This study is evaluating escalating doses of 4D-110, a gene therapy injected into the eye (intravitreal), in patients with choroideremia. Patients with a genetically confirmed disease-causing mutation in the CHM gene are eligible. The study is in Phase 1 and is active but not currently recruiting participants.
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.
Key Inclusion Criteria: * Diagnosis of CHM defined as a pathogenic mutation in the CHM gene, confirmed by genetic testing * Both eyes must have ≥ 34 ETDRS letters (\~20/200) Key Exclusion Criteria: * Clinically significant, active ocular or peri-ocular infection or inflammation in the study eye * Patient has previously received any AAV treatment * Ocular conditions or ocular media opacity in either eye that would preclude the planned treatment (i.e. IVT injection) or interfere with the interpretation of study endpoints (e.g. significant lens opacity)