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

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

Status

Active, not recruiting

Phases

Early Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06088992

Enrollment

Registered

2023-10-18

Start date

2023-01-10

Completion date

2028-10-30

Last updated

2024-09-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Leber Congenital Amaurosis

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.

Interventions

GENETICHG004

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.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

8 Years to 50 Years

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
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)

Countries

China

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026