A study of the safety, pharmacokinetics and efficacy of cannabidiol (GWP42003-P) in children and young adults with Dravet syndrome

• Conditions: Dravet Syndrome; MedDRA version: 18.1Level: PTClassification code 10073677Term: Severe myoclonic epilepsy of infancySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2014-000995-24-PL
• Lead Sponsor: GW Research Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-10-31
• Last Update Posted: 14 March 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 125

Inclusion Criteria

Inclusion Criteria: Part A
For inclusion in Part A of the study, patients must fulfil ALL of the following criteria:
• 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 4 and 10 years (inclusive).
• Patient must have a documented history of DS which is not completely controlled by current AEDs.
• Patient must be experiencing fewer than four convulsive seizures (i.e., tonic-clonic, tonic, clonic, atonic seizures) during the 28-day baseline period.
• Patient must be taking one or more AEDs at a dose which has 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.
Inclusion Criteria: Part B
For inclusion in Part B of the study, patients must fulfil ALL of the following criteria:
• 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 2 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 been stable for at least four weeks.
• All medications or interventions for epilepsy (including ketogenic diet and 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 120
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Exclusion Criteria: Parts A and B
The patient may not enter the study (Part A or Part B) if ANY of the following apply:
• 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 significantly abnormal, in the investigator’s opinion, laboratory values at screening or randomization.
• Patient has clinically relevant abnormalities in the 12-lead ECG measured at screening or randomization.
• Patient has any concurrent cardiovascular conditions, which will, in the investigator’s opinion, interfere with the ability to read 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 consumed grapefruit or grapefruit juice three days prior to screening and/or is unwilling to abstain from consuming these during the study.
• Patient has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP(s), 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 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 an investigational product 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 A1 or B1) or randomization (Visit A2 or B2) (Alanine aminotransferase [ALT] >5 × upper limit of normal [ULN] and total bilirubin [TBL] >2 × ULN) OR the ALT or Aspartate aminotransferase (AST) >3 × ULN and (TBL >2 × ULN or international normalized ratio

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified