Epidiolex Pilot Trial for Presymptomatic Treatment of Sturge-Weber Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Safe completion as assessed by the number of participants with no significant side effects
研究概览
简要总结
Patients with Sturge-Weber syndrome (SWS) are frequently affected by seizures, and seizures are associated with poorer neurological outcomes. To date there is no established means of predicting or preventing seizure onset. Cannabidiol (Epidiolex) was well tolerated in an open label study in this population. This trial will evaluate whether Epidiolex in presymptomatic Sturge-Weber patients may delay the onset of seizures and improve neurological outcome.
详细描述
This is an open-label, prospective dose escalation study to determine the safety and tolerability of presymptomatic treatment of SWS patients with Epidiolex. The investigators hypothesize that Epidiolex will be well tolerated and delay seizure onset in SWS patients. The total number of subjects to be consented for this safety study=10 babies with SWS; the initial funded phase will be with 6 subjects. Interim analysis will be completed after 6 subjects; the remaining 4 will be enrolled as warranted for 6 months on Epidiolex. Subjects will be dosed as recommended clinically (starting at 2.5 mg/kg/day divided bid, with weekly increases over the next 4 weeks to a maximum dose of 20 mg/kg/day) as tolerated. Neurologic visits during the trial, the bloodwork, EEGs, and motor assessments will be done as research procedures. Once the first dose of the study drug is administered, a blood draw will be done at 4-hour and 12-hour post study drug administration. A mini-PK will be similarly performed once the maximum maintenance dose is achieved.
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
入排标准
- 年龄范围
- 1 Month 至 18 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of Sturge-Weber Syndrome.
- •Age 1 to 18 months of age, inclusive.
- •Neuroimaging demonstrating involvement of 3 or more lobes, or bilateral involvement.
- •No history of seizures.
- •Patient's parent or legal guardian provides written informed consent prior to treatment initiation.
排除标准
- •Any severe and/or uncontrolled medical conditions at randomization including, but not limited to the following:
- •Liver disease such as cirrhosis, decompensated liver disease, and chronic hepatitis (i.e. quantifiable HBVDNA and/or positive HbsAg, quantifiable HCV-RNA). Specifically the patient should not have either AST or ALT more than 1.5 times the upper limit of normal. The Child- Pugh Score must be A (mild) which is a score of 5-6 (minimum/normal score=5). Child-Turcotte-Pugh (CTP) Calculator - Clinical Calculators - Hepatitis C Online (uw.edu)
- •Uncontrolled diabetes as defined by fasting serum glucose > 1.5 ULN
- •Active (acute or chronic) or uncontrolled severe infections
- •Active, bleeding diathesis
- •Any other neurological diagnosis that increases the risk of seizure or neurodevelopmental disability.
- •Patients who have had a major surgery or significant traumatic injury within 4 weeks of study entry, patients who have not recovered from the side effects of any major surgery (defined as requiring general anesthesia), or patients that may require major surgery during the course of the study.
- •Prior treatment with any investigational drug or use of any other cannabis product within the preceding 4 weeks prior to study entry.
- •Use of aspirin.
- •Allergic reaction/hypersensitivity to Epidiolex or other forms of cannabidiol.
- •Concern for non-compliance to medical regimens, or concern that the patient will not be able to complete the entire study, whether due to reliability or logistical barriers. This includes those in foster care, or those unable to keep follow-up appointments, maintain close contact with Principal Investigator, or complete all necessary studies to maintain safety.
- •Use of any seizure medication (whether for seizures or any other reason) (e.g. valproate, clobazam).
- •Use of chronic medications other than a multi-vitamin and/or vitamin D supplements.
研究组 & 干预措施
Presymptomatic Epidiolex Treatment
Epidiolex will be started at 2.5 mg/kg/day divided twice daily. The dose will increase weekly as tolerated over the next 5 weeks to a target dose of 20 mg/kg/day.
干预措施: Epidiolex 100 mg/mL Oral Solution (Drug)
结局指标
主要结局
Safe completion as assessed by the number of participants with no significant side effects
时间窗: 6 months
Safe completion as assessed by the number of participants with no seizure onset
时间窗: 6 months
次要结局
- Sturge-Weber Syndrome Neuroscore (SWS-NRS)(baseline, week 5, week 12, and week 24)
- Milestone checklist(baseline, week 5, week 12, and week 24)
- Modified Early Motor Questionnaire (EMQ)(Baseline, week 24)
- Pediatric Evaluation of Disability Inventory Computer Adaptive Test (PEDI-CAT)(Baseline, week 24)
- Modified Erhardt Developmental Prehension Assessment (EDPA)(Baseline, week 24)