Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
Recruiting
Phase 1
Phase 2
Interventional Study
Retinal Dystrophy Due to Biallelic ABCA4 Mutations
Stargardt Disease 1
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 6 and older
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 15 (estimated)
- Sponsor
- VeonGen Therapeutics GmbH · Industry
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About This Trial
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.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
To be eligible for study entry, subjects must satisfy all the following criteria:
1. Written informed consent.
2. Subjects aged ≥ 6 years.
3. Clinical diagnosis of a macular lesion phenotypically consistent with a recessive hereditary macular dystrophy (Stargardt disease).
4. C…
Contacts