Dijck, Anke Van

Ganaxolone Fragile X Clinical Trial Showed Disappointing Results

Hannah Maria Miles Dijck, Anke Van • Kooy, Frank • Research Updates • University of Antwerp, Belgium February 7, 2019February 12, 2019GABA, ganaxolone, mGluR, NIH, seizure, sleep

Ganaxolone, an experimental drug from Marinus Pharmaceuticals which targets GABA receptors, did not show promise for Fragile X syndrome in a clinical trial.

Clinical Trial of Ganaxolone in Patients with Fragile X Syndrome

FRAXA Research Foundation 2014 Grants • 2015 Grants • Clinical • Dijck, Anke Van • Has Results • Kooy, Frank • Research Grant Results • Treatment Targets • Trials and Studies • University of Antwerp, Belgium September 2, 2017March 9, 2020drug tolerance, GABA, GABA-A, ganaxolone, glutamate, mGluR, seizure, sleep

With a $90,000 grant from FRAXA Research Foundation funded during 2014-2015, Dr. Frank Kooy and colleagues at the University of Antwerp are conducting a double blind crossover trial of ganaxolone in patients with Fragile X syndrome. Results of this study were mixed (see Marinus: Results from Phase 2 Exploratory Clinical Study Support Continued Development of Ganaxolone in Fragile X Syndrome.)