Trial · NCT02038036
Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy.
NCT02038036, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2021-07-20
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy. Trials, record NCT02038036. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02038036
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy.
- NCT number
- NCT02038036
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 149 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Polycythemia Vera
- Interventions
- Best Available Therapy, Ruxolitinib
- First posted
- 2014-01-16
- Last updated
- 2021-07-20
- Results first posted
- 2021-07-20
Source ClinicalTrials.gov
Registered outcomes25
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants Achieving Hematocrit (Hct) Control at Week 28 | Week 28 | Proportion of patients achieving Hct control at Week 28 was defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 28, with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8. Phlebotomy eligibility was defined by: * Confirmed Hct \> 45% that is at least 3 percentage points higher than the Hct obtained at Baseline Or * Confirmed Hct \> 48% The confirmation occurred 2 to 14 days subsequent to the initial observation. |
| Secondary | Number of Participants Achieving a Complete Hematological Remission at Week 28 | Week 28 | Proportion of patients achieving a complete hematological remission at Week 28 was defined by: * Hct control at Week 28 defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 28, with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8, and * WBC \< 10 x109/L at Week 28, and * Platelets ≤ 400 x 109/L at Week 28 |
| Secondary | Number of Participants Achieving a Hematocrit (Hct) Control at Week 52 and Week 80 | Week 52 and 80 | Proportion of patients achieving a Hct control at Week 52 was defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 52, and no more than one phlebotomy eligibility occurring post randomization and prior to Week 8 \- Endpoint for Week 80 was defined, similarly. |
| Secondary | Number of Participants Achieving a Complete Hematological Remission at Week 52 and Week 80 | Week 52 and 80 | Proportion of patients achieving a complete hematological remission at Week 52, was defined by: * Hct control at Week 52, as defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 52 with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8, and * White Blood Count (WBC) \< 10 x10\^9/L at Week 52, and * Platelets ≤ 400 x 10\^9/L at Week 52 * Endpoint for Week 80 was defined, similarly. |
| Secondary | Number of Participants With Phlebotomies Over Time | Baseline to Week 260 | Phlebotomy eligibility was defined by Confirmed Hct \> 45% that is at least 3 percentage points higher than the Hct obtained at Baseline Or Confirmed Hct \> 48%. The confirmation occurred 2 to 14 days subsequent to the initial observation. |
| Secondary | Change From Baseline in Hematocrit (Hct) at Each Visit | Baseline, Week 4, 8, 12, 16, 20, 24, 28, 40, 52, 66, 80, 92, 104, 117, 130, 143, 156, 169, 182, 195, 208, 221, 234, 247 and 260 | Hematocrit is the volume percentage of red blood cells (RBC) in the blood. |
| Secondary | Change From Baseline in Hematocrit (Hct) at Each Scheduled Visit After Crossover in Participants Randomized to BAT Who Cross Over to Ruxolitinib | Baseline (last assessment before cross over), Week 4, 8, 12, 16, 20, 24, 28, 40, 52, 64, 76, 89, 102, 115, 128, 141, 154, 167, 180, 193, 206, 219 and 232 after cross-over | Hematocrit is the percentage of red blood cells (RBC) in the blood. |
| Secondary | Spleen Length by Visit | Week 4, 8, 12, 16, 20, 24, 28, 40, 52, 66, 80, 92, 104, 117, 130, 143, 156, 169, 182, 195, 208, 221, 234, 247 and 260 | Spleen length was assessed by manual palpation at every study visit. |
| Secondary | Change From Baseline in Eastern Cooperative Oncology Group (ECOG) Performance Status to Week 28 | Baseline and Week 28 | The ECOG scale of performance status described the level of functioning of participants in terms of their ability to care for themselves, daily activity, and physical ability. The ECOG performance was recorded as per ECOG performance status grades ranging from 0 (fully active, able to carry on all pre-disease performance without restriction) to 5 (dead). |
| Secondary | Number of Participants Achieving a Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 28 | Week 28 | Proportion of patients achieving a partial remission at Week 28, based on the ELN and IWG-MRT criteria, as defined by: * Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) score reduction of greater than or equal to 10 points from baseline to Week 28, and * Hct control defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 28, with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8, and * WBC \< 10 x10\^9/L at Week 28, and * Platelets ≤ 400 x 10\^9/L at Week 28, and * No palpable spleen at Week 28, and * No hemorrhagic or thrombotic events, and * No transformation into post-PV myelofibrosis, myelodysplastic syndrome (IWG-MRT criteria) or acute leukemia (WHO criteria). |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 registered outcomesOutcomes26
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants Achieving Hematocrit (Hct) Control at Week 28 | Week 28 | Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Achieving a Complete Hematological Remission at Week 28 | Week 28 | Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Achieving a Hematocrit (Hct) Control at Week 52 and Week 80 | Week 52 and 80 | Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Achieving a Complete Hematological Remission at Week 52 and Week 80 | Week 52 and 80 | Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Phlebotomies Over Time | Baseline to Week 260 | Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Hematocrit (Hct) at Each Visit | Baseline, Week 4, 8, 12, 16, 20, 24, 28, 40, 52, 66, 80, 92, 104, 117, 130, 143, 156, 169, 182, 195, 208, 221, 234, 247 and 260 | Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. Data after crossover for participants randomized to BAT are reported as separate outcome measure. | volume percentage of RBC in blood | MEAN |
