Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)

Early Phase 1

Recruiting

• Conditions: Leber Congenital Amaurosis
• Interventions: Genetic: HG004

• Registration Number: NCT06088992
• Lead Sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

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 mutationsin RPE65 gene.

Detailed Description

Not available

Study Timeline

• Study Start: 2023-01-10
• Study First Submitted: 2023-10-12
• Study First Submitted QC: 2023-10-12
• Study First Posted: 2023-10-18
• Status Verified: 2023-11
• Last Update Submitted: 2023-11-21
• Last Update Posted: 2023-11-24
• Primary Completion: 2024-01-30
• Study Completion: 2024-12-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

• Male or females between 8 and 50 years of age at the time of signing theinformed consent form.
• Willing to adhere to protocol as evidenced by written informed consent orparental permission and subject assent.
• Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) andmolecular diagnosis of LCA due to RPE65 mutations.
• Ability to perform tests of visual and retinal function.
• Visual acuity of ≤ 20/160 or visual field less than 20 degrees in the eye to beinjected.
• Acceptable hematology, clinical chemistry, and urine laboratory parameters.

Exclusion Criteria

• OCT examination determined that the outer nuclear layer was not visible inthe planned injection area (Bleb) in the study eye.
• Presence of epiretinal membrane by OCT.
• Complicating systemic diseases or clinically significant abnormal baselinelaboratory values.
• Complicating systemic diseases would include those in which the diseaseitself, or the treatment for the disease, can alter ocular function.
• Prior ocular surgery within six months.
• Prior gene therapy or oligonucleotide therapy treatments.
• Any other condition that would not allow the potential subject to completefollow-up examinations during the study and would, in the opinion of theinvestigator, make the potential subject unsuitable for the study.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
HG004	HG004	-

Primary Outcome Measures

Name	Time	Method
Incidence and severity of ocular and systemic adverse events	26 weeks	Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)

Trial Locations

Locations (1)

Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine; 🇨🇳 Shanghai, Shanghai, China