A Phase 3 interventional study of GWP42003-P in Infantile Spasms, sponsored by Jazz Pharmaceuticals. Completed at 7 sites in 2 countries. Open to participants aged 1 Month to 24 Months. Per ClinicalTrials.gov, last updated 2022-09-02.
Sponsored by Jazz Pharmaceuticals · Phase 3, Interventional, and Treatment
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.
145 studies on the registry are indexed under Spasm; 15 are open to participants now.
This study's enrollment of 9 is below the median of 52 across 117 interventional studies indexed under Spasm.
Browse Spasm studies →Jazz Pharmaceuticals is the lead sponsor of 167 studies on the registry; 20 are open to participants now.
Of its 39 completed or terminated interventional studies of FDA-regulated products, 28 (72%) have results posted.
Counted across the registry records on this site, refreshed daily.
Only participants who completed the pilot or pivotal phases of the trial may proceed to take part in this open-label extension phase of the trial.
Key eligibility criteria for the blinded phase were as follows:
Key Inclusion Criteria:
Key Exclusion Criteria:
Administered orally, up to the target dose recommended by the data safety monitoring committee. Participants continue at the target dose, or the highest tolerated dose up to the target dose, for a total of 12 months' treatment.
Drug: GWP42003-P
Clear, colorless to yellow solution containing cannabidiol dissolved in the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring.
Also known as: CBD, Cannabidiol
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
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
| Milestone | GWP42003-P OS |
|---|---|
| Started | 9 |
| Completed | 2 |
| Not completed | 7 |
| Withdrew: Withdrawal by subject | 3 |
| Withdrew: Lack of efficacy | 3 |
| Withdrew: Other | 1 |
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.
| Participants | GWP42003-P OS |
|---|---|
| Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs) | 7 |
| Participants | GWP42003-P OS |
|---|---|
| Day 19, Low | 3 |
| Day 19, High | 4 |
| Day 29, Low | 3 |
| Day 29, High | 4 |
| Day 43, Low | 1 |
| Day 43, High | 2 |
| Day 71, Low | 1 |
| Day 71, High | 3 |
| Day 127, Low | 1 |
| Day 127, High | 3 |
| Day 211, Low | 1 |
| Day 211, High | 2 |
| Day 295, Low | 2 |
| Day 295, High | 0 |
| Day 379, Low | 4 |
| Day 379, High | 4 |
| Day 389, Low | 1 |
| Day 389, High | 4 |
| Participants | GWP42003-P OS |
|---|---|
| Day 19, Low | 6 |
| Day 19, High | 7 |
| Day 29, Low | 6 |
| Day 29, High | 6 |
| Day 43, Low | 4 |
| Day 43, High | 5 |
| Day 71, Low | 2 |
| Day 71, High | 4 |
| Day 127, Low | 4 |
| Day 127, High | 3 |
| Day 211, Low | 2 |
| Day 211, High | 3 |
| Day 295, Low | 2 |
| Day 295, High | 3 |
| Day 379, Low | 5 |
| Day 379, High | 7 |
| Day 389, Low | 3 |
| Day 389, High | 2 |
Clinical relevance was determined by the investigator.
| Participants | GWP42003-P OS |
|---|---|
| Day 19 | 0 |
| Day 29 | 0 |
| Day 43 | 0 |
| Day 71 | 1 |
| Day 127 | 0 |
| Day 211 | 0 |
| Day 295 | 0 |
| Day 379 | 0 |
| Day 389 | 0 |
Clinical significance was determined by the investigator.
| Participants | GWP42003-P OS |
|---|---|
| Number of Participants With Clinically Significant Electrocardiogram Findings | 0 |
Clinical significance was determined by the investigator.
| Participants | GWP42003-P OS |
|---|---|
| Number of Participants With Clinically Significant Vital Sign Findings | 0 |
Clinical significance was determined by the investigator.
| Participants | GWP42003-P OS |
|---|---|
| Number of Participants With Clinically Significant Physical Examination Findings | 0 |
Clinical spasms were determined by video-electroencephalography (VEEG) for at least 8 hours and up to 24 hours.
| Participants | GWP42003-P OS |
|---|---|
| Day 29 | 1 |
| Day 43 | 2 |
| Day 127 | 1 |
| Day 211 | 1 |
| Day 295 | 1 |
| Day 379 | 3 |
Clinical spasms were determined by VEEG for at least 8 hours and up to 24 hours.
