在现实生活中对法国处方 Epidyolex 的一组参与者进行长期随访 (OPERA)
在现实生活中对法国处方 Epidyolex 的多中心、非干预、前瞻性队列参与者进行长期随访
This is a multi-center, non-interventional and prospective study of patients receiving Epidyolex as part of standard clinical practice in France. The study will state an overview of patient characteristics and clinical history (including age, sex, diagnosis, duration of epilepsy, predominant seizure type, previous medications, current co-medications and Epidyolex dose), and an evaluation of retention rates, safety profile, seizure activity, changes in executive function and quality of life measured in a 2-year follow-up period.
研究概览
研究类型
注册 (实际的)
联系人和位置
学习地点
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Amiens、法国、80054
- CHU Amiens-service adulte
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Amiens、法国、80054
- CHU Amiens-service enfant
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Angers、法国、49100
- CHU Angers - service adulte
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Angers、法国、49100
- CHU Angers - service enfant
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Bordeaux、法国、33000
- CHU Bordeaux - service adulte
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Bordeaux、法国、33000
- CHU Bordeaux,neuropédiatrie
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Bron、法国、69500
- GH Est - Hôpital Femme Mère Enfant
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Bron、法国、69677
- HCL - Lyon - service adulte
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Dijon、法国、21000
- CHU Dijon - service neurophysiologie clinique
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Grenoble、法国、38700
- CHU Grenoble - service adulte
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Grenoble、法国、38700
- CHU Grenoble - service enfant
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Le Kremlin-Bicêtre、法国、94270
- APHP Kremlin Bicetre - service enfant
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Lille、法国、59037
- CHU Lille - service enfant
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Lille、法国、59037
- Hôpital Salengro - service adulte
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Marseille、法国、13005
- APHM - Marseille - service adulte
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Marseille、法国、13005
- Hôpital de la Timone - service enfant
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Nancy、法国、54000
- CHU Nancy - service adulte
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Nancy、法国、54000
- CHU Nancy - service enfant
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Paris、法国、75013
- APHP Pitié Salpetriere- service adulte
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Paris、法国、75019
- APHP Robert Debré - service enfant
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Paris、法国、75743
- APHP Necker - service enfant
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Rennes、法国、35000
- CHU Rennes - service adulte
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Rennes、法国、35200
- CHU Rennes - service enfant
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Strasbourg、法国、67200
- CHU Strasbourg - service enfant
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Strasbourg、法国、67200
- Hôpital de Hautepierre - service adulte
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Toulouse、法国、31059
- CHU Toulouse - service adulte
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Toulouse、法国、31059
- CHU Toulouse - service enfant
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Tours、法国、37000
- Hôpital Bretonneau - service adulte
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Tours、法国、37044
- Hôpital Clocheville - service enfant
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
取样方法
研究人群
Participants for whom Epidyolex is prescribed according to the approved therapeutic indications and criteria of the summary of product Characteristics (SmPC). Participants will be recruited from approximately 15 facilities specializing in treating rare epilepsies in France.
描述
Key Inclusion Criteria:
- Individuals for whom treatment with Epidyolex has been initiated by a physician with experience in the treatment of epilepsy.
- The participant and/or parent(s)/legal representative is willing and able to give informed consent/assent for participation in the study.
Key Exclusion Criteria:
- Previously initiated with Epidyolex before the start of the study (especially during the French early access program (EAP)).
学习计划
研究是如何设计的？
设计细节
- 观测模型：COHORT
- 时间观点：PROSPECTIVE
队列和干预
团体/队列 |
干预/治疗 |
|---|---|
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Epidyolex
Participants will receive Epidyolex in accordance with their routine clinical practice as prescribed by their physician. |
Administered as an oral solution
其他名称：
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研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
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Retention Rate Percentage After Initiation of Treatment With Epidyolex
大体时间：Up to 24 months post-dose.
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Retention rate will be measured through assessing the frequency of seizures and the length of time on treatment before a seizure event. |
Up to 24 months post-dose.
