A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

Summary

This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The blinded phase only will be described in this record. Participants will receive 1 of 2 doses of GWP42003-P or matching placebo. The primary clinical hypothesis is that there will be a difference between GWP42003-P and placebo in their effect on seizure frequency.

Timeline

Start

2016-04-06

Primary completion

2019-01-22

Completion

2019-02-26

Outcome

Met primary endpoint

registry GWP42003-P 25 mg/kg/Day vs Placebo; p =0.0009; Treatment ratio 0.699 (95% CI 0.567 to 0.861); Mixed Models Analysis NCT02544763 ↗

registry GWP42003-P 50 mg/kg/Day vs Placebo; p =0.0018; Treatment ratio 0.715 (95% CI 0.580 to 0.881); Mixed Models Analysis NCT02544763 ↗

Publications

• Burke C, Crossan C, Tyas E, Hemstock M, Lee D, Bowditch S. A Cost-Utility Analysis of Add-On Cannabidiol Versus Usual Care Alone for the Treatment of Seizures Associated with Tuberous Sclerosis Complex in England and Wales. Pharmacoecon Open. 2024 Jul;8(4):611-626. doi: 10.1007/s41669-024-00474-x. Epub 2024 Mar 5.
• Wu JY, Cock HR, Devinsky O, Joshi C, Miller I, Roberts CM, Sanchez-Carpintero R, Checketts D, Sahebkar F. Time to onset of cannabidiol treatment effect and resolution of adverse events in tuberous sclerosis complex: Post hoc analysis of randomized controlled phase 3 trial GWPCARE6. Epilepsia. 2022 May;63(5):1189-1199. doi: 10.1111/epi.17199. Epub 2022 Mar 4.
• Thiele EA, Bebin EM, Filloux F, Kwan P, Loftus R, Sahebkar F, Sparagana S, Wheless J. Long-term cannabidiol treatment for seizures in patients with tuberous sclerosis complex: An open-label extension trial. Epilepsia. 2022 Feb;63(2):426-439. doi: 10.1111/epi.17150. Epub 2021 Dec 27.
• Thiele EA, Bebin EM, Bhathal H, Jansen FE, Kotulska K, Lawson JA, O'Callaghan FJ, Wong M, Sahebkar F, Checketts D, Knappertz V; GWPCARE6 Study Group. Add-on Cannabidiol Treatment for Drug-Resistant Seizures in Tuberous Sclerosis Complex: A Placebo-Controlled Randomized Clinical Trial. JAMA Neurol. 2021 Mar 1;78(3):285-292. doi: 10.1001/jamaneurol.2020.4607.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Cannabidiol	Other / unclassified	25 mg/kg	Oral
Subject	Cannabidiol	Other / unclassified	50 mg/kg	Oral

Indications