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    Summary
    EudraCT Number:2020-003268-25
    Sponsor's Protocol Code Number:RC31/19/0504
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-08-10
    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 ConcernedFrance - ANSM
    A.2EudraCT number2020-003268-25
    A.3Full title of the trial
    Hydroxychloroquine in isolated cutaneous mastocytosis patients or indolent systemic mastocytosis with associated skin involvement patients: proof of concept study
    Hydroxychloroquine dans la mastocytose cutanée isolée ou systémique indolente avec atteinte cutanée associée : étude de preuve de concept
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Hydroxychloroquine in isolated cutaneous mastocytosis patients or indolent systemic mastocytosis with associated skin involvement patients: proof of concept study
    Hydroxychloroquine dans la mastocytose cutanée isolée ou systémique indolente avec atteinte cutanée associée : étude de preuve de concept
    A.3.2Name or abbreviated title of the trial where available
    HCQMa
    HCQMa
    A.4.1Sponsor's protocol code numberRC31/19/0504
    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 SponsorCHU Toulouse
    B.1.3.4CountryFrance
    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 supportGIRGI-SOHO
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCHU Toulouse
    B.5.2Functional name of contact pointFlorine LEGAY
    B.5.3 Address:
    B.5.3.1Street Address2 rue viguerie - TSA 80035
    B.5.3.2Town/ cityToulouse cedex 9
    B.5.3.3Post code31059
    B.5.3.4CountryFrance
    B.5.4Telephone number+3305-61-77-84-34
    B.5.5Fax number05-61-77-84-11
    B.5.6E-maillegay.f@chu-toulouse.fr
    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 Plaquenil
    D.2.1.1.2Name of the Marketing Authorisation holdersanofi-aventis France
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 namePlaquenil (Hydroxychloroquineà
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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
    mastocytosis
    mastocytose
    E.1.1.1Medical condition in easily understood language
    mastocytosis
    mastocytose
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10042949
    E.1.2Term Systemic mastocytosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate at 12 months the effect of hydroxychloroquine administered at a dose of 6 to 6.5mg / kg / day on mast cell activation symptoms as pruritus and / or flush moderate to severe in patients with isolated cutaneous mastocytosis and / or indolent systemic with associated skin involvement.
    L’objectif principal de cette étude sera d’évaluer à 12 mois l’effet de l’hydroxychloroquine administrée à la dose de 6 à 6,5mg/kg/jour sur les symptômes d’activation mastocytaire type prurit et/ou flush modéré à sévère chez les patients atteints d’une mastocytose cutanée isolée et/ou systémique indolente avec atteinte cutanée associée
    E.2.2Secondary objectives of the trial
    1) To assess the objective response rate of mast cell mass at 12 months.
    2) To assess the effect of hydroxychloroquine on other symptoms of mast cell activation such as: diarrhea, pollakiuria, arthralgia and discomfort at 12 months.
    3) To assess the tolerance of hydroxychloroquine.
    4) To assess the association between changes in HCQ blood levels and changes in symptoms of mast cell activation
    1) D’évaluer le taux de réponse objective de la masse mastocytaire à 12 mois.
    2) D’évaluer l’effet de l’hydroxychloroquine sur les autres symptômes d’activation mastocytaire comme : diarrhées, pollakiurie, arthralgies et malaises à 12 mois.
    3) D’évaluer la tolérance de l’hydroxychloroquine.
    4) D’évaluer l’association entre l’évolution du taux sanguin d’HCQ et l’évolution des symptômes d’activation mastocytaire.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Age > 18 years
    2) Isolated Cutaneous mastocytosis or indolent systemic mastocytosis with associated skin lesions defined according to WHO criteria (and / or international standards for cutaneous mastocytosis)
    3) Patient with at least one disability defined by the presence of the following symptoms assessed as moderate to severe:
    a. Cutaneous pruritus with score ≥ 5 on a VAS scale from 0 to 10
    b. Number of flushs / week ≥ 7
    4) Skin KIT mutation known
    5) Performance scale: OMS/ECOG ≤ 1
    6) Woman and man of childbearing age under effective contraception
    1) Age supérieur à 18 ans
    2) Mastocytose cutanée ou systémique indolente avec une atteinte cutanée associée définie selon les critères OMS (et/ou les critères internationaux pour la mastocytose cutanée)
    3) Patient avec au moins un handicap défini par la présence d’un des symptômes suivants évalués comme modéré à sévère :
    a. Prurit cutané avec score ≥ 5 sur une échelle EVA de 0 à 10
    b. Nombre de flushs/semaine ≥ 7
    4) Statut de la mutation kit connu dans la peau
    5) Echelle de performance : OMS/ECOG ≤ 1
    6) Femme et homme en âge de procréer sous contraception efficace
    E.4Principal exclusion criteria
    1) Non-symptomatic mastocytosis and / or without skin involvement
    2) Systemic mastocytosis with associated hematologic neoplasm (SM-AHN)
    3) Aggressive systemic mastocytosis
    4) Mast cell leukemia
    5) History of ophthalmic disease and / or cardiac conduction disorders against-indicating the use of hydroxychloroquine
    6) Treatment with citalopram, escitalopram, hydroxyzine, domperidone, piperaquine due to the increased risk of ventricular rhythm disorders, especially torsades de pointes
    7) Specific anti-tumor treatment (chemotherapy, radiotherapy) of less than 4 weeks before inclusion.
