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.
Excerpt | Relevance | Reference |
---|---|---|
" 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." | 3.11 | The 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) |
Timeframe | Studies, this research(%) | All Research% |
---|---|---|
pre-1990 | 0 (0.00) | 18.7374 |
1990's | 0 (0.00) | 18.2507 |
2000's | 0 (0.00) | 29.6817 |
2010's | 2 (50.00) | 24.3611 |
2020's | 2 (50.00) | 2.80 |
Authors | Studies |
---|---|
Mascarenhas, J | 2 |
Passamonti, F | 1 |
Burbury, K | 1 |
El-Galaly, TC | 1 |
Gerds, A | 1 |
Gupta, V | 1 |
Higgins, B | 1 |
Wonde, K | 1 |
Jamois, C | 1 |
Kovic, B | 1 |
Huw, LY | 1 |
Katakam, S | 1 |
Maffioli, M | 1 |
Mesa, R | 1 |
Palmer, J | 1 |
Bellini, M | 1 |
Ross, DM | 1 |
Vannucchi, AM | 1 |
Yacoub, A | 1 |
Ritchie, EK | 1 |
Marcellino, BK | 1 |
Farnoud, N | 1 |
Cassinat, B | 1 |
Lu, M | 1 |
Verger, E | 1 |
McGovern, E | 1 |
Patel, M | 1 |
Medina-Martinez, J | 1 |
Levine, MF | 1 |
Arango Ossa, JE | 1 |
Zhou, Y | 1 |
Kosiorek, H | 1 |
Mehrotra, M | 1 |
Houldsworth, J | 1 |
Dueck, A | 1 |
Rossi, M | 1 |
Kiladjian, JJ | 1 |
Rampal, RK | 1 |
Hoffman, R | 1 |
Foucar, CE | 1 |
Stein, BL | 1 |
Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
---|---|---|---|---|---|---|---|
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 2 | 27 participants (Actual) | Interventional | 2018-02-21 | Terminated (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] |
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
Intervention | Percentage of Participants (Number) |
---|---|
Ruxolitinib-Naïve Participants With Splenomegaly | 40 |
Ruxolitinib-Naïve Participants Without Splenomegaly | 100 |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 0 |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 0 |
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)
Intervention | Percentage of Participants (Number) |
---|---|
Ruxolitinib-naïve Participants With Splenomegaly | 33.3 |
Ruxolitinib-naïve Participants Without Splenomegaly | 100 |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 75.0 |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 0 |
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
Intervention | Percentage of Participants (Number) |
---|---|
Ruxolitinib-Naïve Participants With Splenomegaly | 44.4 |
Ruxolitinib-Naïve Participants Without Splenomegaly | 100 |
Total Ruxolitinib-Naïve Participants | 54.5 |
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)
Intervention | Percentage of Participants (Number) |
---|---|
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 75.0 |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 0 |
Total Ruxolitinib-Resistant or Intolerant Participants | 60 |
"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)
Intervention | Percentage of Participants (Number) |
---|---|
Ruxolitinib-Naïve Participants With Splenomegaly | 0 |
Ruxolitinib-Naïve Participants Without Splenomegaly | 0 |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 0 |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 0 |
"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)
Intervention | Percentage of Participants (Number) |
---|---|
Ruxolitinib-Naïve Participants With Splenomegaly | 0 |
Ruxolitinib-Naïve Participants Without Splenomegaly | 0 |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 0 |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 0 |
"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)
Intervention | Percentage of Participants (Number) |
---|---|
Ruxolitinib-naïve Participants With Splenomegaly | 0 |
Ruxolitinib-naïve Participants Without Splenomegaly | 0 |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 0 |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 0 |
"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
Intervention | Percentage of Participants (Number) |
---|---|
Ruxolitinib-naïve Participants With Splenomegaly | 100 |
Ruxolitinib-naïve Participants Without SplenomegalyEdit | 100 |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 100 |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 100 |
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
Intervention | Percentage of Participants (Number) |
---|---|
Ruxolitinib-Naïve Participants With Splenomegaly | 44.4 |
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
Intervention | Percentage of Participants (Number) |
---|---|
Ruxolitinib-Naïve Participants Without Splenomegaly | 100 |
"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
Intervention | Score on a Scale (Mean) | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
EORTC QLQ-C30 Scores: Cognitive function Baseline | EORTC QLQ-C30 Scores: Cognitive function Cycle 2, Day 1 | EORTC QLQ-C30 Scores: Cognitive function Cycle 3 Day 28 | EORTC QLQ-C30 Scores: Cognitive function Cycle 5 Day 28 | EORTC QLQ-C30 Scores: Cognitive function Cycle 8 (Week 32) | EORTC QLQ-C30 Scores: Cognitive function Cycle 11 Day 28 | EORTC QLQ-C30 Scores: Cognitive function Cycle 14 Day 28 | EORTC QLQ-C30 Scores: Cognitive function Cycle 17 Day 28 | EORTC QLQ-C30 Scores: Cognitive function Cycle 20 Day 28 | EORTC QLQ-C30 Scores: Cognitive function Final Visit | EORTC QLQ-C30 Scores: Diarrhea Baseline | EORTC QLQ-C30 Scores: Diarrhea Cycle 2 Day 1 | EORTC QLQ-C30 Scores: Diarrhea Cycle 3 Day 28 | EORTC QLQ-C30 Scores: Diarrhea Cycle 5 Day 28 | EORTC QLQ-C30 Scores: Diarrhea Cycle 8 (Week 32) | EORTC QLQ-C30 Scores: Diarrhea Cycle 11 Day 28 | EORTC QLQ-C30 Scores: Diarrhea Cycle 14 Day 28 | EORTC QLQ-C30 Scores: Diarrhea Cycle 17 Day 28 | EORTC QLQ-C30 Scores: Diarrhea Cycle 20 Day 28 | EORTC QLQ-C30 Scores: Diarrhea Final visit | EORTC QLQ-C30 Scores: Emotional functioning Baseline | EORTC QLQ-C30 Scores: Emotional functioning Cycle 1 Day 28 | EORTC QLQ-C30 Scores: Emotional functioning Cycle 3 Day 28 | EORTC QLQ-C30 Scores: Emotional functioning Cycle 5 Day 28 | EORTC QLQ-C30 Scores: Emotional functioning Cycle 8 (Week 32) | EORTC QLQ-C30 Scores: Emotional functioning Cycle 11 Day 28 | EORTC QLQ-C30 Scores: Emotional functioning Cycle 14 Day 28 | EORTC QLQ-C30 Scores: Emotional functioning Cycle 17 Day 28 | EORTC QLQ-C30 Scores: Emotional functioning Cycle 20 Day 28 | EORTC QLQ-C30 Scores: Emotional functioning