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    Summary
    EudraCT Number:2021-000103-20
    Sponsor's Protocol Code Number:202000638
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-07-15
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2021-000103-20
    A.3Full title of the trial
    An explorative randomized, placebo-controlled and double-blind intervention crossover study:
    Transvamix (100mg/mL THC / 50mg/mL CBD) to treat chronic pain in Epidermolysis Bullosa
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study on the effect of Transvamix on pain in adults with epidermolysis bullosa.
    Een onderzoek naar het Effect van Transvamix op Pijn bij Volwassenen met Epidermolysis Bullosa.
    A.4.1Sponsor's protocol code number202000638
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorUniversity Medical Center Groningen
    B.1.3.4CountryNetherlands
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportDEBRA-UK
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity Medical Center Groningen
    B.5.2Functional name of contact pointPrincipal Investigator
    B.5.3 Address:
    B.5.3.1Street AddressHanzeplein 1
    B.5.3.2Town/ cityGroningen
    B.5.3.3Post code9713GZ
    B.5.3.4CountryNetherlands
    B.5.6E-maila.p.wolff@umcg.nl
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameTransvamix (100mg/mL THC, 50mg/mL CBD)
    D.3.4Pharmaceutical form Concentrate for oromucosal solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSublingual use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTHC
    D.3.9.3Other descriptive nameDELTA-9-TETRAHYDROCANNABINOL
    D.3.9.4EV Substance CodeSUB27785
    D.3.10 Strength
    D.3.10.1Concentration unit % percent
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCBD
    D.3.9.1CAS number 13956-29-1
    D.3.9.3Other descriptive nameCANNABIDIOL
    D.3.9.4EV Substance CodeSUB26600
    D.3.10 Strength
    D.3.10.1Concentration unit % percent
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product Yes
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboConcentrate for oromucosal solution
    D.8.4Route of administration of the placeboSublingual use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Genetic Epidermolysis Bullosa
    E.1.1.1Medical condition in easily understood language
    Inherited blistering skin condition
    E.1.1.2Therapeutic area Diseases [C] - Symptoms and general pathology [C23]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the effect of Transvamix, relative to placebo, on participant reported pain scores of the quality “unpleasantness” in EB patients with chronic pain.
    E.2.2Secondary objectives of the trial
    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.
    E.2.3Trial contains a sub-study No
    E.3Principal 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.
    E.4Principal 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
    E.5 End points
    E.5.1Primary 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.
    E.5.1.1Timepoint(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)
    E.5.2Secondary 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-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]).
    E.5.2.1Timepoint(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)

    *End of first exposure to Transvamix OR placebo (day 14)
    *End of second exposure to Transvamix OR placebo (day 42)

    ^ Continuous (Day 1 to 42)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 2
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 12
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally Information not present in EudraCT
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state16
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Regular treatment/care will continue as per care as usual. Participation in trial does not alter this.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-07-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-07-15
    P. End of Trial
    P.End of Trial StatusOngoing
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