Trial Outcomes & Findings for Phase 3 Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms: Open-label Extension Phase (GWPCARE7) (NCT NCT02954887)
NCT ID: NCT02954887
Last Updated: 2022-09-02
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
9 participants
Primary outcome timeframe
From signing of informed consent up to Day 417
Results posted on
2022-09-02
Participant Flow
Participant milestones
| Measure |
GWP42003-P OS
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open Label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Overall Study
STARTED
|
9
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
7
|
Reasons for withdrawal
| Measure |
GWP42003-P OS
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open Label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
3
|
|
Overall Study
Lack of Efficacy
|
3
|
|
Overall Study
Other
|
1
|
Baseline Characteristics
Phase 3 Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms: Open-label Extension Phase (GWPCARE7)
Baseline characteristics by cohort
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open Label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Age, Continuous
|
12.2 years
STANDARD_DEVIATION 5.56 • n=5 Participants
|
|
Age, Customized
Infants and toddlers (28 days-23 months)
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From signing of informed consent up to Day 417Population: Safety Analysis Set: all participants who had received ≥ 1 dose of GWP42003-P
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.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs)
|
7 Participants
|
PRIMARY outcome
Timeframe: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389Population: Safety Analysis Set. Only participants with evaluable data were analyzed.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 19, Low
|
3 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 19, High
|
4 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 29, Low
|
3 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 29, High
|
4 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 43, Low
|
1 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 43, High
|
2 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 71, Low
|
1 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 71, High
|
3 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 127, Low
|
1 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 127, High
|
3 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 211, Low
|
1 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 211, High
|
2 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 295, Low
|
2 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 295, High
|
0 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 379, Low
|
4 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 379, High
|
4 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 389, Low
|
1 Participants
|
|
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Day 389, High
|
4 Participants
|
PRIMARY outcome
Timeframe: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389Population: Safety Analysis Set. Only participants with evaluable data were analyzed.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 19, Low
|
6 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 19, High
|
7 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 29, Low
|
6 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 29, High
|
6 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 211, Low
|
2 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 211, High
|
3 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 295, Low
|
2 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 295, High
|
3 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 379, Low
|
5 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 379, High
|
7 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 389, Low
|
3 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 389, High
|
2 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 43, Low
|
4 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 43, High
|
5 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 71, Low
|
2 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 71, High
|
4 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 127, Low
|
4 Participants
|
|
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Day 127, High
|
3 Participants
|
PRIMARY outcome
Timeframe: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389Population: Safety Analysis Set. Only participants with evaluable data were analyzed.
Clinical relevance was determined by the investigator.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Number of Participants With Any Clinically Relevant Urinalysis Parameter Value
Day 19
|
0 Participants
|
|
Number of Participants With Any Clinically Relevant Urinalysis Parameter Value
Day 29
|
0 Participants
|
|
Number of Participants With Any Clinically Relevant Urinalysis Parameter Value
Day 43
|
0 Participants
|
|
Number of Participants With Any Clinically Relevant Urinalysis Parameter Value
Day 71
|
1 Participants
|
|
Number of Participants With Any Clinically Relevant Urinalysis Parameter Value
Day 127
|
0 Participants
|
|
Number of Participants With Any Clinically Relevant Urinalysis Parameter Value
Day 211
|
0 Participants
|
|
Number of Participants With Any Clinically Relevant Urinalysis Parameter Value
Day 295
|
0 Participants
|
|
Number of Participants With Any Clinically Relevant Urinalysis Parameter Value
Day 379
|
0 Participants
|
|
Number of Participants With Any Clinically Relevant Urinalysis Parameter Value
Day 389
|
0 Participants
|
PRIMARY outcome
Timeframe: From signing of informed consent up to Day 389Population: Safety Analysis Set
Clinical significance was determined by the investigator.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Number of Participants With Clinically Significant Electrocardiogram Findings
|
0 Participants
|
PRIMARY outcome
Timeframe: From signing of informed consent up to Day 389Population: Safety Analysis Set
Clinical significance was determined by the investigator.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Number of Participants With Clinically Significant Vital Sign Findings
|
0 Participants
|
PRIMARY outcome
Timeframe: From signing of informed consent up to Day 389Population: Safety Analysis Set
Clinical significance was determined by the investigator.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Number of Participants With Clinically Significant Physical Examination Findings
|
0 Participants
|
SECONDARY outcome
Timeframe: Days 29, 43, 127, 211, 295, and 379Population: Safety Analysis Set. Only participants with evaluable data were analyzed.
