No registrations found.
ID
Source
Brief title
Health condition
Epidermolysis Bullosa
Sponsors and support
Intervention
Outcome measures
Primary outcome
• 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 outcome
• 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.
Background summary
There is an unmet need for more effective pain alleviation in EB patients. EB patient anecdotes on the use of cannabinoid-based medicines (CBMs) are in line with current science aimed at assessing the effectiveness of CBMs for chronic pain conditions. Until now evidence on the effectiveness of CBMs is moderate and is inconclusive. As the pain quality item “unpleasantness” delineates EB pain, we hypothesize the modulation of affective pain processing in the brain by way of intervention with Transvamix (comprising THC and CBD) - objectified by functional magnetic resonance imaging (fMRI).
Study objective
As the pain quality item “unpleasantness” delineates EB pain, we hypothesize the modulation of affective pain processing in the brain by way of intervention with Transvamix (a CBM comprising THC and CBD) - objectified by functional magnetic resonance imaging (fMRI).
Study design
0, 2 & 8 weeks
Intervention
Transvamix oil (THC 10%, CBD 5%)
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
- 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
Recruitment
IPD sharing statement
Plan description
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL9347 |
| Other | METC UMCG : METc: 2021076 |