Trial Outcomes & Findings for Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures (NCT NCT04485104)

NCT ID: NCT04485104

Last Updated: 2025-10-31

Results Overview

• Recruitment status: TERMINATED
• Study phase: PHASE3
• Target enrollment: 3 participants
• Primary outcome timeframe: From start of treatment to the post-treatment safety follow-up visit, up to 62 weeks
• Results posted on: 2025-10-31

Participant Flow

A total of 3 participants who met all inclusion criteria and no exclusion criteria were enrolled in the study at 3 sites in the United States.

The study duration will be up to approximately 62 weeks, including a 4-week screening/baseline period, a 52-week dose optimisation treatment period (which includes a fixed 2-week titration period followed by flexible dose optimisation), a 10-day taper period, and a safety follow-up period (4 weeks after the end-of-taper visit).

Participant milestones

Measure	Tuberous Sclerosis Complex Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Overall Study STARTED	2	1
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	2	1

Reasons for withdrawal

Measure	Tuberous Sclerosis Complex Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Overall Study Physician Decision	2	0
Overall Study Decision by sponsor	0	1

Baseline Characteristics

Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures

Baseline characteristics by cohort

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Total n=3 Participants Total of all reporting groups
Age, Categorical <=18 years	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: From start of treatment to the post-treatment safety follow-up visit, up to 62 weeks

Population: Treatment-emergent adverse events (TEAEs) were assessed in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	2 participants	1 participants

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: Blood pressure was assessed in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Mean Change From Baseline in Blood Pressure Systolic blood pressure	-3.0 mmHg Standard Deviation 9.90	11.0 mmHg Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Mean Change From Baseline in Blood Pressure Diastolic blood pressure	13.5 mmHg Standard Deviation 19.09	-2.0 mmHg Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: Pulse rate was assessed in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Mean Change From Baseline in Pulse Rate	-11.0 beats per minute Standard Deviation 1.41	11.0 beats per minute Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: Respiratory rate was assessed in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Mean Change From Baseline in Respiratory Rate	-10.0 breaths/minute Standard Deviation 16.97	0 breaths/minute Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: Body temperature was assessed in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Mean Change From Baseline in Body Temperature	-0.05 degrees Celsius Standard Deviation 0.354	-0.10 degrees Celsius Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: Height was assessed in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Mean Change From Baseline in Height	1.95 centimeters Standard Deviation 1.344	4.20 centimeters Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: Body weight was assessed in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Mean Change From Baseline in Body Weight	0.15 kilogram Standard Deviation 0.354	0.60 kilogram Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: Heart rate was assessed in participants with available data in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=1 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Mean Change From Baseline in Heart Rate	-10.0 beats/minute Standard Deviation NA Standard deviation cannot be calculated for 1 person.	8.0 beats/minute Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: RR interval was assessed in participants with available data in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=1 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Mean Change From Baseline in RR Interval	48.0 millisecond Standard Deviation NA Standard deviation cannot be calculated for 1 person.	-45.0 millisecond Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: PR interval was assessed in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Mean Change From Baseline in PR Interval	11.0 millisecond Standard Deviation 12.73	-2.0 millisecond Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: QRS duration was assessed in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Mean Change From Baseline in QRS Duration	3.0 millisecond Standard Deviation 1.41	6.0 millisecond Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: QT interval was assessed in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Mean Change From Baseline in QT Interval	0 millisecond Standard Deviation 8.49	0 millisecond Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: QTcB and QTcF were assessed in participants with available data in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=1 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Mean Change From Baseline in QTcB and QTcF QTcB	-10.0 millisecond Standard Deviation NA Standard deviation cannot be calculated for 1 person.	16.0 millisecond Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Mean Change From Baseline in QTcB and QTcF QTcF	-4.0 millisecond Standard Deviation NA Standard deviation cannot be calculated for 1 person.	9.0 millisecond Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: Clinically significant change in laboratory parameters were assessed in participants with available data in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Number of Participants With a Clinically Significant Change in Laboratory Parameters Alanine aminotransferase	0 participants	1 participants
Number of Participants With a Clinically Significant Change in Laboratory Parameters Aspartate aminotransferase	0 participants	1 participants
Number of Participants With a Clinically Significant Change in Laboratory Parameters Gamma Glutamyltransferase	0 participants	1 participants

PRIMARY outcome

Timeframe: From baseline up to the end of taper follow-up visit (Visit 20), up to 62 weeks