Clinical spasms were determined by video-electroencephalography (VEEG) for at least 8 hours and up to 24 hours.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Number of Participants Free of Clinical Spasms
Day 29
|
1 Participants
|
|
Number of Participants Free of Clinical Spasms
Day 43
|
2 Participants
|
|
Number of Participants Free of Clinical Spasms
Day 127
|
1 Participants
|
|
Number of Participants Free of Clinical Spasms
Day 211
|
1 Participants
|
|
Number of Participants Free of Clinical Spasms
Day 295
|
1 Participants
|
|
Number of Participants Free of Clinical Spasms
Day 379
|
3 Participants
|
SECONDARY outcome
Timeframe: Days 29, 43, 127, 211, 295, and 379Population: Safety Analysis Set. Only participants with evaluable data were analyzed.
Clinical spasms were determined by VEEG for at least 8 hours and up to 24 hours.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Percentage of Participants Free of Clinical Spasms
Day 295
|
11.1 percentage of participants
|
|
Percentage of Participants Free of Clinical Spasms
Day 379
|
33.3 percentage of participants
|
|
Percentage of Participants Free of Clinical Spasms
Day 29
|
11.1 percentage of participants
|
|
Percentage of Participants Free of Clinical Spasms
Day 43
|
22.2 percentage of participants
|
|
Percentage of Participants Free of Clinical Spasms
Day 127
|
11.1 percentage of participants
|
|
Percentage of Participants Free of Clinical Spasms
Day 211
|
11.1 percentage of participants
|
SECONDARY outcome
Timeframe: Days 29, 43, 127, 211, 295, and 379Population: Safety Analysis Set. Only participants with evaluable data were analyzed.
Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Number of Participants With a Resolution of Hypsarrhythmia
Day 29
|
1 Participants
|
|
Number of Participants With a Resolution of Hypsarrhythmia
Day 43
|
0 Participants
|
|
Number of Participants With a Resolution of Hypsarrhythmia
Day 127
|
1 Participants
|
|
Number of Participants With a Resolution of Hypsarrhythmia
Day 211
|
0 Participants
|
|
Number of Participants With a Resolution of Hypsarrhythmia
Day 295
|
1 Participants
|
|
Number of Participants With a Resolution of Hypsarrhythmia
Day 379
|
3 Participants
|
SECONDARY outcome
Timeframe: Days 29, 43, 127, 211, 295, and 379Population: Safety Analysis Set. Only participants with evaluable data were analyzed.
Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Percentage of Participants With a Resolution of Hypsarrhythmia
Day 29
|
11.1 percentage of participants
|
|
Percentage of Participants With a Resolution of Hypsarrhythmia
Day 43
|
0 percentage of participants
|
|
Percentage of Participants With a Resolution of Hypsarrhythmia
Day 127
|
11.1 percentage of participants
|
|
Percentage of Participants With a Resolution of Hypsarrhythmia
Day 211
|
0 percentage of participants
|
|
Percentage of Participants With a Resolution of Hypsarrhythmia
Day 295
|
11.1 percentage of participants
|
|
Percentage of Participants With a Resolution of Hypsarrhythmia
Day 379
|
33.3 percentage of participants
|
SECONDARY outcome
Timeframe: Days 19, 29, 127, 211, 295, and 379Population: Safety Analysis Set
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.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 19, Clonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 19, Tonic-Clonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 19, Atonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 19, Myoclonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 19, Focal
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 19, Absence
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 29, Myoclonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 29, Focal
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 29, Absence
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 29, Not Done
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 127, Clonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 127, Myoclonic
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 127, Focal
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 127, Absence
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 127, No Done
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 211, Clonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 211, Tonic-Clonic
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 211, Atonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 211, Myoclonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 211, Focal
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 211, Absence
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 379, Clonic
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 379, Myoclonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 379, Focal
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 19, Not Done
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 29, Clonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 29, Tonic-Clonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 29, Atonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 127, Tonic-Clonic
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 127, Atonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 211, Not Done
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 295, Clonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 295, Tonic-Clonic
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 295, Atonic
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 295, Myoclonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 295, Focal
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 295, Absence
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 295, Not Done
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 379, Tonic-Clonic
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 379, Atonic
|
0 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 379, Absence
|
1 Participants
|
|
Number of Participants Experiencing Spasms and Seizures by Subtype
Day 379, Not Done
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline; Days 29, 43, 71, 127, 211, 295, and 379Population: Safety Analysis Set
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.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Caregiver Global Impression of Change (CGIC)
Day 29, Much Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 29, Very Much Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 29, Not Done
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 43, Very Much Improved
|
2 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 43, Much Improved
|
1 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 43, Slightly Improved
|
3 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 43, No Change
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 43, Slightly Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 43, Much Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 43, Very Much Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 71, Much Improved
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 71, Slightly Improved
|
1 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 71, Much Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 71, Very Much Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 71, Not Done
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 127, Slightly Improved
