NCT05603312

Brief Summary

The objective of this clinical trial was 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Oct 2022

Geographic Reach
1 country

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 5, 2022

Completed
7 days until next milestone

First Submitted

Initial submission to the registry

October 12, 2022

Completed
21 days until next milestone

First Posted

Study publicly available on registry

November 2, 2022

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 6, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 6, 2024

Completed
12 months until next milestone

Results Posted

Study results publicly available

September 5, 2025

Completed
Last Updated

September 5, 2025

Status Verified

September 1, 2025

Enrollment Period

1.9 years

First QC Date

October 12, 2022

Results QC Date

August 4, 2025

Last Update Submit

September 2, 2025

Conditions

Parkinson's Disease

Outcome Measures

Primary Outcomes (2)

  • Incidence of Adverse Events Related to the Treatment

    The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related treatment-emergent adverse events.

    Baseline to Week 26

  • Incidence of Serious Adverse Events Related to the Treatment

    The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related serious treatment-emergent adverse events.

    Baseline to Week 26

Study Arms (3)

AAV-GAD Low Dose

EXPERIMENTAL

Eligible participants received bilateral infusion of AAV-GAD low dose into the STN

Genetic: AAV-GAD Low Dose

AAV-GAD High Dose

EXPERIMENTAL

Eligible participants received bilateral infusion of AAV-GAD high dose into the STN

Genetic: AAV-GAD High Dose

Sham Surgery

SHAM COMPARATOR

Eligible participants underwent a sham surgical procedure

Procedure: Sham Surgery

Interventions

AAV-GAD Low DoseGENETIC

Bilateral infusion of AAV-GAD low dose

AAV-GAD Low Dose
AAV-GAD High DoseGENETIC

Bilateral infusion of AAV-GAD high dose

AAV-GAD High Dose
Sham SurgeryPROCEDURE

Sham infusion

Sham Surgery

Eligibility Criteria

Age25 Years - 85 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Confirmed Parkinson's Disease
  • Levodopa responsiveness for at least 12 months
  • UPDRS Part 3 score of ≥25 points in the "off" state

You may not qualify if:

  • 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 the Montreal Cognitive Assessment (MoCA) ≤ 20
  • Beck Depression Inventory-II score of ≥ 20

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Yale University - Yale New Haven Hospital

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 DisordersBasal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMovement DisordersSynucleinopathiesNeurodegenerative Diseases

Results Point of Contact

Title
Program Manager
Organization
MeiraGTx, LLC
patients@meiragtx.com
646-860-7982

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: Participants are enrolled in one of three groups in parallel for the duration of the study.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 12, 2022

First Posted

November 2, 2022

Study Start

October 5, 2022

Primary Completion

September 6, 2024

Study Completion

September 6, 2024

Last Updated

September 5, 2025

Results First Posted

September 5, 2025

Record last verified: 2025-09

Data Sharing

IPD Sharing
Will not share

Locations

US(6)