Population: New types of seizures were assessed in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Number of Participants With Emergence of New Types of Seizures	1 participants	0 participants

PRIMARY outcome

Timeframe: Predose, 3 hours and 6 hours post dose at End of Treatment (Week 52)

Population: Pharmacokinetics were assessed and reported for participants when samples were collected. For both participants with Tuberous Sclerosis Complex, it was not possible to collect PK samples at 6hr post dose.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Plasma Concentrations of GWP42003-P and Its Major Metabolites CBD (pre-dose)	55.3 ng/mL Standard Deviation 46.3	99.8 ng/mL Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Plasma Concentrations of GWP42003-P and Its Major Metabolites CBD (3-hour post-dose)	456 ng/mL Standard Deviation NA Standard deviation cannot be calculated for 1 person.	427 ng/mL Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Plasma Concentrations of GWP42003-P and Its Major Metabolites CBD (6-hour post-dose)	—	149 ng/mL Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Plasma Concentrations of GWP42003-P and Its Major Metabolites 7-OH-CBD (pre-dose)	37.8 ng/mL Standard Deviation 29.7	123 ng/mL Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Plasma Concentrations of GWP42003-P and Its Major Metabolites 7-OH-CBD (3-hour post-dose)	161 ng/mL Standard Deviation NA Standard deviation cannot be calculated for 1 person.	580 ng/mL Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Plasma Concentrations of GWP42003-P and Its Major Metabolites 7-OH-CBD (6-hour post-dose)	—	292 ng/mL Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Plasma Concentrations of GWP42003-P and Its Major Metabolites 7-COOH-CBD (pre-dose)	2790 ng/mL Standard Deviation 884	7460 ng/mL Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Plasma Concentrations of GWP42003-P and Its Major Metabolites 7-COOH-CBD (3-hour post-dose)	4870 ng/mL Standard Deviation NA Standard deviation cannot be calculated for 1 person.	10700 ng/mL Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Plasma Concentrations of GWP42003-P and Its Major Metabolites 7-COOH-CBD (6-hour post-dose)	—	9680 ng/mL Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: Day 1 up to Taper Period, up to Week 52

Population: Indication-specific total countable seizures where assessed in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 30-57: <= -50% to > -75% change (reduction)	0 participants	1 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 30-57: <= -75% (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 142-169: <= -75% (reduction)	1 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 170-197: > 25% (increase)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 58-85: > -25% to 0% (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 58-85: <= -25% to > -50% change (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 58-85: <= -50% to > -75% change (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 58-85: <= -75% (reduction)	0 participants	1 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 86-113: > 25% (increase)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 86-113: >= 0% to <=25% (increase)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 86-113: > -25% to 0% (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 86-113: <= -25% to > -50% change (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 86-113: <= -50% to > -75% change (reduction)	0 participants	1 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 86-113: <= -75% (reduction)	1 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 114-141: > 25% (increase)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 114-141: >= 0% to <=25% (increase)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 114-141: > -25% to 0% (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 114-141: <= -25% to > -50% change (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 114-141: <= -50% to > -75% change (reduction)	0 participants	1 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 114-141: <= -75% (reduction)	1 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 142-169: > 25% (increase)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 142-169: >= 0% to <=25% (increase)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 142-169: > -25% to 0% (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 142-169: <= -25% to > -50% change (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 142-169: <= -50% to > -75% change (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 170-197: >= 0% to <=25% (increase)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 170-197: > -25% to 0% (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 170-197: <= -25% to > -50% change (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 170-197: <= -50% to > -75% change (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 170-197: <= -75% (reduction)	1 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Taper Period: > 25% (increase)	1 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Taper Period: >= 0% to <=25% (increase)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Taper Period: > -25% to 0% (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Taper Period: <= -25% to > -50% change (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Taper Period: <= -50% to > -75% change (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Taper Period: <= -75% (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 1-29: > 25% (increase)	2 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 1-29: >= 0% to <=25% (increase)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 1-29: > -25% to 0% (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 1-29: <= -25% to > -50% change (reduction)	0 participants	1 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 1-29: <= -50% to > -75% change (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 1-29: <= -75% (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 30-57: > 25% (increase)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 30-57: >= 0% to <=25% (increase)	1 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 30-57: > -25% to 0% (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 30-57: <= -25% to > -50% change (reduction)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 58-85: > 25% (increase)	0 participants	0 participants
Number of Participants Based on Percentage Change From Baseline in Indication-Specific Total Countable Seizures as Recorded by Caregivers Day 58-85: >= 0% to <=25% (increase)	1 participants	0 participants

PRIMARY outcome

Timeframe: At Day 365 (EOT)

Population: CGI-S was assessed and reported for the participant with Dravet Syndrome. As both participants with Tuberous Sclerosis Complex consented under a previous protocol version that did not include CGI S, this assessment was not collected for these participants.

