Trials / NCT04517149
4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
Underway, not enrolling Phase 1 / Phase 2 Gene therapy
- Sponsor
- 4D Molecular Therapeutics
- Treatment
- 4D-125 IVT Injection; Observational
- Gene
- RPGR
- Conditions
- X-Linked Retinitis Pigmentosa
- Ages
- 12 Years to no upper limit
- Enrollment
- 21 participants
- Started
- 2020-06-09
- Main results expected
- 2026-06
- Results posted
- Not yet
- Record last updated
- 2025-03-21
What the study is testing
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).
Where it runs
United States (8)
- University of Colorado, Aurora, Colorado
- Vitreo Retinal Associates, Gainesville, Florida
- University of Michigan Kellogg Eye Center, Ann Arbor, Michigan
- Columbia University Medical Center/Edward Harkness Eye Institute, New York, New York
- Duke University Eye Center/Dept. of Ophthalmology, Durham, North Carolina
- Casey Eye Institute, Oregon Health and Science University, Portland, Oregon
- Retina Foundation of the Southwest, Dallas, Texas
- University of Utah John A. Moran Eye Center, Salt Lake City, Utah