Polycythemia Vera
Conditions
Keywords
INCB018424
Brief summary
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).
Interventions
DRUGruxolitinib tablets
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.
Sponsors
Novartis Pharmaceuticals
Incyte Corporation
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participants diagnosed with PV for at least 24 weeks prior to screening according to the 2008 World Health Organization criteria * Participants resistant to or intolerant of hydroxyurea * Participants with a phlebotomy requirement * Participants with splenomegaly (palpable or non-palpable) and a spleen volume, as measured by MRI (or CT in applicable participants ), of greater than or equal to 450 cubic centimeters * Participants with an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
Exclusion criteria
* Women who are pregnant or nursing * Participants with inadequate liver or renal function * Participants with significant bacterial, fungal, parasitic, or viral infection requiring treatment * Participants with an active malignancy within the past 5 years, excluding specific skin cancers * Participants with known active hepatitis or HIV positivity * Participants who have previously received treatment with a JAK inhibitor * Participants being treated with any investigational agent
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Percentage of Participants Achieving a Primary Response at Week 32 | 32 Weeks | 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). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The Percentage of Participants Achieving a Durable Primary Response at Week 48 | 48 Weeks | 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. |
| The Percentage of Participants Achieving Complete Hematological Remission at Week 32 | 32 Weeks | 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. |
| The Percentage of Participants Who Achieved a Durable Hematocrit Control at Week 48 | 48 Weeks | 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. |
| The Percentage of Participants Who Achieved Durable Spleen Volume Reduction at Week 48 | 48 Weeks | 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. |
| Estimated Duration of the Primary Response | Through study completion, analysis was conducted when all participants had completed the Week 80 visit or discontinued the study | 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. |
| The Percentage of Participants Who Achieved a Durable Complete Hematological Remission at Week 48 | 48 Weeks | 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. |
| The Percentage of Participants Achieving a Durable Complete or Partial Clinicohematologic Response at Week 48 | 48 Weeks | 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. |
| Estimated Duration of the Complete Hematological Remission | Through study completion, analysis was conducted when all participants had completed the Week 80 visit or discontinued the study | 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. |
| Duration of the Absence of Phlebotomy Eligibility | 256 Weeks | 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. |
| Duration of Reduction in Spleen Volume | 256 Weeks | 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. |
| Duration of The Overall Clinicohematologic Response | 256 Weeks | 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. |
| The Percentage of Participants Who Achieved Overall Clinicohematologic Response at Week 32 | 32 Weeks | 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. |
Countries
Argentina, Australia, Belgium, Canada, China, France, Germany, Hungary, Italy, Japan, Netherlands, Russia, South Korea, Spain, Thailand, Turkey (Türkiye), United Kingdom, United States
Participant flow
Recruitment details
Participants may be treated beyond 256 weeks due to the 14 day visit window.
Participants by arm
| Arm | Count |
|---|---|
| Ruxolitinib Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy | 110 |
| Best Available Therapy 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. | 112 |
| Total | 222 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 15 | 13 |
| Overall Study | Death | 2 | 3 |
| Overall Study | Disease progression | 12 | 11 |
| Overall Study | Lost to Follow-up | 2 | 1 |
| Overall Study | Non-compliance with study treatment | 1 | 0 |
| Overall Study | Participant decision | 10 | 15 |
| Overall Study | Physician Decision | 2 | 7 |
| Overall Study | Protocol deviation | 1 | 1 |
Baseline characteristics
| Characteristic | Ruxolitinib | Best Available Therapy | Total |
|---|---|---|---|
| Age, Continuous | 61.1 years STANDARD_DEVIATION 10.48 | 59.1 years STANDARD_DEVIATION 10.25 | 60.1 years STANDARD_DEVIATION 10.39 |
| Age, Customized < 60 years | 49 participants | 54 participants | 103 participants |
| Age, Customized ≥ 60 years | 61 participants | 58 participants | 119 participants |
| Body Mass Index (BMI) | 25.8 kg/m^2 STANDARD_DEVIATION 3.82 | 26.1 kg/m^2 STANDARD_DEVIATION 4.24 | 25.9 kg/m^2 STANDARD_DEVIATION 4.03 |
| ECOG performance status 0 | 76 participants | 77 participants | 153 participants |
| ECOG performance status 1 | 31 participants | 34 participants | 65 participants |
| ECOG performance status 2 | 3 participants | 1 participants | 4 participants |
| Height | 172.2 cm STANDARD_DEVIATION 8.44 | 173.3 cm STANDARD_DEVIATION 9.79 | 172.8 cm STANDARD_DEVIATION 9.14 |
| Race/Ethnicity, Customized Asian | 11 participants | 16 participants | 27 participants |
| Race/Ethnicity, Customized Black/African American | 1 participants | 0 participants | 1 participants |
| Race/Ethnicity, Customized White/Caucasian | 98 participants | 96 participants | 194 participants |
| Sex: Female, Male Female | 44 Participants | 32 Participants | 76 Participants |
| Sex: Female, Male Male | 66 Participants | 80 Participants | 146 Participants |
| Weight | 76.6 kg STANDARD_DEVIATION 14.09 | 79.0 kg STANDARD_DEVIATION 17.34 | 77.8 kg STANDARD_DEVIATION 15.83 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 105 / 110 | 104 / 111 | 107 / 110 |
