Phase 2 N=14 Randomized Triple-blind Treatment

A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants

Parkinson's Disease

Bottom Line

View on ClinicalTrials.gov: NCT05603312 ↗

Enrolled (actual)

Serious AEs

21.4%

Results posted

Sep 2025

Primary outcome: Primary: Incidence of Adverse Events Related to the Treatment — 0; 1; 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: AAV-GAD Low Dose (Genetic); AAV-GAD High Dose (Genetic); Sham Surgery (Procedure)
Age: Adult, Older Adult · 25+ yrs
Sex: All
Sponsor: MeiraGTx, LLC
Primary completion: Sep 2024

Outcome Measures

Outcome	Result	p-value
PRIMARY Incidence of Adverse Events Related to the Treatment	0; 1; 0	—
PRIMARY Incidence of Serious Adverse Events Related to the Treatment	0; 0; 0	—

Summary

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.

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 full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT05603312). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.