A Double-blind, Randomized, Placebo-controlled Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P, CBD) as Add-on Therapy in Patients With Tuberous Sclerosis Complex Who Experience Inadequately-controlled Seizures
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 224
- 试验地点
- 44
- 主要终点
- Percent Change From Baseline in the Number of Tuberous Sclerosis Complex (TSC)-Associated Seizures During the Treatment Period (Maintenance and Titration)
研究概览
简要总结
This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The blinded phase only will be described in this record. Participants will receive 1 of 2 doses of GWP42003-P or matching placebo. The primary clinical hypothesis is that there will be a difference between GWP42003-P and placebo in their effect on seizure frequency.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 1 Year 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant has a well-documented clinical history of epilepsy.
- •Participant has a clinical diagnosis of Tuberous Sclerosis Complex (TSC) according to the criteria agreed by the 2012 International TSC Consensus Conference.
- •All medications or interventions for epilepsy (including ketogenic diet and any neurostimulation devices for epilepsy) must have been stable for 1 month prior to screening and the participant is willing to maintain a stable regimen throughout the trial.
排除标准
- •Participant has a history of pseudo-seizures.
- •Participant has clinically significant unstable medical conditions other than epilepsy.
- •Participant has an illness in the 4 weeks prior to screening or randomization, other than epilepsy, which in the opinion of the investigator could affect seizure frequency.
- •Participant has undergone general anesthetic in the 4 weeks prior to screening or randomization.
- •Participant has undergone surgery for epilepsy in the 6 months prior to screening.
- •Participant is being considered for epilepsy surgery or any procedure involving general anesthesia.
- •Participant has been taking felbamate for less than 1 year prior to screening.
- •Participant is taking an oral mTOR inhibitor.
- •Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the Investigational Medicinal Product (IMP), such as sesame oil.
- •Participant has any history of suicidal behavior or any suicidal ideation of type 4 or 5 on the C-SSRS in the last month or at screening.
- •Participant is currently using or has in the past used recreational or medicinal cannabis, or cannabinoid-based medications, within the 3 months prior to screening and is unwilling to abstain for the duration for the study.
- •Participant has tumor growth which, in the opinion of the Investigator, could affect the primary endpoint.
- •Participant has significantly impaired hepatic function at the screening or randomization visit
- •Participant has received an IMP within the 12 weeks prior to the screening visit.
研究组 & 干预措施
25 mg/kg/day GWP42003-P
100 mg/mL GWP42003-P oral solution taken twice daily (morning and evening).
干预措施: GWP42003-P (Drug)
50 mg/kg/day GWP42003-P
100 mg/mL GWP42003-P oral solution taken twice daily (morning and evening).
干预措施: GWP42003-P (Drug)
Placebo
Placebo oral solution matching 100 mg/mL GWP42003-P.
干预措施: Placebo (Drug)
结局指标
主要结局
Percent Change From Baseline in the Number of Tuberous Sclerosis Complex (TSC)-Associated Seizures During the Treatment Period (Maintenance and Titration)
时间窗: Baseline; up to Week 16
TSC-associated seizures included: focal motor seizures without impairment of consciousness or awareness (Type 1 focal motor); focal seizures with impairment of consciousness or awareness (Type 2 focal); focal seizures evolving to bilateral generalized convulsive seizures (Type 3 focal); and tonic-clonic, tonic, clonic, or atonic seizures that are countable. Percent change from Baseline was calculated as the (post-Baseline value minus the Baseline value) divided by the Baseline value x 100.
次要结局
- Percent Change From Baseline in Total Seizures During the Treatment Period (Maintenance and Titration)(Baseline; up to Week 16)
- Number of Participants Considered Treatment Responders During the Treatment Period (Maintenance and Titration)(Baseline; up to Week 16)
- Change From Baseline in the Caregiver Global Impression of Change (CGIC) or Participant Global Impression of Change (PGIC) Score at the Participant's Last Visit(Baseline; up to Week 16)
- Number of Participants With Any Severe Treatment-emergent Adverse Event (TEAE)(up to approximately Week 22)