| percentage of participants | GWP42003-P OS |
|---|---|
| Day 29 | 11.1 |
| Day 43 | 22.2 |
| Day 127 | 11.1 |
| Day 211 | 11.1 |
| Day 295 | 11.1 |
| Day 379 | 33.3 |
Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
| Participants | GWP42003-P OS |
|---|---|
| Day 29 | 1 |
| Day 43 | 0 |
| Day 127 | 1 |
| Day 211 | 0 |
| Day 295 | 1 |
| Day 379 | 3 |
Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
| percentage of participants | GWP42003-P OS |
|---|---|
| Day 29 | 11.1 |
| Day 43 | 0 |
| Day 127 | 11.1 |
| Day 211 | 0 |
| Day 295 | 11.1 |
| Day 379 | 33.3 |
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.
| Participants | GWP42003-P OS |
|---|---|
| Day 19, Clonic | 0 |
| Day 19, Tonic-Clonic | 0 |
| Day 19, Atonic | 0 |
| Day 19, Myoclonic | 0 |
| Day 19, Focal | 1 |
| Day 19, Absence | 0 |
| Day 19, Not Done | 0 |
| Day 29, Clonic | 0 |
| Day 29, Tonic-Clonic | 0 |
| Day 29, Atonic | 0 |
| Day 29, Myoclonic | 0 |
| Day 29, Focal | 1 |
| Day 29, Absence | 0 |
| Day 29, Not Done | 0 |
| Day 127, Clonic | 0 |
| Day 127, Tonic-Clonic | 1 |
| Day 127, Atonic | 0 |
| Day 127, Myoclonic | 1 |
| Day 127, Focal | 0 |
| Day 127, Absence | 1 |
| Day 127, No Done | 0 |
| Day 211, Clonic | 0 |
| Day 211, Tonic-Clonic | 1 |
| Day 211, Atonic | 0 |
| Day 211, Myoclonic | 0 |
| Day 211, Focal | 0 |
| Day 211, Absence | 1 |
| Day 211, Not Done | 0 |
| Day 295, Clonic | 0 |
| Day 295, Tonic-Clonic | 1 |
| Day 295, Atonic | 1 |
| Day 295, Myoclonic | 0 |
| Day 295, Focal | 0 |
| Day 295, Absence | 1 |
| Day 295, Not Done | 0 |
| Day 379, Clonic | 1 |
| Day 379, Tonic-Clonic | 1 |
| Day 379, Atonic | 0 |
| Day 379, Myoclonic | 0 |
| Day 379, Focal | 1 |
| Day 379, Absence | 1 |
| Day 379, Not Done | 0 |
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.
| participants | GWP42003-P OS |
|---|---|
| Day 29, Very Much Improved | 1 |
| Day 29, Much Improved | 1 |
| Day 29, Slightly Improved | 5 |
| Day 29, No Change | 1 |
| Day 29, Slightly Worse | 0 |
| Day 29, Much Worse | 0 |
| Day 29, Very Much Worse | 0 |
| Day 29, Not Done | 0 |
| Day 43, Very Much Improved | 2 |
| Day 43, Much Improved | 1 |
| Day 43, Slightly Improved | 3 |
| Day 43, No Change | 0 |
| Day 43, Slightly Worse | 0 |
| Day 43, Much Worse | 0 |
| Day 43, Very Much Worse | 0 |
| Day 43, Not Done | 0 |
| Day 71, Very Much Improved | 3 |
| Day 71, Much Improved | 0 |
| Day 71, Slightly Improved | 1 |
| Day 71 No Change | 0 |
| Day 71, Slightly Worse | 1 |
| Day 71, Much Worse | 0 |
| Day 71, Very Much Worse | 0 |
| Day 71, Not Done | 0 |
| Day 127, Very Much Improved | 2 |
| Day 127, Much Improved | 2 |
| Day 127, Slightly Improved | 0 |
| Day 127, No Change | 0 |
| Day 127, Slightly Worse | 0 |
| Day 127, Much Worse | 0 |
| Day 127, Very Much Worse | 0 |
| Day 127, Not Done | 0 |
| Day 211, Very Much Improved | 1 |
| Day 211, Much Improved | 2 |
| Day 211, Slightly Improved | 0 |
| Day 211, No Change | 0 |
| Day 211, Slightly Worse | 0 |
| Day 211, Much Worse | 0 |
| Day 211, Very Much Worse | 0 |
| Day 211, Not Done | 0 |
| Day 295, Very Much Improved | 1 |
| Day 295, Much Improved | 2 |
| Day 295, Slightly Improved | 0 |
| Day 295, No Change | 0 |
| Day 295, Slightly Worse | 0 |
| Day 295, Much Worse | 0 |
| Day 295, Very Much Worse | 0 |
| Day 295, Not Done | 0 |
| Day 379, Very Much Improved | 2 |
| Day 379, Much Improved | 1 |
| Day 379, Slightly Improved | 3 |
| Day 379, No Change | 0 |
| Day 379, Slightly Worse | 1 |
| Day 379, Much Worse | 1 |
| Day 379, Very Much Worse | 0 |
| Day 379, Not Done | 1 |
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.
