Epidermolysis Bullosa
Conditions
Interventions
Transvamix oil (THC 10%, CBD 5%)
Sponsors
University Medical Center of Groningen
Eligibility
Inclusion criteria
Inclusion criteria: - Clinical diagnosis, supplemented by genetic analysis, immunofluorescent diagnostics or electron microscopy of congenital epidermolysis bullosa (EB). Including the subtypes recessive dystrophic EB, dominant dystrophic EB, junctional EB and EB simplex. - At least 16 years of age from the date of onset of participation. - Can read and write in the Dutch language. - Mentally competent and legally able to appreciate informed consent. - Reporting an average pain or pruritus mean score =4 on NRS (0-10) averaged throughout the previous week at one of the following times of day: morning, afternoon or evening. - Negative COVID-19 testing will be required prior to participation
Exclusion criteria
Exclusion criteria: - Patients enrolled in other clinical trials that do not allow for a deviation in treatment. - Have experienced myocardial infarction or clinically significant cardiac dysfunction within the last 12 months or have had a cardiac disorder that, in the opinion of the investigator would have put the participant at risk of a clinically significant arrhythmia or myocardial infarction. - Patients with known psychotic disorder (including the use of antipsychotic medications), or a history of suicidal ideation. - Female patients of child-bearing potential and male participants whose partner was of child-bearing potential, unless willing to ensure that they or their partner used effective contraception. - Patients who have had significantly impaired renal or hepatic function in the last 12 months. - The patient is currently using or has used cannabis or cannabinoid-based medications within 30 days of study entry and was unwilling to abstain for the duration of the study. - Patients unwilling or unable to refrain from driving road vehicles and/or using potentially dangerous machinery where sufficient concentration is necessary. - Patients unable to stay within the Netherlands for the duration of the study period. - History of addiction and/or hospital admission due to addiction to recreational or pharmaceutical drugs. - Patients with contradictions for MRI determined through the MRI safety form
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| •To determine the effect of Transvamix, relative to placebo, on participant reported pain scores of the quality “unpleasantness” in EB patients with chronic pain. | — |
Secondary
| Measure | Time frame |
|---|---|
| • To explore the effect of Transvamix, relative to placebo, on participant reported general pain quality scores, and pain intensity, in EB patients with chronic pain. • To explore the effect of Transvamix, relative to placebo, on pain self-efficacy in EB patients with chronic pain. • To explore the effect of Transvamix, relative to placebo on pruritus intensity in EB patients with chronic pain. • To explore the effect of Transvamix, relative to placebo on brain connectivity in areas corresponding to the affective pain modulation circuit. • To determine the sub-side effect threshold maintenance dose achieved during titration of Transvamix and placebo. • To determine the frequency and participant-reported burden of adverse-events encountered during titration of Transvamix and placebo. | — |
Contacts
Public ContactNicholas Schrader
Universitair Medisch Centrum Groningen
Outcome results
None listed