Trial · NCT03952039
An Efficacy and Safety Study of Fedratinib Compared to Best Available Therapy in Subjects With DIPSS-intermediate or High-risk Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis and Previously Treated With Ruxolitinib
NCT03952039, Phase 3, Completed, Celgene; results first posted 2024-01-30
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Efficacy and Safety Study of Fedratinib Compared to Best Available Therapy in Subjects With DIPSS-intermediate or High-risk Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis and Previously Treated With Ruxolitinib. Trials, record NCT03952039. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03952039
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
An Efficacy and Safety Study of Fedratinib Compared to Best Available Therapy in Subjects With DIPSS-intermediate or High-risk Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis and Previously Treated With Ruxolitinib
- NCT number
- NCT03952039
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 202 participants, actual
- Lead sponsor
- Celgene
- Conditions
- Primary Myelofibrosis, Post-Polycythemia Vera, Myelofibrosis
- Interventions
- FEDRATINIB, Best Available Therapy (BAT)
- First posted
- 2019-05-16
- Last updated
- 2025-08-28
- Other registries' IDs
- eudract:2018-003411-21
- Results first posted
- 2024-01-30
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Spleen Volume Response Rate (RR) | From Screening to end of Cycle 6 (1 cycle = 28 days), approximately 196 days | Percentage of participants who have ≥ 35% spleen volume reduction (SVR) at end of cycle 6 A Cochran-Mantel-Haenszel (CMH) test to adjust for planned stratification factors (spleen size by palpation, platelet counts and refractory/relapsed or intolerance to ruxolitinib treatment). The RRs and 95% confidence intervals (CI) will be provided for each arm as well as for the difference in RRs and 95% confidence interval of the difference for fedratinib to BAT. |
| Secondary | Symptom Response Rate (SRR) | From Cycle 1 Dose 1 to end of Cycle 6, approximately 168 days | Percentage of participants with ≥ 50% reduction in total symptom scores measured by Myelofibrosis Symptom Assessment Form (MFSAF) 4.0 at end of cycle 6. Subjects with a missing TSS at the end of cycle 6 or who had disease progression before the end of the cycle 6 will be considered non-responders. MFSAF measures the sum of 7 symptoms on a scale from 0 to 10 (0 being absent and 10 being the worst imagineable). The lower the score the better. A Cochran-Mantel-Haenszel (CMH) test to adjust for planned stratification factors (spleen size by palpation, platelet counts and refractory/relapsed or intolerance to ruxolitinib treatment). The SRRs and 95% confidence intervals (CI) will be provided for each arm as well as for the difference in SRRs and 95% confidence interval of the difference for fedratinib to BAT. |
| Secondary | Spleen Volume Response Rate (RR25) | From Screening to end of Cycle 6 (1 cycle = 28 days), approximately 196 days | Percentage of participants who have ≥ 25% spleen volume reduction (SVR) at end of cycle 6 A Cochran-Mantel-Haenszel (CMH) test to adjust for planned stratification factors (spleen size by palpation, platelet counts and refractory/relapsed or intolerance to ruxolitinib treatment). The RRs and 95% confidence intervals (CI) will be provided for each arm as well as for the difference in RRs and 95% confidence interval of the difference for fedratinib to BAT. |
| Secondary | Number of Participants With All Grade Treatment Emergent Adverse Events (AEs) and Grade 3 to 4 Treatment Emergent AEs | From Cycle 1 Dose 1 to end of Cycle 6, approximately 168 days | Number of participants with all grade adverse events (AEs) and grade 3 to 4 AEs |
| Secondary | Number of Participants With Hematology Laboratory Abnormalities | From Cycle 1 Dose 1 to end of Cycle 6, approximately 168 days | Number of participants with hematology laboratory abnormalities in the following parameters: hemoglobin (decreased), leukocytes (increase and decrease), lymphocytes (decreased), neutrophils (segmented and band form decreased), Platelets (decreased). |
| Secondary | Spleen Response Rate by Palpation (RRP) | From Cycle 1 Dose 1 to end of Cycle 6, approximately 168 days | Spleen response rate by palpation is the percentage of participants at the end of cycle 6 with a spleen response according to the IWG-MRT 2013 criteria. A baseline splenomegaly that is palpable at 5-10 cm, below the LCM, becomes not palpable\*\* or A baseline splenomegaly that is palpable at \> 10 cm, below the LCM, decreases by ≥ 50%\*\* Participants with a missing spleen size assessment at the end of cycle 6 including those who meet the criteria for progression of splenomegaly before end of cycle 6 will be considered not to be responders. |
| Secondary | Durability of Spleen Volume Response (DR) | From baseline to end of treatment visit, Approximately on average 53 weeks up to 151 weeks. | Durability of spleen volume response (DR) by MRI/CT is defined as the date from the first documented spleen response (ie, ≥ 35% reduction in spleen volume) to the date of subsequent progressive disease (PD) in spleen volume per the IWG-MRT 2013 criteria or death, whichever is earlier. In the absence of an event before the analysis is performed, the DR will be censored at the date of the last valid assessment performed before the analysis performed date. |
| Secondary | Durability of Spleen Response by Palpation (DRP) | From baseline to end of treatment visit, Approximately on average 53 weeks up to 151 weeks. | Durability of spleen response by palpation (DRP) is defined as time from the date of the first documented palpable spleen response, according to the IWG-MRT 2013 to the date of the subsequent PD in spleen size according to the IWG-MRT 2013 or death, whichever is earlier. Durability of spleen response by palpation according to the IWG-MRT 2013 criteria will be calculated for subjects that have an enlarged spleen at baseline (≥ 5 cm below LCM), and that have a spleen response by palpation. In the absence of an event before the analysis is performed, the DRP will be censored at the date of the last valid assessment performed before the analysis performed date. |
| Secondary | Durability of Symptoms Response (DSR) | From baseline to end of treatment visit, Approximately on average 53 weeks up to 151 weeks. | The DSR is defined as time from the first documented response in TSS (ie, reduction in TSS ≥ 50%) measured by MFSAF version 4.0 to the first documented TSS reduction \< 50%. In the absence of TSS reduction \< 50% before the analysis performed, the DSR will be censored at the date of the last valid assessment performed before the analysis performed date. |
| Secondary | Assessment of the Effectiveness of Risk Mitigation Strategy for ≥3 Grade Gastrointestinal Adverse Events and Any Grade Wernickes Encephalopathy | From Screening to end of Cycle 6 (1 cycle = 28 days), approximately 196 days | Number of participants with a CTCAE Grade ≥3 of nausea, diarrhea, or vomiting and any grade wernickes encephalopathy. |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Spleen Volume Response Rate (RR) | From Screening to end of Cycle 6 (1 cycle = 28 days), approximately 196 days | Intent to Treat Population | Percentage of Participants | NUMBER |
| SECONDARY | Symptom Response Rate (SRR) | From Cycle 1 Dose 1 to end of Cycle 6, approximately 168 days | ITT population with non-zero baseline TSS | Percentage of Participants | NUMBER |
| SECONDARY | Spleen Volume Response Rate (RR25) | From Screening to end of Cycle 6 (1 cycle = 28 days), approximately 196 days | Intent to Treat Population | Percentage of Participants | NUMBER |
| SECONDARY | Number of Participants With All Grade Treatment Emergent Adverse Events (AEs) and Grade 3 to 4 Treatment Emergent AEs | From Cycle 1 Dose 1 to end of Cycle 6, approximately 168 days | Safety Population | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Hematology Laboratory Abnormalities | From Cycle 1 Dose 1 to end of Cycle 6, approximately 168 days | Safety Population | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Spleen Response Rate by Palpation (RRP) | From Cycle 1 Dose 1 to end of Cycle 6, approximately 168 days | Intent to Treat Population with baseline spleen size ≥5 cm below the lower costal margin (LCM) | Percentage of Participants | NUMBER |
| SECONDARY | Durability of Spleen Volume Response (DR) | From baseline to end of treatment visit, Approximately on average 53 weeks up to 151 weeks. | Participants with spleen volume response at any time | Weeks | MEDIAN |
| SECONDARY | Durability of Spleen Response by Palpation (DRP) | From baseline to end of treatment visit, Approximately on average 53 weeks up to 151 weeks. | Participants with spleen response by palpation at any time | Weeks | MEDIAN |
