Safety Study of Subthalamic Nucleus Gene Therapy for Parkinson's Disease
Phase I Study of Subthalamic GAD Gene Transfer in Medically Refractory Parkinson's Disease Patients
1 other identifier
interventional
12
1 country
1
Brief Summary
The purpose of this study is to determine the safety of using a modified virus to transfer a gene called GAD into a region of the brain called the subthalamic nucleus in patients with advanced Parkinson's disease. The overall goal of this approach is to ultimately normalize the flow of information in several brain regions responsible for movement, to ultimately improve function in patients with this disorder. The current study is primarily designed to evaluate the safety of this approach, but patients are also being monitored for possible signs of effectiveness as well.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Aug 2003
Typical duration for phase_1
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
August 1, 2003
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2005
CompletedFirst Submitted
Initial submission to the registry
September 15, 2005
CompletedFirst Posted
Study publicly available on registry
September 19, 2005
CompletedMarch 27, 2008
September 1, 2005
September 15, 2005
March 26, 2008
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Safety
Secondary Outcomes (2)
Improvement in brain metabolism measured by PET scans
Improvement in standard clinical rating scales
Interventions
Eligibility Criteria
You may qualify if:
- Duration of disease: Greater than 5 years
- Idiopathic Parkinson's disease
- Parkinson's medication stable for 3 months
- Absence of dementia
- Hoehn and Yahr rating: 3 or greater and/or UPDRS: 30 or more in "off" state and/or Complications of l-dopa therapy limiting effective use
You may not qualify if:
- Poor candidate for any surgery
- Significant dementia
- Secondary parkinsonism
- Severe autonomic symptoms
- Atypical Parkinson's disease
- History of substance abuse
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Neurologix, Inc.lead
- Weill Medical College of Cornell Universitycollaborator
- North Shore University Hospitalcollaborator
Study Sites (1)
North Shore University Hospital
Manhasset, New York, 11030, United States
Related Publications (2)
Luo J, Kaplitt MG, Fitzsimons HL, Zuzga DS, Liu Y, Oshinsky ML, During MJ. Subthalamic GAD gene therapy in a Parkinson's disease rat model. Science. 2002 Oct 11;298(5592):425-9. doi: 10.1126/science.1074549.
PMID: 12376704BACKGROUNDKaplitt 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: 17586305DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Michael G Kaplitt, MD PhD
Weill Medical College of Cornell University
- PRINCIPAL INVESTIGATOR
Matthew J During, MD
Weill Medical College of Cornell University
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
September 15, 2005
First Posted
September 19, 2005
Study Start
August 1, 2003
Study Completion
August 1, 2005
Last Updated
March 27, 2008
Record last verified: 2005-09