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

Phase 3

Completed

Brief 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.

Recruitment & Eligibility

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
Placebo comparator	Placebo	once daily X 28 days, orally, empty stomach, approximately same time each day
SAR302503 500 mg	SAR302503	once daily X 28 days, orally, empty stomach, approximately same time each day
SAR302503 400 mg	SAR302503	once daily X 28 days, orally, empty stomach, approximately same time each day

Primary Outcome Measures

Name	Time	Method
Response Rate (RR), defined as the proportion of patients who have a ≥35% reduction in volume of spleen size at the end of Cycle 6, and confirmed 4 weeks thereafter	6 months

Secondary Outcome Measures

Name	Time	Method
Symptom Response Rate (SRR): Proportion of patients with ≥50% reduction from baseline to the end of Cycle 6 in the total symptom score.	6 months	This assessment will be conducted through the modified MFSAF diary, which will be completed during the week prior to Day 1 of each treatment cycle up to Cycle 6, and during the week prior to the end of Cycle 6.
OS (overall survival) of either 400 mg/day or 500 mg/day of IMP as compared to placebo.	approximately 5 years
PFS (progression free survival) of either 400 mg/day or 500 mg/day of IMP as compared to placebo.	approximately 5 years
Proportion of patients who have ≥25% reduction in volume of spleen size at end of Cycle 6, and confirmed 4 weeks thereafter.	6 months
Duration of spleen response, measured by MRI (or CT scan in patients with contraindications for MRI.	2 years
Clinical and laboratory events graded by the NCI CTCAE v4.03.	approximately 5 years

Locations (101): Investigational Site Number 840014
🇺🇸
Scottsdale, Arizona, United States
Investigational Site Number 840001
🇺🇸
La Jolla, California, United States
Investigational Site Number 840012
🇺🇸
La Jolla, California, United States
Investigational Site Number 840006
🇺🇸
Los Angeles, California, United States
Investigational Site Number 840013
🇺🇸
Baton Rouge, Louisiana, United States
Investigational Site Number 840008
🇺🇸
Rochester, Minnesota, United States
Investigational Site Number 840009
🇺🇸
Newark, New Jersey, United States
Investigational Site Number 840002
🇺🇸
Canton, Ohio, United States
Investigational Site Number 840004
🇺🇸
Houston, Texas, United States
Investigational Site Number 036001
🇦🇺
Box Hill, Australia
Scroll for more (91 remaining)
Investigational Site Number 840014
🇺🇸Scottsdale, Arizona, United States