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 127, No Change
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 127, Slightly Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 127, Much Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 127, Very Much Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 211, Slightly Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 211, Much Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 211, Very Much Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 211, Not Done
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 295, Very Much Improved
|
1 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 295, Much Improved
|
2 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 295, Slightly Improved
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 295, No Change
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 295, Slightly Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 295, Much Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 295, Very Much Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 295, Not Done
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 379, Very Much Improved
|
2 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 379, Much Improved
|
1 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 379, Slightly Improved
|
3 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 379, No Change
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 379, Slightly Worse
|
1 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 379, Much Worse
|
1 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 29, Very Much Improved
|
1 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 29, Much Improved
|
1 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 29, Slightly Improved
|
5 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 29, No Change
|
1 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 29, Slightly Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 43, Not Done
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 71, Very Much Improved
|
3 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 71 No Change
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 71, Slightly Worse
|
1 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 127, Very Much Improved
|
2 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 127, Much Improved
|
2 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 127, Not Done
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 211, Very Much Improved
|
1 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 211, Much Improved
|
2 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 211, Slightly Improved
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 211, No Change
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 379, Very Much Worse
|
0 participants
|
|
Caregiver Global Impression of Change (CGIC)
Day 379, Not Done
|
1 participants
|
SECONDARY outcome
Timeframe: Baseline; Days 29, 43, 71, 127, 211, 295, and 379Population: Safety Analysis Set
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.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Physician Global Impression of Change (PGIC)
Day 29, Very Much Improved
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 29, Much Improved
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 29, Slightly Improved
|
2 participants
|
|
Physician Global Impression of Change (PGIC)
Day 29, No Change
|
4 participants
|
|
Physician Global Impression of Change (PGIC)
Day 29, Slightly Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 29, Much Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 29, Very Much Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 29, Not Done
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 43, Very Much Improved
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 43, Much Improved
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 43, Slightly Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 71, Much Improved
|
2 participants
|
|
Physician Global Impression of Change (PGIC)
Day 71, Slightly Improved
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 127, No Change
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 127, Slightly Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 127, Much Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 127, Very Much Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 127, Not Done
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 295, No Change
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 295, Slightly Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 295, Much Worse
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 43, Slightly Improved
|
4 participants
|
|
Physician Global Impression of Change (PGIC)
Day 43, No Change
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 43, Much Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 43, Very Much Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 43, Not Done
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 71, Very Much Improved
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 71, No Change
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 71, Slightly Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 71, Much Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 71, Very Much Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 71, Not Done
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 127, Very Much Improved
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 127, Much Improved
|
2 participants
|
|
Physician Global Impression of Change (PGIC)
Day 127, Slightly Improved
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 211, Very Much Improved
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 211, Much Improved
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 211, Slightly Improved
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 211, No Change
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 211, Slightly Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 211, Much Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 211, Very Much Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 211, Not Done
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 295, Very Much Improved
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 295, Much Improved
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 295, Slightly Improved
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 295, Very Much Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 295, Not Done
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 379, Very Much Improved
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 379, Much Improved
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 379, Slightly Improved
|
3 participants
|
|
Physician Global Impression of Change (PGIC)
Day 379, No Change
|
2 participants
|
|
Physician Global Impression of Change (PGIC)
Day 379, Slightly Worse
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 379, Much Worse
|
1 participants
|
|
Physician Global Impression of Change (PGIC)
Day 379, Very Much Worse
|
0 participants
|
|
Physician Global Impression of Change (PGIC)
Day 379, Not Done
|
1 participants
|
SECONDARY outcome
Timeframe: Days 29, 43, 127, 211, 295, and 379Population: Safety Analysis Set. Only participants with evaluable data were analyzed.
