Efficacy of Cannabidiol in the Management of Self-injuries in Children and Adolescents With Severe Neurodevelopmental Disorders. (CBD-SIB)
研究概览
详细说明
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.
研究类型
注册 (估计的)
阶段
- 阶段2
联系人和位置
学习联系方式
- 姓名:Laure CHOUPEAUX, Msc
- 电话号码:+33 01 44 38 17 11
- 邮箱:laure.choupeaux@aphp.fr
研究联系人备份
- 姓名:Pauline CHASTE, MD; PhD
- 电话号码:01 44 49 57 59
- 邮箱:pauline.chaste@aphp.fr
学习地点
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Île-de-France Region
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Paris、Île-de-France Region、法国、75015
- Hôpital Necker Enfants malades
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接触:
- Pauline CHASTE
- 邮箱:pauline.chaste@aphp.fr
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Paris、Île-de-France Region、法国、75013
- Hôpital Pitié-Salpêtrière
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接触:
- David COHEN, MD, PHD
- 邮箱:david.cohen@aphp.fr
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参与标准
资格标准
适合学习的年龄
- 孩子
接受健康志愿者
描述
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
学习计划
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:不适用
- 介入模型:单组作业
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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实验性的: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.
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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Efficacy of cannabidiol
大体时间: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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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Efficacy of cannabidiol
大体时间: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
大体时间: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
大体时间: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
大体时间: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
大体时间: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
大体时间: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
大体时间: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
大体时间: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
大体时间: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: 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
大体时间: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
|
|
Efficacy of cannabidiol / frequency of Aggressive/destructive behaviour
大体时间: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
大体时间: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
大体时间: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
大体时间: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
大体时间: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.
大体时间: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.
大体时间: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®
大体时间: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
大体时间: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
大体时间: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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合作者和调查者
调查人员
- 首席研究员:Pauline CHASTE, MD; PhD、Assistance Publique - Hôpitaux de Paris
- 研究主任:Maryse PAGNIER, MD; PhD、Assistance Publique - Hôpitaux de Paris
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
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
与本研究相关的术语
关键字
其他研究编号
- APHP240090
- 2025-521161-27-00 (克蒂斯)
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