4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
X-Linked Retinitis Pigmentosa
About this trial
This is an interventional treatment trial for X-Linked Retinitis Pigmentosa focused on measuring XLRP, Gene therapy
Eligibility Criteria
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
Sites / Locations
- University of Colorado
- Vitreo Retinal Associates
- University of Michigan Kellogg Eye Center
- Columbia University Medical Center/Edward Harkness Eye Institute
- Duke University Eye Center/Dept. of Ophthalmology
- Casey Eye Institute, Oregon Health and Science University
- Retina Foundation of the Southwest
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Other
4D-125 Dose Exploration
4D-125 Dose Expansion
Observational
Dose 1 and Dose 2 4D-125 will be administered at the assigned dose level as a single dose, IVT injection on Day 1. The contralateral eye may also be dosed with 4D-125 as a single dose, IVT injection provided the subject is eligible and provides consent.
4D-125 will be administered at the assigned dose level as a single dose, IVT injection on Day 1 in both adults and pediatric participants. For adult participants only, the contralateral eye may also be dosed with 4D-125 as a single dose, IVT injection provided the subject is eligible and provides consent.
Natural History