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

NCT06088992 | Active Not Recruiting Early Phase 1

• 1 other identifier: HG00401
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of ocular and systemic adverse events

  Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)

  26 weeks

Study Arms (1)

HG004

EXPERIMENTAL

Genetic: HG004

Interventions

HG004 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.

HG004

Eligibility Criteria

• Age: 8 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Xinhua Hospital, Shanghai Jiao Tong University School of Medicine: lead
• HuidaGene Therapeutics Co., Ltd.: collaborator

Study Sites (1)

Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine

Shanghai, Shanghai Municipality, China

Location

MeSH Terms

Conditions

Leber Congenital Amaurosis

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Diseases

Study Officials

• Peiquan Zhao

  Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: October 12, 2023
• First Posted: October 18, 2023
• Study Start: January 10, 2023
• Primary Completion: October 30, 2024
• Study Completion (Estimated): October 30, 2028
• Last Updated: September 19, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)