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

Sierra Oncology

Status and phase: Completed; Phase 2

Conditions: Primary Myelofibrosis (PMF); Postessential Thrombocythemia (Post-ET) Myelofibrosis; Post-polycythemia Vera (Post-PV) Myelofibrosis

Treatments: Drug: MMB

Study type: Interventional

Funder types: Industry

Identifiers

NCT02515630

GS-US-352-1672

Details and patient eligibility

About

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

Enrollment: 41 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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41 participants in 1 patient group

Momelotinib

Experimental group

Description:

MMB for 24 weeks (± 7 days)

Treatment:

Drug: MMB