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    The EU Clinical Trials Register currently displays   42869   clinical trials with a EudraCT protocol, of which   7063   are clinical trials conducted with subjects less than 18 years old.
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    EudraCT Number:2018-004244-33
    Sponsor's Protocol Code Number:ATYR1923-C-002
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-07-15
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedGermany - PEI
    A.2EudraCT number2018-004244-33
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled Multiple Ascending Dose Study of Intravenous ATYR1923 in Patients with Pulmonary Sarcoidosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A safety study with multiple doses of intravenous (in the vein) ATYR1923 in patients with pulmonary sarcoidosis
    A.4.1Sponsor's protocol code numberATYR1923-C-002
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03824392
    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 organisationVoisin Consulting
    B.5.2Functional name of contact pointClinical Trial Operations
    B.5.3 Address:
    B.5.3.1Street Address64 avenue Pierre Grenier
    B.5.3.2Town/ cityBoulogne-Billancourt
    B.5.3.3Post code92100
    B.5.4Telephone number+4121 353 79 20
    B.5.5Fax number+4121 353 78 01
    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 nameATYR1923
    D.3.2Product code ATYR1923
    D.3.4Pharmaceutical form 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 INNNot yet available
    D.3.9.1CAS number N/A
    D.3.9.2Current sponsor codeATYR1923
    D.3.9.3Other descriptive nameiMod.Fc, H-Fcv3-N4
    D.3.10 Strength
    D.3.10.1Concentration unit g/l gram(s)/litre
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    E.1.1.1Medical condition in easily understood language
    The disease is characterized by the buildup of immune system cells in organs that form small clusters called granulomas, a type of inflammation of the involved tissues.
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to evaluate the safety and tolerability of multiple ascending intravenous (IV) doses of ATYR1923 in patients with pulmonary sarcoidosis.
    E.2.2Secondary objectives of the trial
    The secondary objectives are:
    - To assess the potential steroid-sparing effect of multiple ascending doses of ATYR1923 in patients with pulmonary sarcoidosis.
    - To assess the potential immunogenicity of multiple ascending IV doses of ATYR1923 in patients with pulmonary sarcoidosis.
    - To characterize the pharmacokinetics (PK) of multiple ascending IV doses of ATYR1923 in patients with pulmonary sarcoidosis.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female patients aged ≥18 to ≤70 years inclusive at time of informed consent
    2. Diagnosis of pulmonary sarcoidosis for ≥6 months (cutaneous and ocular involvement allowed), defined as:
    • Histologically proven diagnosis of sarcoidosis by bronchoscopy, biopsy (any organ) or bronchioalveolar lavage.
    • Evidence of parenchymal lung involvement by historical radiological evidence (eg, computed tomography [CT], magnetic resonance imaging [MRI], 18F-FDG PET/CT or chest X-ray) or on the Screening 18F-FDG PET/CT.
    3. Must have symptomatic and/or active pulmonary sarcoidosis as evidence by:
    • Clinical findings of dyspnea, as indicated by a Modified Medical Research Council (MRC) Dyspnea Scale grade of at least 1; and
    • FVC% predicted ≥50%; and
    • Positive 18F-FDG PET/CT scan showing increased metabolic activity (maximum standardized update value [SUVmax] >1) in lung parenchyma within 4 weeks prior to Day 1.
    4. Must be receiving treatment with 10 to 25 mg/day of oral prednisone (or oral equivalent; eg, methylprednisolone), at a stable dose for ≥4 weeks prior to Day 1, and be determined by the Investigator to be capable of undergoing the protocol-specified steroid taper regimen.
    • Treatment with one oral immunomodulatory therapy (eg, methotrexate, azathioprine, hydroxychloroquine) at a stable dose for ≥1 months prior to Day 1 is allowed but not required. The dose of this therapy should remain constant throughout the study.
    5. Body weight ≥45 kg and <160 kg.
    6. Female patients may be of childbearing potential or of non-childbearing potential (either surgically sterilized or at least 1 year postmenopausal (confirmed by amenorrhea duration of at least 12 months and serum follicle-stimulating hormone [FSH] ≥30 mIU/mL).
    • Female patients of childbearing potential must be non-pregnant and non-lactating, and have a negative pregnancy test at Screening (serum) and at Day 1 (urine) prior to first study drug infusion.
    • Additionally, female patients of childbearing potential must be willing to use highly-effective contraception from Screening until 90 days after the last follow-up visit.
    7. Male patients, if not infertile or surgically sterilized, must agree to use highly-effective contraception and not donate sperm from Screening until 90 days after the last follow-up visit.
    8. Provision of written informed consent.
    9. Be able to communicate well with the Investigator and site personnel, and agree to comply with all study procedures and requirements.
