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

September 13, 2024 updated by: HuidaGene Therapeutics Co., Ltd.

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)

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

20

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

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.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: HG004
Drug: HG004
Low dose Medium dose High dose

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of ocular and non-ocular adverse events
Time Frame: 52 weeks
52 weeks

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in Best Corrected Visual Acuity (BCVA) of letters based on the Early Treatment Diabetic Retionpathy Study (ETDRS) chart
Time Frame: 52 weeks
52 weeks
Change from baseline in visual fields of full-field stimulus threshold test in log cd.s/m2.
Time Frame: 52 weeks
52 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

HuidaGene Therapeutics Co., Ltd.

Collaborators

Cholgene Therapeutics, Inc.

Investigators

  • 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 Major Dates

Study Start (Actual)

October 31, 2023

Primary Completion (Estimated)

December 1, 2025

Study Completion (Estimated)

December 1, 2025

Study Registration Dates

First Submitted

May 9, 2023

First Submitted That Met QC Criteria

June 13, 2023

First Posted (Actual)

June 18, 2023

Study Record Updates

Last Update Posted (Actual)

September 19, 2024

Last Update Submitted That Met QC Criteria

September 13, 2024

Last Verified

September 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HG00402

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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