Phase 3
N=149
Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy.
Polycythemia Vera
Bottom Line
View on ClinicalTrials.gov: NCT02038036 ↗Enrolled (actual)
149
Serious AEs
31.9%
Results posted
Jul 2021
Primary outcome: Primary: Number of Participants Achieving Hematocrit (Hct) Control at Week 28 — 46; 14 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Best Available Therapy (Drug); Ruxolitinib (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Sep 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Achieving Hematocrit (Hct) Control at Week 28 |
46; 14 | — |
| SECONDARY Number of Participants Achieving a Complete Hematological Remission at Week 28 |
17; 4 | — |
| SECONDARY Number of Participants Achieving a Hematocrit (Hct) Control at Week 52 and Week 80 |
44; 5; 35; 2 | — |
| SECONDARY Number of Participants Achieving a Complete Hematological Remission at Week 52 and Week 80 |
17; 3; 18; 2 | — |
| SECONDARY Number of Participants With Phlebotomies Over Time |
12; 29; 7; 17; 4; 2 | — |
| SECONDARY Change From Baseline in Hematocrit (Hct) at Each Visit |
-0.65; 1.25; -1.22; 1.63; -2.33; 1.70 | — |
| SECONDARY Change From Baseline in Hematocrit (Hct) at Each Scheduled Visit After Crossover in Participants Randomized to BAT Who Cross Over to Ruxolitinib |
-2.44; -4.24; -5.73; -6.27; -5.76; -5.29 | — |
| SECONDARY Spleen Length by Visit |
0.00; 0.04; 0.00; 0.01; 0.00; 0.01 | — |
| SECONDARY Change From Baseline in Eastern Cooperative Oncology Group (ECOG) Performance Status to Week 28 |
49; 17; 2; 1; 0; 0 | — |
| SECONDARY Number of Participants Achieving a Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 28 |
7; 0 | — |
| SECONDARY Number of Participants Who Achieved Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 52 and Week 80 |
5; 0; 4; 0 | — |
| SECONDARY Number of Participants Achieving a Hematocrit (Hct) Control at Week 104, Week 156, Week 208 and Week 260. |
9; 25; 9; 21; 7; 18 | — |
| SECONDARY Number of Participants Achieving a Complete Hematological Remission at Week 104, Week 156, Week 208 and Week 260 |
15; 19; 11; 9 | — |
| SECONDARY Number of Participants Who Achieved Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 104, Week 156, Week 208 and Week 260. |
4; 9; 4; 0 | — |
| SECONDARY Number of Participants With Transformation Free Survival Events |
4; 3 | — |
| SECONDARY Number of Participants With Overall Survival (OS) Events |
3; 6 | — |
| SECONDARY Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) |
-8.43; 0.40; -9.86; 1.37; -9.14; 1.41 | — |
| SECONDARY Change From Baseline in Total Scores of MPN-SAF by Visit in Patients From BAT Group Who Cross Over to Ruxolitinib After Crossover |
-8.00; -9.76; -9.40; -9.15; -8.46; -8.58 | — |
| SECONDARY Change From Baseline in Score as Per European Quality of Life 5-Dimension 5-level (EQ-5D-5L) Questionnaire |
4.24; 0.04; 7.62; -2.73; 6.35; -3.12 | — |
| SECONDARY Change From Baseline in EQ-5D-5L VAS, by Visit in Patients From BAT Group Who Cross Over to Ruxolitinib After Crossover |
4.54; 4.62; 6.58; 6.38; 5.26; 4.71 | — |
| SECONDARY Change From Baseline in Work Productivity and Activity Impairment (WPAI) Questionnaire |
-5.50; -0.40; -4.88; -4.35; 4.50; 4.79 | — |
| SECONDARY Change From Baseline in Work Productivity and Activity Impairment Questionnaire (WPAI), by Visit in Patients From BAT Group Who Cross Over to Ruxolitinib After Crossover |
-7.45; -3.90; -2.66; -1.67; 7.06; 1.12 | — |
| SECONDARY Patient Global Impression of Change (PGIC) |
10; 0; 22; 8; 21; 7 | — |
| SECONDARY Summary of Patient Global Impression of Change (PGIC), by Visit in Patients From BAT Group Who Cross Over to Ruxolitinib After Crossover |
10; 13; 13; 16; 0; 0 | — |
| SECONDARY Number of Participants Developing Thrombosis |
0; 0 | — |
Summary
This study compared the efficacy and safety of ruxolitinib to Best Available Therapy (BAT) in patients with polycythemia vera (PV) who were hydroxyurea (HU) resistant or intolerant and did not have a palpable spleen.
Eligibility Criteria
Inclusion Criteria
Confirmed diagnosis of PV according to the 2008 World Health Organization criteria, Non-palpable spleen, Phlebotomy dependent, Resistant to or intolerant of hydroxyurea, ECOG performance status of 0, 1 or 2.
Exclusion Criteria
Inadequate liver or renal function, Significant bacterial, fungal, parasitic, or viral infection requiring treatment, Active malignancy within the past 5 years, excluding specific skin cancers, Previously received treatment with a JAK inhibitor, Being treated with any investigational agent, Women who are pregnant or nursing.
Other inclusion/exclusion criteria apply.
Data sourced from ClinicalTrials.gov (NCT02038036). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.