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    Clinical Trial Results:
    A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects with Diffuse Cutaneous Systemic Sclerosis

    Summary
    EudraCT number
    		 2020-000134-17
    Trial protocol
    		 GB   DE   BE   IT   PL  
    Global end of trial date
    14 Feb 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    22 Dec 2024
    First version publication date
    22 Dec 2024
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    MT-7117-G02
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04440592
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Mitsubishi Tanabe Pharma America, Inc.
    Sponsor organisation address
    525 Washington Boulevard, Suite 1100, Jersey City , United States, 07310
    Public contact
    Mitsubishi Tanabe Pharma Europe Ltd (MTPE), General Information, +44 2070655000, regulatory@mt-pharma-eu.com
    Scientific contact
    Mitsubishi Tanabe Pharma Europe Ltd (MTPE), General Information, +44 2070655000, regulatory@mt-pharma-eu.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
    14 Feb 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Feb 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of MT-7117 treatment in participants with diffuse cutaneous systemic sclerosis (dcSSc) using the American College of Rheumatology Composite Response Index in Diffuse Systemic Sclerosis (ACR CRISS) at Week 52.
    Protection of trial subjects
    The study was conducted in accordance with the 2013 (Fortaleza) revision of the 1964 Declaration of Helsinki, Good Clinical Practice (GCP) as required by the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH) guidelines, applicable regional and local legislation, and standard operating procedures (SOPs) in place at Mitsubishi Tanabe Pharma America Inc. (MTPA). Prior to undergoing any study-specific procedure, all legally competent participants were required to provide consent in writing to participate in the study. An Informed Consent Form (ICF) was given to each participants, which contained all regulatory, ICH and data protection requirements, as applicable, in language understandable to the participant. If a participant was legally incompetent, the enrollment of such a participant was required to be in accordance with all applicable laws, and consent sought by the Investigator from the participant’s legally authorized representative. Either the Investigator or a designated person, qualified to meet any applicable local regulations, who was equally knowledgeable about the study explained the aims, methods, anticipated benefits and potential hazards of the study and any discomfort it could entail. A corresponding written explanation was also provided, and the subject allowed sufficient time to consider the study information. Clinical monitoring was conducted to confirm the ethical conduct of the study at the investigational site(s).
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    05 Feb 2021
    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
    Canada: 1
    Country: Number of subjects enrolled
    United States: 18
    Country: Number of subjects enrolled
    Poland: 26
    Country: Number of subjects enrolled
    Spain: 13
    Country: Number of subjects enrolled
    United Kingdom: 5
    Country: Number of subjects enrolled
    Belgium: 7
    Country: Number of subjects enrolled
    Germany: 4
    Country: Number of subjects enrolled
    Italy: 2
    Worldwide total number of subjects
    76
    EEA total number of subjects
    52
    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)
    63
    From 65 to 84 years
    13
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 A total of 76 participants were enrolled in North America and Europe, between 05 February 2021 and 14 February 2024.

    Pre-assignment
    Screening details
    		 After providing informed consent, male and female participants were eligible to participate if aged 18 or older, with documented diagnosis of systemic sclerosis (SSc), as defined using the 2013 ACR/European League Against Rheumatism (EULAR) criteria and with disease duration ≤ 5 years.

    Period 1
    Period 1 title
    Overall Study (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
    Yes

    Arm title
    		 MT-7117
    Arm description
    		 Participants with dcSSc took oral tablets of 300mg MT-7117 once daily for the duration of the study. If the participant continuously presented significant intolerable AEs at the following scheduled or unscheduled visit and the Investigator deemed it necessary, the daily dose of study drug could have been reduced.
    Arm type
    		 Experimental

    Investigational medicinal product name
    MT-7117
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral administration once daily in the morning with or without food.

    Arm title
    		 Placebo
    Arm description
    		 Participants with dcSSc took oral tablets of placebo to match MT-7117 once daily for the duration of the study.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral administration once daily in the morning with or without food.

