- ICH GCP
- 미국 임상 시험 레지스트리
- 임상시험 NCT07564245
Efficacy of Cannabidiol in the Management of Self-injuries in Children and Adolescents With Severe Neurodevelopmental Disorders. (CBD-SIB)
연구 개요
상세 설명
The objective of this project is to study the effect of cannabidiol in children with severe neurodevelopmental disorders who engage in significant self-injury, both on the frequency and severity of self-injury and on nonverbal expressions of pain. We expect a positive effect on both of these parameters, as well as a correlation between a reduction in nonverbal expressions of pain and a reduction in the frequency and severity of self-injury. If our expectations are confirmed, this would contribute to expanding the use of cannabidiol, verifying its tolerability in this patient population, and understanding the factors associated with a positive response to this unconventional treatment in order to clarify its indications.
The experimental treatment is the same for all participants. It consists of a cannabidiol (CBD) solution for oral administration: EPIDYOLEX® will be administered orally in two equal doses per day, 12 hours apart, preferably with breakfast and dinner.
연구 유형
등록 (추정된)
단계
- 2 단계
연락처 및 위치
연구 연락처
- 이름: Laure CHOUPEAUX, Msc
- 전화번호: +33 01 44 38 17 11
- 이메일: laure.choupeaux@aphp.fr
연구 연락처 백업
- 이름: Pauline CHASTE, MD; PhD
- 전화번호: 01 44 49 57 59
- 이메일: pauline.chaste@aphp.fr
연구 장소
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Île-de-France Region
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Paris, Île-de-France Region, 프랑스, 75015
- Hopital Necker Enfants Malades
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연락하다:
- Pauline CHASTE
- 이메일: pauline.chaste@aphp.fr
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Paris, Île-de-France Region, 프랑스, 75013
- Hôpital Pitié-Salpêtrière
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연락하다:
- David COHEN, MD, PHD
- 이메일: david.cohen@aphp.fr
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참여기준
자격 기준
공부할 수 있는 나이
- 어린이
건강한 자원 봉사자를 받아들입니다
설명
Inclusion Criteria:
- Age between 5 years and 17 years 6 months;
- Weight between 12 and 49.9 kg;
- Clinical diagnosis of severe neurodevelopmental disorder including severe to profound intellectual developmental disorder, characterized in the DSM-5 by a need for help with any daily activity (meals, dressing, toileting, elimination) and the need for constant supervision;
Severe self-injuries during the last 7 days defined by BPI-01, i.e.:
- at least one type of self-injuries assessed as severe intensity occurring at least once every 3 hours while awake or
- at least two types of self-injuries assessed as severe intensity occurring at least once every 6 hours each while awake;
- Self-injuries refractory to treatment with atypical neuroleptic (RISPERIDONE, ARIPIPRAZOLE, etc.) at a dosage deemed effective by the investigator in view of the patient's weight, age and background, for a minimum duration of 30 days (except in the case of poor tolerance by the patient);
- No change in drug and non-drug treatments such as rehabilitative care (psychomotricity, occupational therapy, speech therapy, intervention by a specialist educator) for at least one month;
- In WOCBP and with active sexual life: negatives hCG and use of highly effective contraceptive measure until 18 days after the end of the treatment.
- The participant has a caregiver who can reliably accompany him/her to all clinic visits, provide reliable assessments, help him/her to comply with study requirements, and interact with him/her on a regular basis.
- Signature of Consent of both holders of parental authority;
- Affiliation to social security regimen.
