This trial consists of 3 parts: a pilot safety phase, a pivotal randomized controlled phase, and an open-label extension phase. The open-label extension phase only will be described in this record. All participants will receive GWP42003-P.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
9
Clear, colorless to yellow solution containing cannabidiol dissolved in the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring.
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Le Bonheur Children's Hospital
Memphis, Tennessee, United States
Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs)
TEAEs were collected in members of the Safety Population, comprised of all participants who received at least 1 dose of GWP42003-P. TEAEs are defined as all adverse events not present prior to the first investigational medicinal product (IMP) or placebo administration or any event already present that worsened in severity or frequency following IMP.
Time frame: From signing of informed consent up to Day 417
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Time frame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Time frame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389
Number of Participants With Any Clinically Relevant Urinalysis Parameter Value
Clinical relevance was determined by the investigator.
Time frame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389
Number of Participants With Clinically Significant Electrocardiogram Findings
Clinical significance was determined by the investigator.
Time frame: From signing of informed consent up to Day 389
Number of Participants With Clinically Significant Vital Sign Findings
Clinical significance was determined by the investigator.
Time frame: From signing of informed consent up to Day 389
Number of Participants With Clinically Significant Physical Examination Findings
Clinical significance was determined by the investigator.
Time frame: From signing of informed consent up to Day 389
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Valley Health Clinical Research
Winchester, Virginia, United States
Uniwersyteckie Centrum Kliniczne
Gdansk, Poland
Centrum Medyczne POMOC
Lodz, Poland
Number of Participants Free of Clinical Spasms
Clinical spasms were determined by video-electroencephalography (VEEG) for at least 8 hours and up to 24 hours.
Time frame: Days 29, 43, 127, 211, 295, and 379
Percentage of Participants Free of Clinical Spasms
Clinical spasms were determined by VEEG for at least 8 hours and up to 24 hours.
Time frame: Days 29, 43, 127, 211, 295, and 379
Number of Participants With a Resolution of Hypsarrhythmia
Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
Time frame: Days 29, 43, 127, 211, 295, and 379
Percentage of Participants With a Resolution of Hypsarrhythmia
Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
Time frame: Days 29, 43, 127, 211, 295, and 379
Number of Participants Experiencing Spasms and Seizures by Subtype
Caregivers recorded the participant's spasms and seizures by category in a daily diary. Subtypes of spasms and seizure included, clonic, tonic-clonic, myoclonic, focal, and absence.
Time frame: Days 19, 29, 127, 211, 295, and 379
Caregiver Global Impression of Change (CGIC)
The CGIC is a single-question assessment completed by the caregiver. The question assessed the status of the participant's condition since treatment start. The caregiver provided a rating 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.
Time frame: Baseline; Days 29, 43, 71, 127, 211, 295, and 379
Physician Global Impression of Change (PGIC)
The PGIC is a single-question assessment completed by the investigator. The question assessed the status of the participant's condition since treatment start. The investigator provided a rating 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.
Time frame: Baseline; Days 29, 43, 71, 127, 211, 295, and 379
Number of Responders
A responder is defined as a participant experiencing a resolution of hypsarrhythmia and free of spasms. Test for responders was conducted by VEEG for at least 8 hours and up to 24 hours.
Time frame: Days 29, 43, 127, 211, 295, and 379
Percentage of Responders
A responder is defined as a participant experiencing a resolution of hypsarrhythmia and free of spasms. Test for responders was conducted by VEEG for at least 8 hours and up to 24 hours.
Time frame: Days 29, 43, 127, 211, 295, and 379
Change From Baseline in Height
A positive change indicates an increase in the average participant's height. A negative change indicates a decrease in the average participant's height. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389
Change From Baseline in Body Weight.
A positive change indicates an increase in the average participant's weight. A negative change indicates a decrease in the average participant's weight. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389
Change From Baseline in Head Circumference
A positive change indicates an increase in the average participant's head circumference. A negative change indicates a decrease in the average participant's head circumference. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline (Day 1 of PIlot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389
Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Score
The Vineland-II scores were assessed by the participant's caregiver. Caregivers were asked to score questions in the following categories: the participant's communication, daily living, physical activity, problem behaviors, and social skills and relationships. Scoring was slightly different for each section, but generally ranged from "usually" (2) to "never" (0). The total score is calculated as the sum of standard scores from the domains and converted into the adaptive behavior composite score (ranging from 20 to 160). Higher scores represent greater levels of functioning, and lower scores represent lower levels of functioning. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline (Day 1 of Pilot Study); Day 211, Day 379
Number of Participants With Relapse of Spasms
Analysis could not be conducted for this outcome measure because the study met No Go Criteria. The Pilot Phase concluded after 9 participants completed treatment and demonstrated continued hypsarrhythmia and spasms on follow-up VEEG. The Pivotal Phase was not initiated; however, participants completing the Pilot Phase could roll into the Open Label Extension Phase for up to 1 year.
Time frame: Day 16 to Day 379
Percentage of Participants With Relapse of Spasms
Analysis could not be conducted for this outcome measure because the study met No Go Criteria. The Pilot Phase concluded after 9 participants completed treatment and demonstrated continued hypsarrhythmia and spasms on follow-up VEEG. The Pivotal Phase was not initiated; however, participants completing the Pilot Phase could roll into the Open Label Extension Phase for up to 1 year.
Time frame: Day 16 to Day 379
Average Time to Cessation of Spasms
Analysis could not be conducted for this outcome measure because the study met No Go Criteria. The Pilot Phase concluded after 9 participants completed treatment and demonstrated continued hypsarrhythmia and spasms on follow-up VEEG. The Pivotal Phase was not initiated; however, participants completing the Pilot Phase could roll into the Open Label Extension Phase for up to 1 year.
Time frame: Day 1 to Day 379
Average Time to Relapse
Analysis could not be conducted for this outcome measure because the study met No Go Criteria. The Pilot Phase concluded after 9 participants completed treatment and demonstrated continued hypsarrhythmia and spasms on follow-up VEEG. The Pivotal Phase was not initiated; however, participants completing the Pilot Phase could roll into the Open Label Extension Phase for up to 1 year.
Time frame: Day 16 to Day 379