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This study examines the gene therapy 4D-125, injected into the eye, in patients with X-linked retinitis pigmentosa (an inherited eye/retinal disease). It can enroll male patients with a hemizygous non-syndromic RPGR mutation confirmed by genetic testing. It is being conducted in Phase 1/Phase 2 and is currently not recruiting but still ongoing (active, not recruiting).
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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