TriangleNCT01437787 · CITED IN SOURCE DOCUMENTS
Phase III Study of SAR302503 in Intermediate-2 and High Risk Patients With Myelofibrosis
An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.
- Phase
- PHASE3
- Status at capture
- TERMINATED
- Registry last update
- 2026-08-11
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.
Open the original ClinicalTrials.gov record → · Download preserved record
What did the study report?
Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.
Participant flow
Baseline characteristics
Response Rate (RR): Percentage of Participants Who Had a >=35% Reduction From Baseline in Volume of Spleen Size at The End Cycle 6
Symptom Response Rate (SRR): Percentage of Participants With >=50% Reduction From Baseline in the Total Symptom Score at End of Cycle 6
Percentage of Participants Who Had >=25% Reduction From Baseline in Volume of Spleen Size at End of Cycle 6
Adverse events
Limitations, agreements, and source contact
Who could take part
- eligibility 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 (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.
- healthy Volunteers
- false
- minimum Age
- 18 Years
- sex
- ALL
- std Ages
- ADULT
- OLDER_ADULT
Treatment arms and interventions
- arm Groups
- description
- once daily X 28 days, orally, empty stomach, approximately same time each day
- intervention Names
- Drug: Placebo
- label
- Placebo comparator
- type
- PLACEBO_COMPARATOR
- description
- once daily X 28 days, orally, empty stomach, approximately same time each day
- intervention Names
- Drug: SAR302503
- label
- SAR302503 400 mg
- type
- EXPERIMENTAL
- description
- once daily X 28 days, orally, empty stomach, approximately same time each day
- intervention Names
- Drug: SAR302503
- label
- SAR302503 500 mg
- type
- EXPERIMENTAL
- interventions
- arm Group Labels
- SAR302503 400 mg
- SAR302503 500 mg
- description
- Pharmaceutical form:capsule Route of administration: oral
- name
- SAR302503
- type
- DRUG
- arm Group Labels
- Placebo comparator
- description
- Pharmaceutical form:capsule Route of administration: oral
- name
- Placebo
- type
- DRUG
Study design
- allocation
- RANDOMIZED
- intervention Model
- CROSSOVER
- masking Info
- masking
- DOUBLE
- who Masked
- PARTICIPANT
- INVESTIGATOR
- primary Purpose
- TREATMENT
Enrollment
- count
- 289
- type
- ACTUAL
Registered outcome plans (not posted results)
- primary Outcomes
- description
- 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).
- measure
- Response Rate (RR): Percentage of Participants Who Had a >=35% Reduction From Baseline in Volume of Spleen Size at The End Cycle 6
- time Frame
- Baseline, Week 24
- secondary Outcomes
- description
- 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.
- measure
- Symptom Response Rate (SRR): Percentage of Participants With >=50% Reduction From Baseline in the Total Symptom Score at End of Cycle 6
- time Frame
- Baseline, Week 24
- description
- 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.
- measure
- Percentage of Participants Who Had >=25% Reduction From Baseline in Volume of Spleen Size at End of Cycle 6
- time Frame
- Baseline, Week 24
Full study description
- 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.
Source references
- references
- citation
- 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.
- pmid
- 26181658
- type
- DERIVED
Source notices and limitations
Discovery and provenance
- release id
- ctgov-registry-7ad944f6deaf1f5cd6122126
- edge id
- nct-edge-a87e4306480b2dc2fb10
- requested nct id
- NCT01437787
- primary nct id
- NCT01437787
- source kind
- fda_spl_label
- source record id
- c328016b-fc3e-4d6f-99b3-8d2c13b362f3
- source file sha256
- a9df7a5786dc51557d38f2a0881b5b5e9ae02ae396a6798e39ac96f586bcc5e6
- payload sha256
- 2a4361704593ba80b8f644c9130cbb32863e95a0aca9e4c51003434a674fb7d7
- payload
- application numbers
- NDA212327
- clinical indication matching status
- not_inferred
- end exclusive
- true
- id
- nct-edge-a87e4306480b2dc2fb10
- link basis
- explicit_NCT_identifier_in_acquired_source
- nct id
- NCT01437787
- occurrences
- context exact
- 14 CLINICAL STUDIES JAKARTA JAKARTA (NCT01437787) was a double-blind, randomized, placebo-controlled trial in patients with intermediate-2 or high-risk myelofibrosis, post-polycythemia vera myelofibrosis or p
- end
- 48
- exact text
- NCT01437787
- start
- 37
- offset unit
- unicode_code_points
- source file
- /tmp/cancer-full-research/indications/raw/labels-00600.json
- source file sha256
- a9df7a5786dc51557d38f2a0881b5b5e9ae02ae396a6798e39ac96f586bcc5e6
- source json pointer
- /results/53/clinical_studies/0
- source kind
- fda_spl_label
- source record id
- c328016b-fc3e-4d6f-99b3-8d2c13b362f3
- source string basis
- parsed_JSON_string_unmodified
- source string sha256
- c09d57a0f64fb4ba024bb39dda637feccc2c520592f2b489a3835893c8375ef5
- spl effective time
- 20260720
- spl set id
- f0f55a2a-4e0c-4cba-8571-03e1424486d7
Preserved source evidence · Independent clinical review pending · Not medical advice