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.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Number of Responders
Day 43
|
0 Participants
|
|
Number of Responders
Day 127
|
0 Participants
|
|
Number of Responders
Day 211
|
0 Participants
|
|
Number of Responders
Day 295
|
1 Participants
|
|
Number of Responders
Day 379
|
3 Participants
|
|
Number of Responders
Day 29
|
1 Participants
|
SECONDARY outcome
Timeframe: Days 29, 43, 127, 211, 295, and 379Population: Safety Analysis Set. Only participants with evaluable data were analyzed.
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.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Percentage of Responders
Day 29
|
11.1 percentage of participants
|
|
Percentage of Responders
Day 43
|
0 percentage of participants
|
|
Percentage of Responders
Day 127
|
0 percentage of participants
|
|
Percentage of Responders
Day 211
|
0 percentage of participants
|
|
Percentage of Responders
Day 295
|
11.1 percentage of participants
|
|
Percentage of Responders
Day 379
|
33.3 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389Population: Safety Analysis Set. Only participants with evaluable data were analyzed.
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.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Change From Baseline in Height
Baseline (Day 1 of Pilot Study)
|
75.04 centimeters
Standard Deviation 9.557
|
|
Change From Baseline in Height
Day 29, Open-label Extension (OLE)
|
0.85 centimeters
Standard Deviation 0.980
|
|
Change From Baseline in Height
Day 43, OLE
|
0.92 centimeters
Standard Deviation 1.530
|
|
Change From Baseline in Height
Day 71, OLE
|
3.10 centimeters
Standard Deviation 0.652
|
|
Change From Baseline in Height
Day 127, OLE
|
3.38 centimeters
Standard Deviation 1.377
|
|
Change From Baseline in Height
Day 211, OLE
|
5.33 centimeters
Standard Deviation 1.258
|
|
Change From Baseline in Height
Day 295, OLE
|
8.17 centimeters
Standard Deviation 1.528
|
|
Change From Baseline in Height
Day 379, OLE
|
5.31 centimeters
Standard Deviation 4.036
|
|
Change From Baseline in Height
Day 389, OLE
|
3.55 centimeters
Standard Deviation 4.085
|
SECONDARY outcome
Timeframe: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389Population: Safety Analysis Set. Only participants with evaluable data were analyzed.
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.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Change From Baseline in Body Weight.
Baseline (Day 1 of Pilot Study)
|
9.92 kilograms
Standard Deviation 3.245
|
|
Change From Baseline in Body Weight.
Day 29, OLE
|
0.39 kilograms
Standard Deviation 0.309
|
|
Change From Baseline in Body Weight.
Day 43, OLE
|
0.75 kilograms
Standard Deviation 0.809
|
|
Change From Baseline in Body Weight.
Day 71, OLE
|
1.10 kilograms
Standard Deviation 0.430
|
|
Change From Baseline in Body Weight.
Day 127, OLE
|
1.25 kilograms
Standard Deviation 0.420
|
|
Change From Baseline in Body Weight.
Day 211, OLE
|
1.17 kilograms
Standard Deviation 0.503
|
|
Change From Baseline in Body Weight.
Day 295, OLE
|
2.10 kilograms
Standard Deviation 0.889
|
|
Change From Baseline in Body Weight.
Day 379, OLE
|
1.19 kilograms
Standard Deviation 0.862
|
|
Change From Baseline in Body Weight.
Day 389, OLE
|
0.98 kilograms
Standard Deviation 0.637
|
SECONDARY outcome
Timeframe: Baseline (Day 1 of PIlot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389Population: Safety Analysis Set. Only participants with evaluable data were analyzed.
