Study Stopped
Due to financial reasons
Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease
Phase 2 Safety and Efficacy Study Evaluating Glutamic Acid Decarboxylase Gene Transfer to Subthalamic Nuclei in Subjects With Advanced Parkinson's Disease
1 other identifier
interventional
44
1 country
7
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
7 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 24, 2008
CompletedFirst Posted
Study publicly available on registry
March 26, 2008
CompletedStudy Start
First participant enrolled
August 1, 2008
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2010
CompletedFebruary 22, 2012
February 1, 2012
2.3 years
March 24, 2008
February 20, 2012
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Evaluate a change in UPDRS scores
Interventions
One-time bilateral administration of rAAV-GAD at 1X10\^12 vector genomes in 35 uL.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Neurologix, Inc.lead
Study Sites (7)
Stanford University
Stanford, California, 94305-5401, United States
University of Colorado
Aurora, Colorado, 80045, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Henry Ford Health Systems - Franklin Pointe Medical
Southfield, Michigan, 48034, United States
University of Rochester
Rochester, New York, 14642, United States
Wake Forest University Health Science Center
Winston-Salem, North Carolina, 27157, United States
The Ohio State University
Columbus, Ohio, 43210, United States
Related Publications (4)
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.
PMID: 17586305BACKGROUNDFeigin 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.
PMID: 18042721BACKGROUNDLeWitt 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.
PMID: 21419704RESULTNiethammer 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.
PMID: 28405611DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 24, 2008
First Posted
March 26, 2008
Study Start
August 1, 2008
Primary Completion
December 1, 2010
Last Updated
February 22, 2012
Record last verified: 2012-02