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A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

X-Linked Retinitis Pigmentosa Clinical Trial

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

Clinical Trial Summary

This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501) to an untreated control group in male participants with X-linked retinitis pigmentosa caused by RPGR mutations.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04850118
Study type Interventional
Source Beacon Therapeutics
Contact Serva Health
Phone 855-467-2364
Email ProviderSupport@scenictrials.com
Status Recruiting
Phase Phase 2/Phase 3
Start date March 14, 2024
Completion date October 2029
View Details
See also
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Enrolling by invitation NCT06275620 - A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN) Phase 2
Completed NCT03116113 - A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112 Phase 1/Phase 2
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Active, not recruiting NCT04312672 - Long-term Follow-up Gene Therapy Study for RPGR- XLRP
Completed NCT04868916 - An Observational Study of Japanese Participants With X-linked Retinitis Pigmentosa
Completed NCT03252847 - Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR) Phase 1/Phase 2
Recruiting NCT05874310 - Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa Early Phase 1
Active, not recruiting NCT04671433 - Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Phase 3
Active, not recruiting NCT04794101 - Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Phase 3
Completed NCT03314207 - Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)
Active, not recruiting NCT03316560 - Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations Phase 1/Phase 2
Active, not recruiting NCT06333249 - A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE) Phase 2