8 completed, 6 active not recruiting, 1 enrolling by invitation, 1 recruiting. 8 Phase 2, 4 Phase 1, 4 Phase 3, 1 Early Phase 1.
Follow X-Linked Retinitis Pigmentosa: new trials by email Literature Review of X-Linked Retinitis Pigmentosa trials Research Report on X-Linked Retinitis Pigmentosa
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP) AAV5-hRKp.RPGR, No intervention (Follow-Up assessment) - X-Linked Retinitis Pigmentosa | Phase 2 | Active not recruiting | MeiraGTx Ocular UK Ltd | 24 | 2024-10-10 | 2026-09-25 |
| A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP rAAV2tYF-GRK1-hRPGRco, Control - X-Linked Retinitis Pigmentosa | Phase 2 / Phase 3 | Active not recruiting | Beacon Therapeutics | 85 | 2024-03-14 | 2026-04-07 |
| A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN) AGTC-501 (high dose and standard corticosteroid regimen), AGTC-501 (low dose and standard corticosteroid regimen), AGTC-501 (high dose and modified corticosteroid regimen) - X-Linked Retinitis Pigmentosa | Phase 2 | Enrolling by invitation | Beacon Therapeutics | 24 | 2023-11-14 | 2024-10-30 |
| Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa FT-002 - X-Linked Retinitis Pigmentosa | Early Phase 1 | Recruiting | Frontera Therapeutics | 18 | 2023-02-01 | 2023-05-24 |
| An Observational Study of Japanese Participants With X-linked Retinitis Pigmentosa X-Linked Retinitis Pigmentosa | Completed | Janssen Pharmaceutical K.K. | 15 | 2021-07-26 | 2025-04-25 | |
| A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE) rAAV2tYF-GRK1-RPGR - X-Linked Retinitis Pigmentosa | Phase 2 | Active not recruiting | Beacon Therapeutics | 14 | 2021-04-13 | 2024-08-12 |
| Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Genetic: AAV5-hRKp.RPGR Intermediate Dose, Genetic: AAV5-hRKp.RPGR Low Dose - X-Linked Retinitis Pigmentosa | Phase 3 | Active not recruiting | MeiraGTx Ocular UK Ltd | 97 | 2020-12-04 | 2026-09-25 |
| Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Genetic: AAV5-hRKp.RPGR - X-Linked Retinitis Pigmentosa | Phase 3 | Completed | MeiraGTx Ocular UK Ltd | 105 | 2020-12-04 | 2026-09-24 |
| 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) 4D-125 IVT Injection, Observational - X-Linked Retinitis Pigmentosa | Phase 1 / Phase 2 | Active not recruiting | 4D Molecular Therapeutics | 21 | 2020-06-09 | 2025-03-21 |
| Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa BIIB111, BIIB112 - Choroideremia, X-Linked Retinitis Pigmentosa | Phase 3 | Completed | NightstaRx Ltd, a Biogen Company | 277 | 2018-06-04 | 2026-07-06 |
| Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations rAAV2tYF-GRK1-RPGR - X-Linked Retinitis Pigmentosa | Phase 1 / Phase 2 | Active not recruiting | Beacon Therapeutics | 29 | 2018-04-16 | 2024-05-20 |
| Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP) X-Linked Retinitis Pigmentosa | Completed | Beacon Therapeutics | 14 | 2017-12-01 | 2023-01-11 | |
| Natural History of the Progression of X-Linked Retinitis Pigmentosa Other: Assessments - X-Linked Retinitis Pigmentosa | Completed | NightstaRx Ltd, a Biogen Company | 201 | 2017-09-13 | 2023-02-10 | |
| Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR) AAV5-RPGR - X-Linked Retinitis Pigmentosa | Phase 1 / Phase 2 | Completed | MeiraGTx UK II Ltd | 49 | 2017-07-31 | 2025-01-08 |
| Long-term Follow-up Gene Therapy Study for RPGR- XLRP AAV5-hRKp.RPGR - X-Linked Retinitis Pigmentosa | Completed | MeiraGTx Ocular UK Ltd | 42 | 2017-07-31 | 2026-09-25 | |
| A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112 BIIB112 - X-Linked Retinitis Pigmentosa | Phase 1 / Phase 2 | Completed | Biogen | 50 | 2017-03-08 | 2024-01-18 |
Retinal Degeneration (5) · Choroideremia (1) · Cone-Rod Dystrophy, X-Linked, Type 1 (1) · Rare Diseases (1)
Eye Diseases (6) · Eye Diseases, Hereditary (6) · Retinal Diseases (5) · Choroid Diseases (1) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1) · Disease Attributes (1) · Genetic Diseases, Inborn (1) · Genetic Diseases, X-Linked (1) · Pathologic Processes (1) · Pathological Conditions, Signs and Symptoms (1) · Uveal Diseases (1)
Cotoretigene Toliparvovec (3) · Watchful Waiting (1)
Beacon Therapeutics (5) · MeiraGTx Ocular UK Ltd (4) · NightstaRx Ltd, a Biogen Company (2) · 4D Molecular Therapeutics (1) · Biogen (1) · Frontera Therapeutics (1) · Janssen Pharmaceutical K.K. (1) · MeiraGTx UK II Ltd (1)
United States (14) · United Kingdom (9) · Canada (5) · France (4) · Netherlands (4) · Denmark (3) · Belgium (2) · Finland (2) · Germany (2) · Israel (2) · Italy (2) · Spain (2) · Switzerland (2) · Australia (1) · Brazil (1)
Carrie Reichley (1) · Gezhi Xu (1) · James Bainbridge, Prof (1) · Matthew Feinsod, MD (1) · Ruifang Sui (1) · Schonmei Lee, MD (1)
PMID 40547876, PMID 36757689, PMID 32094925, PMID 37895218, PMID 38423215, PMID 38871269, PMID 39680391, PMID 40535564
2016 (1), 2017 (1), 2018 (1)