Hereditary Epidermolysis BullosaPhase 2EBCBDNCT05651607
Evaluation of the Efficacy of CANNABIDIOL on the Pruritus in Children With Hereditary Epidermolysis Bullosa
A Phase 2 study testing Cannabidiol in people with Hereditary Epidermolysis Bullosa, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Hereditary Epidermolysis Bullosa
- What is being tested?
- Cannabidiol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteFrance
- Study dates
- 7 Sep 2023to 17 Nov 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Show all 10Show fewer
- 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)
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the detailed description
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
- Conditions
- Hereditary Epidermolysis Bullosa
- Being tested
- Cannabidiol
- Keywords
- hereditary epidermolysis bullosa, Cannabidiol, Prutitus, Pilot Study
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CannabidiolDrug
Also called Epidyolex
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
| Study group | Role | What they receive |
|---|---|---|
| Cannabidiol (Epidyolex) | Experimental | Not described Receives: Drug: Cannabidiol |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Day 30
Secondary outcomes (10)
Change of pruritus Visual Analog Scale scores
Change of the mean pruritus VAS scores recorded on D27, D28 and D29 compared to the mean pruritus VAS scores on D-3, D-2 and D-1 . The scale is scored in a range of 0-10 with 10 representing worst imaginable pruritus.
Measured: Day 30
Evaluation of tolerance of Cannabidiol
Serious and non-serious side effects
Measured: Day 48
Clinically significant decrease of chronic pain scores
Loss of at least 2 points on the mean pain scores (VAS or FLACC according to the age of patient) recorded on D27, D28 and D29 compared to the mean pain scores recorded on D-3, D-2 and D-1 (D0 = inclusion and start of treatment). The Face, Legs, Activity, Cry, Consolability (FLACC) scale is a measurement used to assess pain for children between the ages of 2 months and 7 years or individuals that are unable to communicate their pain. The scale is scored in a range of 0-10 with 10 representing severe pain.
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 pain.
Measured: Day 30
Change of chronic pain scores
Change of the mean pain scores (Visual Analogue Scale or Face, Legs, Activity, Cry, Consolability scale according to the age of patient) recorded on D27, D28 and D29 compared to the mean pain scores on D-3, D-2 and D-1 The Face, Legs, Activity, Cry, Consolability (FLACC) scale is a measurement used to assess pain for children between the ages of 2 months and 7 years or individuals that are unable to communicate their pain. The scale is scored in a range of 0-10 with 10 representing severe pain.
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 pain.
Measured: Day 30
Change of pruritus Visual Analog Scale score during cares
Change of the mean of maximum pruritus VAS scores recorded during the last two cares before D30 compared to the mean of maximum pruritus VAS scores recorded during the last two cares before D0. 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.
Measured: Day 30
Change of acute pain score during cares
Change of the mean of maximum acute pain scores (VAS or FLACC according to the age of patient) recorded during the last two cares before D30 compared to the mean of maximum acute pain scores recorded during the last two cares before D0. The Face, Legs, Activity, Cry, Consolability (FLACC) scale is a measurement used to assess pain for children between the ages of 2 months and 7 years or individuals that are unable to communicate their pain. The scale is scored in a range of 0-10 with 10 representing severe pain.
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 pain.
Measured: Day 30
Change of quality of sleep
Quality of sleep measured by the patient or his family on a scale of 0 to 10, with 10 representing excellent sleep quality, at D30 compared to D0
Measured: Day 30
Change of quality of life
Quality of life measured by the patient or his family via the Dermatology Life Quality Index (DLQI child) at D30 compared to D0.
The Dermatology Life Quality Index (DLQI child) is scored in a range of 0-30 with 30 representing extremely important effect on the patient's quality life.
Measured: Day 30
Systemic passage of CANNABIDIOL
Systemic passage measured at D30 by blood test 4 hours after the morning intake and before the midday intake of CANNABIDIOL.
