Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome: A Phase II Trial
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- List Sorting Working Memory Test
研究概览
简要总结
The purpose of this study is to better understand the utility of cannabidiol (CBD/ Epidiolex) for improving the treatment of cognitive impairments in Sturge-Weber syndrome (SWS).
详细描述
The investigators hope to gain an understanding of the utility of pharmaceutical grade CBD used for the treatment of cognitive impairments in SWS in this open-label study. Anecdotal evidence from a phase I trial investigating the use of CBD for medically refractory seizures suggests CBD may also have a beneficial effect on cognition, mood, and behavior. The investigators hypothesize that CBD/ Epidiolex will improve SWS brain function resulting in improved cognitive function, social interactions, mood, motor function and behavior, as well as reduced migraines. This is an open-label prospective oral drug trial of Epidiolex in 10 subjects. Assessments will be done at baseline and repeated after 6 months on study drug.
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
- 3 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants with Sturge-Weber syndrome brain involvement as defined on neuroimaging (n=10 subjects, male and female, ages 3 to 50 years of age) and the following:
- •Cognitive impairment defined as a cognitive neuroscore greater than or equal to 2 at screening.
- •Anti-epileptic, mood or behavioral drugs (if on) at stable doses for a minimum of 4 weeks prior to enrollment.
- •If present, VNS must be on stable setting for a minimum of 3 months prior to enrollment.
- •If on ketogenic or Atkins diet, must be on stable ratio for a minimum of 3 months prior to enrollment.
- •Previous subjects who fail at any point to meet continuation criteria and withdraw early may be considered for re-enrollment under new subject ID as long as the above inclusion criteria are met. The determination of whether to re-enroll will be made by the PI and sponsor on a case-by-case basis. Re-enrollment can occur no earlier than 4 weeks after the final, post-weaning follow-up visit under the old subject ID.
- •Written informed consent obtained from the patient or the patient's legal representative must be obtained prior to beginning treatment.
排除标准
- •Patients with any severe and/or uncontrolled medical conditions at randomization such as:
- •Liver disease such as cirrhosis, decompensated liver disease, and chronic hepatitis (i.e. quantifiable HBV-DNA and/or positive HbsAg, quantifiable HCV-RNA)
- •Uncontrolled diabetes as defined by fasting serum glucose greater than 1.5
- •Active (acute or chronic) or uncontrolled severe infections
- •Active, bleeding diathesis
- •Patients who have 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.
- •Patients who start or discontinue a seizure, mood or behavioral medication in the 4 weeks leading up to screening.
- •Prior treatment with any investigational drug or use of any other cannabis product within the preceding 4 weeks prior to study entry.
- •Patients with a history of non-compliance to medical regimens or who are considered potentially unreliable or will not be able to complete the entire study. This includes those in foster care, or those unable to keep follow-up appointments, maintain close contact with the Principal Investigator, or complete all necessary studies to maintain safety.
- •Pregnant or nursing (lactating) women, where pregnancy is defined as the state of the female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
研究组 & 干预措施
Cannabidiol/ Epidiolex
All subjects will receive the experimental Epidiolex (cannabidiol) oral solution to be taken at home twice a day, and will be treated on an outpatient basis. The drug will be taken for 24 weeks unless the subject chooses to participate in the extension phase of the study, in which case the subject will continue to receive the drug for one additional year or until the drug is approved for clinical use for the treatment of cognitive impairments in patients with Sturge-Weber syndrome.
干预措施: Cannabidiol (Drug)
结局指标
主要结局
List Sorting Working Memory Test
时间窗: Baseline, Follow-up (6 months)
Data on cognitive function was collected using the List Sorting Working Memory Test from the NIH Toolbox. Data was collected on working memory performance, which was transformed into a t-score from 0 to 100 where a higher t-score indicates better performance. T-score of 50 indicates the population mean with a standard deviation of 10. Data was collected at baseline and after 6 months on study drug.
次要结局
- Seizure Frequency(Baseline, Follow-up (6 months))
- Migraine Severity(Baseline, Follow-up (6 months))
- Modified House Classification Scores(Baseline, Follow-up (6 months))
- Pediatric Evaluation of Disability Inventory Computer Adapted Test(Baseline, Follow-up (6 months))
- Social Responsiveness Scale, Second Edition (SRS-2)(Baseline, Follow-up (6 months))
- Behavioral Assessment System for Children, Third Edition (BASC-3)(Baseline, Follow-up (6 months))
- Screen for Child Anxiety Related Disorders (SCARED)(Baseline, Follow-up (6 months))
- Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV)(Baseline, Follow-up (6 months))
- Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-55)(Baseline, Follow-up (6 months))
- Erhardt Developmental Prehension Assessment Scores(Baseline, Follow-up (6 months))
- Picture Vocabulary Test(Baseline, Follow-up (6 months))
- Pediatric Neurological Quality of Life (Neuro-QoL)(Baseline, Follow-up (6 months))
- Safety of Epidiolex(Baseline, Follow-up (6 months))
- ABILHAND Questionnaire(Baseline, Follow-up (6 months))
- Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2)(Baseline, Follow-up (6 months))
- Neuroscore(Baseline, Follow-up (6 months))
- Adult Neurological Quality of Life (Neuro-QoL)(Baseline, Follow-up (6 months))
- Port-wine Birthmark Score(Baseline, Follow-up (6 months))
研究者
Anne Comi, MD
Principal Investigator, Director Sturge-Weber Center, Kennedy Krieger Institute, Professor Johns Hopkins University School of Medicine
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.