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A Safety and Efficacy Study of HG004 in Subjects With Leber Congenital Amaurosis

Primary Purpose

Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations

Status
Not yet recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
HG004
Sponsored by
HuidaGene Therapeutics Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Leber Congenital Amaurosis focused on measuring IRD, LCA, LCA2

Eligibility Criteria

6 Years - 50 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Research Site
  • Research Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

HG004

Arm Description

Outcomes

Primary Outcome Measures

Incidence of ocular and non-ocular adverse events

Secondary Outcome Measures

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

Full Information

First Posted
May 9, 2023
Last Updated
September 28, 2023
Sponsor
HuidaGene Therapeutics Co., Ltd.
Collaborators
Cholgene Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05906953
Brief Title
A Safety and Efficacy Study of HG004 in Subjects With Leber Congenital Amaurosis
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)
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
September 2023 (Anticipated)
Primary Completion Date
December 2025 (Anticipated)
Study Completion Date
December 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
HuidaGene Therapeutics Co., Ltd.
Collaborators
Cholgene Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
Keywords
IRD, LCA, LCA2

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
HG004
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
HG004
Intervention Description
Low dose Medium dose High dose
Primary Outcome Measure Information:
Title
Incidence of ocular and non-ocular adverse events
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
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
Title
Change from baseline in visual fields of full-field stimulus threshold test in log cd.s/m2.
Time Frame
52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Study Director
Phone
+862125076143
Email
HG00402@huidagene.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
HuidaGene Therapeutics Co., Ltd.
Official's Role
Study Director
Facility Information:
Facility Name
Research Site
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Study Director
Email
HG00402@huidagene.com
Facility Name
Research Site
City
Shanghai
State/Province
Shanghai
Country
China
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Study Director
Email
HG00402@huidagene.com

12. IPD Sharing Statement

Learn more about this trial

A Safety and Efficacy Study of HG004 in Subjects With Leber Congenital Amaurosis

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