Trial · NCT01243944
Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 (INCB018424) Tablets Versus Best Available Care: (The RESPONSE Trial)
NCT01243944, Phase 3, Completed, Incyte Corporation; results first posted 2015-03-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 (INCB018424) Tablets Versus Best Available Care: (The RESPONSE Trial). Trials, record NCT01243944. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01243944
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 (INCB018424) Tablets Versus Best Available Care: (The RESPONSE Trial)
- NCT number
- NCT01243944
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 222 participants, actual
- Lead sponsor
- Incyte Corporation
- Conditions
- Polycythemia Vera
- Interventions
- ruxolitinib tablets, Best Available Therapy (BAT)
- First posted
- 2010-11-19
- Last updated
- 2019-03-06
- Results first posted
- 2015-03-06
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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 | 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. |
| Secondary | 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. |
| Secondary | 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. |
| Secondary | 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. |
| Secondary | 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. |
| Secondary | 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. |
| Secondary | 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. |
| Secondary | 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. |
| Secondary | 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. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | The Percentage of Participants Achieving a Primary Response at Week 32 | 32 Weeks | Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. | percentage of participants | NUMBER |
| SECONDARY | The Percentage of Participants Achieving a Durable Primary Response at Week 48 | 48 Weeks | Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. | percentage of participants | NUMBER |
| SECONDARY | The Percentage of Participants Achieving Complete Hematological Remission at Week 32 | 32 Weeks | Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. | percentage of participants | NUMBER |
| SECONDARY | The Percentage of Participants Who Achieved a Durable Complete Hematological Remission at Week 48 | 48 Weeks | Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. | percentage of participants | NUMBER |
| SECONDARY | The Percentage of Participants Who Achieved a Durable Hematocrit Control at Week 48 | 48 Weeks | Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. | percentage of participants | NUMBER |
| SECONDARY | The Percentage of Participants Who Achieved Durable Spleen Volume Reduction at Week 48 | 48 Weeks | Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. | percentage of participants | NUMBER |
| SECONDARY | 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 | 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. | probability | NUMBER |
| SECONDARY | The Percentage of Participants Who Achieved Overall Clinicohematologic Response at Week 32 | 32 Weeks | Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. | percentage of participants | NUMBER |
| SECONDARY | The Percentage of Participants Achieving a Durable Complete or Partial Clinicohematologic Response at Week 48 | 48 Weeks | Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. | percentage of participants | NUMBER |
| SECONDARY | 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 | 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. | probability | NUMBER |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group100
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| The Percentage of Participants Achieving a Primary Response at Week 32 | Ruxolitinib | 22.7 | 95% Confidence Interval 15.3 to 31.7 | percentage of participants | |
| The Percentage of Participants Achieving a Primary Response at Week 32 | Best Available Therapy | 0.9 | 95% Confidence Interval 0.0 to 4.9 | percentage of participants | |
| The Percentage of Participants Achieving a Durable Primary Response at Week 48 | Ruxolitinib | 20.0 | 95% Confidence Interval 13.0 to 28.7 | percentage of participants | |
| The Percentage of Participants Achieving a Durable Primary Response at Week 48 | Best Available Therapy | 0.9 | 95% Confidence Interval 0.0 to 4.9 | percentage of participants | |
| The Percentage of Participants Achieving Complete Hematological Remission at Week 32 | Ruxolitinib | 23.6 | 95% Confidence Interval 16.1 to 32.7 | percentage of participants | |
| The Percentage of Participants Achieving Complete Hematological Remission at Week 32 | Best Available Therapy | 8.0 | 95% Confidence Interval 3.7 to 14.7 | percentage of participants | |
| The Percentage of Participants Who Achieved a Durable Complete Hematological Remission at Week 48 | Ruxolitinib | 20.9 | 95% Confidence Interval 13.7 to 29.7 | percentage of participants | |
| The Percentage of Participants Who Achieved a Durable Complete Hematological Remission at Week 48 | Best Available Therapy | 0.9 | 95% Confidence Interval 0.0 to 4.9 | percentage of participants | |
| The Percentage of Participants Who Achieved a Durable Hematocrit Control at Week 48 | Ruxolitinib | 54.5 | 95% Confidence Interval 44.8 to 64.1 | percentage of participants | |
| The Percentage of Participants Who Achieved a Durable Hematocrit Control at Week 48 | Best Available Therapy | 1.8 | 95% Confidence Interval 0.2 to 6.3 | percentage of participants |
1 to 10 of 100Source ClinicalTrials.gov
See all 100 results by groupParticipant flow10
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ruxolitinib | 110 | |
| Overall Study | STARTED | Best Available Therapy | 112 | |
| Overall Study | Treated | Ruxolitinib | 110 | |
| Overall Study | Treated | Best Available Therapy | 111 | |
| Overall Study | Followed for Survival | Ruxolitinib | 22 | |
| Overall Study | Followed for Survival | Best Available Therapy | 21 | |
| Overall Study | COMPLETED | Ruxolitinib | 65 | |
| Overall Study | COMPLETED | Best Available Therapy | 61 | |
| Overall Study | NOT COMPLETED | Ruxolitinib | 45 | |
| Overall Study | NOT COMPLETED | Best Available Therapy | 51 |
Source ClinicalTrials.gov
Adverse events423
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Ruxolitinib - Through Week 32 | 20 | 110 | |
| other | Anaemia | Blood and lymphatic system disorders | Best Available Therapy - Through Week 32 | 3 | 111 | |
| other | Anaemia | Blood and lymphatic system disorders | Ruxolitinib - Week 256 Close Out | 38 | 110 | |
| other | Headache | Nervous system disorders | Ruxolitinib - Through Week 32 | 18 | 110 | |
| other | Headache | Nervous system disorders | Best Available Therapy - Through Week 32 | 21 | 111 | |
| other | Headache | Nervous system disorders | Ruxolitinib - Week 256 Close Out | 25 | 110 | |
| other | Diarrhoea | Gastrointestinal disorders | Ruxolitinib - Through Week 32 | 16 | 110 | |
| other | Diarrhoea | Gastrointestinal disorders | Best Available Therapy - Through Week 32 | 8 | 111 | |
| other | Diarrhoea | Gastrointestinal disorders | Ruxolitinib - Week 256 Close Out | 30 | 110 | |
| other | Fatigue | General disorders | Ruxolitinib - Through Week 32 | 16 | 110 |
1 to 10 of 423Source ClinicalTrials.gov
See all 423 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ruxolitinib - Through Week 32 | ||
| Total, all-cause mortality | Best Available Therapy - Through Week 32 | ||
| Total, all-cause mortality | Ruxolitinib - Week 256 Close Out | ||
| Total, other adverse events | Ruxolitinib - Through Week 32 | 105 | 110 |
| Total, other adverse events | Best Available Therapy - Through Week 32 | 104 | 111 |
| Total, other adverse events | Ruxolitinib - Week 256 Close Out | 107 | 110 |
| Total, serious adverse events | Ruxolitinib - Through Week 32 | 15 | 110 |
| Total, serious adverse events | Best Available Therapy - Through Week 32 | 10 | 111 |
| Total, serious adverse events | Ruxolitinib - Week 256 Close Out | 44 | 110 |
Source ClinicalTrials.gov
Papers6
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
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