The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02224573




Registration number
NCT02224573
Ethics application status
Date submitted
21/08/2014
Date registered
25/08/2014
Date last updated
3/02/2022

Titles & IDs
Public title
An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and 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 Adults With Inadequately Controlled Dravet or Lennox-Gastaut Syndromes.
Secondary ID [1] 0 0
2014-001834-27
Secondary ID [2] 0 0
GWEP1415
Universal Trial Number (UTN)
Trial acronym
GWPCARE5
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Condition category
Condition code
Neurological 0 0 0 0
Epilepsy
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

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

Experimental: GWP42003-P -


Treatment: Drugs: GWP42003-P


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

Outcomes
Primary outcome [1] 0 0
Number of Participants With Any Treatment-emergent Adverse Event (TEAE) Occurring in =5% of Participants in Any Treatment Group
Timepoint [1] 0 0
up to Week 260
Primary outcome [2] 0 0
Number of Participants With Clinically Significant Changes in the Indicated Vital Sign Values From the Pre-randomization Baseline of the Core Study at Any Time Post-dose
Timepoint [2] 0 0
up to Week 260
Primary outcome [3] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Body Mass Index (BMI)
Timepoint [3] 0 0
up to Week 260
Primary outcome [4] 0 0
Number of Participants With Clinically Significant Electrocardiogram (ECG) Values at the Pre-randomization Baseline of the Core Study and at Any Time Post-dose
Timepoint [4] 0 0
up to Week 260
Primary outcome [5] 0 0
Number of Participants With the Indicated Responses to Questions Regarding Suicidal Ideation and Behavior Using the Children's Columbia-Suicide Severity Rating Scale (C-SSRS) at Day 1 and at Any Time Post-dose
Timepoint [5] 0 0
up to Week 260
Primary outcome [6] 0 0
Mean Cannabis Withdrawal Scale Score at End of Taper (EOT), the Post-Taper Safety Call, and at Safety Follow-up
Timepoint [6] 0 0
up to Week 260
Primary outcome [7] 0 0
Mean Pediatric Cannabinoid Withdrawal Scale (PCWS) Score at the End of Treatment, the End of Taper, the Post-Taper Safety Call, and at Safety Follow-up
Timepoint [7] 0 0
up to Week 260
Primary outcome [8] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Red Blood Cells (RBCs) Values
Timepoint [8] 0 0
up to Week 260
Primary outcome [9] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Hemoglobin Levels
Timepoint [9] 0 0
up to Week 260
Primary outcome [10] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Hematocrit Values
Timepoint [10] 0 0
up to Week 260
Primary outcome [11] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Erythrocyte Mean Corpuscular Volume
Timepoint [11] 0 0
up to Week 260
Primary outcome [12] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Erythrocyte Mean Corpuscular Hemoglobin
Timepoint [12] 0 0
up to Week 260
Primary outcome [13] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes, and Neutrophils
Timepoint [13] 0 0
up to Week 260
Primary outcome [14] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in the Percentage of Basophils, Eosinophils, Lymphocytes, Monocytes, and Neutrophils in White Blood Cells (WBCs)
Timepoint [14] 0 0
up to Week 260
Primary outcome [15] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Sodium, Potassium, Blood Urea Nitrogen, Glucose, Calcium, and High-Density Lipoprotein (HDL) Cholesterol
Timepoint [15] 0 0
up to week 260
Primary outcome [16] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Creatinine (Jaffe), Creatinine (Enzymatic), and Bilirubin
Timepoint [16] 0 0
up to Week 260
Primary outcome [17] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Creatinine Clearance (Schwartz) and Creatinine Clearance (Cockcroft-Gault)
Timepoint [17] 0 0
up to Week 260
Primary outcome [18] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Alkaline Phosphatase, Aspartate Aminotransferase, Alanine Aminotransferase, and Gamma Glutamyl Transferase
