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An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT Disease

NCT07063251 · Status: RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2025-08-27

No results posted yet for this study

Summary

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 properly, toxic substances like A2E accumulate and damage the retinal pigment epithelium (RPE), leading to vision loss.

There are currently no approved treatments for STGD1. HG005 is an investigational gene therapy designed to deliver a healthy copy of the ABCA4 gene to the retina. Because the gene is too large to fit into a single AAV (adeno-associated virus) vector, HG005 used two AAV vectors that work together in retinal cells to produce the full-length, functional ABCA4 protein. The goal of HG005 is to restore normal waste removal, protect retinal cells from further damage, and slow or stop vision loss.

Conditions

  • Stargardt Disease Type 1 (STGD1)

Interventions

GENETIC

HG005

Once subretinal injection; The duration of the study is about 52 weeks for each subject, including a 4 weeks screening period, enrollment visit, treatment visit, and 56 weeks follow-up period.

Sponsors & Collaborators

  • HuidaGene Therapeutics Co., Ltd.

    lead INDUSTRY

Principal Investigators

  • Study Director · HuidaGene Therapeutics Co., Ltd.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-08-20
Primary Completion
2027-02-28
Completion
2028-02-28

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07063251 on ClinicalTrials.gov