Genetic Epidermolysis Bullosa
Conditions
Interventions
Product Name: Transvamix (100mg/mL THC, 50mg/mL CBD)
Pharmaceutical Form: Concentrate for oromucosal solution
INN or Proposed INN: THC
Other descriptive name: DELTA-9-TETRAHYDROCANNABINOL
Concentratio
Sponsors
University Medical Center Groningen
Eligibility
Sex/Gender
All
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. - COVID-19 testing will be required prior to participation if deemed necessary by governing guidelines. Are the trial subjects under 18? yes Number of subjects for this age range: 2 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 12 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
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 |
|---|---|
| Main Objective: 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 Objective: 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. ;Primary end point(s): • A reduction of 30% and 50% of mean pain scores (of the pain quality “unpleasantness”) between Transvamix and placebo group. Will be measured as the difference in mean scores pre- (baseline) and post- intervention (Transvamix and placebo) for each group using the pain quality item “unpleasantness” from the pain quality assessment scale (PQAS), a 20-item numerical rating scale (NRS 0-10) survey on pain qualities. ;Timepoint(s) of evaluation of this end point: Baseline (day -1) End of first exposure to Transvamix OR placebo (day 14) End of second exposure to Transvamix OR placebo (day 42) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • The difference of mean scores of pain qualities between Transvamix and placebo. Will be measured as the difference of the mean of all PQAS pain quality items (which have received a score >0 on NRS) between Transvamix and Placebo. • The difference of mean scores of pain intensity between Transvamix and placebo. Will be measured as daily changes in pain intensity (1-14 days placebo, 1-14 days Transvamix) using the VAS-Pain ([no pain] 0-100 [worst pain imaginable] unit scale) for pain intensity. The change score (delta) from baseline to Transvamix (day 14) and Placebo (day 14) will be calculated. • To determine the effect of Transvamix on pain self-efficacy Will be measured using the PSEQ survey pre-intervention (1x baseline) and during intervention (1x Transvamix, 1x placebo), a 10-item survey on the ability to continue normal life given ongoing pain. • The difference of mean scores of pruritus intensity between Transvamix and placebo. Will be measured as daily changes in pruritus intensity (1-14 days placebo, 1-14 days Transvamix) using the VAS-Pruritus ([no pruritus] 0-100 [worst pruritus imaginable] unit scale) for pruritus intensity. The change score (delta) from baseline to Transvamix (day 14) and Placebo (day 14) will be calculated. • The difference in brain connectivity of the seed regions (anterior cingulate cortex and amygdala) to the brain cortex between Transvamix and placebo. Will be measured using voxel-based analysis of blood oxygen-level dependent functional magnetic resonance imaging (fMRI-BOLD). • *To determine sub-side effect threshold maintenance dose achieved of Transvamix during titration and the respective interindividual variability (CV%). Will be measured as the dose administered on day 14 of placebo and Transvamix interventions. CV% is measured as inter-participant variance of dose achieved on day 14 of placebo and Transvamix interventions. • ^To inventorize how many and which participant-experienced adverse-ev | — |
Countries
Netherlands
Contacts
Public ContactPrincipal Investigator
University Medical Center Groningen
Outcome results
None listed