Exclusion Criteria:
- 1. Underweight corresponding to a body mass index below the IOTF 18.5 curve on the International Obesity Task Force's body mass index curves for girls and boys 2. Hypersensitivity to the active substance (cannabidiol) or to any of the excipients (refined sesame oil, anhydrous ethanol, sucralose, strawberry flavor, benzyl alcohol); 3. Treatment with or consumption of cannabidiol in the 12 weeks prior to inclusion; 4. Consumption of cannabis in the 12 weeks prior to inclusion; 5. Usual treatment (excluding treatment of self-injuries) containing molecules interacting with cannabidiol (rifampicin, carbamazepine, enzalutamide, mitotane, St. John's wort, clobazam, valproate, stiripentol, phenytoin, lamotrigine, everolimus, theophylline, tizanidine, bupropion, efavirenz, diflunisal, propofol, fenofibrate, gemfibrozil, morphine, lorazepam, repaglinide, warfarin, sirolimus, tacrolimus, digoxin); 6. Elevated transaminases > 3N or total bilirubin > 2N; 7. Known current heart failure; 8. Known current terminal renal failure (GFR < 15 ml/min/1.73 m2); 9. Known current moderate or severe hepatic insufficiency (Child-Pugh B or C); 10. Known current epilepsy or history of epilepsy, even stabilized, requiring treatment currently or not, whatever the type; 11. Pregnancy or breastfeeding; 12. Inability of the patient or entourage to comply with the study protocol; 13. Patient currently participating in another interventional research (category 1 or 2), clinical trial or clinical investigation, or who are in the exclusion period following him/her participation in another study of this type, as defined in the protocol of that study.
14. Participant related to a person involved in the study at the investigational site
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 해당 없음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
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실험적: Cannabidiol
Multicentre phase II study evaluating the efficacy of CANNABIDIOL (EPIDYOLEX®) in reducing self-injuries and non-verbal expressions of pain in children and adolescents with severe neurodevelopmental disorders.
The experimental treatment is the same for all participants will last 63 days.
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EPIDYOLEX® will be administered orally in two equivalent doses taken 12 hours apart each day.
It will be started at a dose of 5 mg/kg/day and gradually increased to 25 mg/kg/day over a titration period of 28 days.
The target dose of 25 mg/kg/day will be maintained for 35 days.
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Efficacy of cannabidiol
기간: Day 63
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Efficacy is evaluate by reducing the frequency of self-injuries.
Cannabidiol will be considered effective at V8 Day 63 in clinically significant reductions in the frequency of self-injuries if the frequency score on the 'Self-injurious behaviour subdomain of the BPI-01 is reduced by 30% or more at the V8 Day 63 visit compared to the V2 Day 0 visit.
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Day 63
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Efficacy of cannabidiol
기간: Day 29
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Cannabidiol will be considered effective at Visit6 Day29 in clinically significant reduction in the frequency of self-injuries if the frequency score in the 'Self-injurious behaviour' sub-domain of the BPI-01 is reduced by 30% or more at the Visit6 Day29 visit compared with the Visit2 Day 0 visit.
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Day 29
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Efficacy of cannabidiol / severity of self-injuries
기간: Day 29
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the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V6 Day29. For this criteria: severity score in the 'Self-injurious behaviour' sub-domain of BPI-01 |
Day 29
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Efficacy of cannabidiol / severity of self-injuries
기간: Day 63
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the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V8 Day 63. For this criteria: severity score in the 'Self-injurious behaviour' sub-domain of BPI-01 |
Day 63
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Efficacy of cannabidiol / severity of Stereotyped behaviour
기간: Day 29
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The efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day0 and visits V6 Day29. For this criteria: severity score for the BPI-01 sub-domain 'Stereotyped behaviour |
Day 29
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Efficacy of cannabidiol / severity of Stereotyped behaviour
기간: Day 63
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the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V8 Day 63. For this criteria: severity score for the BPI-01 sub-domain 'Stereotyped behaviour |
Day 63
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Efficacy of cannabidiol / frequency of Stereotyped behaviour
기간: Day 29
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The efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V6 Day 29. For this criteria: frequency score for the BPI-01 sub-domain 'Stereotyped behaviour |
Day 29
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Efficacy of cannabidiol / frequency of Stereotyped behaviour
기간: Day 63
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the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V8 Day 63. For this criteria: frequency score for the BPI-01 sub-domain 'Stereotyped behaviour |
