A Phase 1/2 Safety Study in Subjects With CHM (Choroideremia) Gene Mutations Using an Adeno-Associated Virus Serotype 2 Vector to Deliver the Normal Human CHM Gene [AAV2-hCHM] to the Retina

AAV2-hCHM-101

This clinical study evaluates the safety and tolerability of AAV2-hCHM in participants with Choroideremia gene mutations.

2015-01-15

2022-10-12

2022-10-12

Completed

Early phase I

15

Inclusion Criteria: * Male at least 18 years of age diagnosed with CHM gene mutation * Central visual field (VF) \<30° in any of the 24 meridians (using Goldmann perimetry III4e isopter) in the eye to be injected * Any evidence of functioning outer retinal cells within the central 10° Exclusion Criteria: * Previous history of ocular inflammatory disease (uveitis) * Prior intraocular surgery within six months * Participation in a previous gene therapy research trial within one year of enrollment or participation in any other ocular gene therapy trial * Participation in a clinical study with an investigational drug in the past six months * Grossly asymmetrical disease, or other eye morbidity, which may render the contralateral eye ineffective as a control * Visual acuity \<20/200 on standard Early Treatment of Diabetic Retinopathy Study (ETDRS) testing in the eye to be injected * Presence of disease which may preclude the participant from participation in this trial * Use of medications known to be neuroprotective or retino-toxic that could potentially interfere with the disease process and/or cause ocular adverse events; individuals who discontinue use of these compounds for 6 months may become eligible * Identification by the investigator as being unable or unwilling to perform/be compliant with study procedures.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 15, 'type': 'ACTUAL'}}

2024-01-25