Tuberous Sclerosis Complex (TSC) MedDRA version: 19.0 Level: PT Classification code 10045138 Term: Tuberous sclerosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Cannabidiol (CBD)
Product Code: GWP42003-P
Pharmaceutical Form: Oral solution
INN or Proposed INN: N/A
CAS Number: 13956-29-1
Current Sponsor code: GWP42003-P
Other descriptive name: CAN
Sponsors
GW Research Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • 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 history of epilepsy, with compatible electroencephalogram (EEG) and clinical history. • Clinical diagnosis of TSC according to criteria agreed by the 2012 International Tuberous Sclerosis Complex Consensus Conference. • 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). Are the trial subjects under 18? yes Number of subjects for this age range: 84 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 108 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • 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 is taking felbamate, and they have been taking it for less than one year prior to screening. • Patient is taking an oral (mTOR) inhibitor. • Active suicidal plan/intent in the past six months, or a history of suicide attempt in the last two years, or more than one lifetime suicide attempt. • 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 within the 12 weeks prior to the screening visit.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Blinded Phase: To evaluate the efficacy of GWP42003-P as add-on therapy in reducing the frequency of seizures when compared with placebo in patients with TSC. Open-label Extension: To evaluate via the adverse events (AE) profile the long term safety and tolerability of GWP42003-P as add-on therapy in children and adults with TSC who experience inadequately-controlled seizures.;Secondary Objective: Blinded Phase: • To evaluate the effect of GWP42003-P compared with placebo on antiepileptic measures. • To evaluate the safety and tolerability of GWP42003-P compared with placebo. • To determine the pharmacokinetics (PK) following single and multiple doses of GWP42003-P. Open-label Extension: • To evaluate the long term effects of GWP42003-P, as add-on therapy, on antiepileptic measures. • To evaluate the long term safety and tolerability of GWP42003.;Primary end point(s): Blinded Phase: The primary endpoint is the percentage change from baseline in number of TSC-associated seizures (average per 28 days) during the treatment period (maintenance and titration) in patients taking GWP42003-P compared with placebo. Open-label Extension: The safety of GWP42003-P will be evaluated by assessing the incidence, type and severity of AEs.;Timepoint(s) of evaluation of this end point: Baseline (Visit 2 to Visit 3) and the last 28 days of the evaluable period. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The following endpoints will be compared between treatment groups over the 16-week, double-blind treatment period (all changes relative to baseline): -Change in number of total seizures (percentage change from baseline and responder analyses). -The plasma concentration/time curve of CBD and its major metabolites Safety and Tolerability: -Clinical laboratory parameters. -ECG. -Physical examination parameters (including height and weight). -Vital signs. -C-SSRS (19+ years) or C-SSRS Children’s (6–18 years) score, where applicable. -Number of inpatient hospitalizations due to epilepsy. -Abuse liability. -Effects on menstruation cycles (in females). Open-label Extension Will include secondary endpoints as in the core study, with the addition of percentage change in the TSC associated seizures and with removal of PK analysis.;Timepoint(s) of evaluation of this end point: Blinded phase (seizures) - Baseline (Visit 2 to Visit 3) and the last 28 days of the evaluable period. Blinded phase (other efficacy) - Visits 3, 4, 5, 6, 7, 8 , 9 and 10. Blinded phase (safety) - Visits 3, 4, 5, 6, 7, 8, 9 and 10. OLE - ALL visit beginning at B1. | — |
Countries
Australia, France, Netherlands, Poland, Spain, United Kingdom, United States
Contacts
Public ContactGW Research Ltd Switchboard
GW Research Ltd
Outcome results
None listed