NCT04517149Active, not recruitingPhase 1/2
4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
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
Source: ClinicalTrials.gov · Last updated 21 Mar 2025
Brief summary (registry, in English)
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).
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 21 (actual)
- First submitted
- 14 Aug 2020
- Start date
- 9 Jun 2020
- Primary completion date
- 30 Jun 2026
- Lead sponsor
- 4D Molecular Therapeutics
- Diseases
- Retinitis pigmentosa
- NCT number
- NCT04517149
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 21
- From 9 Jun 2020 to the primary completion date, 30 Jun 2026
- 8 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Patient has previously received any AAV treatment
Other illness
- Pre-existing eye conditions or surgical complications that would preclude participation in an interventional clinical trial or interfere with the interpretation of study endpoints
Criteria the registry did not label
- Natural History Key Inclusion Criteria:
- Male, ≥ 6 years of age at the time of informed consent
- Hemizygous non-syndromic RPGR mutation confirmed by genetic testing
- Interventional Key Inclusion Criteria:
- Male, ≥12 years of age
- Hemizygous non-syndromic RPGR mutation confirmed by genetic testing
- Phase 1 Dose Exploration: At least one eye amenable to IVT injection and BCVA ≤ 78 ETDRS letters (\~20/32) and ≥ 34 ETDRS letters (\~20/200)
- Phase 2 Dose Expansion: At least one eye amenable to IVT injection AND both eyes must have BCVA ≥ 34 ETDRS letters (\~20/200)
Age in the registry: 12 years to - · Sex: Male · Does not accept healthy volunteers
Sites (8)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University of Michigan Kellogg Eye Center Coordinator of this site? Confirm its status | Ann Arbor | United States | Unknown status |
| University of Colorado Coordinator of this site? Confirm its status | Aurora | United States | Unknown status |
| Retina Foundation of the Southwest Coordinator of this site? Confirm its status | Dallas | United States | Unknown status |
| Duke University Eye Center/Dept. of Ophthalmology Coordinator of this site? Confirm its status | Durham | United States | Unknown status |
| Vitreo Retinal Associates Coordinator of this site? Confirm its status | Gainesville | United States | Unknown status |
| Columbia University Medical Center/Edward Harkness Eye Institute Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| Casey Eye Institute, Oregon Health and Science University Coordinator of this site? Confirm its status | Portland | United States | Unknown status |
| University of Utah John A. Moran Eye Center Coordinator of this site? Confirm its status | Salt Lake City | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.