    Number of subjects in period 1
    		MT-7117 Placebo
    Started
    38
    38
    Completed
    28
    36
    Not completed
    10
    2
         Consent withdrawn by subject
    1
    1
         Adverse event, non-fatal
    8
    -
         Other
    -
    1
         Lost to follow-up
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    MT-7117
    Reporting group description
    		 Participants with dcSSc took oral tablets of 300mg MT-7117 once daily for the duration of the study. If the participant continuously presented significant intolerable AEs at the following scheduled or unscheduled visit and the Investigator deemed it necessary, the daily dose of study drug could have been reduced.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants with dcSSc took oral tablets of placebo to match MT-7117 once daily for the duration of the study.

    Reporting group values
    		 MT-7117 Placebo Total
    Number of subjects
    		 38 38 76
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 34 29 63
        From 65-84 years
    		 4 9 13
        85 years and over
    		 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 49.2 ( 12.5 ) 54.2 ( 12.4 ) -
    Gender categorical
    Units: Subjects
        Female
    		 26 30 56
        Male
    		 12 8 20
    Race
    Units: Subjects
        White
    		 33 32 65
        Black or African American
    		 2 1 3
        Asian
    		 0 1 1
        American Indian or Alaska Native
    		 0 1 1
        Native Hawaiian or Pacific Islander
    		 1 0 1
        Other
    		 2 3 5
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 7 7 14
        Non-Hispanic or Latino
    		 31 29 60
        Other
    		 0 2 2

    End points

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    End points reporting groups
    Reporting group title
    MT-7117
    Reporting group description
    		 Participants with dcSSc took oral tablets of 300mg MT-7117 once daily for the duration of the study. If the participant continuously presented significant intolerable AEs at the following scheduled or unscheduled visit and the Investigator deemed it necessary, the daily dose of study drug could have been reduced.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants with dcSSc took oral tablets of placebo to match MT-7117 once daily for the duration of the study.

    Primary: ACR CRISS Composite Score at Week 52

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    End point title
    		 ACR CRISS Composite Score at Week 52
    End point description
    The ACR CRISS is a composite endpoint that calculates probability of improvement from baseline, incorporating change in modified Rodnan Skin Score (mRSS), percentage predicted forced vital capacity (%pFVC), physician and patient global assessments, and Health Assessment Questionnaire Disability Index (HAQ-DI). The outcome was a continuous variable between 0.0 and 1.0 with higher score indicating improvement in symptoms. A higher score indicated greater improvement. An ACR CRISS score of 0.60 or greater indicated that a participant improved on treatment and a score of less than 0.60 suggested that a participant had not improved. Intent-to-treat (ITT) population: Included all randomized participants who received at least 1 dose of study drug.
    End point type
    Primary
    End point timeframe
    Week 52
    End point values
    		 MT-7117 Placebo
    Number of subjects analysed
    38
    38
    Units: ACR CRISS score
    median (full range (min-max))
        ACR CRISS score
    0.8537 (0.000 to 1.000)
    0.8502 (0.000 to 1.000)
    Statistical analysis title
    		 MT-7117 versus (vs) placebo
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.9514
    Method
    		 Hodges-Lehman
    Parameter type
    		 Location shift
    Point estimate
    -0.0038
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.1243
         upper limit
    		 0.1168