Day 63
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Efficacy of cannabidiol / severity of Aggressive/destructive behaviour
기간: Day 29
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the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V6 Day 29. For this criteria: severity score for the BPI-01 sub-domain 'Aggressive/destructive behaviour' |
Day 29
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Efficacy of cannabidiol / severity of Aggressive/destructive behaviour
기간: Day 63
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the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V8 Day 63. For this criteria: severity score for the BPI-01 sub-domain 'Aggressive/destructive behaviour' |
Day 63
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Efficacy of cannabidiol / frequency of Aggressive/destructive behaviour
기간: Day 29
|
the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V6 Day 29. For this criteria: frequency score for the BPI-01 sub-domain 'Aggressive/destructive behaviour' |
Day 29
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Efficacy of cannabidiol / frequency of Aggressive/destructive behaviour
기간: Day 63
|
the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V8 Day 63. For this criteria: frequency score for the BPI-01 sub-domain 'Aggressive/destructive behaviour' |
Day 63
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Efficacy of cannabidiol / the frequency of non-verbal expressions of pain
기간: Day 29
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the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V6 Day 29 in GED-DI score
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Day 29
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Efficacy of cannabidiol / the frequency of non-verbal expressions of pain
기간: Day 63
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the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V8 Day 63 in GED-DI score
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Day 63
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Efficacy of cannabidiol / quality of life
기간: Day 29
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the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V6 Day 29 in QI-Disability score
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Day 29
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Efficacy of cannabidiol / quality of life
기간: Day 63
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the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V8 Day 63 in QI-Disability score
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Day 63
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Efficacy of cannabidiol / stress related to the child experienced by the parents.
기간: Day 29
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the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V6 Day 29 in Parental Stress Scale (PSS) score
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Day 29
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Efficacy of cannabidiol / stress related to the child experienced by the parents.
기간: Day 63
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the efficacy of the treatment will be assessed by the percentage change in percentage scores between visit V2 Day 0 and visits V8 Day 63 in Parental Stress Scale (PSS) score
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Day 63
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Safety of EPIDYOLEX®
기간: Day 81
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serious and non-serious clinical and biological adverse events
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Day 81
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Correlation between the evolution of the frequency of self-injuries and the evolution of the frequency of non-verbal expressions of pain
기간: Day 29
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Correlation between the evolution of the frequency of self-injuries (frequency score in the BPI-01 subdomain 'Self-injurious behaviour') and the evolution of the frequency of non-verbal expressions of pain (GED-DI score)
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Day 29
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Correlation between the evolution of the frequency of self-injuries and the evolution of the frequency of non-verbal expressions of pain
기간: Day 63
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Correlation between the evolution of the frequency of self-injuries (frequency score in the BPI-01 subdomain 'Self-injurious behaviour') and the evolution of the frequency of non-verbal expressions of pain (GED-DI score)
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Day 63
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공동 작업자 및 조사자
수사관
- 수석 연구원: Pauline CHASTE, MD; PhD, Assistance Publique - Hôpitaux de Paris
- 연구 책임자: Maryse PAGNIER, MD; PhD, Assistance Publique - Hôpitaux de Paris
연구 기록 날짜
연구 주요 날짜
연구 시작 (추정된)
기본 완료 (추정된)
연구 완료 (추정된)
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
이 연구와 관련된 용어
키워드
- 통증 평가
- 부모의 스트레스
- 신경 발달 장애
- Epidyolex
- Cannabidiol, self-injuries
- Vineland Adaptive Behaviour II
- Behavior Problems Inventory - 01 (BPI-01)
- Pain Evaluation- Intellectual Disability (GED-DI)
- Quality of life inventory - Disability (QI-Disability)
- Parental Stress Scale (PSS)
- Non Communicating Children's Pain
기타 연구 ID 번호
- APHP240090
- 2025-521161-27-00 (씨티스)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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