HG005 two-vector gene therapy for Stargardt disease in children and teens
Official title: An Investigator-initiated, Open-label, Dose-escalation Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of HG005 in Pediatric Patients With Stargardt Disease (STGD1) Caused by Biallelic ABCA4 Mutations
Run by HuidaGene Therapeutics Co., Ltd. · NCT07063251 · Data updated 2026-11-12
This trial tries a two-part gene therapy (HG005) injected into the eye to restore ABCA4 function and slow vision loss in children and teens with Stargardt disease type 1.
Quick facts
| Phase | Early Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 6 Years to 17 Years |
| Sex | All |
| Enrollment | 6 (estimated) |
| Sponsor | HuidaGene Therapeutics Co., Ltd. Industry-sponsored |
| Locations | 1 site (Shanghai) |
| Status | Recruiting |
| Started | August 20, 2025 |
| Main results expected | February 28, 2027 |
| Study ends | February 28, 2028 |
| Registry record updated | August 27, 2025 |
| Trial ID | NCT07063251 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Children and adolescents aged 6–17 with a clinical diagnosis of Stargardt disease type 1, at least one ABCA4 allele on each chromosome, well-defined macular atrophy in both eyes, and meeting study visual acuity and lab requirements are ideal candidates.
✕Probably not a fit
Patients with active intraocular inflammation, recent eye infection or surgery, corneal dystrophy, a history of HIV or hepatitis, prior gene or cell therapy, or very advanced retinal damage are unlikely to benefit from this early-phase intervention.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This early-phase 1 interventional study tests safety, tolerability, and preliminary efficacy of HG005, a dual AAV gene therapy designed to deliver a full-length ABCA4 protein to retinal cells. The approach uses two cooperating AAV vectors intended to reconstitute the large ABCA4 gene in the retina. Eligible participants are children and adolescents (ages 6–17) with clinical STGD1, biallelic ABCA4 variants, and well-defined macular atrophy who meet visual acuity and laboratory criteria. Outcomes will focus on safety and tolerability with exploratory measures of vision and retinal structure over follow-up visits at the single study site.
Why it matters
Potential benefit: If successful, HG005 could restore ABCA4 protein function in retinal cells and slow or halt progressive central vision loss in STGD1 patients.
How similar studies have gone: AAV gene therapy has produced clear clinical benefit for other inherited retinal diseases (for example, RPE65), but dual-AAV strategies for the large ABCA4 gene are experimental and currently have limited clinical proof-of-concept.
Who can join: the full rules
Show full inclusion / exclusion 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 dystrophy. * Meet visual acuity criteria based on ETDRS letter chart * Subject must agree to contraception during the study. * Acceptable hematology, clinical chemistry, urine laboratory, and protocol required eye examination. Exclusion Criteria: * Presence of active intraocular inflammation or uveitis history in either eye; * Presence of ocular or periocular infection history in either eye within 2 weeks prior to selection; * History or presence of corneal dystrophy in the study eye; * History of HIV or hepatitis A, B, or C infection; * Previous treatment with any gene therapy or cell therapy (e.g., stem cell transplantation); * Additional intraocular surgery in study eye 3 months prior to baseline visit; * Participation in an oral therapeutic STGD clinical trial within 3 months (or within 5 half-lives after last dose) prior to Screening * Any concomitant treatment that, in the opinion of the investigator, might interfere with the surgical procedure or healing process of the eye * Any other conditions that would not allow the potential subject to complete follow-up examinations during the study and would, in the opinion of the investigator, make the potential subject unsuitable for the study.
Where it's running
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Eye & ENT Hospital of Fudan University
Shanghai, China Recruiting
Study contacts
- Study coordinator: Study Director
- Email: HG00501@huidagene.com
- Phone: +86 021-25076143
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.