Clinical Trial: Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy.
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: Randomized, Open Label, Multicenter Phase IIIb Study Evaluating the Efficacy and Safety of Ruxolitinib Versus Best Available Therapy in Patients With Polycythemia Vera Who
Brief Summary: This trial will compare the efficacy and safety of ruxolitinib to Best Available Therapy (BAT) in patients with polycythemia vera (PV) who are hyroxyurea (HU) resistant or intolerant and do not have a palpable spleen.
Detailed Summary:
Sponsor: Novartis Pharmaceuticals
Current Primary Outcome: Efficacy of ruxolitinib to Best Available Therapy (BAT) as assessed by Hct control at Week 28 [ Time Frame: Week 28 ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Efficacy of ruxolitinib to BAT as assessed by peripheral blood count remission at Week 28. [ Time Frame: Week 28 ]Proportion of patients achieving a peripheral blood count remission at Week 28 as defined by: Hct < 45% at Week 16 and maintained until Week 28, and WBC < 10 x10E9/L at Week 16 and maintained until Week 28, and Platelets ≤ 400 x 10E9/L at Week 16 and maintained until Week 28, and No phlebotomy from Week 4 to Week 28, with no more than one phlebotomy occurring post randomization and prior to the Week 4 visit.
- Efficacy of ruxolitinib to BAT as assessed by durable Hct control at Week 52 [ Time Frame: Week 52 ]Proportion of patients achieving a Hct control at Week 52 as defined by: Hct < 45% at Week 16 and maintained until Week 52, and no phlebotomy from Week 4 to Week 28, with no more than one phlebotomy occurring post randomization and prior to the Week 4 visit, and, in the BAT arm, no change in the treatment regimen or crossover to the ruxolitinib arm.
- Efficacy of ruxolitinib to BAT as assessed by durable peripheral blood count remission at Week 52. [ Time Frame: Week 52 ]Proportion of patients achieving a peripheral blood count remission at Week 52 as defined by: •Hct < 45% at Week 16 and maintained until Week 52, and WBC < 10 x10E9/L at Week 16 and maintained until Week 52, and Platelets ≤ 400 x 10E9/L at Week 16 and maintained until Week 52, and, no phlebotomy from Week 4 to Week 52, with no more than one phlebotomy occurring post randomization and prior to the Week 4 visit, and in the BAT arm, no change in the treatment regimen or crossover to the ruxolitinib arm.
- Change in ECOG status [ Time Frame: Week 28 ]Change in ECOG status from baseline to Week 28
- Change in spleen length [ Time Frame: Week 4, 8, 12, 16, 20, 24, 28, 40, 52/End of Treatment ]Change from Baseline in spleen length at each scheduled visit
- Efficacy of ruxolitinib to BAT as assessed by partial remission based on the ELN and IWG-MRT criteria at Week 28 [ Time Frame: Week 28 ]Proportion of patients achieving a partial remission at Week 28, based on the ELN and IWG-MRT criteria, as defined by: •MPN-SAF TSS score reduction of greater than or equal to 10 points from baseline to Week 16 and maintained until Week 28 (for patients with a baseline score of 20 or more), and •Hct < 45% at Week 16 and maintained until Week 28, and •No phlebotomy from Week 4 to Week 28, with no more than one phlebotomy occurring post randomization and prior to the Week 4 visit, and •WBC < 10 x10E9/L at Week 16 and maintained until Week 28, and •Platelets ≤ 400 x 10E9/L at Week 16 and maintained until Week 28, and •No palpable spleen at Week 16 and maintained until Week 28, and •No hemorrhagic or thrombotic events, and •No transformation into post-PV myelofibrosis, myelodysplastic syndrome, or acute leukemia
- Efficacy of ruxolitinib to BAT as assessed by durable partial remission based on the ELN and IWG-MRT criteria at Week 52. [ Time Frame: Week 52 ]Proportion of patients who achieved partial remission at Week 52, based on the ELN and IWG-MRT criteria, as defined by: •MPN-SAF TSS score reduction of greater than or equal to 10 points from baseline to Week 16 and maintained until Week 52 (for patients with a baseline score of 20 or more), and •Hct < 45% at Week 16 and maintained until Week 52, and •No phlebotomy from Week 4 to Week 52, with no more than one phlebotomy occurring post randomization and prior to the Week 4 visit, and •WBC < 10 x10E9/L at Week 16 and maintained until Week 52, and •Platelets ≤ 400 x 10E9/L at Week 16 and maintained until Week 52, and •No palpable spleen at Week 16 and maintained until Week 52, and •No hemorrhagic or thrombotic events, and •No transformation into post-PV myelofibrosis, myelodysplastic syndrome (IWG-MRT criteria) or acute leukemia (WHO criteria), and •In the BAT arm, no change in the treatment regimen or crossover to the ruxolitinib arm.
- Efficacy of BAT patients after they cross over to ruxolitinib [ Time Frame: +4, +8, +12, +16, +20, +24 weeks after crossover ]Among patients randomized to BAT who cross over to ruxolitinib, proportion of patients achieving Hct < 45% at each visit after cross over
- Changes in MPN-SAF TSS [ Time Frame: Week 4, 8, 16, 28, 40, 52/End of Treatment ]Proportion of patients achieving at least a 50% reduction from baseline in MPN-SAF TSS score at each visit where it is measured.
- Resolution of disease related symptoms in MPN-SAF TSS at Week 28. [ Time Frame: Week 28 ]Proportion of patients achieving a resolution of disease related symptoms in MPN-SAF TSS at Week 28 defined as: • MPN-SAF TSS score reduction of greater than or equal to 10 points from baseline to Week 16 and maintained until Week 28 (for patients with a baseline score of 20 or mor
Original Secondary Outcome: Same as current
Information By: Novartis
Dates:
Date Received: January 14, 2014
Date Started: March 25, 2014
Date Completion: April 20, 2020
Last Updated: March 7, 2017
Last Verified: March 2017
