Momelotinib in Transfusion-Dependent Adults With Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF)

• Status: Completed
• Trial end date: 2017-08-15
• Target enrollment: 0
• Participant gender: All

Summary

This study will evaluate the transfusion independence response rate in transfusion-dependent adults with myelofibrosis after treatment with momelotinib (MMB).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Gilead Sciences; Sierra Oncology, Inc.
• Treatments: N-(cyanomethyl)-4-(2-((4-(4-morpholinyl)phenyl)amino)-4-pyrimidinyl)benzamide

Criteria

Key Inclusion 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

Key Exclusion Criteria:

• 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

Note: Other protocol defined Inclusion/Exclusion criteria may apply.