An Efficacy and Safety Trial of Fedratinib in Subjects With DIPSS, Intermediate or High-Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis and Previously Treated With Ruxolitinib (FREEDOM)
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|ClinicalTrials.gov Identifier: NCT03755518|
Recruitment Status : Recruiting
First Posted : November 28, 2018
Last Update Posted : January 31, 2020
This is Single-Arm, Open-Label Efficacy and Safety Trial of Fedratinib in Subjects with DIPSS (Dynamic International Prognostic Scoring System)-Intermediate or High- Risk Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (post-PV MF), or Post-Essential Thrombocythemia Myelofibrosis (post-ET MF) and Previously Treated with Ruxolitinib.
The primary objective of the study is to evaluate the percentage of subjects with at least a 35% reduction of spleen volume and one of the secondary objectives is to evaluate the safety of fedratinib
|Condition or disease||Intervention/treatment||Phase|
|Primary Myelofibrosis Post-Polycythemia Vera Myelofibrosis||Drug: FEDRATINIB||Phase 3|
Expanded Access : An investigational treatment associated with this study is available outside the clinical trial. More info ...
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||110 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 3b, Multicenter, Single-arm, Open-label Safety AND Efficacy Study of Fedratinib in Subjects With DIPSS-Intermediate or High-Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis|
|Actual Study Start Date :||March 27, 2019|
|Estimated Primary Completion Date :||June 6, 2022|
|Estimated Study Completion Date :||November 28, 2023|
Experimental: Administration of Fedratinib 400mg/day
Self-administered Investigational Product (IP) (400 mg/day) on an outpatient basis, once daily preferably with food during an evening meal at the same time each day in consecutive 4-week (28-day) cycles.
A potent and selective inhibitor of JAK2 kinase activity
- Proportion of subjects who have a ≥ 35% SVR at end of Cycle 6 [ Time Frame: At the end of Cycle 6 (each cycle is 28 days) ]Spleen volume response rate (RR)
- Adverse Event(s) [ Time Frame: Up to 12 months post last dose ]Number of participants with adverse event
- Proportion of subjects who have ≥ 50% reduction in spleen size by [ Time Frame: At the end of Cycle 6 (each cycle is 28 days) ]Spleen response rate by palpation (RRP)
- Symptom response rate (SRR) [ Time Frame: At the end of Cycle 6 (each cycle is 28 days) ]Is defined as the proportion of subjects with ≥ 50% reduction from baseline to the end of Cycle 6 in total symptom score (TSS) measured by MFSAF version 4.0.
- To evaluate durability of spleen volume response (DR) [ Time Frame: From enrollment until treatment discontinuation (estimation of 12 months) ]Is defined as time from the first documented spleen response (ie, ≥ 35% reduction in spleen volume) to the first documented spleen volume reduction < 35%.
- To evaluate the durability of spleen response by palpation (DRP) [ Time Frame: From enrollment until treatment discontinuation (estimation of 12 months) ]Is defined as time from the first documented palpable spleen response, according to the IWG-MRT 2013 to the time of the first documented loss of response according to the IWG-MRT 2013.
- Durability of symptoms response (DSR) [ Time Frame: Up to 30 days post last dose ]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%.
- Gastrointestinal Adverse Events [ Time Frame: Up to 30 days post last dose ]Incidence of subjects with Grade 3 or higher Gastrointestinal events (nausea, diarrhea, or vomiting) according to CTCAE v5.0
- Wernicke encephalopathy (WE) events [ Time Frame: Up to 30 days post last dose ]Occurrence of confirmed Wernicke encephalopathy events
- Wernicke encephalopathy (WE) thiamine monitoring [ Time Frame: Up to 30 days post last dose ]Monitoring and correction of thiamine levels as appropriate
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03755518
|Contact: Daniel Aversa, MSemail@example.com|
|Study Director:||Daniela Buglio, MD||Celgene|