7 completed, 4 active not recruiting, 2 unknown, 1 enrolling by invitation. 10 Phase 2, 6 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 10 | Active not recruiting 4, Completed 3, Unknown 2, Enrolling by invitation 1 |
| Phase 1 | 6 | Completed 3, Unknown 2, Active not recruiting 1 |
| Phase 3 | 1 | Active not recruiting 1 |
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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 Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa Adeno-associated virus vector expressing a human RPGR gene - X-Linked Retinitis Pigmentosa (XLRP) | Phase 2 | Active not recruiting | Beacon Therapeutics | 10 | 2025-09-10 | 2026-06-25 |
| 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 |
| 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 |
| 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 | |
| Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial) AGTC-402 - Achromatopsia | Phase 1 / Phase 2 | Unknown | Beacon Therapeutics | 24 | 2017-08-03 | 2022-07-22 |
| Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial) rAAV2tYF-PR1.7-hCNGB3 - Achromatopsia | Phase 1 / Phase 2 | Unknown | Beacon Therapeutics | 32 | 2016-04-11 | 2022-07-22 |
| Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS) rAAV2tYF-CB-hRS1 - X-linked Retinoschisis | Phase 1 / Phase 2 | Completed | Beacon Therapeutics | 27 | 2015-05-01 | 2023-06-12 |
| Clinical and Genetic Characterization of Individuals With Achromatopsia Achromatopsia | Completed | Beacon Therapeutics | 56 | 2013-06-01 | 2017-10-13 | |
| Clinical Evaluation of Patients With X-linked Retinoschisis Dorzolamide 2% TID or brinzolamide 1% TID - X-linked Retinoschisis, XLRS | Completed | Beacon Therapeutics | 66 | 2012-11-01 | 2017-11-17 | |
| Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency rAAV1-CB-hAAT - Alpha-1 Antitrypsin Deficiency | Phase 2 | Completed | Beacon Therapeutics | 9 | 2010-06-01 | 2019-03-28 |
| Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis rAAV2-CB-hRPE65 - Leber Congenital Amaurosis | Phase 1 / Phase 2 | Completed | Beacon Therapeutics | 12 | 2009-06-17 | 2017-12-28 |
| Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency rAAV1-CB-hAAT - Alpha 1-Antitrypsin Deficiency | Phase 1 | Completed | University of Massachusetts, Worcester | 9 | 2006-02-01 | 2016-12-15 |
Retinal Degeneration (7) · Color Vision Defects (3) · Retinoschisis (2) · alpha 1-Antitrypsin Deficiency (2) · Achromatopsia 3 (1) · Eye Diseases (1) · Leber Congenital Amaurosis (1) · Nervous System Diseases (1) · Neurologic Manifestations (1) · Sensation Disorders (1) · Signs and Symptoms (1) · Vision Disorders (1)
brinzolamide (1) · dorzolamide (1)
United States (14) · Australia (1) · Canada (1) · Ireland (1) · Israel (1) · United Kingdom (1)
David Jacobs, MD, MBA (2) · Matt Feinsod, MD (2) · Terence R. Flotte, MD (2) · Bruce C. Trapnell, MD (1) · Carrie Reichley (1) · J Timothy Stout, MD, PhD, MBA (1) · Mark L Brantly, MD (1) · Matthew Feinsod, MD (1) · Noel G. McElvaney, MB, BCh, BAO (1) · None None (1) · Robert A. Sandhaus, MD, PhD (1) · Theresa Heah, MD (1)
PMID 40547876, PMID 21609134, PMID 24231351, PMID 27479814, PMID 29869534, PMID 30194931, PMID 30551202, PMID 38888288, PMID 39680391
R01HL069877 (2), R24EY022023 (2), 2009 (1)