This Phase 2, multicenter, open-label study is evaluating CK0801 for the treatment of aplastic anemia in adults with persistent transfusion dependence after at least one prior line of therapy or intolerance to standard-of-care treatment. CK0801 is an allogeneic cord blood-derived regulatory T-cell therapy administered intravenously. The study is designed to assess safety and clinical activity, including hematologic response, transfusion independence, duration of response, survival outcomes, and patient-reported outcomes. Exploratory assessments include immune reconstitution, biomarkers, pharmacokinetics, immunogenicity, and donor-specific antibodies. The primary endpoint is overall response at Day 180.
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Primary Outcome-Overall Response Rate (ORR) at Day 180
Timeframe: Day +180
Overall Response Rate at Additional Timepoints
Timeframe: Days 28, 56, 100, and 365