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Safety Study of Subthalamic Nucleus Gene Therapy for Parkinson's Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00195143
Recruitment Status : Completed
First Posted : September 19, 2005
Last Update Posted : March 27, 2008
Weill Medical College of Cornell University
North Shore University Hospital
Information provided by:
Neurologix, Inc.

Brief Summary:
The purpose of this study is to determine the safety of using a modified virus to transfer a gene called GAD into a region of the brain called the subthalamic nucleus in patients with advanced Parkinson's disease. The overall goal of this approach is to ultimately normalize the flow of information in several brain regions responsible for movement, to ultimately improve function in patients with this disorder. The current study is primarily designed to evaluate the safety of this approach, but patients are also being monitored for possible signs of effectiveness as well.

Condition or disease Intervention/treatment Phase
Parkinson's Disease Genetic: Surgical infusion of AAV-GAD into the subthalamic nucleus Phase 1

Detailed Description:
This study involves treatment of patients with medically refractory Parkinson's disease (PD) with gene therapy. The patients are chosen from a population of patients who would normally be candidates for standard deep brain stimulation (DBS) surgery for PD. These patients respond to medical therapy, but develop substantially reduced responses over time, often with severe fluctuations in their condition between a functional and severely non-functional state. Some patients also develop dose-limiting side effects from medication, including involuntary movements called dyskinesias and nightmares. When there are no medical contraindications, DBS is often performed in these patients to try to quiet hyperactive brain regions such as the subthalamic nucleus (STN). In PD, the STN is overactive due to a loss of GABA inputs to this region, which normally reduces neuronal firing. In turn, the STN drives other brain regions, including the globus pallidus (GPi) and substantia nigra (SNr), which are also hyperactive and which also have reduced GABA inputs. The goal of this gene therapy trial is to introduce the gene for glutamic acid decarboxylase (GAD) into the STN using an adeno-associated virus (AAV) vector, in order to permit the STN to produce it's own GABA, as well as release GABA into the GPi and SNr targets, which also have reduced GABA inputs. This is anticipated to restore a more normal pattern of information flow from this basal ganglia circuit to the thalamus and higher cortical structures in order to reduce the motor symptoms of PD, while eliminating complications arising from inserting DBS electrodes and batteries into the human body.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 12 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Study of Subthalamic GAD Gene Transfer in Medically Refractory Parkinson's Disease Patients
Study Start Date : August 2003
Study Completion Date : August 2005

Resource links provided by the National Library of Medicine

Primary Outcome Measures :
  1. Safety

Secondary Outcome Measures :
  1. Improvement in brain metabolism measured by PET scans
  2. Improvement in standard clinical rating scales

Information from the National Library of Medicine

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Ages Eligible for Study:   25 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Duration of disease: Greater than 5 years
  • Idiopathic Parkinson's disease
  • Parkinson's medication stable for 3 months
  • Absence of dementia
  • Hoehn and Yahr rating: 3 or greater and/or UPDRS: 30 or more in "off" state and/or Complications of l-dopa therapy limiting effective use

Exclusion Criteria:

  • Poor candidate for any surgery
  • Significant dementia
  • Secondary parkinsonism
  • Severe autonomic symptoms
  • Atypical Parkinson's disease
  • History of substance abuse

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00195143

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United States, New York
North Shore University Hospital
Manhasset, New York, United States, 11030
Sponsors and Collaborators
Neurologix, Inc.
Weill Medical College of Cornell University
North Shore University Hospital
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Principal Investigator: Michael G Kaplitt, MD PhD Weill Medical College of Cornell University
Principal Investigator: Matthew J During, MD Weill Medical College of Cornell University
Publications automatically indexed to this study by Identifier (NCT Number):
Layout table for additonal information Identifier: NCT00195143    
Other Study ID Numbers: 0902-478
First Posted: September 19, 2005    Key Record Dates
Last Update Posted: March 27, 2008
Last Verified: September 2005
Keywords provided by Neurologix, Inc.:
Parkinson's disease
Subthalamic nucleus
Adeno-associated virus
Gene therapy
Additional relevant MeSH terms:
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Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases