Hematopoietic NeoplasmPhase 3JAKARTANCT01437787
Phase III Study of SAR302503 in Intermediate-2 and High Risk Patients With Myelofibrosis
A Phase 3 study testing SAR302503 in people with Hematopoietic Neoplasm, run by Bristol-Myers Squibb at 101 sites in 25 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematopoietic Neoplasm
- What is being tested?
- SAR302503
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 289 participants
- Where
- 101 sitesin 25 countries
- Study dates
- 31 Dec 2011to 30 Jun 2014
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
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- 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
Who cannot join
- 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
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- 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.
Trial locations and contacts
101 sites in 25 countries.
101 sites in 25 countries
United Kingdom10 sites
Investigational Site Number 826006
Belfast, BT9 7AB
Investigational Site Number 826003
Birmingham, B9 5SS
Investigational Site Number 826002
Glasgow, G12 0YN
Investigational Site Number 826004
Leeds, LS9 7TF
Investigational Site Number 826001
London, SE1 9RT
Investigational Site Number 826005
London, W12 0HS
Investigational Site Number 826007
Manchester, M20 4BX
Investigational Site Number 826008
Newcastle upon Tyne, NE7 7DN
Investigational Site Number 826009
Oxford, OX3 7LJ
Investigational Site Number 826010
Southampton, SO16 6YD
United States9 sites
Investigational Site Number 840013
Baton Rouge, Louisiana, 70808
Investigational Site Number 840002
Canton, Ohio, 44718
Investigational Site Number 840004
Houston, Texas, 77030
Investigational Site Number 840001
La Jolla, California, 92093
Investigational Site Number 840012
La Jolla, California, 92093
Investigational Site Number 840006
Los Angeles, California, 90033
Investigational Site Number 840009
Newark, New Jersey, 07112
Investigational Site Number 840008
Rochester, Minnesota, 55905
Investigational Site Number 840014
Scottsdale, Arizona, 85259-5499
France7 sites
Investigational Site Number 250006
Marseille, 13273
Investigational Site Number 250005
Nantes, 44093
Investigational Site Number 250004
Nîmes, 30029
Investigational Site Number 250002
Pierre-Bénite, 69495
Investigational Site Number 250007
Poitiers, 86000
Investigational Site Number 250003
Toulouse, 31000
Investigational Site Number 250001
Villejuif, 94805
Russia7 sites
Investigational Site Number 643009
Moscow, 125167
Investigational Site Number 643001
Moscow, 125284
Investigational Site Number 643010
Nizhny Novgorod, 603126
Investigational Site Number 643008
Petrozavodsk, 185019
Investigational Site Number 643005
Saint Petersburg, 191024
Investigational Site Number 643004
Saint Petersburg, 197341
Investigational Site Number 643007
Volgograd, 400138
South Korea7 sites
Investigational Site Number 410002
Bundang-Gu, 463-707
Investigational Site Number 410004
Seoul, 110-744
Investigational Site Number 410003
Seoul, 120-752
Investigational Site Number 410001
Seoul, 135-710
Investigational Site Number 410005
Seoul, 137-701
Investigational Site Number 410006
Seoul, 138-878
Investigational Site Number 410007
Seoul
Italy6 sites
Investigational Site Number 380002
Bergamo, 24127
Investigational Site Number 380007
Bologna, 40138
Investigational Site Number 380004
Florence, 50134
Investigational Site Number 380001
Pavia, 27100
Investigational Site Number 380006
Pavia, 27100
Investigational Site Number 380003
Varese, 21100
Australia5 sites
Investigational Site Number 036001
Box Hill, 3128
Investigational Site Number 036005
Herston, 4029
Investigational Site Number 036003
Randwick, 2031
Investigational Site Number 036004
Tweed Heads, 2485
Investigational Site Number 036002
Wodonga, 3690
Hungary5 sites
Investigational Site Number 348002
Budapest, 1097
Investigational Site Number 348001
Debrecen, 4032
Investigational Site Number 348007
Győr, 9023
Investigational Site Number 348006
Kecskemét, 6000
Investigational Site Number 348003
Miskolc, 3529
Poland5 sites
Investigational Site Number 616005
Brzozów, 36-200
Investigational Site Number 616002
Gdansk, 80-952
Investigational Site Number 616006
Lodz, 93-510
Investigational Site Number 616010
Warsaw, 02-106
Investigational Site Number 616003
Wroclaw, 50-367
Romania5 sites
Investigational Site Number 642003
Brasov
Investigational Site Number 642004
Bucharest, 022328
Investigational Site Number 642002
Bucharest, 030171
Investigational Site Number 642006
Bucharest
Investigational Site Number 642001
Timișoara
Germany4 sites
Investigational Site Number 276006
Aachen, 52074
Investigational Site Number 276007
Bonn, 53127
Investigational Site Number 276008
Dresden, 01307
Investigational Site Number 276001
Mannheim, 68167
Portugal4 sites
Investigational Site Number 620005
Coimbra, 3000-075
Investigational Site Number 620004
Lisbon, 1169-050
Investigational Site Number 620001
Lisbon, 1649-035
Investigational Site Number 620003
Porto, 4200-072
Brazil3 sites
Investigational Site Number 076002
