Thirteen clinical centers across the U.S. are conducting a large-scale Fragile X clinical trial of Novartis AFQ056 (an mGluR5 antagonist) with children. With funding from the National Institutes of Health through the NeuroNext network, Dr. Elizabeth Berry-Kravis and colleagues aim to show effects of a targeted treatment — the mGluR5 blocker for Fragile X that normalizes brain plasticity in Fragile X mice but failed in previous adult human trials — can be better evaluated by studying effects on learning in young children.
In this study, 100 subjects with FXS, age 32 months to 6 years of age will enter a 12-month blinded treatment phase during which they are randomized to AFQ056 or placebo followed by an 8-month (open label) extension phase in which all participants will be treated with active drug.
Details about the clinical trial can be found on ClinicalTrials.gov. In October of 2016 Theodore Coutilish wrote about Elizabeth M. Berry-Kravis and the introduction to this trail.
If all goes well with with this trial, the entire Fragile X field will have new tools for future trials of promising new drug treatments.
To find out about enrollment, please contact Katherine J Friedmann, RN at (312) 942-9841 or firstname.lastname@example.org
For those who are curious, NeuroNext is an NINDS initiative that has an large amount of money and funds exploratory trials in neurological conditions. They awarded a whopping $11.5 million for this trial!