drugset / Trial / NCT00195143
Safety Study of Subthalamic Nucleus Gene Therapy for Parkinson's Disease
Phase 1
Completed
12 enrolled
Neurologix, Inc.
North Shore University Hospital · collabWeill Medical College of Cornell University · collab
Non-randomizedSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2003-08
- Primary completion
- —
- Completion
- 2005-08
Publications
- Background 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.
- 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.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAV-GAD | Gene therapy (AAV / viral vector) | — | Other |