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    Summary
    EudraCT Number:2022-001012-26
    Sponsor's Protocol Code Number:ATYR1923-C-004
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-08-17
    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 ConcernedSpain - AEMPS
    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
    Estudio de fase 3, aleatorizado, en doble ciego y controlado con placebo, para evaluar la eficacia y la seguridad de efzofitimod intravenoso en pacientes con sarcoidosis pulmonar
    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
    Estudio para evaluar la efectividad y la seguridad de efzofitimod en pacientes con sarcoidosis pulmonar
    A.3.2Name or abbreviated title of the trial where available
    EFZO-FIT
    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
    Sarcoidosis pulmonar
    E.1.1.1Medical condition in easily understood language
    Pulmonary sarcoidosis
    Sarcoidosis pulmonar
    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
    Evaluar la eficacia del efzofitimod en pacientes con sarcoidosis pulmonar
    E.2.2Secondary objectives of the trial
    To assess the safety and tolerability of efzofitimod in patients with pulmonary sarcoidosis
    Evaluar la seguridad y tolerabilidad del efzofitimod en pacientes con sarcoidosis pulmonar
    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
    - Diagnóstico confirmado de sarcoidosis pulmonar desde hace al menos 6 meses, definido por los siguientes criterios: diagnóstico de sarcoidosis documentado y demostrado histológicamente por biopsia de tejido y pruebas documentadas de afectación del parénquima pulmonar mediante pruebas radiológicas previas.
    - Pruebas de sarcoidosis pulmonar sintomática, demostrada por los siguientes criterios: grado de la escala de disnea modificada del Consejo de Investigaciones Médicas (MRC, por sus siglas en inglés) de al menos 1 y Puntuación del KSQ-Lung <=70.
    - Los pacientes deberán estar recibiendo tratamiento con OCS desde hace >= 3 meses con una dosis inicial entre >=7,5 y <=25 mg/día.
    - Peso corporal >=40 kg y <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
    - Tratamiento con >1 inmunosupresor oral.
    - Tratamiento con inmunomoduladores biológicos, como los inhibidores del factor de necrosis tumoral alfa (FNT-alfa) o antifibróticos o inhibidores de la interleucina.
    - Probabilidad de fibrosis pulmonar significativa, como lo demuestran uno o más de los siguientes factores: Fibrosis en la TAC de alta resolución >20 % en la Selección; FVC <50 % del teórico y Puntuación del KSQ-Lung <30.
    - Hipertensión pulmonar clínicamente significativa que requiera tratamiento con vasodilatadores.
    - Pacientes con sarcoidosis cardíaca, neurosarcoidosis o sarcoidosis renal.
    - Sarcoidosis cutánea u ocular clínicamente significativas.
    - Antecedentes de síntomas adisonianos que impidieron los intentos anteriores de reducción progresiva de los OCS.
    - Fumador activo y empedernido de productos que contienen tabaco/nicotina.
    - Antecedentes de síndrome antisintetasa o positivo para Jo-1 en el momento basal.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in mean daily OCS dose post-taper
    Cambio con respecto al valor basal en la dosis media diaria de OCS después de su reducción progresiva
    E.5.1.1Timepoint(s) of evaluation of this end point
    At week 48
    En la semana 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
    - Tasa anual de cambio del valor absoluto de la FVC
    - Porcentaje de cambio con respecto al valor basal en la dosis media diaria de OCS después de su reducción progresiva.
    - Cambio de la puntuación del KSQ-Lung desde el valor basal en la Semana 48.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At week 48
    En la semana 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 concerned3
    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
    última visita último paciente (LVLS)
    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 months3
    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 state20
    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.
    Después de completar la visita de fin de estudio (EOS), los pacientes pueden tener la opción de participar en un estudio de extensión ciego.
    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 Decision2022-10-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-10-04
    P. End of Trial
    P.End of Trial StatusOngoing
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