A clinical trial evaluating the safety and efficacy of a single subretinal injection of AGTC-501 in participants with X-linked retinitis pigmentosa caused by RPGR mutations
A clinical trial evaluating the safety and efficacy of a single subretinal injection of AGTC-501 in participants with X-linked retinitis pigmentosa caused by RPGR mutations
| Trial Fact Box | |
|---|---|
| NCT Code | NCT04850118 |
| Sponsor | Beacon Therapeutics |
| Study type(s) | Interventional |
| Phase | Phase 2 / Phase 3 |
| Enrollment (Actual) | 85 |
| Study start (Actual) | 14 March 2024 |
| Primary Completion (Estimated) | 30 June 2026 |
| Study Completion (Estimated) | 31 October 2031 |
Official Title:
A Randomized, Controlled, Masked, Multi-center Study Evaluating the Efficacy, Safety, and Tolerability of Two Doses of AGTC-501 Compared to an Untreated Control Group in Male Participants With X-linked Retinitis Pigmentosa
Brief Summary:
This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501/laruparetigene zovaparvovec )) to an untreated control group in male participants with X-linked retinitis pigmentosa caused by RPGR mutations.
Detailed Description:
This study is a randomized, controlled, masked, multi-center study evaluating and comparing 2 doses of AGTC-501 to an untreated control group. A single subretinal injection of AGTC-501 Dose 1 or Dose 2 will be administered in participants in 2 treatment groups while participants in the untreated control group will be followed and evaluated, after which they will be evaluated to determine eligibility to receive treatment with AGTC-501 Dose 2.
Approximately 75 eligible male participants between 12 and 50 years of age (inclusive) will be randomized in a 1:1:1 ratio to 1 of 3 groups.
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