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    Clinical Trial Results:
    A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria

    Summary
    EudraCT number
    		 2019-004226-16
    Trial protocol
    		 NO   SE   GB   DE   FI   IT  
    Global end of trial date
    26 Jul 2022

    Results information
    Results version number
    		 v1(current)
    This version publication date
    15 Jul 2023
    First version publication date
    15 Jul 2023
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    MT-7117-G01
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04402489
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 jRCT: jRCT2080225355
    Sponsors
    Sponsor organisation name
    Mitsubishi Tanabe Pharma America (MTPA), Inc.
    Sponsor organisation address
    525 Washington Boulevard, Suite 1100, Jersey City, United States, 07310
    Public contact
    General Information, Mitsubishi Tanabe Pharma Europe Ltd, regulatory@mt-pharma-eu.com
    Scientific contact
    General Information, Mitsubishi Tanabe Pharma Europe Ltd, regulatory@mt-pharma-eu.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-002850-PIP02-21
    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
    26 Jul 2022
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    26 Jul 2022
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with Erythropoietic Protoporphyria (EPP)or X-Linked Protoporphyria (XLP).
    Protection of trial subjects
    The study was conducted in accordance with the 2013 (Fortaleza) revision of the Declaration of Helsinki, Good Clinical Practice as required by the International Council for Harmonisation guidelines, applicable regional and local legislation, and standard operating procedures (SOPs) in place at MTPA. Clinical monitoring at the investigational site(s) was delegated to the Contract Research Organization (CRO), along with the responsibility to conform to the CRO’s ethical standards and SOPs. An external independent Data Monitoring Committee was established to perform regular review of safety data to ensure the ongoing safety of participating subjects until the last subject completed the Double blind extension period. A subject was withdrawn from study if ANY of the following criteria were met: 1. The subject requests to voluntarily withdraw from further participation in study. 2. The subject is significantly noncompliant with the protocol. 3. Continuing in the study would be detrimental to the subject’s safety in the opinion of the Investigator, e.g., a. The subject experiences intolerable AEs, SAEs, or AESIs. b. The subject has clinically significant changes in safety parameters at any of the post-dose time points, as confirmed with a repeat assessment performed as soon as possible after the initial out-of-range result. c. The subject experiences any clinically significant adverse findings from postbaseline nevi evaluation and adverse change is demonstrated histologically and confirmed by central pathologist. Visual evaluation of change will not be acceptable criteria for permanent discharge from treatment. d. Development of any clinically significant liver dysfunction, as follows: i. ALT or AST >8 × ULN. ii. ALT or AST >5 × ULN for more than 2 weeks. iii. Elevated total bilirubin >2 × ULN and ALT or AST >3 × ULN or iv. Symptoms consistent with liver dysfunction (e.g., fatigue, nausea, vomiting, abdominal pain or tenderne
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Jun 2020
    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
    Norway: 3
    Country: Number of subjects enrolled
    Spain: 11
    Country: Number of subjects enrolled
    Sweden: 5
    Country: Number of subjects enrolled
    United Kingdom: 14
    Country: Number of subjects enrolled
    Germany: 6
    Country: Number of subjects enrolled
    Italy: 21
    Country: Number of subjects enrolled
    United States: 93
    Country: Number of subjects enrolled
    Japan: 11
    Country: Number of subjects enrolled
    Australia: 12
    Country: Number of subjects enrolled
    Canada: 8
    Worldwide total number of subjects
    184
    EEA total number of subjects
    46
    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)
    37
    Adults (18-64 years)
    138
    From 65 to 84 years
    9
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A sufficient number of subjects were screened to ensure the planned sample size was achieved. Each subject were screened according to the criteria. Only subjects eligible for the study were randomized.

    Pre-assignment
    Screening details
    		 Subjects attended the screening visit (Visit 1) up to 6 weeks before Randomization (Visit 2), to confirm eligibility, obtain pre-study safety assessments including nevi evaluation, and to receive instruction on how to use the electronic sunlight exposure diary (SED).

