Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Public ClinicalTrials.gov record NCT04794101. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Study identification
- NCT ID
- NCT04794101
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Janssen Research & Development, LLC
- Industry
- Enrollment
- 97 participants
Conditions and interventions
Conditions
Interventions
- Genetic: AAV5-hRKp.RPGR Intermediate Dose Biological
- Genetic: AAV5-hRKp.RPGR Low Dose Biological
Biological
Eligibility (public fields only)
- Age range
- 3 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 3, 2020
- Primary completion
- Sep 19, 2029
- Completion
- Dec 6, 2029
- Last update posted
- Apr 12, 2026
2020 – 2029
United States locations
- U.S. sites
- 9
- U.S. states
- 7
- U.S. cities
- 9
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Shiley Eye Institute Jacobs Retina Center | La Jolla | California | 92093 0946 | — |
| Childrens Hospital Los Angeles | Los Angeles | California | 90027 | — |
| Stanford Health Care | Palo Alto | California | 94303 | — |
| VitreoRetinal Associates, PA | Gainesville | Florida | 32607 | — |
| Emory University | Atlanta | Georgia | 30322 | — |
| Massachusetts Eye and Ear Infirmary | Boston | Massachusetts | 02114 | — |
| Univ of Michigan Medical Center | Ann Arbor | Michigan | 48105 | — |
| Duke Eye Center | Durham | North Carolina | 27705 | — |
| University of Pittsburgh Medical Center (UPMC) | Pittsburgh | Pennsylvania | 15213 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 16 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04794101, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 12, 2026 · Synced Apr 23, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04794101 live on ClinicalTrials.gov.