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    Summary
    EudraCT Number:2020-005948-33
    Sponsor's Protocol Code Number:CDFV890A12201
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2022-01-17
    Trial results View 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 number2020-005948-33
    A.3Full title of the trial
    An open-label, single arm phase II study of DFV890 to assess the safety, tolerability and efficacy in participants with familial cold auto-inflammatory syndrome (FCAS)
    Etude de phase II, en ouvert, à un seul bras, pour évaluer la sécurité d’emploi, la tolérance et l’efficacité du DFV890 chez des patients atteints d’un syndrome
    familial auto-inflammatoire au froid (FCAS)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of safety, tolerability and efficacy of DFV890 in participants with familial cold auto-inflammatory syndrome (FCAS)
    A.4.1Sponsor's protocol code numberCDFV890A12201
    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 SponsorNovartis Pharma AG
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma S.A.S
    B.5.2Functional name of contact pointInformation&Communication Médicales
    B.5.3 Address:
    B.5.3.1Street Address8/10 rue Henry Sainte Claire Deville, CS 40150
    B.5.3.2Town/ cityRueil-Malmaison
    B.5.3.3Post code92563
    B.5.3.4CountryFrance
    B.5.4Telephone number+33 1 5547 6600
    B.5.5Fax number+33 1 5547 6100
    B.5.6E-mailicm.phfr@novartis.com
    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 nameDFV890
    D.3.2Product code DFV890
    D.3.4Pharmaceutical form Film-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 INNNot yet established
    D.3.9.2Current sponsor codeDFV890
    D.3.9.4EV Substance CodeSUB208521
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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
    Familial Cold Autoinflammatory Syndrome (FCAS)
    Urticaire familiale au froid
    E.1.1.1Medical condition in easily understood language
    Familial Cold Autoinflammatory Syndrome (FCAS)
    Urticaire familiale au froid
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10068850
    E.1.2Term Cryopyrin associated periodic syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the efficacy of DFV890 to reduce cold-induced inflammation in participants with FCAS.
    Evaluer l'efficacité du DFV890 dans la réduction de l'inflammation induite par le froid chez des patients atteints de FCAS.
    E.2.2Secondary objectives of the trial
    - To assess safety and tolerability of DFV890.
    - To assess the efficacy of DFV890 to improve the signs and symptoms of FCAS.
    - To assess the effect of DFV890 on patient reported outcomes.
    - Evaluer la sécurité d’emploi et la tolérance du DFV890 ;
    - Evaluer l'efficacité du DFV890 dans l’amélioration des signes et symptômes d'un FCAS ;
    - Evaluer l'effet du DFV890 sur les résultats rapportés par le patient (PRO);
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Written informed consent must be obtained before any study-specific assessment is performed.
    - Body mass index within the range of 18-35 kg/m2.
    - Patients with a genetic diagnosis of FCAS.
    - Patients with a clinical history and investigations consistent with FCAS.
    -Recueil du formulaire de consentement éclairé par écrit et signé avant toute évaluation spécifique à l'étude.
    - Indice de masse corporelle (IMC) compris entre 18 et 35 kg/m2 (IMC = poids corporel (kg) / [taille (m)]2) au moment de la sélection.
    -Diagnostic génétique de FCAS (cette analyse peut être réalisée dans le cadre de la sélection si elle n'est pas déja disponible dans le dossier du patient).
    -Antécédents et investigations cliniques compatibles avec la FCAS, en l'absence d'antécédents ou de diagnostic d'amylose et/ou de lesions organiques (par ex. surdité, oedème périorbitaire, lymphadénopathie et sérite).
    E.4Principal exclusion criteria
    - Participants currently being treated with anti-rejection and/or immunomodulatory drugs and the treatment cannot be discontinued or switched to a different medication.
    - Clinically significant, suspected active or chronic bacterial (including Mycobacterium tuberculosis), viral or fungal infection within 30 days prior to dosing.
    - Patients with innate (e.g. TLR immunodeficiencies, defects in IFN-γ signaling) or acquired immune deficiencies (e.g. AIDS).
    - Presence of human immunodeficiency virus (HIV) infection, hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (anti-HBc), or hepatitis C antibodies at screening.
    - Live vaccines within 4 weeks of dosing.
    - Pregnant or nursing (lactating) women.
    - Women of child-bearing potential unless they are using highly effective methods of contraception.
    Other protocol-defined inclusion/exclusion criteria may apply.
    - Traitement actuel par des médicaments anti-rejet et/ou immunomodulateurs qui ne peut pas être interrompu ou remplacé par un autre médicament dans les 28 jours ou 5 demi-vies (la durée la plus longue prévalant si la réglementation locale l'exige), ou jusqu'à ce que l’effet pharmacodynamique attendu soit revenu à la baseline pour les anticorps thérapeutiques immunomodulateurs, avant le test de dépistage par provocation au froid et pendant la durée de l'étude.
    - Infection bactérienne, virale ou fongique cliniquement significative, suspectée active ou chronique (y compris Mycobacterium tuberculosis) dans les 30 jours précédant l'administration.
    -Déficits immunitaires innés (par ex. Toll-Like Récepteurs (TLR), défauts de la voie de signalisation de l'interféron gamma (IFN-γ)) ou déficits immunitaires acquis (par ex. SIDA).
    -Présence d'une infection par le virus de l'immunodéficience humaine (VIH), de l'antigène de surface de l'hépatite B (AgHBs) ou de l’antigène de la nucléocapside du virus de l'hépatite B (anti-HBc), ou d'anticorps de l'hépatite C à la sélection.
    -Vaccination par vaccins vivants dans les 4 semaines précédant le jour 1 (c.-à-d. l première dose de DFV890).
    -Femmes en âge de procréer, à savoir toute femme physiologiquement apte à être enceinte, sauf si elles utilisent des méthodes de contraception très efficaces pendant toute la durée du traitement et pendant 10 jours après l’arrêt du traitement
    à l'étude.
    D'autres critères d'inclusion/exclusion définis par le protocole peuvent s'appliquer.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline for white cell count (WCC).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Pre-baseline to post-baseline.
    E.5.2Secondary end point(s)
    - Number and severity of safety assessments and adverse events.
    - Change from baseline in physician assessed disease scores.
    - Change from baseline in Patient’s global assessment.
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Number and severity of safety assessments and adverse events: up to End of Study.
    - Change from baseline in physician assessed disease scores: pre-baseline to post-baseline
    - Change from baseline in Patient’s global assessment: pre-baseline to post-baseline.
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability.
    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 Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA3
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    France
    Germany
    Italy
    United Kingdom
    United States
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days3
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days28
    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: 3
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 4
    F.4.2.2In the whole clinical trial 6
    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)
    Continuing care should be provided by the investigator and/or referring physician. No further study treatment will be provided.
    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 Decision2022-02-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-03-01
    P. End of Trial
    P.End of Trial StatusCompleted
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