Back to resultsA Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants
Primary Purpose
Parkinson Disease
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
AAV-GAD Dose 1
AAV-GAD Dose 2
Sham Surgery
Sponsored by
About this trial
This is an interventional treatment trial for Parkinson Disease
Eligibility 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
Sites / Locations
- Yale University - Yale New Haven Hospital
- Henry Ford West Bloomfield HospitalRecruiting
- Weill Cornell MedicineRecruiting
- Columbia University Irving Medical CenterRecruiting
- Thomas Jefferson UniversityRecruiting
- West Virginia UniversityRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Sham Comparator
Arm Label
AAV-GAD Dose 1 treatment group
AAV-GAD Dose 2 treatment group
Sham treatment group
Arm Description
Eligible participants will receive bilateral infusion of AAV-GAD Dose 1 into the STN
Eligible participants will receive bilateral infusion of AAV-GAD Dose 2 into the STN
Eligible participants will undergo a sham surgical procedure
Outcomes
Primary Outcome Measures
Safety and tolerability of AAV-GAD delivered to the STN in patients with Parkinson's Disease
Number of participants with adverse events and serious adverse events
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05603312
Brief Title
A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants
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
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 5, 2022 (Actual)
Primary Completion Date
March 1, 2024 (Anticipated)
Study Completion Date
March 1, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
MeiraGTx, LLC
4. Oversight
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
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Parkinson Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Model Description
Participants are enrolled in one of three groups in parallel for the duration of the study.
Masking
ParticipantInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
14 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
AAV-GAD Dose 1 treatment group
Arm Type
Experimental
Arm Description
Eligible participants will receive bilateral infusion of AAV-GAD Dose 1 into the STN
Arm Title
AAV-GAD Dose 2 treatment group
Arm Type
Experimental
Arm Description
Eligible participants will receive bilateral infusion of AAV-GAD Dose 2 into the STN
Arm Title
Sham treatment group
Arm Type
Sham Comparator
Arm Description
Eligible participants will undergo a sham surgical procedure
Intervention Type
Genetic
Intervention Name(s)
AAV-GAD Dose 1
Intervention Description
Bilateral infusion of AAV-GAD Dose 1
Intervention Type
Genetic
Intervention Name(s)
AAV-GAD Dose 2
Intervention Description
Bilateral infusion of AAV-GAD Dose 2
Intervention Type
Procedure
Intervention Name(s)
Sham Surgery
Intervention Description
Sham infusion
Primary Outcome Measure Information:
Title
Safety and tolerability of AAV-GAD delivered to the STN in patients with Parkinson's Disease
Description
Number of participants with adverse events and serious adverse events
Time Frame
Baseline to Week 26
Other Pre-specified Outcome Measures:
Title
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"
Description
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.
Time Frame
Baseline to Week 26
10. Eligibility
Sex
All
Minimum Age & Unit of Time
25 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
Eligibility 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
MeiraGTx Clinical Project Manager
Phone
+44 (0)2038664320
Email
mgtgad025@meiragtx.com
Facility Information:
Facility Name
Yale University - Yale New Haven Hospital
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06510
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Lindsey Fraczkiewicz
Email
lindsey.fraczkiewicz@yale.edu
First Name & Middle Initial & Last Name & Degree
Zion Zibly, MD
Facility Name
Henry Ford West Bloomfield Hospital
City
West Bloomfield
State/Province
Michigan
ZIP/Postal Code
48322
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kelly Tundo, RN, BSN,CCRP
Email
KCIACH1@hfhs.org
First Name & Middle Initial & Last Name & Degree
Jason Schwalb, MD,FAANS
Facility Name
Weill Cornell Medicine
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Meem Mahmud
Email
has9059@med.cornell.edu
First Name & Middle Initial & Last Name & Degree
Harini Sarva, MD
Facility Name
Columbia University Irving Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Natasha Desai
Email
nd2528@cumc.columbia.edu
First Name & Middle Initial & Last Name & Degree
Kimberly Kwei, MD
Facility Name
Thomas Jefferson University
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sara Thalheimer
Email
Sara.Thalheimer@jefferson.edu
First Name & Middle Initial & Last Name & Degree
Chengyuan Wu, MD
Facility Name
West Virginia University
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26506
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Shannon Filburn, RN, BSN, MBA
Email
sfilburn@hsc.wvu.edu
First Name & Middle Initial & Last Name & Degree
Ali Rezai, MD
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants
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