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    Summary
    EudraCT Number:2020-004281-19
    Sponsor's Protocol Code Number:1.5
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-10-22
    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 ConcernedSweden - MPA
    A.2EudraCT number2020-004281-19
    A.3Full title of the trial
    Investigator initiated clinical trial of dantrolene as a treatment for Darier disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Investigator initiated clinical trial of dantrolene as a treatment for Darier disease
    A.4.1Sponsor's protocol code number1.5
    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 SponsorRegion Stockholm Karolinska University Hospital (Dep. of Dermatology)
    B.1.3.4CountrySweden
    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 supportRegion Stockholm / Karolinska University Hospital (Dep. of Dermatology)
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRegion Stockholm Karolinska University Hospital (Dep. of Dermatology)
    B.5.2Functional name of contact pointJakob Wikström
    B.5.3 Address:
    B.5.3.1Street AddressEugeniavägen 3
    B.5.3.2Town/ cityStockholm
    B.5.3.3Post code17164
    B.5.3.4CountrySweden
    B.5.4Telephone numberSwede739611019
    B.5.6E-mailjakob.wikstrom@ki.se
    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 Yes
    D.2.1.1.1Trade name Dantrium (dantrolene)
    D.2.1.2Country which granted the Marketing AuthorisationSweden
    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 nameDantrium (dantrolene)
    D.3.2Product code ATC code M03CA01
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDantrolene sodium
    D.3.9.1CAS number 14663-23-1
    D.3.9.4EV Substance CodeSUB01553MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Darier disease is a rare and severe autosomal dominant skin disorder.
    E.1.1.1Medical condition in easily understood language
    Darier disease is a rare and severe autosomal dominant skin disorder.
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    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
    Primary outcome
    Does dantrolene treatment reduce skin area affected?
    The primary outcome will be reduction of skin area affected using the Eczema Area and Severity Index (EASI; scale of 0-72). A 20 % reduction will be considered the minimal clinically important difference.
    E.2.2Secondary objectives of the trial
    Secondary outcome
    Does dantrolene treatment improve patient quality of life?


    As a secondary outcome, improvement in dermatological quality of life index (DLQI) will be used. A 4 points improvement in DLQI will be considered the minimal clinically important difference.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    DD research subjects will be recruited at the dermatology clinic at the Karolinska University hospital, by advertisement in the Swedish Society for Dermato-Venereology as well as from the very active patient social media networks. Since DD is a familial disorder, participants will also be recruited through previous included participants. We plan to recruit 14 participants.
    The inclusion criteria will be histopathologically verified diagnosis combined with typical skin symptoms of hyperkeratotic papules and erythema, or clinical symptoms combined with family history in which the relative has a histopathologically verified diagnosis. Written consent to participate in the study will also be an inclusion criteria. For female participants, adequate contraception should be used. A negative pregnancy test (blood-HCG) will be a requirement. Contraceptive requirements may also apply to male participants.
    E.4Principal exclusion criteria
    Subjects must not be included in the study if any of the following criteria are met:
    Pregnancy, breastfeeding, or planned pregnancy.
    Recent acute illness (past 4 weeks)
    Active substance abuse
    Kidney or liver disease including steatosis, cirrhosis and hepatitis B/C
    Heart failure
    Pulmonary disease such as COPD (but not common asthma)
    Abnormal blood chemistry (assed by blood samples)
    Known or suspected allergies against dantrolene or vehicle
    Systemic treatment for Dariers disease that cannot be paused during the study (e.g. acitretin)
    Mental inability, reluctance or language difficulties that result in difficulty understanding the meaning of study participation
    Participation or recent participation in a clinical study with an investigational product (within 30 days).
    Concomitant Medication that may interact:
    antihistamines (e.g., cetirizine, doxylamine, diphenhydramine, hydroxyzine, loratadine)
    antipsychotics (e.g., chlorpromazine, clozapine, haloperidol, olanzapine, quetiapine, risperidone)
    apalutamide
    benzodiazepines (e.g., alprazolam, diazepam, lorazepam)
    bosentan
    brimonidine
    buspirone
    calcium channel blockers (e.g., amlodipine, diltiazem, verapamil)
    cannabis

    chloral hydrate
    dabrafenib
    deferasirox
    dexamethasone
    efavirenz
    enzalutamide
    estrogens (e.g., conjugated estrogen, estradiol, ethinyl estradiol)
    etravirine
    methadone
    mirtazapine
    mitotane
    modafinil
    muscle relaxants (e.g., baclofen, cyclobenzaprine, methocarbamol, orphenadrine)
    nabilone
    narcotic pain relievers (e.g., codeine, fentanyl, morphine, oxycodone)
    pramipexole
    rifabutin
    rifampin
    ropinirole
    rotigotine
    St. John's wort
    scopolamine
    seizure medications (e.g., carbamazepine, clobazam, levetiracetam, phenobarbital, phenytoin, primidone, topiramate, valproic acid, zonisamide)
    selective serotonin reuptake inhibitors (SSRIs; e.g., citalopram, fluoxetine, paroxetine, sertraline)
    siltuximab
    sodium oxybate
    tapentadol
    thalidomide
    tocilizumab
    tramadol
    tricyclic antidepressants (e.g., clomipramine, desipramine, imipramine)
    zolpidem
    zopiclone
    E.5 End points
    E.5.1Primary end point(s)
    The study ends when the last subject (the 14th) has completed the last follow-up. The study may be prematurely terminated if it appears that the treatment involved a large number of serious adverse events (SAE) or if recruitment of subjects cannot be met within reasonable time limits. If the study is prematurely terminated or suspended, the investigator should immediately inform the subjects about this and ensure appropriate treatment and follow-up. The regulatory authority should be informed as soon as possible, but no later than within 15 days. We view early termination as unlikely since dantrolene is an established clinical drug and we will only use 12.5 % (100 mg) of the maximal dose (800 mg).
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 6 months of active treatment plus 3 months until the follow up visit, i.e. 9 months.
    E.5.2Secondary end point(s)
    Same as above.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Same as above.
    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 Yes
    E.6.4Safety No
    E.6.5Efficacy No
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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
    The study ends when the last subject (the 14th) has completed the last follow-up.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 14
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 14
    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 Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state14
    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)
    Not different.
    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 Decision2022-06-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-11
    P. End of Trial
    P.End of Trial StatusOngoing
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