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4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
Sponsor: 4D Molecular Therapeutics
Summary
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).
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
Key Details
Gender
MALE
Age Range
12 Years - Any
Study Type
INTERVENTIONAL
Enrollment
21
Start Date
2020-06-09
Completion Date
2029-05
Last Updated
2025-03-21
Healthy Volunteers
No
Conditions
Interventions
4D-125 IVT Injection
4D-125 drug product developed for gene therapy, which comprises an AAV capsid variant (4D-R100) carrying a codon-optimized human Retinitis Pigmentosa GTPase Regulator transgene.
Observational
Natural History
Locations (8)
University of Colorado
Aurora, Colorado, United States
Vitreo Retinal Associates
Gainesville, Florida, United States
University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, United States
Columbia University Medical Center/Edward Harkness Eye Institute
New York, New York, United States
Duke University Eye Center/Dept. of Ophthalmology
Durham, North Carolina, United States
Casey Eye Institute, Oregon Health and Science University
Portland, Oregon, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
University of Utah John A. Moran Eye Center
Salt Lake City, Utah, United States