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

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.

Timeline

Start

2011-12

Primary completion

2014-06

Completion

2014-06

Outcome

Met primary endpoint

paper The primary end point was achieved by 35 of 96 (36% [95% CI, 27%-46%]) and 39 of 97 (40% [95% CI, 30%-50%]) PMID 26181658 ↗

paper vs 1 of 96 (1% [95% CI, 0%-3%]) in the placebo group (P < .001). PMID 26181658 ↗

Publications

• Pardanani A, Harrison C, Cortes JE, Cervantes F, Mesa RA, Milligan D, Masszi T, Mishchenko E, Jourdan E, Vannucchi AM, Drummond MW, Jurgutis M, Kuliczkowski K, Gheorghita E, Passamonti F, Neumann F, Patki A, Gao G, Tefferi A. Safety and Efficacy of Fedratinib in Patients With Primary or Secondary Myelofibrosis: A Randomized Clinical Trial. JAMA Oncol. 2015 Aug;1(5):643-51. doi: 10.1001/jamaoncol.2015.1590.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Fedratinib	Small molecule	400 mg	Oral
Subject	Fedratinib	Small molecule	500 mg	Oral

Indications