A Phase 2, Open-label, Translational Biology Study of Momelotinib in Transfusion-Dependent Subjects With Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF)
Description
Brief Summary
This study will evaluate the transfusion independence response rate in transfusion-dependent
adults with myelofibrosis after treatment with momelotinib (MMB).
Phase
Phase 2 - takes the treatment one step further, assessing the activity of a particular therapy in a disease, often building upon leads from the Phase I trial. While patients are generally required to be previously untreated, participation in a Phase II trial doesn't usually preclude the patient from getting the standard treatment after they've received the investigational agent. At best they are allowed to get a new drug they wouldn't be able to get otherwise that may turn out to be better for their disease.Inclusion and Exclusion Criteria
- Diagnosis of PMF or Post PV/ET-MF
- Requires myelofibrosis therapy, in the opinion of the investigator
- High risk OR intermediate-2 risk defined by dynamic international prognostic scoring system (DIPSS) OR intermediate-1 risk defined by DIPSS and associated with symptomatic splenomegaly and/or hepatomegaly
- Transfusion dependent at baseline, defined as ≥ 4 U red blood cell (RBC) transfusion in the 8 weeks prior to first dose of MMB
- Acceptable organ function as evidenced by the following:
- Platelet Count ≥ 50 x 10^9/L
- Aspartate aminotransferase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤ 3 x upper limit of normal (ULN) or AST or ALT ≤ 5 x ULN if liver is involved by disease process as judged by the investigator
- Serum creatinine ≤ 2.0 mg/dL or calculated creatinine clearance of ≥ 60 mL/min
- Direct bilirubin ≤ 2.0 x ULN
- Life expectancy of > 24 weeks
- Males and females of childbearing potential who engage in heterosexual intercourse must agree to use protocol specified method(s) of contraception
- Lactating females must agree to discontinue nursing before MMB administration
- Able to understand and willing to sign the informed consent form
- Prior splenectomy
- Splenic irradiation within 3 months prior to the first dose of MMB
- Prior treatment with MMB
- Known positive status of human immunodeficiency virus (HIV)
- Chronic active or acute viral hepatitis A, B, or C infection (testing required for hepatitis B and C), or hepatitis B or C carrier
- Use of strong cytochrome P450 3A4 (CYP3A4) inducer within 2 weeks prior to the first dose of MMB
- Uncontrolled intercurrent illness per protocol
- Treatment with a Janus kinase (JAK) inhibitor within 21 days of the planned first dose of MMB
- Presence of peripheral neuropathy ≥ Common Terminology Criteria for Adverse Events (CTCAE) Grade 2
- Unwilling or unable to undergo a MRI per requirements in the study protocol
- Unwilling to consent to genomics sampling
Sites
Please contact the trial administrator to learn more about where you can participate in this trial. Please use the contact form on the right side.
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