A randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of cannabidiol (GWP42003-P) in children and young adults with Dravet syndrome.
- Conditions
- Dravet syndrome10039911
- Registration Number
- NL-OMON44663
- Lead Sponsor
- GW Research Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 30
• Patient and/or parent(s)/legal representative must be willing and able
to give informed assent/consent for participation in the study.;• Patient and their caregiver must be willing and able (in the
investigator's opinion) to comply with all study requirements.;• Patient must be male or female aged between two and 18 years
(inclusive).;• Patient must have a documented history of DS which is not completely
controlled by current AEDs.;• Patient must be experiencing four or more convulsive seizures (i.e.,
tonic-clonic, tonic, clonic, atonic seizures) during the first 28 days of the
baseline period.;• Patient must be taking one or more AEDs at a dose which has/have
been stable for at least four weeks.;• All medications or interventions for epilepsy (including ketogenic diet
and vagus nerve stimulation [VNS]) must have been stable for four
weeks prior to screening and patient and caregiver are willing to
maintain a stable regimen throughout the study. The ketogenic diet and
VNS treatments are not counted as an AED.;• Patient and/or parent(s)/legal representative is willing to allow his or
her primary care practitioner and consultant to be notified of
participation in the study.;• Patient has completed their Interactive Voice Response System (IVRS)
telephone diary on at least 25 days of the baseline period; patients who
are non-compliant will be deemed ineligible to continue.
• Patient has clinically significant unstable medical conditions other than
epilepsy.;• Patient has had clinically relevant symptoms or a clinically significant
illness in the four weeks prior to screening or randomization, other than
epilepsy.;• Patient has clinically significant abnormal laboratory values, in the
investigator's opinion, at screening or randomization.;• Patient has clinically relevant abnormalities in the ECG measured at
screening or randomization.;• Patient has any concurrent cardiovascular conditions which will, in the
investigator's opinion, interfere with the ability to assess their ECGs.;• Patient has a history or presence of alcohol or substance abuse within
the last two years prior to the study or daily consumption of five or more
alcohol-containing beverages.;• Patient is currently using, or has in the past used, recreational or
medicinal cannabis, or synthetic cannabinoid-based medications
(including Sativex®) within the three months prior to study entry.;• Patient is unwilling to abstain from using recreational or medicinal
cannabis, or synthetic cannabinoid based medications (including
Sativex®) during the study.;• Patient has a history of symptoms (e.g., dizziness, light-headedness,
blurred vision, palpitations, weakness, syncope) related to a drop in
blood pressure due to postural changes.;• Patient has ingested alcohol in the 24-hour period prior to the first
study visit and/or is unwilling to abstain from drinking alcohol
throughout the treatment period.;• Patient has any known or suspected hypersensitivity to cannabinoids
or any of the excipients of the IMPs (e.g., sesame oil).;• Female patient is of child bearing potential or male patient's partner is
of child bearing potential; unless willing to ensure that they or their
partner use highly effective contraception for the duration of the study
and for three months thereafter. Highly effective methods of
contraception are defined as those, alone or in combination, that result
in a low failure rate (i.e., less than 1% per year) when used consistently
and correctly. Such methods include hormonal contraceptives,
intrauterine devices/hormone-releasing systems, bilateral tubal
occlusion, vasectomized partner or sexual abstinence.;• Female patient who is pregnant (positive pregnancy test), lactating or
planning pregnancy during the course of the study and for three months
thereafter.;• Patient has been part of a clinical trial involving another IMP in the
previous six months.;• Patient is taking felbamate and they have been taking it for less than
one year prior to screening.;• Any other significant disease or disorder which, in the opinion of the
investigator, may either put the patient at risk because of participation
in the study, may influence the result of the study, or affect the patient's
ability to participate in the study.;• Patient has significantly impaired hepatic function at screening (Visit
1) or randomization (Visit 2), defined as any of the
following:
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST)
>5 x upper limit of normal (ULN).
- ALT or AST >3 x ULN and (total bilirubin [TBL] >2 x ULN or
international normalized ratio [INR] >1.5).
- ALT or AST >3 × ULN with the presence of fatigue, nausea, vomiting,
right upper quadrant pain or tenderness, fever, rash, and/or
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary endpoint is the mean percentage change from baseline in total<br /><br>convulsive seizure frequency during the treatment period (Day 1 to the end of<br /><br>the evaluable period) in patients taking GWP42003-P compared with placebo.</p><br>
- Secondary Outcome Measures
Name Time Method