CDKL5 deficiency disorder (CDD) is a rare, X-linked, developmental and epileptic encephalopathy characterised by severe global developmental impairment and seizures that can begin in the first few months after birth and are often treatment refractory. Ganaxolone, an investigational neuroactive steroid, reduced seizure frequency in an open-label, phase 2 trial that included patients with CDD. We aimed to further assess the efficacy and safety of ganaxolone in patients with CDD-associated refractory epilepsy.
Safety and efficacy of ganaxolone in patients with CDKL5 deficiency disorder: results from the double-blind phase of a randomised, placebo-controlled, phase 3 trial / Elia M Pestana Knight , Sam Amin , Nadia Bahi-Buisson , Tim A Benke , J Helen Cross , Scott T Demarest , Heather E Olson , Nicola Specchio , Thomas R Fleming , Alex A Aimetti , Maciej Gasior , Orrin Devinsky , Elena Belousova, Oleg Belyaev, Bruria Ben-Zeev, Andreas Brunklaus, Michael A Ciliberto, Francesca Darra, Ronald Davis, Valentina De Giorgis, Olga Doronina, Michael Fahey, Renzo Guerrini, Peter Heydemann, Olga Khaletskaya, Pawel Lisewski, Eric D Marsh, Ahsan N Moosa, Scott Perry, Sunny Philip, Rajsekar R Rajaraman, Ben Renfroe, Russell P Saneto, Ingrid E Scheffer, Yoshimi Sogawa, Bernhardt Suter, Matthew T Sweney, Daniel Tarquinio, Pierangelo Veggiotti, Geoff Wallace, Judy Weisenberg, Angus Wilfong, Elaine C Wirrell, Muhammad Zafar, Marta Zolnowska. - In: LANCET NEUROLOGY. - ISSN 1474-4465. - ELETTRONICO. - (2022), pp. 417-427. [10.1016/S1474-4422(22)00077-1]
Safety and efficacy of ganaxolone in patients with CDKL5 deficiency disorder: results from the double-blind phase of a randomised, placebo-controlled, phase 3 trial
Renzo Guerrini;
2022
Abstract
CDKL5 deficiency disorder (CDD) is a rare, X-linked, developmental and epileptic encephalopathy characterised by severe global developmental impairment and seizures that can begin in the first few months after birth and are often treatment refractory. Ganaxolone, an investigational neuroactive steroid, reduced seizure frequency in an open-label, phase 2 trial that included patients with CDD. We aimed to further assess the efficacy and safety of ganaxolone in patients with CDD-associated refractory epilepsy.
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