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Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease

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ClinicalTrials.gov Identifier: NCT00643890
Recruitment Status : Terminated (Due to financial reasons)
First Posted : March 26, 2008
Last Update Posted : February 22, 2012
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Parkinson's Disease Genetic: Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 44 participants
Allocation: Randomized
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Phase 2 Safety and Efficacy Study Evaluating Glutamic Acid Decarboxylase Gene Transfer to Subthalamic Nuclei in Subjects With Advanced Parkinson's Disease
Study Start Date : August 2008
Actual Primary Completion Date : December 2010

Resource links provided by the National Library of Medicine

Intervention Details:
  • Genetic: Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus
    One-time bilateral administration of rAAV-GAD at 1X10^12 vector genomes in 35 uL.

Primary Outcome Measures :
  1. Evaluate a change in UPDRS scores

Information from the National Library of Medicine

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Ages Eligible for Study:   30 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

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 ClinicalTrials.gov identifier (NCT number): NCT00643890

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United States, California
Stanford University
Stanford, California, United States, 94305-5401
United States, Colorado
University of Colorado
Aurora, Colorado, United States, 80045
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, Michigan
Henry Ford Health Systems - Franklin Pointe Medical
Southfield, Michigan, United States, 48034
United States, New York
University of Rochester
Rochester, New York, United States, 14642
United States, North Carolina
Wake Forest University Health Science Center
Winston-Salem, North Carolina, United States, 27157
United States, Ohio
The Ohio State University
Columbus, Ohio, United States, 43210
Sponsors and Collaborators
Neurologix, Inc.
Publications of Results:
Other Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Neurologix, Inc.
ClinicalTrials.gov Identifier: NCT00643890    
Other Study ID Numbers: NRGX-GAD-02
First Posted: March 26, 2008    Key Record Dates
Last Update Posted: February 22, 2012
Last Verified: February 2012
Keywords provided by Neurologix, Inc.:
Parkinson's disease
Gene transfer
Gene therapy
Subthalamic nucleus
Adeno-associated virus
Study placed in the following topic categories:
Ganglion Cysts
Movement Disorders
Parkinson Disease
Basal Ganglia Diseases
Central Nervous System Diseases
Parkinsonian Disorders
Neurodegenerative Diseases
Brain Diseases
Additional relevant MeSH terms:
Nervous System Diseases
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