| NCT ID | Title | Condition | Status |
|---|---|---|---|
| Phase 2 | |||
| NCT06092034 | A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease | Danon Disease | Recruiting |
| NCT04248439 | Gene Therapy for Fanconi Anemia, Complementation Group A | Fanconi Anemia Complementation Group A | Active, Not Recruiting |
| Phase 1 | |||
| NCT05885412 | A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated… | PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) | Recruiting |
| NCT07137338 | A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in… | Dilated Cardiomyopathy (DCM) | Recruiting |
| Not specified | |||
| NCT06644742 | PKP2-ACM Natural History Study | Cardiomyopathies | Recruiting |
| NCT06214507 | Danon Disease Natural History Study | Danon Disease | Recruiting |
| NCT06282432 | Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) | Leukocyte Adhesion Deficiency | Active, Not Recruiting |
| NCT07486752 | BAG3-DCM Natural History Study | Dilated Cardiomyopathy (DCM) | Not Yet Recruiting |
| NCT04437771 | Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene… | Fanconi Anemia Complementation Group A | Active, Not Recruiting |
| NCT07527975 | Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the… | Fanconi Anemia | Enrolling by Invitation |