    Secondary: Change in HAQ-DI from Baseline at Weeks 16, 26, 39, and 52

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    End point title
    		 Change in HAQ-DI from Baseline at Weeks 16, 26, 39, and 52
    End point description
    The HAQ-DI is a self-administered instrument consists of 20 questions referring to eight component sets consisting of dressing/grooming, arising, eating, walking, hygiene, reach, grip, and activities. Each item was scored on a 4-point scale from 0 to 3: 0 = Without any difficulty; 1 = With some difficulty; 2 = With much difficulty; 3 = Unable to do. Overall score, ranging from 0 to 3, was calculated as the sum of component set scores and divided by the number of component sets answered. A negative change from baseline indicated improvement. ITT population: Included all randomized participants who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    From Baseline, Week 16, Week 26, Week 39, and Week 52
    End point values
    		 MT-7117 Placebo
    Number of subjects analysed
    37
    38
    Units: Score on a Scale
    arithmetic mean (standard deviation)
        Change from baseline (BL) at Week 16
    -0.1439 ( 0.3803 )
    -0.0395 ( 0.3900 )
        Change from BL at Week 26
    -0.0847 ( 0.3360 )
    -0.0691 ( 0.4481 )
        Change from BL at Week 39
    -0.0927 ( 0.3178 )
    -0.0439 ( 0.5147 )
        Change from BL at Week 52
    -0.1111 ( 0.4265 )
    -0.0208 ( 0.5102 )
    Statistical analysis title
    		 MT-7117 vs Placebo Week 16
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    75
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1908
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    -0.1185
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.2976
         upper limit
    		 0.0605
    Statistical analysis title
    		 MT-7117 vs Placebo Week 26
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    75
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.7757
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    -0.027
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.2154
         upper limit
    		 0.1614
    Statistical analysis title
    		 MT-7117 vs Placebo Week 39
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    75
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.5994
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    -0.0549
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.2625
         upper limit
    		 0.1527
    Statistical analysis title
    		 MT-7117 vs Placebo Week 52
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    75
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1739
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    -0.1508
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.3699
         upper limit
    		 0.0683

    Secondary: Change in Patient Global Assessment (PtGA) from Baseline at Weeks 16, 26, 39, and 52

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    End point title
    		 Change in Patient Global Assessment (PtGA) from Baseline at Weeks 16, 26, 39, and 52
    End point description
    The PtGA was used to assess the participants’ rating of their overall disease activity. Participants rated their perceived health on an 11 point scale from 0 (excellent) to 10 (extremely poor). A negative change from baseline indicated improvement in symptoms. ITT population: Included all randomized participants who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    From Baseline, Week 16, Week 26, Week 39, and Week 52
    End point values
    		 MT-7117 Placebo
    Number of subjects analysed
    38
    38
    Units: Score on a Scale
    arithmetic mean (standard deviation)
        Change from BL at Week 16
    -0.8 ( 1.8 )
    -0.3 ( 1.9 )
        Change from BL at Week 26
    0.0 ( 2.5 )
    -0.4 ( 2.6 )
        Change from BL at Week 39
    -0.7 ( 2.2 )
    -0.5 ( 2.3 )
        Change from BL at Week 52
    -0.5 ( 2.3 )
    -0.3 ( 2.5 )
    Statistical analysis title
    		 MT-7117 vs Placebo Week 16
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1902
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    -0.48
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.2
         upper limit
    		 0.24
    Statistical analysis title
    		 MT-7117 vs Placebo Week 26
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.5075
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    0.33
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.66
         upper limit
    		 1.32
    Statistical analysis title
    		 MT-7117 vs Placebo Week 39
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.799
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    -0.12
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.1
         upper limit
    		 0.85
    Statistical analysis title
    		 MT-7117 vs Placebo Week 52
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.606
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    -0.28
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.34
         upper limit
    		 0.79

    Secondary: Change in %pFVC from Baseline at Weeks 16, 26, 39, and 52

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    End point title
    		 Change in %pFVC from Baseline at Weeks 16, 26, 39, and 52
    End point description
    %pFVC was measured to assess pulmonary function. ITT population: Included all randomized participants who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    From Baseline, Week 16, Week 26, Week 39, and Week 52
    End point values
    		 MT-7117 Placebo
    Number of subjects analysed
    38
    38
    Units: %pFVC
    arithmetic mean (standard deviation)
        Change from BL at Week 16
    0.754 ( 4.938 )
    -1.058 ( 5.749 )
        Change from BL at Week 26
    0.904 ( 5.669 )
    -0.691 ( 5.847 )
        Change from BL at Week 39
    -1.201 ( 6.293 )
    -1.457 ( 5.600 )
        Change from BL at Week 52
    0.048 ( 6.917 )
    -2.906 ( 5.101 )
    Statistical analysis title
    		 MT-7117 vs Placebo Week 16
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1441
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    1.968
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.691
         upper limit
    		 4.627
    Statistical analysis title
    		 MT-7117 vs Placebo Week 26
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.2261
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    1.708
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.084
         upper limit
    		 4.5
    Statistical analysis title
    		 MT-7117 vs Placebo Week 39
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.5901
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    0.763
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -2.053
         upper limit
    		 3.578
    Statistical analysis title
    		 MT-7117 vs Placebo Week 52
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1071
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    2.556
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.571
         upper limit
    		 5.683