    8) Concomitant specific anti-mast cell treatment
    9) Inclusion in another trial with an experimental therapeutic molecule
    10) Change symptomatic treatment (including dosage) in the 4 weeks preceding the inclusion visit
    11) Inability to give informed consent
    12) Inability to undergo medical monitoring for geographical, social or psychic
    13) Patients with major surgery scheduled in the next two weeks screening
    14) Patient without health insurance
    15) Pregnancy, Breastfeeding
    16) Vulnerable Patient, defined as:
    - Esperance survival < 6 months
    - Patient with another uncontrolled severe disease
    17) Patient under juridical protection
    1) Mastocytose non symptomatique et/ou sans atteinte cutanée associée
    2) Mastocytose systémique associée à une autre hémopathie néoplasique (MS-AHN)
    3) Mastocytose systémique agressive
    4) Leucémie mastocytaire
    5) Antécédents de maladie ophtalmologique et/ou de troubles de conduction cardiaque contre-indiquant l’utilisation d’hydroxychloroquine
    6) Traitement par citalopram, escitalopram, hydroxyzine, dompéridone, pipéraquine en raison du risque majoré de troubles du rythme ventriculaire, notamment de torsades de pointe
    7) Traitement spécifique anti tumoral (chimiothérapie, radiothérapie) de moins de 4 semaines précédant l'inclusion.
    8) Traitement concomitant anti mastocytaire spécifique
    9) Inclusion dans un autre essai thérapeutique avec une molécule expérimentale (pendant la présente étude)
    10) Changement de traitement symptomatique (y compris du dosage) dans les 4 semaines précédant la visite d’inclusion
    11) Impossibilité de donner un consentement éclairé
    12) Impossibilité de se soumettre à un suivi médical pour des raisons géographique, sociale ou psychique
    13) Patient ayant une chirurgie majeure programmée dans les deux semaines suivantes le screening
    14) Patient sans couverture sociale
    15) Grossesse, allaitement
    16) Patient vulnérable, défini comme :
    - Esperance de survie < 6 mois
    - Patient avec une autre maladie sévère non-contrôlée
    17) Patient sous un régime de protection juridique des majeurs (sauvegarde de justice, tutelle, curatelle, institutionnalisées, ou sous mandat de protection future)

    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of this study is the change of mast cell activation symptoms as pruritus and/or flushes between the start of treatment and 12 months later.
    Le critère de jugement principal de cette étude sera la variation des symptômes d’activation mastocytaire type prurit et/ou flush entre le début du traitement et 12 mois plus tard.
    E.5.1.1Timepoint(s) of evaluation of this end point
    12months after the tratment begining
    12 mois après le début du traitement
    E.5.2Secondary end point(s)
    The secondary endpoints are:
    - The difference on mast cell mass between the start of treatment and 12 months later.
    - The difference of other mast cell activation symptoms such as diarrhea, pollakiuria, arthralgia and discomfort between the start of treatment and 12 months later.
    - The safety of hydroxychloroquine treatment.
    - The correlation of the effectiveness of treatment with the hydroxychloroquine and level of serum HCQ will be performed by the Bland-Altman test.
    Les critères de jugement secondaires seront :
    - la variation de la masse mastocytaire entre le début du traitement et 12 mois plus tard.
    - la variation des autres symptômes d’activation mastocytaire comme les diarrhées, la pollakiurie, les arthralgies et les malaises entre le début du traitement et 12 mois plus tard.
    - la tolérance par un examen clinique et si besoin paraclinique selon la décision de l’investigateur; la tolérance sera évaluée pendant la période de l’étude.
    - le taux sanguin d’hydroxychloroquine
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 months after the begining of the treatment and during the treatment
    12 mois après le début du traitement et pendant le traitement
    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 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 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.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    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 No
    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
    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 months25
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months25
    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: 30
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 30
    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 state30
    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)
    None
    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 Decision2020-10-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-10-08
    P. End of Trial
    P.End of Trial StatusOngoing
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