Final visit | EORTC QLQ-C30 Scores: Nausea and vomiting Baseline | EORTC QLQ-C30 Scores: Nausea and vomiting Cycle 2 Day 1 | EORTC QLQ-C30 Scores: Nausea and vomiting Cycle 3 Day 28 | EORTC QLQ-C30 Scores: Nausea and vomiting Cycle 5 Day 28 | EORTC QLQ-C30 Scores: Nausea and vomiting Cycle 8 (Week 32) | EORTC QLQ-C30 Scores: Nausea and vomiting Cycle 11 Day 28 | EORTC QLQ-C30 Scores: Nausea and vomiting Cycle 14 Day 28 | EORTC QLQ-C30 Scores: Nausea and vomiting Cycle 20 Day 28 | EORTC QLQ-C30 Scores: Nausea and vomiting Final visit | EORTC QLQ-C30 Scores: Social functioning Baseline | EORTC QLQ-C30 Scores: Social functioning Cycle 2 Day 1 | EORTC QLQ-C30 Scores: Social functioning Cycle 3 Day 28 | EORTC QLQ-C30 Scores: Social functioning Cycle 5 Day 28 | EORTC QLQ-C30 Scores: Social functioning Cycle 8 (Week 32) | EORTC QLQ-C30 Scores: Social functioning Cycle 11 Day 28 | EORTC QLQ-C30 Scores: Social functioning Cycle 14 Day 28 | EORTC QLQ-C30 Scores: Social functioning Cycle 17 Day 28 | EORTC QLQ-C30 Scores: Social functioning Cycle 20 Day 28 | EORTC QLQ-C30 Scores: Social functioning Final visit | EORTC QLQ-C30 Scores: Physical functioning Baseline | EORTC QLQ-C30 Scores: Physical functioning Cycle 2 Day 1 | EORTC QLQ-C30 Scores: Physical functioning Cycle 3 Day 28 | EORTC QLQ-C30 Scores: Physical functioning Cycle 5 Day 28 | EORTC QLQ-C30 Scores: Physical functioning Cycle 8 (Week 32) | EORTC QLQ-C30 Scores: Physical functioning Cycle 11 Day 28 | EORTC QLQ-C30 Scores: Physical functioning Cycle 14 Day 28 | EORTC QLQ-C30 Scores: Physical functioning Cycle 17 Day 28 | EORTC QLQ-C30 Scores: Physical functioning Cycle 20 Day 28 | EORTC QLQ-C30 Scores: Physical functioning Final visit | EORTC QLQ-C30 Scores: Global health status/QoL Baseline | EORTC QLQ-C30 Scores: Global health status/QoL Cycle 2 Day 1 | EORTC QLQ-C30 Scores: Global health status/QoL Cycle 3 Day 28 | EORTC QLQ-C30 Scores: Global health status/QoL Cycle 5 Day 28 | EORTC QLQ-C30 Scores: Global health status/QoL Cycle 8 (Week 32) | EORTC QLQ-C30 Scores: Global health status/QoL Cycle 11 Day 28 | EORTC QLQ-C30 Scores: Global health status/QoL Cycle 14 Day 28 | EORTC QLQ-C30 Scores: Global health status/QoL Cycle 17 Day 28 | EORTC QLQ-C30 Scores: Global health status/QoL Cycle 20 Day 28 | EORTC QLQ-C30 Scores: Global health status/QoL Final visit | EORTC QLQ-C30 Scores: Role functioning Baseline | EORTC QLQ-C30 Scores: Role functioning Cycle 2 Day 1 | EORTC QLQ-C30 Scores: Role functioning Cycle 3 Day 28 | EORTC QLQ-C30 Scores: Role functioning Cycle 5 Day 28 | EORTC QLQ-C30 Scores: Role functioning Cycle 8 (Week 32) | EORTC QLQ-C30 Scores: Role functioning Cycle 11 Day 28 | EORTC QLQ-C30 Scores: Role functioning Cycle 14 Day 28 | EORTC QLQ-C30 Scores: Role functioning Cycle 17 Day 28 | EORTC QLQ-C30 Scores: Role functioning Cycle 20 Day 28 | EORTC QLQ-C30 Scores: Role functioning Final Visit | |
Ruxolitinib-Naïve Participants | 65.83 | 11.11 | 7.02 | 1.19 | 6.06 | 8.33 | 5.56 | 0.00 | 0 | 4.44 | 8.33 | -5.56 | 1.75 | 7.14 | 9.09 | 5.56 | 5.56 | 25.00 | 0 | 13.33 | 65.83 | 13.43 | 14.04 | 16.07 | 14.39 | 6.94 | 5.56 | -8.33 | 0 | 3.33 | 10.00 | -4.63 | -1.75 | -2.38 | 7.58 | 2.78 | -2.78 | 0 | 11.11 | 67.50 | 4.63 | -1.75 | 5.95 | 0.00 | 0.00 | 0.00 | -4.17 | 0 | 0.00 | 86.33 | 1.48 | -2.81 | 1.43 | 5.45 | -1.11 | -1.11 | 1.67 | 0 | -4.44 | 61.25 | 2.31 | 7.89 | 7.14 | 9.09 | 1.39 | 2.78 | 10.42 | 25.00 | -3.33 | 74.17 | 7.41 | 2.63 | 7.14 | 15.15 | -2.78 | 5.56 | 8.33 | 16.67 | -13.33 |
Ruxolitinib-Resistant or Intolerant Participants | 76.19 | 8.33 | 6.67 | 11.11 | -3.33 | 0 | 0 | -8.33 | 0 | 6.67 | 14.29 | 5.56 | 0 | 5.56 | 20.00 | 0 | 0 | 0 | 0 | 6.67 | 64.29 | 15.28 | 8.33 | 6.94 | 5.00 | 16.67 | -4.17 | 0 | 8.33 | 16.67 | 2.38 | 8.33 | 3.33 | 8.33 | 16.67 | 0.00 | -8.33 | 0 | 6.67 | 76.19 | 0.00 | 3.33 | -2.78 | -6.67 | 0 | 0 | 0 | 0 | -6.67 | 81.90 | 2.22 | 2.67 | -2.22 | -4.00 | -10.00 | 0.00 | 0 | 0 | -2.67 | 60.71 | 1.39 | 11.67 | 0.00 | -8.33 | 8.33 | 8.33 | 16.67 | 25.00 | 13.33 | 76.19 | -8.33 | 0.00 | -2.78 | 0.00 | -8.33 | 0 | 0 | 0 | 6.67 |
"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
Intervention | Score on a Scale (Mean) | |||||||||
---|---|---|---|---|---|---|---|---|---|---|
Baseline (Cycle 1 Day 1) | Cycle 2 Day 1 | Cycle 3 Day 28 | Cycle 5 Day 28, | Week 32 | Cycle 11 Day 28 | Cycle 14 Day 28 | Cycle 17 Day 28 | Cycle 20 Day 28 | Final Visit | |
Ruxolitinib-Naïve Participants | 31.95 | -5.06 | -6.38 | -7.00 | -8.20 | -4.60 | -4.67 | -3.25 | -12.00 | -5.92 |
Ruxolitinib-Resistant or Intolerant Participants | 26.00 | 0.80 | -8.00 | -9.50 | -5.00 | -7.50 | -10.50 | -12.00 | -8.00 | -7.20 |
"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
Intervention | Millimeters of mercury (mmHg) (Mean) | |||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle1, Day 15 | Cycle 1, Day 22 | Cycle 2, Day 1 | Cycle 2, Day 15 | Cycle 3 Day 1 | Cycle 3, Day 15 | Cycle 4, Day 1 | Cycle 5, Day 1 | Cycle 6, Day 1 | Cycle 7, Day 1 | Cycle 8, Day 1 | Cycle 9, Day 1 | Cycle 10, Day 1 | Cycle 11, Day 1 | Cycle 12, Day 1 | Cycle 13, Day 1 | Cycle 14, Day 1 | Cycle 15, Day 1 | Cycle 16, Day 1 | Cycle 17, Day 1 | Final visit | |
Ruxolitinib-naïve Participants Without Splenomegaly | 73.2 | 10.0 | 2.4 | 3.0 | 2.5 | 5.8 | 2.0 | 2.7 | 5.0 | 3.3 | 4.5 | 5.0 | 4.0 | 5.0 | 10.0 | 6.0 | 4.0 | 19.0 | 10.0 | 11.0 | 11.0 | 3.8 |
"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
Intervention | Millimeters of mercury (mmHg) (Mean) | ||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle1, Day 15 | Cycle 1, Day 22 | Cycle 2, Day 1 | Cycle 2, Day 15 | Cycle 3 Day 1 | Cycle 3, Day 15 | Cycle 4, Day 1 | Cycle 5, Day 1 | Cycle 6, Day 1 | Cycle 7, Day 1 | Cycle 8, Day 1 | Cycle 9, Day 1 | Cycle 10, Day 1 | Cycle 11, Day 1 | Cycle 12, Day 1 | Cycle 13, Day 1 | Cycle 14, Day 1 | Cycle 15, Day 1 | Cycle 16, Day 1 | Cycle 17, Day 1 | Cycle 18, Day 1 | Cycle 19, Day 1 | Cycle 20, Day 1 | Final visit | |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 72.3 | -1.8 | -2.2 | -0.2 | -5.5 | -4.0 | -7.5 | -0.4 | -5.0 | -2.0 | -5.3 | 0.5 | -2.7 | -2.3 | 1.0 | 2.0 | 2.0 | -9.0 | -2.0 | 7.0 | 3.0 | -2.0 | 5.0 | -1.0 | -2.0 |
"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
