Clinical Research Directory
Browse clinical research sites, groups, and studies.
A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa
Sponsor: Janssen Pharmaceutical K.K.
Summary
The purpose of the study is to assess the safety and tolerability of bilateral subretinal delivery of adeno-associated virus vector with a serotype 5 capsid human rhodopsin kinase promoter. retinitis pigmentosa guanosine triphosphatase regulator (AAV5-hRKp.RPGR).
Official title: Phase 3 Study to Evaluate the Safety and Efficacy of AAV5-hRKp.RPGR for the Treatment of Japanese X-linked Retinitis Pigmentosa Associated With Pathogenic Variants in Retinitis Pigmentosa GTPase Regulator (RPGR)
Key Details
Gender
All
Age Range
5 Years - Any
Study Type
INTERVENTIONAL
Enrollment
4
Start Date
2023-09-12
Completion Date
2030-11-12
Last Updated
2026-03-13
Healthy Volunteers
No
Conditions
Interventions
AAV5-hRKp.RPGR
AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.
AAV5-hRKp.RPGR
AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.
Locations (1)
National Hospital Organization Tokyo Medical Center
Meguro-ku, Japan