A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)

May 7, 2026 updated by: Janssen Research & Development, LLC

An Open-label, Multicenter, Phase 2 Follow-on Study for Second Eye Treatment of Patients Previously Treated With a Recombinant Adeno-associated Virus Vector (AAV5 hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)

The purpose of this study is to assess the safety and tolerability of subretinal delivery of Adeno-associated Virus Vector (AAV5 hRKp.RPGR) gene therapy in adults and children with X-linked retinitis pigmentosa.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

24

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • London, United Kingdom, EC1V 2PD
        • Moorfields Eye Hospital
  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts Eye and Ear Infirmary
    • Michigan
      • Ann Arbor, Michigan, United States, 48105
        • University of Michigan Kellogg Eye Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Have been treated with AAV5-hRKp.RPGR in study MGT009 and have completed or is currently enrolled in Study MGT010
  • Must sign an informed consent form indicating that they understand the purpose and procedures of the study and is willing to participate in the study
  • Willing to adhere to the protocol and long-term follow-up

Exclusion Criteria:

- There are no specific exclusion criteria to enroll in this study

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1
Participants will receive a dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 depending on the dosage administered in study MGT009 (NCT03252847) in the past. After receiving the treatment, participants will be assessed for safety.
Biological: AAV5-hRKp.RPGR
AAV5-hRKp.RPGR will be administered sub-retinally.
Other Names:
  • JNJ-74765340, botaretigene sparoparvovec
Experimental: Cohort 2
Participants will receive the treatment dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 in the second eye once the safety will be determined in Cohort 1.
Biological: AAV5-hRKp.RPGR
AAV5-hRKp.RPGR will be administered sub-retinally.
Other Names:
  • JNJ-74765340, botaretigene sparoparvovec
Other: Cohort 3
The participants who are not willing to undergo surgery or are not eligible for surgery will be assessed in this cohort. They will be assessed yearly until 5 years after their initial eye surgery in previous study MGT009.
Other: No intervention (Follow-Up assessment)
Participants will not receive any intervention and will undergo follow-up assessment.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Adverse Events (AE)
Time Frame: From baseline up to 5.5 years
An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non investigational) product. An AE does not necessarily have a causal relationship with the intervention. An AE can be any unfavorable and unintended sign (including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal (investigational or non investigational) product, whether or not related to that medicinal (investigational or non investigational) product.
From baseline up to 5.5 years
Change from Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Scores
Time Frame: Baseline, Months 12, 24, 36, 48 and 60
Change from baseline in BCVA by ETDRS chart letter scores in monocular assessment of the second treated eye will be assessed. BCVA was measured using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. An increase in the number of letters read correctly means that vision has improved and vice-versa.
Baseline, Months 12, 24, 36, 48 and 60
Change from Baseline in Low Luminance Visual Acuity (LLVA) by ETDRS Chart Letter Score
Time Frame: Baseline, Months 12, 24, 36, 48 and 60
Change in LLVA by ETDRS chart letter scores from baseline in monocular assessment of second treated eye will be assessed. Visual function assessments included LLVA assessment in each eye by ETDRS letters. The letter score ranges from 0 (worse score) to 100 (best score), and a gain in LLVA from baseline indicates an improvement in visual acuity.
Baseline, Months 12, 24, 36, 48 and 60

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees (MRS10)
Time Frame: Baseline, Months 12, 24, 36, 48 and 60
Change from baseline in MRS10 in static perimetry of the second treated eye will be measured.
Baseline, Months 12, 24, 36, 48 and 60
Change from Baseline in Mean Retinal Sensitivity Within the Full Visual Field (MRS90)
Time Frame: Baseline, Months 12, 24, 36, 48 and 60
Change from baseline in MRS90 in static perimetry of the second treated eye will be assessed.
Baseline, Months 12, 24, 36, 48 and 60
Change from Baseline in the Low Luminance Questionnaire (LLQ) Domain Scores
Time Frame: Baseline, Month 12
LLQ is a 32-item disease-specific questionnaire to assess self reported visual problems under low luminance and at night. The range of responses is different for different questions, with the overall pattern being that "1" means "No difficulty at all" and that increasingly higher numbers mean increasingly more difficult.
Baseline, Month 12
Change in Retinal Function as Assessed by Pointwise Response in Static Perimetry
Time Frame: From Month 12 up to Month 60
Pointwise response in static perimetry within the full visual field in the second treated eye will be assessed.
From Month 12 up to Month 60
Change in Retinal Function as Assessed by Pointwise Response in Static Perimetry Within Central 30 Degrees Visual Field
Time Frame: From Month 12 up to Month 60
Pointwise response in static perimetry within the central 30 degrees visual field in the second treated eye will be assessed.
From Month 12 up to Month 60
Change from Baseline in Low Luminance Visual Acuity (LLVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score
Time Frame: Baseline, Months 12, 24, 36, 48 and 60
Change in LLVA by ETDRS chart letter scores from baseline in monocular assessment of second treated eye will be assessed. Visual function assessments included LLVA assessment in each eye by ETDRS letters. The letter score ranges from 0 (worse score) to 100 (best score), and a gain in LLVA from baseline indicates an improvement in visual acuity.
Baseline, Months 12, 24, 36, 48 and 60
Change from Baseline in Best Corrected Visual Acuity (BCVA) by ETDRS Chart Letter Scores
Time Frame: Baseline, Months 12, 24, 36, 48 and 60
Change from baseline in BCVA by ETDRS chart letter scores in monocular assessment of the second treated eye will be assessed. BCVA was measured using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. An increase in the number of letters read correctly means that vision has improved and vice-versa.
Baseline, Months 12, 24, 36, 48 and 60

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Janssen Research & Development, LLC

Investigators

  • Study Director: Janssen Research & Development, LLC Clinical Trial, Janssen Research & Development, LLC

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 10, 2024

Primary Completion (Estimated)

October 24, 2030

Study Completion (Estimated)

October 24, 2030

Study Registration Dates

First Submitted

October 16, 2024

First Submitted That Met QC Criteria

October 16, 2024

First Posted (Actual)

October 17, 2024

Study Record Updates

Last Update Posted (Actual)

May 8, 2026

Last Update Submitted That Met QC Criteria

May 7, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 74765340RPG2001 (Other Identifier: Janssen Research & Development, LLC)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

The data sharing policy of Johnson & Johnson Innovative Medicine is available at innovativemedicine.jnj.com/our-innovation/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

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on X-Linked Retinitis Pigmentosa

Clinical Trials on AAV5-hRKp.RPGR

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Türkiye

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe