NCT05603312

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

A Randomized, Double-Blind, Sham-Controlled Study to Evaluate the Safety and Tolerability of Glutamic Acid Decarboxylase Gene Transfer to the Subthalamic Nuclei in Participants With Parkinson's Disease

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: MeiraGTx, LLC · Industry
• Sex: All
• Age: 25 Years – 85 Years
• Healthy volunteers: Not accepted

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.

Detailed description

The planned length of participation in the study for each participant was approximately 7 months, including a screening period of up to 40 days, randomization, surgery, and a follow-up period of 26 weeks.

Conditions

• Parkinson's Disease

Interventions

Type	Name	Description
GENETIC	AAV-GAD Low Dose	Bilateral infusion of AAV-GAD low dose
GENETIC	AAV-GAD High Dose	Bilateral infusion of AAV-GAD high dose
PROCEDURE	Sham Surgery	Sham infusion

Timeline

• Start date: 2022-10-05
• Primary completion: 2024-09-06
• Completion: 2024-09-06
• First posted: 2022-11-02
• Last updated: 2025-09-05
• Results posted: 2025-09-05

Locations

6 sites across 1 country: United States

Regulatory

• FDA-regulated drug study
• FDA-regulated device study