed up to 15 months

Intervention	Months (Median)
Stratum 1	5.5263
Stratum 2	3.9145

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Early Response Rate (RR) (Stratum 1 Only)

Early RR evaluated in stratum 1 to see if bevacizumab (bev) would be added to GFL treatment (tx). Patients with stable disease (SD) pre 5th cycle of tx had bev added. Response was evaluated by Response Evaluation Criteria In Solid Tumors (RECIST) version 1.0. Per RECIST and CT scan: Complete Response (CR), disappearance of all target lesions; Partial Response (PR), >=30% decrease in sum of longest diameter (LD) of target lesions; SD, neither sufficient shrinkage in sum of LD of target lesions to be PR nor increase of >=20%; Progressive Disease (PD), increase in existing lesions or new lesions. (NCT00786643)
Timeframe: After 4 cycles of treatment (approximately 56 days)

Intervention	Participants (Number)
	Complete response (CR)	Partial response (PR)	Stable disease (SD)	Progressive disease (PD)	Not evaluable (NE)
Stratum 1	0	5	7	6	2

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Change in Macular Volume in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week Two Compared to Baseline

Macular volume was assessed by spectral-domain optical coherence tomography (Cirrus HD-OCT; Carl Zeiss Meditec, Dublin, CA), a non-invasive imaging technique that uses long-wavelength light to capture micrometer-resolution cross-sectional images from biological tissue. (NCT01376362)
Timeframe: Baseline and 2 Weeks

Intervention	mm^3 (Mean)
Interferon Gamma-1b	0.00

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Change in Macular Volume in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week One Compared to Baseline

Intervention	mm^3 (Mean)
Interferon Gamma-1b	-0.02

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Change in Macular Volume in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week Two Compared to Baseline

Intervention	mm^3 (Mean)
Interferon Gamma-1b	0.06

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Change in Macular Volume in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week One Compared to Baseline

Intervention	mm^3 (Mean)
Interferon Gamma-1b	0.14

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Change in Intraocular Pressure (IOP) in the Study Eye at Week Two Compared to Baseline

Intraocular pressure was recorded using a standard Goldmann applanation tonometer, a device for the measurement of intraocular pressure between 0 to 78 mm Hg. (NCT01376362)
Timeframe: Baseline and 2 Weeks

Intervention	mm Hg (Mean)
Interferon Gamma-1b	0.40

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Change in Intraocular Pressure (IOP) in the Study Eye at Week One Compared to Baseline

Intervention	mm Hg (Mean)
Interferon Gamma-1b	0.40

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Change in Intraocular Pressure (IOP) in the Fellow Eye at Week Two Compared to Baseline

Intervention	mm Hg (Mean)
Interferon Gamma-1b	-2.20

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Change in Intraocular Pressure (IOP) in the Fellow Eye at Week One Compared to Baseline

Intervention	mm Hg (Mean)
Interferon Gamma-1b	-1.60

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Change in Excess Central Macular Thickening in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week Two Compared to Baseline

Central macular thickness was assessed by spectral-domain optical coherence tomography (Cirrus HD-OCT; Carl Zeiss Meditec, Dublin, CA), a non-invasive imaging technique that uses long-wavelength light to capture micrometer-resolution cross-sectional images from biological tissue. (NCT01376362)
Timeframe: Baseline and 2 Weeks

Intervention	µm (Mean)
Interferon Gamma-1b	-47.40

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Change in Excess Central Macular Thickening in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week One Compared to Baseline

Intervention	µm (Mean)
Interferon Gamma-1b	-38.60

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Change in Excess Central Macular Thickening in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week One Compared to Baseline

Intervention	µm (Mean)
Interferon Gamma-1b	12.00

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Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Study Eye at Week Two Compared to Baseline

Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20. (NCT01376362)
Timeframe: Baseline and 2 Weeks

Intervention	ETDRS Letters (Mean)
Interferon Gamma-1b	4.00

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Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Study Eye at Week One Compared to Baseline

Intervention	ETDRS Letters (Mean)
Interferon Gamma-1b	0.20

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Proportion of Participants With a Visual Loss of 15 or More Early Treatment Diabetic Retinopathy Study (ETDRS) Letters in the Study Eye

Intervention	Percentage of Participants (Number)
Interferon Gamma-1b	0

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Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week One Compared to Baseline

Intervention	ETDRS Letters (Mean)
Interferon Gamma-1b	1.60

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Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week Two Compared to Baseline

Intervention	ETDRS Letters (Mean)
Interferon Gamma-1b	3.20

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Change in Excess Central Macular Thickening in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week Two Compared to Baseline

Intervention	µm (Mean)
Interferon Gamma-1b	30.20

Intervention	Adverse Events (Number)
Interferon Gamma-1b	7

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Changes in Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week 2 Compared to Baseline

"Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20.~A positive change value indicates improvement of the outcome. A negative change value indicates worsening of the outcome." (NCT01468337)
Timeframe: Baseline and Week 2

Intervention	ETDRS letters (Mean)
Interferon Gamma-1b	0.80

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Changes in Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week 48 Compared to Baseline

Intervention	ETDRS letters (Mean)
Interferon Gamma-1b	4.67

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Changes in Best-corrected Visual Acuity (BCVA) in the Study Eye at Week 2 Compared to Baseline

Intervention	ETDRS letters (Mean)
Interferon Gamma-1b	0.4

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Changes in Best-corrected Visual Acuity (BCVA) in the Study Eye at Week 48 Compared to Baseline

Intervention	ETDRS letters (Mean)
Interferon Gamma-1b	2.67

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Number of Participants Who Withdrew From the Study

(NCT01468337)
Timeframe: Week 48

Intervention	participants (Number)
Interferon Gamma-1b	0

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Change in Class I Major Histocompatibility Complex (MHC) Expression After Treatment With IFN Gamma

It would be highly relevant to observe marked increase macrophages (effect size > 2.5). Four patients gives over 90% power to detect such a large increase with a two-tailed alpha of 0.05. (NCT01957709)
Timeframe: Baseline to up to 2 weeks post-surgery

Intervention	percentage of MHC Class I+ on tumor cell (Median)
	Pre-treatment	Post-treatment
Basic Science (Interferon Gamma and MHC Expression)	8.91	26.6

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Changes in Immune Response

To examine changes in the immune response to MRCL and SS by examining changes in the immune infiltrates, antibody response and antigen specific T cell response before and after IFNg treatment. (NCT01957709)
Timeframe: Baseline to 2 weeks post biopsy

Intervention	percentage of T cells (Median)
	Pre-treatment	Post-treatment
Basic Science (Interferon Gamma and MHC Expression)	0.14	0.82

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MHC Class II Expression

To determine whether systemic administration of IFNg will increase class II MHC expression in SS and MRCL tumors. (NCT01957709)
Timeframe: Baseline to 2 weeks post biopsy.

Intervention	percentage of MHC Class II on tumor cell (Median)
	Pre-treatment	Post-treatment
Basic Science (Interferon Gamma and MHC Expression)	2.556	6.125

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Change in Total Friedreich Ataxia Rating Scale (FARS) Score

The Friedreich Ataxia Rating Scale (FARS) is neurological rating scale specifically developed and validated for FRDA. The FARS includes assessments of stance, gait, upper and lower limb coordination, speech, proprioception and strength. In addition to the standard neurological examination, the FARS contains three quantitative performance measures and a component that assesses activities of daily living (ADL). Quantitative performance measures include the nine-hole peg test, and a timed 25-foot walk. FARS scores correlate significantly with functional disability, activities of daily living scores and disease duration. The scores from the three subscales are added to generate a total score ranging from 0 to 159, with a higher score indicating a greater level of disability. (NCT01965327)
Timeframe: FARS score was calculated at the beginning and conclusion of treatment (baseline and 12 weeks)

Intervention	units on a scale (Mean)
Interferon Gamma-1b (ACTIMMUNE)	-4.98

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Change in Whole Blood Frataxin Levels

Assessment of the change in whole blood frataxin levels as assessed by lateral flow assay using an immunoassay for frataxin. Frataxin levels in the blood were measured at each study visit. Change in frataxin level at the end of treatment (week 12) relative to frataxin level at baseline was analyzed. (NCT01965327)
Timeframe: Frataxin levels were measured at the beginning and conclusion of treatment (baseline and 12 weeks)

Intervention	percentage of baseline frataxin level (Mean)
Interferon Gamma-1b (ACTIMMUNE)	-1.5

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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 3

Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Day 3 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Day 3

,,,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	278	319	745	260
Central Retinal Thickness at Baseline in Study Eye	307	619	702	1413
Central Retinal Thickness at Day 3 in Fellow Eye	277	320	656	243
Central Retinal Thickness at Day 3 in Study Eye	330	587	607	1393
Difference at Day 3 From Baseline in Fellow Eye	-1	1	-89	-17
Difference at Day 3 From Baseline in Study Eye	23	-32	-95	-20

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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 5

Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Week 5 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 5

,,,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	278	319	745	260
Central Retinal Thickness at Baseline in Study Eye	307	619	702	1413
Central Retinal Thickness at Week 5 in Fellow Eye	282	316	591	243
Central Retinal Thickness at Week 5 in Study Eye	385	576	509	1089
Difference at Week 5 From Baseline in Fellow Eye	4	-3	-154	-17
Difference at Week 5 From Baseline in Study Eye	78	-43	-193	-324

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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 52

Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Week 52 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 52

Intervention	μm (Number)
	Participant 001	Participant 003	Participant 004
Central Retinal Thickness at Week 52 in Fellow Eye	277	472	233
Difference at Week 52 From Baseline in Fellow Eye	-1	-273	-27

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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 52

Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Week 52 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 52

,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	278	319	745	260
Central Retinal Thickness at Baseline in Study Eye	307	619	702	1413
Central Retinal Thickness at Week 52 in Study Eye	309	497	445	1221
Difference at Week 52 From Baseline in Study Eye	2	-122	-257	-192

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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 8

Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Week 8 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 8

,,,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	278	319	745	260
Central Retinal Thickness at Baseline in Study Eye	307	619	702	1413
Central Retinal Thickness at Week 8 in Fellow Eye	280	313	657	250
Central Retinal Thickness at Week 8 in Study Eye	370	545	486	1020
Difference at Week 8 From Baseline in Fellow Eye	2	-6	-88	-10
Difference at Week 8 From Baseline in Study Eye	63	-74	-216	-393

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Change in Central Visual Field Sensitivity at Day 2 and Week 5 Compared to Baseline.

Change in central visual field sensitivity as measured by microperimetry testing at Day 2 and Week 5 compared to baseline in both study and fellow eyes. (NCT02338973)
Timeframe: Day 2 and Week 5

Intervention	dB (Mean)
	Day 2 Study Eye	Day 2 Fellow Eye	Week 5 Study Eye	Week 5 Fellow Eye
Interferon Gamma-1b	-0.3	-1.4	-1.1	-1.7

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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 1

Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Day 1 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Day 1

,,,,,

Intervention	mm^3 (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Difference at Day 1 From Baseline in Fellow Eye	0.25	0.05	-1.35	-0.05
Difference at Day 1 From Baseline in Study Eye	0.1	-0.2	-0.95	1.4
Subretinal Fluid Volume at Baseline in Fellow Eye	7.1	11.2	18.55	9.25
Subretinal Fluid Volume at Baseline in Study Eye	7.25	14.15	16.1	36.3
Subretinal Fluid Volume at Day 1 in Study Eye	7.35	13.95	15.15	37.7
Subretinal Fluid Volume at Day1 in Fellow Eye	7.35	11.25	17.2	9.2

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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 2

Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Day 2 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Day 2

,,,,,

Intervention	mm^3 (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Difference at Day 2 From Baseline in Fellow Eye	-0.1	-0.3	-3	-0.2
Difference at Day 2 From Baseline in Study Eye	-0.15	-0.05	-0.95	1
Subretinal Fluid Volume at Baseline in Fellow Eye	7.1	11.2	18.55	9.25
Subretinal Fluid Volume at Baseline in Study Eye	7.25	14.15	16.1	36.3
Subretinal Fluid Volume at Day 2 in Fellow Eye	7	10.9	15.55	9.05
Subretinal Fluid Volume at Day 2 in Study Eye	7.1	14.1	15.15	37.3

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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 3

Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Day 3 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Day 3

,,,,,

Intervention	mm^3 (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Difference at Day 3 From Baseline in Fellow Eye	0.35	0.05	-1.55	-0.15
Difference at Day 3 From Baseline in Study Eye	0.25	-0.4	-0.8	-1.3
Subretinal Fluid Volume at Baseline in Fellow Eye	7.1	11.2	18.55	9.25
Subretinal Fluid Volume at Baseline in Study Eye	7.25	14.15	16.1	36.3
Subretinal Fluid Volume at Day 3 in Fellow Eye	7.45	11.25	17	9.1
Subretinal Fluid Volume at Day 3 in Study Eye	7.5	13.75	15.3	35

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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 2

Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Week 2 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 2

,,,,,

Intervention	mm^3 (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Difference at Week 2 From Baseline in Fellow Eye	0.2	-0.2	-1.55	-0.25
Difference at Week 2 From Baseline in Study Eye	0.35	-1.05	-1.8	-3.8
Subretinal Fluid Volume at Baseline in Fellow Eye	7.1	11.2	18.55	9.25
Subretinal Fluid Volume at Baseline in Study Eye	7.25	14.15	16.1	36.3
Subretinal Fluid Volume at Week 2 in Fellow Eye	7.3	11	17	9
Subretinal Fluid Volume at Week 2 in Study Eye	7.6	13.1	14.3	32.5

