Back to resultsLong-term Follow-up of Glutamic Acid Decarboxylase (GAD) Gene Transfer in Parkinson's Disease
Primary Purpose
Parkinson Disease
Status
Enrolling by invitation
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
AAV-GAD
Sponsored by
About this trial
This is an interventional treatment trial for Parkinson Disease
Eligibility Criteria
Inclusion Criteria: Previously enrolled in Study MGT-GAD-025. Exclusion Criteria: None
Sites / Locations
- Henry Ford West Bloomfield Hospital
- Weill Cornell Medicine
- Columbia University Irving Medical Center
- Thomas Jefferson University
- West Virginia University
Arms of the Study
Arm 1
Arm 2
Arm Type
No Intervention
Experimental
Arm Label
Follow-up group
Active treatment group
Arm Description
Participants who were randomized to immediate treatment in Study MGT-GAD-025 will transition directly to a long-term follow-up schedule to complete an additional 54 months of follow-up. All study participants are to be followed for a period of 60 months after vector administration.
Participants who were randomized to sham surgery in Study MGT-GAD-025 will transition to an active treatment schedule including open-label bilateral treatment upon confirmation of continued eligibility. Upon completion of the treatment period, participants will enter the long-term follow-up schedule to complete a total of 60 months of follow-up.
Outcomes
Primary Outcome Measures
Number of participants with study drug-related adverse events and serious adverse events
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05894343
Brief Title
Long-term Follow-up of Glutamic Acid Decarboxylase (GAD) Gene Transfer in Parkinson's Disease
Official Title
Long-term Follow-up 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
June 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
May 19, 2023 (Actual)
Primary Completion Date
May 2028 (Anticipated)
Study Completion Date
May 2028 (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 study is to evaluate the long-term safety of AAV-GAD delivered bilaterally to the subthalamic nuclei (STN) in participants with Parkinson's disease.
Detailed Description
The duration of individual participation in this study will be approximately 54 months for participants who received AAV-GAD in Study MGT-GAD-025 and approximately 60 months for participants who received sham surgery in Study MGT-GAD-025.
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 will be assigned to the follow-up or active treatment group based on the group to which they were randomized in Study MGT-GAD-025.
Masking
None (Open Label)
Allocation
Randomized
Enrollment
14 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Follow-up group
Arm Type
No Intervention
Arm Description
Participants who were randomized to immediate treatment in Study MGT-GAD-025 will transition directly to a long-term follow-up schedule to complete an additional 54 months of follow-up. All study participants are to be followed for a period of 60 months after vector administration.
Arm Title
Active treatment group
Arm Type
Experimental
Arm Description
Participants who were randomized to sham surgery in Study MGT-GAD-025 will transition to an active treatment schedule including open-label bilateral treatment upon confirmation of continued eligibility. Upon completion of the treatment period, participants will enter the long-term follow-up schedule to complete a total of 60 months of follow-up.
Intervention Type
Genetic
Intervention Name(s)
AAV-GAD
Intervention Description
Bilateral infusion of AAV-GAD
Primary Outcome Measure Information:
Title
Number of participants with study drug-related adverse events and serious adverse events
Time Frame
From study start until Month 60 post treatment
10. Eligibility
Sex
All
Minimum Age & Unit of Time
25 Years
Maximum Age & Unit of Time
86 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Previously enrolled in Study MGT-GAD-025.
Exclusion Criteria:
None
Facility Information:
Facility Name
Henry Ford West Bloomfield Hospital
City
West Bloomfield
State/Province
Michigan
ZIP/Postal Code
48322
Country
United States
Facility Name
Weill Cornell Medicine
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Columbia University Irving Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Thomas Jefferson University
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Facility Name
West Virginia University
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26506
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Long-term Follow-up of Glutamic Acid Decarboxylase (GAD) Gene Transfer in Parkinson's Disease
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