An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT Disease
Recruiting
Early Phase 1
Interventional Study
Stargardt Disease Type 1 (STGD1)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 6 – 17
- Sex
- Any
- Trial phase
- Early Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 6 (estimated)
- Sponsor
- HuidaGene Therapeutics Co., Ltd. · Industry
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About This Trial
Stargardt disease type 1 (STGD1) is a rare genetic eye condition that causes progressive vision loss, often beginning in childhood or adolescence. It is the most common form of inherited macular degeneration and can lead to legal blindness. STGD1 is caused by mutations in the ABCA4 gene, which normally helps clear waste from the photoreceptor cells in the retina. When ABCA4 gene doesn't function p…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Patient ≥ 6 and ≤17 years at the time of signing informed consent, with clinical diagnosis of Stargardt disease;
* At least one ABCA4 allele on each chromosome;
* Both eyes must have well-defined macular atrophic lesions consistent with the diagnosis of Stargardt macular dystr…
Contacts