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NCT Number: NCT06088992

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

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Leber Congenital Amaurosis Eye Diseases Eye Diseases, Hereditary Retinal Diseases

Age range

8 year–50 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine

Shanghai, Shanghai Municipality, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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.

Primary outcomes

  1. Incidence and severity of ocular and systemic adverse events

    Time frame: 26 weeks

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

Sponsors and collaborators

Lead sponsor

Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Other

Collaborators

  • HuidaGene Therapeutics Co., Ltd.

Registry information

Official study title

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)

Important dates

Study start
2023
Primary completion
2024
Study completion
2028
First posted
Oct 18, 2023
Registry last updated
Sep 19, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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