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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
14
Bilateral infusion of AAV-GAD low dose
Bilateral infusion of AAV-GAD high dose
Sham infusion
Yale University - Yale New Haven Hospital
New Haven, Connecticut, United States
Henry Ford West Bloomfield Hospital
West Bloomfield, Michigan, United States
Weill Cornell Medicine
New York, New York, United States
Columbia University Irving Medical Center
New York, New York, United States
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.
Time frame: 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.
Time frame: Baseline to Week 26
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Thomas Jefferson University
Philadelphia, Pennsylvania, United States
West Virginia University
Morgantown, West Virginia, United States