Lennox-Gastaut syndrome, Dravet syndrome, tuberous sclerosis complex
Conditions
Interventions
Intervention Drug: GWP42003-P oral solution
For DS and LGS, targeted maintenance dosage of 10mg/kg administered twice daily (b.i.d.) orally.
For TSC, targeted maintenance dosage of 12.5 mg/kg admini
Sponsors
Yamamoto Tomoyuki
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Participant must be Japanese. 2.Participan must fulfill conditions-specific criteria for Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC). 3.Male or female >= 1 to = 1 to =< 18 years of age, has been on a stable dose of clobazam for at least 4 weeks prior to screening.
Exclusion criteria
Exclusion criteria: 1. Etiology of seizures is a progressive neurologic disease. Participants with TSC will not be excluded from study participation unless there is a progressive tumor. 2. Has had an anoxic episode requiring resuscitation within 6 months of screening. 3. Has clinically significant medical conditions other than epilepsy. 4. Has undergone surgery for epilepsy in the 26 weeks prior to screening. 5. Has clinically significant abnormal laboratory values, in the investigator's opinion, at screening or enrollment. 6. Has a history of pseudo-seizures. 7. Has clinically significant unstable medical conditions other than epilepsy. 8. Any history of suicidal behaviour or any suicidal ideation of type 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS)/Children's C-SSRS in the last month, or at screening. This criterion applies only to participants >= 4 years of age. 9. Known or suspected hypersensitivity to cannabinoids, or any of the excipients of study intervention, such as sesame oil. 10. In the opinion of the investigator, the participant has clinically significant abnormalities in the 12-lead electrocardiogram (ECG) measured at screening or enrollment, or any concurrent cardiovascular conditions, which will interfere with the ability to read their ECGs. 11.Cohort 2 Only Participant has current or recent stiripentol use within 28 days prior to screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A (Short-term efficacy and safety) Primary Safety 1. Treatment-emergent adverse events 2. Clinical laboratory parameters 3. Change in children's Columbia-Suicide Severity Rating Scale (C-SSRS) ideation score 4. Change in the number of suicide attempts in the children's Columbia-Suicide Severity Rating Scale (C-SSRS) 5. Vital signs measurements 6. 12-lead electrocardiogram (ECG) measurements (heart rate and PR, QRS, and QT intervals) 7. Effects of menstruation cycles in female participants Primary Efficacy 1. Percentage change from baseline in indication-associated seizure frequency during treatment period for all indications combined Part B (Long-term safety) 1. Treatment-emergent adverse events 2. Clinical laboratory parameters 3. Change in children's C-SSRS ideation score 4. Change in the number of suicide attempts in the children's C-SSRS 5. Vital signs measurements 6. 12-lead ECG measurements (heart rate and PR, QRS, and QT intervals) 7. Effects of menstruation cycles in female participants | — |
Contacts
Public ContactTomoyuki Yamamoto
Jazz Pharmaceuticals Japan K.K
Outcome results
None listed