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

NCT05651607 | Completed Phase 2

• 2 other identifiers: APHP2207412022-003411-28
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

Hereditary epidermolysis bullosa (HEB) is a heterogeneous group of rare genetic diseases, characterized by fragility of the skin and mucous membranes, which results in the appearance of mucocutaneous bullae and erosions during minimal trauma. Pruritus is a neuropathic pain mainly related to activation of unmyelinated cutaneous C nerve fibers and is very common in patients with HEB. It is the cause of trophic disorders, aggravation of certain wounds, appearance of new bubbles. In addition, this chronic pruritus can also have a major psychological impact on the patient and his family. However, these therapies used in the pruritus of patients with HEB have often proven to be ineffective. In order to improve the quality of life of children and their families, research into new therapies to limit this chronic pruritus is necessary. Among phytocannabinoids, CANNABIDIOL (CBD) should be clearly distinguished from Delta-9-tetrahydrocannabinol (THC). Indeed, CBD is an "inverse" agonist of the CB2 receptor, it acts by reducing the effect of this receptor, while THC is an agonist of the CB1 and CB2 receptors. Thus, CBD has antipsychotic, anxiolytic, antiemetic, anti-inflammatory and anti-epileptic effects, unlike THC which has psychotic, relaxation effects, impairs cognitive function and memory. Cannabinoids are involved in the physiopathology in pruritus at the level of the peripheral nervous system via the CB1 and TRPV1 receptors, and also at the level of the central nervous system thanks to the CB1 and CB2 receptors. In addition, inflammation plays an important role in the physiopathology of pruritus and this is reduced via the activation of CB2 receptors, expressed in immune cells. Various studies with promising results have examined the effect of cannabinoids in pruritus. No serious adverse effects have been reported and the rare adverse effects that have been observed are reversible upon discontinuation of treatment. The research project seeks to estimate the efficacy of CANNABIDIOL in the pruritus of 10 children with severe hereditary epidermolysis bullosa. Pruritus is assessed before the start of treatment, then after one month of taking oral treatment, three times a day. The effectiveness of taking the treatment will also be assessed on pain, on the impact on sleep and on overall quality of life. The tolerance of CANNABIDIOL will be well monitored. The systemic passage of CANNABIDIOL is measured during a routine blood test 1 month after treatment.

Conditions

Hereditary Epidermolysis Bullosa

Keywords

hereditary epidermolysis bullosa; Cannabidiol; Prutitus; Pilot Study

Outcome Measures

Primary Outcomes (1)

• Clinically significant decrease of pruritus Visual Analog Scale (VAS) scores

  Loss of at least 2 points on the mean pruritus VAS scores recorded on D27, D28 and D29 compared to the mean pruritus scores recorded on D-3, D-2 and D-1 (D0 = inclusion and start of treatment). The Visual Analogue Scale (VAS) is a scale consisting of a 10cm long line and a single question. The scale is scored in a range of 0-10 with 10 representing worst imaginable pruritus.

  Day 30

Secondary Outcomes (10)

• Change of pruritus Visual Analog Scale scores

  Day 30

• Evaluation of tolerance of Cannabidiol

  Day 48

• Clinically significant decrease of chronic pain scores

  Day 30

• Change of chronic pain scores

  Day 30

• Change of pruritus Visual Analog Scale score during cares

  Day 30

Study Arms (1)

Cannabidiol (Epidyolex)

EXPERIMENTAL

Drug: Cannabidiol

Interventions

Cannabidiol DRUG

Oral solution, taken 3 times a day (morning, noon and evening), 5 mg/kg/day from day D0 to day D4. If not effective and well tolerated, dose increase : 10 mg/kg/day from day D5 to day D9, then 20 mg/kg/day from day D10 to day D29, maximum 800 mg/day

Also known as: Epidyolex

Cannabidiol (Epidyolex)

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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
• No change in treatment or care for at least one month
• Consent of parents
• Affiliated to social security

You may not qualify if:

• 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)

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• HELEBOR: collaborator
• Fondation Apicil: collaborator
• Lions Club International Foundation: collaborator
• URC-CIC Paris Descartes Necker Cochin: collaborator

Study Sites (1)

Hôpital Necker Enfants Malades

Paris, 75015, France

Location

Related Publications (11)

• Sivesind TE, Maghfour J, Rietcheck H, Kamel K, Malik AS, Dellavalle RP. Cannabinoids for the Treatment of Dermatologic Conditions. JID Innov. 2022 Jan 13;2(2):100095. doi: 10.1016/j.xjidi.2022.100095. eCollection 2022 Mar.

