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    EudraCT Number:2015-001912-36
    Sponsor's Protocol Code Number:ATYR1940-C-005
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2015-06-22
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2015-001912-36
    A.3Full title of the trial
    An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients with Facioscapulohumeral Muscular Dystrophy (FSHD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Exposure of ATYR1940 in Adult Patients with Genetic Myopathy
    A.4.1Sponsor's protocol code numberATYR1940-C-005
    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
    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.3.9.3Other descriptive nameATYR1940
    D.3.9.4EV Substance CodeSUB129952
    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. 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Facioscapulohumeral muscular dystrophy
    E.1.1.1Medical condition in easily understood language
    Genetic myopathy
    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
    Evaluate the safety, tolerability, and immunogenicity of long-term treatment with intravenous (IV) ATYR1940 in adult patients with facioscapulohumeral muscular dystrophy (FSHD) previously enrolled in clinical study ATYR1940-C-002
    E.2.2Secondary objectives of the trial
    • Evaluate the effects of long-term ATYR1940 treatment on clinically relevant measures of muscle strength and function, including: Quantitative Muscle Testing (QMT) and Manual muscle testing (MMT), as determined by the Investigator
    • Evaluate the effects of long-term ATYR1940 treatment on patient-reported quality of life (QoL)
    • Evaluate the effects of long-term ATYR1940 treatment on muscle disease burden, based on skeletal muscle magnetic resonance imaging (MRI)
    • Explore biological and pharmacodynamic (PD) changes in the inflammatory immune state in peripheral blood
    • Determine the long-term systemic exposure to ATYR1940 through pharmacokinetic (PK) sampling
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Enrolled in ≥Cohort 2 and completed the double-blind treatment period in the parent study.
    2. Demonstrated, in the Investigator’s opinion, acceptable tolerability of study drug.
    3. In the Investigator’s opinion, patient has shown acceptable compliance with study drug and the study procedures in the parent study and is willing and able to comply with all procedures in the current study.
    4. Is, in the opinion of the Investigator and Sponsor, a suitable candidate for continued study drug treatment.
    5. Provided written informed consent after the nature of the study has been explained and prior to the performance of any research-related procedures.
    E.4Principal exclusion criteria
    1. At any time during participation in the parent study, met a study drug discontinuation criterion, including, but not limited to:
    a. Jo-1 Ab levels ≥1.5 U/mL.
    b. Clinical evidence of a generalized infusion-related reaction (IRR).
    c. Clinical evidence of a National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) (version 4.03) ≥Grade 2, study-drug-related SAE.
    d. Pregnancy.
    e. Progression of disease that, in the opinion of the Investigator, precluded further participation in the study.
    f. Withdrawal of consent.
    g. Other findings that, at the discretion of the Investigator and/or Sponsor, indicated that study drug administration should be discontinued.
    2. Is expected to require treatment with curcumin or systemic albuterol (intermittent inhaled albuterol is permissible) during study participation; or use of a product that putatively enhances muscle growth (e.g., insulin-like growth factor, growth hormone) or activity (e.g., Coenzyme Q, Coenzyme A, creatine, L-carnitine) on a chronic basis; or statin treatment initiation or significant adjustment to statin regimen (stable, chronic statin use is permissible).
    3. Planned to receive any vaccination during study participation.
    4. Abnormal baseline findings, medical condition(s), or laboratory findings that, in the Investigator’s opinion, might jeopardize the patient's safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study.
    5. Evidence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, dermatological, or gastrointestinal disease, or has a condition that requires immediate surgical intervention or other treatment or may not allow safe participation.
    6. If female and of childbearing potential (premenopausal and not surgically sterile), has a positive pregnancy test at entry or is unwilling to use contraception from the time of entry through the 1-month Follow-up visit. Acceptable methods of birth control include abstinence, barrier methods, hormones, or intra-uterine device.
    7. If male, is unwilling to use a condom plus spermicide during sexual intercourse from the time of entry through the 1 month Follow-up visit"
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability:
    • Change from baseline in physical examination, including neurological examination.
    • Incidence of AEs, including serious and severe AEs.
    • Change from baseline in safety laboratory test results.
    • Change from baseline in electrocardiogram (ECG) findings.
    • Change from baseline in vital sign measurements and pulmonary evaluations (pulmonary function tests and pulse oximetry).
    • Anti-drug antibody (ADA) titers and Jo-1 antibody levels.
    • Exploratory characterization of immune response to ATYR1940.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Physical Exam: at eligibility check, W4, every 3M and at 1 and 4-week FU
    AEs: weekly and at 1, 4 and 12-week FU
    Safety lab tests: at eligibility check, W1 to W4, monthly and at 1, 4 and 12-week FU
    ECG: at W1, W2, W4, every 3M and at 1 and 4-week FU
    Vital signs: at eligibility check, weekly and at 1, 4 and 12-week FU
    Pulmonary function tests: at W1, every 3M and at 4-week FU
    Pulse Oximetry: at W1 to W4, every 3M and at 1, 4 and 12-week FU
    Jo-1 Ab: at eligibility check, weekly from W2 and at 1, 4 and 12-week FU
    ADA: at eligibility check, W2, W3, every 3M and at 1, 4 and 12-week FU
    Plasma complement factors: at W1, W3, W16, every 3M starting at W24 and at 1-week FU
    Serum complement and tryptase levels: at W1, W16, every 3M starting at W24 and at 1-week FU
    E.5.2Secondary end point(s)
    Muscle strength and function:
    • Muscle strength, based on Quantitative muscle testing (QMT) and Manual muscle testing (MMT)
    • Lower extremity muscle function based on the Vignos scale

    Quality of life:
    • INQoL questionnaire
    • FSHD-HI questionnaire
    • Additional measures of quality of life (e.g., sleep status)

    Pharmacodynamic effects of ATYR1940:
    • Muscle disease burden on lower extremity skeletal muscle MRI
    • Changes in FSHD-related inflammatory immune state in peripheral blood

    Systemic exposure:
    • PK sampling
    E.5.2.1Timepoint(s) of evaluation of this end point
    QMT & MMT & Vignos scale & INQoL & sleep status: at eligibility check, every 3M and at 1-week FU
    FSHD-HI: at eligibility check and every 3M
    Skeletal Muscle Surveillance MRI: at eligibility check, at M3 and then every 6M and at 4-week FU
    PBMCs & biomarkers: at eligibility check, at W4, every 3M and at 1 and 4-week FU
    Pharmacokinetics: at W1 to W4, every 3M and at 1-week FU
    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 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. trial type description
    Phase 1b/2a: Safety, Tolerability, Biological Activity and Systemic Exposure
    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 Information not present in EudraCT
    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
    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
    Last visit of the last patient
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    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: 32
    F.1.3Elderly (>=65 years) No
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 32
    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)
    Subjects will return to their standard of care treatment as determined by their physician after completion of the trial.
    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 Decision2015-06-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-08-04
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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