NCT01523171

Phase II, Open Label, Single Arm Study of SAR302503 In Myelofibrosis Patients Previously Treated With Ruxolitinib

A Phase II, Multicenter, Open Label, Single Arm Study of SAR302503 in Subjects Previously Treated With Ruxolitinib and With a Current Diagnosis of Intermediate or High-Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 97 (actual)
• Sponsor: Bristol-Myers Squibb · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: \- To evaluate the efficacy of once daily dose of SAR302503 in subjects previously treated with ruxolitinib and with a current diagnosis of intermediate-1 with symptoms, Intermediate-2 or high-risk primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (Post-PV MF), or post-essential thrombocythemia myelofibrosis (Post-ET MF) based on the reduction of spleen volume at the end of 6 treatment cycles; Secondary Objectives: * To evaluate the effect of SAR302503 on Myelofibrosis (MF) associated symptoms as measured by the modified Myelofibrosis Symptom Assessment Form (MFSAF) diary * To evaluate the durability of splenic response * To evaluate the splenic response to SAR302503 by palpation at the end of Cycle 6 * To evaluate the splenic response to SAR302503 at the end of Cycle 3 * To evaluate the effect of SAR302503 on the Janus kinase 2 (JAK2) V617F allele burden * To evaluate the safety and tolerability of SAR302503 in this population * To evaluate plasma concentrations of SAR302503 for population PK analysis, if warranted

Detailed description

The expected duration of the 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 EOT visit for subjects who will not continue the treatment after completing the 6 cycles of SAR302503, or discontinue the treatment early for any reasons as well as a follow-up visit which should occur 30 days after the last administration of SAR302503. Patients who continue to benefit clinically will be allowed to remain on study medication 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

Timeline

• Start date: 2012-04-01
• Primary completion: 2014-04-01
• Completion: 2014-04-01
• First posted: 2012-02-01
• Last updated: 2025-03-05

Locations

42 sites across 10 countries: United States, Austria, Belgium, Canada, France, Germany, Italy, Netherlands, Spain, United Kingdom