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
Study 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).
Eligibility
Sex
MALE
Min age
12 Years
Max age
—
Healthy volunteers
No
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)
Key Exclusion Criteria (all cohorts)
* Patient has previously received any AAV treatment
* Pre-existing eye conditions or surgical complications that would preclude participation in an interventional clinical trial or interfere with the interpretation of study endpoints
Primary outcome measure(s)
Incidence and severity of TEAEs and serious adverse events (SAEs), including clinically significant changes in safety parameters — 24 months to 60 Months
Trial sites (8)
Facility
City
Region
Status
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
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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