    Period 1
    Period 1 title
    26 weeks Double-Blind Treatment
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 placebo
    Arm description
    		 -
    Arm type
    		 Placebo

    Investigational medicinal product name
    MT-7117 matching placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects self-administered 2 tablets of investigational medicinal product (IMP) with or without food each morning

    Arm title
    		 MT-7117 low dose
    Arm description
    		 -
    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
    Subjects self-administered 2 tablets of investigational medicinal product (IMP) with or without food each morning. Subjects received 1 tablet of MT-7117 and 1 tablet of matching placebo.

    Investigational medicinal product name
    MT-7117 matching placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects self-administered 2 tablets of investigational medicinal product (IMP) with or without food each morning. Subjects received 1 tablet of MT-7117 and 1 tablet of matching placebo.

    Arm title
    		 MT-7117 high dose
    Arm description
    		 -
    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
    Subjects self-administered 2 tablets of investigational medicinal product (IMP) with or without food each morning.

    Number of subjects in period 1
    		placebo MT-7117 low dose MT-7117 high dose
    Started
    61
    63
    60
    Completed
    57
    60
    58
    Not completed
    4
    3
    2
         Consent withdrawn by subject
    3
    2
    2
         Physician decision
    1
    -
    -
         Adverse event, non-fatal
    -
    1
    -
    Period 2
    Period 2 title
    26 Weeks Double-Blind Extention
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo -> low dose MT-7117
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    MT-7117 matching placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects self-administered 2 tablets of investigational medicinal product (IMP) with or without food each morning. Subjects received 1 tablet of MT-7117 and 1 tablet of matching placebo.

    Investigational medicinal product name
    MT-7117
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects self-administered 2 tablets of investigational medicinal product (IMP) with or without food each morning. Subjects received 1 tablet of MT-7117 and 1 tablet of matching placebo.

    Arm title
    		 Placebo -> high dose MT-7117
    Arm description
    		 -
    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
    Subjects self-administered 2 tablets of investigational medicinal product (IMP) with or without food each morning.

    Arm title
    		 MT-7117 low dose
    Arm description
    		 -
    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
    Subjects self-administered 2 tablets of investigational medicinal product (IMP) with or without food each morning. Subjects received 1 tablet of MT-7117 and 1 tablet of matching placebo.

    Investigational medicinal product name
    MT-7117 matching placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects self-administered 2 tablets of investigational medicinal product (IMP) with or without food each morning. Subjects received 1 tablet of MT-7117 and 1 tablet of matching placebo.

    Arm title
    		 MT-7117 high dose
    Arm description
    		 -
    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
    Subjects self-administered 2 tablets of investigational medicinal product (IMP) with or without food each morning.

    Number of subjects in period 2 [1]
    		Placebo -> low dose MT-7117 Placebo -> high dose MT-7117 MT-7117 low dose MT-7117 high dose
    Started
    28
    28
    56
    55
    Completed
    27
    28
    55
    52
    Not completed
    1
    0
    1
    3
         Adverse event, serious fatal
    -
    -
    -
    1
         Consent withdrawn by subject
    -
    -
    1
    2
         Lost to follow-up
    1
    -
    -
    -
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: Not all subjects took the offer to participate in a 6-month extension which all participants were on active drug but were double-blinded to dose starting immediately after the end of DBT period. Subjects who received MT-7117 during the DBT period continued the DBE in the same treatment arm and remained blinded. Subjects who received placebo were randomized to receive MT-7117 low or high dose in 1:1 ratio for the DBE period.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    placebo
    Reporting group description
    		 -

    Reporting group title
    MT-7117 low dose
    Reporting group description
    		 -

    Reporting group title
    MT-7117 high dose
    Reporting group description
    		 -

    Reporting group values
    		 placebo MT-7117 low dose MT-7117 high dose Total
    Number of subjects
    		 61 63 60 184
    Age categorical
    Units: Subjects
        Adolescents (12-17 years)
    		 12 13 12 37
        Adults 18-65
    		 47 45 46 138
        >65
    		 2 5 2 9
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 34.1 ± 15.6 33.5 ± 17.1 34.4 ± 15.3 -
    Gender categorical
    Units: Subjects
        Female
    		 30 30 33 93
        Male
    		 31 33 27 91
    Subject analysis sets

    Subject analysis set title
    DBT ITT1 Placebo
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.

