Overview

This trial is active, not recruiting.

Condition polycythemia vera
Treatments best available therapy, ruxolitinib
Phase phase 3
Targets JAK, JAK1, JAK2
Sponsor Novartis Pharmaceuticals
Start date March 2014
End date April 2020
Trial size 149 participants
Trial identifier NCT02038036, CINC424B2401

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
52 patients- at a starting dose of 10 mg bid. Dose may be adjusted based on efficacy and safety parameters up to a maximum dose of 25 mg bid.
ruxolitinib
Supplied as tablets. Starting dose of 10 mg bid. Based on efficacy and safety parameters, dose may be increased or decreased. Maximum dose allowed is 25mg bid.
(Active Comparator)
52 patients - as selected by the investigator from: Hydroxyurea, IFN/PEG-IFN, popobroman, anagrelide, IMIDs, or observation
best available therapy BAT
Best Available Therapy as selected by the investigator from the below: 1. Hydroxyurea 2. IFN/PEG-IFN 3. Pipobroman 4. Anagrelide 5. IMIDs 6. Observation only

Primary Outcomes

Measure
Efficacy of ruxolitinib to Best Available Therapy (BAT) as assessed by Hct control at Week 28
time frame: Week 28

Secondary Outcomes

Measure
Efficacy of ruxolitinib to BAT as assessed by peripheral blood count remission at Week 28.
time frame: Week 28
Efficacy of ruxolitinib to BAT as assessed by durable Hct control at Week 52
time frame: Week 52
Efficacy of ruxolitinib to BAT as assessed by durable peripheral blood count remission at Week 52.
time frame: Week 52
Change in ECOG status
time frame: Week 28
Change in spleen length
time frame: Week 4, 8, 12, 16, 20, 24, 28, 40, 52/End of Treatment
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
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
Efficacy of BAT patients after they cross over to ruxolitinib
time frame: +4, +8, +12, +16, +20, +24 weeks after crossover
Changes in MPN-SAF TSS
time frame: Week 4, 8, 16, 28, 40, 52/End of Treatment
Resolution of disease related symptoms in MPN-SAF TSS at Week 28.
time frame: Week 28
Changes in patient reported outcomes
time frame: Week 4, 8, 16, 28, 40, 52/End of Treatment
Safety of ruxolitinib and Best Available Therapy
time frame: Week 28, 52/EoT
Safety of BAT patients after they crossover
time frame: +52/EoT

Eligibility Criteria

Male or female participants at least 18 years old.

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.

Additional Information

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 Are Hydroxyurea Resistant or Intolerant (Response 2)
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Novartis.