FX-LEARN – Clinical Trial for Language Learning of AFQ056 in Children

FX-LEARN – Clinical Trial for Language Learning of AFQ056 in Children

The purpose of this NeuroNEXT study is to find out if the drug AFQ056, made by the pharmaceutical company Novartis, is safe and has beneficial effects on language learning in children who have fragile X syndrome (FXS). The study also aims to find out if a structured language intervention can help children with fragile X syndrome communicate better.

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Why Did Fragile X Clinical Trials of mGluR Antagonists Fail?

Why Did Fragile X Clinical Trials of mGluR Antagonists Fail?
Drug Tolerance and Dose Range Problems May Have Been the CulpritsAndy Tranfaglia and his dad, Mike Tranfaglia In my opinion, the Fragile X clinical trials of AFQ056 sponsored by Novartis failed because of a dose range that was inadequate for fragile X, and because of the unexpected development of tolerance. Dosage problems are relatively easy to correct, but tolerance to the degree we observed may be a kind of fatal flaw, at least if we're talking about mGluR5 antagonists.  The mGluR Theory of Fragile X is still probably correct; it's just that no one (least of all Novartis) expected tolerance to this drug -- indeed, I'm not sure they would agree that's what happened. I think we saw a much better response than most people because our son, Andy, was also on minocycline, effectively augmenting the response, and perhaps delaying the development of tolerance.  This may be a clue to understanding the mechanism of tolerance,Read more

Fragile X Clinical Trial: Novartis trial results are in, and they’re not pretty

AFQ056 Fragile X Clinical Trial showed Negative Results This year's Gordon Conference just finished, and Novartis presented their results for the first time (though advisors and advocates had been given a private peak months ago.) To say that the trial results for AFQ056 were disappointing would be the understatement of the century! While the company has already announced that the adult and adolescent trials failed to meet their pre-designated endpoints, the numbers looked really bad. This wasn't a case of the drug working, but placebo effects leading to an outcome that wasn't statistically significant; in this case, the effect of the drug was statistically significant, but in the wrong direction! So, what went wrong? The evidence for using mGluR5 antagonists in fragile X was really strong going into these trials---in fact, about as good as it ever gets. The drug itself was an advanced compound that had been studied extensively.Read more

Novartis Discontinues Development of mavoglurant (AFQ056) for Fragile X Syndrome

Novartis Clinical Trials in Fragile X Ended Novartis has announced that the company will be discontinuing its development program in Fragile X for its lead mGluR5 antagonist, mavoglurant (AFQ056), following negative results in a large international clinical trial in adults (reported in the Fall of 2013) and most recently, in a trial in adolescents. In both placebo-controlled trials, patients taking mavoglurant did not show improvement over placebo in any outcome measures. Novartis has also announced that the current open-label extension phase of the trial will be closed, but patients will be allowed to continue on the medication until their next scheduled clinic visit, or August 29, whichever comes first. No more of the drug will be dispensed to trial participants, but mavoglurant which has already been dispensed will not be recalled. We at FRAXA are disappointed by the negative results, but wish to thank Novartis for conducting superb clinical trials (at great

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Spotlight on two families – experiences on the extension trial of AFQ056

Spotlight on two families – experiences on the extension trial of AFQ056

Experiences on the Extension Trial of AFQ056 Alex Kowal and Taylor Stevenson, two young men who have Fragile X syndrome, have each completed a clinical trial and are now taking an experimental medication from Novartis, as part of a long term extension trial. “For the first time in his life, Alex is happy”, says his mom, Lisa.  Meet Taylor Stevenson   Meet Alex Kowal  

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