    Secondary: Change in Physician Global Assessment (PhGA) from Baseline at Weeks 16, 26, 39, and 52

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    End point title
    		 Change in Physician Global Assessment (PhGA) from Baseline at Weeks 16, 26, 39, and 52
    End point description
    The PhGA was used to assess the physician’s rating of overall health of the participant. Physicians rated the perceived health of the participant on an 11-point scale from 0 (excellent) to 10 (extremely poor). A negative change from baseline indicated improvement in symptoms. ITT population: Included all randomized participants who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    From Baseline, Week 16, Week 26, Week 39, and Week 52
    End point values
    		 MT-7117 Placebo
    Number of subjects analysed
    38
    38
    Units: Score on a Scale
    arithmetic mean (standard deviation)
        Change from BL at Week 16
    -1.6 ( 1.9 )
    -1.7 ( 1.8 )
        Change from BL at Week 26
    -1.6 ( 2.1 )
    -1.7 ( 2.1 )
        Change from BL at Week 39
    -2.1 ( 2.5 )
    -2.2 ( 2.3 )
        Change from BL at Week 52
    -2.0 ( 2.9 )
    -1.7 ( 2.2 )
    Statistical analysis title
    		 MT-7117 vs Placebo Week 16
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.5319
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    -0.24
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.98
         upper limit
    		 0.51
    Statistical analysis title
    		 MT-7117 vs Placebo Week 26
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.8085
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    -0.11
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.97
         upper limit
    		 0.76
    Statistical analysis title
    		 MT-7117 vs Placebo Week 39
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.5004
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    -0.32
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.26
         upper limit
    		 0.62
    Statistical analysis title
    		 MT-7117 vs Placebo Week 52
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.3485
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    -0.5
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.57
         upper limit
    		 0.56

    Secondary: Change in mRSS from Baseline at Weeks 16, 26, 39, and 52

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    End point title
    		 Change in mRSS from Baseline at Weeks 16, 26, 39, and 52
    End point description
    mRSS evaluated a participant's skin thickness which was assessed by palpation and rated on a scale ranged from 0 (normal) to 3 (severe skin thickening) across 17 different body sites. The total score was the sum of the individual skin scores from all of these sites and ranged from 0 to 51 units. A negative change from baseline indicated improvement in skin symptoms. ITT population: Included all randomized participants who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    From Baseline, Week 16, Week 26, Week 39, and Week 52
    End point values
    		 MT-7117 Placebo
    Number of subjects analysed
    38
    38
    Units: Score on a Scale
    arithmetic mean (standard deviation)
        Change from BL at Week 16
    -3.2 ( 4.7 )
    -3.6 ( 3.9 )
        Change from BL at Week 26
    -4.9 ( 5.5 )
    -5.3 ( 6.9 )
        Change from BL at Week 39
    -6.4 ( 7.0 )
    -6.2 ( 7.5 )
        Change from BL at Week 52
    -6.6 ( 8.2 )
    -6.5 ( 7.9 )
    Statistical analysis title
    		 MT-7117 vs Placebo Week 16
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.7639
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    0.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.7
         upper limit
    		 2.3
    Statistical analysis title
    		 MT-7117 vs Placebo Week 26
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.8134
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    0.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -2.5
         upper limit
    		 3.2
    Statistical analysis title
    		 MT-7117 vs Placebo Week 39
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.8556
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    -0.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -3.7
         upper limit
    		 3
    Statistical analysis title
    		 MT-7117 vs Placebo Week 52
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.7776
    Method
    		 Mixed models analysis
    Parameter type
    		 Least square mean difference vs Placebo
    Point estimate
    -0.5
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -4.3
         upper limit
    		 3.2

