Evaluation of the Efficacy of CANNABIDIOL on the Pruritus in Children With Hereditary Epidermolysis Bullosa

Experimental scheme M-3 to M-1: pre-inclusion Proposal of the study and information of the families followed in the dermatology department during a visit as part of the care pathway. Explanation of protocol.

D0: inclusion

During conventional hospitalization or day hospitalization of the patient in the dermatology department for monitoring of hereditary epidermolysis bullosa (EBH) :

• Signature of the consent of the legal guardians after verification of the eligibility criteria and information given on the protocol
• Collection of pruritus and pain scores
• Clinical skin examination with measurement of the severity score of epidermolysis bullosa EBDASI (Epidermolysis Bullosa Disease Activity and Scarring Index).
• List of treatments and medical devices of the patient
• Quality of sleep measured by the patient or his family on a scale of 0 to 10
• Quality of life measured by the patient or his family via the child DLQI questionnaire

• st intake of CANNABIDIOL D0 to D4: 5 mg/kg/day in 3 doses (morning, noon and evening). on D5: If efficacy (mean VAS for pruritus <3 on D4), continue at the same dosage. If ineffective or partially effective (VAS pruritus ≥3) and in the event of good tolerance, increase to 10 mg/kg/day in 3 doses (morning, noon and evening).

on D10: If efficacy (mean VAS pruritus <3 on D9), continue at the same dosage. If ineffective or partially effective (VAS pruritus ≥3), and in the event of good tolerance, increase to 20 mg/kg/day in 3 doses (morning, noon and evening) if previous dosage at 10 mg/kg/day, or increase to 10 mg/kg/day if previous dosage at 5 mg/kg/day.

In case of intolerance at D5, D10, D14 or D21: decrease to the previous dosage, or interruption if the dosage was 5 mg/kg/day.

D30(+/-2): end of treatment consultation The end-of-treatment consultation takes place D30 (+/-2) after the start of treatment.

• Collection of pruritus and pain scores
• Clinical skin examination with measurement of the EBDASI score
• Quality of sleep measured by the patient or his family on a scale of 0 to 10
• Quality of life measured by the patient or his family via the child DLQI questionnaire
• During a systematic blood test, collection of two more tubes for CANNABIDIOL dosage and a liver test

D48 (+2): phone call by the investigating doctor to monitor the occurrence of adverse events

Inclusion criteria

• Minor patient between 2 and 17 years and 10 months
• Suffering from distrophic recessive epidermolysis bullosa
• Patient weight less than or equal to 40 kg
• With pruritus not relieved by conventional treatments with mean VAS greater than or equal to 4/10 the 3 days preceding inclusion
• No change in treatment or care for at least one month
• Consent of parents
• Affiliated to social security

Exclusion criteria

• Hypersensitivity to the active substance or to any of the excipients (Refined sesame oil, anhydrous ethanol, sucralose, strawberry flavor, includes benzyl alcohol)
• Consumption of cannabis or cannabidiol
• Severe renal impairment defined by GFR less than 29 ml/min
• Moderate to severe hepatic impairment defined by a Child-Pugh B or C score or an AST and/or ALT level greater than 3 times normal and/or bilirubin more than 2 times normal
• with clinically or ultrasound sign(s) of moderate to severe cardiac insufficiency, defined by LVEF less than 45% and stage II to IV of the NYHA classification
• Taking a tricyclic antidepressant treatment with anti-H4 antihistamine action or a neurokinin-1 receptor antagonist in the previous month
• Participating to an interventional research (category 1 or 2)
• Modification of at least one background treatment in the previous month
• Proven pregnancy or breastfeeding patient
• Patient deprived of their liberty by decision of a judicial or administrative authority (Article L. 1121-6 of the Public Health Code)