Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age6-50
SponsorHuidaGene Therapeutics Co., Ltd.
About this trial
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.
Eligibility criteria
Qualifiers
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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- HG004
Treatment groups
20 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov