Quote:
Originally Posted by Carolyn
Neurologix Initiates Recruitment for Phase 2 Parkinson's Disease Trial
Tuesday August 19, 8:00 am ET
Multicenter Study of Novel Gene Transfer Approach
http://biz.yahoo.com/bw/080819/20080819005335.html?.v=1
FORT LEE, N.J.--(BUSINESS WIRE)--Neurologix, Inc. (OTCBB:NRGX - News), 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.
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This is another example of sham surgery for placebo control plus additional risk for study participants from gene therapy, an unknown risk. Carolyn, do you know how long the control patients are under anisthesia? Are there provisions for crossover to treatment for patients who receive the placebo? Is that follow-on guaranteed by the company? Were the FDA Patient consultants asked to represent patient views in the design decisions?
These are a few of my questions to the FDA and the company based on our pipeliner experiences with many clinical trials. We have asked FDA to consider participation in a round table discussion on the issues around sham surgery, The question in this case is what is the value of the data gained by a burr hole and exposure to anesthesia as designed to trick the patients into uncertainty about whether they got the treatment. On the face of it, to me this design would undermine the therapeutic impact for those who got the treatment as well. In order to think through the logic of the designs carefully, we need to hold this workshop as soon as possible, with FDA and scientists. I expect that PDF will sponsor the meeting this fall. In meantime I would like to hear responses from PWP who are eligible for this study what factors they consider important in entering this clinical trial.
Lets get the discussion started to find out how PWP perceive the risks and benefits, to let them know how we and other PWP feel. Then we can either endorse the practice or condemn it. If we do nothing they will continue to ask for this risk to be taken on by volunteers desperate for relief.
Perry
Perry