Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease
- Conditions
- Parkinson's Disease
- Registration Number
- NCT00643890
- Lead Sponsor
- Neurologix, Inc.
- Brief Summary
The purpose of this study is to determine the safety and efficacy of AAV-GAD gene transfer into the subthalamic nucleus (STN) region of the brain. This study involves the treatment of subjects with medically refractory Parkinson's disease (PD). The gene transfer product, a disabled virus with a gene called GAD, will be infused into the STN bilaterally using stereotactic surgical techniques. The overall goal of this approach is to normalize the activity of the STN and reduce the motor symptoms of PD.
Because the change in UPDRS demonstrated a positive outcome, the sham surgery subjects from the blinded portion of the study will be invited to crossover into the Open-label Arm portion of the study. The Open-label Arm will further evaluate the safety and efficacy of AAV-GAD gene transfer into the subthalamic nucleus (STN) region of the brain.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 44
- Idiopathic Parkinson's disease
- Duration of disease for at least 5 years
- Levodopa responsiveness for at least 12 months
- UPDRS Part 3 score ≥ 25 or more in "off" state
- Past history of brain surgery for PD
- Beck Depression Inventory Score ≥ 20
- Any history of cerebral insult or central nervous system infection
- Cognitive impairment score < 130 on the Mattis Dementia Rating Scale
- Focal neurological deficits
- Evidence of significant medical or psychiatric disorders
- Secondary Parkinsonism
- Atypical Parkinson's disease
- History of substance abuse
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Evaluate a change in UPDRS scores
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (7)
Stanford University
🇺🇸Stanford, California, United States
University of Colorado
🇺🇸Aurora, Colorado, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Henry Ford Health Systems - Franklin Pointe Medical
🇺🇸Southfield, Michigan, United States
University of Rochester
🇺🇸Rochester, New York, United States
Wake Forest University Health Science Center
🇺🇸Winston-Salem, North Carolina, United States
The Ohio State University
🇺🇸Columbus, Ohio, United States
Stanford University🇺🇸Stanford, California, United States