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

Tracking Information
First Received Date ^ICMJE	March 24, 2008
Last Updated Date	February 20, 2012
Start Date ^ICMJE	August 2008
Primary Completion Date	December 2010 (final data collection date for primary outcome measure)
Current Primary Outcome Measures ^ICMJE (submitted: June 6, 2011)	Evaluate a change in UPDRS scores [ Designated as safety issue: No ]
Original Primary Outcome Measures ^ICMJE	Not Provided
Change History	Complete list of historical versions of study NCT00643890 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ^ICMJE	Not Provided
Original Secondary Outcome Measures ^ICMJE	Not Provided
Current Other Outcome Measures ^ICMJE	Not Provided
Original Other Outcome Measures ^ICMJE	Not Provided

Descriptive Information
Brief Title ^ICMJE	Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease
Official Title ^ICMJE	Phase 2 Safety and Efficacy Study Evaluating Glutamic Acid Decarboxylase Gene Transfer to Subthalamic Nuclei in Subjects With Advanced Parkinson's Disease
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.
Detailed Description	Not Provided
Study Type ^ICMJE	Interventional
Study Phase	Phase 2
Study Design ^ICMJE	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Condition ^ICMJE	Parkinson's Disease
Intervention ^ICMJE	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.
Study Arm (s)	Not Provided
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.
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information
Recruitment Status ^ICMJE	Terminated
Estimated Enrollment ^ICMJE	44
Completion Date	Not Provided
Primary Completion Date	December 2010 (final data collection date for primary outcome measure)
Eligibility Criteria ^ICMJE	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
Gender	Both
Ages	30 Years to 75 Years
Accepts Healthy Volunteers	No
Contacts ^ICMJE	Contact information is only displayed when the study is recruiting subjects
Location Countries ^ICMJE	United States

Administrative Information
NCT Number ^ICMJE	NCT00643890
Other Study ID Numbers ^ICMJE	NRGX-GAD-02
Has Data Monitoring Committee	Yes
Responsible Party	Neurologix, Inc.
Study Sponsor ^ICMJE	Neurologix, Inc.
Collaborators ^ICMJE	Not Provided
Investigators ^ICMJE	Not Provided
Information Provided By	Neurologix, Inc.
Verification Date	February 2012
^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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