실제 환경에서 프랑스에서 Epidyolex를 처방한 참가자 코호트에 대한 장기 후속 조치 (OPERA)
실제 환경에서 프랑스에서 Epidyolex를 처방한 참가자의 다기관, 비간섭, 전향적 코호트의 장기 후속 조치
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.
연구 개요
연구 유형
등록 (실제)
연락처 및 위치
연구 장소
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Amiens, 프랑스, 80054
- CHU Amiens-service adulte
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Amiens, 프랑스, 80054
- CHU Amiens-service enfant
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Angers, 프랑스, 49100
- CHU Angers - service adulte
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Angers, 프랑스, 49100
- CHU Angers - service enfant
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Bordeaux, 프랑스, 33000
- CHU Bordeaux - service adulte
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Bordeaux, 프랑스, 33000
- CHU Bordeaux,neuropédiatrie
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Bron, 프랑스, 69500
- GH Est - Hôpital Femme Mère Enfant
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Bron, 프랑스, 69677
- HCL - Lyon - service adulte
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Dijon, 프랑스, 21000
- CHU Dijon - service neurophysiologie clinique
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Grenoble, 프랑스, 38700
- CHU Grenoble - service adulte
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Grenoble, 프랑스, 38700
- CHU Grenoble - service enfant
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Le Kremlin-Bicêtre, 프랑스, 94270
- APHP Kremlin Bicetre - service enfant
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Lille, 프랑스, 59037
- CHU Lille - service enfant
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Lille, 프랑스, 59037
- Hôpital Salengro - service adulte
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Marseille, 프랑스, 13005
- APHM - Marseille - service adulte
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Marseille, 프랑스, 13005
- Hôpital de la Timone - service enfant
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Nancy, 프랑스, 54000
- CHU Nancy - service adulte
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Nancy, 프랑스, 54000
- CHU Nancy - service enfant
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Paris, 프랑스, 75013
- APHP Pitié Salpetriere- service adulte
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Paris, 프랑스, 75019
- APHP Robert Debré - service enfant
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Paris, 프랑스, 75743
- APHP Necker - service enfant
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Rennes, 프랑스, 35000
- CHU Rennes - service adulte
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Rennes, 프랑스, 35200
- CHU Rennes - service enfant
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Strasbourg, 프랑스, 67200
- CHU Strasbourg - service enfant
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Strasbourg, 프랑스, 67200
- Hôpital de Hautepierre - service adulte
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Toulouse, 프랑스, 31059
- CHU Toulouse - service adulte
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Toulouse, 프랑스, 31059
- CHU Toulouse - service enfant
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Tours, 프랑스, 37000
- Hôpital Bretonneau - service adulte
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Tours, 프랑스, 37044
- Hôpital Clocheville - service enfant
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
샘플링 방법
연구 인구
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.
설명
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)).
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 관찰 모델: COHORT
- 시간 관점: PROSPECTIVE
코호트 및 개입
그룹/코호트 |
개입 / 치료 |
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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
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Retention Rate Percentage After Initiation of Treatment With Epidyolex
기간: 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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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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발작 빈도의 기준선 대비 백분율 변화(28일당 평균)
기간: 투약 후 최대 24개월.
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투약 후 최대 24개월.
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응답률 50%
기간: 투약 후 최대 24개월.
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투약 후 최대 24개월.
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응답률 75%
기간: 투약 후 최대 24개월.
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투약 후 최대 24개월.
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100% 응답자(발작 자유) 비율
기간: 투약 후 최대 24개월.
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투약 후 최대 24개월.
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발작이 없는 일수(28일당 평균)
기간: 투약 후 최대 24개월.
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투약 후 최대 24개월.
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지난 28일 동안 가장 긴 발작 없는 날
기간: 투약 후 최대 24개월.
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투약 후 최대 24개월.
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참가자에게 투여된 Epidyolex의 모달 복용량
기간: 투약 후 최대 24개월.
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투약 후 최대 24개월.
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참가자에게 투여된 Epidyolex의 최대 용량
기간: 투약 후 최대 24개월.
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투약 후 최대 24개월.
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병용 항경련제(AED) 및 기타 의약품의 일일 평균 복용량의 기준선에서 변경
기간: 투약 후 최대 24개월.
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투약 후 최대 24개월.
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Change From Baseline in Behavior Rating Inventory of Executive Function, Preschool Version (BRIEF-P) Score in Participants Aged ≤5 Years
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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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공동 작업자 및 조사자
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (실제)
연구 완료 (실제)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
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