- Conditions
- Leukemia, High Risk Acute Lymphoblastic Leukemia, High Risk Acute Myeloid Leukemia, Relapse Leukemia, MDS (Myelodysplastic Syndrome), Relapsed Non-Hodgkin Lymphoma, Acquired Aplastic Anemia, Inherited BMF Syndrome, Immunodeficiency, Primary Immune Regulatory Disorder, Hemoglobinopathies, Bone Marrow Failure, Inborn Errors of Metabolism, HLH
- Interventions
- Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3, Phase 2 Maximum Tolerated Dose determined in Phase 1, Phase 2 Established Dose from prior study, NCT03810196
- Device
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 1 Month to 25 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
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