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

Jazz Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Seizures; Tuberous Sclerosis Complex

Treatments: Drug: GWP42003-P; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02544763

2015-002154-12 (EudraCT Number)

GWEP1521 Blinded Phase

Details and patient eligibility

About

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.

Enrollment: 224 patients

Sex: All

Ages: 1 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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224 participants in 3 patient groups, including a placebo group

25 mg/kg/day GWP42003-P

Experimental group

Description:

100 mg/mL GWP42003-P oral solution taken twice daily (morning and evening).

Treatment:

Drug: GWP42003-P

50 mg/kg/day GWP42003-P

Experimental group

Description:

100 mg/mL GWP42003-P oral solution taken twice daily (morning and evening).

Treatment:

Drug: GWP42003-P

Placebo

Placebo Comparator group

Description:

Placebo oral solution matching 100 mg/mL GWP42003-P.

Treatment:

Drug: Placebo