| NCT Number | NCT01962415 |
| Lead Sponsor | Paul Szabolcs |
| Conditions | Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA) |
| Enrollment | 100 participants |
| Start Date | 2014-02-04 |
| Primary Completion | 2026-11 (estimated) |
| Study Completion | 2027-11 (estimated) |
| Updated on ClinicalTrials.gov | 2025-12-15 |