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RECRUITINGPHASE3

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)

NCT05926583•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).

Eligibility

Age

5 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Participants who are Japanese male or female aged 5 years or older
•Participants diagnosed as X-linked retinitis pigmentosa (XLRP) (generalized rod-cone dystrophy) associated with pathogenic or likely pathogenic variants in the retinitis pigmentosa guanosine triphosphatase regulator(RPGR) gene
•Has evidence of preserved retinal function as defined by a mean retinal sensitivity of greater than or equal to (\>=) 2 decibel (dB) by Octopus static perimetry and evidence of preserved outer retinal structure (namely the presence of discernible ellipsoid zone) as determined by spectral domain-optical coherence tomography (SD-OCT) in both eyes
•Otherwise, healthy participant on the basis of clinical laboratory tests performed at screening. If the results of the serum chemistry panel or hematology outside the normal reference ranges, the participant may be included only if the investigator judges the abnormalities or deviations from normal to be not clinically significant or to be appropriate and reasonable for the population under study. This determination must be recorded in the participant's source documents and initialed by the investigator

Exclusion Criteria

•Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the AAV5-hRKp.RPGR administration
•Is unable to perform the imaging assessments as required (for example: reliable static perimetry \[reliability factor less than or equal to {\<=}19\], optical coherence tomography \[OCT\], or fundus autofluorescence \[FAF\]).
•Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule

Locations (1)

National Hospital Organization Tokyo Medical Center

Meguro-ku, Japan

Key Dates

Start Date

September 12, 2023

Primary Completion Date

June 30, 2027

Completion Date

November 12, 2030

Last Updated

March 13, 2026

Enrollment

ESTIMATED participants

Conditions

Retinitis Pigmentosa

Interventions

AAV5-hRKp.RPGR

GENETIC

AAV5-hRKp.RPGR

GENETIC

Contacts

Study Contact

CONTACT

844-434-4210 Participate-In-This-Study1@its.jnj.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 13, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa

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