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    Summary
    EudraCT Number:2022-000506-10
    Sponsor's Protocol Code Number:APHP211036
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2022-04-19
    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 number2022-000506-10
    A.3Full title of the trial
    Bariticinib in the treatment of new-onset juvenile dermatomyositis :a phase II trial
    Baricitinib dans le traitement de la dermatomyosite juvénile d’apparition récente: un essai de phase II.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Bariticinib in the treatment of new-onset juvenile dermatomyositis :a phase II trial
    Baricitinib dans le traitement de la dermatomyosite juvénile d’apparition récente: un essai de phase II.
    A.3.2Name or abbreviated title of the trial where available
    MYOCIT
    MYOCIT
    A.4.1Sponsor's protocol code numberAPHP211036
    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 SponsorAssistance Publique des Hôpitaux de Paris
    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 supportDGOS
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAssistance Publique des Hôpitaux de Paris
    B.5.2Functional name of contact pointDRCI, Hôpital St Louis
    B.5.3 Address:
    B.5.3.1Street Address1 avenue Claude Vellefaux
    B.5.3.2Town/ cityPARIS
    B.5.3.3Post code75010
    B.5.3.4CountryFrance
    B.5.4Telephone number33144841712
    B.5.5Fax number33144841701
    B.5.6E-mailalexandra.bruneau@aphp.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 OLUMIANT® 2 mg comprimés pélliculés
    D.2.1.1.2Name of the Marketing Authorisation holderEli Lilly Nederland B.V.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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.3.4Pharmaceutical form Coated tablet
    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 INNBaricitinib
    D.3.9.3Other descriptive nameBaricitinib
    D.3.9.4EV Substance CodeSUB180983
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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
    New-onset juvenile dermatomyositis
    Dermatomyosite juvénile d'apparition récente
    E.1.1.1Medical condition in easily understood language
    New-onset juvenile dermatomyositis
    Dermatomyosite juvénile d'apparition récente
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10008521
    E.1.2Term Childhood dermatomyositis
    E.1.2System Organ Class 100000004858
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the efficacy of baricitinib in patients with a new-onset JDM at week 24 according to PRINTO 20 level of improvement
    Évaluer l'efficacité du baricitinib chez les patients présentant un DMJ d'apparition récente à la semaine 24 selon le niveau d'amélioration PRINTO 20
    E.2.2Secondary objectives of the trial
    To assess the efficacy of baricitinib :
    - according to PRINTO 20, 50, 70, and 90 levels of improvement and to the criteria of clinically inactive disease
    - on global disease activity of various extramuscular organ systems according to the MYOACT
    - on interstitial lung disease
    - on JDM-related-skin disease according to the cutaneous DM disease area and severity index
    To determine the response rate to baricitinib by the TIS using the 2016 ACR/EULAR myositis response criteria
    To assess the safety of baracitinib, the reduction in oral corticosteroids, a correlation between the muscle biopsy score and the response to baricitinib, and between PK and response to baricitinib
    To assess the following measures in order to identify biomarkers of JDM activity and predictive of the response to baricitinib at W24:
    o cytokine circulating levels independently or jointly at V0
    o genes expression within 800 genes related to immunity analyzed at V0
    To characterize the PK of baricitinib
    Evaluer l'efficacité du baricitinib :
    - selon les niveaux d'amélioration PRINTO 20, 50,70, 90 et les critères de maladie cliniquement inactive.
    - sur l'activité globale de la maladie sur les organes extra-musculaires selon l’échelle MYOACT.
    - sur la pneumopathie interstitielle.
    - sur les maladies cutanées liées à la DMJ selon l’échelle « CDASI »
    Déterminer le taux de réponse au baricitinib selon le score TIS en utilisant les critères de réponse des myosites de l’ACR/EULAR 2016
    Évaluer la sécurité du baracitinib, la réduction des corticostéroïdes oraux, la corrélation entre la PK et la réponse au baricitinib, et la corrélation entre le score de la biopsie musculaire et la réponse au baricitinib
    Évaluer les niveaux circulants de cytokines à V0 et l’expression des gènes parmi les 800 gènes liés à l'immunité analysés à V0 afin d'identifier les biomarqueurs de l'activité de la DMJ et ceux prédictifs de la réponse au baricitinib à S24.
    Caractériser la PK du baracitinib
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Patient aged 3-18 years with new-onset juvenile dermatomyositis, according to the ENMC 2018 dermatomyositis classification criteria
    - Muscle weakness at MMT and/or CMAS (MMT < 74 and/or CMAS < 45)
    - Seropositivity for chickenpox
    - Negative βHCG
    - Informed consent form signed by the patient or child’ s parents
    - Patient affiliated to a social security regime
    - Patient âgé de 3 à 18 ans atteint d'une dermatomyosite juvénile d'apparition récente, selon les critères de classification ENMC 2018 des dermatomyosites
    - Faiblesse musculaire au MMT et/ou CMAS (MMT < 74 et/ou CMAS < 45)
    - Séropositivité pour la varicelle
    - βHCG négatif
    - Formulaire de consentement éclairé signé par le patient ou les parents de l'enfant
    - Patient affilié à un régime de sécurité sociale
    E.4Principal exclusion criteria
    - Amyopathic dermatomyositis (without muscle weakness)
    - Inability to be treated by oral way or to take pills
    - Previous treatment with JAK inhibitor
    - Previous treatment of JDM with immunosuppressive drugs or biologics other than corticosteroids. Previous treatment with prednisone was allowed if the daily dose was greater than 1 mg/kg for no more than 1 month.
