Page last updated: 2024-11-03

4-aminobenzoic acid and Erythremia

4-aminobenzoic acid has been researched along with Erythremia in 4 studies

para-Aminobenzoates: Benzoic acids, salts, or esters that contain an amino group attached to carbon number 4 of the benzene ring structure.
4-aminobenzoate : An aromatic amino-acid anion that is the conjugate base of 4-aminobenzoic acid.

Research Excerpts

ExcerptRelevanceReference
" Overall, the idasanutlin dosing regimen showed clinical activity and rapidly reduced JAK2 allele burden in patients with HU-resistant/- intolerant PV but was associated with low-grade gastrointestinal toxicity, leading to poor long-term tolerability."3.11The MDM2 antagonist idasanutlin in patients with polycythemia vera: results from a single-arm phase 2 study. ( Bellini, M; Burbury, K; El-Galaly, TC; Gerds, A; Gupta, V; Higgins, B; Huw, LY; Jamois, C; Katakam, S; Kovic, B; Maffioli, M; Mascarenhas, J; Mesa, R; Palmer, J; Passamonti, F; Ross, DM; Vannucchi, AM; Wonde, K; Yacoub, A, 2022)

Research

Studies (4)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's0 (0.00)29.6817
2010's2 (50.00)24.3611
2020's2 (50.00)2.80

Authors

AuthorsStudies
Mascarenhas, J2
Passamonti, F1
Burbury, K1
El-Galaly, TC1
Gerds, A1
Gupta, V1
Higgins, B1
Wonde, K1
Jamois, C1
Kovic, B1
Huw, LY1
Katakam, S1
Maffioli, M1
Mesa, R1
Palmer, J1
Bellini, M1
Ross, DM1
Vannucchi, AM1
Yacoub, A1
Ritchie, EK1
Marcellino, BK1
Farnoud, N1
Cassinat, B1
Lu, M1
Verger, E1
McGovern, E1
Patel, M1
Medina-Martinez, J1
Levine, MF1
Arango Ossa, JE1
Zhou, Y1
Kosiorek, H1
Mehrotra, M1
Houldsworth, J1
Dueck, A1
Rossi, M1
Kiladjian, JJ1
Rampal, RK1
Hoffman, R1
Foucar, CE1
Stein, BL1

Clinical Trials (1)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
A Phase II, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Idasanutlin Monotherapy in Patients With Hydroxyurea-Resistant/Intolerant Polycythemia Vera[NCT03287245]Phase 227 participants (Actual)Interventional2018-02-21Terminated (stopped due to The Sponsor decided to discontinue the development of idasanutlin in the polycythemia vera indication.)
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

Percentage of All Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants Who Achieved Complete Hematologic Remission at Cycle 11 Day 28

Complete hematologic remission requires all of the following: Hct control without phlebotomy between Weeks 32 and Cycle 11 Day 28; WBC count ≤10 × 10^9/L at Cycle 11 Day 28; and Platelet count ≤400 × 10^9/L at Week 32. Hct control is defined as protocol-specified ineligibility for phlebotomy. Eligibility for phlebotomy is defined as a Hct level ≥45% that was ≥3% higher than baseline level or a Hct level of >48%. (NCT03287245)
Timeframe: Cycle 11 Day 28

InterventionPercentage of Participants (Number)
Ruxolitinib-Naïve Participants With Splenomegaly40
Ruxolitinib-Naïve Participants Without Splenomegaly100
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly0
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly0

Percentage of All Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants Who Achieved Complete Hematologic Response at Week 32

Complete hematologic response requires all of the following: Hct control without phlebotomy; White blood cell (WBC) count ≤10 × 10^9/Liter (L) at Week 32; and Platelet count ≤400 × 10^9/L at Week 32. Hct control is defined as protocol-specified ineligibility for phlebotomy between Weeks 8 to 32 and ≤1 instance of phlebotomy eligibility between first dose and Week 8. Eligibility for phlebotomy is defined as a Hct level ≥45% that was ≥3% higher than baseline level or a Hct level of >48%. (NCT03287245)
Timeframe: Week 32 (Cycle 8 Day 28)

InterventionPercentage of Participants (Number)
Ruxolitinib-naïve Participants With Splenomegaly33.3
Ruxolitinib-naïve Participants Without Splenomegaly100
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly75.0
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly0

Percentage of All Ruxolitinib-Naïve Participants (Irrespective of Spleen Size) Who Achieved Hct Control Without Phlebotomy at Week 32

Hct control is defined as protocol-specified ineligibility for phlebotomy between Weeks 8 to 32 and ≤1 instance of phlebotomy eligibility between first dose and Week 8. Eligibility for phlebotomy is defined as a Hct level ≥45% that was ≥3% higher than baseline level or a Hct level of >48%. (NCT03287245)
Timeframe: Week 32

InterventionPercentage of Participants (Number)
Ruxolitinib-Naïve Participants With Splenomegaly44.4
Ruxolitinib-Naïve Participants Without Splenomegaly100
Total Ruxolitinib-Naïve Participants54.5

Percentage of All Ruxolitinib-Resistant or Intolerant Participants Who Achieved Hct Control Without Phlebotomy at Week 32

Hct control is defined as protocol-specified ineligibility for phlebotomy between Weeks 8 to 32 and ≤1 instance of phlebotomy eligibility between first dose and Week 8. Eligibility for phlebotomy is defined as a Hct level ≥45% that was ≥3% higher than baseline level or a Hct level of >48%. (NCT03287245)
Timeframe: From Baseline to Week 32 (Cycle 8 Day 28)

InterventionPercentage of Participants (Number)
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly75.0
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly0
Total Ruxolitinib-Resistant or Intolerant Participants60

Percentage of Participants With Clinical Laboratory Abnormalities: Clinical Chemistry Parameters

"Clinical chemistry parameter laboratory values falling outside the standard reference range were to be recorded as either high or low.~There was no clinical chemistry abnormalities identified. The study was pre-maturely terminated by the sponsor's decision, therefore did not reach the end of study." (NCT03287245)
Timeframe: Baseline to end of study (up to 2 years)

InterventionPercentage of Participants (Number)
Ruxolitinib-Naïve Participants With Splenomegaly0
Ruxolitinib-Naïve Participants Without Splenomegaly0
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly0
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly0

Percentage of Participants With Clinical Laboratory Abnormalities: Hematology Parameters.

"Hematology parameter laboratory values falling outside the standard reference range were planned to be recorded as either high or low.~There was no clinical laboratory abnormalities identified. The study was pre-maturely terminated by the sponsor's decision, therefore did not reach the end of study." (NCT03287245)
Timeframe: Baseline to end of study (up to 2 years)

InterventionPercentage of Participants (Number)
Ruxolitinib-Naïve Participants With Splenomegaly0
Ruxolitinib-Naïve Participants Without Splenomegaly0
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly0
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly0

Percentage of Participants With Clinical Laboratory Abnormalities: Urinalysis Parameters

"Urinalysis parameter laboratory values falling outside the standard reference range were planned to be recorded as either high or low.~There was no clinical laboratory (urinalysis) abnormalities identified. The study was pre-maturely terminated by the sponsor's decision, therefore did not reach the end of study." (NCT03287245)
Timeframe: Baseline to end of study (up to 2 years)

InterventionPercentage of Participants (Number)
Ruxolitinib-naïve Participants With Splenomegaly0
Ruxolitinib-naïve Participants Without Splenomegaly0
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly0
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly0

Percentage of Participants With Concomitant Medications

"Participants with Concomitant Medications used from 28 days prior to screening until the final visit or end of study (EOS) were reported.~The result data not derived due to early termination of the study by the sponsor's decision and did not reach the end of study." (NCT03287245)
Timeframe: Overall Study Period

InterventionPercentage of Participants (Number)
Ruxolitinib-naïve Participants With Splenomegaly100
Ruxolitinib-naïve Participants Without SplenomegalyEdit100
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly100
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly100

Percentage of Ruxolitinib-Naïve Participants With Splenomegaly at Baseline Who Achieved Composite Response at Week 32

Composite response is defined as hematocrit (Hct) control without phlebotomy and ≥35% decrease in spleen size by imaging at Week 32. Hct control is defined as protocol-specified ineligibility for phlebotomy between Weeks 8 to 32 and ≤1 instance of phlebotomy eligibility between first dose and Week 8. Eligibility for phlebotomy is defined as a Hct of ≥45% that was ≥3% higher than baseline level or a Hct of >48%. One Cycle is 28 Days. (NCT03287245)
Timeframe: Week 32

InterventionPercentage of Participants (Number)
Ruxolitinib-Naïve Participants With Splenomegaly44.4

Percentage of Ruxolitinib-Naïve Participants Without Splenomegaly at Baseline Who Achieved Hematocrit (Hct) Control Without Phlebotomy at Week 32

Hct control is defined as protocol-specified ineligibility for phlebotomy between Weeks 8 to 32 and ≤1 instance of phlebotomy eligibility between first dose and Week 8. Eligibility for phlebotomy is defined as a Hct level ≥45% that was ≥3% higher than baseline level or a Hct level of >48%. (NCT03287245)
Timeframe: Week 32

InterventionPercentage of Participants (Number)
Ruxolitinib-Naïve Participants Without Splenomegaly100

Baseline and Mean Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) Scores Over Time

"Reported EORTC QLQ-C30 Scores include: Cognitive function, Diarrhea Emotional functioning, Nausea and vomiting, Social functioning, Physical functioning, Global health status/QoL and Role functioning. The questions use a 4-point scale (1 'Not at all' to 4 'Very much'; 2 questions used 7-point scale [1 'very poor' to 7 'Excellent']). Scores are averaged and transformed to 0-100 scale; higher score=better level of functioning or greater degree of symptoms.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the end of study." (NCT03287245)
Timeframe: Baseline (Cycle 1 Day 1), Cycle 2 Day 1, Cycles 3, 5, 8, 11, 14, 17, 20 Day 28 and Final Visit

,
InterventionScore on a Scale (Mean)
EORTC QLQ-C30 Scores: Cognitive function BaselineEORTC QLQ-C30 Scores: Cognitive function Cycle 2, Day 1EORTC QLQ-C30 Scores: Cognitive function Cycle 3 Day 28EORTC QLQ-C30 Scores: Cognitive function Cycle 5 Day 28EORTC QLQ-C30 Scores: Cognitive function Cycle 8 (Week 32)EORTC QLQ-C30 Scores: Cognitive function Cycle 11 Day 28EORTC QLQ-C30 Scores: Cognitive function Cycle 14 Day 28EORTC QLQ-C30 Scores: Cognitive function Cycle 17 Day 28EORTC QLQ-C30 Scores: Cognitive function Cycle 20 Day 28EORTC QLQ-C30 Scores: Cognitive function Final VisitEORTC QLQ-C30 Scores: Diarrhea BaselineEORTC QLQ-C30 Scores: Diarrhea Cycle 2 Day 1EORTC QLQ-C30 Scores: Diarrhea Cycle 3 Day 28EORTC QLQ-C30 Scores: Diarrhea Cycle 5 Day 28EORTC QLQ-C30 Scores: Diarrhea Cycle 8 (Week 32)EORTC QLQ-C30 Scores: Diarrhea Cycle 11 Day 28EORTC QLQ-C30 Scores: Diarrhea Cycle 14 Day 28EORTC QLQ-C30 Scores: Diarrhea Cycle 17 Day 28EORTC QLQ-C30 Scores: Diarrhea Cycle 20 Day 28EORTC QLQ-C30 Scores: Diarrhea Final visitEORTC QLQ-C30 Scores: Emotional functioning BaselineEORTC QLQ-C30 Scores: Emotional functioning Cycle 1 Day 28EORTC QLQ-C30 Scores: Emotional functioning Cycle 3 Day 28EORTC QLQ-C30 Scores: Emotional functioning Cycle 5 Day 28EORTC QLQ-C30 Scores: Emotional functioning Cycle 8 (Week 32)EORTC QLQ-C30 Scores: Emotional functioning Cycle 11 Day 28EORTC QLQ-C30 Scores: Emotional functioning Cycle 14 Day 28EORTC QLQ-C30 Scores: Emotional functioning Cycle 17 Day 28EORTC QLQ-C30 Scores: Emotional functioning Cycle 20 Day 28EORTC QLQ-C30 Scores: Emotional functioning Final visitEORTC QLQ-C30 Scores: Nausea and vomiting BaselineEORTC QLQ-C30 Scores: Nausea and vomiting Cycle 2 Day 1EORTC QLQ-C30 Scores: Nausea and vomiting Cycle 3 Day 28EORTC QLQ-C30 Scores: Nausea and vomiting Cycle 5 Day 28EORTC QLQ-C30 Scores: Nausea and vomiting Cycle 8 (Week 32)EORTC QLQ-C30 Scores: Nausea and vomiting Cycle 11 Day 28EORTC QLQ-C30 Scores: Nausea and vomiting Cycle 14 Day 28EORTC QLQ-C30 Scores: Nausea and vomiting Cycle 20 Day 28EORTC QLQ-C30 Scores: Nausea and vomiting Final visitEORTC QLQ-C30 Scores: Social functioning BaselineEORTC QLQ-C30 Scores: Social functioning Cycle 2 Day 1EORTC QLQ-C30 Scores: Social functioning Cycle 3 Day 28EORTC QLQ-C30 Scores: Social functioning Cycle 5 Day 28EORTC QLQ-C30 Scores: Social functioning Cycle 8 (Week 32)EORTC QLQ-C30 Scores: Social functioning Cycle 11 Day 28EORTC QLQ-C30 Scores: Social functioning Cycle 14 Day 28EORTC QLQ-C30 Scores: Social functioning Cycle 17 Day 28EORTC QLQ-C30 Scores: Social functioning Cycle 20 Day 28EORTC QLQ-C30 Scores: Social functioning Final visitEORTC QLQ-C30 Scores: Physical functioning BaselineEORTC QLQ-C30 Scores: Physical functioning Cycle 2 Day 1EORTC QLQ-C30 Scores: Physical functioning Cycle 3 Day 28EORTC QLQ-C30 Scores: Physical functioning Cycle 5 Day 28EORTC QLQ-C30 Scores: Physical functioning Cycle 8 (Week 32)EORTC QLQ-C30 Scores: Physical functioning Cycle 11 Day 28EORTC QLQ-C30 Scores: Physical functioning Cycle 14 Day 28EORTC QLQ-C30 Scores: Physical functioning Cycle 17 Day 28EORTC QLQ-C30 Scores: Physical functioning Cycle 20 Day 28EORTC QLQ-C30 Scores: Physical functioning Final visitEORTC QLQ-C30 Scores: Global health status/QoL BaselineEORTC QLQ-C30 Scores: Global health status/QoL Cycle 2 Day 1EORTC QLQ-C30 Scores: Global health status/QoL Cycle 3 Day 28EORTC QLQ-C30 Scores: Global health status/QoL Cycle 5 Day 28EORTC QLQ-C30 Scores: Global health status/QoL Cycle 8 (Week 32)EORTC QLQ-C30 Scores: Global health status/QoL Cycle 11 Day 28EORTC QLQ-C30 Scores: Global health status/QoL Cycle 14 Day 28EORTC QLQ-C30 Scores: Global health status/QoL Cycle 17 Day 28EORTC QLQ-C30 Scores: Global health status/QoL Cycle 20 Day 28EORTC QLQ-C30 Scores: Global health status/QoL Final visitEORTC QLQ-C30 Scores: Role functioning BaselineEORTC QLQ-C30 Scores: Role functioning Cycle 2 Day 1EORTC QLQ-C30 Scores: Role functioning Cycle 3 Day 28EORTC QLQ-C30 Scores: Role functioning Cycle 5 Day 28EORTC QLQ-C30 Scores: Role functioning Cycle 8 (Week 32)EORTC QLQ-C30 Scores: Role functioning Cycle 11 Day 28EORTC QLQ-C30 Scores: Role functioning Cycle 14 Day 28EORTC QLQ-C30 Scores: Role functioning Cycle 17 Day 28EORTC QLQ-C30 Scores: Role functioning Cycle 20 Day 28EORTC QLQ-C30 Scores: Role functioning Final Visit
Ruxolitinib-Naïve Participants65.8311.117.021.196.068.335.560.0004.448.33-5.561.757.149.095.565.5625.00013.3365.8313.4314.0416.0714.396.945.56-8.3303.3310.00-4.63-1.75-2.387.582.78-2.78011.1167.504.63-1.755.950.000.000.00-4.1700.0086.331.48-2.811.435.45-1.11-1.111.670-4.4461.252.317.897.149.091.392.7810.4225.00-3.3374.177.412.637.1415.15-2.785.568.3316.67-13.33
Ruxolitinib-Resistant or Intolerant Participants76.198.336.6711.11-3.3300-8.3306.6714.295.5605.5620.0000006.6764.2915.288.336.945.0016.67-4.1708.3316.672.388.333.338.3316.670.00-8.3306.6776.190.003.33-2.78-6.670000-6.6781.902.222.67-2.22-4.00-10.000.0000-2.6760.711.3911.670.00-8.338.338.3316.6725.0013.3376.19-8.330.00-2.780.00-8.330006.67

Baseline and Mean Change From Baseline Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) Over Time

"MPN-SAF Total Symptom Score is the sum of the following 10 items: early satiety, abdominal discomfort, inactivity, concentration issues, night sweats, Itching, Bone pain, Fever and Unintentional weight loss last 6 months and fatigue. The participant provides a severity score for each symptom on a scale of 0 as a minimum score (none/absent) to 10 (worst imaginable) as a maximum score.~Baseline (Cycle 1, Day 1) MPN-SAF total symptom score and the mean change from baseline score at each timepoint are reported. The change from baseline score was calculated by subtracting the post-baseline score from the baseline score.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the end of study." (NCT03287245)
Timeframe: Baseline (Cycle 1 Day 1), Cycle 2 Day 1, Days 28 of Cycles 3, 5, 8 (Week 32), 11, 14, 17 ) Day 28, Cycle 5 Day 28, End of Cycle 8 (Week 32), and Final Visit

,
InterventionScore on a Scale (Mean)
Baseline (Cycle 1 Day 1)Cycle 2 Day 1Cycle 3 Day 28Cycle 5 Day 28,Week 32Cycle 11 Day 28Cycle 14 Day 28Cycle 17 Day 28Cycle 20 Day 28Final Visit
Ruxolitinib-Naïve Participants31.95-5.06-6.38-7.00-8.20-4.60-4.67-3.25-12.00-5.92
Ruxolitinib-Resistant or Intolerant Participants26.000.80-8.00-9.50-5.00-7.50-10.50-12.00-8.00-7.20

Change From Baseline in Diastolic Blood Pressure

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionMillimeters of mercury (mmHg) (Mean)
BaselineCycle1, Day 15Cycle 1, Day 22Cycle 2, Day 1Cycle 2, Day 15Cycle 3 Day 1Cycle 3, Day 15Cycle 4, Day 1Cycle 5, Day 1Cycle 6, Day 1Cycle 7, Day 1Cycle 8, Day 1Cycle 9, Day 1Cycle 10, Day 1Cycle 11, Day 1Cycle 12, Day 1Cycle 13, Day 1Cycle 14, Day 1Cycle 15, Day 1Cycle 16, Day 1Cycle 17, Day 1Final visit
Ruxolitinib-naïve Participants Without Splenomegaly73.210.02.43.02.55.82.02.75.03.34.55.04.05.010.06.04.019.010.011.011.03.8

Change From Baseline in Diastolic Blood Pressure

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionMillimeters of mercury (mmHg) (Mean)
BaselineCycle1, Day 15Cycle 1, Day 22Cycle 2, Day 1Cycle 2, Day 15Cycle 3 Day 1Cycle 3, Day 15Cycle 4, Day 1Cycle 5, Day 1Cycle 6, Day 1Cycle 7, Day 1Cycle 8, Day 1Cycle 9, Day 1Cycle 10, Day 1Cycle 11, Day 1Cycle 12, Day 1Cycle 13, Day 1Cycle 14, Day 1Cycle 15, Day 1Cycle 16, Day 1Cycle 17, Day 1Cycle 18, Day 1Cycle 19, Day 1Cycle 20, Day 1Final visit
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly72.3-1.8-2.2-0.2-5.5-4.0-7.5-0.4-5.0-2.0-5.30.5-2.7-2.31.02.02.0-9.0-2.07.03.0-2.05.0-1.0-2.0

Change From Baseline in Diastolic Blood Pressure

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionMillimeters of mercury (mmHg) (Mean)
BaselineCycle1, Day 15Cycle 1, Day 22Cycle 2, Day 1Cycle 2, Day 15Cycle 3 Day 1Cycle 3, Day 15Cycle 4, Day 1Cycle 5, Day 1Cycle 6, Day 1Cycle 7, Day 1Cycle 8, Day 1Cycle 9, Day 1Cycle 10, Day 1Cycle 11, Day 1Cycle 12, Day 1Cycle 13, Day 1Cycle 14, Day 1Cycle 15, Day 1Cycle 16, Day 1Cycle 17, Day 1Cycle 18, Day 1Cycle 19, Day 1Cycle 20, Day 1Cycle 21, Day 1Cycle 22, Day 1Final visit
Ruxolitinib-naïve Participants With Splenomegaly76.35.13.66.14.16.21.85.51.75.92.97.84.13.79.79.89.05.67.09.811.35.04.012.5-3.022.05.8

Change From Baseline in Diastolic Blood Pressure

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionMillimeters of mercury (mmHg) (Mean)
BaselineCycle1, Day 15Cycle 1, Day 22Cycle 2, Day 1Cycle 2, Day 15Cycle 3 Day 1Cycle 3, Day 15Cycle 4, Day 1Cycle 5, Day 1Cycle 6, Day 1Cycle 7, Day 1Cycle 8, Day 1Cycle 9, Day 1Cycle 10, Day 1Cycle 11, Day 1Cycle 12, Day 1Cycle 13, Day 1Cycle 14, Day 1Cycle 15, Day 1Cycle 16, Day 1Cycle 17, Day 1Cycle 18, Day 1Cycle 19, Day 1Cycle 20, Day 1Cycle 21, Day 1Cycle 22, Day 1Cycle 23, Day 1Final visit
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly65.00.0-1.03.016.05.08.01.01.012.030.010.04.03.024.08.010.011.07.06.07.03.08.02.07.08.07.011.0

Change From Baseline in Electrocardiogram Parameters: PQ(PR), QRS, QT, QTcB, QTcF, and RR Durations

"Single 12-lead ECGs was obtained using an ECG machine that automatically calculates the heart rate and measures PR, QRS, QT, and QTc intervals. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Day 1, hours 4 and 6, Day 2 pre-dose and 24 Hour, Day 5 pre-dose, 4 and 6 Hour), Cycle 2 (Day 1 pre-dose only), Cycle 3 (Day 1 pre-dose, 4 and 6 Hour), Cycle 4 (Day 1 pre-dose and 4 Hour)

,
InterventionMillisecond (msec) (Mean)
PR Duration BaselinePQ(PR) Durations Cycle 1, Day 1, 4 hourPQ(PR) Durations Cycle 1, Day 1, 6 hourPQ(PR) Durations Cycle 1, Day 2, 24 hourPQ(PR) Durations Cycle ,1 Day 5, pre-dosePQ(PR) Durations Cycle 1, Day 5, 4 hourPQ(PR) Durations Cycle 1, Day 5, 6 hourPQ(PR) Durations Cycle 2, Day1, pre-doseQRS Duration BaselineQRS Cycle 1 Day 1 (4 H)QRS Cycle 1 Day 1 (6 H)QRS Cycle 1 Day 2 (24 H)QRS Cycle 1 Day 5 (PREDOSE)QRS Cycle 1 Day 5 (4 H)QRS Cycle 1 Day 5 (6 H)QRS Cycle 2 Day 1 (PREDOSE)QT Duration BaselineQT Duration Cycle 1 Day 1 (4 H)QT Duration Cycle 1 Day 1 (6 H)QT Duration Cycle 1 Day 2 (24 H)QT Duration Cycle 1 Day 5 (PREDOSE)QT Duration Cycle 1 Day 5 (4 H)QT Duration Cycle 1 Day 5 (6 H)QT Duration Cycle 2 Day 1 (PREDOSE)QTcB baselineQTcB - Bazett's Correction Formula Cycle 1 Day 1 (4 H)QTcB - Bazett's Correction Formula Cycle 1 Day 1 (6 H)QTcB - Bazett's Correction Formula Cycle 1 Day 2 (24 H)QTcB - Bazett's Correction Formula Cycle 1 Day 5 (PREDOSE)QTcB - Bazett's Correction FormulaCycle 1 Day 5 (4 H)QTcB - Bazett's Correction Formula Cycle 1 Day 5 (6 H)QTcB - Bazett's Correction Formula Cycle 2 Day 1 (PREDOSE)QTcF Durations Fridericia's Correction Formula BaselineQTcF Durations Fridericia's Correction Formula Cycle 1 Day 1 (4 H)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 1 (6 H)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 2 (24 H)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 5 (PREDOSE)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 5 (4 H)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 5 (6 H)QTcF Durations Fridericia's Correction Formula Cycle 2 Day 1 (PREDOSE)RR Duration BaselineRR Duration Cycle 1 Day 1 4 HRR Duration Cycle 1 Day 1 6 HRR Duration Cycle 1 Day 2 (24 H)RR Duration Cycle 1 Day 5 (PREDOSE)RR Duration Cycle 1 Day 5 (4 H)RR Duration Cycle 1 Day 5 (6 H)RR Duration Cycle 2 Day 1 (PREDOSE)
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly152.00-2.006.000.670.40-1.207.60-2.3383.331.330.333.000.00-0.402.001.00386.005.67-1.337.008.003.207.6019.00424.5013.836.334.50-9.20-4.60-1.600.17411.1711.673.505.33-3.20-2.001.606.83835.83-36.50-31.178.8367.8024.2033.0078.17
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly196.004.000.00-12.00-4.0000-8.0094.000-2.00-2.002.0000-4.00392.0036.0036.0004.00-12.00-4.0012.00444.00-30.005.00-14.00-7.000.00-12.00-23.00426.00-8.0016.00-9.00-3.00-4.006.00-11.00779.00292.00130.0054.0043.00-47.00-56.00144.00

Change From Baseline in Electrocardiogram Parameters: PQ(PR), QRS, QT, QTcB, QTcF, and RR Durations

"Single 12-lead ECGs was obtained using an ECG machine that automatically calculates the heart rate and measures PR, QRS, QT, and QTc intervals. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Day 1, hours 4 and 6, Day 2 pre-dose and 24 Hour, Day 5 pre-dose, 4 and 6 Hour), Cycle 2 (Day 1 pre-dose only), Cycle 3 (Day 1 pre-dose, 4 and 6 Hour), Cycle 4 (Day 1 pre-dose and 4 Hour)

InterventionMillisecond (msec) (Mean)
PR Duration BaselinePQ(PR) Durations Cycle 1, Day 1, 4 hourPQ(PR) Durations Cycle 1, Day 1, 6 hourPQ(PR) Durations Cycle 1, Day 2, pre-dosePQ(PR) Durations Cycle 1, Day 2, 24 hourPQ(PR) Durations Cycle ,1 Day 5, pre-dosePQ(PR) Durations Cycle 1, Day 5, 4 hourPQ(PR) Durations Cycle 1, Day 5, 6 hourPQ(PR) Durations Cycle 2, Day1, pre-doseQRS Duration BaselineQRS Cycle 1 Day 1 (4 H)QRS Cycle 1 Day 1 (6 H)QRS Cycle 1 Day 2 (PREDOSE)QRS Cycle 1 Day 2 (24 H)QRS Cycle 1 Day 5 (PREDOSE)QRS Cycle 1 Day 5 (4 H)QRS Cycle 1 Day 5 (6 H)QRS Cycle 2 Day 1 (PREDOSE)QT Duration BaselineQT Duration Cycle 1 Day 1 (4 H)QT Duration Cycle 1 Day 1 (6 H)QT Duration Cycle 1 Day 2 (PREDOSE)QT Duration Cycle 1 Day 2 (24 H)QT Duration Cycle 1 Day 5 (PREDOSE)QT Duration Cycle 1 Day 5 (4 H)QT Duration Cycle 1 Day 5 (6 H)QT Duration Cycle 2 Day 1 (PREDOSE)QTcB baselineQTcB - Bazett's Correction Formula Cycle 1 Day 1 (4 H)QTcB - Bazett's Correction Formula Cycle 1 Day 1 (6 H)QTcB - Bazett's Correction Formula Cycle 1 Day 2 (PREDOSE)QTcB - Bazett's Correction Formula Cycle 1 Day 2 (24 H)QTcB - Bazett's Correction Formula Cycle 1 Day 5 (PREDOSE)QTcB - Bazett's Correction FormulaCycle 1 Day 5 (4 H)QTcB - Bazett's Correction Formula Cycle 1 Day 5 (6 H)QTcB - Bazett's Correction Formula Cycle 2 Day 1 (PREDOSE)QTcF Durations Fridericia's Correction Formula BaselineQTcF Durations Fridericia's Correction Formula Cycle 1 Day 1 (4 H)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 1 (6 H)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 2 (PREDOSE)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 2 (24 H)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 5 (PREDOSE)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 5 (4 H)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 5 (6 H)QTcF Durations Fridericia's Correction Formula Cycle 2 Day 1 (PREDOSE)RR Duration BaselineRR Duration Cycle 1 Day 1 4 HRR Duration Cycle 1 Day 1 6 HRR Duration Cycle 1 Day 2 (PREDOSE)RR Duration Cycle 1 Day 2 (24 H)RR Duration Cycle 1 Day 5 (PREDOSE)RR Duration Cycle 1 Day 5 (4 H)RR Duration Cycle 1 Day 5 (6 H)RR Duration Cycle 2 Day 1 (PREDOSE)
Ruxolitinib-naïve Participants Without Splenomegaly153.60-2.60-4.40-6.001.00-12.00-6.25-2.75-5.7583.804.604.002.001.754.003.75-0.257.75392.6016.8011.00-18.002.75-8.7515.254.7510.50419.6021.008.40-410.00-0.75-10.001.50-4.757.75410.0018.8010.00-20.000.00-9.254.00-2.258881.00-5.6014.0018.0017.25-0.7579.7549.5023.75

Change From Baseline in Electrocardiogram Parameters: PQ(PR), QRS, QT, QTcB, QTcF, and RR Durations

