The primary aim of Part A of the study to assess the efficacy and tolerability of GWP42003-P compared to placebo as an adjunctive treatment for children with Epilepsy with myoclonic-atonic seizures (EMAS) -associated seizures. Part B of this study will be conducted to evaluate the long-term safety and tolerability of GWP42003-P in participants with EMAS.
The duration of study participation in Part A will be approximately 26 weeks, which includes a 1- to 3-week Screening Period, 4-week Baseline Observation Period, 14-week Dose Optimization Treatment Period, 10-day Taper Period, and a Safety Follow-up Period (4 weeks after end of taper visit). Participants will be randomized centrally in a 1:1 ratio to receive either GWP42003-P or matching placebo. Randomization will be stratified by clobazam use (on/off) and age of seizure onset (3 years of age and younger or older than 3 years of age). Upon completion of the double-blind phase (Part A), participants will have an option to continue in a 54-week open-label extension (Part B).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
3
oral solution
oral solution
Children's Hospital of Alabama
Birmingham, Alabama, United States
University of California Davis Health
Sacramento, California, United States
Part A: Percent Change From Baseline in Epilepsy With Myoclonic-atonic Seizures (EMAS) Associated Seizure Frequency (Myoclonic-atonic, Atonic, Tonic, Clonic, or Tonic-clonic) Over the 14-week Treatment Period
Time frame: Baseline; up to 14 weeks
Part B: Number of Participants With Treatment-emergent Adverse Events
Time frame: up to Week 54
Part B: Number of Participants With Clinically Significant Vital Sign Values
Time frame: up to Week 50
Part B: Number of Participants With Clinically Significant Physical Examination Values
Time frame: up to Week 48
Part B: Number of Participants With Clinically Significant 12-lead Electrocardiogram (ECG) Values
Time frame: up to Week 48
Part B: Number of Participants With Clinically Significant Laboratory Test Values
Time frame: up to Week 48
Part B: Number of Participants With Changes in Tanner Staging
Time frame: up to Week 48
Part B: Number of Participants With a Change in Columbia-Suicide Severity Rating Scale (C-SSRS) Ideation Scores
Time frame: up to Week 54
Part B: Number of Participants With a Change in the Number of Suicide Attempts Per C-SSRS Scores
Time frame: up to Week 54
Part A: Number of Participants Who Achieve ≥ 50% Reduction From Baseline in EMAS-associated Seizures Over the 14-week Treatment Period
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Healthcare of Atlanta
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, United States
Mayo Clinic
Rochester, Minnesota, United States
Wake Forest Baptist Health Sciences, Department of Neurology
Winston-Salem, North Carolina, United States
Cincinnati Children's Hospital Medical Center - TS Clinic
Cincinnati, Ohio, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Dell's Children's Hospital
Austin, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
...and 4 more locations
Time frame: Baseline; up to 14 weeks
Part A: Total Seizure Frequency Over the 14-week Treatment Period
Time frame: Baseline; up to 14 weeks
Part A: Caregiver Global Impression of Change (CGIC) Score at Week 14
Time frame: Week 14
Part A: Physician Global Impression of Change (PGIC) Score at Week 14
Time frame: Week 14
Part A: Number of Participants Who Achieve ≥ 25%, ≥ 50%, ≥ 75%, and 100% Reduction From Baseline in Total Seizures Over the 14-week Treatment Period
Time frame: Baseline; up to 14 weeks
Part A: Change From Baseline in the Number of EMAS-associated Seizure-free Days Over the 14-week Treatment Period
Time frame: Baseline; up to 14 weeks
Part A: Number of Participants With at Least 25% and 50% Reduction From Baseline in the Number of Days Per Week With Myoclonic Seizures During the 14-week Treatment Period
Time frame: Baseline; up to 14 weeks
Part A: Time to Baseline Seizure Frequency
Time frame: up to 14 weeks
Part A: Number of Participants With Treatment-emergent Adverse Events
A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
Time frame: From the time of informed consent signing up to 27 weeks.
Part A: Number of Participants With Clinically Significant Laboratory Test Values
Time frame: up to 27 weeks
Part A: Number of Participants With Clinically Significant Vital Sign Values
Time frame: up to 27 weeks
Part A: Number of Participants With Clinically Significant Physical Examination Values
Time frame: up to 27 weeks
Part A: Number of Participants With Clinically Significant 12-lead ECG Values
Time frame: up to 27 weeks
Part A: Number of Participants With Changes in Tanner Staging
Time frame: up to Day 99
Part A: Number of Participants With a Change in C-SSRS Ideation Scores
Time frame: up to 27 weeks
Part A: Number of Participants With a Change in the Number of Suicide Attempts Per C-SSRS Scores
Time frame: up to 27 weeks
Part B: Percent Change From Baseline in EMAS-associated Seizure Frequency (Myoclonic-atonic, Atonic, Tonic, Clonic, or Tonic-clonic) Over the 48-week Open-label Treatment Period
Time frame: Baseline; up to 48 weeks
Part B: Number of Participants Achieving ≥50% Reduction From Baseline in EMAS-associated Seizures Over the 48-week Open-label Treatment Period
Time frame: Baseline; up to 48 weeks
Part B: Total Seizure Frequency Over the 48-week Open-label Treatment Period
Time frame: up to Week 48
Part B: CGIC Score at Weeks 14, 24, and 48
Time frame: Weeks 14, 24, and 48
Part B: PGIC Score at Weeks 14, 24, and 48
Time frame: Weeks 14, 24, and 48
Part B: Number of Participants Who Achieve ≥ 25%, ≥ 50%, ≥ 75%, and 100% Reduction From Baseline in Total Seizures Over the 48-week Open-label Treatment Period
Time frame: Baseline; up to 48 weeks
Part B: Change From Baseline in the Number of EMAS-associated Seizure-free Days Over the 48-week Open-label Treatment Period
Time frame: Baseline; up to 48 weeks
Part B: Number of Participants With at Least 25% and 50% Reduction From Baseline in the Number of Days Per Week With Myoclonic Seizures During the 48-week Open-label Treatment Period
Time frame: Baseline; up to 48 weeks