    Secondary: ACR CRISS Score at Weeks 16, 26, and 39

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    End point title
    		 ACR CRISS Score at Weeks 16, 26, and 39
    End point description
    The ACR CRISS is a composite endpoint that calculates probability of improvement from baseline, incorporating change in modified Rodnan Skin Score (mRSS), percentage predicted forced vital capacity (%pFVC), physician and patient global assessments, and Health Assessment Questionnaire Disability Index (HAQ-DI). The outcome was a continuous variable between 0.0 and 1.0 with higher score indicating improvement in symptoms. A higher score indicated greater improvement. An ACR CRISS score of 0.60 or greater indicated that a participant improved on treatment and a score of less than 0.60 suggested that a participant had not improved. Intent-to-treat (ITT) population: Included all randomized participants who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    Week 16, Week 26, and Week 39
    End point values
    		 MT-7117 Placebo
    Number of subjects analysed
    38
    38
    Units: ACR CRISS Score
    median (full range (min-max))
        Week 16
    0.0349 (0.000 to 1.000)
    0.1236 (0.000 to 1.000)
        Week 26
    0.2626 (0.000 to 1.000)
    0.6232 (0.000 to 1.000)
        Week 39
    0.3890 (0.000 to 1.000)
    0.5289 (0.000 to 1.000)
    Statistical analysis title
    		 MT-7117 vs Placebo Week 16
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.939
    Method
    		 Hodges-Lehman
    Parameter type
    		 Location shift
    Point estimate
    0.0047
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.1156
         upper limit
    		 0.125
    Statistical analysis title
    		 MT-7117 vs Placebo Week 26
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.616
    Method
    		 Hodges-Lehman
    Parameter type
    		 Location shift
    Point estimate
    -0.0446
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.2191
         upper limit
    		 0.1298
    Statistical analysis title
    		 MT-7117 vs Placebo Week 39
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.6589
    Method
    		 Hodges-Lehman
    Parameter type
    		 Location shift
    Point estimate
    -0.0308
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.1677
         upper limit
    		 0.106

    Secondary: ACR CRISS Score Responders (CRISS>=0.6) at Weeks 16, 26, 39, and 52

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    End point title
    		 ACR CRISS Score Responders (CRISS>=0.6) at Weeks 16, 26, 39, and 52
    End point description
    The ACR CRISS is a composite endpoint that calculates probability of improvement from baseline, incorporating change in modified Rodnan Skin Score (mRSS), percentage predicted forced vital capacity (%pFVC), physician and patient global assessments, and Health Assessment Questionnaire Disability Index (HAQ-DI). The outcome was a continuous variable between 0.0 and 1.0 with higher score indicating improvement in symptoms. A higher score indicated greater improvement. An ACR CRISS score of 0.60 or greater indicated that a participant improved on treatment and a score of less than 0.60 suggested that a participant had not improved. Participants experiencing a response are reported. Intent-to-treat (ITT) population: Included all randomized participants who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    Week 16, Week 26, Week 39 and Week 52
    End point values
    		 MT-7117 Placebo
    Number of subjects analysed
    38
    38
    Units: Responders
    number (not applicable)
        Week 16
    10
    8
        Week 26
    10
    18
        Week 39
    11
    15
        Week 52
    14
    18
    Statistical analysis title
    		 MT-7117 vs Placebo Week 16
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.295
    Method
    		 Mixed models analysis
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    1.826
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.589
         upper limit
    		 5.664
    Statistical analysis title
    		 MT-7117 vs Placebo Week 26
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.181
    Method
    		 Mixed models analysis
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    0.487
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.169
         upper limit
    		 1.402
    Statistical analysis title
    		 MT-7117 vs Placebo Week 39
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.601
    Method
    		 Mixed models analysis
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    0.758
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.267
         upper limit
    		 2.154
    Statistical analysis title
    		 MT-7117 vs Placebo Week 52
    Comparison groups
    MT-7117 v Placebo
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.913
    Method
    		 Mixed models analysis
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    1.061
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.363
         upper limit
    		 3.096

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    60 weeks
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.1
    Reporting groups
    Reporting group title
    MT-7117
    Reporting group description
    		 Participants with dcSSc took 300mg of MT-7117 daily for the duration of the study. If the participant continuously presented significant intolerable AEs at the following scheduled or unscheduled visit and the Investigator deemed it necessary, the daily dose of study drug could have been reduced.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants with dcSSc took oral tablets of placebo to match MT-7117 once daily for the duration of the study.