Intervention | Millimeters of mercury (mmHg) (Mean) | ||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle1, Day 15 | Cycle 1, Day 22 | Cycle 2, Day 1 | Cycle 2, Day 15 | Cycle 3 Day 1 | Cycle 3, Day 15 | Cycle 4, Day 1 | Cycle 5, Day 1 | Cycle 6, Day 1 | Cycle 7, Day 1 | Cycle 8, Day 1 | Cycle 9, Day 1 | Cycle 10, Day 1 | Cycle 11, Day 1 | Cycle 12, Day 1 | Cycle 13, Day 1 | Cycle 14, Day 1 | Cycle 15, Day 1 | Cycle 16, Day 1 | Cycle 17, Day 1 | Cycle 18, Day 1 | Cycle 19, Day 1 | Cycle 20, Day 1 | Cycle 21, Day 1 | Cycle 22, Day 1 | Final visit | |
Ruxolitinib-naïve Participants With Splenomegaly | 76.3 | 5.1 | 3.6 | 6.1 | 4.1 | 6.2 | 1.8 | 5.5 | 1.7 | 5.9 | 2.9 | 7.8 | 4.1 | 3.7 | 9.7 | 9.8 | 9.0 | 5.6 | 7.0 | 9.8 | 11.3 | 5.0 | 4.0 | 12.5 | -3.0 | 22.0 | 5.8 |
"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
Intervention | Millimeters of mercury (mmHg) (Mean) | |||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle1, Day 15 | Cycle 1, Day 22 | Cycle 2, Day 1 | Cycle 2, Day 15 | Cycle 3 Day 1 | Cycle 3, Day 15 | Cycle 4, Day 1 | Cycle 5, Day 1 | Cycle 6, Day 1 | Cycle 7, Day 1 | Cycle 8, Day 1 | Cycle 9, Day 1 | Cycle 10, Day 1 | Cycle 11, Day 1 | Cycle 12, Day 1 | Cycle 13, Day 1 | Cycle 14, Day 1 | Cycle 15, Day 1 | Cycle 16, Day 1 | Cycle 17, Day 1 | Cycle 18, Day 1 | Cycle 19, Day 1 | Cycle 20, Day 1 | Cycle 21, Day 1 | Cycle 22, Day 1 | Cycle 23, Day 1 | Final visit | |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 65.0 | 0.0 | -1.0 | 3.0 | 16.0 | 5.0 | 8.0 | 1.0 | 1.0 | 12.0 | 30.0 | 10.0 | 4.0 | 3.0 | 24.0 | 8.0 | 10.0 | 11.0 | 7.0 | 6.0 | 7.0 | 3.0 | 8.0 | 2.0 | 7.0 | 8.0 | 7.0 | 11.0 |
"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)
Intervention | Millisecond (msec) (Mean) | |||||||||||||||||||||||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
PR Duration Baseline | PQ(PR) Durations Cycle 1, Day 1, 4 hour | PQ(PR) Durations Cycle 1, Day 1, 6 hour | PQ(PR) Durations Cycle 1, Day 2, 24 hour | PQ(PR) Durations Cycle ,1 Day 5, pre-dose | PQ(PR) Durations Cycle 1, Day 5, 4 hour | PQ(PR) Durations Cycle 1, Day 5, 6 hour | PQ(PR) Durations Cycle 2, Day1, pre-dose | QRS Duration Baseline | QRS 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 Baseline | QT 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 baseline | QTcB - 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 Baseline | QTcF 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 Baseline | RR Duration Cycle 1 Day 1 4 H | RR Duration Cycle 1 Day 1 6 H | 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-Resistant or Intolerant Participants With Splenomegaly | 152.00 | -2.00 | 6.00 | 0.67 | 0.40 | -1.20 | 7.60 | -2.33 | 83.33 | 1.33 | 0.33 | 3.00 | 0.00 | -0.40 | 2.00 | 1.00 | 386.00 | 5.67 | -1.33 | 7.00 | 8.00 | 3.20 | 7.60 | 19.00 | 424.50 | 13.83 | 6.33 | 4.50 | -9.20 | -4.60 | -1.60 | 0.17 | 411.17 | 11.67 | 3.50 | 5.33 | -3.20 | -2.00 | 1.60 | 6.83 | 835.83 | -36.50 | -31.17 | 8.83 | 67.80 | 24.20 | 33.00 | 78.17 |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 196.00 | 4.00 | 0.00 | -12.00 | -4.00 | 0 | 0 | -8.00 | 94.00 | 0 | -2.00 | -2.00 | 2.00 | 0 | 0 | -4.00 | 392.00 | 36.00 | 36.00 | 0 | 4.00 | -12.00 | -4.00 | 12.00 | 444.00 | -30.00 | 5.00 | -14.00 | -7.00 | 0.00 | -12.00 | -23.00 | 426.00 | -8.00 | 16.00 | -9.00 | -3.00 | -4.00 | 6.00 | -11.00 | 779.00 | 292.00 | 130.00 | 54.00 | 43.00 | -47.00 | -56.00 | 144.00 |
"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)
Intervention | Millisecond (msec) (Mean) | |||||||||||||||||||||||||||||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
PR Duration Baseline | PQ(PR) Durations Cycle 1, Day 1, 4 hour | PQ(PR) Durations Cycle 1, Day 1, 6 hour | PQ(PR) Durations Cycle 1, Day 2, pre-dose | PQ(PR) Durations Cycle 1, Day 2, 24 hour | PQ(PR) Durations Cycle ,1 Day 5, pre-dose | PQ(PR) Durations Cycle 1, Day 5, 4 hour | PQ(PR) Durations Cycle 1, Day 5, 6 hour | PQ(PR) Durations Cycle 2, Day1, pre-dose | QRS Duration Baseline | QRS 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 Baseline | QT 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 baseline | QTcB - 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 Baseline | QTcF 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 Baseline | RR Duration Cycle 1 Day 1 4 H | RR Duration Cycle 1 Day 1 6 H | RR 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 Splenomegaly | 153.60 | -2.60 | -4.40 | -6.00 | 1.00 | -12.00 | -6.25 | -2.75 | -5.75 | 83.80 | 4.60 | 4.00 | 2.00 | 1.75 | 4.00 | 3.75 | -0.25 | 7.75 | 392.60 | 16.80 | 11.00 | -18.00 | 2.75 | -8.75 | 15.25 | 4.75 | 10.50 | 419.60 | 21.00 | 8.40 | -410.00 | -0.75 | -10.00 | 1.50 | -4.75 | 7.75 | 410.00 | 18.80 | 10.00 | -20.00 | 0.00 | -9.25 | 4.00 | -2.25 | 8 | 881.00 | -5.60 | 14.00 | 18.00 | 17.25 | -0.75 | 79.75 | 49.50 | 23.75 |
"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)
Intervention | Millisecond (msec) (Mean) | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
PR Duration Baseline | PQ(PR) Durations Cycle 1, Day 1, 4 hour | PQ(PR) Durations Cycle 1, Day 1, 6 hour | PQ(PR) Durations Cycle 1, Day 2, pre-dose | PQ(PR) Durations Cycle 1, Day 2, 24 hour | PQ(PR) Durations Cycle ,1 Day 5, pre-dose | PQ(PR) Durations Cycle 1, Day 5, 4 hour | PQ(PR) Durations Cycle 1, Day 5, 6 hour | PQ(PR) Durations Cycle 2, Day1, pre-dose | PQ(PR) Durations Cycle 3, Day 1, pre-dose | PQ(PR) Durations Cycle 3, Day 1, 4 hour | PQ(PR) Durations Cycle 3, Day 1, 6 hour | PQ(PR) Durations Cycle 4, Day 1, pre-dose | PQ(PR) Durations Cycle 4, Day 1, 4 hour | QRS Duration Baseline | QRS 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 Baseline | QT 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 baseline | QTcB - 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 H | QTcF Durations Fridericia's Correction Formula Baseline | QTcF 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 Baseline | RR Duration Cycle 1 Day 1 4 H | RR Duration Cycle 1 Day 1 6 H | RR 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 Splenomegaly | 158.93 | 0.60 | -2.80 | -14.00 | 1.93 | -1.00 | -8.00 | -4.77 | 1.43 | -6.00 | -8.00 | -6.00 | -20.00 | -20.00 | 90.87 | -1.47 | -1.27 | -4.00 | 1.07 | 0.13 | -1.08 | -0.92 | 0.64 | -4.00 | -4.00 | -4.00 | -2.00 | -2.00 | 396.67 | -7.33 | -9.40 | -6.00 | -10.21 | -6.20 | -4.31 | -10.31 | 5.00 | -2.00 | -10.00 | -4.00 | -16.00 | -16.00 | 427.67 | 3.80 | 8.00 | -15.00 | 3.71 | -11.07 | -62.00 | -2.92 | -4.54 | 3.93 | -16.00 | 6.00 | 8.00 | -62.00 | -62.00 | 417.93 | -1.27 | 1.00 | -12.00 | -2.36 | -10.47 | -0.08 | -7.85 | 3.36 | -11.00 | 0.00 | 4.00 | -45.00 | -45.00 | 861.47 | -38.87 | -65.67 | 28.00 | -51.50 | 25.87 | -26.23 | -21.46 | 7.14 | 48.00 | -59.00 | -43.00 | 182.00 | 182.00 |
"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)
Intervention | Beats per Minute (Mean) | |||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1, Day 1, 4 Hour | Cycle 1, Day 1, 6 Hour | Cycle 1 Day 2, pre-dose | Cycle 1 Day 2, 24 Hour | Cycle 1 Day 5, pre-dose | Cycle 1 Day 5, 4 hour | Cycle 1 Day 5, 6 hour | Cycle 2, Day 1, pre-dose | Cycle 3 Day 1, pre-dose | Cycle 3 Day 1, 4 hour | Cycle 3 Day 1, 6 hour | Cycle 4 Day 1, pre-dose | Cycle 4 Day 1, 4 hour | |
Ruxolitinib-naïve Participants With Splenomegaly | 71.47 | 4.40 | 6.20 | -3.00 | 4.71 | -1.47 | 2.69 | 2.23 | -1.43 | -5.00 | 7.00 | 5.00 | -16.00 | -16.00 |
"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)
Intervention | Beats per Minute (Mean) | |||||||
---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1, Day 1, 4 Hour | Cycle 1, Day 1, 6 Hour | Cycle 1 Day 2, 24 Hour | Cycle 1 Day 5, pre-dose | Cycle 1 Day 5, 4 hour | Cycle 1 Day 5, 6 hour | Cycle 2, Day 1, pre-dose | |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 77.00 | -21.00 | -11.00 | -5.00 | -4.00 | 5.00 | 6.00 | -12.00 |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 73.17 | 2.50 | 3.00 | -0.67 | -5.60 | -2.20 | -2.80 | -6.17 |
"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)
Intervention | Beats per Minute (Mean) | ||||||||
---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1, Day 1, 4 Hour | Cycle 1, Day 1, 6 Hour | Cycle 1 Day 2, pre-dose | Cycle 1 Day 2, 24 Hour | Cycle 1 Day 5, pre-dose | Cycle 1 Day 5, 4 hour | Cycle 1 Day 5, 6 hour | Cycle 2, Day 1, pre-dose | |
Ruxolitinib-naïve Participants Without Splenomegaly | 69.80 | 0.60 | -0.60 | -1.00 | -1.75 | -0.75 | -5.00 | -3.75 | -1.50 |
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
Intervention | Degrees Celsius (C) (Mean) | |||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1 Day 15 | Cycle 1 Day 22 | Cycle 2 Day 1 | Cycle 2 Day 15 | Cycle 3 Day 1 | Cycle 3 Day 15 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12 Day 1 | Cycle 13 Day 1 | Cycle 14 Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17 Day 1 | Final Visit | |
Ruxolitinib-naïve Participants Without Splenomegaly | 36.40 | 0.42 | 0.04 | 0.05 | 0.18 | 0.13 | -0.03 | -0.17 | -0.10 | -0.10 | -0.20 | -0.05 | 0.05 | 0.30 | -0.20 | 0.30 | -0.70 | -0.10 | -0.30 | -0.80 | 0.60 | 0.26 |
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
Intervention | Degrees Celsius (C) (Mean) | ||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1 Day 15 | Cycle 1 Day 22 | Cycle 2 Day 1 | Cycle 2 Day 15 | Cycle 3 Day 1 | Cycle 3 Day 15 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12 Day 1 | Cycle 13 Day 1 | Cycle 14 Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17 Day 1 | Cycle 18 Day 1 | Cycle 19 Day 1 | Cycle 20 Day 1 | Final Visit | |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 36.47 | 0.18 | 0.15 | 0.17 | 0.23 | 0.17 | 0.10 | 0.06 | 0.10 | 0.12 | 0.20 | 0.20 | -0.07 | 0.10 | -0.10 | -0.20 | 0 | -0.30 | -0.40 | -0.10 | 0.00 | -0.20 | 0 | 0 | 0.08 |
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
Intervention | Degrees Celsius (C) (Mean) | ||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1 Day 15 | Cycle 1 Day 22 | Cycle 2 Day 1 | Cycle 2 Day 15 | Cycle 3 Day 1 | Cycle 3 Day 15 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12 Day 1 | Cycle 13 Day 1 | Cycle 14 Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17 Day 1 | Cycle 18 Day 1 | Cycle 19 Day 1 | Cycle 20 Day 1 | Cycle 21 Day 1 | Cycle 22 Day 1 | Final Visit | |
Ruxolitinib-naïve Participants With Splenomegaly | 36.50 | 0.13 | 0.03 | -0.05 | 0.04 | -0.09 | -0.04 | -0.05 | 0.05 | -0.03 | 0.01 | -0.01 | 0.03 | -0.08 | -0.07 | -0.10 | 0.00 | 0.00 | -0.10 | 0.03 | 0.03 | 0.07 | -0.15 | 0.00 | 0 | -0.50 | -0.08 |
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
Intervention | Degrees Celsius (C) (Mean) | |||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1 Day 15 | Cycle 1 Day 22 | Cycle 2 Day 1 | Cycle 2 Day 15 | Cycle 3 Day 1 | Cycle 3 Day 15 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12 Day 1 | Cycle 13 Day 1 | Cycle 14 Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17 Day 1 | Cycle 18 Day 1 | Cycle 19 Day 1 | Cycle 20 Day 1 | Cycle 21 Day 1 | Cycle 22 Day 1 | Cycle 23 Day 1 | Final Visit | |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 37.10 | -0.30 | -0.50 | -0.60 | -0.30 | 0.10 | -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 |
"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
Intervention | Beats per Minute (Mean) | |||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1 Day 15 | Cycle 1 Day 22 | Cycle 2 Day 1 | Cycle 2 Day 15 | Cycle 3 Day 1 | Cycle 3 Day 15 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12, Day 1 | Cycle 13, Day 1 | Cycle 14, Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17, Day 1 | Final Visit | |
Ruxolitinib-naïve Participants Without Splenomegaly | 71.6 | 5.4 | 8.2 | 1.8 | 1.8 | -2.0 | 10.8 | 1.7 | -4.0 | -4.3 | 1.5 | -1.0 | 0.0 | 8.5 | -2.0 | -6.0 | -5.0 | -2.0 | 10.0 | -2.0 | 9.0 | 0.2 |
"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
Intervention | Beats per Minute (Mean) | ||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1 Day 15 | Cycle 1 Day 22 | Cycle 2 Day 1 | Cycle 2 Day 15 | Cycle 3 Day 1 | Cycle 3 Day 15 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12, Day 1 | Cycle 13, Day 1 | Cycle 14, Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17, Day 1 | Cycle 18, Day 1 | Cycle 19, Day 1 | Cycle 20, Day 1 | Final Visit | |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 76.7 | -0.7 | -5.8 | -9.3 | -5.8 | -4.2 | -2.2 | -6.6 | -3.6 | -1.6 | -1.0 | 0.5 | 0.3 | 2.3 | 2.5 | -1.0 | -1.0 | -4.0 | -2.0 | -2.0 | 1.0 | -3.0 | 6.0 | 4.0 | 0.2 |
"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