| SECONDARY | Durability of Symptoms Response (DSR) | From baseline to end of treatment visit, Approximately on average 53 weeks up to 151 weeks. | Participants with a symptom response at any time | Weeks | MEDIAN |
| SECONDARY | Assessment of the Effectiveness of Risk Mitigation Strategy for ≥3 Grade Gastrointestinal Adverse Events and Any Grade Wernickes Encephalopathy | From Screening to end of Cycle 6 (1 cycle = 28 days), approximately 196 days | Safety Population | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group116
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Spleen Volume Response Rate (RR) | Fedratinib | 35.8 | 95% Confidence Interval 27.7 to 44.6 | Percentage of Participants | |
| Spleen Volume Response Rate (RR) | Best Available Therapy (BAT) | 6.0 | 95% Confidence Interval 1.7 to 14.6 | Percentage of Participants | |
| Symptom Response Rate (SRR) | Fedratinib | 34.1 | 95% Confidence Interval 25.9 to 43.1 | Percentage of Participants | |
| Symptom Response Rate (SRR) | Best Available Therapy (BAT) | 16.9 | 95% Confidence Interval 8.8 to 28.3 | Percentage of Participants | |
| Spleen Volume Response Rate (RR25) | Fedratinib | 47.0 | 95% Confidence Interval 38.3 to 55.8 | Percentage of Participants | |
| Spleen Volume Response Rate (RR25) | Best Available Therapy (BAT) | 13.4 | 95% Confidence Interval 6.3 to 24.0 | Percentage of Participants | |
| Number of Participants With All Grade Treatment Emergent Adverse Events (AEs) and Grade 3 to 4 Treatment Emergent AEs | Fedratinib | All Grade AEs | 132 | Participants | |
| Number of Participants With All Grade Treatment Emergent Adverse Events (AEs) and Grade 3 to 4 Treatment Emergent AEs | Best Available Therapy (BAT) | All Grade AEs | 65 | Participants | |
| Number of Participants With All Grade Treatment Emergent Adverse Events (AEs) and Grade 3 to 4 Treatment Emergent AEs | Fedratinib | Grade 3 and 4 AEs | 88 | Participants | |
| Number of Participants With All Grade Treatment Emergent Adverse Events (AEs) and Grade 3 to 4 Treatment Emergent AEs | Best Available Therapy (BAT) | Grade 3 and 4 AEs | 29 | Participants |
1 to 10 of 116Source ClinicalTrials.gov
See all 116 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Fedratinib | 134 | |
| Overall Study | STARTED | Best Available Therapy (BAT) | 67 | |
| Overall Study | Participants Who Crossed Over | Fedratinib | 0 | |
| Overall Study | Participants Who Crossed Over | Best Available Therapy (BAT) | 46 | |
| Overall Study | COMPLETED | Fedratinib | 43 | |
| Overall Study | COMPLETED | Best Available Therapy (BAT) | 28 | |
| Overall Study | NOT COMPLETED | Fedratinib | 91 | |
| Overall Study | NOT COMPLETED | Best Available Therapy (BAT) | 39 |
Source ClinicalTrials.gov
Adverse events498
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 | Fedratinib | 55 | 134 | |
| other | Anaemia | Blood and lymphatic system disorders | Best Available Therapy (BAT) | 24 | 67 | |
| other | Anaemia | Blood and lymphatic system disorders | Fedratinib After Crossover | 19 | 46 | |
| other | Leukocytosis | Blood and lymphatic system disorders | Fedratinib | 4 | 134 | |
| other | Leukocytosis | Blood and lymphatic system disorders | Best Available Therapy (BAT) | 4 | 67 | |
| other | Leukocytosis | Blood and lymphatic system disorders | Fedratinib After Crossover | 2 | 46 | |
| other | Neutropenia | Blood and lymphatic system disorders | Fedratinib | 9 | 134 | |
| other | Neutropenia | Blood and lymphatic system disorders | Best Available Therapy (BAT) | 2 | 67 | |
| other | Neutropenia | Blood and lymphatic system disorders | Fedratinib After Crossover | 4 | 46 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Fedratinib | 48 | 134 |
1 to 10 of 498Source ClinicalTrials.gov
See all 498 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Fedratinib | 43 | 134 |
| Total, all-cause mortality | Best Available Therapy (BAT) | 7 | 67 |
| Total, all-cause mortality | Fedratinib After Crossover | 11 | 46 |
| Total, other adverse events | Fedratinib | 128 | 134 |
| Total, other adverse events | Best Available Therapy (BAT) | 57 | 67 |
| Total, other adverse events | Fedratinib After Crossover | 45 | 46 |
| Total, serious adverse events | Fedratinib | 72 | 134 |
| Total, serious adverse events | Best Available Therapy (BAT) | 21 | 67 |
| Total, serious adverse events | Fedratinib After Crossover | 16 | 46 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 39265613 | DERIVED |
Source PubMed
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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