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

Launched by HUIDAGENE THERAPEUTICS CO., LTD. · Jul 11, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new gene therapy called HG005 for Stargardt disease type 1 (STGD1), a rare inherited eye condition that causes gradual vision loss starting in childhood or adolescence. Stargardt disease happens because of a faulty gene called ABCA4, which normally helps clear waste from the retina, the part of the eye responsible for sharp, central vision. When this gene doesn’t work correctly, harmful substances build up and damage the retina, leading to vision problems. HG005 aims to deliver a healthy copy of this gene to the retina using a special method, with the hope of protecting the eye cells and slowing down or stopping further vision loss.

This study is looking for children and teenagers between 6 and 17 years old who have been diagnosed with Stargardt disease and meet certain eye health criteria. Both eyes need to show specific signs of the disease, and participants should have a certain level of vision based on an eye chart test. Those interested should be free from certain eye infections, inflammations, and other health issues, and must agree to use contraception during the study. Although the trial is not yet recruiting, participants can expect careful eye exams and monitoring throughout the study to see if the treatment is safe, well-tolerated, and helpful in protecting their vision. This trial offers hope for a condition that currently has no approved treatments.

Gender

ALL

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 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.