GWEP1521 - Study of GWP42003-P in Tuberous Sclerosis Complex
Research type
Research Study
Full title
A double-blind, randomized, placebo-controlled study to investigate the efficacy and safety of cannabidiol (GWP42003-P,CBD) as add-on therapy in patients with tuberous sclerosis complex who experience inadequately-controlled focal seizures
IRAS ID
187560
Contact name
Finbar O'Callaghan
Contact email
Finbar.O'Callaghan@gosh.nhs.uk
Sponsor organisation
GW Research Ltd
Eudract number
2015-002154-12
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 5 months, 28 days
Research summary
The study duration has since been updated to 1 year, 26 weeks and 3 days.\n\nThis clinical study is being conducted by GW Research Ltd to determine the safety and efficacy of the drug GWP42003-P in children and adults with tuberous sclerosis complex (TSC) who experience inadequately-controlled focal seizures. It is a Phase III, double-blind, two-part, multicentre study. In the blinded phase participants will receive either GWP42003-P or placebo. However, they will not know which treatment they have been assigned. In the open-label extension phase all participants will receive GWP42003-P.\nParticipants will be aged between 2 and 65 years old and will be randomly assigned one of two doses of GWP42003-P or placebo. The ratio of GWP42003-P high dose to GWP42003-P low dose to placebo will be 1:1:1, with participants having a 66% chance of receiving GWP42003-P and a 33% chance of receiving placebo.\nThe blinded phase will last up to 25 weeks, including a dosing period lasting 15 weeks. Participants will attend 8 or 9 clinic visits and 2 additional visits over the phone. Safety telephone calls will be made to the participants every 2 days during titration and 1 week after the end of titration.\nThe open-label extension phase will continue until GWP42003-P is granted market authorization in TSC. In the first year visits will occur at 2, 4, 12, 26, 39 and 52 weeks after the enrolment visit. After the first year, assessment visits to the clinic will be every 26 weeks with safety phone calls made every 4 weeks and visits in between to dispense study drug.\nAssessments include vital signs, physical examination, blood/urine samples, electrocardiogram and monitoring of adverse events. The participant’s caregiver will complete questionnaires on quality of life and impression of change. The participant will complete a battery of cognitive assessments and be asked about their mental health. A diary will be completed by the participant or caregiver throughout the study.
REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
16/NE/0312
Date of REC Opinion
2 Dec 2016
REC opinion
Further Information Favourable Opinion