Timepoint [18] 0 0
up to Week 260
Primary outcome [19] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Albumin and Protein
Timepoint [19] 0 0
up to Week 260
Primary outcome [20] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Prolactin
Timepoint [20] 0 0
up to Week 260
Primary outcome [21] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Prothrombin Time
Timepoint [21] 0 0
up to Week 260
Primary outcome [22] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Prothrombin International Normalized Ratio
Timepoint [22] 0 0
up to Week 260
Primary outcome [23] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Insulin-like Growth Factor-1 (IGF-1) Levels
Timepoint [23] 0 0
up to Week 260
Primary outcome [24] 0 0
Mean Subject/Caregiver Global Impression of Change (S/CGIC) Score
Timepoint [24] 0 0
Baseline; up to Week 260
Primary outcome [25] 0 0
Percent Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Total Seizure Frequency
Timepoint [25] 0 0
Baseline; Week 49 to 60, Week 97 to 108, Week 145 to 156, Week 205 to 216, Week 253 to 264, and Last 12 Weeks
Primary outcome [26] 0 0
Percent Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Drop Seizure Frequency in Participants With Lennox-Gastaut Syndrome
Timepoint [26] 0 0
Baseline; Week 49 to 60, Week 97 to 108, Week 145 to 156, Week 205 to 216, and Last 12 Weeks
Primary outcome [27] 0 0
Percent Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Convulsive Seizure Frequency in Participants With Dravet Syndrome
Timepoint [27] 0 0
Baseline; Week 49 to 60, Week 97 to 108, Week 145 to 156, Week 205 to 216, Week 253 to 264, and Last 12 Weeks
Primary outcome [28] 0 0
Number of Participants With Convulsive and Non-convulsive Seizures Greater Than 30 Minutes in Duration (Status Epilepticus)
Timepoint [28] 0 0
Baseline; At last 12 weeks
Secondary outcome [1] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Quality of Life in Childhood Epilepsy (QOLCE) Scores
Timepoint [1] 0 0
Baseline; up to Week 260
Secondary outcome [2] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment QOL in Epilepsy Scores (QOLIE-31-P) in Participants With Lennox-Gastaut Syndrome
Timepoint [2] 0 0
Baseline; up to Week 260
Secondary outcome [3] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Vineland Adaptive Behavior Scale, Second Edition (Vineland-II) Scores
Timepoint [3] 0 0
Baseline; up to Week 260
Secondary outcome [4] 0 0
Number of Participants With Inpatient Hospitalizations Due to Epilepsy Since the Previous Visit
Timepoint [4] 0 0
Week 48, 104, 156, 208, and End of Treatment (up to Week 260 based on when the participant discontinued treatment)
Secondary outcome [5] 0 0
Number of Treatment Responders With Greater Than or Equal to 50% Reduction in Total Seizures
Timepoint [5] 0 0
Baseline; Week 49 to 60, Week 97 to 108, Week 145 to 156, Week 205 to 216, Week 253 to 264, and Last 12 Weeks
Secondary outcome [6] 0 0
Number of Participants With Changes in the Average Duration of Seizure Subtypes as Assessed by the Participant/Caregiver Global Impression of Change in Seizure Duration (S/CGICSD)
Timepoint [6] 0 0
Up to Week 260
Secondary outcome [7] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Delis-Kaplan Executive Function System (D-KEFS) Visual Scanning Completion Time Scaled Score
Timepoint [7] 0 0
Baseline; up to Week 260
Secondary outcome [8] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in D-KEFS Number Sequencing Completion Time Scaled Score
Timepoint [8] 0 0
Baseline; up to Week 260
Secondary outcome [9] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in D-KEFS Letter Sequencing Completion Time Scaled Score
Timepoint [9] 0 0
Baseline; up to Week 260
Secondary outcome [10] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in D-KEFS Number-letter Switching Completion Time Scaled Score
Timepoint [10] 0 0
Baseline; up to Week 260
Secondary outcome [11] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in D-KEFS Motor Speed Completion Time Scaled Score
Timepoint [11] 0 0
Baseline; up to Week 260
