Gene Therapy
Total Trials
14
As Lead Sponsor
13
As Collaborator
1
Total Enrollment
411
NCT00430768
Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency
Phase: Phase 1
Role: Collaborator
Start: Feb 28, 2006
Completion: Jan 31, 2015
NCT00749957
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
Phase: Phase 1/2
Role: Lead Sponsor
Start: Jun 17, 2009
Completion: Sep 22, 2017
NCT01054339
Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency
Phase: Phase 2
Start: Jun 30, 2010
Completion: Oct 31, 2015
NCT02331173
Clinical Evaluation of Patients With X-linked Retinoschisis
Phase: N/A
Start: Nov 30, 2012
Completion: Oct 31, 2016
NCT01846052
Clinical and Genetic Characterization of Individuals With Achromatopsia
Start: Jun 30, 2013
Completion: Apr 30, 2017
NCT02416622
Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)
Start: May 31, 2015
Completion: May 9, 2023
NCT02599922
Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)
Start: Apr 11, 2016
Completion: Jul 31, 2026
NCT02935517
Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)
Start: Aug 3, 2017
Completion: Aug 31, 2026
NCT03314207
Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)
Start: Dec 1, 2017
Completion: Feb 10, 2022
NCT03316560
Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
Start: Apr 16, 2018
Completion: Mar 31, 2025
NCT06333249
A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)
Start: Apr 13, 2021
Completion: Feb 28, 2027
NCT06275620
A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
Start: Nov 14, 2023
Completion: Dec 31, 2029
NCT04850118
A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
Phase: Phase 2/3
Start: Mar 14, 2024
Completion: Oct 31, 2029
NCT07174726
A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa
Start: Sep 10, 2025
Completion: Dec 15, 2030
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