Try our beta test site
IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...

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

This study has been completed.
Weill Medical College of Cornell University
North Shore University Hospital
Information provided by:
Neurologix, Inc. Identifier:
First received: September 15, 2005
Last updated: March 26, 2008
Last verified: September 2005
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 Intervention Phase
Parkinson's Disease
Genetic: Surgical infusion of AAV-GAD into the subthalamic nucleus
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase I Study of Subthalamic GAD Gene Transfer in Medically Refractory Parkinson's Disease Patients

Resource links provided by NLM:

Further study details as provided by Neurologix, Inc.:

Primary Outcome Measures:
  • Safety

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

Estimated Enrollment: 12
Study Start Date: August 2003
Estimated Study Completion Date: August 2005
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.

Ages Eligible for Study:   25 Years to 75 Years   (Adult, Senior)
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00195143

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
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
  More Information

Publications automatically indexed to this study by Identifier (NCT Number): Identifier: NCT00195143     History of Changes
Other Study ID Numbers: 0902-478
Study First Received: September 15, 2005
Last Updated: March 26, 2008

Keywords provided by Neurologix, Inc.:
Parkinson's disease
Subthalamic nucleus
Adeno-associated virus
Gene therapy

Additional relevant MeSH terms:
Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases processed this record on April 24, 2017