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

March 3, 2025 updated by: Bristol-Myers Squibb

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

289

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Box Hill, Australia, 3128
        • Investigational Site Number 036001
      • Herston, Australia, 4029
        • Investigational Site Number 036005
      • Randwick, Australia, 2031
        • Investigational Site Number 036003
      • Tweed Heads, Australia, 2485
        • Investigational Site Number 036004
      • Wodonga, Australia, 3690
        • Investigational Site Number 036002
  • Austria
      • Wien, Austria, 1090
        • Investigational Site Number 040001
  • Belgium
      • Antwerpen, Belgium, 2060
        • Investigational Site Number 056003
      • Leuven, Belgium, 3000
        • Investigational Site Number 056001
  • Brazil
      • Jau, Brazil, 17210-120
        • Investigational Site Number 076002
      • Porto Alegre, Brazil, 90110-270
        • Investigational Site Number 076004
      • Rio De Janeiro, Brazil, 20230-130
        • Investigational Site Number 076001
  • Canada
      • Montreal, Canada, H1T 2M4
        • Investigational Site Number 124001
      • Montreal, Canada, H2W 1S6
        • Investigational Site Number 124003
      • Saint John, Canada, E2L 4L2
        • Investigational Site Number 124002
  • France
      • Marseille, France, 13273
        • Investigational Site Number 250006
      • Nantes Cedex 01, France, 44093
        • Investigational Site Number 250005
      • Nimes, France, 30029
        • Investigational Site Number 250004
      • Pierre Benite Cedex, France, 69495
        • Investigational Site Number 250002
      • Poitiers, France, 86000
        • Investigational Site Number 250007
      • Toulouse, France, 31000
        • Investigational Site Number 250003
      • Villejuif Cedex, France, 94805
        • Investigational Site Number 250001
  • Germany
      • Aachen, Germany, 52074
        • Investigational Site Number 276006
      • Bonn, Germany, 53127
        • Investigational Site Number 276007
      • Dresden, Germany, 01307
        • Investigational Site Number 276008
      • Mannheim, Germany, 68167
        • Investigational Site Number 276001
  • Hungary
      • Budapest, Hungary, 1097
        • Investigational Site Number 348002
      • Debrecen, Hungary, 4032
        • Investigational Site Number 348001
      • Györ, Hungary, 9023
        • Investigational Site Number 348007
      • Kecskemét, Hungary, 6000
        • Investigational Site Number 348006
      • Miskolc, Hungary, 3529
        • Investigational Site Number 348003
  • Ireland
      • Dublin, Ireland, DUBLIN 8
        • Investigational Site Number 372002
      • Galway, Ireland
        • Investigational Site Number 372001
  • Israel
      • Haifa, Israel, 31048
        • Investigational Site Number 376003
      • Tel Hashomer, Israel, 52621
        • Investigational Site Number 376002
  • Italy
      • Bergamo, Italy, 24127
        • Investigational Site Number 380002
      • Bologna, Italy, 40138
        • Investigational Site Number 380007
      • Firenze, Italy, 50134
        • Investigational Site Number 380004
      • Pavia, Italy, 27100
        • Investigational Site Number 380001
      • Pavia, Italy, 27100
        • Investigational Site Number 380006
      • Varese, Italy, 21100
        • Investigational Site Number 380003
  • Korea, Republic of
      • Bundang-Gu, Korea, Republic of, 463-707
        • Investigational Site Number 410002
      • Seoul, Korea, Republic of, 110-744
        • Investigational Site Number 410004
      • Seoul, Korea, Republic of, 135-710
        • Investigational Site Number 410001
      • Seoul, Korea, Republic of, 120-752
        • Investigational Site Number 410003
      • Seoul, Korea, Republic of, 137-701
        • Investigational Site Number 410005
      • Seoul, Korea, Republic of, 138-878
        • Investigational Site Number 410006
      • Seoul, Korea, Republic of
        • Investigational Site Number 410007
  • Lithuania
      • Kaunas, Lithuania, LT-50009
        • Investigational Site Number 440001
      • Klaipeda, Lithuania, LT-92288
        • Investigational Site Number 440002
  • Mexico
      • Queretaro, Mexico, 76000
        • Investigational Site Number 484001
  • Poland
      • Brzozow, Poland, 36-200
        • Investigational Site Number 616005
      • Gdansk, Poland, 80-952
        • Investigational Site Number 616002
      • Lodz, Poland, 93-510
        • Investigational Site Number 616006
      • Warszawa, Poland, 02-106
        • Investigational Site Number 616010
      • Wroclaw, Poland, 50-367
        • Investigational Site Number 616003
  • Portugal
      • Coimbra, Portugal, 3000-075
        • Investigational Site Number 620005
      • Lisboa, Portugal, 1649-035
        • Investigational Site Number 620001
      • Lisboa, Portugal, 1169-050
        • Investigational Site Number 620004
      • Porto, Portugal, 4200-072
        • Investigational Site Number 620003
  • Romania
      • Brasov, Romania
        • Investigational Site Number 642003
      • Bucharest, Romania, 022328
        • Investigational Site Number 642004
      • Bucuresti, Romania, 030171
        • Investigational Site Number 642002
      • Bucuresti, Romania
        • Investigational Site Number 642006
      • Timisoara, Romania
        • Investigational Site Number 642001
  • Russian Federation
      • Moscow, Russian Federation, 125167
        • Investigational Site Number 643009
      • Moscow, Russian Federation, 125284
        • Investigational Site Number 643001
      • Nizhny Novgorod, Russian Federation, 603126
        • Investigational Site Number 643010
      • Petrozavodsk, Russian Federation, 185019
        • Investigational Site Number 643008
      • St-Petersburg, Russian Federation, 197341
        • Investigational Site Number 643004
      • St.-Petersburg, Russian Federation, 191024
        • Investigational Site Number 643005
      • Volgograd, Russian Federation, 400138
        • Investigational Site Number 643007
  • Singapore
      • Singapore, Singapore, 119228
        • Investigational Site Number 702002
      • Singapore, Singapore, 169608
        • Investigational Site Number 702001
  • South Africa
      • Johannesburg, South Africa, 2013
        • Investigational Site Number 710003
      • Parktown, South Africa, 2193
        • Investigational Site Number 710002
  • Spain
      • Barcelona, Spain, 08036
        • Investigational Site Number 724001
      • Madrid, Spain, 28046
        • Investigational Site Number 724002
  • Sweden
      • Stockholm, Sweden, 14186
        • Investigational Site Number 752001
      • Uddevalla, Sweden, 451 80
        • Investigational Site Number 752002
  • Taiwan
      • Changhua, Taiwan, 500
        • Investigational Site Number 158002
      • Kaohsiung, Taiwan, 833
        • Investigational Site Number 158003
      • Taipei, Taiwan, 112
        • Investigational Site Number 158001
  • United Kingdom
      • Belfast, United Kingdom, BT9 7AB
        • Investigational Site Number 826006
      • Birmingham, United Kingdom, B9 5SS
        • Investigational Site Number 826003
      • Glasgow, United Kingdom, G12 0YN
        • Investigational Site Number 826002
      • Leeds, United Kingdom, LS9 7TF
        • Investigational Site Number 826004
      • London, United Kingdom, SE1 9RT
        • Investigational Site Number 826001
      • London, United Kingdom, W12 0HS
        • Investigational Site Number 826005
      • Manchester, United Kingdom, M20 4BX
        • Investigational Site Number 826007
      • Newcastle Upon Tyne, United Kingdom, NE7 7DN
        • Investigational Site Number 826008
      • Oxford, United Kingdom, OX3 7LJ
        • Investigational Site Number 826009
      • Southampton, United Kingdom, SO16 6YD
        • Investigational Site Number 826010
  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85259-5499
        • Investigational Site Number 840014
    • California
      • La Jolla, California, United States, 92093
        • Investigational Site Number 840001
      • La Jolla, California, United States, 92093
        • Investigational Site Number 840012
      • Los Angeles, California, United States, 90033
        • Investigational Site Number 840006
    • Louisiana
      • Baton Rouge, Louisiana, United States, 70808
        • Investigational Site Number 840013
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Investigational Site Number 840008
    • New Jersey
      • Newark, New Jersey, United States, 07112
        • Investigational Site Number 840009
    • Ohio
      • Canton, Ohio, United States, 44718
        • Investigational Site Number 840002
    • Texas
      • Houston, Texas, United States, 77030
        • Investigational Site Number 840004

