search
Back to results

Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)

Primary Purpose

Leber Congenital Amaurosis

Status
Recruiting
Phase
Early Phase 1
Locations
China
Study Type
Interventional
Intervention
HG004
Sponsored by
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Leber Congenital Amaurosis

Eligibility Criteria

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

Inclusion Criteria: Male or females between 8 and 50 years of age at the time of signing theinformed consent form. Willing to adhere to protocol as evidenced by written informed consent orparental permission and subject assent. Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) andmolecular diagnosis of LCA due to RPE65 mutations. Ability to perform tests of visual and retinal function. Visual acuity of ≤ 20/160 or visual field less than 20 degrees in the eye to beinjected. Acceptable hematology, clinical chemistry, and urine laboratory parameters. Exclusion Criteria: OCT examination determined that the outer nuclear layer was not visible inthe planned injection area (Bleb) in the study eye. Presence of epiretinal membrane by OCT. Complicating systemic diseases or clinically significant abnormal baselinelaboratory values. Complicating systemic diseases would include those in which the diseaseitself, or the treatment for the disease, can alter ocular function. Prior ocular surgery within six months. Prior gene therapy or oligonucleotide therapy treatments. Any other condition that would not allow the potential subject to completefollow-up examinations during the study and would, in the opinion of theinvestigator, make the potential subject unsuitable for the study.

Sites / Locations

  • Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of MedicineRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

HG004

Arm Description

Outcomes

Primary Outcome Measures

Incidence and severity of ocular and systemic adverse events
Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)

Secondary Outcome Measures

Full Information

First Posted
October 12, 2023
Last Updated
October 12, 2023
Sponsor
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Collaborators
HuidaGene Therapeutics Co., Ltd.
search

1. Study Identification

Unique Protocol Identification Number
NCT06088992
Brief Title
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)
Official Title
An Investigator-Initiated Open-Label, Multiple-Dose Clinical Study to Evaluate the Safety,Tolerability, and Efficacy of Gene Therapy for 2Leber's Congenital Amaurosis With RPE65 Mutation (LCA2)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
January 10, 2023 (Actual)
Primary Completion Date
January 30, 2024 (Anticipated)
Study Completion Date
December 30, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Collaborators
HuidaGene Therapeutics Co., Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
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 mutationsin RPE65 gene.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leber Congenital Amaurosis

7. Study Design

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

8. Arms, Groups, and Interventions

Arm Title
HG004
Arm Type
Experimental
Intervention Type
Genetic
Intervention Name(s)
HG004
Intervention Description
Method of Administration: Once unilateralsubretinal injection; The duration of the study isabout 60 weeks for each subject including a 8-weekscreening period, enrollment/baseline visit,treatment visit, and 52 weeks follow-up period.
Primary Outcome Measure Information:
Title
Incidence and severity of ocular and systemic adverse events
Description
Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)
Time Frame
26 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
8 Years
Maximum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or females between 8 and 50 years of age at the time of signing theinformed consent form. Willing to adhere to protocol as evidenced by written informed consent orparental permission and subject assent. Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) andmolecular diagnosis of LCA due to RPE65 mutations. Ability to perform tests of visual and retinal function. Visual acuity of ≤ 20/160 or visual field less than 20 degrees in the eye to beinjected. Acceptable hematology, clinical chemistry, and urine laboratory parameters. Exclusion Criteria: OCT examination determined that the outer nuclear layer was not visible inthe planned injection area (Bleb) in the study eye. Presence of epiretinal membrane by OCT. Complicating systemic diseases or clinically significant abnormal baselinelaboratory values. Complicating systemic diseases would include those in which the diseaseitself, or the treatment for the disease, can alter ocular function. Prior ocular surgery within six months. Prior gene therapy or oligonucleotide therapy treatments. Any other condition that would not allow the potential subject to completefollow-up examinations during the study and would, in the opinion of theinvestigator, make the potential subject unsuitable for the study.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Director Study
Phone
+862125076143
Email
HG00401@huidagene.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Peiquan Zhao
Organizational Affiliation
Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine
Official's Role
Principal Investigator
Facility Information:
Facility Name
Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine
City
Shanghai
State/Province
Shanghai
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Peiquan Zhao
Phone
+86 13311620396

12. IPD Sharing Statement

Learn more about this trial

Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)

We'll reach out to this number within 24 hrs