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

NCT06646289 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 74765340RPG2001
• Study Type: interventional
• Target: 24
• Locations: 2 countries
• Sites: 3

Brief Summary

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.

Conditions

X-Linked Retinitis Pigmentosa

Outcome Measures

Primary Outcomes (3)

• Number of Participants With Adverse Events (AE)

  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

  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

  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

Study Arms (3)

Cohort 1

EXPERIMENTAL

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

Cohort 2

EXPERIMENTAL

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

Cohort 3

OTHER

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)

Interventions

AAV5-hRKp.RPGR BIOLOGICAL

AAV5-hRKp.RPGR will be administered sub-retinally.

Also known as: JNJ-74765340, botaretigene sparoparvovec

Cohort 1; Cohort 2

No intervention (Follow-Up assessment) OTHER

Participants will not receive any intervention and will undergo follow-up assessment.

Cohort 3

Eligibility Criteria

• Age: 5 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (3)

Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, 02114, United States

Location

University of Michigan Kellogg Eye Center

Ann Arbor, Michigan, 48105, United States

Location

Moorfields Eye Hospital

London, EC1V 2PD, United Kingdom

Location

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 16, 2024
• First Posted: October 17, 2024
• Study Start: October 10, 2024
• Primary Completion (Estimated): October 24, 2030
• Study Completion (Estimated): October 24, 2030
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

US (2); GB (1)