Phase 3 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

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-001897-25-GB

Enrollment

225

Registered

2011-08-17

Start date

2011-10-03

Completion date

Unknown

Last updated

2019-02-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Primary myelofibrosis, post-polycythemia vera myelofibrosis, or post- essential thrombocythemia myelofibrosis MedDRA version: 20.1 Level: LLT Classification code 10018864 Term: Haematopoietic neoplasm NOS System Organ Class: 100000004864

Interventions

Product Code: SAR302503 Pharmaceutical Form: Capsule, hard Current Sponsor code: SAR302503 Concentration unit: mg milligram(s) Concentra

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Diagnosis of Primary Myelofibrosis (MF) or Post-Polycythemia Vera MF or Post-Essential Thrombocythemia MF, according to the 2008 World Health Organization and International Working Group of Myelofibrosis Research and Treatment (IWG-MRT) criteria. MF classified as high-risk or intermediate-risk level 2, as defined by modified IWG-MRT criteria. Enlarged spleen, palpable at least 5 cm below costal margin. At least 18 years of age. Eastern Cooperative Oncology Group performance status of 0, 1, or 2 at study entry. The following laboratory values within 14 days prior to the initiation of IMP or placebo: Absolute Neutrophil Count (ANC) =1.0 x 10exp9/L Platelet count =50 x 10exp9/L Serum creatinine =1.5 x Upper Limit of Normal (ULN) Serum amylase and lipase =1.5 x ULN Direct bilirubin =2.0 x ULN Aspartate aminotransferase or alanine aminotransferase =3 x ULN; higher values (ie, =5 x ULN) are allowed if clinically compatible with hepatic extramedullary hematopoiesis. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 112 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 113

Exclusion criteria

Exclusion criteria: Splenectomy. Any chemotherapy (eg, hydroxyurea), immunomodulatory drug therapy (eg, thalidomide, interferon-alpha), Anagrelide, immunosuppressive therapy, corticosteroids >10 mg/day prednisone or equivalent, or growth factor treatment (eg, erythropoietin), or hormones (eg, androgens, danazol) within 14 days prior to initiation of IMP or placebo; darbepoetin use within 28 days prior to initiation of IMP or placebo. Patients who have had exposure to hydroxyurea (eg, hydrea) in the past may be enrolled into the study as long as it has not been administered within 14 days prior to initiation of IMP or placebo. Major surgery within 28 days or radiation within 6 months prior to initiation of IMP or placebo. Prior treatment with a Janus Kinase 2 (JAK2) inhibitor.

Design outcomes

Primary

Measure	Time frame
Main 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 Objective: 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. ;Primary end point(s): 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;Timepoint(s) of evaluation of this end point: 6 months	—

Measure

Time frame

Main 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 Objective: 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. ;Primary end point(s): 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;Timepoint(s) of evaluation of this end point: 6 months

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Secondary

Measure	Time frame
Timepoint(s) of evaluation of this end point: 6 months to approximately 5 year(s); Secondary end point(s): Symptom Response Rate (SRR): Proportion of patients with =50% reduction from baseline to the end of Cycle 6 in the total symptom score. 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, at end of Cycle 6, the EOT visit, and the 30-day follow-up visit. OS (overall survival) of either 400 mg/day or 500 mg/day of IMP as compared to placebo. PFS (progression free survival) of either 400 mg/day or 500 mg/day of IMP as compared to placebo. Proportion of patients who have =25% reduction in volume of spleen size at end of Cycle 6, and confirmed 4 weeks thereafter. Duration of spleen response, measured by MRI (or CT scan in patients with contraindications for MRI. Safety, as assessed by clinical, laboratory, ECG, and vital sign events; graded by the NCI CTCAE v4.03.	—

Measure

Time frame

Timepoint(s) of evaluation of this end point: 6 months to approximately 5 year(s); Secondary end point(s): Symptom Response Rate (SRR): Proportion of patients with =50% reduction from baseline to the end of Cycle 6 in the total symptom score. 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, at end of Cycle 6, the EOT visit, and the 30-day follow-up visit. OS (overall survival) of either 400 mg/day or 500 mg/day of IMP as compared to placebo. PFS (progression free survival) of either 400 mg/day or 500 mg/day of IMP as compared to placebo. Proportion of patients who have =25% reduction in volume of spleen size at end of Cycle 6, and confirmed 4 weeks thereafter. Duration of spleen response, measured by MRI (or CT scan in patients with contraindications for MRI. Safety, as assessed by clinical, laboratory, ECG, and vital sign events; graded by the NCI CTCAE v4.03.

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Countries

Australia, Austria, Belgium, Brazil, Canada, Czech Republic, Germany, Hungary, Ireland, Israel, Italy, Korea, Republic of, Lithuania, Mexico, Portugal, Russian Federation, Singapore, South Africa, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States

Contacts

Public ContactMedical Information

Aventis Pharma Limited trading as Sanofi-aventis

uk-medicalinformation@sanofi-aventis.com+4401483505515

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026