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
A Phase 3, multicenter, open-label, randomized study to evaluate the efficacy and safety of
fedratinib compared to best available therapy (BAT) 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 35% spleen volume reduction
in the fedratinib and the BAT arms.
This Phase 3, multicenter, randomized, two-arm, open-label study will include subjects with
intermediate or high-risk (as per the DIPSS score) primary myelofibrosis (PMF),
postpolycythemia vera myelofibrosis (post-PV MF), or post-essential thrombocythemia
myelofibrosis (post-ET MF). This study will be conducted in compliance with International
Council for Harmonisation (ICH) Good Clinical Practices (GCPs).
Study design includes:
A 28-day Screening Period
2:1 Randomization to fedratinib or best available therapy (BAT)
Stratification at Randomization according to:
Spleen size by palpation: < 15 cm below left costal margin (LCM) versus 15 cm
Platelets 50 to < 100 x 109/L versus platelets 100 x 109/L
Refractory or relapsed to ruxolitinib treatment versus intolerant to ruxolitinib
Study Treatment Period (time on study drug plus 30 days after last dose)
Subjects are allowed to crossover from BAT to the fedratinib arm after the Cycle 6
response assessment or before the Cycle 6 response assessment in the event of a
confirmed progression of splenomegaly by MRI/CT scan
A Survival Follow-up Period for progression and survival
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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