    Subject analysis set title
    DBT ITT1 MT-7117 low dose
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.

    Subject analysis set title
    DBT ITT1 MT-7117 high dose
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.

    Subject analysis sets values
    		 DBT ITT1 Placebo DBT ITT1 MT-7117 low dose DBT ITT1 MT-7117 high dose
    Number of subjects
    60
    63
    60
    Age categorical
    Units: Subjects
        Adolescents (12-17 years)
    11
    13
    12
        Adults 18-65
    47
    45
    46
        >65
    2
    5
    2
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    34.4 ± 15.6
    33.5 ± 17.1
    34.4 ± 15.3
    Gender categorical
    Units: Subjects
        Female
    29
    30
    33
        Male
    31
    33
    27

    End points

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    End points reporting groups
    Reporting group title
    placebo
    Reporting group description
    		 -

    Reporting group title
    MT-7117 low dose
    Reporting group description
    		 -

    Reporting group title
    MT-7117 high dose
    Reporting group description
    		 -
    Reporting group title
    Placebo -> low dose MT-7117
    Reporting group description
    		 -

    Reporting group title
    Placebo -> high dose MT-7117
    Reporting group description
    		 -

    Reporting group title
    MT-7117 low dose
    Reporting group description
    		 -

    Reporting group title
    MT-7117 high dose
    Reporting group description
    		 -

    Subject analysis set title
    DBT ITT1 Placebo
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.

    Subject analysis set title
    DBT ITT1 MT-7117 low dose
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.

    Subject analysis set title
    DBT ITT1 MT-7117 high dose
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.

    Primary: Change from baseline in average daily sunlight exposure time (minutes) to first prodromal symptom (burning, tingling, itching, or stinging) associated with sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset at Week 26 (Visit 7)

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    End point title
    		 Change from baseline in average daily sunlight exposure time (minutes) to first prodromal symptom (burning, tingling, itching, or stinging) associated with sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset at Week 26 (Visit 7)
    End point description
    End point type
    Primary
    End point timeframe
    Week 26 (Visit 7)
    End point values
    		 DBT ITT1 Placebo DBT ITT1 MT-7117 low dose DBT ITT1 MT-7117 high dose
    Number of subjects analysed
    60
    63
    60
    Units: minute
    least squares mean (standard error)
        Least Square Mean (SE)
    20.59 ± 10.29
    30.39 ± 10.04
    43.29 ± 10.13
    Statistical analysis title
    		 Change from BL at Week 26 (DBT EOT) - lower dose
    Comparison groups
    DBT ITT1 Placebo v DBT ITT1 MT-7117 low dose
    Number of subjects included in analysis
    123
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.496
    Method
    		 mixed-effect model for repeated measures
    Parameter type
    		 Least Square Mean Difference vs Placebo
    Point estimate
    9.8
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -18.52
         upper limit
    		 38.12
    Variability estimate
    Standard error of the mean
    Dispersion value
    14.35
    Statistical analysis title
    		 Change from BL at Week 26 (DBT EOT) - higher dose
    Comparison groups
    DBT ITT1 Placebo v DBT ITT1 MT-7117 high dose
    Number of subjects included in analysis
    120
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.116
    Method
    		 mixed-effect model for repeated measures
    Parameter type
    		 Least Square Mean Difference vs Placebo
    Point estimate
    22.7
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -5.68
         upper limit
    		 51.09
    Variability estimate
    Standard error of the mean
    Dispersion value
    14.38

