| Phase 1/2 | 1 | |
| Phase 3 | 2 |
| NCT ID | Title | Condition | Status | Updated |
|---|---|---|---|---|
| Phase 3 | ||||
| NCT06149403 | A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH… | MPS-IH (Hurler Syndrome) | Active, Not Recruiting | 2025-12-22 |
| NCT04283227 | OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) | Lysosomal Storage Diseases | Active, Not Recruiting | 2025-09-05 |
| Phase 1/2 | ||||
| NCT03488394 | Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of… | Mucopolysaccharidosis IH | Active, Not Recruiting | 2026-07-16 |