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

Leber Congenital Amaurosis Clinical Trial

Official title:

A Phase 1/2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects With RPE65-associated Leber Congenital Amaurosis Type 2 (LCA2)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05906953
• Other study ID #: HG00402
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: October 31, 2023
• Est. completion date: December 2025

Study information

• Verified date: November 2023
• Source: HuidaGene Therapeutics Co., Ltd.
• Contact: Study Director
• Phone: +862125076143
• Email: HG00402[@]huidagene.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: December 2025
• Est. primary completion date: December 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 50 Years

Eligibility

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.

Related Conditions & MeSH terms

• Blindness
• Leber Congenital Amaurosis
• Retinal Diseases

Intervention

Drug:
• HG004
Low dose Medium dose High dose

Locations

Country	Name	City	State
China	Research Site	Shanghai	Shanghai
United States	Research Site	Houston	Texas
United States	Research Site	Sacramento	California

Sponsors (2)

Lead Sponsor	Collaborator
HuidaGene Therapeutics Co., Ltd.	Cholgene Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of ocular and non-ocular adverse events		52 weeks
Secondary	Change from baseline in Best Corrected Visual Acuity (BCVA) of letters based on the Early Treatment Diabetic Retionpathy Study (ETDRS) chart		52 weeks
Secondary	Change from baseline in visual fields of full-field stimulus threshold test in log cd.s/m2.		52 weeks