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

Phase 1

Completed

• Conditions: Parkinson's Disease

• Registration Number: NCT05603312
• Lead Sponsor: MeiraGTx, LLC

Brief 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.

Study Timeline

• Study Start: 2022-10-05
• Study First Submitted: 2022-10-12
• Study First Submitted QC: 2022-10-28
• Study First Posted: 2022-11-02
• Primary Completion: 2024-09-06
• Study Completion: 2024-09-06
• Results First Submitted: 2025-08-04
• Status Verified: 2025-09
• Results First Submitted QC: 2025-09-02
• Last Update Submitted: 2025-09-02
• Results First Posted: 2025-09-05
• Last Update Posted: 2025-09-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 14

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
Incidence of Adverse Events Related to the Treatment	Baseline to Week 26	The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related treatment-emergent adverse events.
Incidence of Serious Adverse Events Related to the Treatment	Baseline to Week 26	The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related serious treatment-emergent adverse events.

Trial Locations

Locations (6)

Yale University - Yale New Haven Hospital; 🇺🇸 New Haven, Connecticut, United States

Henry Ford West Bloomfield Hospital; 🇺🇸 West Bloomfield, Michigan, United States

Weill Cornell Medicine; 🇺🇸 New York, New York, United States

Columbia University Irving Medical Center; 🇺🇸 New York, New York, United States

Thomas Jefferson University; 🇺🇸 Philadelphia, Pennsylvania, United States

West Virginia University; 🇺🇸 Morgantown, West Virginia, United States