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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Fragile X Clinical Trial on Novartis’s AFQ056 Opens Enrollment

Fragile X Clinical Trial on Novartis’s AFQ056 Opens Enrollment

Elizabeth M. Berry-Kravis, MD, PhD has informed us that Rush University Medical Center in Chicago is enrolling the first patient in the NeuroNext learning trial for children ages 3-6 this week. This is the start of a large-scale fragile X clinical trial of Novartis AFQ056 (an mGluR5 antagonist) with children.

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New Fragile X Clinical Trial for Children launching in June 2017

New Fragile X Clinical Trial for Children launching in June 2017

Rush University Medical Center Professor Elizabeth M. Berry-Kravis, MD, PhD, begins a large-scale clinical trial to study effects of AFQ056, an mGluR5 blocker, on learning in young children BERRY tenacious! You can say a lot about Elizabeth M. Berry-Kravis, MD, PhD: • University of Notre Dame and University of Chicago educated. • Professor of Pediatrics, Neurological Sciences, Biochemistry, Rush University Medical Center. • Board certified in neurology with a special qualification in child neurology. • Expert in Fragile X syndrome and other neurogenetic diseases. You can also say she’ll talk your ears off. Literally! And much of it may go over your head. Yes, she often talks in industry jargon. Plenty of it. OK, we get it. She’s smart. Super smart. She’s caring. Super caring. She’s knowledgeable. Super knowledgeable. But did you know she’s tenacious? Incredibly, super-duper, berry tenacious? Good thing for us she’s on our side. No doubt her

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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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