| participants | GWP42003-P OS |
|---|---|
| Day 29, Very Much Improved | 1 |
| Day 29, Much Improved | 1 |
| Day 29, Slightly Improved | 2 |
| Day 29, No Change | 4 |
| Day 29, Slightly Worse | 0 |
| Day 29, Much Worse | 0 |
| Day 29, Very Much Worse | 0 |
| Day 29, Not Done | 0 |
| Day 43, Very Much Improved | 1 |
| Day 43, Much Improved | 0 |
| Day 43, Slightly Improved | 4 |
| Day 43, No Change | 1 |
| Day 43, Slightly Worse | 0 |
| Day 43, Much Worse | 0 |
| Day 43, Very Much Worse | 0 |
| Day 43, Not Done | 0 |
| Day 71, Very Much Improved | 1 |
| Day 71, Much Improved | 2 |
| Day 71, Slightly Improved | 1 |
| Day 71, No Change | 1 |
| Day 71, Slightly Worse | 0 |
| Day 71, Much Worse | 0 |
| Day 71, Very Much Worse | 0 |
| Day 71, Not Done | 0 |
| Day 127, Very Much Improved | 1 |
| Day 127, Much Improved | 2 |
| Day 127, Slightly Improved | 1 |
| Day 127, No Change | 0 |
| Day 127, Slightly Worse | 0 |
| Day 127, Much Worse | 0 |
| Day 127, Very Much Worse | 0 |
| Day 127, Not Done | 0 |
| Day 211, Very Much Improved | 0 |
| Day 211, Much Improved | 1 |
| Day 211, Slightly Improved | 1 |
| Day 211, No Change | 1 |
| Day 211, Slightly Worse | 0 |
| Day 211, Much Worse | 0 |
| Day 211, Very Much Worse | 0 |
| Day 211, Not Done | 0 |
| Day 295, Very Much Improved | 0 |
| Day 295, Much Improved | 1 |
| Day 295, Slightly Improved | 0 |
| Day 295, No Change | 1 |
| Day 295, Slightly Worse | 0 |
| Day 295, Much Worse | 1 |
| Day 295, Very Much Worse | 0 |
| Day 295, Not Done | 0 |
| Day 379, Very Much Improved | 0 |
| Day 379, Much Improved | 1 |
| Day 379, Slightly Improved | 3 |
| Day 379, No Change | 2 |
| Day 379, Slightly Worse | 1 |
| Day 379, Much Worse | 1 |
| Day 379, Very Much Worse | 0 |
| Day 379, Not Done | 1 |
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.
| Participants | GWP42003-P OS |
|---|---|
| Day 29 | 1 |
| Day 43 | 0 |
| Day 127 | 0 |
| Day 211 | 0 |
| Day 295 | 1 |
| Day 379 | 3 |
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.
| percentage of participants | GWP42003-P OS |
|---|---|
| Day 29 | 11.1 |
| Day 43 | 0 |
| Day 127 | 0 |
| Day 211 | 0 |
| Day 295 | 11.1 |
| Day 379 | 33.3 |
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.