| SECONDARY | Change From Baseline in Hematocrit (Hct) at Each Scheduled Visit After Crossover in Participants Randomized to BAT Who Cross Over to Ruxolitinib | Baseline (last assessment before cross over), Week 4, 8, 12, 16, 20, 24, 28, 40, 52, 64, 76, 89, 102, 115, 128, 141, 154, 167, 180, 193, 206, 219 and 232 after cross-over | Cross-over set consisted of all participants randomized to the BAT arm, who crossed over and received at least one dose of ruxolitinib. | Volume percentage of RBC in blood | MEAN |
| SECONDARY | Spleen Length by Visit | Week 4, 8, 12, 16, 20, 24, 28, 40, 52, 66, 80, 92, 104, 117, 130, 143, 156, 169, 182, 195, 208, 221, 234, 247 and 260 | Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. Data after crossover for participants randomized to BAT were not collected. | cm | MEAN |
| SECONDARY | Change From Baseline in Eastern Cooperative Oncology Group (ECOG) Performance Status to Week 28 | Baseline and Week 28 | Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Achieving a Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 28 | Week 28 | Full Analysis Set (FAS) included all participants to whom study treatment was assigned by randomization and had valid measurements for the outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 outcomesResults by group484
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants Achieving Hematocrit (Hct) Control at Week 28 | Ruxolitinib | 46 | Participants | ||
| Number of Participants Achieving Hematocrit (Hct) Control at Week 28 | Best Available Therapy (BAT) | 14 | Participants | ||
| Number of Participants Achieving a Complete Hematological Remission at Week 28 | Ruxolitinib | 17 | Participants | ||
| Number of Participants Achieving a Complete Hematological Remission at Week 28 | Best Available Therapy (BAT) | 4 | Participants | ||
| Number of Participants Achieving a Hematocrit (Hct) Control at Week 52 and Week 80 | Ruxolitinib | Week 52 | 44 | Participants | |
| Number of Participants Achieving a Hematocrit (Hct) Control at Week 52 and Week 80 | Best Available Therapy (BAT) | Week 52 | 5 | Participants | |
| Number of Participants Achieving a Hematocrit (Hct) Control at Week 52 and Week 80 | Ruxolitinib | Week 80 | 35 | Participants | |
| Number of Participants Achieving a Hematocrit (Hct) Control at Week 52 and Week 80 | Best Available Therapy (BAT) | Week 80 | 2 | Participants | |
| Number of Participants Achieving a Complete Hematological Remission at Week 52 and Week 80 | Ruxolitinib | Week 52 | 17 | Participants | |
| Number of Participants Achieving a Complete Hematological Remission at Week 52 and Week 80 | Best Available Therapy (BAT) | Week 52 | 3 | Participants |
1 to 10 of 484Source ClinicalTrials.gov
See all 484 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Core Study | STARTED | Ruxolitinib | 74 | |
| Core Study | STARTED | Best Available Therapy (BAT) | 75 | |
| Core Study | Full Analysis Set | Ruxolitinib | 74 | |
| Core Study | Full Analysis Set | Best Available Therapy (BAT) | 75 | |
| Core Study | Crossover Set | Ruxolitinib | 0 | |
| Core Study | Crossover Set | Best Available Therapy (BAT) | 58 | |
| Core Study | COMPLETED | Ruxolitinib | 59 | |
| Core Study | COMPLETED | Best Available Therapy (BAT) | 61 | |
| Core Study | NOT COMPLETED | Ruxolitinib | 15 | |
| Core Study | NOT COMPLETED | Best Available Therapy (BAT) | 14 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events570
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 | 27 | 74 | |
| other | Anaemia | Blood and lymphatic system disorders | Best Available Therapy | 1 | 75 | |
| other | Anaemia | Blood and lymphatic system disorders | All Crossover Patients | 19 | 58 | |
| other | Leukocytosis | Blood and lymphatic system disorders | Ruxolitinib | 5 | 74 | |
| other | Leukocytosis | Blood and lymphatic system disorders | Best Available Therapy | 4 | 75 | |
| other | Leukocytosis | Blood and lymphatic system disorders | All Crossover Patients | 3 | 58 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Ruxolitinib | 5 | 74 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Best Available Therapy | 6 | 75 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | All Crossover Patients | 3 | 58 | |
| other | Thrombocytosis | Blood and lymphatic system disorders | Ruxolitinib | 8 | 74 |
1 to 10 of 570Source ClinicalTrials.gov
See all 570 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ruxolitinib | 1 | 74 |
| Total, all-cause mortality | Best Available Therapy | 1 | 75 |
| Total, all-cause mortality | All Crossover Patients | 3 | 58 |
| Total, other adverse events | Ruxolitinib | 73 | 74 |
| Total, other adverse events | Best Available Therapy | 56 | 75 |
| Total, other adverse events | All Crossover Patients | 56 | 58 |
| Total, serious adverse events | Ruxolitinib | 34 | 74 |
| Total, serious adverse events | Best Available Therapy | 9 | 75 |
| Total, serious adverse events | All Crossover Patients | 23 | 58 |
Source ClinicalTrials.gov
Papers4
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 comparisons6
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
Ruxolitinib vs Best Available Therapy (BAT): Number of Participants Achieving a Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 28 | Trial comparisons |
| Ruxolitinib vs Best Available Therapy (BAT): Number of Participants Achieving a Complete Hematological Remission at Week 28 | Trial comparisons |
| Ruxolitinib vs Best Available Therapy (BAT): Number of Participants With Transformation Free Survival Events | Trial comparisons |
| Ruxolitinib vs Best Available Therapy (BAT): Number of Participants Developing Thrombosis | Trial comparisons |
| Ruxolitinib vs Best Available Therapy (BAT): Number of Participants Achieving Hematocrit (Hct) Control at Week 28 | Trial comparisons |
| Ruxolitinib vs Best Available Therapy (BAT): Number of Participants With Overall Survival (OS) Events | Trial comparisons |
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.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02038036
GET https://conductscience.org/api/trials/v1/NCT02038036/registered?page=2