Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

Plain-language summary

This study evaluates HG004, a gene therapy, in people with Leber congenital amaurosis, an inherited vision loss disease caused by mutations in the RPE65 gene. Patients aged 6 to 50 with a clinically and molecularly confirmed diagnosis who meet certain vision criteria may take part. The study is in Phase 1/Phase 2 and is currently enrolling participants.

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

Trial details

Phase

PHASE1/PHASE2

Status

Recruiting

Sponsor

HuidaGene Therapeutics Co., Ltd.

Intervention

HG004

Start date

10/31/2023

Estimated completion

12/1/2025

Countries

United States, China

Related genes

Locations

• Sacramento, United States
• Houston, United States
• 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:

* Male or females between 6 and 50 years of age at the time of signing the informed consent form.
* Willing to adhere to protocol as evidenced by written informed consent or parental permission and subject assent.
* Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) and molecular diagnosis of LCA due to RPE65 mutations.
* Ability to perform tests of visual and retinal function.
* Visual acuity of ≤ 20/80 or visual field less than 20 degrees in the eye to be injected.
* Acceptable hematology, clinical chemistry, and urine laboratory parameters.

Exclusion Criteria:

* Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery (e.g., glaucoma requiring upcoming surgery, corneal or significant lenticular opacities).
* Presence of epiretinal membrane by OCT.
* Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
* Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function.
* Prior ocular surgery within six months.
* Prior gene therapy or oligonucleotide therapy treatments.
* Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.