A Phase 2, Randomized, Open-label, Study of Momelotinib in Participants With Anemia Due to Low-risk Myelodysplastic Syndrome
The goal of this clinical trial is to determine if momelotinib is safe and effective for people with low-risk myelodysplastic syndromes (LR-MDS). The trial will also examine how the body processes the drug. Participants will receive different doses of momelotinib to find the best dose by evaluating effectiveness in improving red blood cell transfusion requirements and safety.
• Age ≥18 years or of legal age of consent in the jurisdiction in which the study is taking place, at the time of signing the informed consent form (ICF).
• Documented diagnosis of MDS according to the World Health Organization classifications with an Revised International Prognostic Scoring System (IPSS-R) classification of very low, low, or intermediate risk disease, with an overall risk score ≤3.5 and bone marrow blasts \< 5%.
• Received only one prior line of treatment with either Erythropoiesis-stimulating agent (ESA) or luspatercept for LR-MDS-related anemia that is relapsed/refractory to therapy. Participants intolerant OR ineligible to prior ESA or luspatercept will fulfill this inclusion criterion provided the definition below is met.
‣ Refractory to prior treatment: documentation of loss of erythroid (E) response or never achieved HI-E response as defined by the IWG 2018 criteria.
⁃ Intolerant to prior treatment: documentation of reasons for discontinuation of prior ESA containing regimen, either as single agent or combination (e.g., G-CSF) or luspatercept due to intolerance or adverse event.
⁃ ESA ineligible: low chance of response to ESA based on endogenous serum erythropoietin level \> 200 U/L for participants not previously treated with ESAs.
• Red blood cell transfusion dependence, defined as requiring ≥3 units of Packed red blood cells (pRBC) transfused over 16-week period in at least 2 transfusions episodes during the 16 weeks preceding randomization. Documentation of a participant's transfusion policy during this 16-week period is required.
• A female participant is eligible to participate if she is not pregnant or breastfeeding and one of the following conditions applies:
• Is a woman of non-childbearing potential (WONCBP). OR
• Is a woman of childbearing potential (WOCBP) and using a contraceptive method.
• Is capable of giving signed informed consent.
• Eastern Cooperative Oncology Group performance status ≤2.
• Adequate organ function.