Actively Recruiting
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
Led by HuidaGene Therapeutics Co., Ltd. · Updated on 2024-09-19
20
Participants Needed
3
Research Sites
108 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female aged 6 to 50 years at the time of consent
- Willing to follow study protocol with informed consent or parental permission and assent
- Clinically confirmed diagnosis of Leber congenital amaurosis with RPE65 gene mutation
- Able to perform visual and retinal function tests
- Visual acuity of 20/80 or worse, or visual field less than 20 degrees in the eye to be treated
- Acceptable blood and urine lab test results
You will not qualify if you...
- Existing eye conditions that prevent planned surgery or affect study results, such as glaucoma needing surgery or corneal/lens opacities
- Epiretinal membrane detected by OCT imaging
- Systemic diseases or abnormal lab test results that affect eye function
- Eye surgery within the past six months
- Previous gene therapy or oligonucleotide treatments
- Any condition causing inability to comply with study or increased risk from participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Research Site
Sacramento, California, United States, 95817
Actively Recruiting
2
Research Site
Houston, Texas, United States, 77707
Actively Recruiting
3
Research Site
Shanghai, Shanghai Municipality, China
Actively Recruiting
Research Team
S
Study Director
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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