Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease
NCT ID: NCT00643890
Last Updated: 2012-02-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE2
44 participants
INTERVENTIONAL
2008-08-31
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
TREATMENT
QUADRUPLE
Interventions
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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 Criteria
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Inclusion Criteria
* 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
* 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
30 Years
75 Years
ALL
No
Sponsors
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Neurologix, Inc.
INDUSTRY
Responsible Party
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Locations
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Stanford University
Stanford, California, United States
University of Colorado
Aurora, Colorado, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Henry Ford Health Systems - Franklin Pointe Medical
Southfield, Michigan, United States
University of Rochester
Rochester, New York, United States
Wake Forest University Health Science Center
Winston-Salem, North Carolina, United States
The Ohio State University
Columbus, Ohio, United States
Countries
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References
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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. doi: 10.1016/S0140-6736(07)60982-9.
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. doi: 10.1073/pnas.0706006104. Epub 2007 Nov 27.
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. doi: 10.1016/S1474-4422(11)70039-4.
Niethammer M, Tang CC, LeWitt PA, Rezai AR, Leehey MA, Ojemann SG, Flaherty AW, Eskandar EN, Kostyk SK, Sarkar A, Siddiqui MS, Tatter SB, Schwalb JM, Poston KL, Henderson JM, Kurlan RM, Richard IH, Sapan CV, Eidelberg D, During MJ, Kaplitt MG, Feigin A. Long-term follow-up of a randomized AAV2-GAD gene therapy trial for Parkinson's disease. JCI Insight. 2017 Apr 6;2(7):e90133. doi: 10.1172/jci.insight.90133.
Other Identifiers
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NRGX-GAD-02
Identifier Type: -
Identifier Source: org_study_id
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