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. (Show More)
Indication	Parkinson's Disease
Compound Name	AAV-GAD
Sponsor	MeiraGTx, LLC
Funder Type	Industry
Status	Completed
Enrollment Count	14

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
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type
Dose 1	1 x 10^12 vg/mL
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	6
Locations	United States

Has US IND	True
Recent Updates	Initiation of Phase III design discussions in H2 2024