    Secondary: Patient Global Impression of Change (PGIC) at Week 26

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    End point title
    		 Patient Global Impression of Change (PGIC) at Week 26
    End point description
    End point type
    Secondary
    End point timeframe
    Week 26
    End point values
    		 DBT ITT1 Placebo DBT ITT1 MT-7117 low dose DBT ITT1 MT-7117 high dose
    Number of subjects analysed
    56
    59
    56
    Units: point
        least squares mean (standard error)
    3.25 ± 1.14
    2.41 ± 0.14
    1.82 ± 0.14
    Statistical analysis title
    		 Patient Global Impression of Change at week26
    Comparison groups
    DBT ITT1 Placebo v DBT ITT1 MT-7117 high dose
    Number of subjects included in analysis
    112
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.001
    Method
    		 mixed-effect model for repeated measures
    Parameter type
    		 Least Square Mean Difference vs Placebo
    Point estimate
    -1.43
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.83
         upper limit
    		 -1.03
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.2
    Statistical analysis title
    		 Patient Global Impression of Change at week26
    Comparison groups
    DBT ITT1 Placebo v DBT ITT1 MT-7117 low dose
    Number of subjects included in analysis
    115
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.001
    Method
    		 mixed-effect model for repeated measures
    Parameter type
    		 Least Square Mean Difference vs Placebo
    Point estimate
    -0.84
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.23
         upper limit
    		 -0.44
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.2

    Secondary: Total number of sunlight-induced pain events defined as prodrome symptoms (burning, tingling, itching, or stinging) with pain rating of 1-10 on the Likert scale during the 26-week double-blind treatment period.

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    End point title
    		 Total number of sunlight-induced pain events defined as prodrome symptoms (burning, tingling, itching, or stinging) with pain rating of 1-10 on the Likert scale during the 26-week double-blind treatment period.
    End point description
    End point type
    Secondary
    End point timeframe
    During the 26-week double-blind treatment period
    End point values
    		 DBT ITT1 Placebo DBT ITT1 MT-7117 low dose DBT ITT1 MT-7117 high dose
    Number of subjects analysed
    60
    63
    60
    Units: pain rating
        geometric mean (standard deviation)
    23.90 ± 30.93
    15.87 ± 22.05
    13.78 ± 18.70
    Statistical analysis title
    		 Total number of sunlight-induced pain events
    Comparison groups
    DBT ITT1 Placebo v DBT ITT1 MT-7117 low dose
    Number of subjects included in analysis
    123
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [1]
    P-value
    		 = 0.199
    Method
    		 see analysis type comment
    Parameter type
    		 incidence rate ratio
    Point estimate
    0.76
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.49
         upper limit
    		 1.16
    Notes
    [1] - Negative binomial regression model with log link was used.
    Statistical analysis title
    		 Total number of sunlight-induced pain events
    Comparison groups
    DBT ITT1 Placebo v DBT ITT1 MT-7117 high dose
    Number of subjects included in analysis
    120
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [2]
    P-value
    		 = 0.006
    Method
    		 see analysis type comment
    Parameter type
    		 incidence rate ratio
    Point estimate
    0.55
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.36
         upper limit
    		 0.84
    Notes
    [2] - Negative binomial regression model with log link was used.

    Secondary: Change from baseline for total score in the domain of pain intensity in the PROMIS-57 at Week 26

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    End point title
    		 Change from baseline for total score in the domain of pain intensity in the PROMIS-57 at Week 26
    End point description
    End point type
    Secondary
    End point timeframe
    week 26
    End point values
    		 DBT ITT1 Placebo DBT ITT1 MT-7117 low dose DBT ITT1 MT-7117 high dose
    Number of subjects analysed
    49
    59
    56
    Units: total score
        least squares mean (standard error)
    -1.42 ± 0.18
    -1.56 ± 0.16
    -1.65 ± 0.17
    Statistical analysis title
    		 Change from Baseline for Total Score in Pain
    Comparison groups
    DBT ITT1 Placebo v DBT ITT1 MT-7117 low dose
    Number of subjects included in analysis
    108
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.55
    Method
    		 mixed-effect model for repeated measures
    Parameter type
    		 Least Square Mean Difference vs Placebo
    Point estimate
    -0.15
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.62
         upper limit
    		 0.33
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.24
    Statistical analysis title
    		 Change from Baseline for Total Score in Pain
    Comparison groups
    DBT ITT1 Placebo v DBT ITT1 MT-7117 high dose
    Number of subjects included in analysis
    105
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.343
    Method
    		 mixed-effect model for repeated measures
    Parameter type
    		 Least Square Mean Difference vs Placebo
    Point estimate
    -0.23
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.72
         upper limit
    		 0.25
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.25