  PMID: 35199092 BACKGROUND

• Sheriff T, Lin MJ, Dubin D, Khorasani H. The potential role of cannabinoids in dermatology. J Dermatolog Treat. 2020 Dec;31(8):839-845. doi: 10.1080/09546634.2019.1675854. Epub 2019 Oct 10.

  PMID: 31599175 BACKGROUND

• Eagleston LRM, Kalani NK, Patel RR, Flaten HK, Dunnick CA, Dellavalle RP. Cannabinoids in dermatology: a scoping review. Dermatol Online J. 2018 Jun 15;24(6):13030/qt7pn8c0sb.

  PMID: 30142706 BACKGROUND

• Avila C, Massick S, Kaffenberger BH, Kwatra SG, Bechtel M. Cannabinoids for the treatment of chronic pruritus: A review. J Am Acad Dermatol. 2020 May;82(5):1205-1212. doi: 10.1016/j.jaad.2020.01.036. Epub 2020 Jan 25.

  PMID: 31987788 BACKGROUND

• Stander S, Reinhardt HW, Luger TA. [Topical cannabinoid agonists. An effective new possibility for treating chronic pruritus]. Hautarzt. 2006 Sep;57(9):801-7. doi: 10.1007/s00105-006-1180-1. German.

  PMID: 16874533 BACKGROUND

• Okusanya BO, Asaolu IO, Ehiri JE, Kimaru LJ, Okechukwu A, Rosales C. Medical cannabis for the reduction of opioid dosage in the treatment of non-cancer chronic pain: a systematic review. Syst Rev. 2020 Jul 28;9(1):167. doi: 10.1186/s13643-020-01425-3.

  PMID: 32723354 BACKGROUND

• Schrader NHB, Gorell ES, Stewart RE, Duipmans JC, Harris N, Perez VA, Tang JY, Wolff AP, Bolling MC. Cannabinoid use and effects in patients with epidermolysis bullosa: an international cross-sectional survey study. Orphanet J Rare Dis. 2021 Sep 6;16(1):377. doi: 10.1186/s13023-021-02010-0.

  PMID: 34488820 BACKGROUND

• Chelliah MP, Zinn Z, Khuu P, Teng JMC. Self-initiated use of topical cannabidiol oil for epidermolysis bullosa. Pediatr Dermatol. 2018 Jul;35(4):e224-e227. doi: 10.1111/pde.13545. Epub 2018 May 22.

  PMID: 29786144 BACKGROUND

• Schrader NHB, Duipmans JC, Molenbuur B, Wolff AP, Jonkman MF. Combined tetrahydrocannabinol and cannabidiol to treat pain in epidermolysis bullosa: a report of three cases. Br J Dermatol. 2019 Apr;180(4):922-924. doi: 10.1111/bjd.17341. Epub 2018 Nov 14.

  PMID: 30347109 BACKGROUND

• Welponer T, Diem A, Nahler G, Laimer M. Purified oral cannabidiol for pain management in severe recessive dystrophic epidermolysis bullosa. Indian J Dermatol Venereol Leprol. 2022 May-Jun;88(4):551-552. doi: 10.25259/IJDVL_71_2021. No abstract available.

  PMID: 35593277 BACKGROUND

• Mayrand L, Bonigen J, Marquant F, Schinkel N, Bellon N, Bataille P, Fettouche I, Chaumon S, Elie C, Bodemer C, Greco C. Therapeutic efficacy of cannabidiol on pruritus and pain in children with recessive dystrophic epidermolysis bullosa: a pilot study. Br J Dermatol. 2026 Jan 27;194(2):375-378. doi: 10.1093/bjd/ljaf400. No abstract available.

  PMID: 41105816 RESULT

MeSH Terms

Interventions

Cannabidiol

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Study Officials

• Christine BODEMER, MD, PhD

  Assistance Publique - Hôpitaux de Paris

  STUDY DIRECTOR

• Lara MAYRAND, Resident

  Assistance Publique - Hôpitaux de Paris

  STUDY CHAIR

• Céline GRECO, MD, PhD

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

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: December 7, 2022
• First Posted: December 15, 2022
• Study Start: September 7, 2023
• Primary Completion: November 17, 2023
• Study Completion: November 17, 2023
• Last Updated: March 5, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)