Authors :
Presenting Author: Elia Pestana-Knight, MD – Cleveland Clinic
Sam Amin, MD – University Hospitals Bristol and Weston; Nadia Bahi-Buisson, MD, PhD – Necker Enfants Malades University Hospital; Orrin Devinsky, MD – New York University Langone Comprehensive Epilepsy Center; Eric Marsh, MD, PhD – University of Pennsylvania Perelman School of Medicine and Division of Neurology; Rajsekar Rajaraman, MD – UCLA Mattel Children's Hospital; Alex Aimetti, PhD – Marinus Pharmaceuticals, Inc.; Eva Rybak, PharmD – Marinus Pharmaceuticals, Inc.; Fanhui Kong, PhD – Marinus Pharmaceuticals, Inc.; Ian Miller, MD – Marinus Pharmaceuticals, Inc.; Joseph Hulihan, MD – Marinus Pharmaceuticals, Inc.; Scott Demarest, MD, MSCS – University of Colorado School of Medicine
Rationale:
In the 17-week double-blind phase 3 Marigold Study (
NCT03572933), 101 patients aged 2–19 years with CDD were treated with adjunctive ganaxolone (GNX) oral suspension which significantly reduced median 28-day major motor seizure frequency (MMSF) versus placebo. In the open-label extension (OLE) phase of the study, an additional 24 months of treatment with GNX further reduced MMSF versus baseline in patients who continued treatment. While the primary outcome of the OLE was to evaluate safety, here we also describe secondary endpoints such as responder analyses, reduction in the number of days with seizures, Caregiver Global Impression of Change–Improvement (GCI-I), and CGI–Seizure Intensity and Duration (CGI-CSID) score through 24 months.
Methods:
Patients who continued into the OLE underwent a four week blinded crossover from their maintenance dose of double-blind study medication to open-label GNX. The target dose of open-label GNX was 21 mg/kg TID (for patients
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