"Single 12-lead ECGs was obtained using an ECG machine that automatically calculates the heart rate and measures PR, QRS, QT, and QTc intervals. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Day 1, hours 4 and 6, Day 2 pre-dose and 24 Hour, Day 5 pre-dose, 4 and 6 Hour), Cycle 2 (Day 1 pre-dose only), Cycle 3 (Day 1 pre-dose, 4 and 6 Hour), Cycle 4 (Day 1 pre-dose and 4 Hour)

InterventionMillisecond (msec) (Mean)
PR Duration BaselinePQ(PR) Durations Cycle 1, Day 1, 4 hourPQ(PR) Durations Cycle 1, Day 1, 6 hourPQ(PR) Durations Cycle 1, Day 2, pre-dosePQ(PR) Durations Cycle 1, Day 2, 24 hourPQ(PR) Durations Cycle ,1 Day 5, pre-dosePQ(PR) Durations Cycle 1, Day 5, 4 hourPQ(PR) Durations Cycle 1, Day 5, 6 hourPQ(PR) Durations Cycle 2, Day1, pre-dosePQ(PR) Durations Cycle 3, Day 1, pre-dosePQ(PR) Durations Cycle 3, Day 1, 4 hourPQ(PR) Durations Cycle 3, Day 1, 6 hourPQ(PR) Durations Cycle 4, Day 1, pre-dosePQ(PR) Durations Cycle 4, Day 1, 4 hourQRS Duration BaselineQRS Cycle 1 Day 1 (4 H)QRS Cycle 1 Day 1 (6 H)QRS Cycle 1 Day 2 (PREDOSE)QRS Cycle 1 Day 2 (24 H)QRS Cycle 1 Day 5 (PREDOSE)QRS Cycle 1 Day 5 (4 H)QRS Cycle 1 Day 5 (6 H)QRS Cycle 2 Day 1 (PREDOSE)QRS Cycle 3 Day 1 (PREDOSE)QRS Cycle 3 Day 1 (4 H)QRS Cycle 3 Day 1 (6 H)QRS Cycle 4 Day 1 (PREDOSE)QRS Cycle 4 Day 1 (4 H)QT Duration BaselineQT Duration Cycle 1 Day 1 (4 H)QT Duration Cycle 1 Day 1 (6 H)QT Duration Cycle 1 Day 2 (PREDOSE)QT Duration Cycle 1 Day 2 (24 H)QT Duration Cycle 1 Day 5 (PREDOSE)QT Duration Cycle 1 Day 5 (4 H)QT Duration Cycle 1 Day 5 (6 H)QT Duration Cycle 2 Day 1 (PREDOSE)QT Durations Cycle 3 Day 1 (PREDOSE)QT Durations Cycle 3 Day 1 (4 H)QT Durations Cycle 3 Day 1 (6 H)QT Durations Cycle 4 Day 1 (PREDOSE)QT Durations Cycle 4 Day 1 (4 H)QTcB baselineQTcB - Bazett's Correction Formula Cycle 1 Day 1 (4 H)QTcB - Bazett's Correction Formula Cycle 1 Day 1 (6 H)QTcB - Bazett's Correction Formula Cycle 1 Day 2 (PREDOSE)QTcB - Bazett's Correction Formula Cycle 1 Day 2 (24 H)QTcB - Bazett's Correction Formula Cycle 1 Day 5 (PREDOSE)QTcB - Bazett's Correction Formula Cycle 4 Day 1 (4 H)QTcB - Bazett's Correction FormulaCycle 1 Day 5 (4 H)QTcB - Bazett's Correction Formula Cycle 1 Day 5 (6 H)QTcB - Bazett's Correction Formula Cycle 2 Day 1 (PREDOSE)QTcB - Bazett's Correction Formula Cycle 3 Day 1 (PREDOSE)QTcB - Bazett's Correction Formula Cycle 3 Day 1 (4 H)QTcB - Bazett's Correction Formula Cycle 3 Day 1 (6 H)QTcB - Bazett's Correction Formula Cycle 4 Day 1 (PREDOSE)QTcB - Bazett's Correction Formula Cycle 4 Day 1, 4 HQTcF Durations Fridericia's Correction Formula BaselineQTcF Durations Fridericia's Correction Formula Cycle 1 Day 1 (4 H)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 1 (6 H)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 2 (PREDOSE)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 2 (24 H)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 5 (PREDOSE)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 5 (4 H)QTcF Durations Fridericia's Correction Formula Cycle 1 Day 5 (6 H)QTcF Durations Fridericia's Correction Formula Cycle 2 Day 1 (PREDOSE)QTcF Durations Fridericia's Correction Formula Cycle 3 Day 1 (PREDOSE)QTcF Durations Fridericia's Correction Formula Cycle 3 Day 1 (4 H)QTcF Durations Fridericia's Correction Formula Cycle 3 Day 1 (6 H)QTcF Durations Fridericia's Correction Formula Cycle 4 Day 1 (PREDOSE)QTcF Durations Fridericia's Correction Formula Cycle 4 Day 1 (4 H)RR Duration BaselineRR Duration Cycle 1 Day 1 4 HRR Duration Cycle 1 Day 1 6 HRR Duration Cycle 1 Day 2 (PREDOSE)RR Duration Cycle 1 Day 2 (24 H)RR Duration Cycle 1 Day 5 (PREDOSE)RR Duration Cycle 1 Day 5 (4 H)RR Duration Cycle 1 Day 5 (6 H)RR Duration Cycle 2 Day 1 (PREDOSE)RR Duration Cycle 3 Day 1 (PREDOSE)RR Duration Cycle 3 Day 1 (4 H)RR Duration Cycle 3 Day 1 (6 H)RR Duration Cycle 4 Day 1 (PREDOSE)RR Duration Cycle 4 Day 1 (4 H)
Ruxolitinib-naïve Participants With Splenomegaly158.930.60-2.80-14.001.93-1.00-8.00-4.771.43-6.00-8.00-6.00-20.00-20.0090.87-1.47-1.27-4.001.070.13-1.08-0.920.64-4.00-4.00-4.00-2.00-2.00396.67-7.33-9.40-6.00-10.21-6.20-4.31-10.315.00-2.00-10.00-4.00-16.00-16.00427.673.808.00-15.003.71-11.07-62.00-2.92-4.543.93-16.006.008.00-62.00-62.00417.93-1.271.00-12.00-2.36-10.47-0.08-7.853.36-11.000.004.00-45.00-45.00861.47-38.87-65.6728.00-51.5025.87-26.23-21.467.1448.00-59.00-43.00182.00182.00

Change From Baseline in Heart Rate, as Measured by Electrocardiogram

"Single 12-lead ECGs was obtained using an ECG machine that automatically calculates the heart rate and measures PR, QRS, QT, and QTc intervals. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Day 1, hours 4 and 6, Day 2 pre-dose and 24 Hour, Day 5 pre-dose, 4 and 6 Hour), Cycle 2 (Day 1 pre-dose only), Cycle 3 (Day 1 pre-dose, 4 and 6 Hour), Cycle 4 (Day 1 pre-dose and 4 Hour)

InterventionBeats per Minute (Mean)
BaselineCycle 1, Day 1, 4 HourCycle 1, Day 1, 6 HourCycle 1 Day 2, pre-doseCycle 1 Day 2, 24 HourCycle 1 Day 5, pre-doseCycle 1 Day 5, 4 hourCycle 1 Day 5, 6 hourCycle 2, Day 1, pre-doseCycle 3 Day 1, pre-doseCycle 3 Day 1, 4 hourCycle 3 Day 1, 6 hourCycle 4 Day 1, pre-doseCycle 4 Day 1, 4 hour
Ruxolitinib-naïve Participants With Splenomegaly71.474.406.20-3.004.71-1.472.692.23-1.43-5.007.005.00-16.00-16.00

Change From Baseline in Heart Rate, as Measured by Electrocardiogram

"Single 12-lead ECGs was obtained using an ECG machine that automatically calculates the heart rate and measures PR, QRS, QT, and QTc intervals. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Day 1, hours 4 and 6, Day 2 pre-dose and 24 Hour, Day 5 pre-dose, 4 and 6 Hour), Cycle 2 (Day 1 pre-dose only), Cycle 3 (Day 1 pre-dose, 4 and 6 Hour), Cycle 4 (Day 1 pre-dose and 4 Hour)

,
InterventionBeats per Minute (Mean)
BaselineCycle 1, Day 1, 4 HourCycle 1, Day 1, 6 HourCycle 1 Day 2, 24 HourCycle 1 Day 5, pre-doseCycle 1 Day 5, 4 hourCycle 1 Day 5, 6 hourCycle 2, Day 1, pre-dose
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly77.00-21.00-11.00-5.00-4.005.006.00-12.00
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly73.172.503.00-0.67-5.60-2.20-2.80-6.17

Change From Baseline in Heart Rate, as Measured by Electrocardiogram

"Single 12-lead ECGs was obtained using an ECG machine that automatically calculates the heart rate and measures PR, QRS, QT, and QTc intervals. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Day 1, hours 4 and 6, Day 2 pre-dose and 24 Hour, Day 5 pre-dose, 4 and 6 Hour), Cycle 2 (Day 1 pre-dose only), Cycle 3 (Day 1 pre-dose, 4 and 6 Hour), Cycle 4 (Day 1 pre-dose and 4 Hour)

InterventionBeats per Minute (Mean)
BaselineCycle 1, Day 1, 4 HourCycle 1, Day 1, 6 HourCycle 1 Day 2, pre-doseCycle 1 Day 2, 24 HourCycle 1 Day 5, pre-doseCycle 1 Day 5, 4 hourCycle 1 Day 5, 6 hourCycle 2, Day 1, pre-dose
Ruxolitinib-naïve Participants Without Splenomegaly69.800.60-0.60-1.00-1.75-0.75-5.00-3.75-1.50

Change From Baseline in Oral Temperature

Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The study was pre-maturely terminated by the sponsor's decision, therefore did not reach the end of study. (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionDegrees Celsius (C) (Mean)
BaselineCycle 1 Day 15Cycle 1 Day 22Cycle 2 Day 1Cycle 2 Day 15Cycle 3 Day 1Cycle 3 Day 15Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12 Day 1Cycle 13 Day 1Cycle 14 Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17 Day 1Final Visit
Ruxolitinib-naïve Participants Without Splenomegaly36.400.420.040.050.180.13-0.03-0.17-0.10-0.10-0.20-0.050.050.30-0.200.30-0.70-0.10-0.30-0.800.600.26

