A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution as Adjunctive Therapy With Vigabatrin as Initial Therapy in Patients With Infantile Spasms
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 2
- 试验地点
- 5
- 主要终点
- Percentage of Participants Considered Complete Responders
研究概览
简要总结
The primary purpose of this study was to evaluate the efficacy, safety, and tolerability of Cannabidiol Oral Solution (CBD) as adjunctive therapy with vigabatrin as initial therapy, compared to vigabatrin alone in the treatment of infants newly diagnosed with Infantile Spasms (IS).
详细描述
This was a randomized, double-blind, placebo-controlled, parallel-group study in which participants were randomized in a 1:1 ratio to 1 of 2 treatment groups. During the Initial Treatment Period, participants received either vigabatrin plus CBD or vigabatrin plus matching placebo and were dosed approximately every 12 hours, with a meal. This study was comprised of five periods: Screening, Initial Treatment, Extended Treatment, Taper, and Follow up Periods, with a maximum duration of approximately 140 days.
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 Month 至 24 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Parent(s)/caregiver(s) fully comprehends and signs the informed consent form, understands all study procedures, and can communicate satisfactorily with the Investigator and study coordinator, in accordance with applicable laws, regulations, and local requirements.
- •Clinical diagnosis of Infantile Spasms, confirmed by video-EEG (including at least one cluster of electroclinical spasms [≥3 in any 10-minute epoch] and hypsarrythmia) obtained during the Screening Period and read by a central reader.
- •General good health (defined as the absence of any clinically relevant abnormalities as determined by the Investigator) based on physical and neurological examinations, medical history, and clinical laboratory values completed during the Screening Visit).
- •In the opinion of the investigator, the parent(s)/caregiver(s) is (are) willing and able to comply with the study procedures and visit schedules.
排除标准
- •Is considered by the investigator, for any reason (including, but not limited to, the risks described as precautions, warnings, and contraindications in the current version of the Investigator's Brochure for Cannabidiol Oral Solution) to be an unsuitable candidate to receive the study drug.
- •Known or suspected allergy to cannabidiol.
- •History of an allergic reaction or a known or suspected sensitivity to any substance that is contained in the investigational product formulation.
- •Use of any cannabidiol/cannabis product within 30 days of study entry.
- •Participant is diagnosed or suspected of having tuberous sclerosis.
- •Participant has received treatment with either vigabatrin, ACTH, or high-dose steroids previously.
- •Previous or concomitant therapy with felbamate, clobazam, valproic acid, or the ketogenic diet.
- •Participant currently on any disallowed CYP3A4-related medication (phenytoin, fluvoxamine, carbamazepine, and St. John's Wort).
- •Previously received any investigational drug or device or investigational therapy within 30 days before Screening.
- •Clinically significant abnormal laboratory values, including: liver function tests (LFTs) such as albumin, direct bilirubin, total bilirubin, aspartate aminotransferase (AST), and alanine aminotransferase (ALT) ≥3 times the upper limit of normal (ULN). The investigator may deem the participant eligible if he or she judges the laboratory values to be not clinically significant.
研究组 & 干预措施
CBD with Vigabatrin
Participants received up to 40 milligrams per kilogram per day (mg/kg/day) divided twice daily (BID) of CBD with food. Participants also received up to 150 mg/kg/day BID of vigabatrin with food.
干预措施: Cannabidiol Oral Solution (Drug)
CBD with Vigabatrin
Participants received up to 40 milligrams per kilogram per day (mg/kg/day) divided twice daily (BID) of CBD with food. Participants also received up to 150 mg/kg/day BID of vigabatrin with food.
干预措施: Vigabatrin (Drug)
Placebo with Vigabatrin
Participants received a matching placebo to CBD with food, and also received up to 150 mg/kg/day BID of vigabatrin with food.
干预措施: Placebo (Drug)
Placebo with Vigabatrin
Participants received a matching placebo to CBD with food, and also received up to 150 mg/kg/day BID of vigabatrin with food.
干预措施: Vigabatrin (Drug)
结局指标
主要结局
Percentage of Participants Considered Complete Responders
时间窗: Up to Day 15
Complete response is defined as complete resolution of spasms and hypsarrhythmia confirmed by 24-hour video-electroencephalogram (EEG).
次要结局
- Percentage of Participants With Resolution of Hypsarrhythmia(Up to Day 15)
- Investigator Impression of Efficacy and Tolerability of Study Drug Clinical Global Impression- Global Improvement (CGI-I)(Day 15)
- Percentage of Participants With Increase in Number of Spasm-Free Days Between Day 1 and Day 15(Up to Day 15)
- Percentage of Participants With Complete Response During the Initial Treatment Period Who Relapse During the Extended Treatment Period(Up to Day 75)
- Percentage of Participants With Resolution of Infantile Spasms(Up to Day 15)
- Time to Relapse During the Extended Treatment Period(Up to Day 75)