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Active Not RecruitingNCT05926583

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

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)

Status
Active Not Recruiting
Phase
Phase 3
Study type
Interventional
Enrollment
4 (actual)
Sponsor
Janssen Pharmaceutical K.K. · Industry
Sex
All
Age
5 Years
Healthy volunteers
Not accepted

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).

Conditions

Interventions

TypeNameDescription
GENETICAAV5-hRKp.RPGRAAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.
GENETICAAV5-hRKp.RPGRAAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.

Timeline

Start date
2023-09-12
Primary completion
2027-06-30
Completion
2030-11-12
First posted
2023-07-03
Last updated
2026-04-13

Locations

1 site across 1 country: Japan

Source: ClinicalTrials.gov record NCT05926583. Inclusion in this directory is not an endorsement.