AFQ056 for Language Learning in Children With FXS

What is the purpose of this trial?

Brief Summary:

The overall goals are to change the paradigm for development of mechanism targeted pharmacotherapy in neurodevelopmental disorders and provide a definitive test of the mGluR theory in humans by determining whether AFQ056, an mGluR5 negative modulator, can enhance neural plasticity in the form of language learning during an intensive language intervention in very young children with fragile X syndrome. This trial therefore will use an innovative but exploratory new trial design to develop a different way to examine efficacy of an agent with substantial support as a drug targeting CNS plasticity in preclinical models of a developmental disorder. If the design is successful, this trial can serve as a model for future trials of mechanistically-targeted treatments operating on neural plasticity in other neurodevelopmental disorders.

Participation Guidelines

Ages: 6 years and younger

Gender: Both

National Institute of Neurological Disorders and Stroke

Start Date: 03/18/2019

End Date: 07/01/2020

Last Updated: 03/27/2019

Study HIC#: 2000024167

Get Involved

For more information about this study, contact:
Sara Jasak
+1 203-737-7629

If you would prefer to contact a member of the Help us Discover team about this trial and other similar trials, please email or call 1-877-978-8348.

Trial Image


Pamela Ventola

Principal Investigator