Recruiting
Phase 1
Phase 2

HG004

Sponsor:

HuidaGene Therapeutics Co., Ltd.

Code:

NCT05906953

Conditions

Leber Congenital Amaurosis

Inherited Retinal Diseases Caused by RPE65 Mutations

Eligibility Criteria

Sex: All

Age: 6 - 50

Healthy Volunteers: Not accepted

Interventions

HG004

Study Details

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 mutations in RPE65 gene.

Conditions

Leber Congenital Amaurosis

Inherited Retinal Diseases Caused by RPE65 Mutations

Study ID

NCT05906953

Start date

Oct 31, 2023

Status verified date

Sep, 2024

Completion date

Dec, 2025

Anticipated

Primary completion date

Dec, 2025

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6 - 50

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Male or females between 6 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 ≤ 20/80 or visual field less than 20 degrees in the eye to be injected.
  • Acceptable hematology, clinical chemistry, and urine laboratory parameters.

Exclusion Criteria:

  • Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery (e.g., glaucoma requiring upcoming surgery, corneal or significant lenticular opacities).
  • 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 condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.

Study Design

Enrollment

20 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: HG004

Interventions

HG004

Low dose Medium dose High dose

Primary outcome measure

  • Incidence of ocular and non-ocular adverse events [ Time Frame: 52 weeks ]

Central Contacts and Locations

Central contacts

Study Director

732-318-9873HG00402@huidagene.com

Locations

Research Site

Recruiting

Sacramento, California, United States, 95817

Contacts

Study Director

HG00402@huidagene.com

Research Site

Recruiting

Houston, Texas, United States, 77707

Contacts

Study Director

HG00402@huidagene.com

More Information

Sponsor

HuidaGene Therapeutics Co., Ltd.

Last update posted

Sep 19, 2024

Last verified

Sep, 2024

Keywords

  • IRD
  • LCA
  • LCA2

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by HuidaGene Therapeutics Co., Ltd. on 2024-09-19.