An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants with Stargardt Disease (STGD1) (CELESTE)

Condition studied

Stargardt Disease (STGD1)

Investigational medicinal product(s)

Eligibility

Primary endpoint

To measure the number and severity of treatment related adverse events following treatment with AAVB-039

Endpoint detail

Change in the area of atrophy (assessed via short wavelength fundus autofluorescence (SW-FAF), following treatment with AAVB-039., Change in ellipsoid zone (EZ) loss following treatment with AAVB-039., Change in macular sensitivity assessed via microperimetry following treatment with AAVB-039, Change in visual acuity following treatment with AAVB-039