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Gene Therapy Trial Report

Summary

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


NCTID NCT05906953 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
Disease Ontology Term DOID:0110016; DOID:0110353
Compound Name HG004
Sponsor HuidaGene Therapeutics Co., Ltd.
Funder Type Industry
Recruitment Status
Enrollment Count 20 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant RPE65
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed medium dose
Dose 3 Undisclosed high dose

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-05-09
Completion Date 2025-12
Last Update 2024-09-19

Participation Criteria


Eligible Age 6 Years - 50 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 3
Locations United States,China

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates First patient dosed November 2023

Resources/Links