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 was 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)
Development Status	Active
Indication	Parkinson's Disease
Disease Ontology Term	DOID:14330
Compound Name	AAV-GAD
Sponsor	MeiraGTx, LLC
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	14 (ACTUAL)
Results Posted	View Results

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
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type	none
Dose 1	7.0E10 vg
Dose 2	21E10 vg

Eligible Age	25 Years - 85 Years
Standard Ages	Adult, Older adult
Sexes Eligible for Study	ALL
Eligibility Criteria	Inclusion Criteria: * Confirmed Parkinson's Disease * Levodopa responsiveness for at least 12 months * UPDRS Part 3 score of ≥25 points in the "off" state Exclusion Criteria: * History of brain surgery to treat Parkinson's Disease * Any history of cerebral insult or central nervous system infection * Atypical Parkinson's Disease * Focal or lateralized neurologic deficits * Evidence of significant medical or psychiatric disorders * Cognitive impairment as defined by the Montreal Cognitive Assessment (MoCA) ≤ 20 * Beck Depression Inventory-II score of ≥ 20 View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	6
Locations	United States

Has US IND	True
FDA Designations	Regenerative Medicine Advanced Therapy
Recent Updates	Initiation of Phase III design discussions in H2 2024