| serious Total, serious adverse events | 15 / 110 | 10 / 111 | 44 / 110 |
Outcome results
Primary
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
Population: Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ruxolitinib | The Percentage of Participants Achieving a Primary Response at Week 32 | 22.7 percentage of participants |
| Best Available Therapy | The Percentage of Participants Achieving a Primary Response at Week 32 | 0.9 percentage of participants |
p-value: <0.000195% CI: [5.04, 1337]Exact Cochran-Mantel-Haenszel
Secondary
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
Population: Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. Duration of the reduction in spleen volume was pre-specified in the protocol to be analyzed for the ruxolitinib arm only considering the study design which allowed crossover for the BAT arm beginning at Week 32.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Ruxolitinib | Duration of Reduction in Spleen Volume | 16 weeks | 1.00 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 32 weeks | 1.00 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 48 weeks | 1.00 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 64 weeks | 1.00 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 80 weeks | 1.00 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 96 weeks | 0.98 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 112 weeks | 0.95 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 128 weeks | 0.95 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 144 weeks | 0.95 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 160 weeks | 0.93 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 176 weeks | 0.93 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 192 weeks | 0.93 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 208 weeks | 0.87 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 224 weeks | 0.72 probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 240 weeks | NA probability |
| Ruxolitinib | Duration of Reduction in Spleen Volume | 256 weeks | NA probability |
Secondary
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
Population: Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. Duration of the absence of phlebotomy eligibility was pre-specified in the protocol to be analyzed for the ruxolitinib arm only considering the study design which allowed crossover for the BAT arm beginning at Week 32.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 224 weeks | 0.73 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 16 weeks | 1.00 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 32 weeks | 1.00 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 48 weeks | 0.97 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 64 weeks | 0.92 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 80 weeks | 0.91 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 96 weeks | 0.91 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 112 weeks | 0.87 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 128 weeks | 0.84 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 144 weeks | 0.84 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 160 weeks | 0.82 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 176 weeks | 0.79 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 192 weeks | 0.77 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 208 weeks | 0.73 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 240 weeks | 0.73 probability |
| Ruxolitinib | Duration of the Absence of Phlebotomy Eligibility | 256 weeks | 0.73 probability |
Secondary
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
Population: Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. Duration of the overall clinicohematologic response was pre-specified in the protocol to be analyzed for the ruxolitinib arm only considering the study design which allowed crossover for the BAT arm beginning at Week 32.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 208 weeks | 0.67 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 16 weeks | 1.00 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 32 weeks | 0.99 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 48 weeks | 0.96 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 64 weeks | 0.91 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 80 weeks | 0.88 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 96 weeks | 0.88 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 112 weeks | 0.85 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 128 weeks | 0.82 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 144 weeks | 0.82 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 160 weeks | 0.80 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 176 weeks | 0.75 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 192 weeks | 0.70 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 224 weeks | 0.67 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 240 weeks | 0.67 probability |
| Ruxolitinib | Duration of The Overall Clinicohematologic Response | 256 weeks | 0.67 probability |
Secondary
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
Population: Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. Duration of response was pre-specified in the protocol to be analyzed for the ruxolitinib arm only considering the study design which allowed crossover for the BAT arm beginning at Week 32.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 16 weeks | 1.00 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 32 weeks | 1.00 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 48 weeks | 0.88 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 64 weeks | 0.83 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 80 weeks | 0.74 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 96 weeks | 0.74 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 112 weeks | 0.69 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 128 weeks | 0.69 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 144 weeks | 0.65 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 160 weeks | 0.65 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 176 weeks | 0.55 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 192 weeks | 0.55 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 208 weeks | 0.55 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 224 weeks | 0.55 probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 240 weeks | NA probability |
