| NCT Number | NCT04528355 |
| Lead Sponsor | Paul Szabolcs |
| Conditions | Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions |
| Enrollment | 50 participants |
| Start Date | 2020-08-20 |
| Primary Completion | 2027-12-31 (estimated) |
| Study Completion | 2028-06-30 (estimated) |
| Updated on ClinicalTrials.gov | 2026-01-13 |