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

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ClinicalTrials.gov Identifier: NCT05603312
Recruitment Status : Recruiting
First Posted : November 2, 2022
Last Update Posted : March 13, 2023
Sponsor:
Information provided by (Responsible Party):
MeiraGTx UK II Ltd ( MeiraGTx, LLC )

Study Description
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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.

Condition or disease Intervention/treatment Phase
Parkinson Disease Genetic: AAV-GAD Dose 1 Genetic: AAV-GAD Dose 2 Procedure: Sham Surgery Phase 1 Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 14 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Participants are enrolled in one of three groups in parallel for the duration of the study.
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : October 5, 2022
Estimated Primary Completion Date : March 1, 2024
Estimated Study Completion Date : March 1, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: AAV-GAD Dose 1 treatment group
Eligible participants will receive bilateral infusion of AAV-GAD Dose 1 into the STN
 Genetic: AAV-GAD Dose 1
Bilateral infusion of AAV-GAD Dose 1
Experimental: AAV-GAD Dose 2 treatment group
Eligible participants will receive bilateral infusion of AAV-GAD Dose 2 into the STN
 Genetic: AAV-GAD Dose 2
Bilateral infusion of AAV-GAD Dose 2
Sham Comparator: Sham treatment group
Eligible participants will undergo a sham surgical procedure
 Procedure: Sham Surgery
Sham infusion


Outcome Measures
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Primary Outcome Measures :
  1. Safety and tolerability of AAV-GAD delivered to the STN in patients with Parkinson's Disease [ Time Frame: Baseline to Week 26 ]
    Number of participants with adverse events and serious adverse events


Other Outcome Measures:
  1. The effect of AAV-GAD on the Unified Parkinson's Disease Study Rating Scale (MDS-UPDRS) Part 3 (motor function) score in the "off medication state" [ Time Frame: Baseline to Week 26 ]
    mean change from baseline to Weeks 12 and 26 for the AAV-GAD groups compared to the sham group in MDS-UPDRS Part 3 (motor examination) score in the "off" state.


Eligibility Criteria
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Ages Eligible for Study:   25 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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:

  • 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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05603312


Contacts
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Contact: MeiraGTx Clinical Project Manager +44 (0)2038664320 mgtgad025@meiragtx.com

Locations
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United States, Michigan
Henry Ford West Bloomfield Hospital Recruiting
West Bloomfield, Michigan, United States, 48322
Contact: Kelly Tundo, RN, BSN,CCRP       KCIACH1@hfhs.org   
Principal Investigator: Jason Schwalb, MD,FAANS         
United States, New York
Columbia University Irving Medical Center Recruiting
New York, New York, United States, 10032
Contact: Natasha Desai       nd2528@cumc.columbia.edu   
Principal Investigator: Kimberly Kwei, MD         
United States, Pennsylvania
Thomas Jefferson University Recruiting
Philadelphia, Pennsylvania, United States, 19107
Contact: Sara Thalheimer       Sara.Thalheimer@jefferson.edu   
Principal Investigator: Chengyuan Wu, MD         
United States, West Virginia
West Virginia University Recruiting
Morgantown, West Virginia, United States, 26506
Contact: Shannon Filburn, RN, BSN, MBA       sfilburn@hsc.wvu.edu   
Principal Investigator: Ali Rezai, MD         
Sponsors and Collaborators
MeiraGTx, LLC
More Information
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Responsible Party: MeiraGTx, LLC
ClinicalTrials.gov Identifier: NCT05603312    
Other Study ID Numbers: MGT-GAD-025
First Posted: November 2, 2022    Key Record Dates
Last Update Posted: March 13, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: Yes
Additional relevant MeSH terms:
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Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
 Nervous System Diseases
Movement Disorders
Synucleinopathies
Neurodegenerative Diseases