The CGIC/S is a comprehensive neurodevelopmental assessment that covers the following domains: sensory, motor, cognition, emotional/behavioral health, communication, social, and adaptive functioning. This assessment is a 2-question survey per domain to be completed by the clinician. Individual domain scores are reported.

The severity of impairment in each domain is rated by the clinician in a scale of 1 through 7 where 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill. Higher scores indicate poor clinical outcome.

Outcome measures

Measure	Tuberous Sclerosis Complex Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Clinician Global Impression of Severity (CGI/S) Score Cognition	—	4.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Clinician Global Impression of Severity (CGI/S) Score Emotional/Behavioral	—	3.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Clinician Global Impression of Severity (CGI/S) Score Communication	—	4.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Clinician Global Impression of Severity (CGI/S) Score Sensory	—	3.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Clinician Global Impression of Severity (CGI/S) Score Motor	—	3.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Clinician Global Impression of Severity (CGI/S) Score Social	—	1.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Clinician Global Impression of Severity (CGI/S) Score Adaptive Functioning	—	1.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.

PRIMARY outcome

Timeframe: At Day 365 (EOT)

Population: CGI-C was assessed and reported for the participant with Dravet Syndrome. As both participants with Tuberous Sclerosis Complex consented under a previous protocol version that did not include CGI-C, this assessment was not collected for these participants.

The CGI/C is a comprehensive neurodevelopmental assessment that covers the following domains: sensory, motor, cognition, emotional/behavioral health, communication, social, and adaptive functioning. This assessment is a 2-question survey per domain to be completed by the clinician. Individual domain scores are reported.

The severity of impairment in each domain is rated by the clinician in a scale of 1 through 7 where 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill. Higher scores indicate poor clinical outcome.

Outcome measures

Measure	Tuberous Sclerosis Complex Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Clinician Global Impression of Change (CGI/C) Score Sensory	—	3.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Clinician Global Impression of Change (CGI/C) Score Motor	—	3.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Clinician Global Impression of Change (CGI/C) Score Cognition	—	3.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Clinician Global Impression of Change (CGI/C) Score Emotional/Behavioral	—	3.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Clinician Global Impression of Change (CGI/C) Score Communication	—	4.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Clinician Global Impression of Change (CGI/C) Score Social	—	3.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Clinician Global Impression of Change (CGI/C) Score Adaptive Functioning	—	4.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated for 1 person.

SECONDARY outcome

Timeframe: Day 1 up to the taper period, up to Week 52

Population: Treatment response was assessed and reported for participants with available data. The participant with Dravet Syndrome was not assessed at the last 3 timepoints due to early withdrawal from the study.

Treatment Responders are defined as participants with ≥ 50% reduction from baseline in caregiver-reported total countable seizures

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Number of Treatment Responders Day 86-113	1 participants	1 participants
Number of Treatment Responders Day 114-141	1 participants	1 participants
Number of Treatment Responders Day 142-169	1 participants	—
Number of Treatment Responders Day 170-197	1 participants	—
Number of Treatment Responders Day 1-29	0 participants	0 participants
Number of Treatment Responders Day 30-57	0 participants	1 participants
Number of Treatment Responders Day 58-85	0 participants	1 participants
Number of Treatment Responders Taper Period	0 participants	—

SECONDARY outcome

Timeframe: Week 12, and every 4 weeks thereafter, up to date of withdrawal or Week 24, whichever occurs first

Population: Seizure free status is reported for the 2 participants that remained on the study for \> 12 weeks, and data are reported up to the timepoint prior to their withdrawal from the study. One participant with Tuberous Sclerosis Complex withdrew from the study prior to week 12 so their seizure free status could not be assessed.

