NCT01437787

Phase III Study of SAR302503 in Intermediate-2 and High Risk Patients With Myelofibrosis

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, 3-Arm Study of SAR302503 in Patients With Intermediate-2 or High-Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis With Splenomegaly

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 289 (actual)
• Sponsor: Bristol-Myers Squibb · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: * To evaluate the efficacy of daily oral doses of 400 mg or 500 mg of SAR302503 (Investigational Medicinal Product, IMP) compared to placebo in the reduction of spleen volume as determined by magnetic resonance imaging (MRI) (or computed tomography scan in patients with contraindications for MRI). Secondary Objectives: * To evaluate the effect on Myelofibrosis (MF)-associated symptoms (key MF symptoms) as measured by the modified Myelofibrosis Symptom Assessment Form (MFSAF) diary. * To evaluate the Overall Survival of patients treated with either 400 mg/day or 500 mg/day of IMP as compared to placebo. * To evaluate the Progression Free Survival of patients treated with either 400 mg/day or 500 mg/day of IMP as compared to placebo. * To evaluate the durability of splenic response. * To evaluate the safety of IMP.

Detailed description

The expected duration of a patient's treatment in this study is approximately 8 months, based on a maximum 28-day screening period, followed by a ≥6-month (6-cycle) treatment period, and an End Of Treatment (EOT) visit, which should be performed at least 30 days following the last administration of IMP or placebo. Patients who continue to benefit clinically will be allowed to remain on IMP or placebo beyond the 6-month treatment period until the occurrence of disease progression or unacceptable toxicity.

Conditions

• Hematopoietic Neoplasm

Interventions

Type	Name	Description
DRUG	SAR302503	Pharmaceutical form:capsule Route of administration: oral
DRUG	Placebo	Pharmaceutical form:capsule Route of administration: oral

Timeline

• Start date: 2011-12-01
• Primary completion: 2014-06-01
• Completion: 2014-06-01
• First posted: 2011-09-21
• Last updated: 2025-03-05

Locations

101 sites across 25 countries: United States, Australia, Austria, Belgium, Brazil, Canada, France, Germany, Hungary, Ireland, Israel, Italy, Lithuania, Mexico, Poland, Portugal, Romania, Russia, Singapore, South Africa, South Korea, Spain, Sweden, Taiwan, United Kingdom