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

This study has been terminated.
(Due to financial reasons)
Sponsor:
Information provided by (Responsible Party):
Neurologix, Inc.
ClinicalTrials.gov Identifier:
NCT00643890
First received: March 24, 2008
Last updated: February 20, 2012
Last verified: February 2012
  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 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 Intervention Phase
Parkinson's Disease
Genetic: Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:


Further study details as provided by Neurologix, Inc.:

Primary Outcome Measures:
  • Evaluate a change in UPDRS scores [ Designated as safety issue: No ]

Estimated Enrollment: 44
Study Start Date: August 2008
Primary Completion Date: December 2010 (Final data collection date for primary outcome measure)
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.
  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
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 ClinicalTrials.gov identifier: NCT00643890

Locations
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.
  More Information

Publications:
Responsible Party: Neurologix, Inc.
ClinicalTrials.gov Identifier: NCT00643890     History of Changes
Other Study ID Numbers: NRGX-GAD-02
Study First Received: March 24, 2008
Last Updated: February 20, 2012
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:
Nervous System Diseases

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

ClinicalTrials.gov processed this record on October 23, 2014