| Ruxolitinib | Estimated Duration of the Complete Hematological Remission | 256 weeks | NA probability |
Secondary
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
Population: Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. Duration of response was pre-specified in the protocol to be analyzed for the ruxolitinib arm only considering the study design which allowed crossover for the BAT arm beginning at Week 32.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Ruxolitinib | Estimated Duration of the Primary Response | 48 weeks | 0.92 probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 64 weeks | 0.92 probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 80 weeks | 0.92 probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 96 weeks | 0.88 probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 112 weeks | 0.84 probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 128 weeks | 0.84 probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 144 weeks | 0.84 probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 160 weeks | 0.79 probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 176 weeks | 0.79 probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 192 weeks | 0.74 probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 208 weeks | 0.74 probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 224 weeks | 0.74 probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 240 weeks | NA probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 256 weeks | NA probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 16 weeks | 1.00 probability |
| Ruxolitinib | Estimated Duration of the Primary Response | 32 weeks | 1.00 probability |
Secondary
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
Population: Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Ruxolitinib | The Percentage of Participants Achieving a Durable Complete or Partial Clinicohematologic Response at Week 48 | Complete response rate | 7.3 percentage of participants |
| Ruxolitinib | The Percentage of Participants Achieving a Durable Complete or Partial Clinicohematologic Response at Week 48 | Partial response rate | 50.9 percentage of participants |
| Best Available Therapy | The Percentage of Participants Achieving a Durable Complete or Partial Clinicohematologic Response at Week 48 | Complete response rate | 0.9 percentage of participants |
| Best Available Therapy | The Percentage of Participants Achieving a Durable Complete or Partial Clinicohematologic Response at Week 48 | Partial response rate | 0.9 percentage of participants |
Secondary
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
Population: Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ruxolitinib | The Percentage of Participants Achieving a Durable Primary Response at Week 48 | 20.0 percentage of participants |
| Best Available Therapy | The Percentage of Participants Achieving a Durable Primary Response at Week 48 | 0.9 percentage of participants |
p-value: <0.000195% CI: [4.24, 1144]Exact Cochran-Mantel-Haenszel
Secondary
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
Population: Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ruxolitinib | The Percentage of Participants Achieving Complete Hematological Remission at Week 32 | 23.6 percentage of participants |
| Best Available Therapy | The Percentage of Participants Achieving Complete Hematological Remission at Week 32 | 8.0 percentage of participants |
p-value: 0.001695% CI: [1.5, 9.06]Exact Cochran-Mantel-Haenszel
Secondary
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
Population: Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ruxolitinib | The Percentage of Participants Who Achieved a Durable Complete Hematological Remission at Week 48 | 20.9 percentage of participants |
| Best Available Therapy | The Percentage of Participants Who Achieved a Durable Complete Hematological Remission at Week 48 | 0.9 percentage of participants |
Secondary
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
Population: Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ruxolitinib | The Percentage of Participants Who Achieved a Durable Hematocrit Control at Week 48 | 54.5 percentage of participants |
| Best Available Therapy | The Percentage of Participants Who Achieved a Durable Hematocrit Control at Week 48 | 1.8 percentage of participants |
Secondary
The Percentage of Participants Who Achieved Durable Spleen Volume Reduction at Week 48
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
Population: Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ruxolitinib | The Percentage of Participants Who Achieved Durable Spleen Volume Reduction at Week 48 | 37.3 percentage of participants |
| Best Available Therapy | The Percentage of Participants Who Achieved Durable Spleen Volume Reduction at Week 48 | 0.9 percentage of participants |
Secondary
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
Population: Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Ruxolitinib | The Percentage of Participants Who Achieved Overall Clinicohematologic Response at Week 32 | Complete response rate | 8.2 percentage of participants |
| Ruxolitinib | The Percentage of Participants Who Achieved Overall Clinicohematologic Response at Week 32 | Partial response rate | 54.5 percentage of participants |
| Best Available Therapy | The Percentage of Participants Who Achieved Overall Clinicohematologic Response at Week 32 | Complete response rate | 0.9 percentage of participants |
| Best Available Therapy | The Percentage of Participants Who Achieved Overall Clinicohematologic Response at Week 32 | Partial response rate | 18.8 percentage of participants |