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

Phase 1

Completed

• Conditions: Parkinson Disease
• Interventions: Genetic: AAV-GAD Dose 2; Genetic: AAV-GAD Dose 1; Procedure: Sham Surgery

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

Brief Summary

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.

Detailed Description

The planned length of participation in the study for each participant will be approximately 7 months. This includes a screening period of up to 40 days, randomization and 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
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-04
• Last Update Posted: 2024-10-08

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

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Exclusion Criteria

• Past 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 Montreal Cognitive Assessment (MoCA)≤20
• Beck Depression Inventory-II score of ≥ 20

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
AAV-GAD Dose 2 treatment group	AAV-GAD Dose 2	Eligible participants will receive bilateral infusion of AAV-GAD Dose 2 into the STN
AAV-GAD Dose 1 treatment group	AAV-GAD Dose 1	Eligible participants will receive bilateral infusion of AAV-GAD Dose 1 into the STN
Sham treatment group	Sham Surgery	Eligible participants will undergo a sham surgical procedure

Primary Outcome Measures

Name	Time	Method
Safety and tolerability of AAV-GAD delivered to the STN in patients with Parkinson's Disease	Baseline to Week 26	Number of participants with adverse events and serious adverse events

Trial Locations

Locations (6)

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

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