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 (IPSS) (according to Cervantes F. et. al.; at screening).
  • 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

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.
  • Known active (acute or chronic) Hepatitis A, B, or C; and hepatitis B and C carriers
  • AST or ALT ≥2.5 x ULN
  • Total Bilirubin:
  • Exclude if ≥3.0 x ULN
  • Patients with total bilirubin between 1.5-3.0 x ULN must be excluded if the direct bilirubin fraction is ≥25% of the total
  • Prior history of chronic liver disease (eg, chronic alcoholic liver disease, autoimmune hepatitis, sclerosing cholangitis, primary biliary cirrhosis, hemachromatosis, non-alcoholic steatohepatitis [NASH])

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo comparator
once daily X 28 days, orally, empty stomach, approximately same time each day
Drug: Placebo

Pharmaceutical form:capsule

Route of administration: oral
Experimental: SAR302503 400 mg
once daily X 28 days, orally, empty stomach, approximately same time each day
Drug: SAR302503

Pharmaceutical form:capsule

Route of administration: oral
Experimental: SAR302503 500 mg
once daily X 28 days, orally, empty stomach, approximately same time each day
Drug: SAR302503

Pharmaceutical form:capsule

Route of administration: oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
Time Frame: 6 months
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Symptom Response Rate (SRR): Proportion of patients with ≥50% reduction from baseline to the end of Cycle 6 in the total symptom score.
Time Frame: 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.
6 months
OS (overall survival) of either 400 mg/day or 500 mg/day of IMP as compared to placebo.
Time Frame: approximately 5 years
approximately 5 years
PFS (progression free survival) of either 400 mg/day or 500 mg/day of IMP as compared to placebo.
Time Frame: approximately 5 years
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.
Time Frame: 6 months
6 months
Duration of spleen response, measured by MRI (or CT scan in patients with contraindications for MRI.
Time Frame: 2 years
2 years
Clinical and laboratory events graded by the NCI CTCAE v4.03.
Time Frame: approximately 5 years
approximately 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Bristol-Myers Squibb

Investigators

  • Study Director: Clinical Sciences & Operations, Sanofi

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2011

Primary Completion (Actual)

June 1, 2014

Study Completion (Actual)

June 1, 2014

Study Registration Dates

First Submitted

September 16, 2011

First Submitted That Met QC Criteria

September 20, 2011

First Posted (Estimated)

September 21, 2011

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

March 3, 2025

Last Verified

December 1, 2015

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EFC12153
  • 2011-001897-25 (EudraCT Number)
  • U1111-1121-7170 (Other Identifier: UTN)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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