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

• Sponsor: MeiraGTx, LLC (Industry)
• Overall Status: Enrolling by invitation
• CT.gov ID: NCT05894343
• Collaborator: (none)
• Enrollment: 14
• Locations: 5
• Arms: 2
• Anticipated Duration (Months): 59.4
• Patients Per Site: 2.8
• Patients Per Site Per Month: 0

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Parkinson Disease	Genetic: AAV-GAD	Phase 1/Phase 2

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.

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 14 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Intervention 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.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)
• Primary Purpose: Treatment
• Official Title: Long-term Follow-up of Glutamic Acid Decarboxylase Gene Transfer to the Subthalamic Nuclei in Participants With Parkinson's Disease
• Actual Study Start Date: May 19, 2023
• Anticipated Primary Completion Date: May 1, 2028
• Anticipated Study Completion Date: May 1, 2028

Arms and Interventions

Arm	Intervention/Treatment
No Intervention: Follow-up group 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.
Experimental: Active treatment group 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; Bilateral infusion of AAV-GAD

Outcome Measures

Primary Outcome Measures

1. Number of participants with study drug-related adverse events and serious adverse events [From study start until Month 60 post treatment]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 25 Years to 86 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Previously enrolled in Study MGT-GAD-025.

Exclusion Criteria:

• None

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Henry Ford West Bloomfield Hospital	West Bloomfield	Michigan	United States	48322
2	Weill Cornell Medicine	New York	New York	United States	10021
3	Columbia University Irving Medical Center	New York	New York	United States	10032
4	Thomas Jefferson University	Philadelphia	Pennsylvania	United States	19107
5	West Virginia University	Morgantown	West Virginia	United States	26506

Sponsors and Collaborators

• MeiraGTx, LLC

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: MeiraGTx, LLC
• ClinicalTrials.gov Identifier: NCT05894343
• Other Study ID Numbers: MGT-GAD-026
• First Posted: Jun 8, 2023
• Last Update Posted: Jun 8, 2023
• Last Verified: Jun 1, 2023

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: Yes
• Additional relevant MeSH terms: Parkinson Disease

Study Results

No Results Posted as of Jun 8, 2023