CompletedPhase 2

Phase 2 Study in Japanese Patients With Intermediate-2 or High Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis With Splenomegaly

Japan8 participantsStarted 2012-11

Plain-language summary

Primary Objective: \- To evaluate the efficacy of daily oral doses of 300 mg, 400 mg, and 500 mg SAR302503 and combined for the response rate defined with the ≥35% reduction of spleen volume as determined by magnetic resonance imaging (MRI or computed tomography scan \[CT\] in patients with contraindications for MRI). Secondary Objectives: * To evaluate the safety of SAR302503 for both pooled (300, 400, and 500mg) and individual doses population. * To evaluate the pharmacokinetics (PK) of SAR302503 after single and repeat-dose. * To evaluate the effect on Myelofibrosis (MF)-associated symptoms (Key MF symptoms) as measured by the modified Myelofibrosis Symptom Assessment Form (MFSAF). * To evaluate the durability of splenic response. * To evaluate the effect of SAR302503 on bone marrow with regard to changes on reticulin fibrosis.

Who can participate

Age range

20 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria : * Diagnosis of primary or post-polycythemia vera or post-essential thrombocythemia myelofibrosis * Myelofibrosis classified as high-risk or intermediate-risk level 2 * Enlarged spleen, palpable at least 5 cm below costal margin * Active symptoms of myelofibrosis * At least 20 years of age * Eastern Collaborative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2 at study entry * Absence of active malignancy other than myelofibrosis * Written informed consent to participate. Exclusion criteria: * Splenectomy. * Any recent chemotherapy (eg, hydroxyurea), immunomodulatory drug therapy (eg, thalidomide, interferon-alpha), immunosuppressive therapy, corticosteroids \>10 mg/day prednisone or equivalent, or growth factor treatment (eg, erythropoietin), hormones (eg, androgens, danazol) within 14 days prior to initiation of study drug. * Major surgery therapy within 28 days or radiation including spleen radiation within 6 months prior to initiation of study drug. * Concomitant treatment with or use of pharmaceutical or herbal agents known to be moderate or severe inhibitors or inducers CYP3A4. * Active acute infection requiring antibiotics. * Uncontrolled congestive heart failure (New York Heart Association Classification 3 or 4), angina, myocardial infarction, cerebrovascular accident, coronary/peripheral artery bypass graft surgery, transient ischemic attack, or pulmonary embolism within 3 months prior to initiation of study drug. * Participation in…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Response Rate (RR), defined as the proportion of subjects who have a ≥35% reduction as measured by MRI (or CT scan in subjects with contraindications for MRI). - Time Frame:

Timeframe: 24 weeks

Trial details

NCT IDNCT01692366

SponsorBristol-Myers Squibb

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2014-03

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