Fragile X Clinical Trial on Novartis’s AFQ056 Opens Enrollment

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.

Elizabeth Berry-Kravis, MD, PhD, Fragile X researcher

Details about the clinical trial can be found on In October of 2016 Theodore Coutilish wrote about Elizabeth M. Berry-Kravis and the introduction to this trail.

Learn More about AFQ056 for Language Learning in Children With FXS

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

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!

Read the NeuroNext Grant Announcement