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Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease
This study is currently recruiting participants.
Verified by Neurologix, Inc., April 2009
First Received: March 24, 2008   Last Updated: April 23, 2009   History of Changes
Sponsored by: Neurologix, Inc.
Information provided by: Neurologix, Inc.
ClinicalTrials.gov Identifier: NCT00643890
  Purpose

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 patients 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.


Condition Intervention Phase
Parkinson's Disease
 Genetic: Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus
 Phase II

Genetics Home Reference related topics: familial paroxysmal nonkinesigenic dyskinesia Parkinson disease
MedlinePlus related topics: Brain Diseases Degenerative Nerve Diseases Movement Disorders Neurologic Diseases Parkinson's Disease Surgery
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Randomized, Double-Blind, Placebo Control, Safety/Efficacy Study
Official Title: Phase 2 Safety and Efficacy Study Evaluating Glutamic Acid Decarboxylase Gene Transfer to Subthalamic Nuclei in Subjects With Advanced Parkinson's Disease

Further study details as provided by Neurologix, Inc.:

Estimated Enrollment: 44
Study Start Date: August 2008
  Eligibility

Ages Eligible for Study:   30 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00643890

Contacts
Contact: Neurologix Incorporated 1-866-604-8665 info@neurologix.net

Locations
United States, California
Stanford University Not yet recruiting
Stanford, California, United States, 94305-5401
United States, Colorado
University of Colorado Recruiting
Aurora, Colorado, United States, 80045
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
United States, New York
University of Rochester Recruiting
Rochester, New York, United States, 14642
United States, North Carolina
Wake Forest University Health Science Center Recruiting
Winston-Salem, North Carolina, United States, 27157
United States, Ohio
The Ohio State University Recruiting
Columbus, Ohio, United States, 43210
United States, Pennsylvania
University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19106
Sponsors and Collaborators
Neurologix, Inc.
  More Information

Additional Information:
Live webcast to learn more about the Phase 2 clinical trial  This link exits the ClinicalTrials.gov site

Publications:
Kaplitt MG, Feigin A, Tang C, Fitzsimons HL, Mattis P, Lawlor PA, Bland RJ, Young D, Strybing K, Eidelberg D, During MJ. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet. 2007 Jun 23;369(9579):2097-105.
Feigin A, Kaplitt MG, Tang C, Lin T, Mattis P, Dhawan V, During MJ, Eidelberg D. Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease. Proc Natl Acad Sci U S A. 2007 Dec 4;104(49):19559-64. Epub 2007 Nov 27.

Responsible Party: Neurologix, Incorporated
Study ID Numbers: NRGX-GAD-02
Study First Received: March 24, 2008
Last Updated: April 23, 2009
ClinicalTrials.gov Identifier: NCT00643890     History of Changes
Health Authority: United States: Food and Drug Administration

Keywords provided by Neurologix, Inc.:
Keywords:
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:
Neurodegenerative Diseases
Central Nervous System Diseases
Nervous System Diseases
Basal Ganglia Diseases
Brain Diseases
Parkinsonian Disorders
Movement Disorders
Parkinson Disease

Study placed in the following topic categories:
Virus Diseases
Ganglion Cysts
Movement Disorders
Parkinson Disease
Basal Ganglia Diseases
 Central Nervous System Diseases
Parkinsonian Disorders
Neurodegenerative Diseases
Brain Diseases
Cysts

Additional relevant MeSH terms:
Movement Disorders
Parkinson Disease
Nervous System Diseases
Basal Ganglia Diseases
 Central Nervous System Diseases
Parkinsonian Disorders
Neurodegenerative Diseases
Brain Diseases

ClinicalTrials.gov processed this record on May 07, 2009



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