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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 5

Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Week 5 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 5

,,,,,

Intervention	mm^3 (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Difference at Week 5 From Baseline in Fellow Eye	0.2	0	-2.85	-0.15
Difference at Week 5 From Baseline in Study Eye	0.15	-0.25	-2.1	-7.2
Subretinal Fluid Volume at Baseline in Fellow Eye	7.1	11.2	18.55	9.25
Subretinal Fluid Volume at Baseline in Study Eye	7.25	14.15	16.1	36.3
Subretinal Fluid Volume at Week 5 in Fellow Eye	7.3	11.2	15.7	9.1
Subretinal Fluid Volume at Week 5 in Study Eye	7.4	13.9	14	29.1

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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 52

Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Week 52 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 52

,,,,,

Intervention	mm^3 (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Difference at Week 52 From Baseline in Fellow Eye	-0.1	0.1	-4.25	-0.35
Difference at Week 52 From Baseline in Study Eye	-0.35	-1.45	-2.3	-4
Subretinal Fluid Volume at Baseline in Fellow Eye	7.1	11.2	18.55	9.25
Subretinal Fluid Volume at Baseline in Study Eye	7.25	14.15	16.1	36.3
Subretinal Fluid Volume at Week 52 in Fellow Eye	7	11.3	14.3	8.9
Subretinal Fluid Volume at Week 52 in Study Eye	6.9	12.7	13.8	32.3

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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 2

Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Week 2 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 2

,,,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	278	319	745	260
Central Retinal Thickness at Baseline in Study Eye	307	619	702	1413
Central Retinal Thickness at Week 2 in Fellow Eye	276	317	687	234
Central Retinal Thickness at Week 2 in Study Eye	333	530	552	1233
Difference at Week 2 From Baseline in Fellow Eye	-2	-2	-58	-26
Difference at Week 2 From Baseline in Study Eye	26	-89	-150	-180

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Number and Severity of IP-related AEs

The number and severity of adverse events related to the investigation product (IP). (NCT02338973)
Timeframe: Study duration, up to 52 weeks

Intervention	adverse events related to IP (Number)
	Severity: Mild	Severity: Severe
Interferon Gamma-1b	3	1

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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 8

Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Week 8 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 8

,,,,,

Intervention	mm^3 (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Difference at Week 8 From Baseline in Fellow Eye	0.3	-0.3	-1.45	0.15
Difference at Week 8 From Baseline in Study Eye	0.15	-0.65	-2.3	-8.7
Subretinal Fluid Volume at Baseline in Fellow Eye	7.1	11.2	18.55	9.25
Subretinal Fluid Volume at Baseline in Study Eye	7.25	14.15	16.1	36.3
Subretinal Fluid Volume at Week 8 in Fellow Eye	7.4	10.9	17.1	9.4
Subretinal Fluid Volume at Week 8 in Study Eye	7.4	13.5	13.8	27.6

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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 2

Change in BCVA from baseline as compared to Week 2 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 2

,,,,,

Intervention	letters read (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
BCVA at Baseline in Fellow Eye	80	61	76	42
BCVA at Baseline in Study Eye	77	39	69	14
BCVA at Week 2 in Fellow Eye	78	66	67	42
BCVA at Week 2 in Study Eye	75	0	66	23
Difference in BCVA at Week 2 From Baseline in Fellow Eye	-2	5	-9	0
Difference in BCVA at Week 2 From Baseline in Study Eye	-2	-39	-3	9

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Number of Participants Who Withdrew

The number of participants who withdrew early. (NCT02338973)
Timeframe: Study duration, up to 52 weeks

Intervention	Participants (Count of Participants)
Interferon Gamma-1b	0

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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 1

Change in BCVA from baseline as compared to Day 1 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Day 1

,,,,,

Intervention	letters read (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
BCVA at Baseline in Fellow Eye	80	61	76	42
BCVA at Baseline in Study Eye	77	39	69	14
BCVA at Day 1 in Fellow Eye	79	62	70	44
BCVA at Day 1 in Study Eye	76	32	74	18
Difference in BCVA at Day 1 From Baseline in Fellow Eye	-1	1	-6	2
Difference in BCVA at Day 1 From Baseline in Study Eye	-1	-7	5	4

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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 2

Change in BCVA from baseline as compared to Day 2 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Day 2

,,,,,

Intervention	letters read (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
BCVA at Baseline in Fellow Eye	80	61	76	42
BCVA at Baseline in Study Eye	77	39	69	14
BCVA at Day 2 in Fellow Eye	81	58	69	45
BCVA at Day 2 in Study Eye	75	36	75	21
Difference in BCVA at Day 2 From Baseline in Fellow Eye	1	-3	-7	3
Difference in BCVA at Day 2 From Baseline in Study Eye	-2	-3	6	7

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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 3

Change in BCVA from baseline as compared to Day 3 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Day 3

,,,,,

Intervention	letters read (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
BCVA at Baseline in Fellow Eye	80	61	76	42
BCVA at Baseline in Study Eye	77	39	69	14
BCVA at Day 3 in Fellow Eye	78	61	73	47
BCVA at Day 3 in Study Eye	81	41	74	23
Difference in BCVA at Day 3 From Baseline in Fellow Eye	-2	0	-3	5
Difference in BCVA at Day 3 From Baseline in Study Eye	4	2	5	9

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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 5

Change in BCVA from baseline as compared to Week 5 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 5

,,,,,

Intervention	letters read (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
BCVA at Baseline in Fellow Eye	80	61	76	42
BCVA at Baseline in Study Eye	77	39	69	14
BCVA at Week 5 in Fellow Eye	82	65	75	45
BCVA at Week 5 in Study Eye	71	0	72	26
Difference in BCVA at Week 5 From Baseline in Fellow Eye	2	4	-1	3
Difference in BCVA at Week 5 From Baseline in Study Eye	-6	-39	3	12

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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 52

Change in BCVA from baseline as compared to Week 52 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 52

,,,,,

Intervention	letters read (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
BCVA at Baseline in Fellow Eye	80	61	76	42
BCVA at Baseline in Study Eye	77	39	69	14
BCVA at Week 52 in Fellow Eye	78	65	65	44
BCVA at Week 52 in Study Eye	79	47	65	20
Difference in BCVA at Week 52 From Baseline in Fellow Eye	-2	4	-11	2
Difference in BCVA at Week 52 From Baseline in Study Eye	2	8	-4	6

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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 8

Change in BCVA from baseline as compared to Week 8 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 8

,,,,,

Intervention	letters read (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
BCVA at Baseline in Fellow Eye	80	61	76	42
BCVA at Baseline in Study Eye	77	39	69	14
BCVA at Week 8 in Fellow Eye	80	65	70	47
BCVA at Week 8 in Study Eye	74	43	72	24
Difference in BCVA at Week 8 From Baseline in Fellow Eye	0	4	-6	5
Difference in BCVA at Week 8 From Baseline in Study Eye	-3	4	3	10

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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 1

Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Day 1 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Day 1

,,,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	269	285	784	241.5
Central Retinal Thickness at Baseline in Study Eye	297.5	597	713	1393
Central Retinal Thickness at Day 1 in Fellow Eye	270.5	298.5	713.5	232.5
Central Retinal Thickness at Day 1 in Study Eye	314	602	598	1394.5
Difference at Day 1 From Baseline in Fellow Eye	1.5	13.5	-70.5	-9
Difference at Day 1 From Baseline in Study Eye	16.5	5	-115	1.5

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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 2

Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Day 2 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Day 2

,,,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	269	285	784	241.5
Central Retinal Thickness at Baseline in Study Eye	297.5	597	713	1393
Central Retinal Thickness at Day 2 in Fellow Eye	279	304.5	700.5	220
Central Retinal Thickness at Day 2 in Study Eye	309.5	605	613	1392.5
Difference at Day 2 From Baseline in Fellow Eye	10	19.5	-83.5	-21.5
Difference at Day 2 From Baseline in Study Eye	12	8	-100	-0.5

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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 3

Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Day 3 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Day 3

,,,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	269	285	784	241.5
Central Retinal Thickness at Baseline in Study Eye	297.5	597	713	1393
Central Retinal Thickness at Day 3 in Fellow Eye	274	299	682.5	220
Central Retinal Thickness at Day 3 in Study Eye	323	587.5	621	1361.5
Difference at Day 3 From Baseline in Fellow Eye	5	14	-101.5	-21.5
Difference at Day 3 From Baseline in Study Eye	25.5	-9.5	-92	-31.5

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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 2

Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Week 2 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 2

,,,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	269	285	784	241.5
Central Retinal Thickness at Baseline in Study Eye	297.5	597	713	1393
Central Retinal Thickness at Week 2 in Fellow Eye	267	300	700	208
Central Retinal Thickness at Week 2 in Study Eye	330	534	517	1193
Difference at Week 2 From Baseline in Fellow Eye	-2	15	-84	-33.5
Difference at Week 2 From Baseline in Study Eye	32.5	-63	-196	-200

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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 5

Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Week 5 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 5

,,,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	269	285	784	241.5
Central Retinal Thickness at Baseline in Study Eye	297.5	597	713	1393
Central Retinal Thickness at Week 5 in Fellow Eye	276	302	580	206
Central Retinal Thickness at Week 5 in Study Eye	374	605	473	1083
Difference at Week 5 From Baseline in Fellow Eye	7	17	-204	-35.5
Difference at Week 5 From Baseline in Study Eye	76.5	8	-240	-310

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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 52

Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Week 52 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 52

,,,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	269	285	784	241.5
Central Retinal Thickness at Baseline in Study Eye	297.5	597	713	1393
Central Retinal Thickness at Week 52 in Fellow Eye	270	307	457	197
Central Retinal Thickness at Week 52 in Study Eye	302	503	437	1175
Difference at Week 52 From Baseline in Fellow Eye	1	22	-327	-44.5
Difference at Week 52 From Baseline in Study Eye	4.5	-94	-276	-218

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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 8

Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Week 8 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Week 8

,,,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	269	285	784	241.5
Central Retinal Thickness at Baseline in Study Eye	297.5	597	713	1393
Central Retinal Thickness at Week 8 in Fellow Eye	276	224	667	220
Central Retinal Thickness at Week 8 in Study Eye	353	544	456	1000
Difference at Week 8 From Baseline in Fellow Eye	7	-61	-117	-21.5
Difference at Week 8 From Baseline in Study Eye	55.5	-53	-257	-393

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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 1

Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Day 1 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Day 1

,,,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	278	319	745	260
Central Retinal Thickness at Baseline in Study Eye	307	619	702	1413
Central Retinal Thickness at Day 1 in Fellow Eye	275	317	703	258
Central Retinal Thickness at Day 1 in Study Eye	331	607	605	1418
Difference at Day 1 From Baseline in Fellow Eye	-3	-2	-42	-2
Difference at Day 1 From Baseline in Study Eye	24	-12	-97	5

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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 2

Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Day 2 by participant in both study and fellow eyes. (NCT02338973)
Timeframe: Day 2

,,,,,

Intervention	μm (Number)
	Participant 001	Participant 002	Participant 003	Participant 004
Central Retinal Thickness at Baseline in Fellow Eye	278	319	745	260
Central Retinal Thickness at Baseline in Study Eye	307	619	702	1413
Central Retinal Thickness at Day 2 in Fellow Eye	278	318	682	246
Central Retinal Thickness at Day 2 in Study Eye	321	611	624	1427
Difference at Day 2 From Baseline in Fellow Eye	0	-1	-63	-14
Difference at Day 2 From Baseline in Study Eye	14	-8	-78	14

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Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW)

The T25FW is a quantitative measure of lower extremity function. Participants are directed to 1 end of a clearly marked 25-foot course and instructed to walk 25 feet as quickly as possible, but safely. The task is immediately administered again by having the participant walk back the same distance, and the score for the test is the average of the 2 walks (after reciprocal transformation). Participants may use assistive devices when performing this task, with the same assistive device used at each assessment. A negative change from Baseline indicates improvement. (NCT02415127)
Timeframe: Baseline, Week 26

Intervention	1/seconds (Mean)
Interferon γ-1b	-0.006
Placebo	-0.003

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Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score

Participants and/or their caregivers rated 9 areas of daily living skills (speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function) on a 5-point scale (0=normal, 4=greatest loss of function) with allowable increments of 0.5 if the participant or caregiver strongly felt that a task falls between 2 scores. ADL scores can range from 0 (normal) to 36 (greatest loss of function). A negative change from baseline indicates improvement. (NCT02415127)
Timeframe: Baseline, Week 26

Intervention	units on a scale (Mean)
Interferon γ-1b	0.64
Placebo	0.01

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Change From Baseline to Week 26 in Total Friedreich Ataxia Rating Scale Score (FARStot)

The FARS assessment includes neurological signs that specifically reflect neural substrates affected in FA. Based on a neurological examination, bulbar, upper limb, lower limb, peripheral nerve, and upright stability/gait functions are assessed. FARStot scores range from 0 (normal) to 125 (most impairment). A negative change from baseline indicates improvement. (NCT02415127)
Timeframe: Baseline, Week 26

