| 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 | 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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
Change in Macular Volume in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week One 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 1 Week
| Intervention | mm^3 (Mean) |
|---|
| Interferon Gamma-1b | -0.02 |
Change in Macular Volume in the Fellow 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 | 0.06 |
Change in Macular Volume in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week One 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 1 Week
| Intervention | mm^3 (Mean) |
|---|
| Interferon Gamma-1b | 0.14 |
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 |
Change in Intraocular Pressure (IOP) in the Study Eye at Week One 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 1 Week
| Intervention | mm Hg (Mean) |
|---|
| Interferon Gamma-1b | 0.40 |
Change in Intraocular Pressure (IOP) in the Fellow 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 | -2.20 |
Change in Intraocular Pressure (IOP) in the Fellow Eye at Week One 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 1 Week
| Intervention | mm Hg (Mean) |
|---|
| Interferon Gamma-1b | -1.60 |
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 |
Change in Excess Central Macular Thickening in the Study Eye, as Measured by Optical Coherence Tomography (OCT), at Week One 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 1 Week
| Intervention | µm (Mean) |
|---|
| Interferon Gamma-1b | -38.60 |
Change in Excess Central Macular Thickening in the Fellow Eye, as Measured by Optical Coherence Tomography (OCT), at Week One 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 1 Week
| Intervention | µm (Mean) |
|---|
| Interferon Gamma-1b | 12.00 |
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 |
Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Study Eye at Week One 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 1 Week
| Intervention | ETDRS Letters (Mean) |
|---|
| Interferon Gamma-1b | 0.20 |
Proportion of Participants With a Visual Loss of 15 or More Early Treatment Diabetic Retinopathy Study (ETDRS) Letters in the Study Eye
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 | Percentage of Participants (Number) |
|---|
| Interferon Gamma-1b | 0 |
Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week One 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 1 Week
| Intervention | ETDRS Letters (Mean) |
|---|
| Interferon Gamma-1b | 1.60 |
Change in ETDRS Best-corrected Visual Acuity (BCVA) in the Fellow 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 | 3.20 |
Change in Excess Central Macular Thickening in the Fellow 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 | 30.20 |
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 |
Changes in Best-corrected Visual Acuity (BCVA) in the Fellow Eye at Week 48 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 48
| Intervention | ETDRS letters (Mean) |
|---|
| Interferon Gamma-1b | 4.67 |
Changes in Best-corrected Visual Acuity (BCVA) in the Study 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 | 0.4 |
Changes in Best-corrected Visual Acuity (BCVA) in the Study Eye at Week 48 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 48
| Intervention | ETDRS letters (Mean) |
|---|
| Interferon Gamma-1b | 2.67 |
Number of Participants Who Withdrew From the Study
(NCT01468337)
Timeframe: Week 48
| Intervention | participants (Number) |
|---|
| Interferon Gamma-1b | 0 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
Change From Baseline in White Blood Cell Count (WBC)
(NCT02666768)
Timeframe: 6 months
| Intervention | x1000 cells/uL (Median) |
|---|
| Gamma Interferon-1b | -0.5 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |