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

NCT04671433 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 105

Last updated 2026-03-16

No results posted yet for this study

Summary

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

Conditions

  • X-Linked Retinitis Pigmentosa

Interventions

BIOLOGICAL

Genetic: AAV5-hRKp.RPGR

Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group

BIOLOGICAL

Genetic: AAV5-hRKp.RPGR

No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study)

Sponsors & Collaborators

  • Janssen Research & Development, LLC

    lead INDUSTRY

Principal Investigators

  • Janssen Research & Development, LLC Clinical Trial · Janssen Research & Development, LLC

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
PARALLEL

Eligibility

Min Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-12-04
Primary Completion
2024-09-30
Completion
2024-09-30
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Canada
  • Denmark
  • France
  • Israel
  • Italy
  • Netherlands
  • Spain
  • Switzerland
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04671433 on ClinicalTrials.gov