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

NCT05906953 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2024-09-19

No results posted yet for this study

Summary

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

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

Interventions

DRUG

HG004

Low dose Medium dose High dose

Sponsors & Collaborators

  • HuidaGene Therapeutics Co., Ltd.

    lead INDUSTRY

Principal Investigators

  • Study Director · HuidaGene Therapeutics Co., Ltd.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-10-31
Primary Completion
2025-12-31
Completion
2025-12-31
FDA Drug
Yes

Countries

  • United States
  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05906953 on ClinicalTrials.gov