Secondary outcome [12] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Purdue Pegboard Fine Motor Speed (Both Hands Z Score)
Timepoint [12] 0 0
Baseline; up to Week 260
Secondary outcome [13] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Purdue Pegboard Fine Motor Speed (Dominant Hand Z Score)
Timepoint [13] 0 0
Baseline; up to Week 260
Secondary outcome [14] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Purdue Pegboard Fine Motor Speed (Non Dominant Hand Z Score)
Timepoint [14] 0 0
Baseline; up to Week 260
Secondary outcome [15] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Wechsler Adult Intelligence Scale (WAIS) Coding Scaled Score
Timepoint [15] 0 0
Baseline; up to Week 260
Secondary outcome [16] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WAIS Digit Span Backward Scaled Score
Timepoint [16] 0 0
Baseline; up to Week 260
Secondary outcome [17] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WAIS Digit Span Forward Scaled Score
Timepoint [17] 0 0
Baseline; up to Week 260
Secondary outcome [18] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WAIS Longest Digit Span Backward Scaled Score
Timepoint [18] 0 0
Baseline; up to Week 260
Secondary outcome [19] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WAIS Longest Digit Span Forward Scaled Score
Timepoint [19] 0 0
Baseline; up to Week 260
Secondary outcome [20] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Wechsler Abbreviated Scale of Intelligence (WASI)-II Vocabulary T Score
Timepoint [20] 0 0
Baseline; up to Week 260
Secondary outcome [21] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WASI-II Matrix Reasoning T Score
Timepoint [21] 0 0
Baseline; up to Week 260
Secondary outcome [22] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Wechsler Intelligence Scale for Children (WISC) Coding Scaled Score
Timepoint [22] 0 0
Baseline; up to Week 260
Secondary outcome [23] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WISC Digit Span Backward Scaled Score
Timepoint [23] 0 0
Baseline; up to Week 260
Secondary outcome [24] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WISC Digit Span Forward Scaled Score
Timepoint [24] 0 0
Baseline; up to Week 260
Secondary outcome [25] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WISC Longest Digit Span Backward Scaled Score
Timepoint [25] 0 0
Baseline; up to Week 260
Secondary outcome [26] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WISC Longest Digit Span Forward Scaled Score
Timepoint [26] 0 0
Baseline; up to Week 260
Secondary outcome [27] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Wechsler Preschool & Primary Scale of Intelligence (WPPSI)-IV Bug Search T Score
Timepoint [27] 0 0
Baseline; up to Week 260
Secondary outcome [28] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WPPSI-IV Receptive Vocabulary T Score
Timepoint [28] 0 0
Baseline; up to Week 260
Secondary outcome [29] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WPPSI-IV Matrix Reasoning T Score
Timepoint [29] 0 0
Baseline; up to Week 260
Secondary outcome [30] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Expressive One-Word Picture Vocabulary Test (EOWPVT) Scaled Score
Timepoint [30] 0 0
Baseline; up to Week 260
Secondary outcome [31] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in a Developmental NEuroPSYchological Assessment (NEPSY)-2 Word Generation Scaled Score
Timepoint [31] 0 0
Baseline; up to Week 260
Secondary outcome [32] 0 0
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Visual Perception Standard Scaled Score
Timepoint [32] 0 0
Baseline; up to Week 260

Eligibility
Key inclusion criteria
Key

• Participant has completed the treatment phase of their Core Studies: GWEP1332A
[NCT02091206], GWEP1332B [NCT02091375], GWEP1424 [NCT02224703], GWEP1414 [NCT02224560],
GWEP1424 [NCT02224703], and GWEP1423 [NCT02224690].

Key
Minimum age
2 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
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)

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Jazz Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.