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    The EU Clinical Trials Register currently displays   44293   clinical trials with a EudraCT protocol, of which   7351   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2022-001012-26
    Sponsor's Protocol Code Number:ATYR1923-C-004
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-08-10
    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 number2022-001012-26
    A.3Full title of the trial
    A Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of intravenous efzofitimod in patients with pulmonary sarcoidosis
    Etude de phase 3, randomisée, en double aveugle, contrôlée contre placebo, évaluant l’efficacité et la sécurité d’un traitement par efzofitimod intraveineux chez des patients atteints de sarcoïdose pulmonaire
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate the effectiveness and safety of efzofitimod in patients with pulmonary sarcoidosis
    Etude évaluant l’efficacité et la sécurité du efzofitimod chez des patients atteints de sarcoïdose pulmonaire
    A.3.2Name or abbreviated title of the trial where available
    EFZO-FIT
    A.4.1Sponsor's protocol code numberATYR1923-C-004
    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 SponsoraTyr Pharma, Inc.
    B.1.3.4CountryUnited States
    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 supportaTyr Pharma, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationaTyr Pharma, Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address3545 John Hopkins Court, Suite #250
    B.5.3.2Town/ citySan Diego
    B.5.3.3Post codeCA 92121
    B.5.3.4CountryUnited States
    B.5.4Telephone number18587318389
    B.5.6E-mailclinicaltrials@atyrpharma.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 nameEfzofitimod
    D.3.2Product code ATYR1923
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEfzofitimod
    D.3.9.1CAS number 2566615-11-8
    D.3.9.2Current sponsor codeATYR1932
    D.3.9.3Other descriptive nameKRP-R120
    D.3.9.4EV Substance CodeSUB199413
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 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 No
    D.3.11.9Recombinant medicinal product Yes
    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 placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Pulmonary sarcoidosis
    Sarcoïdose pulmonaire
    E.1.1.1Medical condition in easily understood language
    Pulmonary sarcoidosis
    Sarcoïdose pulmonaire
    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 PT
    E.1.2Classification code 10037430
    E.1.2Term Pulmonary sarcoidosis
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    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 efzofitimod in patients with pulmonary sarcoidosis
    Evaluer l’efficacité de l’efzofitimod chez des patients atteints de sarcoïdose pulmonaire
    E.2.2Secondary objectives of the trial
    To assess the safety and tolerability of efzofitimod in patients with pulmonary sarcoidosis
    Evaluer la sécurité et la tolérance de l’efzofitimod chez des patients atteints de sarcoïdose pulmonaire
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Confirmed diagnosis of pulmonary sarcoidosis for at least 6 months, defined by the following criteria: documented histologically proven diagnosis of sarcoidosis by tissue biopsy and documented evidence of parenchymal lung involvement by historical radiological evidence

    - Evidence of symptomatic pulmonary sarcoidosis, as demonstrated by the following criteria: Modified Medical Research Council (MRC) dyspnea scale grade of at least 1 and KSQ-Lung score ≤70

    - Patients must be receiving treatment with OCS of ≥ 3 months with a starting dose between ≥ 7.5 and ≤ 25 mg/day.

    - Body weight ≥ 40 kg and < 160 kg

    - Diagnostic confirmé de sarcoïdose pulmonaire depuis au moins 6 mois, définie par les critères suivants : diagnostic documenté de sarcoïdose histologiquement prouvée par biopsie tissulaire et preuve d’atteinte pulmonaire parenchymateuse documentée par les examens radiologiques historiques

    - Mise en évidence d’une sarcoïdose pulmonaire symptomatique, démontrée par les critères suivants : échelle de dyspnée modifiée du MRC (Medical Research Council) de grade d’au moins 1 et score KSQ-Poumon ≤ 70

    - Les patients doivent recevoir un traitement par corticoïdes oraux (CO) ≥ 3 mois avec une dose de départ entre ≥ 7,5 et ≤ 25 mg/jour

    - Poids corporel ≥ 40 kg et < 160 kg

    E.4Principal exclusion criteria
    - Treatment with > 1 oral immunosuppressant therapy

    - Treatment with biological immunomodulators, such as tumor necrosis factor-alpha (TNF-α) inhibitors or antifibrotics or interleukin inhibitors

    - Likelihood of significant pulmonary fibrosis as shown by any 1 or more of the following: High resolution CT fibrosis > 20% at Screening; FVC % predicted < 50% and KSQ-Lung score < 30

    - Clinically significant pulmonary hypertension requiring treatment with vasodilators

    - Patients with cardiac sarcoidosis, neurosarcoidosis, or renal sarcoidosis

    - Clinically significant cutaneous and ocular sarcoidosis

    - History of Addisonian symptoms that precluded previous OCS taper attempts

    - Is an active, heavy smoker of tobacco/nicotine-containing products

    - History of anti-synthetase syndrome or Jo-1 positive at baseline
    - Traitement par > 1 agent immunosuppresseur oral

    - Traitement par immunomodulateurs biologiques, tels que inhibiteurs du facteur de nécrose tumorale-alpha (TNF-α), antifibrotiques ou inhibiteurs d’interleukines

    - Probabilité de fibrose pulmonaire significative démontrée par au moins 1 des critères suivants : TDM à haute résolution montrant une fibrose > 20 % à la sélection, valeur prédite CVF % < 50 % et score KSQ-Poumon < 30

    - Hypertension pulmonaire cliniquement significative nécessitant un traitement par vasodilatateurs

    - Sarcoïdose cardiaque, neurosarcoïdose ou sarcoïdose rénale

    - Sarcoïdose cutanée et oculaire cliniquement significative

    - Antécédents de symptômes addisoniens ayant empêché les tentatives précédentes de diminution progressive de CO

    - Gros fumeur actif de tabac/produits contenant de la nicotine

    - Antécédents de syndrome anti-synthétase (anti-Jo-1) ou positivité Jo-1 à l’inclusion
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in mean daily OCS dose post-taper
    Modification, par rapport aux valeurs initiales, de la dose quotidienne moyenne de CO après diminution progressive
    E.5.1.1Timepoint(s) of evaluation of this end point
    At week 48
    Semaine 48
    E.5.2Secondary end point(s)
    • Annual rate of change in absolute value of FVC
    • Percent change from baseline in mean daily OCS post-taper
    • Change from baseline in KSQ-Lung score at Week 48
    • Taux annuel de modification de la valeur absolue de CVF
    • Modification en pourcentage, par rapport aux valeurs initiales, de la dose quotidienne moyenne de CO après diminution progressive
    • Modifications, par rapport aux valeurs initiales, du score KSQ-Poumon à la Semaine 48
    E.5.2.1Timepoint(s) of evaluation of this end point
    At week 48
    Semaine 48
    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 Yes
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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
    Japan
    Puerto Rico
    United States
    France
    Netherlands
    Spain
    Germany
    Italy
    United Kingdom
    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
    Dernière visite du dernier patient
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days0
    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: 211
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 53
    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 state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 107
    F.4.2.2In the whole clinical trial 264
    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)
    After completion of the EOS visit, patients may have the option to participate in a blinded extension study.
    Après la visite de fin d'étude, les patients pourront avoir la possibilité de participer à une étude d’extension en aveugle.
    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 Decision2022-10-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-01-17
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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