GWPCARE5 - An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet or Lennox-Gastaut Syndromes

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02224573
• Date of registration: 21/08/2014
• Prospective Registration: Yes
• Primary sponsor: GW Research Ltd
• Public title: GWPCARE5 - An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet or Lennox-Gastaut Syndromes
• Scientific title: An Open Label Extension Study to Investigate the Safety of Cannabidiol (GWP42003-P; CBD) in Children and Young Adults With Inadequately Controlled Dravet or Lennox-Gastaut Syndromes.
• Date of first enrolment: June 2015
• Target sample size: 681
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02224573
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Key inclusion & exclusion criteria

Key Inclusion Criteria:

• Participant has completed the treatment phase of their Core Study.

Key Exclusion Criteria:

- Participant is currently using or has in the past used recreational or medicinal
cannabis, or synthetic cannabinoid-based medications (including Sativex®) within the 3
months prior to study entry other than the investigational medicinal product (IMP)
received during the Core Study and are unwilling to abstain for the duration for the
study..

- Any history of suicidal behavior or any suicidal ideation of type 4 or 5 on the
Columbia-Suicide Severity Rating Scale (C-SSRS) at Visit 1.

- Participant has been part of a clinical trial involving an IMP during the inter-study
period.

- Female participant is of child bearing potential or male participant's partner is of
child bearing potential, unless willing to ensure that they or their partner use
highly effective contraception, for example, hormonal contraceptives, intrauterine
devices/hormone-releasing systems, bilateral tubal occlusion, vasectomized partner or
sexual abstinence, during the study and for 3 months thereafter (however, a male
condom should not be used in conjunction with a female condom).

- Participant has significantly impaired hepatic function at the 'End of Treatment'
visit of their Core Study or at Visit 1 if re-assessed: i) Alanine aminotransferase
(ALT) or aspartate aminotransferase (AST) >5 × upper limit of normal (ULN); ii) ALT or
AST >3 × ULN and (total bilirubin [TBL] >2 × ULN or international normalized ratio
[INR] >1.5); iii) ALT or AST >3 × ULN with the presence of fatigue, nausea, vomiting,
right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia (>5%). This
criterion must be confirmed prior to entering the study.



Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Lennox-Gastaut Syndrome

Dravet Syndrome

Epilepsy

Intervention(s)

Drug: GWP42003-P

Primary Outcome(s)

Number of participants with adverse events and other assessments as a measure of participant safety [Time Frame: Participants will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular participant, or for a maximum of 5 years.]

Secondary Outcome(s)

Mean percentage change in total convulsive seizure frequency, relative to the pre-randomization baseline of the Core Study [Time Frame: Participants will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular participant, or for a maximum of 5 years.]

Mean change in quality of life, relative to the pre-randomization baseline of the Core Study, if assessed during the Core Study [Time Frame: Participants will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular participant, or for a maximum of 5 years.]

Mean percentage change in total non-convulsive seizure frequency, relative to the pre-randomization baseline of the Core Study [Time Frame: Participants will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular participant, or for a maximum of 5 years.]

Mean change in the Caregiver Global Impression of Change (CGIC) or Subject Global Impression of Change (SGIC) score, relative to the pre-randomization baseline of the Core Study, if assessed during the Core Study [Time Frame: Participants will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular participant, or for a maximum of 5 years.]

Mean percentage change in the number of drop seizures, relative to the pre-randomization baseline of the Core Study [Time Frame: Participants will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular participant, or for a maximum of 5 years.]

Mean percentage change in the number of non-drop seizures, relative to the pre-randomization baseline of the Core Study [Time Frame: Participants will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular participant, or for a maximum of 5 years.]

Number of participants experiencing a >25% worsening, -25 to +25% no change, 25-50% improvement, 50-75% improvement, or >75% improvement in convulsive seizures, relative to the pre-randomization baseline of the Core Study [Time Frame: Participants will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular participant, or for a maximum of 5 years.]

Mean percentage change in the frequencies of sub-types of seizures, relative to the pre-randomization baseline of the Core Study [Time Frame: Participants will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular participant, or for a maximum of 5 years.]

Number of participants with LGS considered treatment responders, defined as those with a =25%, =50%, =75%, or 100% reduction in drop seizures, relative to the pre-randomization baseline of the Core Study [Time Frame: Participants will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular participant, or for a maximum of 5 years.]

Number of participants considered treatment responders, defined as those with a =25%, =50%, =75%, or 100% reduction in convulsive seizures, relative to the pre-randomization baseline of the Core Study [Time Frame: Participants will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular participant, or for a maximum of 5 years.]

Number of participants experiencing a >25% worsening, -25 to +25% no change, 25-50% improvement, 50-75% improvement, or >75% improvement in drop seizures, relative to the pre-randomization baseline of the Core Study [Time Frame: Participants will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular participant, or for a maximum of 5 years.]

Secondary ID(s)

GWEP1415

2014-001834-27

Source(s) of Monetary Support

Please refer to primary and secondary sponsors