Change From Baseline in Oral Temperature

Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The study was pre-maturely terminated by the sponsor's decision, therefore did not reach the end of study. (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionDegrees Celsius (C) (Mean)
BaselineCycle 1 Day 15Cycle 1 Day 22Cycle 2 Day 1Cycle 2 Day 15Cycle 3 Day 1Cycle 3 Day 15Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12 Day 1Cycle 13 Day 1Cycle 14 Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17 Day 1Cycle 18 Day 1Cycle 19 Day 1Cycle 20 Day 1Final Visit
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly36.470.180.150.170.230.170.100.060.100.120.200.20-0.070.10-0.10-0.200-0.30-0.40-0.100.00-0.20000.08

Change From Baseline in Oral Temperature

Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The study was pre-maturely terminated by the sponsor's decision, therefore did not reach the end of study. (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionDegrees Celsius (C) (Mean)
BaselineCycle 1 Day 15Cycle 1 Day 22Cycle 2 Day 1Cycle 2 Day 15Cycle 3 Day 1Cycle 3 Day 15Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12 Day 1Cycle 13 Day 1Cycle 14 Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17 Day 1Cycle 18 Day 1Cycle 19 Day 1Cycle 20 Day 1Cycle 21 Day 1Cycle 22 Day 1Final Visit
Ruxolitinib-naïve Participants With Splenomegaly36.500.130.03-0.050.04-0.09-0.04-0.050.05-0.030.01-0.010.03-0.08-0.07-0.100.000.00-0.100.030.030.07-0.150.000-0.50-0.08

Change From Baseline in Oral Temperature

Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value. The study was pre-maturely terminated by the sponsor's decision, therefore did not reach the end of study. (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionDegrees Celsius (C) (Mean)
BaselineCycle 1 Day 15Cycle 1 Day 22Cycle 2 Day 1Cycle 2 Day 15Cycle 3 Day 1Cycle 3 Day 15Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12 Day 1Cycle 13 Day 1Cycle 14 Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17 Day 1Cycle 18 Day 1Cycle 19 Day 1Cycle 20 Day 1Cycle 21 Day 1Cycle 22 Day 1Cycle 23 Day 1Final Visit
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly37.10-0.30-0.50-0.60-0.300.10-0.20-0.50-0.80-0.40-0.60-0.30-0.40-0.60-0.40-0.70-0.70-0.70-0.50-0.50-0.80-0.80-0.50-0.50-0.40-0.70-0.80-0.50

Change From Baseline in Pulse Rate

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionBeats per Minute (Mean)
BaselineCycle 1 Day 15Cycle 1 Day 22Cycle 2 Day 1Cycle 2 Day 15Cycle 3 Day 1Cycle 3 Day 15Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12, Day 1Cycle 13, Day 1Cycle 14, Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17, Day 1Final Visit
Ruxolitinib-naïve Participants Without Splenomegaly71.65.48.21.81.8-2.010.81.7-4.0-4.31.5-1.00.08.5-2.0-6.0-5.0-2.010.0-2.09.00.2

Change From Baseline in Pulse Rate

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionBeats per Minute (Mean)
BaselineCycle 1 Day 15Cycle 1 Day 22Cycle 2 Day 1Cycle 2 Day 15Cycle 3 Day 1Cycle 3 Day 15Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12, Day 1Cycle 13, Day 1Cycle 14, Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17, Day 1Cycle 18, Day 1Cycle 19, Day 1Cycle 20, Day 1Final Visit
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly76.7-0.7-5.8-9.3-5.8-4.2-2.2-6.6-3.6-1.6-1.00.50.32.32.5-1.0-1.0-4.0-2.0-2.01.0-3.06.04.00.2

Change From Baseline in Pulse Rate

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionBeats per Minute (Mean)
BaselineCycle 1 Day 15Cycle 1 Day 22Cycle 2 Day 1Cycle 2 Day 15Cycle 3 Day 1Cycle 3 Day 15Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12, Day 1Cycle 13, Day 1Cycle 14, Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17, Day 1Cycle 18, Day 1Cycle 19, Day 1Cycle 20, Day 1Cycle 21, Day 1Cycle 22, Day 1Final Visit
Ruxolitinib-naïve Participants With Splenomegaly74.74.53.9-1.10.1-2.8-1.21.00.60.41.4-3.9-5.4-0.3-2.5-0.2-2.8-3.8-0.8-1.3-12.7-4.3-9.0-5.02.0-8.0-1.7

Change From Baseline in Pulse Rate

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionBeats per Minute (Mean)
BaselineCycle 1 Day 15Cycle 1 Day 22Cycle 2 Day 1Cycle 2 Day 15Cycle 3 Day 1Cycle 3 Day 15Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12, Day 1Cycle 13, Day 1Cycle 14, Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17, Day 1Cycle 18, Day 1Cycle 19, Day 1Cycle 20, Day 1Cycle 21, Day 1Cycle 22, Day 1Cycle 23, Day 1Final Visit
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly68.07.08.012.09.09.021.024.028.014.021.011.0020.037.021.016.012.014.016.022.016.015.05.017.01419.016.0

Change From Baseline in Respiratory Rate

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionBreaths per Minute (Mean)
BaselineCycle 1 Day 15Cycle 1 Day 22Cycle 2 Day 1Cycle 2 Day 15Cycle 3 Day 1Cycle 3 Day 15Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12 Day 1Cycle 13 Day 1Cycle 14 Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17 Day 1Final visit
Ruxolitinib-naïve Participants Without Splenomegaly17.4-0.20.2-0.30.0-1.50.0-3.3-0.3-0.50-0.50.5-0.51.01.01.01.01.01.01.0-1.2

Change From Baseline in Respiratory Rate

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionBreaths per Minute (Mean)
BaselineCycle 1 Day 15Cycle 1 Day 22Cycle 2 Day 1Cycle 2 Day 15Cycle 3 Day 1Cycle 3 Day 15Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12 Day 1Cycle 13 Day 1Cycle 14 Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17 Day 1Cycle 18 Day 1Cycle 19 Day 1Cycle 20 Day 1Final visit
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly18.2-0.2-0.5-0.4-1.2-1.8-1.0-1.01.0-0.6-2.3-0.8-1.7-0.3-0.51.5-1.01.0-1.0-2.0-2.0-1.0-3.0-3.0-1.8

Change From Baseline in Respiratory Rate

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionBreaths per Minute (Mean)
BaselineCycle 1 Day 15Cycle 1 Day 22Cycle 2 Day 1Cycle 2 Day 15Cycle 3 Day 1Cycle 3 Day 15Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12 Day 1Cycle 13 Day 1Cycle 14 Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17 Day 1Cycle 18 Day 1Cycle 19 Day 1Cycle 20 Day 1Cycle 21 Day 1Cycle 22 Day 1Final visit
Ruxolitinib-naïve Participants With Splenomegaly17.4-0.3-0.5-0.3-0.2-0.7-0.7-0.3-0.4-0.5-0.20.10.4-0.2-0.8-0.4-1.40.80.0-0.31.31.31.52.02.00.0-0.3

Change From Baseline in Respiratory Rate

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionBreaths per Minute (Mean)
BaselineCycle 1 Day 15Cycle 1 Day 22Cycle 2 Day 1Cycle 2 Day 15Cycle 3 Day 1Cycle 3 Day 15Cycle 4 Day 1Cycle 5 Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12 Day 1Cycle 13 Day 1Cycle 14 Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17 Day 1Cycle 18 Day 1Cycle 19 Day 1Cycle 20 Day 1Cycle 21 Day 1Cycle 22 Day 1Cycle 23 Day 1Final visit
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly16.00002.0002.002.02.00-2.002.002.000002.02.02.00000

Change From Baseline in Systolic Blood Pressure

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionMillimeters of mercury (mmHg) (Mean)
BaselineCycle 1, Day 15Cycle 1, Days 22Cycle 2, Day 1Cycle 2 , Day 15Cycle 3, Day 1Cycle 3, Day 15Cycle 4, Day 1Cycle 5, Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12 Day 1Cycle 13 Day 1Cycle 14 Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17 Day 1Final Visit
Ruxolitinib-naïve Participants Without Splenomegaly132.03.6-5.62.8-2.0-3.0-3.5-7.3-2.52.5-4.05.03.0-3.014.0-8.0-8.018.014.08.024.07.6

Change From Baseline in Systolic Blood Pressure

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionMillimeters of mercury (mmHg) (Mean)
BaselineCycle 1, Day 15Cycle 1, Days 22Cycle 2, Day 1Cycle 2 , Day 15Cycle 3, Day 1Cycle 3, Day 15Cycle 4, Day 1Cycle 5, Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12 Day 1Cycle 13 Day 1Cycle 14 Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17 Day 1Cycle 18 Day 1Cycle 19 Day 1Cycle 20 Day 1Final Visit
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly122.33.84.8-4.3-7.27.3-3.89.83.38.610.014.0-4.311.011.06.58.03.04.09.07.09.09.09.012.8

Change From Baseline in Systolic Blood Pressure

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionMillimeters of mercury (mmHg) (Mean)
BaselineCycle 1, Day 15Cycle 1, Days 22Cycle 2, Day 1Cycle 2 , Day 15Cycle 3, Day 1Cycle 3, Day 15Cycle 4, Day 1Cycle 5, Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12 Day 1Cycle 13 Day 1Cycle 14 Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17 Day 1Cycle 18 Day 1Cycle 19 Day 1Cycle 20 Day 1Cycle 21 Day 1Cycle 22 Day 1Final Visit
Ruxolitinib-naïve Participants With Splenomegaly129.34.43.33.05.25.51.22.24.211.24.78.812.611.88.510.813.44.413.67.59.35.72.511.5-18.024.01.3

Change From Baseline in Systolic Blood Pressure

"Vital signs were measured prior to the infusion while the participant was in a seated position. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline, Cycle 1 (Days 15 and 22), Cycle 2 and 3 (Days 1 and 15), Each Cycle 4-23 (Day 1 only) and Final Visit

