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

NCT ID: NCT06088992

Last Updated: 2024-09-19

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-01-10
• Study Completion Date: 2028-10-30

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.

Conditions

Leber Congenital Amaurosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

HG004

Group Type: EXPERIMENTAL

HG004

Intervention Type: GENETIC

Method of Administration: Once unilateralsubretinal injection; The duration of the study isabout 60 weeks for each subject including a 8-weekscreening period, enrollment/baseline visit,treatment visit, and 52 weeks follow-up period.

Interventions

HG004

Method of Administration: Once unilateralsubretinal injection; The duration of the study isabout 60 weeks for each subject including a 8-weekscreening period, enrollment/baseline visit,treatment visit, and 52 weeks follow-up period.

Intervention Type: GENETIC

Eligibility Criteria

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.

• Minimum Eligible Age: 8 Years
• Maximum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

HuidaGene Therapeutics Co., Ltd.

INDUSTRY

Sponsor Role: collaborator

Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

OTHER

Sponsor Role: lead

Responsible Party

Peiquan Zhao

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Peiquan Zhao

Role: PRINCIPAL_INVESTIGATOR

Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine

Locations

Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine

Shanghai, Shanghai Municipality, China

Countries

China

Other Identifiers

HG00401

Identifier Type: -

Identifier Source: org_study_id

NCT06064565

Identifier Type: -

Identifier Source: nct_alias