A Single Arm, Ph1/2, Open-label, Multicenter Trial With Dose-exploration Via Subretinal Injection to Evaluate the Safety and Preliminary Efficacy of VG801 for Treatment of ABCA4 Mutation-associated Recessive Hereditary Retinal Dystrophy (Stargardt Disease)
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: f
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⁃ To be eligible for study entry, subjects must satisfy all the following criteria:
• Written informed consent.
• Subjects aged ≥ 6 years.
• Clinical diagnosis of a macular lesion phenotypically consistent with a recessive hereditary macular dystrophy (Stargardt disease).
• Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compound heterozygotes).
• Poor vision in the study eye.
Locations
Other Locations
China
Shanghai General Hospital
RECRUITING
Shanghai
Contact Information
Primary
Research Coordinator
shiyilunli@sina.com
+86-021-36123569
Time Frame
Start Date: 2024-12-23
Estimated Completion Date: 2026-05
Participants
Target number of participants: 15
Treatments
Experimental: VG801
Participants will receive a single dose of subretinal injection of VG801at Day 0.
Related Therapeutic Areas
Sponsors
Leads: VeonGen Therapeutics GmbH