    E.4Principal exclusion criteria
    1. Current disease presentation consistent with Lofgren’s syndrome (ie, presence of the triad of erythema nodosum, bilateral hilar lymphadenopathy on chest X-ray, and joint pain).
    2. History of severe allergic or anaphylactic reactions to therapeutic proteins or known sensitivity to ATYR1923 or to its inactive components (L histidine, sodium chloride, sucrose, L-methionine, and polysorbate-20).
    3. Treatment (within 6 months of Screening) with biological immunomodulators such as tumor necrosis factor-alpha (TNF-α) inhibitors (eg, infliximab, adalimumab).
    4. Current evidence of clinically significant cardiovascular, neurological, hepatic, renal, hematological, lymphatic, metabolic, or gastrointestinal disease, or has a condition that requires other treatment, may not allow safe participation, or which in the opinion of the Investigator should preclude the patient’s participation in the clinical study.
    5. Clinically significant pulmonary hypertension requiring vasodilator treatment.
    6. Any history of tuberculosis or evidence of active systemic non-tuberculous fungal or mycobacterial infection within 1 year of Screening.
    7. History of clinically significant cardiac, neurological, gastrointestinal, and/or renal manifestations of their sarcoidosis.
    8. Any history of malignancy.
    9. Major surgery within 3 months prior to Day 1 or anticipated surgery during the study.
    10. Any condition that necessitated hospitalization within the 3 months prior to Day 1 or is likely to require so during the study.
    11. Participation in another clinical study of an investigational agent or device within 3 months (small molecules) / 6 months (biologics) or 5 half-lives (if known) of the agent, whichever is longer.
    12. History of or positive results of screening for hepatitis B (hepatitis B surface antigen [HBsAg]), hepatitis C (anti-hepatitis C virus [HCV] antibodies [Ab]) or human immunodeficiency virus (HIV) (HIV Ab type 1 and 2).
    13. Has been an active smoker (including e-cigarettes or e-vaporizers) within 3 months prior to Screening.
    14. Active substance abuse (drugs or alcohol) or history of substance abuse within the 12 months prior to Screening.
    15. Clinically significant abnormalities in the Screening physical examination, vital signs, ECG, or clinical laboratory test results that, in the opinion of the Investigator and Medical Monitor should preclude the patient’s participation in the clinical study.
    16. Patient has received a live vaccination within 8 weeks before Day 1 or inoculation with a live vaccine is planned during study participation.
    17. Jo-1 Ab levels >7 U/mL at Screening, or past history of Jo-1 Ab positivity.
    18. Any other condition or circumstance that, in the opinion of the investigator, would be likely to prevent adequate compliance with the study protocol.
    19. Significant and/or acute illness (eg, change in pulmonary status, infection requiring antibiotics) within 5 days prior to (the first) drug administration that may impact safety assessments, in the opinion of the Investigator.
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability of multiple IV doses of ATYR1923 in patients with pulmonary sarcoidosis based on the following analyses:
    • Incidence of TEAEs and serious adverse events (SAEs).
    E.5.1.1Timepoint(s) of evaluation of this end point
    From Day 1 to end of study.
    E.5.2Secondary end point(s)
    Potential to Decrease Background Oral Corticosteroid Dose
    • Total cumulative steroid dose administered over the primary study period (Day 1 through Week 24).
    • Number of patients who achieve the targeted tapered dose of prednisone 5 mg/day (or equivalent) and maintain it through Week 24.
    • Exposure response analysis comparing steroid dose area under the curve (AUC) with ATYR1923 PK parameters through Week 28.
    Immunogenicity of multiple IV doses of ATYR1923 in patients with pulmonary sarcoidosis based on the following endpoints:
    • Incidence and titer of positive ADA (anti-ATYR1923) and anti-Jo 1 Ab.
    E.5.2.1Timepoint(s) of evaluation of this end point
    From Day 1 to Week 28.
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability, Immunogenicity
    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. 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 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 No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E. trial design description
    Dose escalation
    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 trial4
    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 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
    United States
    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
    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 months0
    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 months0
    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: 30
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 6
    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 state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 14
    F.4.2.2In the whole clinical trial 36
    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)
    Patients with elevated confirmed positive anti-drug antibody (ADA) titers or who have ongoing study drug-related treatment-emergent adverse events (TEAEs) at Week 24/EOS will continue to be followed until resolution or determined by the Investigator to be stable (eg, by telephone contact or unscheduled visit).
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Foundation for Sarcoidosis Research
    G.4.3.4Network Country United States
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-10-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-09-12
    P. End of Trial
    P.End of Trial StatusCompleted
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