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. The study is comprised of two parts: Part 1: Participants will receive different doses of momelotinib to find the best dose by evaluating effectiveness in improving red blood cell transfusion requirements and safety. Part 2: Participants will receive dose selected from Part 1 to assess its impact on improving red blood cell transfusion requirements and safety in LR-MDS.
⁃ • 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.
• Received prior treatment with Erythropoiesis-stimulating agent (ESA) OR luspatercept for LR-MDS-related anemia that is relapsed/refractory to therapy. Participants intolerant 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.
• Red blood cell transfusion dependence, defined as requiring an average of ≥4 units of Packed red blood cells (pRBC) transfused over an 8-week period 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:
• a. Is a woman of non-childbearing potential (WONCBP). OR b. 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.