    Secondary: The percentage of subjects who are responders

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    End point title
    		 The percentage of subjects who are responders
    End point description
    The percentage of subjects who are responders based on average daily sunlight exposure time to first prodromal symptom associated with sunlight exposure between 1 hour post-sunrise and 1 hour pre-sunset defined by within-subject meaningful change of 66 minutes increase from baseline to Week 26
    End point type
    Secondary
    End point timeframe
    week 26
    End point values
    		 DBT ITT1 Placebo DBT ITT1 MT-7117 low dose DBT ITT1 MT-7117 high dose
    Number of subjects analysed
    60
    63
    60
    Units: responders
        Change from baseline ≥66 minutes
    12
    11
    16
    Statistical analysis title
    		 Responder Rate
    Comparison groups
    DBT ITT1 MT-7117 low dose v DBT ITT1 Placebo
    Number of subjects included in analysis
    123
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.642
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    0.805
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.323
         upper limit
    		 2.007
    Statistical analysis title
    		 Responder Rate
    Comparison groups
    DBT ITT1 MT-7117 high dose v DBT ITT1 Placebo
    Number of subjects included in analysis
    120
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.431
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    1.412
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.598
         upper limit
    		 3.336

    Secondary: Change from baseline for total score in the domain of physical function in the PROMIS-57 at Week 26.

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    End point title
    		 Change from baseline for total score in the domain of physical function in the PROMIS-57 at Week 26.
    End point description
    End point type
    Secondary
    End point timeframe
    week 26
    End point values
    		 DBT ITT1 Placebo DBT ITT1 MT-7117 low dose DBT ITT1 MT-7117 high dose
    Number of subjects analysed
    49
    59
    56
    Units: total score
        least squares mean (standard error)
    0.67 ± 0.36
    1.54 ± 0.32
    1.22 ± 0.33
    Statistical analysis title
    		 Change from Baseline for Total Score in Physical
    Comparison groups
    DBT ITT1 Placebo v DBT ITT1 MT-7117 low dose
    Number of subjects included in analysis
    108
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.072
    Method
    		 mixed-effect model for repeated measures
    Parameter type
    		 Least Square Mean Difference vs Placebo
    Point estimate
    0.87
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.08
         upper limit
    		 1.82
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.48
    Statistical analysis title
    		 Change from Baseline for Total Score in Physical
    Comparison groups
    DBT ITT1 Placebo v DBT ITT1 MT-7117 high dose
    Number of subjects included in analysis
    105
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.252
    Method
    		 mixed-effect model for repeated measures
    Parameter type
    		 Least Square Mean Difference vs Placebo
    Point estimate
    0.56
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.4
         upper limit
    		 1.51
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.48

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Screening to Follow up.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.1
    Reporting groups
    Reporting group title
    SAF1 Placebo
    Reporting group description
    		 -

    Reporting group title
    SAF1 MT-7117 low dose
    Reporting group description
    		 -

    Reporting group title
    SAF1 MT-7117 high dose
    Reporting group description
    		 -

    Reporting group title
    SAF2 Placebo -> MT-7117 low dose
    Reporting group description
    		 -

    Reporting group title
    SAF2 Placebo -> MT-7117 high dose
    Reporting group description
    		 -

    Reporting group title
    SAF2 MT-7117 low dose
    Reporting group description
    		 -

    Reporting group title
    SAF2 MT-7117 high dose
    Reporting group description
    		 -

