A Randomized, Double-blind, Placebo-controlled Trial to Investigate the Efficacy and Safety of Cannabidiol (CBD; GWP42003-P) in Infants With Infantile Spasms Following an Initial Open-label Pilot Study
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 9
- 试验地点
- 7
- 主要终点
- Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
研究概览
简要总结
This trial consists of 3 parts: a pilot safety phase, a pivotal randomized controlled phase, and an open-label extension phase. The open-label extension phase only will be described in this record. All participants will receive GWP42003-P.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
盲法说明
Open-label
入排标准
- 年龄范围
- 1 Month 至 24 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant is diagnosed with IS and has failed to respond adequately following treatment with 1 or more approved IS therapies.
排除标准
- •Participant is currently taking or has taken clobazam or any mammalian target of rapamycin (mTOR) inhibitor within the 2 weeks prior to the screening visit.
- •Participant has a QT interval, corrected for heart rate with Bazett's formula (QTcB), of 460 msec or greater on ECG.
- •Participant's caregiver is currently giving or has given recreational or medicinal cannabis, or synthetic cannabinoid-based medications, within the 1 month prior to the screening visit.
- •Participant's caregiver is unwilling to abstain from giving the participant (including the participant's mother abstaining themselves, if breastfeeding)recreational or medicinal cannabis, or synthetic cannabinoid-based medications (other than the study drug) during the trial.
- •Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the study drug, such as sesame oil.
- •Participant has significantly impaired hepatic function at the screening visit.
- •Participant has received an investigational medicinal product as part of a clinical trial within a minimum of 5 half-lives prior to the screening visit.
研究组 & 干预措施
GWP42003-P
Administered orally, up to the target dose recommended by the data safety monitoring committee.
Participants continue at the target dose, or the highest tolerated dose up to the target dose, for a total of 12 months' treatment.
干预措施: GWP42003-P (Drug)
结局指标
主要结局
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
时间窗: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389
Number of Participants With Clinically Significant Vital Sign Findings
时间窗: From signing of informed consent up to Day 389
Clinical significance was determined by the investigator.
Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs)
时间窗: From signing of informed consent up to Day 417
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.
Number of Participants With Clinically Significant Physical Examination Findings
时间窗: From signing of informed consent up to Day 389
Clinical significance was determined by the investigator.
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
时间窗: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389
Number of Participants With Any Clinically Relevant Urinalysis Parameter Value
时间窗: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389
Clinical relevance was determined by the investigator.
Number of Participants With Clinically Significant Electrocardiogram Findings
时间窗: From signing of informed consent up to Day 389
Clinical significance was determined by the investigator.
次要结局
- Number of Participants Free of Clinical Spasms(Days 29, 43, 127, 211, 295, and 379)
- Percentage of Participants With a Resolution of Hypsarrhythmia(Days 29, 43, 127, 211, 295, and 379)
- Number of Participants Experiencing Spasms and Seizures by Subtype(Days 19, 29, 127, 211, 295, and 379)
- Change From Baseline in Height(Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389)
- Number of Participants With a Resolution of Hypsarrhythmia(Days 29, 43, 127, 211, 295, and 379)
- Number of Responders(Days 29, 43, 127, 211, 295, and 379)
- Percentage of Participants Free of Clinical Spasms(Days 29, 43, 127, 211, 295, and 379)
- Physician Global Impression of Change (PGIC)(Baseline; Days 29, 43, 71, 127, 211, 295, and 379)
- Percentage of Responders(Days 29, 43, 127, 211, 295, and 379)
- Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Score(Baseline (Day 1 of Pilot Study); Day 211, Day 379)
- Number of Participants With Relapse of Spasms(Day 16 to Day 379)
- Change From Baseline in Head Circumference(Baseline (Day 1 of PIlot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389)
- Caregiver Global Impression of Change (CGIC)(Baseline; Days 29, 43, 71, 127, 211, 295, and 379)
- Change From Baseline in Body Weight.(Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389)
- Average Time to Cessation of Spasms(Day 1 to Day 379)
- Average Time to Relapse(Day 16 to Day 379)
- Percentage of Participants With Relapse of Spasms(Day 16 to Day 379)