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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癫痫发作频率相对于基线的百分比变化（平均每 28 天）
大体时间：给药后长达 24 个月。
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给药后长达 24 个月。
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50% 回复率
大体时间：给药后长达 24 个月。
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给药后长达 24 个月。
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75% 回复率
大体时间：给药后长达 24 个月。
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给药后长达 24 个月。
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100% 反应者（癫痫发作自由度）率
大体时间：给药后长达 24 个月。
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给药后长达 24 个月。
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无发作天数（平均每 28 天）
大体时间：给药后长达 24 个月。
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给药后长达 24 个月。
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过去 28 天内最长的无发作天数
大体时间：给药后长达 24 个月。
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给药后长达 24 个月。
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给参与者服用 Epidyolex 的模态剂量
大体时间：给药后长达 24 个月。
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给药后长达 24 个月。
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给予参与者的 Epidyolex 最大剂量
大体时间：给药后长达 24 个月。
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给药后长达 24 个月。
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伴随抗癫痫药 (AED) 和其他药物的平均每日剂量相对于基线的变化
大体时间：给药后长达 24 个月。
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给药后长达 24 个月。
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Change From Baseline in Behavior Rating Inventory of Executive Function, Preschool Version (BRIEF-P) Score in Participants Aged ≤5 Years
大体时间：Baseline; Up to 24 months post-dose.
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The BRIEF-P is a 63-item questionnaire which consists of a single Rating Form used by parents, teachers, and day care providers to assess a child's executive functions within the context of their everyday environments--home and preschool. High scores obtained on the BRIEF-P suggest a higher level of dysfunction in a specific domain of executive functions. The BRIEF rating scores is an ordinal set of a technically summarized degree of disorder in functionality: Never = 1 (Minimum), Sometimes = 2 and Often = 3 (Maximum). |
Baseline; Up to 24 months post-dose.
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Change From Baseline in Behavior Rating Inventory of Executive Function (BRIEF) Score in Participants Aged 6 to 18 Years
大体时间：Baseline; Up to 24 months post-dose.
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BRIEF is an 86-item questionnaire used to assess the executive function behaviors at home and at school for children and adolescents ages 5-18. High scores obtained on the BRIEF suggest a higher level of dysfunction in a specific domain of executive functions. The BRIEF rating scores is an ordinal set of a technically summarized degree of disorder in functionality: Never = 1 (Minimum), Sometimes = 2 and Often = 3 (Maximum). |
Baseline; Up to 24 months post-dose.
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Change From Baseline in Behavior Rating Inventory of Executive Function, Adult Version (BRIEF- A) Score in Participants Aged >18 Years
大体时间：Baseline; Up to 24 months post-dose.
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BRIEF-A is a patient-reported scale to measure various aspects of adult executive functioning and self-regulation in the person's everyday environment. It is a 75-item questionnaire that participants rate on a 3-point Likert scale (1 = behavior is never observed to 3 = behavior is often observed). Higher scores indicate greater impairment in executive functioning. |
Baseline; Up to 24 months post-dose.
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Use of Rescue Medication Per Month
大体时间：Up to 24 months post-dose.
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A month is defined as 28 days. |
Up to 24 months post-dose.
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Physician Global Clinical Impression of Change (PGIC) Score
大体时间：Up to 24 months post-dose.
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The PGIC comprises the following question: "Please assess the change in the patient's general functional abilities since enrollment," scored on a seven-point scale from 1 (Very Much Improved) to 7 (Very Much Worse). Lower scores on the scale indicate a better quality of life. |
Up to 24 months post-dose.
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Caregiver Global Impression of Change (CGIC) Score
大体时间：Up to 24 months post-dose.
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The CGIC comprises of the following question: "Since your child started treatment, please assess the status of your child's overall condition (comparing their condition now to their condition before treatment)," scored on a seven-point scale from 1 (Very Much Improved) to 7 (Very Much Worse). Lower scores on the scale indicate a better quality of life. |
Up to 24 months post-dose.
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Change from Baseline in Health Utility Index Mark II (HUI-2) Score
大体时间：Up to 24 months post-dose.
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The HUI-2 is a generic measure of health status and health-related quality of life. There are 15 questions in the questionnaire with 1-week recall completed by the caregiver. Answers to the questionnaire are mapped into a classification system of 7 sets of utility scores (sensation, mobility, emotion, cognition, self-care, pain, and fertility) with a scoring scale of a minimum score of 0.00 (lack of functional capacity or most disabled) to a maximum of 1.00 (full function or no disability). A higher score indicates a better outcome. |
Up to 24 months post-dose.
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合作者和调查者
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (实际的)
研究完成 (实际的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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