    Serious adverse events
    		 MT-7117 Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 38 (10.53%)
    2 / 38 (5.26%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Malignant melanoma in situ
         subjects affected / exposed
    1 / 38 (2.63%)
    0 / 38 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 38 (2.63%)
    0 / 38 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 38 (2.63%)
    0 / 38 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Uterine polyp
         subjects affected / exposed
    0 / 38 (0.00%)
    1 / 38 (2.63%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Organising pneumonia
         subjects affected / exposed
    0 / 38 (0.00%)
    1 / 38 (2.63%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Skin ulcer
         subjects affected / exposed
    1 / 38 (2.63%)
    0 / 38 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Systemic scleroderma
         subjects affected / exposed
    1 / 38 (2.63%)
    0 / 38 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    1 / 38 (2.63%)
    0 / 38 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 MT-7117 Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    35 / 38 (92.11%)
    28 / 38 (73.68%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Melanocytic naevus
         subjects affected / exposed
    12 / 38 (31.58%)
    2 / 38 (5.26%)
         occurrences all number
    18
    2
    Dysplastic naevus
         subjects affected / exposed
    2 / 38 (5.26%)
    1 / 38 (2.63%)
         occurrences all number
    3
    1
    Seborrhoeic keratosis
         subjects affected / exposed
    2 / 38 (5.26%)
    0 / 38 (0.00%)
         occurrences all number
    2
    0
    Investigations
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 38 (5.26%)
         occurrences all number
    0
    2
    Injury, poisoning and procedural complications
    Accidental overdose
         subjects affected / exposed
    0 / 38 (0.00%)
    4 / 38 (10.53%)
         occurrences all number
    0
    4
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 38 (5.26%)
    2 / 38 (5.26%)
         occurrences all number
    2
    2
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    4 / 38 (10.53%)
    3 / 38 (7.89%)
         occurrences all number
    4
    4
    Eye disorders
    Cataract
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 38 (5.26%)
         occurrences all number
    0
    3
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    10 / 38 (26.32%)
    5 / 38 (13.16%)
         occurrences all number
    13
    8
    Nausea
         subjects affected / exposed
    7 / 38 (18.42%)
    2 / 38 (5.26%)
         occurrences all number
    7
    2
    Vomiting
         subjects affected / exposed
    6 / 38 (15.79%)
    2 / 38 (5.26%)
         occurrences all number
    6
    2
    Dysphagia
         subjects affected / exposed
    2 / 38 (5.26%)
    1 / 38 (2.63%)
         occurrences all number
    2
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 38 (0.00%)
    3 / 38 (7.89%)
         occurrences all number
    0
    3
    Oropharyngeal pain
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 38 (5.26%)
         occurrences all number
    0
    2
    Skin and subcutaneous tissue disorders
    Skin discolouration
         subjects affected / exposed
    8 / 38 (21.05%)
    0 / 38 (0.00%)
         occurrences all number
    10
    0
    Pruritus
         subjects affected / exposed
    3 / 38 (7.89%)
    2 / 38 (5.26%)
         occurrences all number
    3
    2
    Skin ulcer
         subjects affected / exposed
    1 / 38 (2.63%)
    2 / 38 (5.26%)
         occurrences all number
    1
    2
    Solar lentigo
         subjects affected / exposed
    2 / 38 (5.26%)
    1 / 38 (2.63%)
         occurrences all number
    2
    1
    Nail pigmentation
         subjects affected / exposed
    2 / 38 (5.26%)
    0 / 38 (0.00%)
         occurrences all number
    2
    0
    Skin hyperpigmentation
         subjects affected / exposed
    16 / 38 (42.11%)
    1 / 38 (2.63%)
         occurrences all number
    17
    1
    Erythema
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 38 (5.26%)
         occurrences all number
    0
    2
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    2 / 38 (5.26%)
