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

Phase 3

Completed

Conditions: Bourneville's disease
Tuberous Sclerosis Complex
10039911

Registration Number: NL-OMON47292

Lead Sponsor: GW Research Ltd

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 25

Inclusion Criteria

The most important inclusion criteria are:;• Patient is male or female aged between one and 65 years inclusive.
• Patient and/or parent(s)/legal representative is willing and able to give informed consent/assent for participation in the study.
• Well-documented clinical history of epilepsy which is not completely controlled by their current AEDs.
• Clinical diagnosis of TSC according to criteria agreed by the 2012 International Tuberous Sclerosis Complex Consensus Conference.
• Taking one or more AEDs at a dose which has been stable for at least four weeks prior to screening.
• All medications or interventions for epilepsy (including ketogenic diet and any neurostimulation devices for epilepsy) must have been stable for one month prior to screening and the patient is willing to maintain a stable regimen throughout the study.;At the end of the baseline period patients must also meet the following criterion:
• Completed at least 90% of calls to IVRS during the first 28 days of the baseline period (a minimum of 25 completed calls).

Exclusion Criteria

The most important exclusion criteria are:;• Patient has a history of pseudo-seizures.
• Patient has undergone general anesthetic in the four weeks prior to screening or randomization.
• Patient has undergone surgery for epilepsy in the six months prior to screening.
• Patient is being considered for epilepsy surgery or any procedure involving general anesthesia during the blinded phase of the study.
• Patient has been taking felbamate for less than one year prior to screening.
• Patient is taking an oral (mTOR) inhibitor.
• Patient is currently using or has in the past used recreational or medicinal cannabis, or cannabinoid-based medications, within the three months prior to screening and is unwilling to abstain for the duration for the study.
• Patient has tumor growth which, in the opinion of the Investigator, could affect the primary endpoint.
• Patient is female and of child bearing potential, or is male whose partner is of child bearing potential, unless willing to ensure that they or their partner use a highly effective method of birth control (e.g., hormonal contraceptives, intrauterine devices/hormone-releasing systems, bilateral tubal occlusion, vasectomized partner, sexual abstinence) during the study and for three months thereafter.
• Female patient who is pregnant (positive pregnancy test), lactating or planning pregnancy during the course of the study and for three months thereafter.
• Patient has received an IMP less than 12 weeks prior to the screening visit.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Blinded Phase:<br /><br>The primary endpoint is the change in number of TSC-associated seizures* during<br /><br>the treatment period (maintenance and titration) in patients taking GWP42003-P<br /><br>compared with placebo.<br /><br><br /><br>Open-label Extension:<br /><br>The safety of GWP42003-P will be evaluated by assessing the incidence, type and<br /><br>severity of AEs.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Blinded Phase<br /><br>The following endpoints will be compared between treatment groups over the<br /><br>16-week, double-blind treatment period (all changes relative to baseline):<br /><br>Key:<br /><br>• Number of patients considered treatment responders defined as those with a >=<br /><br>50% reduction in TSC-associated seizure* frequency.<br /><br>• Change in Caregiver Global Impression of Change (CGIC) or Subject Global<br /><br>Impression of Change (SGIC) score.<br /><br>• Change in total seizures.<br /><br>Other:<br /><br>• Antiepileptic Efficacy Measures<br /><br>• Quality of life<br /><br>• Safety and Tolerability<br /><br><br /><br>Open-label Extension:<br /><br>Will include secondary endpoints as in the core study, with the addition of<br /><br>percentage change in the TSC associated seizures and with removal of PK<br /><br>analysis.</p><br>