Parkinson's Disease Clinical Trials For posting and discussion of clinical trials related to Parkinson's Disease, and for the Parkinson Pipeline Project. All are welcome.


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Old 03-29-2012, 04:59 PM #4
Neuroscientist99 Neuroscientist99 is offline
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Neuroscientist99 Neuroscientist99 is offline
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Default Neurologix update

Quote:
Originally Posted by paula_w View Post
shoot i lost the first one. i hear there are just 34 participants . have they been unblinded? will the placebos not get anything now? that makes me so angry - people get their heads drilled and nothing for it. this is inexcusable. i hope someone in the know will inform us about the details, even the researchers for neurologix. it's important that patients get involved here and it's time for the govt. to bail out these companies who have success and are ready for phase III. i'm starting to think there is no phaseIII in parkinson trials. is a hybrid car worth more than peoples lives - millions having surgery and suffering - who could use the funding in a more deserving way. we have lost the priority of human life and replaced it with money and world control. watch Damascus.

please update if u know anything.
thank you
Dear Paula_w. The Neurologix story is tragic, as clearly the intervention had efficacy, with almost everyone in the study gaining some benefit. Most importantly, there were no significant adverse events, an achievement in itself. A total of 45 people were enrolled in the study and randomized one to one. 22 received the AAV-GAD active treatment, 23 received sham. A few patients randomized to GAD didnt receive the treatment as indicated, either because of missed target (the blinded design prevented revision surgery that is commonly carried out with DBS to ensure correct targeting). Moreover as the device used to deliver the GAD gene (AAV-GAD or NLX-P101) is not a commercial device, but still undergoing development, and so there were a few problems with infusion, pump failures etc. In the end 16 individuals got the treatment as intended. the other subjects either got partial treatment, or missed target or the device slipped out of the target prior to infusion. Of the patients randomize to the burr hole only (sham surgery), 14 of that group has now received the GAD treatment, with no significant problems reported. My understanding is that only 2 of the remaining 9 sham subjects were eligible candidates to receive GAD, and it is tragic that with the demise of NRGX, that the study is terminated. The hope is that the original developers of this technology can move forward with a new trial, fully optimized in every way. The Neurologix Phase 2 was the first of any biological therapy to reach its primary endpoint, with 10 of the subjects at one year having improved by 9 points or more on their UPDRS(motor) OFF scores. Moreover, those who didnt get such a good response on UPDRS still improved with increased ON time of at least 2 hours during the waking day. Hence, although the approach is no cure, it is a significant advance, and so it would be great if the inventors can move it along..
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