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

NCT07063251 | Recruiting Early Phase 1

• 1 other identifier: HG00501
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief 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)

Keywords

Stargardt disease type 1 (STGD1); Stargardt Macular Dystrophy; Stargardt Macular Degeneration; HG005; AAV; Gene Therapy

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of systemic adverse events

  Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)

  26 & 52 weeks

Secondary Outcomes (3)

• Change of study eye versus control eye in Low Luminance Visual Acuity(LLVA)

  52 weeks

• Change of study eye versus control eye in Fundus Autofluorescence (FAF)

  52 weeks

• Change of study eye versus control eye in Optical Coherence Tomography (OCT)

  52 weeks

Study Arms (1)

HG005

EXPERIMENTAL

The study will enroll up to 2 dose cohorts

Genetic: HG005

Interventions

HG005 GENETIC

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.

HG005

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• HuidaGene Therapeutics Co., Ltd.: lead

Study Sites (1)

Eye & ENT Hospital of Fudan University

Shanghai, China

RECRUITING

MeSH Terms

Conditions

Stargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Study Director

  HuidaGene Therapeutics Co., Ltd.

  STUDY DIRECTOR

Central Study Contacts

Study Director

CONTACT

+86 021-25076143; HG00501@huidagene.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 3, 2025
• First Posted: July 14, 2025
• Study Start: August 20, 2025
• Primary Completion (Estimated): February 28, 2027
• Study Completion (Estimated): February 28, 2028
• Last Updated: August 27, 2025

Record last verified: 2025-08

Locations

CN (1)