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

Phase 3
Active, not recruiting
Conditions
X-Linked Retinitis Pigmentosa
Registration Number
NCT04794101
Lead Sponsor
Janssen Research & Development, LLC
Brief Summary

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

Detailed Description

Not available

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
97
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Primary Outcome Measures
NameTimeMethod
Number of Participants with Ocular and Non-ocular Adverse EventsDay 1 - Month 60

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

Number of Participants With Abnormalities in Laboratory AssessmentsDay 1 - Month 60

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

Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Monocular AssessmentFrom Baseline- Month 60

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

Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular AssessmentFrom Baseline - Month 60

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

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (28)

Shiley Eye Institute Jacobs Retina Center

🇺🇸

La Jolla, California, United States

Childrens Hospital Los Angeles

🇺🇸

Los Angeles, California, United States

Stanford Health Care

🇺🇸

Palo Alto, California, United States

VitreoRetinal Associates, PA

🇺🇸

Gainesville, Florida, United States

Emory University

🇺🇸

Atlanta, Georgia, United States

Massachusetts Eye and Ear Infirmary

🇺🇸

Boston, Massachusetts, United States

Univ of Michigan Medical Center

🇺🇸

Ann Arbor, Michigan, United States

Duke Eye Center

🇺🇸

Durham, North Carolina, United States

University of Pittsburgh Medical Center (UPMC)

🇺🇸

Pittsburgh, Pennsylvania, United States

Retina Consultants of Houston

🇺🇸

Bellaire, Texas, United States

Scroll for more (18 remaining)
Shiley Eye Institute Jacobs Retina Center
🇺🇸La Jolla, California, United States

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