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Efficacy of Cannabidiol in the Management of Self-injuries in Children and Adolescents With Severe Neurodevelopmental Disorders. (CBD-SIB)

27 april 2026 bijgewerkt door: Assistance Publique - Hôpitaux de Paris
Self-injuries includes repetitive actions directed at oneself, such as biting, hitting, hitting one's head or limbs, slapping oneself, pulling one's hair, or hitting oneself in the eyes, which can lead to severe injury. They are commonly reported in many neurodevelopmental disorders associated with intellectual development disorders. Faced with the difficulty of treating these self-mutilations, it seems essential to be able to explore new therapeutic avenues. Today, in the rare disease reference centers of the Necker-Enfants malades hospital, neuropathic pain in children, especially those suffering from genetic diseases with skin expression such as primary erythermalgia, is successfully and safely treated with cannabidiol in the event of failure of conventional therapies. It seems essential to be able to confirm the effectiveness of cannabidiol in treating self-injuries in children with neurodevelopmental disorders, and to help understand the mechanisms of action that underlie it. We hypothesize that the nociceptive system is involved in the occurrence of self-injuries and as a target of action of cannabidiol to treat it.

Studie Overzicht

Toestand

Nog niet aan het werven

Interventie / Behandeling

Gedetailleerde beschrijving

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.

Studietype

Ingrijpend

Inschrijving (Geschat)

21

Fase

  • Fase 2

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Studie Contact Back-up

Studie Locaties

    • Île-de-France Region
      • Paris, Île-de-France Region, Frankrijk, 75015
      • Paris, Île-de-France Region, Frankrijk, 75013

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

  • Kind

Accepteert gezonde vrijwilligers

Nee

Beschrijving

Inclusion Criteria:

  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.:

    1. at least one type of self-injuries assessed as severe intensity occurring at least once every 3 hours while awake or
    2. 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. 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.
  9. Signature of Consent of both holders of parental authority;
  10. 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

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: NVT
  • Interventioneel model: Opdracht voor een enkele groep
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: 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.
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.
Andere namen:
  • Epidyolex®

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Efficacy of cannabidiol
Tijdsspanne: Day 63
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.
Day 63

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Efficacy of cannabidiol
Tijdsspanne: Day 29
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.
Day 29
Efficacy of cannabidiol / severity of self-injuries
Tijdsspanne: 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 Day29.

For this criteria: severity score in the 'Self-injurious behaviour' sub-domain of BPI-01

Day 29
Efficacy of cannabidiol / severity of self-injuries
Tijdsspanne: 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 in the 'Self-injurious behaviour' sub-domain of BPI-01

Day 63
Efficacy of cannabidiol / severity of Stereotyped behaviour
Tijdsspanne: Day 29

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
Efficacy of cannabidiol / severity of Stereotyped behaviour
Tijdsspanne: 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 'Stereotyped behaviour

Day 63
Efficacy of cannabidiol / frequency of Stereotyped behaviour
Tijdsspanne: 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 'Stereotyped behaviour

Day 29
Efficacy of cannabidiol / frequency of Stereotyped behaviour
Tijdsspanne: 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 'Stereotyped behaviour

Day 63
Efficacy of cannabidiol / severity of Aggressive/destructive behaviour
Tijdsspanne: 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: severity score for the BPI-01 sub-domain 'Aggressive/destructive behaviour'

Day 29
Efficacy of cannabidiol / severity of Aggressive/destructive behaviour
Tijdsspanne: 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
Efficacy of cannabidiol / frequency of Aggressive/destructive behaviour
Tijdsspanne: 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
Tijdsspanne: 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
Efficacy of cannabidiol / the frequency of non-verbal expressions of pain
Tijdsspanne: 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 in GED-DI score
Day 29
Efficacy of cannabidiol / the frequency of non-verbal expressions of pain
Tijdsspanne: 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 in GED-DI score
Day 63
Efficacy of cannabidiol / quality of life
Tijdsspanne: 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 in QI-Disability score
Day 29
Efficacy of cannabidiol / quality of life
Tijdsspanne: 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 in QI-Disability score
Day 63
Efficacy of cannabidiol / stress related to the child experienced by the parents.
Tijdsspanne: 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 in Parental Stress Scale (PSS) score
Day 29
Efficacy of cannabidiol / stress related to the child experienced by the parents.
Tijdsspanne: 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 in Parental Stress Scale (PSS) score
Day 63
Safety of EPIDYOLEX®
Tijdsspanne: Day 81
serious and non-serious clinical and biological adverse events
Day 81
Correlation between the evolution of the frequency of self-injuries and the evolution of the frequency of non-verbal expressions of pain
Tijdsspanne: Day 29
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)
Day 29
Correlation between the evolution of the frequency of self-injuries and the evolution of the frequency of non-verbal expressions of pain
Tijdsspanne: Day 63
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)
Day 63

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Onderzoekers

  • Hoofdonderzoeker: Pauline CHASTE, MD; PhD, Assistance Publique - Hôpitaux de Paris
  • Studie directeur: Maryse PAGNIER, MD; PhD, Assistance Publique - Hôpitaux de Paris

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Geschat)

1 mei 2026

Primaire voltooiing (Geschat)

1 mei 2029

Studie voltooiing (Geschat)

1 februari 2030

Studieregistratiedata

Eerst ingediend

27 april 2026

Eerst ingediend dat voldeed aan de QC-criteria

27 april 2026

Eerst geplaatst (Werkelijk)

4 mei 2026

Updates van studierecords

Laatste update geplaatst (Werkelijk)

4 mei 2026

Laatste update ingediend die voldeed aan QC-criteria

27 april 2026

Laatst geverifieerd

1 april 2026

Meer informatie

Termen gerelateerd aan deze studie

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

NEE

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Nee

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

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