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

Recruiting now · Phase 1/Phase 2

Conditions studied: Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations

In brief

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.

Key facts

Study ID

NCT05906953

Run by

HuidaGene Therapeutics Co., Ltd.

People needed

20

Starts

2023-10-31

Expected to finish

2025-12-01

Last updated by the study team

2024-09-19

Who can join

Age: 6 and older, up to 50. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Research Site — Sacramento, California, United States (enrolling)
• Research Site — Houston, Texas, United States (enrolling)
• Research Site — Shanghai, Shanghai Municipality, China (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.