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

NCT ID: NCT05603312

Last Updated: 2025-09-05

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

14 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-10-05

Study Completion Date

2024-09-06

Brief Summary

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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

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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

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Parkinson's Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Participants are enrolled in one of three groups in parallel for the duration of the study.
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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AAV-GAD Low Dose

Eligible participants received bilateral infusion of AAV-GAD low dose into the STN

Group Type EXPERIMENTAL

AAV-GAD Low Dose

Intervention Type GENETIC

Bilateral infusion of AAV-GAD low dose

AAV-GAD High Dose

Eligible participants received bilateral infusion of AAV-GAD high dose into the STN

Group Type EXPERIMENTAL

AAV-GAD High Dose

Intervention Type GENETIC

Bilateral infusion of AAV-GAD high dose

Sham Surgery

Eligible participants underwent a sham surgical procedure

Group Type SHAM_COMPARATOR

Sham Surgery

Intervention Type PROCEDURE

Sham infusion

Interventions

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AAV-GAD Low Dose

Bilateral infusion of AAV-GAD low dose

Intervention Type GENETIC

AAV-GAD High Dose

Bilateral infusion of AAV-GAD high dose

Intervention Type GENETIC

Sham Surgery

Sham infusion

Intervention Type PROCEDURE

Eligibility Criteria

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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
Minimum Eligible Age

25 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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MeiraGTx, LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Yale University - Yale New Haven Hospital

New Haven, Connecticut, United States

Site Status

Henry Ford West Bloomfield Hospital

West Bloomfield, Michigan, United States

Site Status

Weill Cornell Medicine

New York, New York, United States

Site Status

Columbia University Irving Medical Center

New York, New York, United States

Site Status

Thomas Jefferson University

Philadelphia, Pennsylvania, United States

Site Status

West Virginia University

Morgantown, West Virginia, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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MGT-GAD-025

Identifier Type: -

Identifier Source: org_study_id

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