4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
Public ClinicalTrials.gov record NCT04517149. 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
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-125 in Males With X-linked Retinitis Pigmentosa (XLRP) Caused by Mutations in the RPGR Gene
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is a Phase 1/2 multicenter study with two parallel parts: an observational natural history cohort and an open-label, prospective interventional trial in males with non-syndromic X-linked retinitis pigmentosa (XLRP) due to mutations in the gene encoding retinitis pigmentosa GTPase regulator (RPGR).
Study identification
- NCT ID
- NCT04517149
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 21 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 12 Years and older
- Sex
- Male
- 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
- Jun 9, 2020 (Actual)
- Primary completion
- Jun 2026 (Estimated)
- Completion
- May 2029 (Estimated)
- Last update posted
- Mar 21, 2025
2020 – 2029
United States locations
- U.S. sites
- 8
- U.S. states
- 8
- U.S. cities
- 8
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Colorado | Aurora | Colorado | 80045 | — |
| Vitreo Retinal Associates | Gainesville | Florida | 32607 | — |
| University of Michigan Kellogg Eye Center | Ann Arbor | Michigan | 48105 | — |
| Columbia University Medical Center/Edward Harkness Eye Institute | New York | New York | 10032 | — |
| Duke University Eye Center/Dept. of Ophthalmology | Durham | North Carolina | 27710 | — |
| Casey Eye Institute, Oregon Health and Science University | Portland | Oregon | 97239 | — |
| Retina Foundation of the Southwest | Dallas | Texas | 75231 | — |
| University of Utah John A. Moran Eye Center | Salt Lake City | Utah | 84132 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04517149, 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 Mar 21, 2025 · Synced Oct 21, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04517149 live on ClinicalTrials.gov.