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

Sponsor

MeiraGTx, LLC (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05603312

Collaborator

(none)

Enrollment

Locations

Arms

10.9

Anticipated Duration (Months)

Patients Per Site

0.4

Patients Per Site Per Month

Study Details

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.

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

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

12 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

Participants are enrolled in one of two groups in parallel for the duration of the study.Participants are enrolled in one of two groups in parallel for the duration of the study.

Masking:

Triple (Participant, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Oct 5, 2022

Anticipated Primary Completion Date :

Sep 1, 2023

Anticipated Study Completion Date :

Sep 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: AAV-GAD treatment group Eligible participants will receive bilateral surgical infusion into the STN	Genetic: AAV-GAD Bilateral infusion of AAV-GAD
Sham Comparator: Sham treatment group Eligible participants will undergo a sham surgical procedure	Procedure: Sham Surgery Sham infusion

Arm

Intervention/Treatment

Experimental: AAV-GAD treatment group

Eligible participants will receive bilateral surgical infusion into the STN

Genetic: AAV-GAD

Bilateral infusion of AAV-GAD

Sham Comparator: Sham treatment group

Eligible participants will undergo a sham surgical procedure

Procedure: Sham Surgery

Sham infusion

Outcome Measures

Primary Outcome Measures

Safety and tolerability of AAV-GAD delivered to the STN in patients with Parkinson's Disease [Baseline to Week 26]
Number of participants with adverse events and serious adverse events

Other Outcome Measures

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" [Baseline to Week 26]
mean change from baseline to Weeks 12 and 26 for the AAV-GAD group compared to the sham group in MDS-UPDRS Part 3 (motor examination) score in the "off" state.

Eligibility Criteria

Criteria

Ages Eligible for Study:

25 Years to 85 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Henry Ford West Bloomfield Hospital	West Bloomfield	Michigan	United States	48322
2	Thomas Jefferson University	Philadelphia	Pennsylvania	United States	19107
3	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:

NCT05603312

Other Study ID Numbers:

MGT-GAD-025

First Posted:

Nov 2, 2022

Last Update Posted:

Jan 4, 2023

Last Verified:

Jan 1, 2023

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

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 Jan 4, 2023