InterventionMillimeters of mercury (mmHg) (Mean)
BaselineCycle 1, Day 15Cycle 1, Days 22Cycle 2, Day 1Cycle 2 , Day 15Cycle 3, Day 1Cycle 3, Day 15Cycle 4, Day 1Cycle 5, Day 1Cycle 6 Day 1Cycle 7 Day 1Cycle 8 Day 1Cycle 9 Day 1Cycle 10 Day 1Cycle 11 Day 1Cycle 12 Day 1Cycle 13 Day 1Cycle 14 Day 1Cycle 15 Day 1Cycle 16 Day 1Cycle 17 Day 1Cycle 18 Day 1Cycle 19 Day 1Cycle 20 Day 1Cycle 21 Day 1Cycle 22 Day 1Cycle 23 Day 1Final Visit
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly106.05.0-5.010.030.006.013.016.08.029.09.026.030.034.06.014.014.05.04.06.013.013.013.07.06.011.019.0

Duration of Complete Hematologic Remission, With a Durable Responder Defined as a Participant in Remission at Week 32 and Cycle 11 Day 28

Complete hematologic remission requires all of the following: Hct control without phlebotomy between Week 32 (Cycle 8 Day 28) and Cycle 11 Day 28; WBC count ≤10 × 10^9/L at Cycle 11 Day 28; and Platelet count ≤400 × 10^9/L at Week 32. Hct control is defined as protocol-specified ineligibility for phlebotomy. Eligibility for phlebotomy is defined as a Hct level ≥45% that was ≥3% higher than baseline level or a Hct level of >48% (NCT03287245)
Timeframe: Week 32 (Cycle 8 Day 28), Cycle 11 Day 28

,,,
InterventionParticipants (Number)
Cycle 11, Day 28Week 32
Ruxolitinib-naïve Participants With Splenomegaly23
Ruxolitinib-naïve Participants Without Splenomegaly12
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly03
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly00

Eastern Cooperative Oncology Group (ECOG) Performance Status Over Time

"The ECOG performance status is a scale used to quantify cancer patients' general well-being and activities of daily life. The scale ranges from 0 to 5, with 0 denoting perfect health and 5 indicating death. The 6 categories are 0=Asymptomatic (Fully active, able to carry on all pre-disease activities without restriction), 1=Symptomatic but completely ambulatory, 2=Symptomatic, < 50% in bed during the day (Ambulatory and capable of all self-care but unable to carry out any work activities. Up and about more than 50% of waking hours), 3=Symptomatic, > 50% in bed, but not bedbound (Capable of only limited self-care, confined to bed or chair 50% or more of waking hours), 4=Bedbound (Completely disabled. Cannot carry on any self-care. Totally confined to bed or chair), 5=Death.~Only baseline data were collectable. The study was pre-maturely terminated by the sponsor's decision, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline

,,,
InterventionPercentage of Participant (Number)
Baseline 0Baseline 1
Ruxolitinib-naïve Participants With Splenomegaly66.733.3
Ruxolitinib-naïve Participants Without Splenomegaly60.040.0
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly50.050.0
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly100.00

Frequency Count of Participant Responses to the Patient Global Impression of Change (PGIC) Question Over Time

"The PGIC is a one-item measure used to assess perceived treatment benefit. Participants were asked Since the start of the treatment you've received in this study, your polycythemia vera (PV) symptoms are: 'very much improved', 'much improved', 'minimally improved', 'no change', 'minimally worse', 'much worse', and 'very much worse'.~The study was pre-maturely terminated by the sponsor's decision, therefore did not reach the end of study." (NCT03287245)
Timeframe: Cycle 2 Day 1, Cycle 3 Day 28, Cycle 5 Day 28, End of Cycle 8 (Week 32), and every 3 cycles thereafter (1 cycle is 28 days) until end of study (up to 2 years)

,
InterventionCount of Participants (Number)
Cycle 2, Day 1 Very Much ImprovedCycle 2, Day 1 Much ImprovedCycle 2, Day 1 Minimally ImprovedCycle 2, Day 1 No ChangeCycle 2, Day 1 Minimally WorseCycle 2, Day 1 Much WorseCycle 2, Day 1 Very Much WorseCycle 2, Day 1 Not AssessedCycle 3 Day 28 Very Much ImprovedCycle 3 Day 28 Much ImprovedCycle 3 Day 28 Minimally ImprovedCycle 3 Day 28 No ChangeCycle 3 Day 28 Minimally WorseCycle 3 Day 28 Much WorseCycle 3 Day 28 Very Much WorseCycle 3 Day 28 Not AssessedCycle 5 Day 28 Very Much ImprovedCycle 5 Day 28 Much ImprovedCycle 5 Day 28 Minimally ImprovedCycle 5 Day 28 No ChangeCycle 5 Day 28 Minimally WorseCycle 5 Day 28 Much WorseCycle 5 Day 28 Very Much WorseCycle 5 Day 28 Not AssessedWeek 32 Very Much ImprovedWeek 32 Much ImprovedWeek 32 Minimally ImprovedWeek 32 No ChangeWeek 32 Minimally WorseWeek 32 Much WorseWeek 32 Very Much WorseWeek 32 Not AssessedCycle 11 Day 28 Very Much ImprovedCycle 11 Day 28 Much ImprovedCycle 11 Day 28 Minimally ImprovedCycle 11 Day 28 No ChangeCycle 11 Day 28 Minimally WorseCycle 11 Day 28 Much WorseCycle 11 Day 28 Very Much WorseCycle 11 Day 28 Not AssessedCycle 14 Day 28 Very Much ImprovedCycle 14 Day 28 Much ImprovedCycle 14 Day 28 Minimally ImprovedCycle 14 Day 28 No changeCycle 14 Day 28 Minimally WorseCycle 14 Day 28 Much WorseCycle 14 Day 28 Very Much WorseCycle 14 Day 28 Not AssessedCycle 17 Day 28 Very Much ImprovedCycle 17 Day 28 Much ImprovedCycle 17 Day 28 Minimally ImprovedCycle 17 Day 28 No ChangeCycle 17 Day 28 Minimally WorseCycle 17 Day 28 Much WorseCycle 17 Day 28 Very Much WorseCycle 17 Day 28 Not AssessedCycle 20 Day 28 Very Much ImprovedCycle 20 Day 28 Much ImprovedCycle 20 Day 28 Minimally ImprovedCycle 20 Day 28 No ChangeCycle 20 Day 28 Minimally WorseCycle 20 Day 28 Much WorseCycle 20 Day 28 Very Much WorseCycle 20 Day 28 Not AssessedFinal Visit Very Much ImprovedFinal Visit Much ImprovedFinal Visit Minimally ImprovedFinal Visit No ChangeFinal Visit Minimally WorseFinal Visit Much WorseFinal Visit Very Much WorseFinal Visit Not Assessed
Ruxolitinib-Naïve Participants045610004463000026210004333110001300100022011000111100000010000033261002
Ruxolitinib-Resistant or Intolerant Participants013110011130000111400000013100000200000002000000020000000000000013100000

Number of Participants With a Durable Response, in Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly at Baseline With Durable Response Lasting at Least 12 Weeks From Week 32

"The number of participants with a durable response lasting at least 12 weeks from Week 32 is analyzed by the type of response achieved: Hct control, complete hematologic response, ELN 2009 response criteria, and composite response, if applicable.~Reported result data start from Cycle 11 Day 28 which is 12 weeks after Week 32 (Cycle 8 Day 28). There was one timepoint for which the participants qualify.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: From Week 32 (Cycle 8 Day 28) and at least 12 weeks after until end of study (up to 2 years)

,
InterventionParticipants (Number)
HCT ControlComposite ResponseELN ResponseComplete Hematologic Response
Ruxolitinib-Naïve Participants With Splenomegaly3042
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly3032

Number of Participants With a Durable Response, in Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly at Baseline With Durable Response Lasting at Least 12 Weeks From Week 32

"The number of participants with a durable response lasting at least 12 weeks from Week 32 is analyzed by the type of response achieved: Hct control, complete hematologic response, ELN 2009 response criteria, and composite response, if applicable.~Reported result data start from Cycle 11 Day 28 which is 12 weeks after Week 32 (Cycle 8 Day 28). There was one timepoint for which the participants qualify.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: From Week 32 (Cycle 8 Day 28) and at least 12 weeks after until end of study (up to 2 years)

,
InterventionParticipants (Number)
HCT ControlComposite ResponseELN ResponseComplete Hematologic Response
Ruxolitinib-Naïve Participants Without Splenomegaly2022
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly0000

Number of Participants With a Duration Response, in All Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants (Irrespective of Spleen Size) With Durable Response Lasting at Least 12 Weeks From Week 32

"The number of participants with a durable response lasting at least 12 weeks from Week 32 is analyzed by the type of response achieved: Hct control, complete hematologic response, ELN 2009 response criteria, and composite response, if applicable.~Reported result data start from Cycle 11 Day 28 which is 12 weeks after Week 32 (Cycle 8 Day 28). There was one timepoint for which the participants qualify.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: From Week 32 (Cycle 8 Day 28) and at least 12 weeks after until end of study (up to 2 years)

,
InterventionParticipants (Number)
HCT ControlComposite ResponseELN ResponseComplete Hematologic Response
All Ruxolitinib-Naïve Participants5064
All Ruxolitinib-Resistant or Intolerant Participants3032

Percentage of All Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants (Irrespective of Spleen Size) by Response Per Modified ELN Criteria

"Complete response (CR) includes all of the following: Hct <45% without phlebotomy; Platelet count ≤400 × 10^9/L; WBC count ≤10 × 10^9/L; Normal spleen size on imaging; and No disease-related symptoms. Partial response (PR): in participants who do not fulfill the criteria for CR: Hct <45% without phlebotomy or response in 3 or more of the other criteria. No response (NR): any response that does not satisfy partial response. Progressive disease (PD): increased bone marrow fibrosis from baseline, and/or transformation to myelofibrosis (MF), myelodysplastic syndrome (MDS) or acute leukemia.~All responders of each category grouped per timepoint include all categories mentioned above. There were no PD responses at any timepoint.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the end of study." (NCT03287245)
Timeframe: Baseline, Day 28 of Cycles 3, 5, 8, 11, 12, 14, 17, 20 and Final visit (28 days after post-last dose)