| centimeters | GWP42003-P OS |
|---|---|
| Baseline (Day 1 of Pilot Study) | 75.04 ± 9.557 |
| Day 29, Open-label Extension (OLE) | 0.85 ± 0.980 |
| Day 43, OLE | 0.92 ± 1.530 |
| Day 71, OLE | 3.10 ± 0.652 |
| Day 127, OLE | 3.38 ± 1.377 |
| Day 211, OLE | 5.33 ± 1.258 |
| Day 295, OLE | 8.17 ± 1.528 |
| Day 379, OLE | 5.31 ± 4.036 |
| Day 389, OLE | 3.55 ± 4.085 |
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.
| kilograms | GWP42003-P OS |
|---|---|
| Baseline (Day 1 of Pilot Study) | 9.92 ± 3.245 |
| Day 29, OLE | 0.39 ± 0.309 |
| Day 43, OLE | 0.75 ± 0.809 |
| Day 71, OLE | 1.10 ± 0.430 |
| Day 127, OLE | 1.25 ± 0.420 |
| Day 211, OLE | 1.17 ± 0.503 |
| Day 295, OLE | 2.10 ± 0.889 |
| Day 379, OLE | 1.19 ± 0.862 |
| Day 389, OLE | 0.98 ± 0.637 |
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.
| centimeters | GWP42003-P OS |
|---|---|
| Baseline (Day 1 of Pilot Study) | 44.71 ± 2.724 |
| Day 29, OLE | -0.01 ± 1.229 |
| Day 43, OLE | 0.54 ± 0.288 |
| Day 71, OLE | 0.70 ± 0.570 |
| Day 127, OLE | 1.50 ± 0.500 |
| Day 211, OLE | 0.75 ± 0.354 |
| Day 295, OLE | -0.3 ± 2.31 |
| Day 379, OLE | 1.14 ± 1.707 |
| Day 389, OLE | 0.90 ± 1.782 |
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.
| score on a scale | GWP42003-P OS |
|---|---|
| Baseline, Day 1 of Pilot Study | 33.6 ± 37.27 |
| Day 211 of OLE | 6.3 ± 3.51 |
| Day 379 of OLE | -5.4 ± 23.42 |
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.
No measurements were reported for this outcome.
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.
No measurements were reported for this outcome.
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.
No measurements were reported for this outcome.
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.
No measurements were reported for this outcome.
Collected over From signing of informed consent up to Day 417. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cannabidiol OS | 0/9 (0%) | 2/9 (22.2%) | 7/9 (77.8%) |
| Event | Cannabidiol OS |
|---|---|
| Enterovirus infectionInfections and infestations | 2/9 |
| PneumoniaInfections and infestations | 2/9 |
| Rhinovirus infectionInfections and infestations | 2/9 |
| Acute respiratory failureRespiratory, thoracic and mediastinal disorders | 1/9 |
| HypoxiaRespiratory, thoracic and mediastinal disorders | 1/9 |
| Pneumonia aspirationRespiratory, thoracic and mediastinal disorders | 1/9 |
| Respiratory distressRespiratory, thoracic and mediastinal disorders | 1/9 |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 1/9 |
| DiarrhoeaGastrointestinal disorders | 1/9 |
| Urinary retentionRenal and urinary disorders | 1/9 |
| Event | Cannabidiol OS |
|---|---|
| Upper respiratory tract congestionRespiratory, thoracic and mediastinal disorders | 3/9 |
| PyrexiaGeneral disorders | 2/9 |
| IrritabilityPsychiatric disorders | 2/9 |
| Blood triglycerides increasedInvestigations | 2/9 |
| AnaemiaBlood and lymphatic system disorders | 2/9 |
| Urinary tract infectionInfections and infestations | 2/9 |
| HypertensionVascular disorders | 1/9 |
| HypotensionVascular disorders | 1/9 |
| Drug toleranceGeneral disorders | 1/9 |
| Sleep disorderPsychiatric disorders | 1/9 |
| Age, Continuous(years) | GWP42003-P OS |
|---|---|
| Mean | 12.2 ± 5.56 |
| Age, Customized(Participants) | GWP42003-P OS |
|---|---|
| Infants and toddlers (28 days-23 months) | 9 |
| Sex: Female, Male(Participants) | GWP42003-P OS |
|---|---|
| Female | 6 |
| Male | 3 |
| Race (NIH/OMB)(Participants) | GWP42003-P OS |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 8 |
| More than one race | 0 |
| Unknown or Not Reported | 1 |
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Jazz Pharmaceuticals