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    EudraCT Number:2016-000624-25
    Sponsor's Protocol Code Number:ATYR1940-C-006
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-04-19
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedDenmark - DHMA
    A.2EudraCT number2016-000624-25
    A.3Full title of the trial
    An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Fascioscapulohumeral Muscular Dystrophy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Fascioscapulohumeral Muscular Dystrophy
    A.4.1Sponsor's protocol code numberATYR1940-C-006
    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
    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 long-term treatment with intravenous (IV) ATYR1940 in patients with limb girdle muscular dystrophy 2B (LGMD2B) or fascioscapulohumeral muscular dystrophy (FSHD) previously enrolled in clinical study ATYR1940-C-003 (Stage 1 only) or ATYR1940-C-004.
    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 and muscle disease burden, based on skeletal muscle MRI.
    • 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. Enrolled in and completed the treatment period in the parent study.
    2. Demonstrated, in the Investigator’s opinion, acceptable tolerability of ATYR1940.
    3. In the Investigator’s opinion, patient has shown acceptable compliance with ATYR1940 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 Investigator’s and Sponsor’s opinion, a suitable candidate for continued ATYR1940 treatment.
    5. Provide 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 ATYR1940 discontinuation criterion, including, but not limited to:
    - Jo-1 antibody (Ab) levels ≥1.5 U/mL.
    - Clinical evidence of a generalized infusion-related reaction (IRR).
    - Clinical evidence of a National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) (version 4.03) ≥Grade 2, study-drug-related serious adverse event (SAE).
    - Pregnancy.
    - Progression of disease that, in the opinion of the Investigator, precluded further participation in the study.
    - Withdrawal of consent.
    - 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. Patient 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 3-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:
    • Incidence of treatment-emergent adverse events (TEAEs) and SAEs overall and by intensity
    • Changes from baseline in:
    - Safety laboratory test results
    - Electrocardiogram (ECG) findings
    - Vital signs measurements
    - Pulmonary evaluations (pulmonary function tests [PFTs] and pulse oximetry)
    - Incidence and level of anti-drug antibodies (ADA) titers and Jo-1 Ab levels
    • Exploratory characterization of immune response to ATYR1940
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety and tolerability:
    - Adverse events & vital signs & pulse oximetry & Jo-1 Ab: at week 1, every week and at 1, 4 and 12-week follow-up
    - Lab test results & ADA: at week 1, every month and at 1, 4 and 12-week follow-up
    - ECG: at week 1, every month and at 1 and 4-week follow-up
    - Pulmonary function tests: at week 1, every 6 weeks or every 3 months and at 4-week follow-up
    E.5.2Secondary end point(s)
    Muscle strength and function:
    • Changes from baseline in the following clinical parameters:
    - Muscle disease burden on lower extremity skeletal muscle MRI
    - Muscle strength, based on manual muscle testing (MMT)
    - Upper and lower extremity muscle function, based on the Brooke and Vignos scales, respectively

    Pharmacodynamics (exploratory):
    • Changes in muscular-dystrophy-related inflammatory immune state in peripheral blood, as assessed by:
    - Circulating immune proteins, such as cytokines
    - Ex vivo inflammatory immune protein (including cytokines) release from peripheral blood mononuclear cells (PBMCs)
    - Immunophenotyping of circulating PBMCs
    • Changes in serum- and/or plasma-based muscle biomarkers

    Quality of life (exploratory):
    • INQoL questionnaire
    • For patients with FSHD, the FSHD-specific Health Inventory (FSHD-HI) questionnaire

    Systemic exposure:
    • Sparse pharmacokinetic (PK) sampling
    E.5.2.1Timepoint(s) of evaluation of this end point
    Muscle strength and function:
    - Surveillance skeletal muscle MRI: at week 1, every 3 months and at 12-week follow-up
    - MMT & Upper and lower extremity muscle function: at week 1, every 3 months and at 1-week follow-up

    - PBMCs & muscle biomarkers: at week 1, every 3 months and at 1 and 4-week follow-up

    Quality of life:
    INQoL & FSHD-HI: at week 1, every 3 months and at 1 and 12-week follow-up

    Systemic exposure:
    - Serum ATYR1940 concentrations: at week 1, every 6 weeks and at 1-week follow-up
    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
    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: Long-Term Safety, Tolerability, and Biological Activity
    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 EEA2
    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
    LVLS. The 12-week follow-up visit is the End-of-Study visit.
    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 Yes
    F.1.1Number of subjects for this age range: 2
    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) Yes
    F. of subjects for this age range: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 8
    F.4.2.2In the whole clinical trial 22
    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 Decision2016-06-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-06-22
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2017-04-18
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