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Completed

NCT Number: NCT05603312

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

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.

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Key information

Conditions

Parkinson's Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Movement Disorders Nervous System Diseases Neurodegenerative Diseases Parkinson Disease Parkinsonian Disorders Synucleinopathies

Age range

25 year–85 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Yale University - Yale New Haven Hospital, New Haven, Connecticut, United States

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

AAV-GAD Low Dose

Genetic

Bilateral infusion of AAV-GAD low dose

AAV-GAD High Dose

Genetic

Bilateral infusion of AAV-GAD high dose

Sham Surgery

Procedure

Sham infusion

Primary outcomes

  1. Incidence of Adverse Events Related to the Treatment

    Time frame: 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.

  2. Incidence of Serious Adverse Events Related to the Treatment

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

MeiraGTx, LLC

Industry

Registry information

Official study title

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

Important dates

Study start
2022
Primary completion
2024
Study completion
2024
First posted
Nov 2, 2022
Registry last updated
Sep 5, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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