A Phase 1/2 Open-label, Multi-centre, Dose-exploration Trial to Evaluate the Safety and Preliminary Efficacy of VG801 via Subretinal Injection in Treatment of Patients with Biallelic ABCA4 Mutation-Associated Retinal Dystrophy.

Condition studied

Biallelic ABCA4 Mutation-Associated Retinal Dystrophy.

Investigational medicinal product(s)

Eligibility

Primary endpoint

Safety Endpoint: Number of Adverse events (ocular and systemic), Safety Endpoint: Evaluation of the humoral immune responses against the AAV capsid, Safety Endpoint: Measurement of vector in blood, tears, and saliva (vector shedding) until two negative samples have been obtained, Safety Endpoint: Hematology, chemistry, and urinalysis, Safety Endpoint: Comprehensive ophthalmic assessments including slit lamp examination coupled with imaging including optical coherence tomography (OCT), fundus photography, intraocular pressure monitoring, and fundus autofluorescence imaging., Preliminary Efficacy Endpoint: Visual Function (BCVA, Pelli Robson contrast test, OCT, fundus photography, IOP, fundus autofluorescence imaging), Preliminary Efficacy Endpoint: Morphological retinal measures (Fundus autofluorescence imaging , Optical coherence tomography), Preliminary Efficacy EndPoint: Local retinal function measures for rods and cones ( Full-field stimulus threshold testing), Preliminary Efficacy Endpoint: Retinal function measures (Goldmann kinetic visual fields, Scotopic and photopic microperimetry), Preliminary Efficacy Endpoint: Mobility function (novel virtual reality mobility test)