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

Leber Congenital Amaurosis Clinical Trial

Official title:

An Investigator-Initiated Open-Label, Multiple-Dose Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy for 2 Leber's Congenital Amaurosis With RPE65 Mutation (LCA2)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06064565
• Other study ID #: HG00401
• Status: Recruiting
• Phase: Early Phase 1
• Start date: January 10, 2023
• Est. completion date: December 30, 2024

Study information

• Verified date: September 2023
• Source: HuidaGene Therapeutics Co., Ltd.
• Contact: Director Study
• Phone: +86 021-25076143
• Email: HG00401[@]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: 9
• Est. completion date: December 30, 2024
• Est. primary completion date: January 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years to 50 Years

Eligibility

Inclusion Criteria:

• Male or females between 8 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 = 20/160 or visual field less than 20 degrees in the eye to be injected.
• Acceptable hematology, clinical chemistry, and urine laboratory parameters.

Exclusion Criteria:

• OCT examination determined that the outer nuclear layer was not visible in the planned injection area (Bleb) in the study eye.
• 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 other condition that would not allow the potential subject to complete follow-up examinations during the study and would, in the opinion of the investigator, make the potential subject unsuitable for the study.

Related Conditions & MeSH terms

• Blindness
• Leber Congenital Amaurosis

Intervention

Genetic:
• HG004
Method of Administration: Once unilateral subretinal injection; The duration of the study is about 60 weeks for each subject including a 8-week screening period, enrollment/baseline visit, treatment visit, and 52 weeks follow-up period.

Locations

Country	Name	City	State
China	Xinhua Hospital affiliated with Shanghai Jiao Tong University School of Medicine	Shanghai	Shanghai

Sponsors (2)

Lead Sponsor	Collaborator
HuidaGene Therapeutics Co., Ltd.	Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence and severity of ocular and systemic adverse events	Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)	Time Frame: 26 weeks