Trials / Recruiting
RecruitingNCT07002398
Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
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
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 15 (estimated)
- Sponsor
- VeonGen Therapeutics GmbH · Industry
- Sex
- All
- Age
- 6 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | VG801 | Administered as specified in the single treatment arm. Study Cohort: Low dose, medium dose and high dose cohort |
Timeline
- Start date
- 2024-12-23
- Primary completion
- 2026-05-01
- Completion
- 2026-05-01
- First posted
- 2025-06-03
- Last updated
- 2025-12-11
Locations
1 site across 1 country: China
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT07002398. Inclusion in this directory is not an endorsement.