    - Previous history of cancer
    - Live vaccine within the 4 weeks before starting baricitinib therapy
    - Current, or recent (< 4 weeks prior to baseline) of active infections, including HBV, HCV, HIV, tuberculosis,
    - Positive blood CMV PCR
    - Creatinine clearance < 40 ml/min
    - Lymphocytes < 0,5x109 cell/L and Neutrophils < 1x109 cell/L
    - Hemoglobin < 8 g/dL
    - Symptomatic herpes herpes simplex infection within 12 weeks prior to inclusion
    - Positivity for antiphospholipids antibodies (Lupus anticoagulant and/or anti-beta2 glycoprotein 1 and/or anti-cardiolipin)
    - History of thrombosis or considered at high risk of venous thrombosis by the investigator
    - History or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, hematological, neurological or neuropsychiatric disorders or any other serious and/or instable illness that, in the opinion of the investigator, could constitute an unacceptable risk, when taking baricitinib. In particular JDM-related-acute severe respiratory distress requiring oxygen is an exclusion criteria
    - Breast-feeding, pregnancy
    - Patient on AME (state medical aid)
    - Participation in another interventional study involving human participants or being in the exclusion period at the end of a previous study involving human participants
    - Dermatomyosite amyopathique (sans faiblesse musculaire)
    - Incapacité à être traité par voie orale ou à prendre des pilules
    - Traitement antérieur avec un inhibiteur de JAK
    - Traitement antérieur de la DMJ avec des médicaments immunosuppresseurs ou des produits biologiques autres que des corticostéroïdes. Un traitement antérieur à la prednisone était autorisé si la dose quotidienne était supérieure à 1 mg/kg pendant un mois au maximum.
    - Antécédents de cancer
    - Injection de vaccin vivant dans les 4 semaines précédant le début du traitement par baricitinib
    - Infections actives actuelles ou récentes (< 4 semaines avant l'inclusion), y compris VHB, VHC, VIH, tuberculose,
    - PCR sanguine positive du CMV
    - Clairance de la créatinine < 40 ml/min
    - Lymphocytes < 0,5x109 cellules/L et Neutrophiles < 1x109 cellules/L
    - Hémoglobine < 8 g/dL
    - Infection herpès simplex symptomatique dans les 12 semaines précédant l'inclusion
    - Positivité pour les anticorps antiphospholipides (Lupus anticoagulant et/ou anti-bêta2 glycoprotéine 1 et/ou anti-cardiolipine)
    - Antécédents de thrombose ou patient considéré par l'investigateur comme présentant un risque élevé de thrombose veineuse
    - Antécédents ou présence de troubles cardiovasculaires, respiratoires, hépatiques, gastro-intestinaux, endocriniens, hématologiques, neurologiques, neuropsychiatriques ou toute autre maladie grave et/ou instable qui, de l'avis de l'investigateur, pourrait constituer un risque inacceptable, lors du traitement par baricitinib. En particulier, détresse respiratoire sévère aiguë liée à la DMJ et nécessitant de l'oxygène.
    - Allaitement, grossesse
    - Patient bénéficiant de l'AME (Aide Médicale d'État)
    - Participation à une autre étude interventionnelle impliquant des participants humains ou être dans la période d'exclusion à la fin d'une étude précédente impliquant des participants humains.
