A study of cannabidiol (GWP42003-P, CBD) in patients with tuberous sclerosis complex who experience seizures.

• Register: EUCTR
• Last refreshed on: 24 January 2022
• Main ID: EUCTR2015-002154-12-PL
• Date of registration: 18/11/2016
• Prospective Registration: Yes
• Primary sponsor: GW Research Ltd.
• Public title: A study of cannabidiol (GWP42003-P, CBD) in patients with tuberous sclerosis complex who experience seizures.
• Scientific title: 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
• Target sample size: 210
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-002154-12
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 4
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Netherlands, Poland, Spain, United Kingdom, United States

Contacts

• Name: GW Research Ltd Switchboard
• Affiliation: GW Research Ltd

• Name: GW Research Ltd Switchboard
• Affiliation: GW Research Ltd

Key inclusion & exclusion 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 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).

Are the trial subjects under 18? yes
Number of subjects for this age range: 94
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 116
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
• Patient has a history of pseudo-seizures.
•Patient has undergone general aesthetic 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 anaesthesia 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 as part of a clinical trial less than 12 weeks prior to the screening visit.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Tuberous Sclerosis Complex (TSC)
MedDRA version: 21.0 Level: LLT Classification code 10045138 Term: Tuberous sclerosis System Organ Class: 100000004850

Intervention(s)

Trade Name: CBD - Oral Solution, is known as Epidyolex 100 mg/ml oral solution,
and is the approved name in the EU.
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: CANNABIDIOL
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Oral use

Primary Outcome(s)

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.

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-P.

Primary end point(s): Blinded Phase:
The primary endpoint is the change in number of TSC-associated seizures during the treatment period (maintenance and titration) compared to baseline 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.

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 Outcome(s)

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):
• Number of patients considered treatment responders defined as those with a = 50% reduction in TSC-associated seizure frequency.
• Change in Caregiver Global Impression of Change (CGIC) or Subject Global Impression of Change (SGIC) score.
• Change in total seizures.
Other:
Antiepileptic efficacy measures
Growth and development (in patients under 18)
Quality of life
Safety and Tolerability

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.

Secondary ID(s)

GWEP1521

2015-002154-12-ES

NCT02544763

Source(s) of Monetary Support

GW Research Ltd

Results

• Results available: Yes
• Date Posted: 26/12/2021
• Date Completed: 11/06/2021
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-002154-12/results