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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants
NCTID
NCT05603312
(View at clinicaltrials.gov)
Description
The objective of this clinical trial is to evaluate the safety and tolerability of adeno-associated virus (AAV)-mediated delivery of glutamic acid decarboxylase (GAD) gene transfer into the subthalamic nuclei (STN) of participants with Parkinson's Disease.
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Development Status
Active
Indication
Parkinson Disease
Disease Ontology Term
DOID:14330
Compound Name
AAV-GAD
Sponsor
MeiraGTx, LLC
Funder Type
Industry
Recruitment Status
Completed
Enrollment Count
14
Results Posted
Not Available
Therapy Information
Target Gene/Variant
GAD1/2
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Overexpression of protective allele/gene
Route of Administration
Intraparenchymal (subthalamic nucleus)
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2
Editor Type
none
Dose 1
7.0E10 vg
Dose 2
21E10 vg
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2022-10-12
Completion Date
2024-09-06
Last Update
2024-10-08
Participation Criteria
Eligible Age
25 Years - 85 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
6
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Initiation of Phase III design discussions in H2 2024
Resources/Links
Clinical Publications
AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial
Network modulation following sham surgery in Parkinson's disease
Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial
Gene therapy reduces Parkinson's disease symptoms by reorganizing functional brain connectivity
Long-term follow-up of a randomized AAV2- GAD gene therapy trial for Parkinson's disease
News and Press Releases
MeiraGTx Announces Positive Data from Randomized, Sham-controlled Clinical Bridging Study of AAV-GAD for the Treatment of Parkinson's Disease
Preclinical Publications
Subthalamic GAD gene therapy in a Parkinson's disease rat model
Related NCTID
Long Term Follow-Up: NCT05894343
Phase 1: NCT00195143
Phase 2: NCT00643890