Intervention	units on a scale (Mean)
Interferon γ-1b	-0.2
Placebo	-0.6

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Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score

The FARS assessment includes neurological signs that specifically reflect neural substrates affected in FA. Based on a neurological examination, bulbar, upper limb, lower limb, peripheral nerve, and upright stability/gait functions were assessed. The FARS-mNeuro score excludes the peripheral nervous system subscale score and the facial and tongue atrophy and fasciculations from the bulbar subscale score. Scores range from 0 (normal) to 93 (most impairment). A negative change from baseline is an improvement. (NCT02415127)
Timeframe: Baseline, Week 26

Intervention	units on a scale (Mean)
Interferon γ-1b	-0.6
Placebo	-1.0

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Change in Bone Turnover Markers Between After Completion of 6-12 Weeks of Treatment

Evaluate for changes in bone turnover markers including TRAP5b, NTX, CTX/TRAP5b ratio after 6-12 weeks of treatment. (NCT02584608)
Timeframe: 6-12 weeks

Intervention	percentage of change (Mean)
	TRAP5B	NTX	CTX/TRAP5b ratio
Treatment	-15.34	-2.09	26.31

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Changes in Bone Resorption Markers From Baseline to 14 Weeks.

Evaluate for changes in bone resorption markers including CTX, NTX/creatinine ratio between baseline and 14 weeks (NCT02584608)
Timeframe: baseline, 14 weeks

Intervention	percentage of change (Mean)
	Percent change of CTX	Percent change of NTX/creatinine ratio
Treatment	2.2	-2.1

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Number of Participants With Positive/Negative Neutralizing Antibody (NAb) and Anti-Drug Antibody (ADA) Tests

NAb testing only for those participants with a positive ADA test. Baseline is defined as the last non-missing measurement/assessment on the date of Week 26 Visit from study HZNP-ACT-301 (NCT02415127). If this measurement was missing or otherwise unavailable, it was the last non-missing measurement/assessment on or prior to first dose in this study. If the participant discontinued the study, then premature withdrawal assessments were mapped to the nearest scheduled visit based on schedule of the assessment and the study day. If the mapped visit was already available then the visit was mapped to the next schedule visit. Last on study assessment is the last non-missing post-baseline assessment for each participant. (NCT02593773)
Timeframe: Baseline/Day 1 (Week 26 of Study HZNP-ACT-301 [NCT02415127]), Week 4, Week 13, Week 26, and Week 28 (follow-up safety visit)

Intervention	participants (Number)
	Baseline ADA = negative	Baseline ADA = positive	Baseline NAb = negative	Baseline NAb = positive	Week 4 ADA = negative	Week 4 ADA = positive	Week 13 ADA = negative	Week 13 ADA = positive	Week 26 ADA = negative	Week 26 ADA = positive	Week 28 (Follow-up) ADA = negative	Week 28 (Follow-up) ADA = positive	Last On Study Assessment ADA = negative	Last On Study Assessment ADA = positive
Interferon γ-1b	85	1	1	0	84	0	79	0	64	0	56	0	85	0

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Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs

An adverse event (AE) is any untoward medical occurrence, whether or not the event is considered related to the investigational product. A TEAE is any adverse change from the subject's baseline condition, including any laboratory test value abnormality judged as clinically significant by the investigator, that occurs on or after the date of the first dose of study drug administered at home and throughout the duration of the clinical study, whether the adverse event is considered related to the treatment or not. An SAE is an AE that results in death, is life-threatening, results in persistent or significant disability or incapacity, inpatient hospitalization or prolongation of an existing hospitalization, is a congenital anomaly or birth defect, or other medically important event. (NCT02593773)
Timeframe: Baseline/Day 1 (Week 26 of Study HZNP-ACT-301 [NCT02415127]) through Week 28 (follow-up safety visit)

Intervention	participants (Number)
	≥ 1 TEAE	≥ 1 Related TEAE	≥ 1 SAE	≥ 1 Related SAE	≥ 1 TEAE Leading to Discontinuation	≥ 1 TEAE Leading to Death
Interferon γ-1b	78	61	4	0	1	1

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Percent Change From Baseline in Bone Mineral Density (BMD)

BMD measured by peripheral quantitative computed tomography (pQCT) in bone area w/ BMD<169mg/m3 (NCT02666768)
Timeframe: 6 months

Intervention	percent change (Median)
Gamma Interferon-1b	0

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Number of Participants With Treatment Related Adverse Events CTCAE v4.0 Grade 3 or Higher

Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 defines a Grade 4 event as having life-threatening consequences, and/or urgent intervention indicated. (NCT02666768)
Timeframe: 12 months

Intervention	Participants (Count of Participants)
Gamma Interferon-1b	0

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Change From Baseline in White Blood Cell Count (WBC)

(NCT02666768)
Timeframe: 6 months

Intervention	x1000 cells/uL (Median)
Gamma Interferon-1b	-0.5

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Change From Baseline in Pain

Survey name: RAND 36-Item Health Survey (Version 1.0) Scale name: Pain Scale range: 0-100; Higher score means less pain (NCT02666768)
Timeframe: 6 months

Intervention	change in units on the pain scale (Least Squares Mean)
Gamma Interferon-1b	-0.8

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Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs

An adverse event (AE) is any untoward medical occurrence, whether or not the event is considered related to the investigational product. A TEAE is any adverse change from the subject's baseline condition, including any laboratory test value abnormality judged as clinically significant by the investigator, that occurs on or after the date of the first dose of study drug administered at home and throughout the duration of the clinical study, whether the adverse event is considered related to the treatment or not. A serious AE (SAE) is an AE that results in death, is life-threatening, results in persistent or significant disability or incapacity, inpatient hospitalization or prolongation of an existing hospitalization, is a congenital anomaly or birth defect, or other medically important event. (NCT02797080)
Timeframe: Baseline/Day 1 (Week 28 Follow-Up Visit for Study HZNP-ACT-302 ([NCT02593773]) through end of study; mean (SD) duration of treatment was 99.2 (58.48) days.

Intervention	participants (Number)
	≥ 1 TEAE	≥ 1 Related TEAE	≥ 1 SAE	≥ 1 Related SAE	≥ 1 TEAE Leading to Discontinuation	≥ 1 TEAE Leading to Death
Interferon γ-1b	21	7	0	0	0	0

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Overall Maximum Tolerated Dose of Actimmune (Interferon Gamma-1b)

Maximum Tolerated Dose of Actimmune (Interferon gamma-1b). (NCT02948426)
Timeframe: Cycle 1 Day 28

Intervention	mcg (Number)
All Participants On Dose Level 1 Through Dose Level 4	50

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Number of Participants With Serious or Non-serious (Any) Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0)

Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT02948426)
Timeframe: Date treatment consent signed to date off study, approximately 10 months and 11 days for dose level 1,12 months and 1 day for dose level 2, 11 months and 4 days for dose level 3, and 12 months and 6 days for dose level 4.