Measured: Day 30
Acceptability of CANNABIDIOL
Measure of acceptability: difficulty of ingesting, difficulty of using the syringe, taste
Measured: Day 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2022
- Study start7 Sep 2023
- Primary completion17 Nov 2023
- Study completion17 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (11)Papers the sponsor has linked to this study.
- Other trials for Hereditary Epidermolysis BullosaStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- HELEBOR, Fondation Apicil, Lions Club International Foundation, URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
- Investigators
- Christine BODEMER, MD, PhD, Study director, Assistance Publique - Hôpitaux de ParisLara MAYRAND, Resident, Study chair, Assistance Publique - Hôpitaux de ParisCéline GRECO, MD, PhD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05651607
- Sponsor's protocol ID
- APHP220741
- Other IDs
- 2022-003411-28
- Acronym
- EBCBD
- Status last verified
- Mar 2026
- First submitted
- 7 Dec 2022
- Last updated
- 5 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 35199092Sheriff 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.
PubMed 31599175Eagleston 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.
PubMed 30142706Avila 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.
PubMed 31987788Stander 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.
PubMed 16874533Okusanya 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.
PubMed 32723354Schrader 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.
PubMed 34488820Chelliah 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.
PubMed 29786144Schrader 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.
PubMed 30347109Welponer 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.
PubMed 35593277Mayrand 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.
PubMed 41105816
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Efficacy of CANNABIDIOL on the Pruritus in Children With Hereditary Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT05651607
- Acronym / sponsor ID
- EBCBD / APHP220741
- Conditions
- Hereditary Epidermolysis Bullosa
- Keywords
- hereditary epidermolysis bullosa, Cannabidiol, Prutitus, Pilot Study
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- HELEBOR, Fondation Apicil, Lions Club International Foundation, URC-CIC Paris Descartes Necker Cochin
- Study start
- 7 Sep 2023 (actual)
- Primary completion
- 17 Nov 2023 (actual)
- Study completion
- 17 Nov 2023 (actual)
- First posted
- 15 Dec 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cannabidiol (Epidyolex) | Experimental | Drug: Cannabidiol |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CannabidiolAlso: EpidyolexOral 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 | Drug | Cannabidiol (Epidyolex) |
Primary outcome
Clinically significant decrease of pruritus Visual Analog Scale (VAS) scores
Time frame: Day 30
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.
10 secondary outcomes
Change of pruritus Visual Analog Scale scores
Time frame: Day 30
Evaluation of tolerance of Cannabidiol
Time frame: Day 48
Clinically significant decrease of chronic pain scores
Time frame: Day 30
Change of chronic pain scores
Time frame: Day 30
Change of pruritus Visual Analog Scale score during cares
Time frame: Day 30
Change of acute pain score during cares
Time frame: Day 30
Change of quality of sleep
Time frame: Day 30
Change of quality of life
Time frame: Day 30
Systemic passage of CANNABIDIOL
Time frame: Day 30
Acceptability of CANNABIDIOL
Time frame: Day 30
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2026.
- Background: PubMed 35199092Sivesind 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.
- Background: PubMed 31599175Sheriff 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.
- Background: PubMed 30142706Eagleston 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.
- Background: PubMed 31987788Avila 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.
- Background: PubMed 16874533Stander 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.
- Background: PubMed 32723354Okusanya 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.
- Background: PubMed 34488820Schrader 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.
- Background: PubMed 29786144Chelliah 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.
- Background: PubMed 30347109Schrader 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.
- Background: PubMed 35593277Welponer 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.
- Results publication: PubMed 41105816Mayrand 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.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christine BODEMER, MD, PhD study director
Assistance Publique - Hôpitaux de Paris
Lara MAYRAND, Resident study chair
Assistance Publique - Hôpitaux de Paris
Céline GRECO, MD, PhD principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cannabidiol in people with Hereditary Epidermolysis Bullosa, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinically significant decrease of pruritus Visual Analog Scale (VAS) scores (time frame: Day 30). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05651607, which also lists 11 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Mar 2026.