Adjunctive Ganaxolone Treatment in Children and Young Adults
Research type
Research Study
Full title
A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Children and Young Adults with Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) Followed by Long-term Open-label Treatment
IRAS ID
248611
Contact name
Helen Cross
Contact email
Sponsor organisation
Marinus Pharmaceuticals, Inc.
Eudract number
2018-001180-23
Duration of Study in the UK
3 years, 4 months, 25 days
Research summary
This study is a double-blind, randomised, placebo-controlled trial of adjunctive ganaxolone treatment in children and young adults with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) followed by long-term open-label treatment
The purpose of this study is to find out if ganaxolone (GNX) reduces seizure activity and if GNX improves attention, sleep, behaviour, and seizure intensity when it is added to patient's current epilepsy treatment which may include medications, as compared to placebo. In addition to this study, GNX is being studied in research studies in adults with seizures, and in women with post-partum depression.
In several studies of seizure activity, the study drug Ganaxolone blocked seizures induced by electroshock in mice and rats.
As of 10 October 2017, approximately 1,587 subjects have received treatment with Ganaxolone in ongoing and completed company-sponsored clinical trials ranging in duration from 1 day to more than 2 years, using doses from 50 to 2,000 mg/day.This study has two phases: double-blind and open-label. The double-blind phase of the study will last 23 weeks. The next 17 weeks of this study are the double-blind treatment phase.
Approximately 70 patients are anticipated to enrol in this study at up to 30 study centres around the world.REC name
London - Harrow Research Ethics Committee
REC reference
18/LO/1450
Date of REC Opinion
6 Dec 2018
REC opinion
Further Information Favourable Opinion