Contact SCGE
Your email
Message
Send
SCGE Consortium Home
About SCGE TCDC
Contact Us
License
Home
Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy
NCTID
NCT02161380
(View at clinicaltrials.gov)
Description
The study is a dose-escalation study, phase 1. The objective of this proposed clinical trial is to evaluate the safety of mitochondrially targeted ND4 gene therapy with the adeno-associated viral vector in appropriate LHON patients.
(Show More)
Development Status
Inactive
Indication
Leber's Hereditary Optic Neuropathy
Disease Ontology Term
DOID:705
Compound Name
ScAAV2-P1ND4v2
Sponsor
Byron Lam
Funder Type
Other
Recruitment Status
Active not recruiting
Enrollment Count
28
Results Posted
View Results
Therapy Information
Target Gene/Variant
ND4
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravitreal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2
Editor Type
Dose 1
5.0E8 vg
Dose 2
2.5E9 vg
Dose 3
1.0E10 vg
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2014-06-06
Completion Date
2025-03-31
Last Update
2024-09-04
Participation Criteria
Eligible Age
>=15 Years
Standard Ages
Child, Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Safe but low efficacy, researchers did not advance development
Resources/Links
Clinical Publications
(Abstract) Leber Hereditary Optic Neuropathy Gene Therapy: Longitudinal Relationships Among Visual Function and Anatomical Measures -ARVO 2023
Leber Hereditary Optic Neuropathy Gene Therapy: Longitudinal Relationships Among Visual Function and Anatomical Measures
Gene Therapy for Leber Hereditary Optic Neuropathy: Initial Results
Gene Therapy for Leber Hereditary Optic Neuropathy: Low- and Medium-Dose Visual Results
Leber Hereditary Optic Neuropathy Gene Therapy: Adverse Events and Visual Acuity Results of All Patient Groups
Preclinical Publications
Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial
Efficient expression of self-complementary AAV in ganglion cells of the ex vivo primate retina
Induction of rapid and highly efficient expression of the human ND4 complex I subunit in the mouse visual system by self-complementary adeno-associated virus
Mutant NADH dehydrogenase subunit 4 gene delivery to mitochondria by targeting sequence-modified adeno-associated virus induces visual loss and optic atrophy in mice
LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile
Protocol
Trial end points and natural history in patients with G11778A Leber hereditary optic neuropathy : preparation for gene therapy clinical trial