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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02544763




Registration number
NCT02544763
Ethics application status
Date submitted
7/09/2015
Date registered
9/09/2015
Date last updated
6/10/2020

Titles & IDs
Public title
A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
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
Secondary ID [1] 0 0
2015-002154-12
Secondary ID [2] 0 0
GWEP1521 Blinded Phase
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Tuberous Sclerosis Complex 0 0
Seizures 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Neurological 0 0 0 0
Epilepsy

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - GWP42003-P
Treatment: Drugs - Placebo

Experimental: 25 mg/kg/day GWP42003-P - 100 mg/mL GWP42003-P oral solution taken twice daily (morning and evening).

Experimental: 50 mg/kg/day GWP42003-P - 100 mg/mL GWP42003-P oral solution taken twice daily (morning and evening).

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


Treatment: Drugs: GWP42003-P
Yellow oily solution containing cannabidiol dissolved in the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring.

Treatment: Drugs: Placebo
Yellow oily solution containing the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percent Change From Baseline in the Number of Tuberous Sclerosis Complex (TSC)-Associated Seizures During the Treatment Period (Maintenance and Titration) - TSC-associated seizures included: focal motor seizures without impairment of consciousness or awareness (Type 1 focal motor); focal seizures with impairment of consciousness or awareness (Type 2 focal); focal seizures evolving to bilateral generalized convulsive seizures (Type 3 focal); and tonic-clonic, tonic, clonic, or atonic seizures that are countable. Percent change from Baseline was calculated as the (post-Baseline value minus the Baseline value) divided by the Baseline value x 100.
Timepoint [1] 0 0
Baseline; up to Week 16
Secondary outcome [1] 0 0
Number of Participants Considered Treatment Responders During the Treatment Period (Maintenance and Titration) - Treatment responders are defined as those participants with a = 50% reduction in TSC-associated seizure frequency. TSC-associated seizures included: focal motor seizures without impairment of consciousness or awareness (Type 1 focal motor); focal seizures with impairment of consciousness or awareness (Type 2 focal); focal seizures evolving to bilateral generalized convulsive seizures (Type 3 focal); and tonic-clonic, tonic, clonic, or atonic seizures that are countable. Participants who withdrew from the trial during the treatment period are considered non-responders.
Timepoint [1] 0 0
Baseline; up to Week 16
Secondary outcome [2] 0 0
Change From Baseline in the Caregiver Global Impression of Change (CGIC) or Participant Global Impression of Change (PGIC) Score at the Participant's Last Visit - The combined caregiver and participant summary uses either the caregiver or participant version if only one version was completed, or the caregiver version if both caregiver and participant versions were completed. The CGIC comprised the following question, to be rated on a 7-point scale (1, Very Much Improved; 2, Much Improved; 3, Slightly Improved; 4, No Change; 5, Slightly Worse; 6, Much Worse; 7, Very Much Worse): "Since your child started treatment, please assess the status of your child's overall condition (comparing their condition now to their condition before treatment)." The SGIC comprised the following question, to be rated on a 7-point scale (1, Very Much Improved; 2, Much Improved; 3, Slightly Improved; 4, No Change; 5, Slightly Worse; 6, Much Worse; 7, Very Much Worse): "Since you started treatment, please assess the status of your overall condition (comparing your condition now to your condition before treatment)."
Timepoint [2] 0 0
Baseline; up to Week 16
Secondary outcome [3] 0 0
Percent Change From Baseline in Total Seizures During the Treatment Period (Maintenance and Titration) - Total seizures included all seizure types combined. Percent change from Baseline was calculated as the (post-Baseline value minus the Baseline value) divided by the Baseline value x 100.
Timepoint [3] 0 0
Baseline; up to Week 16
Secondary outcome [4] 0 0
Number of Participants With Any Severe Treatment-emergent Adverse Event (TEAE) - A TEAE was defined as an AE with a start date on or after the first dose of IMP during the Blinded Phase up to and including the date of first dose of the Open-label Extension (OLE) Phase (OLE Day 1).
Timepoint [4] 0 0
up to approximately Week 22

Eligibility
Key inclusion criteria
Key

- 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
Minimum age
1 Year
Maximum age
65 Years
Gender
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Austin Health - Heidelberg
Recruitment hospital [2] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [3] 0 0
The Royal Melbourne Hospital - Parkville
Recruitment hospital [4] 0 0
Sydney Children's Hospital - Randwick
Recruitment postcode(s) [1] 0 0
- Heidelberg
Recruitment postcode(s) [2] 0 0
- Herston
Recruitment postcode(s) [3] 0 0
- Parkville
Recruitment postcode(s) [4] 0 0
- Randwick
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arkansas
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Colorado
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Maryland
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Minnesota
Country [10] 0 0
United States of America
State/province [10] 0 0
Missouri
Country [11] 0 0
United States of America
State/province [11] 0 0
New York
Country [12] 0 0
United States of America
State/province [12] 0 0
North Carolina
Country [13] 0 0
United States of America
State/province [13] 0 0
Oregon
Country [14] 0 0
United States of America
State/province [14] 0 0
Pennsylvania
Country [15] 0 0
United States of America
State/province [15] 0 0
Tennessee
Country [16] 0 0
United States of America
State/province [16] 0 0
Texas
Country [17] 0 0
United States of America
State/province [17] 0 0
Utah
Country [18] 0 0
United States of America
State/province [18] 0 0
Virginia
Country [19] 0 0
United States of America
State/province [19] 0 0
Washington
Country [20] 0 0
Netherlands
State/province [20] 0 0
Rotterdam
Country [21] 0 0
Netherlands
State/province [21] 0 0
Utrecht
Country [22] 0 0
Poland
State/province [22] 0 0
Bydgoszcz
Country [23] 0 0
Poland
State/province [23] 0 0
Kraków
Country [24] 0 0
Poland
State/province [24] 0 0
Lublin
Country [25] 0 0
Poland
State/province [25] 0 0
Warsaw
Country [26] 0 0
Poland
State/province [26] 0 0
Wroclaw
Country [27] 0 0
Spain
State/province [27] 0 0
Barcelona
Country [28] 0 0
Spain
State/province [28] 0 0
Madrid
Country [29] 0 0
Spain
State/province [29] 0 0
Pamplona
Country [30] 0 0
United Kingdom
State/province [30] 0 0
Cardiff
Country [31] 0 0
United Kingdom
State/province [31] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
GW Research Ltd
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/show/NCT02544763
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications