- Conditions
- Metabolic Disorders, Hematologic, Immune, or Bone Marrow Disorders, Hemoglobinopathies, Non-malignant Disorders
- Interventions
- Treatment Plan 1: Stratum 1, Treatment Plan 2: Strata 2, 3, or 4, GVHD Regimen A: UCB Recipients, GVHD Regimen B: BM Recipients
- Drug
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- Up to 20 Years
- Enrollment
- 220 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2031
- U.S. locations
- 25
- States / cities
- Phoenix, Arizona • Orange, California • San Diego, California + 20 more
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