- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT06064565
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial (LIGHT)
September 26, 2023 updated by: HuidaGene Therapeutics Co., Ltd.
An Investigator-Initiated Open-Label, Multiple-Dose Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy for 2 Leber's Congenital Amaurosis With RPE65 Mutation (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
Study Type
Interventional
Enrollment (Estimated)
9
Phase
- Early 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
- Name: Director Study
- Phone Number: +86 021-25076143
- Email: HG00401@huidagene.com
Study Locations
-
-
Shanghai
-
Shanghai, Shanghai, China
- Recruiting
- Xinhua Hospital affiliated with Shanghai Jiao Tong University School of Medicine
-
Contact:
- Peiquan Zhao, PhD
- Phone Number: +86 13311620396
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-
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 8 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 ≤ 20/160 or visual field less than 20 degrees in the eye to be injected.
- Acceptable hematology, clinical chemistry, and urine laboratory parameters.
Exclusion Criteria:
- OCT examination determined that the outer nuclear layer was not visible in the planned injection area (Bleb) in the study eye.
- 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 other condition that would not allow the potential subject to complete follow-up examinations during the study and would, in the opinion of the investigator, make the potential subject unsuitable for the study.
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
|
Method of Administration: Once unilateral subretinal injection; The duration of the study is about 60 weeks for each subject including a 8-week screening period, enrollment/baseline visit, treatment visit, and 52 weeks follow-up period.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Incidence and severity of ocular and systemic adverse events
Time Frame: Time Frame: 26 weeks
|
Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)
|
Time Frame: 26 weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Director Study, Huidagene Therapeuticd 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)
January 10, 2023
Primary Completion (Estimated)
January 30, 2024
Study Completion (Estimated)
December 30, 2024
Study Registration Dates
First Submitted
September 26, 2023
First Submitted That Met QC Criteria
September 26, 2023
First Posted (Actual)
October 3, 2023
Study Record Updates
Last Update Posted (Actual)
October 3, 2023
Last Update Submitted That Met QC Criteria
September 26, 2023
Last Verified
September 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- HG00401
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
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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ProQR TherapeuticsTerminatedEye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders CongenitalUnited States, Belgium
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Eyecure Therapeutics Inc.Beijing Tongren HospitalUnknownLeber Congenital Amaurosis, Retinitis PigmentosaChina
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QLT Inc.CompletedLCA (Leber Congenital Amaurosis) | RP (Retinitis Pigmentosa)Canada, United States, Germany, Netherlands, United Kingdom
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QLT Inc.CompletedLCA (Leber Congenital Amaurosis) | RP (Retinitis Pigmentosa)United States, Canada, Germany, Netherlands, United Kingdom
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ProQR TherapeuticsRecruitingEye Diseases | Neurologic Manifestations | Retinal Degeneration | Retinal Dystrophies | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders CongenitalBelgium, Brazil, Canada, Germany, Italy, Netherlands, United Kingdom
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University of Campania "Luigi Vanvitelli"Retina Italia OnlusCompletedLeber Congenital Amaurosis 2 | Retinitis Pigmentosa 20Italy
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QLT Inc.CompletedLeber Congenital Amaurosis (LCA) | Retinitis Pigmentosa (RP)United States, Canada, Denmark, Germany, Netherlands, Switzerland, United Kingdom
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ProQR TherapeuticsCompletedLeber's Congenital AmaurosisUnited States, Belgium
Clinical Trials on HG004
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Xinhua Hospital, Shanghai Jiao Tong University...HuidaGene Therapeutics Co., Ltd.RecruitingLeber Congenital AmaurosisChina
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HuidaGene Therapeutics Co., Ltd.Cholgene Therapeutics, Inc.RecruitingLeber Congenital Amaurosis | Inherited Retinal Diseases Caused by RPE65 MutationsUnited States, China