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RECRUITING
NCT05926583
PHASE3

A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa

Sponsor: Janssen Pharmaceutical K.K.

View on ClinicalTrials.gov

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

Interventions

GENETIC

AAV5-hRKp.RPGR

AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.

GENETIC

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