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Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
NCT04671433 · View on ClinicalTrials.gov ↗
Study Summary
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
Conditions Studied
Interventions
- BIOLOGICAL Genetic: AAV5-hRKp.RPGR
Study Locations (20)
Other
- UZ Gent — Ghent
- Rigshospitalet Glostrup — Glostrup Municipality
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts — Paris
- Hadassah Medical Center — Jerusalem
- Azienda Ospedaliero Universitaria Careggi — Florence
- Ospedale San Paolo — Milan
- Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli — Naples
- IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS — Roma
- VUMC Amsterdam — Amsterdam
California
- Shiley Eye Institute Jacobs Retina Center — La Jolla
- Childrens Hospital — Los Angeles
- Stanford Health Care — Palo Alto
Florida
- VitreoRetinal Associates, PA — Gainesville
Georgia
- Emory University — Atlanta
Massachusetts
- Massachusetts General Hospital - Center for Celiac Research and Treatment — Boston
Michigan
- Univ of Michigan Medical Center — Ann Arbor
North Carolina
- Duke Eye Center — Durham
Pennsylvania
- University of Pittsburgh Medical Center (UPMC) — Pittsburgh
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 105 participants |
| Start Date | 2020-12-04 |
| Est. Completion | 2024-09-30 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04671433
The ClinicalTrials.gov registry entry for NCT04671433 describes a study currently listed as completed. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 105 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Janssen Research & Development, which has 442 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with X-Linked Retinitis Pigmentosa appearing as the primary indexed condition, and to 1 intervention — of which Genetic: AAV5-hRKp.RPGR is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04671433 reports 20 study locations spanning 10 distinct geographic areas — top geographies include Other, California, Florida. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04671433 about?
NCT04671433 is a clinical study titled "Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene". A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
What is the current status of trial NCT04671433?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 105 participants. The study started on 2020-12-04. Estimated completion is 2024-09-30.
What conditions does trial NCT04671433 study?
This clinical trial studies the following conditions: X-Linked Retinitis Pigmentosa. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04671433?
The interventions under investigation include: Genetic: AAV5-hRKp.RPGR (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04671433?
This trial is sponsored by Janssen Research & Development, which has 442 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04671433 being conducted?
This trial has 20 study locations across California, Florida, Georgia, Massachusetts, Michigan. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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