This pivotal phase III trial (CINC424B2301) is designed to compare the efficacy and safety of ruxolitinib (INC424) to Best Available Therapy (BAT) in participants with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea (HU).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
222
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
The Percentage of Participants Achieving a Primary Response at Week 32
Primary response was defined as having achieved hematocrit control (the absence of phlebotomy eligibility beginning at the Week 8 visit and continuing through Week 32) and Spleen Volume Reduction (a greater than or equal to 35% reduction from baseline in spleen volume at Week 32).
Time frame: 32 Weeks
The Percentage of Participants Achieving a Durable Primary Response at Week 48
Durable Primary Response was defined as any participant who achieved the primary outcome measure and who maintained their response up to 48 weeks after randomization.
Time frame: 48 Weeks
The Percentage of Participants Achieving Complete Hematological Remission at Week 32
Complete Hematological Remission at Week 32 was defined as any participant who achieved hematocrit control with a platelet count less than or equal to 400 X 10\^9/L and a white blood cell count less than or equal to 10 X 10\^9/L.
Time frame: 32 Weeks
The Percentage of Participants Who Achieved a Durable Complete Hematological Remission at Week 48
Durable Complete Hematological Remission was defined as any participant who achieved Complete Hematological Remission at Week 32 and maintained their response up to 48 weeks after randomization.
Time frame: 48 Weeks
The Percentage of Participants Who Achieved a Durable Hematocrit Control at Week 48
Durable Hematocrit Control was defined as any participant who achieved phlebotomy eligibility independence from Week 8 to Week 32 and maintained hematocrit control up to 48 weeks after randomization.
Time frame: 48 Weeks
The Percentage of Participants Who Achieved Durable Spleen Volume Reduction at Week 48
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Birmingham, Alabama, United States
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Scottsdale, Arizona, United States
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Pomona, California, United States
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Bridgeport, Connecticut, United States
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...and 92 more locations
Durable Spleen Volume Reduction was defined as a participant who achieved at least 35% reduction from baseline in spleen volume at Week 32 and maintained that response 48 weeks after randomization.
Time frame: 48 Weeks
Estimated Duration of the Primary Response
Duration of the primary response is defined as the time from the first occurrence when both components of the primary endpoint are met until the date of the first documented disease progression (end of response). Kaplan-Meier estimates are provided for duration of primary response.
Time frame: Through study completion, analysis was conducted when all participants had completed the Week 80 visit or discontinued the study
The Percentage of Participants Who Achieved Overall Clinicohematologic Response at Week 32
Overall Clinicohematologic Response is defined as any participant who achieved a complete or partial clinicohematologic response per the European LeukemiaNet modified criteria for response in polycythemia vera (PV). A Complete Response (CR) is defined as: hematocrit control, spleen volume reduction at least 35% from baseline, platelet count less than or equal to 400 x 10(9)/L, and white blood cell count less than or equal to 10 x 10(9)/L. A Partial Response (PR) is defined as hematocrit control or response in all 3 of the other criteria.
Time frame: 32 Weeks
The Percentage of Participants Achieving a Durable Complete or Partial Clinicohematologic Response at Week 48
Durable Complete or Partial Clinicohematologic Response was defined as any participant who achieved complete or partial clinicohematologic response per the European LeukemiaNet modified criteria for response in polycythemia vera at Week 32 and maintained that response 48 weeks after randomization.
Time frame: 48 Weeks
Estimated Duration of the Complete Hematological Remission
Duration of the complete hematological remission is defined as the time from the first occurrence of complete hematological remission until the date of the first documented progression (end of response). Kaplan-Meier estimates are provided for duration of complete hematological remission.
Time frame: Through study completion, analysis was conducted when all participants had completed the Week 80 visit or discontinued the study
Duration of the Absence of Phlebotomy Eligibility
Duration of the absence of phlebotomy eligibility is defined as the time from the first occurrence of absence of phlebotomy eligibility until the date of the first documented progression.
Time frame: 256 Weeks
Duration of Reduction in Spleen Volume
Duration of spleen volume reduction is defined as the time from the first occurrence of a \>=35% reduction from baseline in spleen volume until the date of the first documented progression.
Time frame: 256 Weeks
Duration of The Overall Clinicohematologic Response
Duration of the overall clinicohematologic response was defined as the time from the first occurrence of complete response (CR) or partial response (PR) until the date of the first documented disease progression.
Time frame: 256 Weeks