Trial Outcomes & Findings for Efficacy and Safety of GWP42003-P Oral Solution in Children With Epilepsy With Myoclonic-atonic Seizures (NCT NCT05288283)
NCT ID: NCT05288283
Last Updated: 2025-02-03
Results Overview
TERMINATED
PHASE3
3 participants
Baseline; up to 14 weeks
2025-02-03
Participant Flow
Participants were screened for enrollment at 14 activated sites: 10 in the USA and 4 in Italy.
A total of 3 participants were enrolled in Part A of the study. One participant was a screen failure, and 2 underwent randomization. Study was terminated prior to Part B.
Participant milestones
| Measure |
GWP42003-P
Participants who received a dose escalation of GWP42003-P twice a day (BID) orally.
|
Placebo
Participants who received the matching placebo.
|
|---|---|---|
|
Overall Study
STARTED
|
1
|
1
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
| Measure |
GWP42003-P
Participants who received a dose escalation of GWP42003-P twice a day (BID) orally.
|
Placebo
Participants who received the matching placebo.
|
|---|---|---|
|
Overall Study
Study Terminated Early
|
1
|
1
|
Baseline Characteristics
Efficacy and Safety of GWP42003-P Oral Solution in Children With Epilepsy With Myoclonic-atonic Seizures
Baseline characteristics by cohort
| Measure |
GWP42003-P
n=1 Participants
Participants who received a dose escalation of GWP42003-P twice a day (BID) orally.
|
Placebo
n=1 Participants
Participants who received the matching placebo.
|
Total
n=2 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline; up to 14 weeksPopulation: No data collected, as the study was terminated before this endpoint was analyzed.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: up to Week 54Population: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: up to Week 50Population: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: up to Week 48Population: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: up to Week 48Population: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: up to Week 48Population: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: up to Week 48Population: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: up to Week 54Population: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: up to Week 54Population: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline; up to 14 weeksPopulation: No data collected, as the study was terminated before this endpoint was analyzed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline; up to 14 weeksPopulation: No data collected, as the study was terminated before this endpoint was analyzed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 14Population: No data collected, as the study was terminated before this endpoint was analyzed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 14Population: No data collected, as the study was terminated before this endpoint was analyzed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline; up to 14 weeksPopulation: No data collected, as the study was terminated before this endpoint was analyzed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline; up to 14 weeksPopulation: No data collected, as the study was terminated before this endpoint was analyzed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline; up to 14 weeksPopulation: No data collected, as the study was terminated before this endpoint was analyzed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 14 weeksPopulation: No data collected, as the study was terminated before this endpoint was analyzed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From the time of informed consent signing up to 27 weeks.A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
Outcome measures
| Measure |
GWP42003-P
n=1 Participants
Participants who received a dose escalation of GWP42003-P twice a day (BID) orally.
|
Placebo
n=1 Participants
Participants who received the matching placebo.
|
|---|---|---|
|
Part A: Number of Participants With Treatment-emergent Adverse Events
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: up to 27 weeksOutcome measures
| Measure |
GWP42003-P
n=1 Participants
Participants who received a dose escalation of GWP42003-P twice a day (BID) orally.
|
Placebo
n=1 Participants
Participants who received the matching placebo.
|
|---|---|---|
|
Part A: Number of Participants With Clinically Significant Laboratory Test Values
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: up to 27 weeksOutcome measures
| Measure |
GWP42003-P
n=1 Participants
Participants who received a dose escalation of GWP42003-P twice a day (BID) orally.
|
Placebo
n=1 Participants
Participants who received the matching placebo.
|
|---|---|---|
|
Part A: Number of Participants With Clinically Significant Vital Sign Values
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: up to 27 weeksOutcome measures
| Measure |
GWP42003-P
n=1 Participants
Participants who received a dose escalation of GWP42003-P twice a day (BID) orally.
|
Placebo
n=1 Participants
Participants who received the matching placebo.
|
|---|---|---|
|
Part A: Number of Participants With Clinically Significant Physical Examination Values
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: up to 27 weeksOutcome measures
| Measure |
GWP42003-P
n=1 Participants
Participants who received a dose escalation of GWP42003-P twice a day (BID) orally.
|
Placebo
n=1 Participants
Participants who received the matching placebo.
|
|---|---|---|
|
Part A: Number of Participants With Clinically Significant 12-lead ECG Values
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: up to Day 99Population: No data collected, as the study was terminated before this endpoint was analyzed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to 27 weeksOutcome measures
| Measure |
GWP42003-P
n=1 Participants
Participants who received a dose escalation of GWP42003-P twice a day (BID) orally.