Intervention | Beats per Minute (Mean) | ||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1 Day 15 | Cycle 1 Day 22 | Cycle 2 Day 1 | Cycle 2 Day 15 | Cycle 3 Day 1 | Cycle 3 Day 15 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12, Day 1 | Cycle 13, Day 1 | Cycle 14, Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17, Day 1 | Cycle 18, Day 1 | Cycle 19, Day 1 | Cycle 20, Day 1 | Cycle 21, Day 1 | Cycle 22, Day 1 | Final Visit | |
Ruxolitinib-naïve Participants With Splenomegaly | 74.7 | 4.5 | 3.9 | -1.1 | 0.1 | -2.8 | -1.2 | 1.0 | 0.6 | 0.4 | 1.4 | -3.9 | -5.4 | -0.3 | -2.5 | -0.2 | -2.8 | -3.8 | -0.8 | -1.3 | -12.7 | -4.3 | -9.0 | -5.0 | 2.0 | -8.0 | -1.7 |
"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
Intervention | Beats per Minute (Mean) | |||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1 Day 15 | Cycle 1 Day 22 | Cycle 2 Day 1 | Cycle 2 Day 15 | Cycle 3 Day 1 | Cycle 3 Day 15 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12, Day 1 | Cycle 13, Day 1 | Cycle 14, Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17, Day 1 | Cycle 18, Day 1 | Cycle 19, Day 1 | Cycle 20, Day 1 | Cycle 21, Day 1 | Cycle 22, Day 1 | Cycle 23, Day 1 | Final Visit | |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 68.0 | 7.0 | 8.0 | 12.0 | 9.0 | 9.0 | 21.0 | 24.0 | 28.0 | 14.0 | 21.0 | 11.0 | 0 | 20.0 | 37.0 | 21.0 | 16.0 | 12.0 | 14.0 | 16.0 | 22.0 | 16.0 | 15.0 | 5.0 | 17.0 | 14 | 19.0 | 16.0 |
"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
Intervention | Breaths per Minute (Mean) | |||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1 Day 15 | Cycle 1 Day 22 | Cycle 2 Day 1 | Cycle 2 Day 15 | Cycle 3 Day 1 | Cycle 3 Day 15 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12 Day 1 | Cycle 13 Day 1 | Cycle 14 Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17 Day 1 | Final visit | |
Ruxolitinib-naïve Participants Without Splenomegaly | 17.4 | -0.2 | 0.2 | -0.3 | 0.0 | -1.5 | 0.0 | -3.3 | -0.3 | -0.5 | 0 | -0.5 | 0.5 | -0.5 | 1.0 | 1.0 | 1.0 | 1.0 | 1.0 | 1.0 | 1.0 | -1.2 |
"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
Intervention | Breaths per Minute (Mean) | ||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1 Day 15 | Cycle 1 Day 22 | Cycle 2 Day 1 | Cycle 2 Day 15 | Cycle 3 Day 1 | Cycle 3 Day 15 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12 Day 1 | Cycle 13 Day 1 | Cycle 14 Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17 Day 1 | Cycle 18 Day 1 | Cycle 19 Day 1 | Cycle 20 Day 1 | Final visit | |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 18.2 | -0.2 | -0.5 | -0.4 | -1.2 | -1.8 | -1.0 | -1.0 | 1.0 | -0.6 | -2.3 | -0.8 | -1.7 | -0.3 | -0.5 | 1.5 | -1.0 | 1.0 | -1.0 | -2.0 | -2.0 | -1.0 | -3.0 | -3.0 | -1.8 |
"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
Intervention | Breaths per Minute (Mean) | ||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1 Day 15 | Cycle 1 Day 22 | Cycle 2 Day 1 | Cycle 2 Day 15 | Cycle 3 Day 1 | Cycle 3 Day 15 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12 Day 1 | Cycle 13 Day 1 | Cycle 14 Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17 Day 1 | Cycle 18 Day 1 | Cycle 19 Day 1 | Cycle 20 Day 1 | Cycle 21 Day 1 | Cycle 22 Day 1 | Final visit | |
Ruxolitinib-naïve Participants With Splenomegaly | 17.4 | -0.3 | -0.5 | -0.3 | -0.2 | -0.7 | -0.7 | -0.3 | -0.4 | -0.5 | -0.2 | 0.1 | 0.4 | -0.2 | -0.8 | -0.4 | -1.4 | 0.8 | 0.0 | -0.3 | 1.3 | 1.3 | 1.5 | 2.0 | 2.0 | 0.0 | -0.3 |
"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
Intervention | Breaths per Minute (Mean) | |||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1 Day 15 | Cycle 1 Day 22 | Cycle 2 Day 1 | Cycle 2 Day 15 | Cycle 3 Day 1 | Cycle 3 Day 15 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12 Day 1 | Cycle 13 Day 1 | Cycle 14 Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17 Day 1 | Cycle 18 Day 1 | Cycle 19 Day 1 | Cycle 20 Day 1 | Cycle 21 Day 1 | Cycle 22 Day 1 | Cycle 23 Day 1 | Final visit | |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 16.0 | 0 | 0 | 0 | 2.0 | 0 | 0 | 2.0 | 0 | 2.0 | 2.0 | 0 | -2.0 | 0 | 2.0 | 0 | 2.0 | 0 | 0 | 0 | 0 | 2.0 | 2.0 | 2.0 | 0 | 0 | 0 | 0 |
"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
Intervention | Millimeters of mercury (mmHg) (Mean) | |||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1, Day 15 | Cycle 1, Days 22 | Cycle 2, Day 1 | Cycle 2 , Day 15 | Cycle 3, Day 1 | Cycle 3, Day 15 | Cycle 4, Day 1 | Cycle 5, Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12 Day 1 | Cycle 13 Day 1 | Cycle 14 Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17 Day 1 | Final Visit | |
Ruxolitinib-naïve Participants Without Splenomegaly | 132.0 | 3.6 | -5.6 | 2.8 | -2.0 | -3.0 | -3.5 | -7.3 | -2.5 | 2.5 | -4.0 | 5.0 | 3.0 | -3.0 | 14.0 | -8.0 | -8.0 | 18.0 | 14.0 | 8.0 | 24.0 | 7.6 |
"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
Intervention | Millimeters of mercury (mmHg) (Mean) | ||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1, Day 15 | Cycle 1, Days 22 | Cycle 2, Day 1 | Cycle 2 , Day 15 | Cycle 3, Day 1 | Cycle 3, Day 15 | Cycle 4, Day 1 | Cycle 5, Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12 Day 1 | Cycle 13 Day 1 | Cycle 14 Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17 Day 1 | Cycle 18 Day 1 | Cycle 19 Day 1 | Cycle 20 Day 1 | Final Visit | |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 122.3 | 3.8 | 4.8 | -4.3 | -7.2 | 7.3 | -3.8 | 9.8 | 3.3 | 8.6 | 10.0 | 14.0 | -4.3 | 11.0 | 11.0 | 6.5 | 8.0 | 3.0 | 4.0 | 9.0 | 7.0 | 9.0 | 9.0 | 9.0 | 12.8 |
"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
Intervention | Millimeters of mercury (mmHg) (Mean) | ||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1, Day 15 | Cycle 1, Days 22 | Cycle 2, Day 1 | Cycle 2 , Day 15 | Cycle 3, Day 1 | Cycle 3, Day 15 | Cycle 4, Day 1 | Cycle 5, Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12 Day 1 | Cycle 13 Day 1 | Cycle 14 Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17 Day 1 | Cycle 18 Day 1 | Cycle 19 Day 1 | Cycle 20 Day 1 | Cycle 21 Day 1 | Cycle 22 Day 1 | Final Visit | |
Ruxolitinib-naïve Participants With Splenomegaly | 129.3 | 4.4 | 3.3 | 3.0 | 5.2 | 5.5 | 1.2 | 2.2 | 4.2 | 11.2 | 4.7 | 8.8 | 12.6 | 11.8 | 8.5 | 10.8 | 13.4 | 4.4 | 13.6 | 7.5 | 9.3 | 5.7 | 2.5 | 11.5 | -18.0 | 24.0 | 1.3 |
