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

History of Changes

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:

Genetics Home Reference related topics: Parkinson disease Perry syndrome

MedlinePlus related topics: Brain Diseases Degenerative Nerve Diseases Movement Disorders Neurologic Diseases Parkinson's Disease

U.S. FDA Resources

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:

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

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:

LeWitt PA, Rezai AR, Leehey MA, Ojemann SG, Flaherty AW, Eskandar EN, Kostyk SK, Thomas K, Sarkar A, Siddiqui MS, Tatter SB, Schwalb JM, Poston KL, Henderson JM, Kurlan RM, Richard IH, Van Meter L, Sapan CV, During MJ, Kaplitt MG, Feigin A. AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial. Lancet Neurol. 2011 Apr;10(4):309-19.

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, 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
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases

Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases

ClinicalTrials.gov processed this record on May 23, 2013

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