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    Summary
    EudraCT Number:2015-001910-88
    Sponsor's Protocol Code Number:ATYR1940-C-004
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-06-30
    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 ConcernedDenmark - DHMA
    A.2EudraCT number2015-001910-88
    A.3Full title of the trial
    An Open-Label, Intrapatient Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Facioscapulohumeral Muscular Dystrophies
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Facioscapulohumeral Muscular Dystrophies
    A.4.1Sponsor's protocol code numberATYR1940-C-004
    A.5.4Other Identifiers
    Name:IND numberNumber:122045
    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 Address3 rue des Longs Prés
    B.5.3.2Town/ cityBoulogne-Billancourt
    B.5.3.3Post code92100
    B.5.3.4CountryFrance
    B.5.6E-mailclinicaltrialinformation@voisinconsulting.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/15/1448
    D.3 Description of the IMP
    D.3.1Product nameATYR1940
    D.3.2Product code ATYR1940
    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 INNNot available yet
    D.3.9.2Current sponsor codeATYR1940
    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 placeboConcentrate for solution 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
    Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy
    E.1.1.1Medical condition in easily understood language
    Genetic myopathies
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level PT
    E.1.2Classification code 10064087
    E.1.2Term Facioscapulohumeral muscular dystrophy
    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 evaluate the safety, tolerability, and immunogenicity of the weekly and twice weekly intravenous (IV) administration of ATYR1940, at doses of 0.3, 1.0, and 3.0 mg/kg, to patiens with Dysferlinopathy (Limb Girdle Muscular Dystrophy 2B [LGMD2B]) and Facioscapulohumeral Muscular Dystrophy (FSHD).
    E.2.2Secondary objectives of the trial
    To explore the biological and pharmacodynamic (PD) activity of ATYR1940 in patients with LGMD2B and FSHD, based on changes in:
    • Serum-based muscle biomarkers.
    • Inflammatory immune state in peripheral blood.
    • Muscle disease burden.
    • Skeletal muscle strength.
    • Upper and lower extremity muscle function.
    • Quality of life measures.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female aged 18 to 75 years, inclusive.
    2. Provided written informed consent.
    3. In the Investigator’s opinion, is willing and able to complete all study procedures and comply with the study visit schedule.
    4. Established genetically confirmed diagnosis of LGMD2B or FSHD.
    5. Either the presence of a STIR positive muscle with limited fatty infiltration, as determined by the Fischer scoring system, on MRI (FHSD) or an elevated muscle marker (LGMD2B)
    E.4Principal exclusion criteria
    1. Currently receiving treatment with an immunomodulatory agent or history of such treatment, including targeted biological therapies (e.g., etanercept, omalizumab) or corticosteroids within the 3 months before baseline; or high-dose non-steroidal anti-inflammatory agents within 2 weeks before baseline.
    2. Currently receiving curcumin or albuterol or requires such treatment during study participation; use of a product that putatively enhances muscle growth or activity on a chronic basis within 30 days before baseline; or statin treatment initiation or significant adjustment to statin regimen within 3 months before baseline.
    3. Use of an investigational product or device (other than a mobility assistance device) within 30 days before baseline.
    4. History of severe restrictive or obstructive lung disease (including interstitial lung disease, pulmonary fibrosis, or asthma), or evidence of active lung disease on screening chest radiograph.
    5. History of anti-synthetase syndrome, prior Jo-1 Ab-positivity, or Jo 1 Ab level ≥0.6 U/mL on screening.
    6. Acute or clinically relevant Epstein-Barr virus (EBV) or cytomegalovirus (CMV) infection or re-activation.
    7. Chronic infection, such as hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) or a history of tuberculosis.
    8. Vaccination within 8 weeks before baseline or vaccination is planned during study participation.
    9. Symptomatic cardiomyopathy or severe cardiac arrhythmia (including bradyarrythmias), that may, in the Investigator’s opinion, limit the patient’s ability to complete the study protocol.
    10. Gamma-glutamyl transferase (GGT) or serum creatinine levels >2× the upper limit of normal.
    11. Muscle biopsy within 30 days before baseline.
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability
    • Incidence of treatment-emergent AEs and SAEs overall and by intensity.
    • Changes from baseline in safety laboratory test results.
    • Changes from baseline in ECG findings.
    • Changes from baseline in vital signs measurements.
    • Changes from baseline in pulmonary evaluations (PFTs and pulse oximetry).

    Immunogenicity
    • Incidence and level of ADA titers and Jo-1 Ab.
    • Exploratory characterization of immune response to ATYR1940.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety and tolerability
    - AEs & vital signs: throughout the study
    - Safety laboratory test results: Screening, weeks 1, 2 4, 6, 8, 10, 12, 14, 17 and 25 (follow-up period)
    - ECG: Screening, weeks 2, 4, 6 and 10 to 14 (follow-up period)
    - Pulmonary function tests: Screening, week 5, 8, 13 and 17 (follow-up period)
    - Pulse oximetry: weeks 1 to 13

    Immunogenicity:
    - Level of ADA titers: Screening, weeks 4, 6, 8 and 10 to 25 (follow-up period)
    - Level of Jo-1 Ab: Screening, weeks 3 to 13, 14, 17 and 25 (follow-up period)
    - Plasma complement levels: weeks 1, 4, 8, 11 and 14 (follow-up period)
    - Serum complement and tryptase levels: weeks 1 and 14 (follow-up period)
    E.5.2Secondary end point(s)
    Pharmacodynamics
    • Changes in muscular-dystrophy-related inflammatory immune state in peripheral blood.
    • Changes in serum- and/or plasma-based muscle biomarkers.
    • Changes from baseline in the following clinical parameters:
    - Lower extremity skeletal muscle MRI.
    - Muscle strength, based on MMT.
    - Upper and lower extremity muscle function, based on the Brooke and Vignos scales, respectively.
    - Physical activity monitoring.
    - Quality of life based on the INQoL questionnaire.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Pharmacodynamics:
    - PBMCs: Screening, weeks 1, 9, 13 and 14 (follow-up period)
    - Serum/plasma biomarkers: weeks 1, 2, 4, 6, 9, 10, 13 and 14 (follow-up period)
    - Skeletal muscle MRI: Screening, weeks 9, 13 and 14 (follow-up period)
    - MMT & Upper and lower extremity function: Screening, weeks 7, 10 and 14 (follow-up period)
    - INQoL: Screening, weeks 7, 14 and 25 (follow-up period)
    - Physical activity monitor: weeks 1 to 17 (daily)
    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 No
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability, immunogenicity
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Phase 1b/2: Safety, Tolerability, Immunogenicity and Pharmacodynamics
    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 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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 EEA4
    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
    Denmark
    France
    Italy
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    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 years1
    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: 12
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 16
    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)
    Continued treatment with ATYR1940 under an extended treatment protocol is planned as a part of the clinical development of ATYR1940.
    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 Decision2015-09-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-12-17
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-10-05
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