InterventionPercentage of Participants (Number)
Baseline Complete ResponseBaseline Partial ResponseBaseline Progressive DiseaseBaseline No ResponseCycle 3, Day 28 Complete ResponseCycle 3, Day 28 Partial ResponseCycle 3, Day 28 Progressive DiseaseCycle 3, Day 28 No ResponseCycle 5, Day 28 Complete ResponseCycle 5, Day 28 Partial ResponseCycle 5, Day 28 Progressive DiseaseCycle 5, Day 28 No ResponseCycle 8, Day 28 (Week 32) Complete ResponseCycle 8, Day 28 (Week 32) Partial ResponseCycle 8, Day 28 (Week 32) Progressive DiseaseCycle 8, Day 28 (Week 32) No ResponseCycle 11 Day 28 Complete ResponseCycle 11 Day 28 Partial ResponseCycle 11 Day 28 Progressive DiseaseCycle 11 Day 28 No ResponseCycle 14, Day 28 Complete ResponseCycle 14, Day 28 Partial ResponseCycle 14, Day 28 Progressive DiseaseCycle 14, Day 28 No ResponseCycle 17, Day 28 Complete ResponseCycle 17, Day 28 Partial ResponseCycle 17, Day 28 Progressive DiseaseCycle 17, Day 28 No ResponseCycle 20, Day 28 Complete ResponseCycle 20, Day 28 Partial ResponseCycle 20, Day 28 Progressive DiseaseCycle 20, Day 28 No ResponseFinal Visit Complete ResponseFinal Visit Partial ResponseFinal Visit Progressive DiseaseFinal Visit No Response
All Ruxolitinib-Naïve Participants000010.568.4021.112.568.8018.818.254.5027.316.766.7016.716.766.7016.725.025.0050.001000014.321.4064.3

Percentage of All Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants (Irrespective of Spleen Size) by Response Per Modified ELN Criteria

"Complete response (CR) includes all of the following: Hct <45% without phlebotomy; Platelet count ≤400 × 10^9/L; WBC count ≤10 × 10^9/L; Normal spleen size on imaging; and No disease-related symptoms. Partial response (PR): in participants who do not fulfill the criteria for CR: Hct <45% without phlebotomy or response in 3 or more of the other criteria. No response (NR): any response that does not satisfy partial response. Progressive disease (PD): increased bone marrow fibrosis from baseline, and/or transformation to myelofibrosis (MF), myelodysplastic syndrome (MDS) or acute leukemia.~All responders of each category grouped per timepoint include all categories mentioned above. There were no PD responses at any timepoint.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the end of study." (NCT03287245)
Timeframe: Baseline, Day 28 of Cycles 3, 5, 8, 11, 12, 14, 17, 20 and Final visit (28 days after post-last dose)

InterventionPercentage of Participants (Number)
Baseline Complete ResponseBaseline Partial ResponseBaseline Progressive DiseaseBaseline No ResponseCycle 3, Day 28 Complete ResponseCycle 3, Day 28 Partial ResponseCycle 3, Day 28 Progressive DiseaseCycle 3, Day 28 No ResponseCycle 5, Day 28 Complete ResponseCycle 5, Day 28 Partial ResponseCycle 5, Day 28 Progressive DiseaseCycle 5, Day 28 No ResponseCycle 8, Day 28 (Week 32) Complete ResponseCycle 8, Day 28 (Week 32) Partial ResponseCycle 8, Day 28 (Week 32) Progressive DiseaseCycle 8, Day 28 (Week 32) No ResponseCycle 11 Day 28 Complete ResponseCycle 11 Day 28 Partial ResponseCycle 11 Day 28 Progressive DiseaseCycle 11 Day 28 No ResponseCycle 12 Day 28 Complete ResponseCycle 12 Day 28 Partial ResponseCycle 12 Day 28 Progressive DiseaseCycle 12 Day 28 No ResponseCycle 14, Day 28 Complete ResponseCycle 14, Day 28 Partial ResponseCycle 14, Day 28 Progressive DiseaseCycle 14, Day 28 No ResponseCycle 17, Day 28 Complete ResponseCycle 17, Day 28 Partial ResponseCycle 17, Day 28 Progressive DiseaseCycle 17, Day 28 No ResponseCycle 20, Day 28 Complete ResponseCycle 20, Day 28 Partial ResponseCycle 20, Day 28 Progressive DiseaseCycle 20, Day 28 No ResponseFinal Visit Complete ResponseFinal Visit Partial ResponseFinal Visit Progressive DiseaseFinal Visit No Response
All Ruxolitinib-Resistant or Intolerant Participants000028.642.9028.6066.7033.320.040.0040.0050.0050.0010000050050.0000100000100025.0075.0

Percentage of All Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants (Irrespective of Spleen Size) With Durable Response Lasting at Least 12 Weeks From Week 32

"The percentage of participants with a durable response lasting at least 12 weeks from Week 32 is analyzed by the type of response achieved: Hct control, complete hematologic response, ELN 2009 response criteria, and composite response, if applicable.~Reported result data start from Cycle 11 Day 28 which is 12 weeks after Week 32 (Cycle 8 Day 28). There was one timepoint for which the participants qualify. The study was pre-maturely terminated by the sponsor decision, therefore did not reach the end of study." (NCT03287245)
Timeframe: From Week 32 (Cycle 8 Day 28) and at least 12 Weeks after until end of study (up to 2 years)

,
InterventionPercentage of Participants (Number)
After 12 Weeks from Week 32 HCT ControlAfter 12 Weeks from Week 32 Composite ResponseAfter 12 Weeks from Week 32 ELN ResponseAfter 12 Weeks from Week 32 Complete Hematologic Response
All Ruxolitinib-Naïve Participants55.606044.4
All Ruxolitinib-Resistant or Intolerant Participants7506050

Percentage of Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly at Baseline by Response Per Modified European Leukemia Net (ELN) Criteria

"Complete response (CR) includes all of the following: Hct <45% without phlebotomy; Platelet count ≤400 × 10^9/L; WBC count ≤10 × 10^9/L; Normal spleen size on imaging; and No disease-related symptoms. Partial response (PR): in participants who do not fulfill the criteria for CR: Hct <45% without phlebotomy or response in 3 or more of the other criteria. No response (NR): any response that does not satisfy partial response. Progressive disease (PD): increased bone marrow fibrosis from baseline, and/or transformation to myelofibrosis (MF), myelodysplastic syndrome (MDS) or acute leukemia.~All responders of each category grouped per timepoint include all categories mentioned above. There were no PD responses at any timepoint.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the end of study." (NCT03287245)
Timeframe: Baseline, Day 28 of Cycles 3, 5, 8, 11, 12, 14, 17, 20 and Final visit (28 days after post-last dose)

InterventionPercentage of Participants (Number)
Baseline Complete ResponseBaseline Partial ResponseBaseline Progressive DiseaseBaseline No ResponseCycle 3, Day 28 Complete ResponseCycle 3, Day 28 Partial ResponseCycle 3, Day 28 Progressive DiseaseCycle 3, Day 28 No ResponseCycle 5, Day 28 Complete ResponseCycle 5, Day 28 Partial ResponseCycle 5, Day 28 Progressive DiseaseCycle 5, Day 28 No ResponseCycle 8, Day 28 (Week 32) Complete ResponseCycle 8, Day 28 (Week 32) Partial ResponseCycle 8, Day 28 (Week 32) Progressive DiseaseCycle 8, Day 28 (Week 32) No ResponseCycle 11, Day 28 Complete ResponseCycle 11, Day 28 Partial ResponseCycle 11, Day 28 Progressive DiseaseCycle 11, Day 28 No ResponseCycle 12, Day 28 Complete ResponseCycle 12, Day 28 Partial ResponseCycle 12, Day 28 Progressive DiseaseCycle 12, Day 28 No ResponseCycle 14, Day 28 Complete ResponseCycle 14, Day 28 Partial ResponseCycle 14, Day 28 Progressive DiseaseCycle 14, Day 28 No ResponseCycle 17, Day 28 Complete ResponseCycle 17, Day 28 Partial ResponseCycle 17, Day 28 Progressive DiseaseCycle 17, Day 28 No ResponseFinal Visit (28 Days post-last dose) Complete ResponseFinal Visit (28 Days post-last dose) Partial ResponseFinal Visit (28 Days post-last dose) Progressive DiseaseFinal Visit (28 Days post-last dose) No Response
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly000033.350.0016.7080.0020.025.050.0025.00100.0000000010000000100033.3066.7

Percentage of Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly at Baseline by Response Per Modified European Leukemia Net (ELN) Criteria

"Complete response (CR) includes all of the following: Hct <45% without phlebotomy; Platelet count ≤400 × 10^9/L; WBC count ≤10 × 10^9/L; Normal spleen size on imaging; and No disease-related symptoms. Partial response (PR): in participants who do not fulfill the criteria for CR: Hct <45% without phlebotomy or response in 3 or more of the other criteria. No response (NR): any response that does not satisfy partial response. Progressive disease (PD): increased bone marrow fibrosis from baseline, and/or transformation to myelofibrosis (MF), myelodysplastic syndrome (MDS) or acute leukemia.~All responders of each category grouped per timepoint include all categories mentioned above. There were no PD responses at any timepoint.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the end of study." (NCT03287245)
Timeframe: Baseline, Day 28 of Cycles 3, 5, 8, 11, 12, 14, 17, 20 and Final visit (28 days after post-last dose)