Intervention	Participants (Count of Participants)
Dose Level 1 - Sylatron 25µg (0.1 µg/ml); Actimmune 5mg (0.02µg/ml)	3
Dose Level 2 - Monocytes (75x10^6); Sylatron 25µg (0.1µg/ml); Actimmune 5mg (0.02µg/ml)	6
Dose Level 3 - Monocytes (750x10^6); Sylatron 25µg (0.1µg/ml); Actimmune 5mg (0.02µg/ml)	3
Dose Level 4 - Monocytes (750x10^6); Sylatron 250µg (1µg/ml); Actimmune 50mg (0.2µg/ml)	6

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Number of Participants With a Grade 3 or Higher Dose-Limiting Toxicity (DLT)

DLT is defined as an laboratory abnormality or adverse drug reaction (ADR) according to the Common Terminology Criteria for Adverse Events (CTCAE), such as any grade 4 immune mediated adverse event attributed to local tumor response, any grade ≥3 colitis, grade 3 or 4 noninfectious pneumonitis, and any > grade 2 cardiac toxicity which dose not resolve to grade 1 within 3 days of initiation of maximum supportive care that is possibly, probably, or definitely related to the combination of drug. Grade 3 adverse event is severe or medically significant but not immediately life-threatening, Grade 4 is life-threatening, and Grade 5 is death related to adverse event. (NCT02948426)
Timeframe: Cycle 1 Day 28

Intervention	Participants (Count of Participants)
Dose Level 1 - Sylatron 25µg (0.1 µg/ml); Actimmune 5mg (0.02µg/ml)	0
Dose Level 2 - Monocytes (75x10^6); Sylatron 25µg (0.1µg/ml); Actimmune 5mg (0.02µg/ml)	1
Dose Level 3 - Monocytes (750x10^6); Sylatron 25µg (0.1µg/ml); Actimmune 5mg (0.02µg/ml)	0
Dose Level 4 - Monocytes (750x10^6); Sylatron 250µg (1µg/ml); Actimmune 50mg (0.2µg/ml)	0

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Number of Participants With a Response

Response was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Complete Response (CR) is disappearance of all target lesions. Partial Response (PR) is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum of diameters. Progressive Disease (PD) is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study. Stable Disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum of diameters while on study. (NCT02948426)
Timeframe: Participants were evaluated for response by radiographic imaging every 8 weeks, up to 80 weeks

,,,

Intervention	Participants (Count of Participants)
	Complete Response	Partial Response	Stable Disease	Progressive Disease
Dose Level 1 - Sylatron 25µg (0.1 µg/ml); Actimmune 5mg (0.02µg/ml)	0	1	0	1
Dose Level 2 - Monocytes (75x10^6); Sylatron 25µg (0.1µg/ml); Actimmune 5mg (0.02µg/ml)	0	0	2	4
Dose Level 3 - Monocytes (750x10^6); Sylatron 25µg (0.1µg/ml); Actimmune 5mg (0.02µg/ml)	0	0	1	2
Dose Level 4 - Monocytes (750x10^6); Sylatron 250µg (1µg/ml); Actimmune 50mg (0.2µg/ml)	0	1	2	3

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Time to Disease Progression

Time to disease progression is defined as the time from registration to progression, censored at date last known progression-free for those who have not progressed. Progression is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (NCT02948426)
Timeframe: Participants were assessed every 4 weeks by physical exam and 8 weeks by radiographic imaging for disease progression, up to 10 months.

Intervention	Months (Median)
Dose Level 1 - Sylatron 25µg (0.1 µg/ml); Actimmune 5mg (0.02µg/ml)	3.7
Dose Level 2 - Monocytes (75x10^6); Sylatron 25µg (0.1µg/ml); Actimmune 5mg (0.02µg/ml)	3.2
Dose Level 3 - Monocytes (750x10^6); Sylatron 25µg (0.1µg/ml); Actimmune 5mg (0.02µg/ml)	2.0
Dose Level 4 - Monocytes (750x10^6); Sylatron 250µg (1µg/ml); Actimmune 50mg (0.2µg/ml)	3.7

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Overall Maximum Tolerated Dose of Sylatron (Peginterferon Alpha-2b)

Maximum Tolerated Dose of Sylatron (Peginterferon alpha-2b). (NCT02948426)
Timeframe: Cycle 1 Day 28

Intervention	mcg (Number)
All Participants On Dose Level 1 Through Dose Level 4	250

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Overall Maximum Tolerated Dose of Intraperitoneal Autologous Monocytes

Maximum Tolerated Dose of intraperitoneal autologous monocytes. (NCT02948426)
Timeframe: Cycle 1 Day 28

Intervention	Cells (Number)
All Participants On Dose Level 1 Through Dose Level 4	750000000

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Time to Response (TTR)

Will use simple statistics. (NCT03063632)
Timeframe: Time interval between the date of first treatment and the date of response (complete response [CR]/partial response [PR]), up to 2 years

Intervention	days (Median)
Group I (Pembrolizumab, Interferon Gamma-1b)	126

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Rate of Overall Response Duration Beyond 12 Months (ORR12)

Will be assessed per global assessment of mycosis fungoides and Sezary syndrome (confirmed & investigator assessed). Will use binomial distribution. (NCT03063632)
Timeframe: From the time measurement criteria are met for CR or PR (whichever is first recorded) until the first date beyond 12 months that recurrent disease is objectively documented, up to 2 years

Intervention	Participants (Count of Participants)
Group I (Pembrolizumab, Interferon Gamma-1b)	2
Group II (Pembrolizumab, Interferon Gamma-1b)	0

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Overall Response Rate (ORR)

"Participants in Treatment Group 1 will be assessed for response and progression using standard response criteria in patients with Mycosis Fungoides and Sezary syndrome. Per Global Response Score determined by evaluating skin, lymph nodes, internal organs (viscera), and blood specimens: Complete Response (CR), complete disappearance of all clinical evidence of disease; Partial Response (PR), regression of measurable disease.~Participants in Treatment Group 2 will be assessed for response and progression using Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria. Complete Response (CR) disappearance of all lesions and no new lesion; Partial Response (PR), 30% or greater reduction in tumor size and no new lesions.~The ORR is defined as CR combined with PR. Will be assessed using binomial proportion.~Best response at any timepoint was used to determine ORR." (NCT03063632)
Timeframe: Up to 2 years

Intervention	Participants (Count of Participants)
Group I (Pembrolizumab, Interferon Gamma-1b)	6
Group II (Pembrolizumab, Interferon Gamma-1b)	0

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Progression-free Survival (PFS)

Will be assessed using the Kaplan-Meier method (NCT03063632)
Timeframe: Time from enrollment to disease progression or death, whichever occurs earlier, based upon investigator assessment, up to 3 years

Intervention	days (Median)
Group I (Pembrolizumab, Interferon Gamma-1b)	394.0
Group II (Pembrolizumab, Interferon Gamma-1b)	196.5

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Incidence of Adverse Events

Will be graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. (NCT03063632)
Timeframe: Up to 2 years and 1 months

Intervention	Participants (Count of Participants)
Group I (Pembrolizumab, Interferon Gamma-1b)	15
Group II (Pembrolizumab, Interferon Gamma-1b)	12

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Event-free Survival (EFS)

Will be assessed using the Kaplan-Meier method. (NCT03063632)
Timeframe: Termination due to toxicity, initiation of next significant treatment, progressive disease, or death of any cause, up to 2 years

Intervention	days (Median)
Group I (Pembrolizumab, Interferon Gamma-1b)	185.5
Group II (Pembrolizumab, Interferon Gamma-1b)	73.0

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Duration of Response (DOR)

Will be assessed using the Kaplan-Meier method. (NCT03063632)
Timeframe: Time interval between the date of first response (CR/PR) and the date of progression, up to 2 years and 11 months

Intervention	days (Median)
Group I (Pembrolizumab, Interferon Gamma-1b)	505.0

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Phase 1: Recommended Phase 2 Dose (RP2D)

The dose limiting toxicity (DLT) evaluation period for dose escalation will be during the first three weeks. The maximum tolerated dose (MTD) dose level is defined as the highest dose level with ≤1 out of 6 participants experiencing a DLT. If the first dose level experience two or more DLTs, then dose de-escalation will occur. DLT during cycle one (C1) is defined as follows: Non-hematologic or hematologic toxicities that are ≥ grade 3 in severity and probably or definitely related to study therapy which leads to chemotherapy treatment delays > 14 days are considered DLT. The MTD will become the RP2D. (NCT03112590)
Timeframe: 12 weeks

Intervention	mcg/m^2 (Number)
Combination Therapy	75

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Phase 2: Pathologic Complete Response Rate (pCR)

"Pathologic complete response in the breast at definitive surgery after completion of protocol therapy. The pathologic response to treatment will be assessed by an institutional pathologist at Moffitt Cancer Center. The pathologist will evaluate response by the Residual Cancer Burden(RCB) for each participant as described in the online calculator (see RCB link in the More Information section). pCR is defined as no residual invasive carcinoma in the breast and lymph notes at definitive surgery following neoadjuvant therapy" (NCT03112590)
Timeframe: After post therapy surgery - Therapy: approximately 12 weeks per participant

Intervention	percentage of participants (Number)
Combination Therapy	52

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Phase 2: Progression Free Survival (PFS)/Number of Participants Who Progressed

"Progression will be evaluated in this study using the Response Evaluation Criteria in Solid Tumors (RECIST) criteria, version 1.1.~PFS is defined as the time from study therapy to the first occurrence of ipsilateral invasive breast tumor recurrence, ipsilateral locoregional invasive breast cancer recurrence, contralateral invasive breast cancer, distant recurrence, or death from any cause. This is reported as number of participants who progressed." (NCT03112590)
Timeframe: Up to 2 years

Intervention	participants (Number)
Phase 2	1

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Phase 2: Clinical Response

Complete Response (CR) and Partial Response (PR) based upon tumor measurements obtained on physical examination at baseline, after completion of 4 cycles of study therapy. Factors that will be evaluated include: Breast mass(es) - size (longest dimension); Axillary lymph node(s) - size (longest dimension); Skin edema of the breast - present worse, present unchanged, present improved, or absent; Skin erythema of the breast - present worse, present unchanged, present improved, or absent. (NCT03112590)
Timeframe: 12 weeks

Intervention	Participants (Count of Participants)
	Complete Response	Partial Response	Stable Disease	Progressive Disease
Phase 2	25	11	2	1

