Neurologix Initiates Recruitment for Phase 2 Parkinson's Disease Trial
- Category: DNA RNA and Cells
- Published on Tuesday, 19 August 2008 02:00
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Multicenter Study of Novel Gene Transfer Approach
FORT LEE, N.J., USA | August 19, 2008 | Neurologix, Inc., a biotechnology company engaged in the development of innovative gene therapies for the brain and central nervous system, announced today that it has received Institutional Review Board approvals to begin recruiting and enrolling participants for its Phase 2 clinical trial of the company's gene transfer approach to the treatment of advanced Parkinson's disease. The study is designed to evaluate the safety and efficacy of a novel non-dopaminergic approach for reestablishing motor function in Parkinson's patients who are sub-optimally responsive to drug therapies.
The randomized, double-blind, sham-procedure controlled trial will involve up to 10 leading academic research centers across the United States, with the first sites being Massachusetts General Hospital and Wake Forest University Health Sciences. Neurologix expects to enroll a total of 40 study participants in the trial. Twenty participants will receive an infusion of the gene-based treatment bilaterally via a catheter temporarily placed in each subthalamic nucleus (STN) (a deep brain structure that is the main target of surgery to treat Parkinson's disease) by stereotactic surgery. The other 20 participants will receive sterile saline solution into a partial thickness burr hole made into the skull, with no brain infusion.
Study participants will be assessed for treatment effects by standardized Parkinson's disease ratings at multiple time points post-procedure. The primary endpoint for the study will be a clinical assessment of motor function at 6 months using the Unified Parkinson's Disease Rating Scale (UPDRS). All participants in the study will also be monitored for safety for 12 months following the gene transfer procedure. If the primary endpoint is met following the analysis of 6 month data, then the sham-control participants will be offered the opportunity to crossover into an open label study of the Neurologix gene transfer therapy if they continue to meet all entry, medical and surgical criteria.
"Parkinson's disease is a devastating illness for those patients whose symptoms are no longer well controlled by medication alone," said John Mordock, President and Chief Executive Officer of Neurologix. "We are very pleased to initiate this study, which may offer a new therapeutic option that is potentially disease modifying and more consistent with the brain's normal metabolic activity."
For details about participating in the clinical trial, please visit http://clinicaltrials.gov/ct2/show/NCT00643890?term=neurologix&rank=1.
About the Neurologix Gene Transfer Approach to Parkinson's Disease
In Parkinson's disease, patients lose dopamine-producing brain cells, which results in substantial reductions in the activity and amount of GABA (gamma-aminobutyric acid), a major inhibitory neurotransmitter in the brain that helps 'quiet' excessive neuronal firing. This reduction in GABA causes a dysfunction in brain circuitry responsible for coordinating movement. GABA is made by a gene called glutamic acid decarboxylase, or GAD.
Neurologix's gene transfer approach to Parkinson's disease seeks to restore GABA -- and thus improve the patient's motor control -- by inserting the GAD gene back into an area of the brain called the subthalamic nucleus, a key regulatory center for movement.
Neurologix, Inc. (NRGX.OB) is a clinical-stage biotechnology company dedicated to the discovery, development, and commercialization of life-altering gene transfer therapies for serious disorders of the brain and central nervous system (CNS). Neurologix's therapeutic approach is built upon the groundbreaking research of its scientific founders and advisors, whose accomplishments have formed the foundation of gene therapy for neurological illnesses. Current company programs address such conditions as Parkinson's disease, epilepsy and Huntington's chorea, all of which are large markets not adequately served by current therapeutic options. For more information, please visit the Neurologix website at http://www.neurologix.net.