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    Clinical Trial Results:
    A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients with Molecularly Defined Genetic Muscular Dystrophies

    Summary
    EudraCT number
    		 2014-001753-17
    Trial protocol
    		 NL   IT  
    Global end of trial date
    04 Jan 2017

    Results information
    Results version number
    		 v1(current)
    This version publication date
    30 Nov 2018
    First version publication date
    30 Nov 2018
    Other versions
    Summary report(s)
    		 ATYR1940-C-002 CSR synopsis

    Trial information

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    Trial identification
    Sponsor protocol code
    ATYR1940-C-002
    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
    12 Jun 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    21 Dec 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    04 Jan 2017
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Evaluate the safety, tolerability, pharmacokinetics (PK), and immunogenicity of multiple doses of intravenous (IV) ATYR1940 in adults 18 to 65 years of age, inclusive, with FSHD. 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, as well as the pharmacodynamic endpoints. Cohort 4 was optional in the study and the Sponsor elected to not move forward with Cohort 4.
    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
    04 Sep 2014
    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
    Netherlands: 7
    Country: Number of subjects enrolled
    France: 6
    Country: Number of subjects enrolled
    Italy: 5
    Country: Number of subjects enrolled
    United States: 2
    Worldwide total number of subjects
    20
    EEA total number of subjects
    18
    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)
    0
    Adults (18-64 years)
    18
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Cohorts 1 to 3 were completed and the data are described herein. The sponsor elected not to move forward with cohort 4.

    Pre-assignment
    Screening details
    		 A total of 44 patients were screened. Out of that number, 20 patients were randomized.

    Period 1
    Period 1 title
    Treatment period (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    No

    Arm title
    		 Cohort 1 - 0.3 mg/kg
    Arm description
    		 Cohort 1 included 4 patients randomized 3 (ATYR1940) : 1 (placebo). For the purpose of reporting the results, separate arms were created for the placebo patients.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ATYR1940
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Patients received an initial Study Drug infusion of placebo, followed by once weekly IV Study Drug administration (ATYR1940) for 4 weeks. All Study Drug was administered via IV infusion over 30 minutes.

    Arm title
    		 Cohort 2 - 1 mg/kg
    Arm description
    		 Cohort 2 included 8 patients randomized 3 (ATYR1940) : 1 (placebo). For the purpose of reporting the results, separate arms were created for the placebo patients.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ATYR1940
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Patients received an initial Study Drug infusion of placebo, followed by once weekly IV Study Drug administration (ATYR1940) for 4 weeks. All Study Drug was administered via IV infusion over 30 minutes.

    Arm title
    		 Cohort 3 - 3 mg/kg
    Arm description
    		 Cohort 3 included 8 patients randomized 3 (ATYR1940) : 1 (placebo). For the purpose of reporting the results, separate arms were created for the placebo patients.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ATYR1940
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Patients received an initial Study Drug infusion of placebo, followed by once weekly IV Study Drug administration (ATYR1940) for 12 weeks. All Study Drug was administered via IV infusion over 30 minutes.

    Arm title
    		 Cohorts 1, 2 and 3 - Placebo
    Arm description
    		 Cohort 1 included 4 patients randomized 3 (ATYR1940) : 1 (placebo). Cohorts 2 and 3 included 8 patients randomized 3 (ATYR1940) : 1 (placebo). This arm includes the placebo patient from Cohort 1, the 2 placebo patients from Cohort 2 and the 2 placebo patients from Cohort 3.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Patients received an initial Study Drug infusion of placebo, followed by once weekly IV Study Drug administration (placebo) for 4 weeks. All Study Drug was administered via IV infusion over 30 minutes.

    Arm title
    		 Cohort 3 - Placebo
    Arm description
    		 Cohort 3 included 8 patients randomized 3 (ATYR1940) : 1 (placebo). This arm includes the 2 placebo patients from Cohort 3.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Patients received an initial Study Drug infusion of placebo, followed by once weekly IV Study Drug administration (placebo) for 12 weeks. All Study Drug was administered via IV infusion over 30 minutes.

    Number of subjects in period 1
    		Cohort 1 - 0.3 mg/kg Cohort 2 - 1 mg/kg Cohort 3 - 3 mg/kg Cohorts 1, 2 and 3 - Placebo Cohort 3 - Placebo
    Started
    3
    6
    6
    5
    2
    Completed
    3
    6
    6
    5
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1 - 0.3 mg/kg
    Reporting group description
    		 Cohort 1 included 4 patients randomized 3 (ATYR1940) : 1 (placebo). For the purpose of reporting the results, separate arms were created for the placebo patients.

    Reporting group title
    Cohort 2 - 1 mg/kg
    Reporting group description
    		 Cohort 2 included 8 patients randomized 3 (ATYR1940) : 1 (placebo). For the purpose of reporting the results, separate arms were created for the placebo patients.

    Reporting group title
    Cohort 3 - 3 mg/kg
    Reporting group description
    		 Cohort 3 included 8 patients randomized 3 (ATYR1940) : 1 (placebo). For the purpose of reporting the results, separate arms were created for the placebo patients.

    Reporting group title
    Cohorts 1, 2 and 3 - Placebo
    Reporting group description
    		 Cohort 1 included 4 patients randomized 3 (ATYR1940) : 1 (placebo). Cohorts 2 and 3 included 8 patients randomized 3 (ATYR1940) : 1 (placebo). This arm includes the placebo patient from Cohort 1, the 2 placebo patients from Cohort 2 and the 2 placebo patients from Cohort 3.

    Reporting group title
    Cohort 3 - Placebo
    Reporting group description
    		 Cohort 3 included 8 patients randomized 3 (ATYR1940) : 1 (placebo). This arm includes the 2 placebo patients from Cohort 3.

    Reporting group values
    		 Cohort 1 - 0.3 mg/kg Cohort 2 - 1 mg/kg Cohort 3 - 3 mg/kg Cohorts 1, 2 and 3 - Placebo Cohort 3 - Placebo Total
    Number of subjects
    		 3 6 6 5 2 20
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 3 6 5 4 2 18
        From 65-84 years
    		 0 0 1 1 0 2
    Age continuous
    Units: years
        arithmetic mean (full range (min-max))
    		 44.3 (25 to 55) 44.5 (33 to 52) 45.3 (25 to 72) 54 (39 to 66) 56.5 (49 to 64) -
    Gender categorical
    Units: Subjects
        Female
    		 1 4 2 1 0 8
        Male
    		 2 2 4 4 2 12

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1 - 0.3 mg/kg
    Reporting group description
    		 Cohort 1 included 4 patients randomized 3 (ATYR1940) : 1 (placebo). For the purpose of reporting the results, separate arms were created for the placebo patients.

    Reporting group title
    Cohort 2 - 1 mg/kg
    Reporting group description
    		 Cohort 2 included 8 patients randomized 3 (ATYR1940) : 1 (placebo). For the purpose of reporting the results, separate arms were created for the placebo patients.

    Reporting group title
    Cohort 3 - 3 mg/kg
    Reporting group description
    		 Cohort 3 included 8 patients randomized 3 (ATYR1940) : 1 (placebo). For the purpose of reporting the results, separate arms were created for the placebo patients.

    Reporting group title
    Cohorts 1, 2 and 3 - Placebo
    Reporting group description
    		 Cohort 1 included 4 patients randomized 3 (ATYR1940) : 1 (placebo). Cohorts 2 and 3 included 8 patients randomized 3 (ATYR1940) : 1 (placebo). This arm includes the placebo patient from Cohort 1, the 2 placebo patients from Cohort 2 and the 2 placebo patients from Cohort 3.

    Reporting group title
    Cohort 3 - Placebo
    Reporting group description
    		 Cohort 3 included 8 patients randomized 3 (ATYR1940) : 1 (placebo). This arm includes the 2 placebo patients from Cohort 3.

    Primary: Anti-drug antibodies (ADA)

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    End point title
    		 Anti-drug antibodies (ADA) [1]
    End point description
    End point type
    Primary
    End point timeframe
    Screening and weeks 3 to 9 for Cohorts 1 and 2, screening and weeks 3 to 6, 10, 14, 17, 25 for Cohort 3
    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
    		 Cohort 1 - 0.3 mg/kg Cohort 2 - 1 mg/kg Cohort 3 - 3 mg/kg Cohorts 1, 2 and 3 - Placebo Cohort 3 - Placebo
    Number of subjects analysed
    3
    6
    6
    5
    2
    Units: number (frequency) of confirmed positive
    2
    1
    3
    0
    0
    No statistical analyses for this end point

    Primary: Jo-1 antibodies

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    End point title
    		 Jo-1 antibodies [2]
    End point description
    End point type
    Primary
    End point timeframe
    Screening and weeks 3 to 9 for Cohorts 1 and 2, screening and weeks 3 to 14, 17, 25 for Cohort 3
    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
    		 Cohort 1 - 0.3 mg/kg Cohort 2 - 1 mg/kg Cohort 3 - 3 mg/kg Cohorts 1, 2 and 3 - Placebo Cohort 3 - Placebo
    Number of subjects analysed
    3
    6
    6
    5
    2
    Units: number of patients positive or equivocal
    0
    0
    0
    0
    0
    No statistical analyses for this end point

    Primary: Treatment Emergent Adverse Events (TEAEs)

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    End point title
    		 Treatment Emergent Adverse Events (TEAEs) [3]
    End point description
    End point type
    Primary
    End point timeframe
    All study visits until the end of the study
    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
    		 Cohort 1 - 0.3 mg/kg Cohort 2 - 1 mg/kg Cohort 3 - 3 mg/kg Cohorts 1, 2 and 3 - Placebo Cohort 3 - Placebo
    Number of subjects analysed
    3
    6
    6
    5
    2
    Units: number of subjects with at least 1 TEAE
    3
    6
    6
    5
    2
    No statistical analyses for this end point

    Secondary: Manual Muscle Testing (MMT)

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    End point title
    		 Manual Muscle Testing (MMT)
    End point description
    End point type
    Secondary
    End point timeframe
    Cohorts 1 and 2: shift from baseline to Week 6 Cohort 3: shift from baseline to Week 14
    End point values
    		 Cohort 1 - 0.3 mg/kg Cohort 2 - 1 mg/kg Cohort 3 - 3 mg/kg Cohorts 1, 2 and 3 - Placebo Cohort 3 - Placebo
    Number of subjects analysed
    3
    6
    6
    5
    2
    Units: percentage
        arithmetic mean (full range (min-max))
    2.83 (1.0 to 4.4)
    3.07 (0.7 to 5.6)
    0.70 (-5.9 to 9.2)
    1.34 (-3.3 to 7.8)
    -1.40 (-1.5 to -1.3)
    No statistical analyses for this end point

    Secondary: Individualized Neuromuscular Quality of Life (INQoL) - Overall QoL

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    End point title
    		 Individualized Neuromuscular Quality of Life (INQoL) - Overall QoL
    End point description
    End point type
    Secondary
    End point timeframe
    Cohorts 1 and 2: shift from baseline to Week 6 Cohort 3: shift from baseline to Week 14
    End point values
    		 Cohort 1 - 0.3 mg/kg Cohort 2 - 1 mg/kg Cohort 3 - 3 mg/kg Cohorts 1, 2 and 3 - Placebo Cohort 3 - Placebo
    Number of subjects analysed
    3
    5
    6
    5
    2
    Units: PP
        arithmetic mean (full range (min-max))
    2.77 (-5.0 to 6.7)
    -2.98 (-16.1 to 8.4)
    -9.90 (-19.4 to 5.0)
    4.12 (-8.3 to 22.2)
    15.55 (3.9 to 27.2)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From the first dose of study drug to the last follow-up assessment (EOS Visit) or after the end of the study if the TEAE was thought to be related to study drug
    Adverse event reporting additional description
    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
    18.0
    Reporting groups
    Reporting group title
    Cohorts 1, 2 and 3 - Placebo
    Reporting group description
    		 -

    Reporting group title
    Cohort 1 - 0.3 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Cohort 2 - 1 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Cohort 3 - 3 mg/kg
    Reporting group description
    		 -

    Serious adverse events
    		 Cohorts 1, 2 and 3 - Placebo Cohort 1 - 0.3 mg/kg Cohort 2 - 1 mg/kg Cohort 3 - 3 mg/kg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    General disorders and administration site conditions
    Infusion related reaction
    Additional description: Not assessed by the Investigator as serious; upgraded by Sponsor as medically-important, and therefore, an SAE
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Cohorts 1, 2 and 3 - Placebo Cohort 1 - 0.3 mg/kg Cohort 2 - 1 mg/kg Cohort 3 - 3 mg/kg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 5 (100.00%)
    3 / 3 (100.00%)
    6 / 6 (100.00%)
    6 / 6 (100.00%)
    Vascular disorders
    Flushing
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 6 (16.67%)
         occurrences all number
    0
    0
    1
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 5 (40.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    2 / 6 (33.33%)
         occurrences all number
    2
    0
    1
    2
    Presyncope
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    0
    0
    0
    2
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 3 (0.00%)
    2 / 6 (33.33%)
    0 / 6 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 3 (33.33%)
    0 / 6 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    0
    1
    0
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 3 (66.67%)
    0 / 6 (0.00%)
    1 / 6 (16.67%)
         occurrences all number
    0
    2
    0
    1
    Back pain
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 3 (33.33%)
    3 / 6 (50.00%)
    0 / 6 (0.00%)
         occurrences all number
    1
    1
    3
    0
    Myalgia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 3 (33.33%)
    1 / 6 (16.67%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    02 Sep 2014
    2.0 (Amendment 1; 02 September 2014)
    10 Jan 2015
    3.0 (Amendment 2; 10 January 2015)
    05 Feb 2015
    4.0 (Amendment 3; 05 February 2015)

    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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