A phase IIIb, open-label, single arm study to evaluate the efficacy and safety of luspatercept in patients with lower-risk MDS and ring-sideroblastic phenotype (MDS-RS)
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Subject is 18 years of age or older at the time of signing the informed consent form (ICF)
✓. Subject is able to understand and voluntarily sign the ICF prior to any study-related assessments/procedures being conducted
✓. Subject has documented diagnosis of MDS according to WHO classification that meets IPSS-R classification\[3\] of very low-, low-, or intermediate-risk disease, and the following:
✓. Subject must be one of the following:
✓. If previously treated with ESAs or G-CSF/granulocyte-macrophage colony-stimulating factor (GM-CSF), both agents must be discontinued ≥ 4 weeks prior to the date of starting treatment with the Investigational medicinal Product (IMP) in this study
✓. Required RBC transfusions, as documented by the following criteria:
✓. Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2
✓. A female of childbearing potential (FCBP) for this study is defined as a sexually mature woman who: (1) has not undergone a hysterectomy or bilateral oophorectomy; or (2) is not naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (i.e. had menses at any time in the preceding 24 consecutive months). An FCBP participating in the study must:
Exclusion criteria
✕. Prior therapy with disease modifying agents other than HMA or LEN for underlying MDS disease
✕. Previously treated with either luspatercept or sotatercept
What they're measuring
1
RBC-TI rate according to IWG 2018 modified criteria
✕. Secondary MDS, i.e. MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases
✕. Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
✕. Prior allogeneic or autologous stem cell transplant
✕. Known history of diagnosis of acute myeloid leukemia (AML)
✕. Use of any of the following within 5 weeks prior to the first dose of the IMP in this study:
✕. Uncontrolled hypertension, defined as repeated elevations of diastolic blood pressure (DBP) ≥ 100 mmHg despite adequate treatment