Long-term Follow-up Gene Therapy Study for RPGR- XLRP

Janssen Research & Development, LLC42 enrolled

Overview

This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.

Study Type

OBSERVATIONAL

Enrollment

Conditions

X-Linked Retinitis Pigmentosa

Interventions

AAV5-hRKp.RPGRBIOLOGICAL

Participants will not receive any intervention in this study. Participants from the retrospective (MGT009-NCT03252847) study will be follow-up.

Eligibility

Sex: MALEMin age: 5 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Received AAV5-hRKp.RPGR in the MGT009 Study Exclusion Criteria: * Unwilling or unable to meet with the requirements of the study

Locations (5)

Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, United States

Univ of Michigan Medical Center

Ann Arbor, Michigan, United States

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Leeds Teaching Hospitals NHS Trust

Leeds, United Kingdom

Outcomes

Primary Outcomes

Assess the Longer-term Safety of AAV5-hRKp.RPGR Administered to Participants

Longer-term safety of subretinal AAV5-hRKp.RPGR administered to participants will be reported.

Time frame: 60 Months

Secondary Outcomes

Change in Functional Vision of Walk Time in Vision-guided Mobility Assessment (VMA)

Change in functional vision of walk time after treatment administration in VMA (Version 1.0) will be reported.

Time frame: Up to 60 Months

Change in Functional Vision of Low Luminance Questionnaire (LLQ) Domain Scores in Patient Reported Outcome (PRO)

Change in functional vision of LLQ domain scores in PRO will be reported. The instrument is scored by domain, computed by scaling individual items from 0 to 100 and then averaging the individual items for each domain. A higher score reflects higher functional level.

Time frame: Up to 60 Months

Change in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score

The longer-term efficacy of AAV5-hRKp.RPGR in change in BCVA using ETDRS chart letter score will be reported.

Time frame: Up to 60 Months

Change in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Under Low Luminance Conditions

Change in LLVA as assessed by ETDRS chart under low luminance conditions will be reported.

Time frame: Up to 60 Months

Change in Mean Retinal Sensitivity Within the Central 10 Degree Visual Field Excluding Scotoma (MRS10) in Static Perimetry

Change in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be reported.

Data from ClinicalTrials.gov

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