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

Plain-language summary

This study evaluates the safety, tolerability, and efficacy of HG005 in patients with Stargardt disease (an inherited cause of vision loss). It is open to patients aged 6-17 who carry changes in the ABCA4 gene and have well-defined atrophic lesions in the macula. The study is being conducted in Early Phase 1 and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Early Phase 1

Status

Recruiting

Sponsor

HuidaGene Therapeutics Co., Ltd.

Intervention

HG005

Start date

8/20/2025

Estimated completion

2/28/2028

Countries

China

Related genes

Locations

• Shanghai, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.