A safety and efficacy study of GWP42003-P oral solution as adjunctive treatment for Japanese children and adults with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex

JRCT ID: jRCT2031220041

Registered date:25/04/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Lennox-Gastaut syndrome, Dravet syndrome, tuberous sclerosis complex
• Date of first enrollment: 31/10/2022
• Target sample size: 84
• Study type: Interventional

Intervention(s)

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 administered twice daily (b.i.d) orally
Treatment Duration:

Part A: Treatment period will consist of an initial 2-week titration period followed by a 14-week maintenance period.

Part B: 52-week treatment period or until market authorization is granted for GWP42003-P oral solution.

Outcome(s)

Primary Outcome

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

Key inclusion & exclusion criteria

• Age minimum: >= 1age old
• Age maximum: <= 65age old
• Gender: Both

Include 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 <= 65 years of age at time of signing the informed consent form.
4.Taking 1 or more antiepileptic drugs (AEDs) at a stable dose for at least 4 weeks prior to screening.
5.Complete at least 25 days of seizure diary entries during the 28 days of the baseline period.

Exclude 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.

Related Information

• Primary Sponsor: Yamamoto Tomoyuki