U.S., June 4 -- ClinicalTrials.gov registry received information related to the study (NCT07002398) titled 'Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)' on May 23.
Brief Summary: This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt disease) due to biallelic ABCA4 mutations.
Study Start Date: Dec. 23, 2024
Study Type: INTERVENTIONAL
Condition:
Retinal Dystrophy Due to Biallelic ABCA4 Mutations
Stargardt Disease 1
Intervention:
DRUG: VG801
Administered as specified in the single treatmen...