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)

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).

Study Overview

• Status: Recruiting
• Conditions: X-Linked Retinitis Pigmentosa
• Intervention / Treatment: Genetic: AAV5-hRKp.RPGR

• Study Type: Interventional
• Enrollment (Estimated): 4
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Study Contact; Phone Number: 844-434-4210; Email: Participate-In-This-Study@its.jnj.com

Study Locations

• Japan
  • Meguro-ku, Japan, 1528902
    • Recruiting
    • National Hospital Organization Tokyo Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Group 1: AAV5-hRKp.RPGR Low Dose; Participants will receive bilateral subretinal administration of AAV5-hRKp.RPGR low dose, with surgical delivery to the 2 eyes performed within 7 to 21 days apart.	Genetic: AAV5-hRKp.RPGR; AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.
Experimental: Group 2: AAV5-hRKp.RPGR High Dose; Participants will receive bilateral subretinal administration of AAV5 hRKp.RPGR high dose, with surgical delivery to the 2 eyes performed within 7 to 21 days apart.	Genetic: AAV5-hRKp.RPGR; AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants with Adverse Event (AEs)	An AE is any untoward medical occurrence in a clinical study participant administered a investigational or non-investigational medicinal product. An AE does not necessarily have a causal relationship with the treatment.	Up to 60 Months
Number of Participants with Abnormalities in Clinical Laboratory Assessments	Number of participants with abnormalities in clinical laboratory assessment (hematology and serum chemistry) will be reported.	Up to 60 Months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees (MRS10) Excluding Scotoma in Static Perimetry at Week 52	Change from baseline in MRS10 excluding scotoma in static perimetry at week 52 will be reported.	Baseline - Week 52
Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma of Worse-seeing Eye in Static Perimetry at Week 52	Change from baseline in MRS10 excluding scotoma of worse-seeing eye in static perimetry at week 52 will be reported.	Baseline - Week 52
Pointwise Response in Full Visual Field at Week 52	Pointwise response in full visual field at week 52 will be reported.	At Week 52
Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52	Pointwise response in worse-seeing eye in full visual field at week 52 will be reported.	At Week 52
Pointwise Response in the Central 30 Degrees Visual Field at Week 52	Pointwise response in the central 30 degrees visual field at week 52 will be reported.	At Week 52
Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52	Pointwise response in worse-seeing eye in the central 30 degrees visual field at week 52 will be reported.	At Week 52
Change From Baseline in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry at Week 52	Change from baseline in mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry at week 52 will be reported.	Baseline - Week 52
Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) in Patient Reported Outcome - Extreme Lighting Domain Score at Week 52	Change from baseline in mLLQ in patient reported outcome - extreme lighting domain score at week 52 will be reported.	Baseline - Week 52
Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment at Week 52	Change from baseline in low luminance visual acuity by ETDRS chart score in monocular assessment at week 52 will be reported.	Baseline - Week 52
Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Monocular Assessment at Week 52	Change from baseline in BCVA by ETDRS chart letter score in monocular assessment at week 52 will be reported.	Baseline - Week 52
Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Worse-seeing Eye at Week 52	Change from baseline in low luminance visual acuity by ETDRS chart letter score in worse-seeing eye at week 52 will be reported.	Baseline - Week 52

Collaborators and Investigators

• Sponsor: Janssen Pharmaceutical K.K.
• Investigators: Study Director: Janssen Pharmaceutical K.K., Japan Clinical Trial, Janssen Pharmaceutical K.K.

Study record dates

Study Major Dates

• Study Start (Actual): September 12, 2023
• Primary Completion (Estimated): September 16, 2025
• Study Completion (Estimated): October 9, 2029

Study Registration Dates

• First Submitted: June 23, 2023
• First Submitted That Met QC Criteria: June 23, 2023
• First Posted (Actual): July 3, 2023

Study Record Updates

• Last Update Posted (Actual): April 24, 2024
• Last Update Submitted That Met QC Criteria: April 23, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Eye Diseases, Hereditary; Retinal Dystrophies; Retinitis; Retinitis Pigmentosa
• Other Study ID Numbers: CR109143; 74765340RPG3001 (Other Identifier: Janssen Pharmaceutical K.K, Japan)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No