The primary objective is to determine the effect of Transvamix on the pain quality item *unpleasantness*, relative to placebo. Secondary objectives include the assessment of general pain qualities, pain self-efficacy, general pain and pruritus…
ID
Source
Brief title
Condition
- Skin and subcutaneous tissue disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The effect of Transvamix, relative to placebo measured by a reduction in the
pain quality item *unpleasantness* (using the SF-MPQ2) by 30% and 50%.
• 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 SF-MPQ2, a 22-item numerical rating scale (NRS
0-10) survey on pain qualities.
Secondary outcome
• The difference of mean scores of pain qualities between Transvamix and
placebo.
Will be measured as the difference of the mean of all SF-MPQ2 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-events
during titration and maintenance of Transvamix and the patient reported burden
for each adverse event on numeric rating scale (NRS [not burdensome at all]
0-10 [extremely burdensome]).
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 (a CBM comprising THC and CBD) -
objectified by functional magnetic resonance imaging (fMRI).
Study objective
The primary objective is to determine the effect of Transvamix on the pain
quality item *unpleasantness*, relative to placebo. Secondary objectives
include the assessment of general pain qualities, pain self-efficacy, general
pain and pruritus intensity, functional brain connectivity, adverse events and
sub-side effect threshold maintenance dose achieved during titration.
Study design
The study is a two-arm randomized, double-blind, placebo controlled, crossover
intervention study.
Intervention
All participants will receive Transvamix-oil comprising 100mg/mL THC and
50mg/mL CBD, and placebo, to be administered sublingually by a 1mL dosing
syringe.
Study burden and risks
Participants are 16 years or older and mentally competent. They will receive
the study intervention Transvamix-oil, a compounded medication (magistrale
bereiding) readily available on prescription and is currently used by patients
for various medical indications in the Netherlands. Transvamix-oil can produce
side effects that can be discomforting, but are mild, well tolerated, and
disappear within 4-8 hours after administration upon which the following dose
can be reduced to mitigate the recurrence of side-effects. Participants will
undergo 3x 30 minute MRI scans. A daily logbook will be completed at home
electronically - pertaining to the dose administered, possible side-effects and
pain/pruritus scores. All participant reported outcomes are completed
electronically at home.
Hanzeplein 1
Groningen 9700RB
NL
Hanzeplein 1
Groningen 9700RB
NL
Listed location countries
Age
Inclusion criteria
- Clinical diagnosis, supplemented by genetic analysis, immunofluorescent
diagnostics or electron microscopy of congenital epidermolysis bullosa (EB).
Including the EB types 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
Medical products/devices used
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 |
|---|---|
| EudraCT | EUCTR2021-000103-20-NL |
| CCMO | NL76471.042.21 |