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    Clinical Trial Results:
    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

    Summary
    EudraCT number
    		 2016-000624-25
    Trial protocol
    		 DK   IT  
    Global end of trial date
    18 Apr 2017

    Results information
    Results version number
    		 v1(current)
    This version publication date
    23 Dec 2018
    First version publication date
    23 Dec 2018
    Other versions
    Summary report(s)
    		 ATYR1940-C-006 CSR Synopsis

    Trial information

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    Trial identification
    Sponsor protocol code
    ATYR1940-C-006
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 IND number: 122045
    Sponsors
    Sponsor organisation name
    aTyr Pharma, Inc.
    Sponsor organisation address
    3545 John Hopkins Court, Suite #250, San Diego, CA, United States, 92121
    Public contact
    Clinical Trial Operations, Voisin Consulting, clinicaltrialinformation@voisinconsulting.com
    Scientific contact
    Clinical Trial Operations, Voisin Consulting, clinicaltrialinformation@voisinconsulting.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    05 Mar 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    18 Apr 2017
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Apr 2017
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main 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 or ATYR1940-C-004. All clinically significant laboratory abnormalities were reported as adverse events and therefore appear in the Adverse events section of this dataset. As a consequence, the endpoints reported in this dataset are limited to the most relevant safety endpoints.
    Protection of trial subjects
    The study process, benefits and risks of participating in the study were explained to each subject. In addition, if the study drug needed to be stopped for safety, the doctor, his/her staff along with the medical monitor, were to continue to monitor participant's health and determine what treatment should be given (if any) until the symptoms or findings had resolved or until a satisfactory conclusion was reached.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Jun 2016
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    United States: 4
    Country: Number of subjects enrolled
    Denmark: 2
    Worldwide total number of subjects
    8
    EEA total number of subjects
    4
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    1
    Adults (18-64 years)
    7
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Patients who participated in and completed the treatment period in the parent studies.

    Pre-assignment
    Screening details
    		 Patients who completed treatment in either parent study ATYR1940-C-003 or ATYR1940-C-004; demonstrated acceptable tolerability of study drug; were considered by the Investigator to be compliant with study drug and the study procedures; and did not meet any criterion for discontinuation.

    Period 1
    Period 1 title
    Treatment period (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Treatment period
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    ATYR1940
    Investigational medicinal product code
    ATYR1940
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    For the first 12 weeks in this extension study, patients received ATYR1940 at the highest tolerated dose received in the parent study; no dose adjustments were allowed during this 12-week period. After 12 weeks, if the patient demonstrated good tolerability, the ATYR1940 dose may have been increased on a patient-specific basis to a maximum weekly dose of 3 mg/kg. All patients received ATYR1940 on a weekly basis in this study, regardless of the frequency of dosing in the parent study. ATYR1940 was administered via IV infusion over 90 minutes.

    Number of subjects in period 1
    		Treatment period
    Started
    8
    Completed
    8

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Treatment period (overall period)
    Reporting group description
    		 -

    Reporting group values
    		 Treatment period (overall period) Total
    Number of subjects
    		 8 8
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 1 1
        Adults (18-64 years)
    		 7 7
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (full range (min-max))
    		 30.0 (16 to 62) -
    Gender categorical
    Units: Subjects
        Female
    		 1 1
        Male
    		 7 7

    End points

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    End points reporting groups
    Reporting group title
    Treatment period
    Reporting group description
    		 -

    Primary: Incidence of Treatment Emergent Adverse Events (TEAEs)

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    End point title
    		 Incidence of Treatment Emergent Adverse Events (TEAEs) [1]
    End point description
    End point type
    Primary
    End point timeframe
    All study visits until the end of the study.
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No statistical analysis was performed for any of the primary/safety endpoints.
    End point values
    		 Treatment period
    Number of subjects analysed
    8
    Units: number of subjects with at least 1 TEAE
    7
    No statistical analyses for this end point

    Primary: Anti-drug antibodies (ADA)

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    End point title
    		 Anti-drug antibodies (ADA) [2]
    End point description
    End point type
    Primary
    End point timeframe
    Week 1 then every 6 weeks (on-treatment), then 1-4- and 12-weeks post-treatment follow-up.
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No statistical analysis was performed for any of the primary/safety endpoints.
    End point values
    		 Treatment period
    Number of subjects analysed
    8
    Units: number of confirmed positive
    3
    No statistical analyses for this end point

    Primary: Jo-1 antibodies

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    End point title
    		 Jo-1 antibodies [3]
    End point description
    End point type
    Primary
    End point timeframe
    Weekly from Week 1, 1-week follow-up, 4-week follow-up, 12-week follow-up
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No statistical analysis was performed for any of the primary/safety endpoints.
    End point values
    		 Treatment period
    Number of subjects analysed
    8
    Units: number of subjects Jo-1 positive
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From signing of informed consent form to EOS visit
    Adverse event reporting additional description
    Due to the small study size (N=8), TEAEs reported for ≥ 2 patients treated with ATYR1940 are listed in the section below. The number of occurrences per TEAE is not available in the source data, the field "Occurrences all number" therefore corresponds to the number of subjects affected per TEAE.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.1
    Reporting groups
    Reporting group title
    Treatment period
    Reporting group description
    		 -

    Serious adverse events
    		 Treatment period
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 8 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Treatment period
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 8 (87.50%)
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    4 / 8 (50.00%)
         occurrences all number
    4
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 8 (25.00%)
         occurrences all number
    2
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    2 / 8 (25.00%)
         occurrences all number
    2
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    4 / 8 (50.00%)
         occurrences all number
    4
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    2 / 8 (25.00%)
         occurrences all number
    2
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    3 / 8 (37.50%)
         occurrences all number
    3
    Myalgia
         subjects affected / exposed
    3 / 8 (37.50%)
         occurrences all number
    3
    Pain in extremity
         subjects affected / exposed
    3 / 8 (37.50%)
         occurrences all number
    3

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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