    E.5 End points
    E.5.1Primary end point(s)
    PRINTO 20 level of improvement is defined as a 20% or greater improvement in three or more of the six variables of the juvenile dermatomyositis core set, with one or no variable worsening by more than 30% (muscle strength can not be the variable worsening) :
    - muscle strength, assessed with the Childhood Myositis Assessment Scale (CMAS), with 0 the worst score and 52 the best;
    - physician’s global assessment of the patient’s disease activity on a 0–10 cm visual analogue scale (Physician’s VAS), with 0 the best score and 10 the worst;
    - global disease activity assessment through the Disease Activity Score (DAS), with 0 the best score and 20 the worst;
    - functional ability through the Childhood Health Assessment Questionnaire (C-HAQ), with 0 the best score and 3 the worst;
    - parent’s global assessment of the child’s overall wellbeing on a 10 cm visual analogue scale (Parent’s VAS), with 0 representing very good well-being and 10 being very poor wellbeing;
    - health-related quality of life, through the parent version of the Child Health Questionnaire (CHQ-Phs), with a low score indicating worse quality of life
    Le niveau d'amélioration PRINTO 20 est défini comme une amélioration d’au moins 20% et d’au moins trois des six variables de l'ensemble de base de la dermatomyosite juvénile, avec une ou aucune variable s'aggravant de plus de 30% (la force musculaire ne peut pas être la variable s'aggravant) :
    - Force musculaire, évaluée à l'aide de l'échelle d'évaluation de la myosite infantile (CMAS), 0 étant le pire score et 52 le meilleur ;
    - Évaluation globale par le médecin de l'activité de la maladie du patient sur une échelle visuelle analogique de 0 à 10 cm (EVA du médecin), 0 étant le meilleur score et 10 le pire ;
    - Évaluation globale de l'activité de la maladie au moyen du Disease Activity Score (DAS), 0 étant le meilleur score et 20 le pire ;
    - Capacité fonctionnelle grâce au Childhood Health Assessment Questionnaire (C-HAQ), le meilleur score étant 0 et le pire 3 ;
    - L’évaluation globale du bien-être de l'enfant par les parents sur une échelle visuelle analogique de 10 cm (EVA des parents), 0 étant le meilleur score et 10 le pire;
    - La qualité de vie liée à la santé, au moyen de la version parentale du Child Health Questionnaire (CHQ-Phs), un score faible indiquant une moins bonne qualité de vie.
    E.5.1.1Timepoint(s) of evaluation of this end point
    at week 24
    à la semaine 24
    E.5.2Secondary end point(s)
    1) The achievement of the PRINTO 20 levels of improvement
    2) The achievement of the PRINTO 50, 70 and 90 levels of improvement
    3) Relative and absolute variations of TIS
    4) Clinically inactive disease according to the PRINTO criteria
    5) Relative and absolute variations of CDSAI
    6) Relative and absolute variations of MYOACT
    7) Improvement of interstitial lung disease if present at screening: Improvement of pulmonary function tests (improvement of at least 10% of FCV, PTC, and DLCO) and/or improvement of Lung tomodensitomery scored according to a specific scale
    8) Dose of corticosteroids at week 24
    9) Non-compartmental analysis of baricitinib PK
    10)Correlation between PK of baricitinib and disease activity ‘s scores
    11) Measurement of serum IFN - α, IFN-γ, IL-1β, IL-4, Il-5, IL-6, IL-8, IL-10, IL-12p70, IL-22, TNF α
    12) Study of genes expression within 800 genes related to immunity
    13) Assessment of muscle biopsies according to the internationally validated score system (Dr Gitiaux)
    1) L'atteinte du niveau d'amélioration PRINTO 20
    2) L'atteinte des niveaux d'amélioration PRINTO 50, 70 et 90
    3) Les variations relatives et absolues du TIS
    4) Maladie cliniquement inactive selon les critères PRINTO.
    5) Variations relatives et absolues du CDSAI .
    6) Variations relatives et absolues du MYOACT
    7) Amélioration de la pneumopathie interstitielle si elle est présente lors du dépistage : Amélioration des tests de la fonction pulmonaire (amélioration d'au moins 10% du FCV, du PTC et du DLCO) et/ou amélioration de la tomodensitométrie pulmonaire notée selon une échelle spécifique.
    8) Dose de corticostéroïdes à la semaine 24
    9) Analyse non compartimentale de la pharmacocinétique du baricitinib
    10) Corrélation entre la pharmacocinétique du baricitinib et les scores d'activité de la maladie.
    11) Mesure des taux sériques d'IFN -, IFN-γ, IL-1β, IL-4, Il-5, IL-6, IL-8, IL-10, IL-12p70, IL-22, TNF α
    12) Étude de l'expression de 800 gènes liés à l'immunité
    13) Évaluation des biopsies musculaires selon le système de score validé au niveau international (Dr Gitiaux).
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) at weeks 4, 8, 12, 16
    2) at weeks 4, 8, 12, 16, 24
    3) between inclusion and weeks 4, 8, 12, 16, 24
    4) at weeks 4, 8, 12, 24
    5) between inclusion and weeks 4, 8, 12, 16, 24
    6) between inclusion and weeks 4, 8, 12, 16, 24
    7) at screening
    8) at week 24
    11)at inclusion, weeks 4 and 24.
    12) at inclusion, weeks 4 and 24.
    1) aux semaines 4, 8, 12, 16
    2) aux semaines 4, 8, 12, 16, 24
    3) entre l'inclusion et les semaines 4, 8, 12, 16, 24
    4) aux semaines 4, 8, 12, 24
    5) entre l'inclusion et les semaines 4, 8, 12, 16, 24
    6) entre l'inclusion et les semaines 4, 8, 12, 16, 24
    7) au screening
    8) à la semaine 24
    11) à l'inclusion, semaines 4 et 24.
    12) à l'inclusion, semaines 4 et 24.
    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 Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    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 concerned13
    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 months37
    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: 16
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 8
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 No
    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 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)
    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 Decision2022-06-02
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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