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

Part of paid clinical trials in Sacramento, California.

Sponsor
HuidaGene Therapeutics Co., Ltd.
Study ID
NCT05906953
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Inherited Retinal Diseases Caused by RPE65 Mutations
  • Leber Congenital Amaurosis

Eligibility Criteria

Sex
ALL
Age
6 Years - 50 Years
Healthy Volunteers
Not accepted

Interventions

  • HG004 — DRUG
    Low dose Medium dose High dose

Study Details

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 Dates

Start date
Oct 31, 2023
Status verified
Sep 2024
Primary completion
Dec 31, 2025
Completion
Dec 31, 2025

Study Design

Enrollment
20 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: HG004

Primary Outcome Measure

Incidence of ocular and non-ocular adverse events [ Time Frame: 52 weeks ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
Research SiteSacramentoCalifornia95817
Study Director
[email protected]
Research SiteHoustonTexas77707
Study Director
[email protected]

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