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
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Not specified
- Sponsor
- MeiraGTx, LLC
- Enrollment
- 14
- Locations
- 6
- Primary Endpoint
- Incidence of Adverse Events Related to the Treatment
- Status
- Completed
- Last Updated
- 7 months ago
Overview
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.
Detailed Description
The planned length of participation in the study for each participant was approximately 7 months, including a screening period of up to 40 days, randomization, surgery, and a follow-up period of 26 weeks.
Investigators
Eligibility Criteria
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
- •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
Outcomes
Primary Outcomes
Incidence of Adverse Events Related to the Treatment
Time Frame: Baseline to Week 26
The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related treatment-emergent adverse events.
Incidence of Serious Adverse Events Related to the Treatment
Time Frame: Baseline to Week 26
The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related serious treatment-emergent adverse events.