Long-term Follow-up of Glutamic Acid Decarboxylase (GAD) Gene Transfer in Parkinson's Disease

NCT05894343 | Active Not Recruiting Phase 1 DMC FDA Drug FDA Device

• 1 other identifier: MGT-GAD-026
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 6

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.

Conditions

Parkinson's Disease

Outcome Measures

Primary Outcomes (1)

• Number of participants with study drug-related adverse events and serious adverse events

  From study start until Month 60 post treatment

Study Arms (2)

Follow-up group

NO INTERVENTION

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 60 months after vector administration.

Active treatment group

EXPERIMENTAL

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.

Genetic: AAV-GAD

Interventions

AAV-GAD GENETIC

Bilateral infusion of AAV-GAD

Active treatment group

Eligibility Criteria

• Age: 25 Years - 86 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Previously enrolled in Study MGT-GAD-025.

You may not qualify if:

• None

Sponsors & Collaborators

• MeiraGTx, LLC: lead

Study Sites (6)

Yale University

New Haven, Connecticut, 06510, United States

Location

Henry Ford West Bloomfield Hospital

West Bloomfield, Michigan, 48322, United States

Location

Weill Cornell Medicine

New York, New York, 10021, United States

Location

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

West Virginia University

Morgantown, West Virginia, 26506, United States

Location

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: May 30, 2023
• First Posted: June 8, 2023
• Study Start: May 19, 2023
• Primary Completion (Estimated): October 1, 2029
• Study Completion (Estimated): October 1, 2029
• Last Updated: August 17, 2025

Record last verified: 2025-08

Locations

US (6)