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

Parkinson Disease Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05603312
• Other study ID #: MGT-GAD-025
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: October 5, 2022
• Est. completion date: August 2024

Study information

• Verified date: March 2024
• Source: MeiraGTx UK II Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 14
• Est. completion date: August 2024
• Est. primary completion date: August 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 25 Years to 85 Years

Eligibility

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

Related Conditions & MeSH terms

• Parkinson Disease

Intervention

Genetic:
• AAV-GAD Dose 1
Bilateral infusion of AAV-GAD Dose 1
• AAV-GAD Dose 2
Bilateral infusion of AAV-GAD Dose 2

Procedure:
• Sham Surgery
Sham infusion

Locations

Country	Name	City	State
United States	West Virginia University	Morgantown	West Virginia
United States	Yale University - Yale New Haven Hospital	New Haven	Connecticut
United States	Columbia University Irving Medical Center	New York	New York
United States	Weill Cornell Medicine	New York	New York
United States	Thomas Jefferson University	Philadelphia	Pennsylvania
United States	Henry Ford West Bloomfield Hospital	West Bloomfield	Michigan

Sponsors (1)

Lead Sponsor	Collaborator
MeiraGTx, LLC

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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"	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.	Baseline to Week 26
Primary	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	Baseline to Week 26