|
Placebo
n=1 Participants
Participants who received the matching placebo.
|
|---|---|---|
|
Part A: Number of Participants With a Change in C-SSRS Ideation Scores
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: up to 27 weeksOutcome measures
| Measure |
GWP42003-P
n=1 Participants
Participants who received a dose escalation of GWP42003-P twice a day (BID) orally.
|
Placebo
n=1 Participants
Participants who received the matching placebo.
|
|---|---|---|
|
Part A: Number of Participants With a Change in the Number of Suicide Attempts Per C-SSRS Scores
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline; up to 48 weeksPopulation: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline; up to 48 weeksPopulation: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: up to Week 48Population: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Weeks 14, 24, and 48Population: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Weeks 14, 24, and 48Population: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline; up to 48 weeksPopulation: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline; up to 48 weeksPopulation: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline; up to 48 weeksPopulation: Not data was collected, as the study was terminated prior to part B.
Outcome measures
Outcome data not reported
Adverse Events
GWP42003-P
Placebo
Serious adverse events
| Measure |
GWP42003-P
n=1 participants at risk
Participants who received a dose escalation of GWP42003-P twice a day (BID) orally.
|
Placebo
n=1 participants at risk
Participants who received the matching placebo.
|
|---|---|---|
|
Infections and infestations
Gastroenteritis viral
|
100.0%
1/1 • All treatment-emergent adverse events (TEAE) were collected from signing the informed consent up to approximately 27 weeks. No participants entered the open-label extension phase due to study termination.
A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
|
0.00%
0/1 • All treatment-emergent adverse events (TEAE) were collected from signing the informed consent up to approximately 27 weeks. No participants entered the open-label extension phase due to study termination.
A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
|
Other adverse events
| Measure |
GWP42003-P
n=1 participants at risk
Participants who received a dose escalation of GWP42003-P twice a day (BID) orally.
|
Placebo
n=1 participants at risk
Participants who received the matching placebo.
|
|---|---|---|
|
Psychiatric disorders
Irritability
|
100.0%
1/1 • All treatment-emergent adverse events (TEAE) were collected from signing the informed consent up to approximately 27 weeks. No participants entered the open-label extension phase due to study termination.
A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
|
100.0%
1/1 • All treatment-emergent adverse events (TEAE) were collected from signing the informed consent up to approximately 27 weeks. No participants entered the open-label extension phase due to study termination.
A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
|
|
General disorders
Fatigue
|
100.0%
1/1 • All treatment-emergent adverse events (TEAE) were collected from signing the informed consent up to approximately 27 weeks. No participants entered the open-label extension phase due to study termination.
A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
|
0.00%
0/1 • All treatment-emergent adverse events (TEAE) were collected from signing the informed consent up to approximately 27 weeks. No participants entered the open-label extension phase due to study termination.
A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
|
|
Cardiac disorders
Tachycardia
|
100.0%
1/1 • All treatment-emergent adverse events (TEAE) were collected from signing the informed consent up to approximately 27 weeks. No participants entered the open-label extension phase due to study termination.
A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
|
0.00%
0/1 • All treatment-emergent adverse events (TEAE) were collected from signing the informed consent up to approximately 27 weeks. No participants entered the open-label extension phase due to study termination.
A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
|
|
Investigations
International normalised ratio increased
|
100.0%
1/1 • All treatment-emergent adverse events (TEAE) were collected from signing the informed consent up to approximately 27 weeks. No participants entered the open-label extension phase due to study termination.
A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
|
0.00%
0/1 • All treatment-emergent adverse events (TEAE) were collected from signing the informed consent up to approximately 27 weeks. No participants entered the open-label extension phase due to study termination.
A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
|
|
Investigations
Blood triglycerides
|
0.00%
0/1 • All treatment-emergent adverse events (TEAE) were collected from signing the informed consent up to approximately 27 weeks. No participants entered the open-label extension phase due to study termination.
A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
|
100.0%
1/1 • All treatment-emergent adverse events (TEAE) were collected from signing the informed consent up to approximately 27 weeks. No participants entered the open-label extension phase due to study termination.
A TEAE is an adverse event that started or worsened in severity or seriousness following the first dose of the investigational medicinal product.
|
Additional Information
Clinical Trial Disclosure & Transparency
Jazz Pharmaceuticals
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place