    0 / 38 (0.00%)
         occurrences all number
    2
    0
    Systemic scleroderma
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 38 (5.26%)
         occurrences all number
    0
    2
    Infections and infestations
    COVID-19
         subjects affected / exposed
    4 / 38 (10.53%)
    5 / 38 (13.16%)
         occurrences all number
    4
    5
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 38 (5.26%)
    2 / 38 (5.26%)
         occurrences all number
    2
    5
    Bronchitis
         subjects affected / exposed
    2 / 38 (5.26%)
    1 / 38 (2.63%)
         occurrences all number
    2
    1
    Nasopharyngitis
         subjects affected / exposed
    3 / 38 (7.89%)
    5 / 38 (13.16%)
         occurrences all number
    3
    7
    Gastroenteritis
         subjects affected / exposed
    1 / 38 (2.63%)
    2 / 38 (5.26%)
         occurrences all number
    1
    2
    Lower respiratory tract infection
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 38 (5.26%)
         occurrences all number
    0
    2
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    2 / 38 (5.26%)
    1 / 38 (2.63%)
         occurrences all number
    2
    1
    Hypercholesterolaemia
         subjects affected / exposed
    2 / 38 (5.26%)
    0 / 38 (0.00%)
         occurrences all number
    2
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    01 Sep 2020
    Amendment 1: 1. Study exclusion criteria were amended, including prohibited medications. 2. Text of assessments were modified. 3. Reverse translational study objective, endpoint, and analysis of this endpoint were added. 4. ACR CRISS improvement score (secondary endpoint) was modified. 5. COVID-19 pandemic language was added. 6. Language was added to clarify allowed medication during study treatment. 7. Text about rescue medication was clarified. 8. Language was added to clarify treatments for SSc complications. 9. Drug-drug interaction text was added. 10. Role of the dual assessor and melanin assessor were clarified. 11. AESI definition was updated. 12. Contraception language was updated. 13. Composite Response Index for dcSSc language was updated. 14. Schedule of Activities (SoA) was revised.
    02 Dec 2020
    Amendment 2: 1. Study entry criteria was updated to select a more homogeneous population. 2. Exclusion criteria was updated to ensure subject safety. 3. Text was updated to exclude use of nintedanib and language was added for vaccine use. 4. Dual assessor approach language was updated.
    28 Jan 2021
    Amendment 3: 1. Exploratory objective/endpoint was updated. 2. Exclusion criteria was modified. 3. Urine dipstick pregnancy test was added and methodology for dosing in the case of positive results was clarified. 4. Risk/benefit statement was updated to include new findings in the non-clinical pre-and postnatal development study in rats and the COVID-19 pandemic. 5. Rescue therapy was clarified. 6. Composite response index for dcSSc section was updated. 7. PFT test section was updated. 8. Dual assessor approach was clarified. 9. Modified Rodnan skin score section was updated. 10. Pharmacogenomics sampling section was updated. 11. Clinical laboratory abnormalities and other abnormal assessments section was updated. 12. Recording and reporting of SAEs or hepatic AESIs section was updated. 13. Pregnancy section was updated. 14. Impact of COVID-19 section was updated. 15. Analysis of exploratory endpoints section was updated. 16. SoA was amended to reflect new additional pregnancy test.
    20 Dec 2021
    Amendment 4: 1. Eligibility criteria were updated. 2. New exploratory endpoint for the revised ACR-CRISS was added. 3. Withdrawal of individual subjects section was updated. 4. Dose reduction criteria section was updated, and dose interruption section was added. 5. Permitted immunosuppressant therapy section was updated. 6. PFT was updated. 7. Vital signs section was updated. 8. AESI section was updated. 9. Management and evaluation of hepatic AESI section was updated. 10. Analysis of ACR CRISS score improvement proportion was updated. 11. SoA was revised.

    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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