CANNABIDIOL for Severe neurodevelopmental disorders | Self-injurious behavior

Inclusion criteria

• {"criterion_text":"- 1. Age between 5 years and 17 years 6 months;\n- 2. Weight between 12 and 49.9 kg;\n- 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;\n- 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;\n- 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);\n- 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;\n- 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.\n- 8. Consent of both holders of parental authority;\n- 9. Affiliation to social security regimen."}

Exclusion criteria

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

Primary endpoints

• {"endpoint_text":"- 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.","definition_or_measurement_approach":"Reduction of the frequency score on the BPI-01 'Self-Injurious Behavior' subdomain by 30% or more at visit V8 (D56) compared to baseline visit V2 (D0)."}

Secondary endpoints

• {"endpoint_text":"- 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.","definition_or_measurement_approach":"Reduction of the BPI-01 'Self-injurious Behavior' frequency score by 30% or more at V6 (D31) vs V2 (D0)."}
• {"endpoint_text":"- 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;-\tGED-DI score;-\tQI-Disability score;-\tParental Stress Scale (PSS) score.","definition_or_measurement_approach":"Change in percentage scores between baseline (V2 D0) and V6 (D31) and V8 (D56) for multiple scales/subdomains: BPI-01 subdomains (Self-injurious, Stereotyped behaviors, Aggressive/Destructive Behaviors), GED-DI, QI-Disability, Parental Stress Scale (PSS)."}
• {"endpoint_text":"- 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.","definition_or_measurement_approach":"Collection and reporting of serious and non-serious clinical adverse events, with particular attention to listed events (diarrhea, vomiting, decreased appetite, weight loss, somnolence, asthenia, fever, liver enzyme/bilirubin increases, hematologic and renal laboratory changes)."}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Statistical correlation analysis between change in BPI-01 'Self-injurious behavior' frequency score and change in GED-DI score between baseline (V2 D0) and V6 (D31)/V8 (D56)."}

France

Earliest CTIS Part Ii Submission Date

12-09-2025

Latest Decision Or Authorization Date

28-04-2026

Processing Time Days

228

Number Of Sites

3

Number Of Participants

21

Primary sponsor

Full Name

Assistance Publique Hopitaux De Paris

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France

Third parties

• {"country":"","full_name":"Fondation Perce Neige","duties_or_roles":"Source of monetary support","organisation_type":""}