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| NCT06422351 | Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency | NOT_YET_RECRUITING | PHASE2 | 2024-08 | 2029-01 | 2026-11 |
| NCT06092034 | A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease | RECRUITING | PHASE2 | 2023-09-05 | 2032-04 | 2028-04 |
| NCT05885412 | A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM) | RECRUITING | PHASE1 | 2023-08-29 | 2026-09 | 2025-09 |
| NCT04525352 | A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis | TERMINATED | PHASE1 | 2020-11-19 | 2021-05-21 | 2021-05-21 |
| NCT04248439 | Gene Therapy for Fanconi Anemia, Complementation Group A | ACTIVE_NOT_RECRUITING | PHASE2 | 2020-07-15 | 2026-05 | 2026-05 |
| NCT04105166 | Gene Therapy for Pyruvate Kinase Deficiency (PKD) | COMPLETED | PHASE1 | 2020-07-06 | 2025-06-09 | 2025-06-09 |
| NCT04069533 | Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A | ACTIVE_NOT_RECRUITING | PHASE2 | 2019-11-28 | 2025-02 | 2025-02 |
| NCT03812263 | A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I | COMPLETED | PHASE1, PHASE2 | 2019-08-30 | 2023-09-12 | 2023-09-12 |
| NCT03882437 | Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B | UNKNOWN | PHASE1 | 2019-04-17 | 2025-03-24 | 2025-03-24 |
| NCT03825783 | A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I | WITHDRAWN | PHASE1 | 2019-04-15 | 2021-10 | 2021-10 |
| NCT03814408 | A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A | UNKNOWN | PHASE1 | 2019-01-11 | 2022-03 | 2020-12 |