InterventionPercentage of Participants (Number)
Baseline Complete ResponseBaseline Partial ResponseBaseline Progressive DiseaseBaseline No ResponseCycle 3, Day 28 Complete ResponseCycle 3, Day 28 Partial ResponseCycle 3, Day 28 Progressive DiseaseCycle 3, Day 28 No ResponseCycle 5, Day 28 Complete ResponseCycle 5, Day 28 Partial ResponseCycle 5, Day 28 Progressive DiseaseCycle 5, Day 28 No ResponseCycle 8, Day 28 (Week 32) Complete ResponseCycle 8, Day 28 (Week 32) Partial ResponseCycle 8, Day 28 (Week 32) Progressive DiseaseCycle 8, Day 28 (Week 32) No ResponseCycle 11, Day 28 Complete ResponseCycle 11, Day 28 Partial ResponseCycle 11, Day 28 Progressive DiseaseCycle 11, Day 28 No ResponseCycle 14, Day 28 Complete ResponseCycle 14, Day 28 Partial ResponseCycle 14, Day 28 Progressive DiseaseCycle 14, Day 28 No ResponseCycle 17, Day 28 Complete ResponseCycle 17, Day 28 Partial ResponseCycle 17, Day 28 Progressive DiseaseCycle 17, Day 28 No ResponseCycle 20, Day 28 Complete ResponseCycle 20, Day 28 Partial ResponseCycle 20, Day 28 Progressive DiseaseCycle 20, Day 28 No ResponseFinal Visit (28 Days post-last dose) Complete ResponseFinal Visit (28 Days post-last dose) Partial ResponseFinal Visit (28 Days post-last dose) Progressive DiseaseFinal Visit (28 Days post-last dose) No Response
Ruxolitinib-naïve Participants With Splenomegaly0000073.3026.7076.9023.1066.7033.3080.0020.0080020033.3066.7010000020.0080

Percentage of Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly at Baseline With Durable Response Lasting at Least 12 Weeks From Week 32

"The percentage of participants with a durable response lasting at least 12 weeks from Week 32 are analyzed by the type of response achieved: Hct control, complete hematologic response, ELN 2009 response criteria, and composite response, if applicable.~Reported result data start from Cycle 11 Day 28 which is 12 weeks after Week 32 (Cycle 8 Day 28). There was one timepoint for which the participants qualify. The study was pre-maturely terminated by the sponsor decision, therefore did not reach the end of study." (NCT03287245)
Timeframe: From Week 32 (Cycle 8 Day 28) and at least 12 Weeks after until end of study (up to 2 years)

,
InterventionPercentage of Participants (Number)
HCT ControlComposite ResponseELN ResponseComplete Hematologic Response
Ruxolitinib-Naïve Participants With Splenomegaly42.905028.6
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly10007566.7

Percentage of Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly at Baseline by Response Per Modified ELN Criteria

"Complete response (CR) includes all of the following: Hct <45% without phlebotomy; Platelet count ≤400 × 10^9/L; WBC count ≤10 × 10^9/L; Normal spleen size on imaging; and No disease-related symptoms. Partial response (PR): in participants who do not fulfill the criteria for CR: Hct <45% without phlebotomy or response in 3 or more of the other criteria. No response (NR): any response that does not satisfy partial response. Progressive disease (PD): increased bone marrow fibrosis from baseline, and/or transformation to myelofibrosis (MF), myelodysplastic syndrome (MDS) or acute leukemia.~All responders of each category grouped per timepoint include all categories mentioned above. There were no PD responses at any timepoint.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the end of study." (NCT03287245)
Timeframe: Baseline, Day 28 of Cycles 3, 5, 8, 11, 12, 14, 17, 20 and Final visit (28 days after post-last dose)

InterventionPercentage of Participants (Number)
Baseline Complete ResponseBaseline Partial ResponseBaseline Progressive DiseaseBaseline No ResponseCycle 3, Day 28 Complete ResponseCycle 3, Day 28 Partial ResponseCycle 3, Day 28 Progressive DiseaseCycle 3, Day 28 No ResponseCycle 5 Day 28 Complete ResponseCycle 5 Day 28 Partial ResponseCycle 5 Day 28 Progressive DiseaseCycle 5 Day 28 No ResponseCycle 8, Day 28 (Week 32) Complete ResponseCycle 8, Day 28 (Week 32) Partial ResponseCycle 8, Day 28 (Week 32) Progressive DiseaseCycle 8, Day 28 (Week 32) No ResponseCycle 11, day 28 Complete ResponseCycle 11, day 28 Partial ResponseCycle 11, day 28 Progressive DiseaseCycle 11, day 28 No ResponseCycle 14, Day 28 Complete ResponseCycle 14, Day 28 Partial ResponseCycle 14, Day 28 Progressive DiseaseCycle 14, Day 28 No ResponseCycle 17, Day 28 Complete ResponseCycle 17, Day 28 Partial ResponseCycle 17, Day 28 Progressive DiseaseCycle 17, Day 28 No ResponseFinal (28 Days post-last dose) Complete ResponseFinal (28 Days post-last dose) Partial ResponseFinal (28 Days post-last dose) Progressive DiseaseFinal (28 Days post-last dose) No Response
Ruxolitinib-naïve Participants Without Splenomegaly000050500066.733.30010000010000010000010000050.025.00.025.0

Percentage of Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly at Baseline by Response Per Modified ELN Criteria

"Complete response (CR) includes all of the following: Hct <45% without phlebotomy; Platelet count ≤400 × 10^9/L; WBC count ≤10 × 10^9/L; Normal spleen size on imaging; and No disease-related symptoms. Partial response (PR): in participants who do not fulfill the criteria for CR: Hct <45% without phlebotomy or response in 3 or more of the other criteria. No response (NR): any response that does not satisfy partial response. Progressive disease (PD): increased bone marrow fibrosis from baseline, and/or transformation to myelofibrosis (MF), myelodysplastic syndrome (MDS) or acute leukemia.~All responders of each category grouped per timepoint include all categories mentioned above. There were no PD responses at any timepoint.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the end of study." (NCT03287245)
Timeframe: Baseline, Day 28 of Cycles 3, 5, 8, 11, 12, 14, 17, 20 and Final visit (28 days after post-last dose)

InterventionPercentage of Participants (Number)
Baseline Complete ResponseBaseline Partial ResponseBaseline Progressive DiseaseBaseline No ResponseCycle 3, Day 28 Complete ResponseCycle 3, Day 28 Partial ResponseCycle 3, Day 28 Progressive DiseaseCycle 3, Day 28 No ResponseCycle 5 Day 28 Complete ResponseCycle 5 Day 28 Partial ResponseCycle 5 Day 28 Progressive DiseaseCycle 5 Day 28 No ResponseCycle 8, Day 28 (Week 32) Complete ResponseCycle 8, Day 28 (Week 32) Partial ResponseCycle 8, Day 28 (Week 32) Progressive DiseaseCycle 8, Day 28 (Week 32) No ResponseCycle 11, day 28 Complete ResponseCycle 11, day 28 Partial ResponseCycle 11, day 28 Progressive DiseaseCycle 11, day 28 No ResponseCycle 14, Day 28 Complete ResponseCycle 14, Day 28 Partial ResponseCycle 14, Day 28 Progressive DiseaseCycle 14, Day 28 No ResponseCycle 17, Day 28 Complete ResponseCycle 17, Day 28 Partial ResponseCycle 17, Day 28 Progressive DiseaseCycle 17, Day 28 No ResponseCycle 20, Day 28 Complete ResponseCycle 20, Day 28 Partial ResponseCycle 20, Day 28 Progressive DiseaseCycle 20, Day 28 No ResponseFinal (28 Days post-last dose) Complete ResponseFinal (28 Days post-last dose) Partial ResponseFinal (28 Days post-last dose) Progressive DiseaseFinal (28 Days post-last dose) No Response
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly0000000100000100000100000100000100000100000100000100

Percentage of Ruxolitinib-Naïve and Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly at Baseline With Durable Response Lasting at Least 12 Weeks From Week 32

"The percentage of participants with a durable response lasting at least 12 weeks from Week 32 is analyzed by the type of response achieved: Hct control, complete hematologic response, ELN 2009 response criteria, and composite response, if applicable.~Reported result data start from Cycle 11 Day 28 which is 12 weeks after Week 32 (Cycle 8 Day 28). There was one timepoint for which the participants qualify. The study was pre-maturely terminated by the sponsor decision, therefore did not reach the end of study." (NCT03287245)
Timeframe: From Week 32 (Cycle 8 Day 28) and at least 12 Weeks after until end of study (up to 2 years)

,
InterventionPercentage of Participants (Number)
HCT ControlComposite ResponseELN ResponseComplete Hematologic Response
Ruxolitinib-Naïve Participants Without Splenomegaly1000100100
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly0000

Total Number of Participants With Adverse Events by Severity, Graded According to NCI CTCAE v4.0

"An adverse event is any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a study drug, whether or not considered related to the study drug. The adverse event severity grading scale for the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 (NCI CTCAE v4.0) will be used for assessing adverse event severity.~During the final analyses, the focus was on the Adverse Events of severity grades >/=3 as shown below. The extensive listings of all grade AEs are available at request.~The study was pre-maturely terminated by the sponsor's decision due to non-transformative efficacy and poor long-term tolerability of the dosing schedule as well as due to chronic gastrointestinal toxicity, therefore did not reach the planned end of study." (NCT03287245)
Timeframe: Baseline to end of study (up to 2 years)

,,,
InterventionParticipants (Number)
BaselineGrade 3-5 AE
Ruxolitinib-Naïve Participants With Splenomegaly05
Ruxolitinib-Naïve Participants Without Splenomegaly02
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly03
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly00

Reviews

1 review available for 4-aminobenzoic acid and Erythremia

ArticleYear
Novel therapeutic approaches in polycythemia vera.
    Clinical advances in hematology & oncology : H&O, 2018, Volume: 16, Issue:11

    Topics: Clinical Trials, Phase II as Topic; Clinical Trials, Phase III as Topic; Half-Life; Humans; Hydroxyu

2018

Trials

1 trial available for 4-aminobenzoic acid and Erythremia

ArticleYear
The MDM2 antagonist idasanutlin in patients with polycythemia vera: results from a single-arm phase 2 study.
    Blood advances, 2022, 02-22, Volume: 6, Issue:4

    Topics: Humans; Hydroxyurea; Nausea; para-Aminobenzoates; Polycythemia Vera; Proto-Oncogene Proteins c-mdm2;

2022

Other Studies

2 other studies available for 4-aminobenzoic acid and Erythremia

ArticleYear
Drug development challenges in polycythemia vera.
    Blood, 2019, 08-08, Volume: 134, Issue:6

    Topics: Drug Development; Humans; para-Aminobenzoates; Polycythemia Vera; Pyrrolidines

2019
Transient expansion of TP53 mutated clones in polycythemia vera patients treated with idasanutlin.
    Blood advances, 2020, 11-24, Volume: 4, Issue:22

    Topics: Clone Cells; Humans; para-Aminobenzoates; Polycythemia Vera; Proto-Oncogene Proteins c-mdm2; Pyrroli

2020