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

Launched by HUIDAGENE THERAPEUTICS CO., LTD. · Jun 13, 2023

Keywords

ClinConnect Summary

The STAR trial is a clinical study looking at a new gene therapy called HG004 to treat a rare eye condition known as Leber Congenital Amaurosis (LCA), which is caused by changes in a specific gene called RPE65. This trial aims to find out if this treatment is safe and effective for people with LCA. The study is currently recruiting participants, and it is open to individuals aged 6 to 50 years who have been diagnosed with LCA due to RPE65 mutations and have certain levels of vision problems.

To be eligible for the trial, participants should be willing to follow the study's guidelines and have a confirmed diagnosis of LCA. They should also be able to perform certain vision tests. However, individuals with specific eye conditions that could interfere with the study or those who have had recent eye surgeries may not qualify. Participants can expect to receive the gene therapy and will be monitored closely for safety and effectiveness. This study not only aims to help those with LCA but also contributes to the understanding of gene therapy for inherited retinal diseases.

Gender

ALL

Eligibility criteria

• 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.