Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Phase 3Active Not RecruitingNCT04794101

Janssen Research & Development, LLC97 enrolled

Overview

A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

Conditions

X-Linked Retinitis Pigmentosa

Interventions

Genetic: AAV5-hRKp.RPGR Intermediate DoseBIOLOGICAL

Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group

Genetic: AAV5-hRKp.RPGR Low DoseBIOLOGICAL

Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group

Eligibility

Sex: ALLMin age: 3 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Male or female, 3 years of age or older, has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory. Exclusion Criteria: * None

Locations (25)

Shiley Eye Institute Jacobs Retina Center

La Jolla, California, United States

Outcomes

Primary Outcomes

Number of Participants with Ocular and Non-ocular Adverse Events

Number of participants with ocular and non-ocular adverse events will be assessed.

Time frame: Day 1 - Month 60

Number of Participants With Abnormalities in Laboratory Assessments

Number of participants with abnormalities in laboratory assessments will be assessed.

Time frame: Day 1 - Month 60

Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment

Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.

Time frame: From Baseline - Month 60

Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Monocular Assessment

Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.

Time frame: From Baseline- Month 60

Data from ClinicalTrials.gov

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