CBD-SIB - Efficacy of cannabidiol in the management of Self-Injurious Behavior in children and adolescents with severe neurodevelopmental disorders

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Assistance Publique Hopitaux De Paris

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Psychiatry and Psychology [F] - Behavior and Behavior Mechanisms [F01]

Decision date (initial)

2025-10-07

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Fondation Perce Neige

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

To evaluate the efficacy of cannabidiol in reducing the frequency of self-injuries at D56 in children and adolescents with severe neurodevelopmental disorders

Secondary objectives 4

1. 1) Evaluate the efficacy of cannabidiol in reducing the frequency of self-injuries at D31
2. 2) Evaluate the efficacy of cannabidiol at D31 and D56 on: - the severity of self-injuries; - the frequency and severity of stereotypical behaviors, aggressive/destructive behaviors; - the frequency of nonverbal manifestations of pain; - quality of life; - stress related to their child felt by parents
3. 3) Evaluate the safety of EPIDYOLEX®.
4. 4) Evaluate the correlation between the evolution of the frequency of self-injuries and the evolution of the frequency of nonverbal manifestations of pain in children and adolescents with severe neurodevelopmental disorders and treated with cannabidiol.

Conditions and MedDRA coding

Children and adolescents with severe neurodevelopmental disorders

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. 1. Age between 5 years and 17 years 6 months;
2. Weight between 12 and 49.9 kg;
3. 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;
4. Severe self-injuries during the last 7 days defined by BPI-01, i.e.: a. at least one type of self-injuries assessed as severe intensity occurring at least once every 3 hours while awake or b. at least two types of self-injuries assessed as severe intensity occurring at least once every 6 hours each while awake;
5. 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);
6. 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;
7. 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.
8. Consent of both holders of parental authority;
9. Affiliation to social security regimen.

Exclusion criteria 12

1. Hypersensitivity to the active substance (cannabidiol) or to any of the excipients (refined sesame oil, anhydrous ethanol, sucralose, strawberry flavor, benzyl alcohol);
2. Treatment with or consumption of cannabidiol in the 12 weeks prior to inclusion;
3. Consumption of cannabis in the 12 weeks prior to inclusion;
4. 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);
5. Elevated transaminases > 3N or total bilirubin > 2N;
6. Known current heart failure;
7. Known current terminal renal failure (GFR < 15 ml/min/1.73 m2);
8. Known current moderate or severe hepatic insufficiency (Child-Pugh B or C);
9. Known current epilepsy or history of epilepsy, even stabilized, requiring treatment currently or not, whatever the type;
10. Pregnancy or breastfeeding;
11. Inability of the patient or entourage to comply with the study protocol;
12. Participation in other interventional research, clinical trial or clinical investigation

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Cannabidiol will be considered effective at V8 D56 in clinically significant reductions in the frequency of self-injuries if the frequency score on the “Self-Injurious Behavior” subdomain of the BPI-01 is reduced by 30% or more at visit V8 D56 compared to visit V2 D0.

Secondary endpoints 4

1. Cannabidiol will be considered effective at V6 D31 to clinically significantly reduce the frequency of self-injuries if the frequency score in the BPI-01 “Self-injurious Behavior” subdomain is reduced by 30% or more at visit V6 D31 compared to visit V2 D0.
2. efficacy of the treatment assessed by the change in percentage scores between visit V2 D0 and visits V6 D31 and V8 D56: in the BPI-01 “Self-injurious behavior” subdomain; “Stereotyped behaviors” subdomain;“Aggressive/Destructive Behaviors” subdomain;- GED-DI score;- QI-Disability score;- Parental Stress Scale (PSS) score.
3. Serious and non-serious clinical adverse events, in particular: diarrhea, vomiting, decreased appetite, weight loss, drowsiness, asthenia, fever, increased transaminases or total bilirubin, decreased hemoglobin and hematocrit, increased creatinine.
4. Correlation between the change in the frequency of self-injuries (frequency score in the “Self-injurious behavior” subdomain of the BPI-01) between V2 D0 and V6 D31 and V8 D56 and the change in the frequency of non-verbal manifestations of pain (GED-DI score) between V2 D0 and V6 D31 and V8 D56

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Assistance Publique Hopitaux De Paris

Sponsor organisation

Assistance Publique Hopitaux De Paris

Address

Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux

City

Paris Cedex 10

Postcode

75475

Country

France

Scientific contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Pr Pauline CHASTE

Public contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Pr Pauline CHASTE

Locations

1 EU/EEA country · 3 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 7 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications