RECRUITING

Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

Conditions

Leber Congenital Amaurosis Inherited Retinal Diseases Caused by RPE65 Mutations IRD LCA LCA2

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

A Phase 1/2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects with RPE65-associated Leber Congenital Amaurosis Type 2 (LCA2)

Quick Facts

Study Start:2023-10-31

Study Completion:2025-12

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05906953

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Years to 50 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion 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.	* 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.

Inclusion Criteria

Exclusion 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.

* 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.

Contacts and Locations

Study Contact

Study Director

CONTACT

732-318-9873

HG00402@huidagene.com

Principal Investigator

Study Director

STUDY_DIRECTOR

HuidaGene Therapeutics Co., Ltd.

Study Locations (Sites)

Research Site

Sacramento, California, 95817

United States

Research Site

Houston, Texas, 77707

United States

Collaborators and Investigators

Sponsor: HuidaGene Therapeutics Co., Ltd.

Study Director, STUDY_DIRECTOR, HuidaGene Therapeutics Co., Ltd.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-10-31

Study Completion Date2025-12

Study Record Updates

Study Start Date2023-10-31

Study Completion Date2025-12

Terms related to this study

Keywords Provided by Researchers

IRD
LCA
LCA2

Additional Relevant MeSH Terms

Leber Congenital Amaurosis
Inherited Retinal Diseases Caused by RPE65 Mutations