"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
Intervention | Millimeters of mercury (mmHg) (Mean) | |||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline | Cycle 1, Day 15 | Cycle 1, Days 22 | Cycle 2, Day 1 | Cycle 2 , Day 15 | Cycle 3, Day 1 | Cycle 3, Day 15 | Cycle 4, Day 1 | Cycle 5, Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 8 Day 1 | Cycle 9 Day 1 | Cycle 10 Day 1 | Cycle 11 Day 1 | Cycle 12 Day 1 | Cycle 13 Day 1 | Cycle 14 Day 1 | Cycle 15 Day 1 | Cycle 16 Day 1 | Cycle 17 Day 1 | Cycle 18 Day 1 | Cycle 19 Day 1 | Cycle 20 Day 1 | Cycle 21 Day 1 | Cycle 22 Day 1 | Cycle 23 Day 1 | Final Visit | |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 106.0 | 5.0 | -5.0 | 10.0 | 30.0 | 0 | 6.0 | 13.0 | 16.0 | 8.0 | 29.0 | 9.0 | 26.0 | 30.0 | 34.0 | 6.0 | 14.0 | 14.0 | 5.0 | 4.0 | 6.0 | 13.0 | 13.0 | 13.0 | 7.0 | 6.0 | 11.0 | 19.0 |
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
Intervention | Participants (Number) | |
---|---|---|
Cycle 11, Day 28 | Week 32 | |
Ruxolitinib-naïve Participants With Splenomegaly | 2 | 3 |
Ruxolitinib-naïve Participants Without Splenomegaly | 1 | 2 |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 0 | 3 |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 0 | 0 |
"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
Intervention | Percentage of Participant (Number) | |
---|---|---|
Baseline 0 | Baseline 1 | |
Ruxolitinib-naïve Participants With Splenomegaly | 66.7 | 33.3 |
Ruxolitinib-naïve Participants Without Splenomegaly | 60.0 | 40.0 |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 50.0 | 50.0 |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 100.0 | 0 |
"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)
Intervention | Count of Participants (Number) | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Cycle 2, Day 1 Very Much Improved | Cycle 2, Day 1 Much Improved | Cycle 2, Day 1 Minimally Improved | Cycle 2, Day 1 No Change | Cycle 2, Day 1 Minimally Worse | Cycle 2, Day 1 Much Worse | Cycle 2, Day 1 Very Much Worse | Cycle 2, Day 1 Not Assessed | Cycle 3 Day 28 Very Much Improved | Cycle 3 Day 28 Much Improved | Cycle 3 Day 28 Minimally Improved | Cycle 3 Day 28 No Change | Cycle 3 Day 28 Minimally Worse | Cycle 3 Day 28 Much Worse | Cycle 3 Day 28 Very Much Worse | Cycle 3 Day 28 Not Assessed | Cycle 5 Day 28 Very Much Improved | Cycle 5 Day 28 Much Improved | Cycle 5 Day 28 Minimally Improved | Cycle 5 Day 28 No Change | Cycle 5 Day 28 Minimally Worse | Cycle 5 Day 28 Much Worse | Cycle 5 Day 28 Very Much Worse | Cycle 5 Day 28 Not Assessed | Week 32 Very Much Improved | Week 32 Much Improved | Week 32 Minimally Improved | Week 32 No Change | Week 32 Minimally Worse | Week 32 Much Worse | Week 32 Very Much Worse | Week 32 Not Assessed | Cycle 11 Day 28 Very Much Improved | Cycle 11 Day 28 Much Improved | Cycle 11 Day 28 Minimally Improved | Cycle 11 Day 28 No Change | Cycle 11 Day 28 Minimally Worse | Cycle 11 Day 28 Much Worse | Cycle 11 Day 28 Very Much Worse | Cycle 11 Day 28 Not Assessed | Cycle 14 Day 28 Very Much Improved | Cycle 14 Day 28 Much Improved | Cycle 14 Day 28 Minimally Improved | Cycle 14 Day 28 No change | Cycle 14 Day 28 Minimally Worse | Cycle 14 Day 28 Much Worse | Cycle 14 Day 28 Very Much Worse | Cycle 14 Day 28 Not Assessed | Cycle 17 Day 28 Very Much Improved | Cycle 17 Day 28 Much Improved | Cycle 17 Day 28 Minimally Improved | Cycle 17 Day 28 No Change | Cycle 17 Day 28 Minimally Worse | Cycle 17 Day 28 Much Worse | Cycle 17 Day 28 Very Much Worse | Cycle 17 Day 28 Not Assessed | Cycle 20 Day 28 Very Much Improved | Cycle 20 Day 28 Much Improved | Cycle 20 Day 28 Minimally Improved | Cycle 20 Day 28 No Change | Cycle 20 Day 28 Minimally Worse | Cycle 20 Day 28 Much Worse | Cycle 20 Day 28 Very Much Worse | Cycle 20 Day 28 Not Assessed | Final Visit Very Much Improved | Final Visit Much Improved | Final Visit Minimally Improved | Final Visit No Change | Final Visit Minimally Worse | Final Visit Much Worse | Final Visit Very Much Worse | Final Visit Not Assessed | |
Ruxolitinib-Naïve Participants | 0 | 4 | 5 | 6 | 1 | 0 | 0 | 0 | 4 | 4 | 6 | 3 | 0 | 0 | 0 | 0 | 2 | 6 | 2 | 1 | 0 | 0 | 0 | 4 | 3 | 3 | 3 | 1 | 1 | 0 | 0 | 0 | 1 | 3 | 0 | 0 | 1 | 0 | 0 | 0 | 2 | 2 | 0 | 1 | 1 | 0 | 0 | 0 | 1 | 1 | 1 | 1 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 3 | 3 | 2 | 6 | 1 | 0 | 0 | 2 |
Ruxolitinib-Resistant or Intolerant Participants | 0 | 1 | 3 | 1 | 1 | 0 | 0 | 1 | 1 | 1 | 3 | 0 | 0 | 0 | 0 | 1 | 1 | 1 | 4 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 3 | 1 | 0 | 0 | 0 | 0 | 0 | 2 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 2 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 2 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 3 | 1 | 0 | 0 | 0 | 0 | 0 |
"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)
Intervention | Participants (Number) | |||
---|---|---|---|---|
HCT Control | Composite Response | ELN Response | Complete Hematologic Response | |
Ruxolitinib-Naïve Participants With Splenomegaly | 3 | 0 | 4 | 2 |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 3 | 0 | 3 | 2 |
"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)
Intervention | Participants (Number) | |||
---|---|---|---|---|
HCT Control | Composite Response | ELN Response | Complete Hematologic Response | |
Ruxolitinib-Naïve Participants Without Splenomegaly | 2 | 0 | 2 | 2 |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 0 | 0 | 0 | 0 |
"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)
Intervention | Participants (Number) | |||
---|---|---|---|---|
HCT Control | Composite Response | ELN Response | Complete Hematologic Response | |
All Ruxolitinib-Naïve Participants | 5 | 0 | 6 | 4 |
All Ruxolitinib-Resistant or Intolerant Participants | 3 | 0 | 3 | 2 |
"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)
