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

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 202 (actual)
• Sponsor: Celgene · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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 below LCM * Platelets ≥ 50 to \< 100 x 109/L versus platelets ≥ 100 x 109/L * Refractory or relapsed to ruxolitinib treatment versus intolerant to ruxolitinib treatment * 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

Conditions

• Primary Myelofibrosis
• Post-Polycythemia Vera
• Myelofibrosis

Interventions

Type	Name	Description
DRUG	FEDRATINIB	A potent and selective inhibitor of JAK2 kinase activity
DRUG	Best Available Therapy (BAT)	Best available therapy (BAT)

Timeline

• Start date: 2019-09-09
• Primary completion: 2022-12-15
• Completion: 2025-07-28
• First posted: 2019-05-16
• Last updated: 2025-08-28
• Results posted: 2024-01-30

Locations

107 sites across 16 countries: Australia, Austria, Belgium, China, Czechia, France, Germany, Hungary, Ireland, Italy, Netherlands, Poland, Russia, South Korea, Spain, United Kingdom

Regulatory

• FDA-regulated drug study