- Conditions
- Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes, Immunodeficiencies, Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome
- Interventions
- Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
- Biological
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- Up to 30 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2030
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 19, 2026 · Synced May 21, 2026, 8:00 PM EDT