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NCT04794101 · ClinicalTrials.gov registry record · Phase 3
Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
A Phase 3 study of X-Linked Retinitis Pigmentosa, sponsored by Janssen Research & Development.
- Active
- Registry status
- Phase 3
- Development phase
- 97
- Enrollment target
- 20
- Study locations
NCT04794101: Active Phase 3 study of X-Linked Retinitis Pigmentosa, sponsored by Janssen Research & Development.
NCT04794101 is a Phase 3 study of X-Linked Retinitis Pigmentosa that is active but no longer recruiting, run by Janssen Research & Development. The registered enrollment target is 97 participants, above the 46-participant average among 8 other X-Linked Retinitis Pigmentosa trials with a reported enrollment target (111% higher). The trial reports 20 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04794101, a Phase 3 study of X-Linked Retinitis Pigmentosa, is active but no longer recruiting, sponsored by Janssen Research & Development.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 97 participants
- Enrollment target
- 20
- Study locations
Study Summary
A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
Primary Outcome
Number of participants with ocular and non-ocular adverse events will be assessed.
Conditions Studied
Interventions
- BIOLOGICAL Genetic: AAV5-hRKp.RPGR Intermediate Dose
- BIOLOGICAL Genetic: AAV5-hRKp.RPGR Low Dose
Study Locations (20)
Other
- Ghent University Hospital - Ghent
- Rigshospitalet Glostrup - Glostrup Municipality
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts - Paris
- Hadassah Medical Center - Jerusalem
- Azienda Ospedaliero Universitaria Careggi - Florence
- Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli - Naples
- VUMC Amsterdam - Amsterdam
- Radboudumc - Nijmegen
- Hosp Univ Fund Jimenez Diaz - Madrid
- University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical - Basel
California
- Shiley Eye Institute Jacobs Retina Center - La Jolla
- Childrens Hospital Los Angeles - Los Angeles
- Stanford Health Care - Palo Alto
Florida
- VitreoRetinal Associates, PA - Gainesville
Georgia
- Emory University - Atlanta
Massachusetts
- Massachusetts Eye and Ear Infirmary - 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 | 97 participants |
| Start Date | 2020-12-04 |
| Est. Completion | 2029-12-07 |
| Phase | Phase 3 |
What the registry record for NCT04794101 still lists
NCT04794101 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 97 participants, a relatively small participant target, above the 46-participant average among 8 other X-Linked Retinitis Pigmentosa trials with a reported enrollment target (111% higher).
The record links to 1 condition, with X-Linked Retinitis Pigmentosa appearing as the primary indexed condition, and to 2 interventions - of which Genetic: AAV5-hRKp.RPGR Intermediate Dose is the first listed.
NCT04794101 names 20 study sites across 9 states, led by Other, California, Florida.
Frequently Asked Questions
What is clinical trial NCT04794101 about?
NCT04794101 is a clinical study titled "Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene". A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
What is the current status of trial NCT04794101?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 97 participants. The study started on 2020-12-04. Estimated completion is 2029-12-07.
What conditions does trial NCT04794101 study?
This clinical trial studies the following conditions: X-Linked Retinitis Pigmentosa.
What interventions are being tested in trial NCT04794101?
The interventions under investigation include: Genetic: AAV5-hRKp.RPGR Intermediate Dose (BIOLOGICAL), Genetic: AAV5-hRKp.RPGR Low Dose (BIOLOGICAL).
Who is sponsoring clinical trial NCT04794101?
This trial is sponsored by Janssen Research & Development, which has 593 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04794101 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.
Learn More About Clinical Trials
Similar trials for X-Linked Retinitis Pigmentosa
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04794101's enrollment target sits among peer trials
97 2nd of 8 the highest of 8 other X-Linked Retinitis Pigmentosa trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other X-Linked Retinitis Pigmentosa trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
COMPLETED · Phase 3
-
A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)
ACTIVE NOT RECRUITING · Phase 2
-
4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
ACTIVE NOT RECRUITING · Phase 1
-
A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)
ACTIVE NOT RECRUITING · Phase 2
-
A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
ACTIVE NOT RECRUITING · Phase 2
-
Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
ACTIVE NOT RECRUITING · Phase 1
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