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.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Change From Baseline in Head Circumference
Baseline (Day 1 of Pilot Study)
|
44.71 centimeters
Standard Deviation 2.724
|
|
Change From Baseline in Head Circumference
Day 29, OLE
|
-0.01 centimeters
Standard Deviation 1.229
|
|
Change From Baseline in Head Circumference
Day 43, OLE
|
0.54 centimeters
Standard Deviation 0.288
|
|
Change From Baseline in Head Circumference
Day 127, OLE
|
1.50 centimeters
Standard Deviation 0.500
|
|
Change From Baseline in Head Circumference
Day 211, OLE
|
0.75 centimeters
Standard Deviation 0.354
|
|
Change From Baseline in Head Circumference
Day 295, OLE
|
-0.3 centimeters
Standard Deviation 2.31
|
|
Change From Baseline in Head Circumference
Day 379, OLE
|
1.14 centimeters
Standard Deviation 1.707
|
|
Change From Baseline in Head Circumference
Day 71, OLE
|
0.70 centimeters
Standard Deviation 0.570
|
|
Change From Baseline in Head Circumference
Day 389, OLE
|
0.90 centimeters
Standard Deviation 1.782
|
SECONDARY outcome
Timeframe: Baseline (Day 1 of Pilot Study); Day 211, Day 379Population: Safety Analysis Set. Only participants with evaluable data were analyzed.
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.
Outcome measures
| Measure |
GWP42003-P OS
n=9 Participants
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open-label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Score
Day 211 of OLE
|
6.3 score on a scale
Standard Deviation 3.51
|
|
Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Score
Day 379 of OLE
|
-5.4 score on a scale
Standard Deviation 23.42
|
|
Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Score
Baseline, Day 1 of Pilot Study
|
33.6 score on a scale
Standard Deviation 37.27
|
SECONDARY outcome
Timeframe: Day 16 to Day 379Population: Safety Analysis Set
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 16 to Day 379Population: Safety Analysis Set
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 to Day 379Population: Safety Analysis Set
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 16 to Day 379Population: Safety Analysis Set
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.
Outcome measures
Outcome data not reported
Adverse Events
Cannabidiol OS
Serious events: 2 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cannabidiol OS
n=9 participants at risk
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open Label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Gastrointestinal disorders
Diarrhoea
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Renal and urinary disorders
Urinary retention
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Enterovirus infection
|
22.2%
2/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Pneumonia
|
22.2%
2/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Rhinovirus infection
|
22.2%
2/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Bronchiolitis
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Pneumonia bacterial
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Pneumonia klebsiella
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Urinary tract infection bacterial
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Viral upper respiratory infection
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
Other adverse events
| Measure |
Cannabidiol OS
n=9 participants at risk
Following completion of the Pilot Phase (NCT02953548), participants were eligible to participate in the Open Label Extension Phase. Participants continued on the same dose administered in the Pilot Phase for a maximum of 1 year and completed a 10-day taper after completing the study or withdrawing. Participants were administered GWP42003-P oral solution (OS) orally, twice daily, or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose.
|
|---|---|
|
Vascular disorders
Hypertension
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Vascular disorders
Hypotension
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
General disorders
Pyrexia
|
22.2%
2/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
General disorders
Drug tolerance
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Psychiatric disorders
Irritability
|
22.2%
2/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Psychiatric disorders
Sleep disorder
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Investigations
Blood triglycerides increased
|
22.2%
2/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract congestion
|
33.3%
3/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Respiratory, thoracic and mediastinal disorders
Adenoidal hypertrophy
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Blood and lymphatic system disorders
Anaemia
|
22.2%
2/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Nervous system disorders
Somnolence
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Nervous system disorders
Myoclonic epilepsy
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Nervous system disorders
Petit mal epilepsy
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Eye disorders
Pupils unequal
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Ear and labyrinth disorders
Deafness
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Ear and labyrinth disorders
Middle ear effusion
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Gastrointestinal disorders
Gingival pain
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Skin and subcutaneous tissue disorders
Rash
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Skin and subcutaneous tissue disorders
Skin irritation
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Metabolism and nutrition disorders
Feeding intolerance
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Metabolism and nutrition disorders
Fluid overload
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Urinary tract infection
|
22.2%
2/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Viral infection
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Bronchiolitis
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Ear infection
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Nasopharyngitis
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Otitis media
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Pneumonia
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Sinusitis
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
11.1%
1/9 • From signing of informed consent up to Day 417
Treatment-emergent adverse events (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.
|
Additional Information
Medical Enquires
GW Research Ltd
Phone: +44 01223 238170; 18778862810
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60