Momelotinib for the Treatment of Myelofibrosis throughout Hematopoietic Cell Transplantation
This phase I trial studies the side effects and best dose of giving momelotinib before, during, and after hematopoietic cell transplantation (HCT) in treating patients with myelofibrosis (MF). Momelotinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. It was recently approved for the treatment of patients with MF and anemia. Doctors think it may work similarly to other drugs used after HCT and that it may improve outcomes after HCT. Giving momelotinib before, during, and after HCT may be safe, tolerable, and/or effective in treating patients with MF.
Inclusion Criteria
- Participants must have pathologically confirmed primary myelofibrosis (PMF) according to World Health Organization (WHO) criteria or secondary myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria * Intermediate-2/ high-risk disease as per Dynamic International Prognostic Scoring System (IPSS) (DIPSS) Plus criteria OR * Intermediate-1 risk disease with at least one of the following unfavorable features known to impact the survival adversely ** Red cell transfusion dependency ** Unfavorable karyotype ** Platelet count ≤ 100 x 10^9/L ** Presence of a high risk molecular marker associated with worsened overall survival (ASXL1, EZH2, IDH1/2, SRSF2, U2AF1, p53)
- Participants do not have to be receiving treatment with Janus-associated kinase (JAK) inhibitors for MF at the time of enrollment. If participants are receiving JAK inhibitor therapy with agents other momelotinib, participants must agree to be switched to momelotinib to begin cycle 1 day 1 on day -7 from HCT (at the initiation of conditioning)
- Age > 18 years
- Participants must be designated to undergo allogeneic HCT with: * Reduced intensity conditioning regimen, and * Peripheral blood stem cells as a graft source
- Participants who will undergo HCT from the following donor types are eligible: * 6/6 (HLA-A, B, DR) fully matched related donor or * 8/8 (HLA-A, B, DR, C) fully matched unrelated donor. Matching in the unrelated setting must be at the allele level
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 (Karnofsky ≥ 60%)
- The effects of momelotinib on the developing human fetus are unknown. Female patients of childbearing potential must have a negative pregnancy test, as measured by serum or urine testing. Women of childbearing potential: must agree to use highly effective contraception prior to the initial dose/start of the first treatment, during the study, and for at least 1 week after the last dose of momelotinib. Male participants with women of child bearing potential partners must agree to use one of the forms of medically acceptable birth control at start of the first treatment, during the study, and for at least 6 months after the last dose
- Ability to understand and the willingness to sign a written informed consent document
Exclusion Criteria
- Known intolerance or hypersensitivity to any JAK inhibitor, including ruxolitinib, fedratinib, pacritinib, momelotinib or any other JAK inhibitor, its metabolites or formulation excipients
- Has had any major surgery within 28 days prior to randomization
- Has received treatment with an investigational agent within 4 weeks of the first dose of study intervention
- Has received immunosuppressive agents within 28 days
- Prior allogeneic transplant for any hematopoietic disorder
- Had accelerated phase or leukemic transformation (≥ 10% blasts in bone marrow any time prior to HCT)
- Has an active, uncontrolled infection
- Has cirrhosis or current unstable liver or biliary disease per investigator assessment defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal/gastric varices, or persistent jaundice
- Known diagnosis of active hepatitis B or hepatitis C
- History of another malignancy(ies), unless: * The participant has been disease-free for at least 2 years and is deemed by the investigator to be at low risk of recurrence of that malignancy, or * The cancer has been deemed indolent with no progression over the last 2 years, and deemed by the investigator to be at low risk for further progression during the course of study and follow-up * The only prior malignancy was cervical cancer in situ and/or basal cell or squamous cell carcinoma of the skin
- Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT]), alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase [SGPT]) and alkaline phosphatase > 3 × institutional upper limit of normal (ULN)
- Total bilirubin > 1.5 mg/dL, with the exception of participants with Gilbert’s Syndrome provided direct bilirubin is ≤ 1.5 x ULN and participant otherwise meets entry criteria
- Calculated creatinine clearance ≤ 60 mL/min (Cockcroft-Gault formula)
- Have current or a history of congestive heart failure New York Heart Association (NYHA) class 3 or 4, or any history of documented diastolic or systolic dysfunction (left ventricular ejection fraction [LVEF] < 40%, as measured by MUGA scan or echocardiogram) or clinically significant arrhythmia not controlled by standard of care therapy
- Not able to take oral medication or having any clinically significant gastrointestinal abnormalities that may alter absorption, e.g., malabsorption syndrome or major resection of the stomach and/or bowels
- Grade 2 or greater peripheral neuropathy
- Pregnant or lactating women, or women planning to become pregnant or initiating breastfeeding
- To exclude women of childbearing potential: who are unwilling or unable to practice highly effective contraception prior to the initial dose/start of the first treatment, during the study, and for at least 1 week after the last dose. Highly effective contraceptive measures include: * Stable use of combined (estrogen and progestogen containing) hormonal contraception (oral, intravaginal, transdermal) or progestogen-only hormonal contraception (oral, injectable, implantable) associated with inhibition of ovulation initiated 2 or more menstrual cycles prior to screening; * Intrauterine device (IUD); intrauterine hormone-releasing system (IUS); * Sexual abstinence; * Intercourse with vasectomized partner (provided that the male vasectomized partner is the sole sexual partner of the women of childbearing potential [WOCBP] study participant and that the vasectomized partner has obtained medical assessment of surgical success for the procedure)
- To exclude sexually active male participants with WOCBP partners who are unwilling to use the one of the following forms of medically acceptable birth control at start of the first treatment, during the study, and for at least 6 months after the last dose: * Vasectomy with medical assessment of surgical success OR consistent use of a condom * Male participants must also agree not to donate sperm while receiving study drug and for at least 6 months after the last dose
- Patients receiving strong CYP 3A4 inducers during study period
- Patients with major ABO mismatch donors only
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT07104799.
Locations matching your search criteria
United States
Massachusetts
Boston
PRIMARY OBJECTIVE:
I. To identify the maximum tolerated dose (MTD) dose of momelotinib when administered during and after allogeneic HCT.
SECONDARY OBJECTIVES:
I. To detect and categorize, according to severity, momelotinib-related toxicities in participants receiving therapy from initiation of conditioning (day -7) through the first year after allogeneic HCT.
II. To characterize the median duration of momelotinib therapy during and after allogeneic HCT, as a measure of feasibility.
III. Median time to neutrophil engraftment for participants receiving momelotinib during allogeneic HCT.
IV. Median time to platelet engraftment for participants receiving momelotinib during allogeneic HCT.
V. Characterize time to red blood cell transfusion independence.
VI. To characterize the cumulative incidence of primary graft failure in participants receiving momelotinib during and after allogeneic HCT.
VII. To characterize the cumulative incidence of acute graft-versus-host disease (GVHD) in participants receiving momelotinib during and after allogeneic HCT.
VIII. To characterize the cumulative incidence of chronic GVHD in participants receiving momelotinib during and after allogeneic HCT.
IX. To characterize non-relapse mortality (NRM) in participants receiving momelotinib during and after allogeneic HCT.
X. To characterize progression-free survival (PFS) in participants receiving momelotinib during and after allogeneic HCT.
XI. To characterize overall survival (OS) in participants receiving momelotinib during and after allogeneic HCT.
XII. To characterize GVHD-free, relapse-free survival (GRFS) in participants receiving momelotinib during and after allogeneic HCT.
EXPLORATORY OBJECTIVES:
I. To longitudinally characterize measurable residual disease and clonal evolution by next-generation sequencing for participants receiving momelotinib during and after allogeneic HCT.
II. To characterize the burden of red blood cell and platelet transfusions for patients receiving momelotinib during and after allogeneic HCT.
III. To characterize quality of life for participants receiving momelotinib from initiation of conditioning through 1 year after allogeneic HCT.
OUTLINE: This is a dose-escalation study of momelotinib followed by a dose-expansion study.
Starting on day -7 prior to standard of care HCT, patients receive momelotinib orally (PO) once daily (QD) on days 1-28 of each cycle. Cycles repeat every 28 days for up to 13 cycles in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo bone marrow biopsy and/or blood sample collection and computed tomography (CT) throughout the study. Patients also undergo echocardiography or multigated acquisition (MUGA) scan during screening.
After completion of study treatment, patients are followed up at 30 days and then every 6 months for up to 2 years.
Trial PhasePhase I
Trial Typetreatment
Lead OrganizationDana-Farber Harvard Cancer Center
Principal InvestigatorGabriela Soriano Hobbs
- Primary ID25-501
- Secondary IDsNCI-2026-00534
- ClinicalTrials.gov IDNCT07104799