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

A Phase 1/2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects with RPE65-associated Leber Congenital Amaurosis Type 2 (LCA2)

Status

Recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05906953

Enrollment

Registered

2023-06-18

Start date

2023-10-31

Completion date

2025-12-31

Last updated

2024-09-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Inherited Retinal Diseases Caused by RPE65 Mutations, Leber Congenital Amaurosis

Keywords

IRD, LCA, LCA2

Brief summary

The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.

Interventions

DRUGHG004

Low dose Medium dose High dose

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

6 Years to 50 Years

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame
Incidence of ocular and non-ocular adverse events	52 weeks

Secondary

Measure	Time frame
Change from baseline in Best Corrected Visual Acuity (BCVA) of letters based on the Early Treatment Diabetic Retionpathy Study (ETDRS) chart	52 weeks
Change from baseline in visual fields of full-field stimulus threshold test in log cd.s/m2.	52 weeks

Countries

China, United States

Contacts

Primary ContactStudy Director

HG00402@huidagene.com732-318-9873

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026