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

NCT07564245 | Not Yet Recruiting Phase 2

• 2 other identifiers: APHP2400902025-521161-27-00
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Neurodevelopmental Disorders With Severe Self-injuries

Keywords

Neurodevelopmental disorders; 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); Epidyolex; Non Communicating Children's Pain; Pain evaluation; Parental stress

Outcome Measures

Primary Outcomes (1)

• Efficacy of cannabidiol

  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

Secondary Outcomes (20)

• Efficacy of cannabidiol

  Day 29

• Efficacy of cannabidiol / severity of self-injuries

  Day 29

• Efficacy of cannabidiol / severity of self-injuries

  Day 63

• Efficacy of cannabidiol / severity of Stereotyped behaviour

  Day 29

• Efficacy of cannabidiol / severity of Stereotyped behaviour

  Day 63

Study Arms (1)

Cannabidiol

EXPERIMENTAL

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.

Drug: Cannabidiol

Interventions

Cannabidiol DRUG

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.

Also known as: Epidyolex®

Cannabidiol

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• \. Participant related to a person involved in the study at the investigational site

Sponsors & Collaborators

• URC-CIC Paris Descartes Necker Cochin: collaborator
• Fondation Perce Neige: collaborator
• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (2)

Hôpital Pitié-Salpêtrière

Paris, Île-de-France Region, 75013, France

Location

Hopital Necker Enfants malades

Paris, Île-de-France Region, 75015, France

Location

MeSH Terms

Conditions

Neurodevelopmental Disorders

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Mental Disorders

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Study Officials

• Pauline CHASTE, MD; PhD

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

• Maryse PAGNIER, MD; PhD

  Assistance Publique - Hôpitaux de Paris

  STUDY DIRECTOR

Central Study Contacts

Pauline CHASTE, MD; PhD

CONTACT

01 44 49 57 59; pauline.chaste@aphp.fr

Laure CHOUPEAUX, Msc

CONTACT

01 44 38 17 11; laure.choupeaux@aphp.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 27, 2026
• First Posted: May 4, 2026
• Study Start: May 1, 2026
• Primary Completion (Estimated): May 1, 2029
• Study Completion (Estimated): February 1, 2030
• Last Updated: May 4, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2)