This trial has completed enrollment and all participants have finished the trial. Investigators are now analyzing the data and hope to have results in early 2022.
Thirteen centers across the US enrolled children with Fragile X in a large-scale clinical trial of Novartis AFQ056. Dr. Elizabeth Berry-Kravis and colleagues aim to show that this targeted treatment — an mGluR5 blocker for Fragile X which failed in previous adult human trials — can be better evaluated by studying effects on learning in young children.
One hundred subjects with FXS, aged 32 months to 6 years, participated in a 12-month blinded treatment phase during which they were randomized to AFQ056 or placebo, followed by an 8-month (open label) extension phase in which all participants will be treated with active drug.
The purpose of this study is to find out if AFQ056 is safe and has beneficial effects on language learning in children who have Fragile X syndrome. The study also aims to find out if a structured language intervention can improve communication skills with or without the drug.
The phase II trial is coordinated by NeuroNEXT (Network for Excellence in Neuroscience Clinical Trials), with support and funding from the National Institutes of Health (NIH). Dr. Elizabeth Berry-Kravis at Rush University is leading this national Fragile X clinical trial.
If all goes well, the entire Fragile X field will have new methods and tools for future trials of promising new treatments.
For those who are curious, NeuroNext is an NINDS initiative that funds exploratory trials in neurological conditions. They awarded a whopping $11.5 million for this study!