Epidiolex® For The Treatment Of Anxiety Comorbidity in Refractory Pediatric Epilepsy
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- CGI-I
研究概览
简要总结
This is a prospective open label study assessing the efficacy and tolerability of pharmaceutical grade cannabidiol--Epidiolex (R), for the treatment of anxiety in pediatric patients with pediatric epilepsy that has been difficult to treat and requires ongoing use of anticonvulsant medication.
详细描述
This study is an open label, adjunctive, proof of concept clinical trial. Prospective participants will be considered for the study if neurologic status suggests the need for continued treatment, and behavioral characteristics are clinically significant. All participants will receive active treatment, involving flexible dose titration of Epidiolex® and will be monitored for a period of four months. The study will be monitored and overseen by the Johns Hopkins Hospital Institutional Review Board.
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
- 6 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Established diagnosis of epilepsy, characterized by focal or generalized seizures. All participants will have active epilepsy that requires treatment with anticonvulsant medication.
- •No episodes of seizure clusters of status epilepticus within 30 days prior to entry into the study.
- •Established symptoms of anxiety with functional impairment.
- •Baseline behavioral criteria for inclusion will include subscale scores for anxiety above the norm for age and gender inth e 60 days prior to the study on one of the following:
- •Male or female participants equal to or above age 6 and below age 18 at the start of the study. No exclusion will be made on the basis of gender or minority status.
- •Ability to administer medicine orally
- •Previous subjects who failed at any point to meet continuation criteria and withdrew early may be considered for re-enrollment by the PI on a case-by-case basis.
- •Participant or legal caregiver capable of providing informed consent and fully capable of monitoring the subject's disease process and compliance with treatment.
- •Participants who are sexually active, must agree to sexual abstinence, or, to use an approved birth control method for the full duration of study participation.
- •No active use of CBD products within the 14 days prior to screening visit and commitment to only use study product for the duration of the study.
排除标准
- •Baseline lab tests for liver specific transaminase, ALT, over the upper limit of normal (ULN).
- •Previous allergic or hypersensitivity reactions to Epidiolex® or cannabidiol
- •No access to a phone or internet to complete remote visits (in-person visits acceptable for participants without devices)
- •Active substance abuse or dependence
- •Presence of psychotic illness or imminent risk of harm to self or others.
- •Current standing use of benzodiazepines (except as "rescue" medicine)
- •Serious unstable medical or neurologic conditions such as HIV, liver or kidney disease, cancer or diabetes.
- •Presence of Epilepsy Syndrome such as Sturge-Weber Syndrome that will be more suitable as a candidate in alternate research studies.
- •Participation in a previous experimental drug study within 30 days of baseline visit.
- •Cognitive functional capacity or English literacy that is insufficient to assure validity of clinical rating scales
- •Insufficient capacity of caregiver or legal guardian to understand and appropriately consent for study procedures
- •No exclusions for existing AEDs will be absolute, though consideration of additional monitoring will be in place for patients taking clobazam or valproate.
- •Pregnant, planning to become pregnant, breast feeding, or failing to use an appropriate method of contraception.
研究组 & 干预措施
treatment arm
open label treatment intervention
干预措施: Cannabidiol 100 MG/ML (Drug)
结局指标
主要结局
CGI-I
时间窗: 16 weeks
Mean of Clinical Global Impression-Improvement (CGI-I) from baseline. The CGI is rated on a 7-point scale. (lower score represents a better outcome: 1/ High score represents minimum improvement: 7)
次要结局
未报告次要终点
研究者
Jay Salpekar, M.D.
Principal Investigator
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.