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Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)

X-Linked Retinitis Pigmentosa Clinical Trial

Official title:
An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2-.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)

Clinical Trial Summary

The Phase 1 part of the study is a dose escalation of subretinal administration of AAV2/5 vector to assess the safety of this vector in participants with XLRP caused by mutations in RPGR. The Phase 2 part of the study is a cohort expansion of subretinal administration of AAV2/5 vector to assess the safety and efficacy of this vector in participants with XLRP caused by mutations in RPGR.

Clinical Trial Description

This is an open-label phase 1/2 dose-escalation and cohort expansion trial to determine the safety and efficacy of subretinal administration of AAV2/5 vector in participants with XLRP caused by mutations in RPGR. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03252847
Study type Interventional
Source MeiraGTx UK II Ltd
Contact
Status Completed
Phase Phase 1/Phase 2
Start date July 14, 2017
Completion date November 18, 2021
View Details
See also
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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