Outcome measures

Measure	Tuberous Sclerosis Complex n=1 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Number of Participants Who Achieved Seizure-Free Status Week 12	0 participants	0 participants
Number of Participants Who Achieved Seizure-Free Status Week 16	0 participants	0 participants
Number of Participants Who Achieved Seizure-Free Status Week 20	0 participants	0 participants
Number of Participants Who Achieved Seizure-Free Status Week 24	0 participants	—

SECONDARY outcome

Timeframe: Week 12, and every 4 weeks thereafter, up to date of withdrawal or Week 24, whichever occurs first

Population: Percentage of participants still receiving GWP42003-P is reported to the point at which subjects terminated their treatment and withdrew from the study. One participant with Tuberous Sclerosis Complex withdrew from the study prior to week 12. One participant with Tuberous Sclerosis Complex withdrew following the Day 170-197 timepoint. The participant with Dravet Syndrome withdrew following the Day 142-169 timepoint.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Percentage of Participants Still Receiving GWP42003-P Week 20	50 percentage of participants	100 percentage of participants
Percentage of Participants Still Receiving GWP42003-P Week 12	50 percentage of participants	100 percentage of participants
Percentage of Participants Still Receiving GWP42003-P Week 16	50 percentage of participants	100 percentage of participants
Percentage of Participants Still Receiving GWP42003-P Week 24	50 percentage of participants	0 percentage of participants

OTHER_PRE_SPECIFIED outcome

Timeframe: At Day 365 (EOT)

Population: ITQOL-47 score was assessed in participants with available data in the Safety Analysis Set.

The Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) was developed for use in infants and toddlers from 12-months-to-5 years of age and assesses levels of health and well-being. The caregiver will complete the assessment on an electronic device. For each concept, item responses are scored, summed, and transformed on a scale from 0 (worst health) to 100 (best health). Higher scores indicate better clinical outcome.

Outcome measures

Measure	Tuberous Sclerosis Complex n=1 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score General Health Perceptions Score	-20.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.	-20.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score Bodily Pain/Discomfort Zone	0 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.	12.50 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score Overall Health Score	25.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.	0 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score Physical Abilities Score	11.10 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.	-5.60 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score Temperament and Moods Score	-8.30 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.	4.20 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score Combined Behavior Scale	17.90 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.	0 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score Change in Health Score	0 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.	1.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score Family Cohesion Score	-55.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.	15.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score Growth and Development Score	5.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.	-15.0 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score Parental Impact-Emotional Score	-25.00 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.	12.50 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.
Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) Score Parental Impact-Time Score	0 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.	8.30 score on a scale Standard Deviation NA Standard deviation cannot be calculated on 1 person.

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 1 up to Taper Period, up to Week 52

Population: Seizure frequency was assessed in participants with available data in the Safety Analysis Set.

Outcome measures

Measure	Tuberous Sclerosis Complex n=2 Participants Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 Participants Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Percentage Change From Baseline in Indication-Specific Seizure Frequency As Recorded by Caregivers Taper Period	71.90 seizure frequency Standard Deviation NA Standard deviation cannot be calculated for 1 person.	—
Percentage Change From Baseline in Indication-Specific Seizure Frequency As Recorded by Caregivers Day 1-29	56.05 seizure frequency Standard Deviation 26.23	-67.80 seizure frequency Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Percentage Change From Baseline in Indication-Specific Seizure Frequency As Recorded by Caregivers Day 30-57	24.80 seizure frequency Standard Deviation NA Standard deviation cannot be calculated for 1 person.	-89.50 seizure frequency Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Percentage Change From Baseline in Indication-Specific Seizure Frequency As Recorded by Caregivers Day 58-85	2.70 seizure frequency Standard Deviation NA Standard deviation cannot be calculated for 1 person.	-94.70 seizure frequency Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Percentage Change From Baseline in Indication-Specific Seizure Frequency As Recorded by Caregivers Day 86-113	-95.20 seizure frequency Standard Deviation NA Standard deviation cannot be calculated for 1 person.	-80.20 seizure frequency Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Percentage Change From Baseline in Indication-Specific Seizure Frequency As Recorded by Caregivers Day 114-141	-97.60 seizure frequency Standard Deviation NA Standard deviation cannot be calculated for 1 person.	-85.80 seizure frequency Standard Deviation NA Standard deviation cannot be calculated for 1 person.
Percentage Change From Baseline in Indication-Specific Seizure Frequency As Recorded by Caregivers Day 142-169	-97.80 seizure frequency Standard Deviation NA Standard deviation cannot be calculated for 1 person.	—
Percentage Change From Baseline in Indication-Specific Seizure Frequency As Recorded by Caregivers Day 170-197	-88.10 seizure frequency Standard Deviation NA Standard deviation cannot be calculated for 1 person.	—

