Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
NCT ID: NCT05906953
Last Updated: 2024-09-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
20 participants
INTERVENTIONAL
2023-10-31
2025-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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HG004
HG004
Low dose Medium dose High dose
Interventions
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HG004
Low dose Medium dose High dose
Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
6 Years
50 Years
ALL
No
Sponsors
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HuidaGene Therapeutics Co., Ltd.
INDUSTRY
Responsible Party
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Principal Investigators
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Study Director
Role: STUDY_DIRECTOR
HuidaGene Therapeutics Co., Ltd.
Locations
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Research Site
Sacramento, California, United States
Research Site
Houston, Texas, United States
Research Site
Shanghai, Shanghai Municipality, China
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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HG00402
Identifier Type: -
Identifier Source: org_study_id
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