Intervention | Percentage of Participants (Number) | |||||||||||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline Complete Response | Baseline Partial Response | Baseline Progressive Disease | Baseline No Response | Cycle 3, Day 28 Complete Response | Cycle 3, Day 28 Partial Response | Cycle 3, Day 28 Progressive Disease | Cycle 3, Day 28 No Response | Cycle 5, Day 28 Complete Response | Cycle 5, Day 28 Partial Response | Cycle 5, Day 28 Progressive Disease | Cycle 5, Day 28 No Response | Cycle 8, Day 28 (Week 32) Complete Response | Cycle 8, Day 28 (Week 32) Partial Response | Cycle 8, Day 28 (Week 32) Progressive Disease | Cycle 8, Day 28 (Week 32) No Response | Cycle 11 Day 28 Complete Response | Cycle 11 Day 28 Partial Response | Cycle 11 Day 28 Progressive Disease | Cycle 11 Day 28 No Response | Cycle 14, Day 28 Complete Response | Cycle 14, Day 28 Partial Response | Cycle 14, Day 28 Progressive Disease | Cycle 14, Day 28 No Response | Cycle 17, Day 28 Complete Response | Cycle 17, Day 28 Partial Response | Cycle 17, Day 28 Progressive Disease | Cycle 17, Day 28 No Response | Cycle 20, Day 28 Complete Response | Cycle 20, Day 28 Partial Response | Cycle 20, Day 28 Progressive Disease | Cycle 20, Day 28 No Response | Final Visit Complete Response | Final Visit Partial Response | Final Visit Progressive Disease | Final Visit No Response | |
All Ruxolitinib-Naïve Participants | 0 | 0 | 0 | 0 | 10.5 | 68.4 | 0 | 21.1 | 12.5 | 68.8 | 0 | 18.8 | 18.2 | 54.5 | 0 | 27.3 | 16.7 | 66.7 | 0 | 16.7 | 16.7 | 66.7 | 0 | 16.7 | 25.0 | 25.0 | 0 | 50.0 | 0 | 100 | 0 | 0 | 14.3 | 21.4 | 0 | 64.3 |
"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)
Intervention | Percentage of Participants (Number) | |||||||||||||||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline Complete Response | Baseline Partial Response | Baseline Progressive Disease | Baseline No Response | Cycle 3, Day 28 Complete Response | Cycle 3, Day 28 Partial Response | Cycle 3, Day 28 Progressive Disease | Cycle 3, Day 28 No Response | Cycle 5, Day 28 Complete Response | Cycle 5, Day 28 Partial Response | Cycle 5, Day 28 Progressive Disease | Cycle 5, Day 28 No Response | Cycle 8, Day 28 (Week 32) Complete Response | Cycle 8, Day 28 (Week 32) Partial Response | Cycle 8, Day 28 (Week 32) Progressive Disease | Cycle 8, Day 28 (Week 32) No Response | Cycle 11 Day 28 Complete Response | Cycle 11 Day 28 Partial Response | Cycle 11 Day 28 Progressive Disease | Cycle 11 Day 28 No Response | Cycle 12 Day 28 Complete Response | Cycle 12 Day 28 Partial Response | Cycle 12 Day 28 Progressive Disease | Cycle 12 Day 28 No Response | Cycle 14, Day 28 Complete Response | Cycle 14, Day 28 Partial Response | Cycle 14, Day 28 Progressive Disease | Cycle 14, Day 28 No Response | Cycle 17, Day 28 Complete Response | Cycle 17, Day 28 Partial Response | Cycle 17, Day 28 Progressive Disease | Cycle 17, Day 28 No Response | Cycle 20, Day 28 Complete Response | Cycle 20, Day 28 Partial Response | Cycle 20, Day 28 Progressive Disease | Cycle 20, Day 28 No Response | Final Visit Complete Response | Final Visit Partial Response | Final Visit Progressive Disease | Final Visit No Response | |
All Ruxolitinib-Resistant or Intolerant Participants | 0 | 0 | 0 | 0 | 28.6 | 42.9 | 0 | 28.6 | 0 | 66.7 | 0 | 33.3 | 20.0 | 40.0 | 0 | 40.0 | 0 | 50.0 | 0 | 50.0 | 0 | 100 | 0 | 0 | 0 | 50 | 0 | 50.0 | 0 | 0 | 0 | 100 | 0 | 0 | 0 | 100 | 0 | 25.0 | 0 | 75.0 |
"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)
Intervention | Percentage of Participants (Number) | |||
---|---|---|---|---|
After 12 Weeks from Week 32 HCT Control | After 12 Weeks from Week 32 Composite Response | After 12 Weeks from Week 32 ELN Response | After 12 Weeks from Week 32 Complete Hematologic Response | |
All Ruxolitinib-Naïve Participants | 55.6 | 0 | 60 | 44.4 |
All Ruxolitinib-Resistant or Intolerant Participants | 75 | 0 | 60 | 50 |
"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)
Intervention | Percentage of Participants (Number) | |||||||||||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline Complete Response | Baseline Partial Response | Baseline Progressive Disease | Baseline No Response | Cycle 3, Day 28 Complete Response | Cycle 3, Day 28 Partial Response | Cycle 3, Day 28 Progressive Disease | Cycle 3, Day 28 No Response | Cycle 5, Day 28 Complete Response | Cycle 5, Day 28 Partial Response | Cycle 5, Day 28 Progressive Disease | Cycle 5, Day 28 No Response | Cycle 8, Day 28 (Week 32) Complete Response | Cycle 8, Day 28 (Week 32) Partial Response | Cycle 8, Day 28 (Week 32) Progressive Disease | Cycle 8, Day 28 (Week 32) No Response | Cycle 11, Day 28 Complete Response | Cycle 11, Day 28 Partial Response | Cycle 11, Day 28 Progressive Disease | Cycle 11, Day 28 No Response | Cycle 12, Day 28 Complete Response | Cycle 12, Day 28 Partial Response | Cycle 12, Day 28 Progressive Disease | Cycle 12, Day 28 No Response | Cycle 14, Day 28 Complete Response | Cycle 14, Day 28 Partial Response | Cycle 14, Day 28 Progressive Disease | Cycle 14, Day 28 No Response | Cycle 17, Day 28 Complete Response | Cycle 17, Day 28 Partial Response | Cycle 17, Day 28 Progressive Disease | Cycle 17, Day 28 No Response | Final Visit (28 Days post-last dose) Complete Response | Final Visit (28 Days post-last dose) Partial Response | Final Visit (28 Days post-last dose) Progressive Disease | Final Visit (28 Days post-last dose) No Response | |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 0 | 0 | 0 | 0 | 33.3 | 50.0 | 0 | 16.7 | 0 | 80.0 | 0 | 20.0 | 25.0 | 50.0 | 0 | 25.0 | 0 | 100.0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 100 | 0 | 0 | 0 | 0 | 0 | 100 | 0 | 33.3 | 0 | 66.7 |
"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)
Intervention | Percentage of Participants (Number) | |||||||||||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline Complete Response | Baseline Partial Response | Baseline Progressive Disease | Baseline No Response | Cycle 3, Day 28 Complete Response | Cycle 3, Day 28 Partial Response | Cycle 3, Day 28 Progressive Disease | Cycle 3, Day 28 No Response | Cycle 5, Day 28 Complete Response | Cycle 5, Day 28 Partial Response | Cycle 5, Day 28 Progressive Disease | Cycle 5, Day 28 No Response | Cycle 8, Day 28 (Week 32) Complete Response | Cycle 8, Day 28 (Week 32) Partial Response | Cycle 8, Day 28 (Week 32) Progressive Disease | Cycle 8, Day 28 (Week 32) No Response | Cycle 11, Day 28 Complete Response | Cycle 11, Day 28 Partial Response | Cycle 11, Day 28 Progressive Disease | Cycle 11, Day 28 No Response | Cycle 14, Day 28 Complete Response | Cycle 14, Day 28 Partial Response | Cycle 14, Day 28 Progressive Disease | Cycle 14, Day 28 No Response | Cycle 17, Day 28 Complete Response | Cycle 17, Day 28 Partial Response | Cycle 17, Day 28 Progressive Disease | Cycle 17, Day 28 No Response | Cycle 20, Day 28 Complete Response | Cycle 20, Day 28 Partial Response | Cycle 20, Day 28 Progressive Disease | Cycle 20, Day 28 No Response | Final Visit (28 Days post-last dose) Complete Response | Final Visit (28 Days post-last dose) Partial Response | Final Visit (28 Days post-last dose) Progressive Disease | Final Visit (28 Days post-last dose) No Response | |
Ruxolitinib-naïve Participants With Splenomegaly | 0 | 0 | 0 | 0 | 0 | 73.3 | 0 | 26.7 | 0 | 76.9 | 0 | 23.1 | 0 | 66.7 | 0 | 33.3 | 0 | 80.0 | 0 | 20.0 | 0 | 80 | 0 | 20 | 0 | 33.3 | 0 | 66.7 | 0 | 100 | 0 | 0 | 0 | 20.0 | 0 | 80 |
"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)
Intervention | Percentage of Participants (Number) | |||
---|---|---|---|---|
HCT Control | Composite Response | ELN Response | Complete Hematologic Response | |
Ruxolitinib-Naïve Participants With Splenomegaly | 42.9 | 0 | 50 | 28.6 |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 100 | 0 | 75 | 66.7 |
"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)
Intervention | Percentage of Participants (Number) | |||||||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline Complete Response | Baseline Partial Response | Baseline Progressive Disease | Baseline No Response | Cycle 3, Day 28 Complete Response | Cycle 3, Day 28 Partial Response | Cycle 3, Day 28 Progressive Disease | Cycle 3, Day 28 No Response | Cycle 5 Day 28 Complete Response | Cycle 5 Day 28 Partial Response | Cycle 5 Day 28 Progressive Disease | Cycle 5 Day 28 No Response | Cycle 8, Day 28 (Week 32) Complete Response | Cycle 8, Day 28 (Week 32) Partial Response | Cycle 8, Day 28 (Week 32) Progressive Disease | Cycle 8, Day 28 (Week 32) No Response | Cycle 11, day 28 Complete Response | Cycle 11, day 28 Partial Response | Cycle 11, day 28 Progressive Disease | Cycle 11, day 28 No Response | Cycle 14, Day 28 Complete Response | Cycle 14, Day 28 Partial Response | Cycle 14, Day 28 Progressive Disease | Cycle 14, Day 28 No Response | Cycle 17, Day 28 Complete Response | Cycle 17, Day 28 Partial Response | Cycle 17, Day 28 Progressive Disease | Cycle 17, Day 28 No Response | Final (28 Days post-last dose) Complete Response | Final (28 Days post-last dose) Partial Response | Final (28 Days post-last dose) Progressive Disease | Final (28 Days post-last dose) No Response | |
Ruxolitinib-naïve Participants Without Splenomegaly | 0 | 0 | 0 | 0 | 50 | 50 | 0 | 0 | 66.7 | 33.3 | 0 | 0 | 100 | 0 | 0 | 0 | 100 | 0 | 0 | 0 | 100 | 0 | 0 | 0 | 100 | 0 | 0 | 0 | 50.0 | 25.0 | 0.0 | 25.0 |
"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)
Intervention | Percentage of Participants (Number) | |||||||||||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Baseline Complete Response | Baseline Partial Response | Baseline Progressive Disease | Baseline No Response | Cycle 3, Day 28 Complete Response | Cycle 3, Day 28 Partial Response | Cycle 3, Day 28 Progressive Disease | Cycle 3, Day 28 No Response | Cycle 5 Day 28 Complete Response | Cycle 5 Day 28 Partial Response | Cycle 5 Day 28 Progressive Disease | Cycle 5 Day 28 No Response | Cycle 8, Day 28 (Week 32) Complete Response | Cycle 8, Day 28 (Week 32) Partial Response | Cycle 8, Day 28 (Week 32) Progressive Disease | Cycle 8, Day 28 (Week 32) No Response | Cycle 11, day 28 Complete Response | Cycle 11, day 28 Partial Response | Cycle 11, day 28 Progressive Disease | Cycle 11, day 28 No Response | Cycle 14, Day 28 Complete Response | Cycle 14, Day 28 Partial Response | Cycle 14, Day 28 Progressive Disease | Cycle 14, Day 28 No Response | Cycle 17, Day 28 Complete Response | Cycle 17, Day 28 Partial Response | Cycle 17, Day 28 Progressive Disease | Cycle 17, Day 28 No Response | Cycle 20, Day 28 Complete Response | Cycle 20, Day 28 Partial Response | Cycle 20, Day 28 Progressive Disease | Cycle 20, Day 28 No Response | Final (28 Days post-last dose) Complete Response | Final (28 Days post-last dose) Partial Response | Final (28 Days post-last dose) Progressive Disease | Final (28 Days post-last dose) No Response | |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 100 | 0 | 0 | 0 | 100 | 0 | 0 | 0 | 100 | 0 | 0 | 0 | 100 | 0 | 0 | 0 | 100 | 0 | 0 | 0 | 100 | 0 | 0 | 0 | 100 | 0 | 0 | 0 | 100 |
"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)
Intervention | Percentage of Participants (Number) | |||
---|---|---|---|---|
HCT Control | Composite Response | ELN Response | Complete Hematologic Response | |
Ruxolitinib-Naïve Participants Without Splenomegaly | 100 | 0 | 100 | 100 |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 0 | 0 | 0 | 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)
Intervention | Participants (Number) | |
---|---|---|
Baseline | Grade 3-5 AE | |
Ruxolitinib-Naïve Participants With Splenomegaly | 0 | 5 |
Ruxolitinib-Naïve Participants Without Splenomegaly | 0 | 2 |
Ruxolitinib-Resistant or Intolerant Participants With Splenomegaly | 0 | 3 |
Ruxolitinib-Resistant or Intolerant Participants Without Splenomegaly | 0 | 0 |
1 review available for 4-aminobenzoic acid and Erythremia
Article | Year |
---|---|
Novel therapeutic approaches in polycythemia vera.
Topics: Clinical Trials, Phase II as Topic; Clinical Trials, Phase III as Topic; Half-Life; Humans; Hydroxyu | 2018 |
1 trial available for 4-aminobenzoic acid and Erythremia
Article | Year |
---|---|
The MDM2 antagonist idasanutlin in patients with polycythemia vera: results from a single-arm phase 2 study.
Topics: Humans; Hydroxyurea; Nausea; para-Aminobenzoates; Polycythemia Vera; Proto-Oncogene Proteins c-mdm2; | 2022 |
2 other studies available for 4-aminobenzoic acid and Erythremia
Article | Year |
---|---|
Drug development challenges in polycythemia vera.
Topics: Drug Development; Humans; para-Aminobenzoates; Polycythemia Vera; Pyrrolidines | 2019 |
Transient expansion of TP53 mutated clones in polycythemia vera patients treated with idasanutlin.
Topics: Clone Cells; Humans; para-Aminobenzoates; Polycythemia Vera; Proto-Oncogene Proteins c-mdm2; Pyrroli | 2020 |