Ganaxolone treatment in female children with protocadherin 19 (PCDH19)
Research type
Research Study
Full title
A double-blind, randomized, placebo-controlled trial of adjunctive ganaxolone treatment in female children with protocadherin 19 (PCDH19)-related epilepsy followed by long-term open-label treatment
IRAS ID
264810
Contact name
Judith Helen Cross
Contact email
Sponsor organisation
Marinus Pharmaceuticals, Inc.
Eudract number
2018-004496-12
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 8 months, 27 days
Research summary
Ganaxolone (GNX) is an investigational drug proposed for the treatment of uncontrolled seizures due to a confirmed PCDH19 gene mutation. This is a placebo-controlled trial which means some participants will receive the study drug, and some will receive a placebo, which contains no active medicine but will look the same as GNX. This study is double-blind meaning neither the participants nor the investigators will know who is receiving GNX or not. This study is randomised meaning participants will be chosen by chance (like flipping a coin) whether they receive GNX or placebo.
The purpose of this study is to find out:
• if the study drug GNX reduces seizure frequency
• if the study drug GNX improves the participant’s behaviour and sleep when it is added to their current epilepsy treatment.
The Sponsor is gathering information on whether the treatment is safe and effective.
All participants in this study will receive the study drug at some point in the study in addition to their current medication that they take for their condition. Patients do not need to be taking an anti-epileptic drug to be eligible for participation in this study.
There are three phases of this study. If there is no previous recorded history of seizure frequency, participants and their families will be asked to record this for 12 weeks. Following this, the randomised treatment phase lasts for 17 weeks, with 4 clinic visits and 7 phone calls. Following this phase, an open label phase begins and GNX will be given to all participants. This will remain available to participants until the medication is available in the UK by prescription, or the sponsor discontinues development of GNX for PCDH19-related epilepsy, whichever comes first.
Approximately 70 participants are anticipated to enrol in this study at approximately 45 study centres worldwide.REC name
London - Central Research Ethics Committee
REC reference
19/LO/1490
Date of REC Opinion
14 Jan 2020
REC opinion
Further Information Favourable Opinion