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.
Phase:
Phase 2
Details
Lead Sponsor:
Elizabeth Berry-Kravis
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)