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

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 Seizures

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.

Key Inclusion Criteria:

• Participant has a well-documented clinical history of epilepsy.
• Participant has a clinical diagnosis of Tuberous Sclerosis Complex (TSC) according to the criteria agreed by the 2012 International TSC Consensus Conference.
• All medications or interventions for epilepsy (including ketogenic diet and any neurostimulation devices for epilepsy) must have been stable for 1 month prior to screening and the participant is willing to maintain a stable regimen throughout the trial.

Key Exclusion Criteria:

• Participant has a history of pseudo-seizures.
• Participant has clinically significant unstable medical conditions other than epilepsy.
• Participant has an illness in the 4 weeks prior to screening or randomization, other than epilepsy, which in the opinion of the investigator could affect seizure frequency.
• Participant has undergone general anesthetic in the 4 weeks prior to screening or randomization.
• Participant has undergone surgery for epilepsy in the 6 months prior to screening.
• Participant is being considered for epilepsy surgery or any procedure involving general anesthesia.
• Participant has been taking felbamate for less than 1 year prior to screening.
• Participant is taking an oral mTOR inhibitor.
• Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the Investigational Medicinal Product (IMP), such as sesame oil.
• Participant has any history of suicidal behavior or any suicidal ideation of type 4 or 5 on the C-SSRS in the last month or at screening.
• Participant is currently using or has in the past used recreational or medicinal cannabis, or cannabinoid-based medications, within the 3 months prior to screening and is unwilling to abstain for the duration for the study.
• Participant has tumor growth which, in the opinion of the Investigator, could affect the primary endpoint.
• Participant has significantly impaired hepatic function at the screening or randomization visit
• Participant has received an IMP within the 12 weeks prior to the screening visit.