Adverse Events

Tuberous Sclerosis Complex

Serious events: 2 serious events

Other events: 1 other events

Deaths: 0 deaths

Dravet Syndrome

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Measure	Tuberous Sclerosis Complex n=2 participants at risk Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 participants at risk Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Infections and infestations Respiratory syncytial virus bronchiolitis	50.0% 1/2 • Number of events 2 • Adverse events were collected from baseline up to 62 weeks.	0.00% 0/1 • Adverse events were collected from baseline up to 62 weeks.
Nervous system disorders Change in seizure presentation	100.0% 2/2 • Number of events 5 • Adverse events were collected from baseline up to 62 weeks.	0.00% 0/1 • Adverse events were collected from baseline up to 62 weeks.

Other adverse events

Measure	Tuberous Sclerosis Complex n=2 participants at risk Participants with Tuberous Sclerosis Complex who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.	Dravet Syndrome n=1 participants at risk Participants with Dravet Syndrome who received GWP42003-P orally twice daily (b.i.d.) on a titration schedule, with a starting dose of 5 mg/kg/day (2.5 mg/kg b.i.d.) on Day 1, then 10 mg/kg/day (5 mg/kg b.i.d.) on Day 8, followed by a flexible dose optimization from Day 15 to Week 52 based on the clinical judgment of the investigator.
Gastrointestinal disorders Teething	50.0% 1/2 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.	0.00% 0/1 • Adverse events were collected from baseline up to 62 weeks.
General disorders Fatigue	0.00% 0/2 • Adverse events were collected from baseline up to 62 weeks.	100.0% 1/1 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.
Infections and infestations Bronchitis	50.0% 1/2 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.	0.00% 0/1 • Adverse events were collected from baseline up to 62 weeks.
Infections and infestations Ear infection	50.0% 1/2 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.	0.00% 0/1 • Adverse events were collected from baseline up to 62 weeks.
Infections and infestations Pneumonia	50.0% 1/2 • Number of events 3 • Adverse events were collected from baseline up to 62 weeks.	0.00% 0/1 • Adverse events were collected from baseline up to 62 weeks.
Infections and infestations Rhinovirus infection	0.00% 0/2 • Adverse events were collected from baseline up to 62 weeks.	100.0% 1/1 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.
Infections and infestations Upper respiratory tract infection	50.0% 1/2 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.	0.00% 0/1 • Adverse events were collected from baseline up to 62 weeks.
Injury, poisoning and procedural complications Contusion	0.00% 0/2 • Adverse events were collected from baseline up to 62 weeks.	100.0% 1/1 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.
Investigations Alanine aminotransferase increased	0.00% 0/2 • Adverse events were collected from baseline up to 62 weeks.	100.0% 1/1 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.
Investigations Aspartate aminotransferase increased	0.00% 0/2 • Adverse events were collected from baseline up to 62 weeks.	100.0% 1/1 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.
Investigations Gamma-glutamyltransferase increased	0.00% 0/2 • Adverse events were collected from baseline up to 62 weeks.	100.0% 1/1 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.
Nervous system disorders Lethargy	50.0% 1/2 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.	0.00% 0/1 • Adverse events were collected from baseline up to 62 weeks.
Psychiatric disorders Irritability	0.00% 0/2 • Adverse events were collected from baseline up to 62 weeks.	100.0% 1/1 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.
Renal and urinary disorders Proteinuria	0.00% 0/2 • Adverse events were collected from baseline up to 62 weeks.	100.0% 1/1 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/2 • Adverse events were collected from baseline up to 62 weeks.	100.0% 1/1 • Number of events 2 • Adverse events were collected from baseline up to 62 weeks.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	0.00% 0/2 • Adverse events were collected from baseline up to 62 weeks.	100.0% 1/1 • Number of events 1 • Adverse events were collected from baseline up to 62 weeks.

Additional Information

Director Clinical Trial Disclosure & Transparency

Jazz Pharmaceuticals, Inc.

Phone: +1 215-832-3750

Email: ClinicalTrialDisclosure@JazzPharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place