Jaú, 17210-120
Investigational Site Number 076004
Porto Alegre, 90110-270
Investigational Site Number 076001
Rio de Janeiro, 20230-130
Canada3 sites
Investigational Site Number 124001
Montreal, H1T 2M4
Investigational Site Number 124003
Montreal, H2W 1S6
Investigational Site Number 124002
Saint John, E2L 4L2
Taiwan3 sites
Investigational Site Number 158002
Changhua, 500
Investigational Site Number 158003
Kaohsiung City, 833
Investigational Site Number 158001
Taipei, 112
Belgium2 sites
Investigational Site Number 056003
Antwerp, 2060
Investigational Site Number 056001
Leuven, 3000
Ireland2 sites
Investigational Site Number 372002
Dublin, DUBLIN 8
Investigational Site Number 372001
Galway
Israel2 sites
Investigational Site Number 376003
Haifa, 31048
Investigational Site Number 376002
Tel Litwinsky, 52621
Lithuania2 sites
Investigational Site Number 440001
Kaunas, LT-50009
Investigational Site Number 440002
Klaipėda, LT-92288
Singapore2 sites
Investigational Site Number 702002
Singapore, 119228
Investigational Site Number 702001
Singapore, 169608
South Africa2 sites
Investigational Site Number 710003
Johannesburg, 2013
Investigational Site Number 710002
Parktown, 2193
Spain2 sites
Investigational Site Number 724001
Barcelona, 08036
Investigational Site Number 724002
Madrid, 28046
Sweden2 sites
Investigational Site Number 752001
Stockholm, 14186
Investigational Site Number 752002
Uddevalla, 451 80
About this study
Official title: 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).
Read more
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.
Read the 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.
- Conditions
- Hematopoietic Neoplasm
- Being tested
- SAR302503, Placebo
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SAR302503Drug
Pharmaceutical form:capsule
Route of administration: oral
PlaceboDrug
Pharmaceutical form:capsule
Route of administration: oral
| Study group | Role | What they receive |
|---|---|---|
| Placebo comparator | Placebo comparator | once daily X 28 days, orally, empty stomach, approximately same time each day Receives: Drug: Placebo |
| SAR302503 400 mg | Experimental | once daily X 28 days, orally, empty stomach, approximately same time each day Receives: Drug: SAR302503 |
| SAR302503 500 mg | Experimental | once daily X 28 days, orally, empty stomach, approximately same time each day Receives: Drug: SAR302503 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response Rate (RR): Percentage of Participants Who Had a >=35% Reduction From Baseline in Volume of Spleen Size at The End Cycle 6
Spleen volume was determined by magnetic resonance imaging (MRI) (or computed tomography scan in participants with contraindications for MRI) at baseline and at the end of cycle 6 with a confirmatory scan approximately 4 weeks after the end of Cycle 6. The MRI or CT imaging results reviewed in a blinded manner by an Independent Review Committee (IRC). Analysis was performed on intent-to-treat (ITT) population defined as all randomized participants who signed informed consent form (ICF).
Measured: Baseline, Week 24
Secondary outcomes (2)
Symptom Response Rate (SRR): Percentage of Participants With >=50% Reduction From Baseline in the Total Symptom Score at End of Cycle 6
Total symptom score is the averaged value of the daily scores for each of 6 key Myelofibrosis (MF) associated Symptom items (night sweats, pruritus, abdominal discomfort, early satiety [filling up quickly when you eat], pain under ribs on left side, and bone or muscle pain), each item measured on a scale from 0 (absent) to 10 (worst imaginable). A higher score indicates worse symptoms. Analysis was performed on ITT population. Number of participants analysed= participants with available data at end of cycle 6.
Measured: Baseline, Week 24
Percentage of Participants Who Had >=25% Reduction From Baseline in Volume of Spleen Size at End of Cycle 6
Spleen volume was determined by magnetic resonance imaging (MRI) (or computed tomography scan in subjects with contraindications for MRI) at baseline and at the end of cycle 6 with a confirmatory scan approximately 4 weeks after the end of Cycle 6. Analysis was performed on ITT population.
Measured: Baseline, Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2011 (estimated)
- Study start31 Dec 2011
- Primary completion30 Jun 2014
- Study completion30 Jun 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hematopoietic NeoplasmStudies of the same condition, open and under way.
- More trials run by Bristol-Myers SquibbThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 289 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bristol-Myers Squibb
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01437787
- Sponsor's protocol ID
- EFC12153
- Other IDs
- 2011-001897-25, U1111-1121-7170
- Acronym
- JAKARTA
- Status last verified
- Jul 2026
- First submitted
- 16 Sep 2011
- Last updated
- 11 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
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.
PubMed 26181658