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Substance	Relationship Strength	Studies	Classes	Roles
carcinine carcinine: structure. carcinine : A beta-alanine derivative that is the amide obtained by formal condensation of the carboxy group of beta-alanine with the primary amino group of histamine.	2.04	1	beta-alanine derivative; imidazoles; monocarboxylic acid amide; organonitrogen compound; organooxygen compound	antioxidant; crustacean metabolite
ciprofloxacin Ciprofloxacin: A broad-spectrum antimicrobial carboxyfluoroquinoline.. ciprofloxacin : A quinolone that is quinolin-4(1H)-one bearing cyclopropyl, carboxylic acid, fluoro and piperazin-1-yl substituents at positions 1, 3, 6 and 7, respectively.	2.07	1	aminoquinoline; cyclopropanes; fluoroquinolone antibiotic; N-arylpiperazine; quinolinemonocarboxylic acid; quinolone antibiotic; quinolone; zwitterion	antibacterial drug; antiinfective agent; antimicrobial agent; DNA synthesis inhibitor; EC 5.99.1.3 [DNA topoisomerase (ATP-hydrolysing)] inhibitor; environmental contaminant; topoisomerase IV inhibitor; xenobiotic
tyrosine Tyrosine: A non-essential amino acid. In animals it is synthesized from PHENYLALANINE. It is also the precursor of EPINEPHRINE; THYROID HORMONES; and melanin.. tyrosine : An alpha-amino acid that is phenylalanine bearing a hydroxy substituent at position 4 on the phenyl ring.	2.97	4	amino acid zwitterion; erythrose 4-phosphate/phosphoenolpyruvate family amino acid; L-alpha-amino acid; proteinogenic amino acid; tyrosine	EC 1.3.1.43 (arogenate dehydrogenase) inhibitor; fundamental metabolite; micronutrient; nutraceutical
4-butyrolactone 4-Butyrolactone: One of the FURANS with a carbonyl thereby forming a cyclic lactone. It is an endogenous compound made from gamma-aminobutyrate and is the precursor of gamma-hydroxybutyrate. It is also used as a pharmacological agent and solvent.. tetrahydrofuranone : Any oxolane having an oxo- substituent at any position on the tetrahydrofuran ring.. gamma-butyrolactone : A butan-4-olide that is tetrahydrofuran substituted by an oxo group at position 2.	2.04	1	butan-4-olide	metabolite; neurotoxin
pyrroles 1H-pyrrole : A tautomer of pyrrole that has the double bonds at positions 2 and 4.. pyrrole : A five-membered monocyclic heteroarene comprising one NH and four CH units which forms the parent compound of the pyrrole group of compounds. Its five-membered ring structure has three tautomers. A 'closed class'.. azole : Any monocyclic heteroarene consisting of a five-membered ring containing nitrogen. Azoles can also contain one or more other non-carbon atoms, such as nitrogen, sulfur or oxygen.	2.04	1	pyrrole; secondary amine
isoxazoles Isoxazoles: Azoles with an OXYGEN and a NITROGEN next to each other at the 1,2 positions, in contrast to OXAZOLES that have nitrogens at the 1,3 positions.. isoxazole : A monocyclic heteroarene with a structure consisting of a 5-membered ring containing three carbon atoms and an oxygen and nitrogen atom adjacent to each other. It is the parent of the class of isoxazoles.. isoxazoles : Oxazoles in which the N and O atoms are adjacent.	2.07	1	isoxazoles; mancude organic heteromonocyclic parent; monocyclic heteroarene
oxazoles Oxazoles: Five-membered heterocyclic ring structures containing an oxygen in the 1-position and a nitrogen in the 3-position, in distinction from ISOXAZOLES where they are at the 1,2 positions.. 1,3-oxazole : A five-membered monocyclic heteroarene that is an analogue of cyclopentadiene with O in place of CH2 at position 1 and N in place of CH at position 3.. oxazole : An azole based on a five-membered heterocyclic aromatic skeleton containing one N and one O atom.	2.07	1	1,3-oxazoles; mancude organic heteromonocyclic parent; monocyclic heteroarene
alpha-methylhistamine (R)-alpha-methylhistamine : An aralkylamino compound that is histamine bearing a methyl substituent at the alpha-position (the R-enantiomer).	2.05	1	aralkylamino compound; imidazoles	H3-receptor agonist
conessine conessine : A steroid alkaloid that is con-5-enine substituted by a N,N-dimethylamino group at position 3. It has been isolated from the plant species of the family Apocynaceae.	2.04	1	steroid alkaloid; tertiary amino compound	antibacterial agent; antimalarial; H3-receptor antagonist; plant metabolite
jnj-5207852 [no description available]	2.44	2
thioperamide thioperamide: structure given in first source; histamine H3 receptor antagonist	2.45	2	primary aliphatic amine
ciproxifan [no description available]	2.04	1	aromatic ketone
nnc 38-1049 NNC 38-1049: selective histamine H3 receptor antagonist; structure in first source	2.05	1
benzonitrile, 4-(2-(2-((2r)-2-methyl-1-pyrrolidinyl)ethyl)-5-benzofuranyl)- [no description available]	2.05	1
jnj 10181457 [no description available]	2.05	1
pitolisant pitolisant: functions as both inverse agonist and antagonist of histamine H3 receptors; structure in first source	2.05	1	organochlorine compound
fistularin 3 fistularin 3: structure given in first source; isolated from the marine sponge Aplysina archeri	2.07	1

Condition	Relationship Strength	Studies
Anaplastic Astrocytoma [description not available]	2.1	1
Astrocytoma Neoplasms of the brain and spinal cord derived from glial cells which vary from histologically benign forms to highly anaplastic and malignant tumors. Fibrillary astrocytomas are the most common type and may be classified in order of increasing malignancy (grades I through IV). In the first two decades of life, astrocytomas tend to originate in the cerebellar hemispheres; in adults, they most frequently arise in the cerebrum and frequently undergo malignant transformation. (From Devita et al., Cancer: Principles and Practice of Oncology, 5th ed, pp2013-7; Holland et al., Cancer Medicine, 3d ed, p1082)	2.1	1
Animal Diseases Diseases that occur in VERTEBRATE animals.	2.07	1

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interferon-gamma

Description

Cross-References

Synonyms (13)

Protein Targets (2)

Inhibition Measurements

Biological Processes (5)

Molecular Functions (3)

Ceullar Components (4)

Bioassays (9)

Research

Studies (8)

Study Types

Clinical Trials (111)

Trial Overview

Trial Outcomes

Bronchoalveolar Lavage (BAL) to Measure Flow of Cytometry and Cytokine Levels

Sputum Conversion

Chest Cavity Size

Number of Patients With Response

Best Response (BR)

Time to Progression

Early Response Rate (RR) (Stratum 1 Only)

Change in Macular Volume in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week Two Compared to Baseline

Change in Macular Volume in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week One Compared to Baseline

Change in Macular Volume in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week Two Compared to Baseline

Change in Macular Volume in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week One Compared to Baseline

Change in Intraocular Pressure (IOP) in the Study Eye at Week Two Compared to Baseline

Change in Intraocular Pressure (IOP) in the Study Eye at Week One Compared to Baseline

Change in Intraocular Pressure (IOP) in the Fellow Eye at Week Two Compared to Baseline

Change in Intraocular Pressure (IOP) in the Fellow Eye at Week One Compared to Baseline

Change in Excess Central Macular Thickening in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week Two Compared to Baseline

Change in Excess Central Macular Thickening in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week One Compared to Baseline

Change in Excess Central Macular Thickening in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week One Compared to Baseline

Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Study Eye at Week Two Compared to Baseline

Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Study Eye at Week One Compared to Baseline

Proportion of Participants With a Visual Loss of 15 or More Early Treatment Diabetic Retinopathy Study (ETDRS) Letters in the Study Eye

Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week One Compared to Baseline

Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week Two Compared to Baseline

Change in Excess Central Macular Thickening in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week Two Compared to Baseline

Total Number of Ocular Adverse Events Related to Investigational Product

Changes in Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week 2 Compared to Baseline

Changes in Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week 48 Compared to Baseline

Changes in Best-corrected Visual Acuity (BCVA) in the Study Eye at Week 2 Compared to Baseline

Changes in Best-corrected Visual Acuity (BCVA) in the Study Eye at Week 48 Compared to Baseline

Number of Participants Who Withdrew From the Study

Total Number of Non-ocular Adverse Events Related to the Investigational Product

Total Number of Severe Non-ocular Adverse Events Related to the Investigational Product

Total Number of Severe Ocular Adverse Events Related to the Investigational Product

Change in Class I Major Histocompatibility Complex (MHC) Expression After Treatment With IFN Gamma

Changes in Immune Response

MHC Class II Expression

Change in Total Friedreich Ataxia Rating Scale (FARS) Score

Change in Whole Blood Frataxin Levels

Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 3

Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 5

Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 52

Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 52

Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 8

Change in Central Visual Field Sensitivity at Day 2 and Week 5 Compared to Baseline.

Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 1

Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 2

Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 3

Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 2

Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 5

Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 52

Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 2

Number and Severity of IP-related AEs

Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 8

Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 2

Number of Participants Who Withdrew

Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 1

Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 2

Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 3

Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 5

Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 52

Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 8

Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 1

Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 2