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

NCT05906953 | Recruiting Phase 1 FDA Drug

• 1 other identifier: HG00402
• Study Type: interventional
• Target: 20
• Locations: 2 countries
• Sites: 3

Brief 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

Keywords

IRD; LCA; LCA2

Outcome Measures

Primary Outcomes (1)

• Incidence of ocular and non-ocular adverse events

  52 weeks

Secondary Outcomes (2)

• Change from baseline in Best Corrected Visual Acuity (BCVA) of letters based on the Early Treatment Diabetic Retionpathy Study (ETDRS) chart

  52 weeks

• Change from baseline in visual fields of full-field stimulus threshold test in log cd.s/m2.

  52 weeks

Study Arms (1)

HG004

EXPERIMENTAL

Drug: HG004

Interventions

HG004 DRUG

Low dose Medium dose High dose

HG004

Eligibility Criteria

• Age: 6 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• HuidaGene Therapeutics Co., Ltd.: lead

Study Sites (3)

Research Site

Sacramento, California, 95817, United States

RECRUITING

Research Site

Houston, Texas, 77707, United States

RECRUITING

Research Site

Shanghai, Shanghai Municipality, China

RECRUITING

MeSH Terms

Conditions

Leber Congenital Amaurosis

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Diseases

Study Officials

• Study Director

  HuidaGene Therapeutics Co., Ltd.

  STUDY DIRECTOR

Central Study Contacts

Study Director

CONTACT

732-318-9873; HG00402@huidagene.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 9, 2023
• First Posted: June 18, 2023
• Study Start: October 31, 2023
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: September 19, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1); US (2)