CK0801 for Treatment of Aplastic Anemia

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.

Inclusion Criteria: * Age 18 years or older * Diagnosis of aplastic anemia * Transfusion dependent after at least one prior line of treatment, or intolerance to standard-of-care treatment * Female subjects of childbearing potential must have a negative urine or serum pregnancy test * Agrees to comply with all protocol-required procedures, including study-related assessments, visits, and long-term follow-up * Willing and able to provide written informed consent Exclusion Criteria: * Known HIV seropositivity * Uncontrolled infection not responding to appropriate antimicrobial agents after 7 days of therapy; the Protocol PI is the final arbiter of eligibility * Uncontrolled intercurrent illness that, in the opinion of the investigator, would place the subject at greater risk of severe toxicity and/or impair the activity of CK0801 * Pregnant or breastfeeding * Unable to provide consent or, in the opinion of the investigator, unlikely to fully comply with protocol requirements