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Clinical Trial Summary

This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.


Clinical Trial Description

This protocol includes a non-randomized, open-label, Phase 1/2 study (HORIZON). Approximately 30 participants will be enrolled into the dose escalation study (HORIZON). Each participant will receive the study agent by subretinal injection in one eye on a single occasion. Enrollment will begin with the lowest dose and will proceed to higher doses only after review of safety data by a Data and Safety Monitoring Committee (DSMC). There are a total of 15 visits over approximately 36 months, and long-term follow-up evaluations annually at years 4 and 5. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03316560
Study type Interventional
Source Beacon Therapeutics
Contact
Status Active, not recruiting
Phase Phase 1/Phase 2
Start date April 16, 2018
Completion date March 2025

See also
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Recruiting NCT05926583 - A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa Phase 3
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 NCT04517149 - 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) 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 NCT04794101 - Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Phase 3
Active, not recruiting NCT04671433 - 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 NCT06333249 - A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE) Phase 2