    Serious adverse events
    		 SAF1 Placebo SAF1 MT-7117 low dose SAF1 MT-7117 high dose SAF2 Placebo -> MT-7117 low dose SAF2 Placebo -> MT-7117 high dose SAF2 MT-7117 low dose SAF2 MT-7117 high dose
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 61 (0.00%)
    2 / 63 (3.17%)
    2 / 60 (3.33%)
    2 / 28 (7.14%)
    1 / 28 (3.57%)
    1 / 56 (1.79%)
    2 / 54 (3.70%)
         number of deaths (all causes)
    0
    0
    1
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    1
    0
    0
    0
    0
    Investigations
    Hepatic enzyme increased
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    0 / 60 (0.00%)
    2 / 28 (7.14%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 3
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Fibula fracture
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    0 / 60 (0.00%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Congenital, familial and genetic disorders
    Porphyria non-acute
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    0 / 60 (0.00%)
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Myocarditis
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 63 (1.59%)
    0 / 60 (0.00%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pericarditis
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    0 / 60 (0.00%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 56 (1.79%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Oesophagitis
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    0 / 60 (0.00%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    1 / 54 (1.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Cholestasis
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    1 / 60 (1.67%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    1 / 60 (1.67%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Meningitis viral
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 63 (1.59%)
    0 / 60 (0.00%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 3%
    Non-serious adverse events
    		 SAF1 Placebo SAF1 MT-7117 low dose SAF1 MT-7117 high dose SAF2 Placebo -> MT-7117 low dose SAF2 Placebo -> MT-7117 high dose SAF2 MT-7117 low dose SAF2 MT-7117 high dose
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    40 / 61 (65.57%)
    44 / 63 (69.84%)
    44 / 60 (73.33%)
    21 / 28 (75.00%)
    20 / 28 (71.43%)
    34 / 56 (60.71%)
    32 / 54 (59.26%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Melanocytic naevus
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    1 / 61 (1.64%)
    6 / 63 (9.52%)
    8 / 60 (13.33%)
    4 / 28 (14.29%)
    3 / 28 (10.71%)
    3 / 56 (5.36%)
    2 / 54 (3.70%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Dysplastic naevus
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    2 / 60 (3.33%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Investigations
    Aspartate aminotransferase increased
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    1 / 61 (1.64%)
    4 / 63 (6.35%)
    0 / 60 (0.00%)
    3 / 28 (10.71%)
    1 / 28 (3.57%)
    1 / 56 (1.79%)
    1 / 54 (1.85%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Alanine aminotransferase increased
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    1 / 61 (1.64%)
    3 / 63 (4.76%)
    0 / 60 (0.00%)
    2 / 28 (7.14%)
    0 / 28 (0.00%)
    1 / 56 (1.79%)
    2 / 54 (3.70%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Gamma-glutamyltransferase increased
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    5 / 61 (8.20%)
    3 / 63 (4.76%)
    0 / 60 (0.00%)
    2 / 28 (7.14%)
    1 / 28 (3.57%)
    1 / 56 (1.79%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    SARS-CoV-2 test positive
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    0 / 60 (0.00%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    3 / 56 (5.36%)
    1 / 54 (1.85%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Weight decreased
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    0 / 60 (0.00%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    2 / 56 (3.57%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Vaccination complication
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    1 / 61 (1.64%)
    0 / 63 (0.00%)
    2 / 60 (3.33%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    2 / 54 (3.70%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Post vaccination syndrome
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    2 / 61 (3.28%)
    0 / 63 (0.00%)
    0 / 60 (0.00%)
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    2 / 54 (3.70%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Cardiac disorders
    Sinus bradycardia
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    0 / 60 (0.00%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    2 / 54 (3.70%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Nervous system disorders
    Headache
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    3 / 61 (4.92%)
    8 / 63 (12.70%)
    9 / 60 (15.00%)
    2 / 28 (7.14%)
    3 / 28 (10.71%)
    2 / 56 (3.57%)
    2 / 54 (3.70%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    General disorders and administration site conditions
    Fatigue
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    4 / 61 (6.56%)
    3 / 63 (4.76%)
    3 / 60 (5.00%)
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    2 / 56 (3.57%)
    1 / 54 (1.85%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Pyrexia
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    1 / 61 (1.64%)
    2 / 63 (3.17%)
    3 / 60 (5.00%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 56 (1.79%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Gastrointestinal disorders
    Nausea
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    4 / 61 (6.56%)
    4 / 63 (6.35%)
    16 / 60 (26.67%)
    1 / 28 (3.57%)
    5 / 28 (17.86%)
    1 / 56 (1.79%)
    2 / 54 (3.70%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Diarrhoea
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    1 / 61 (1.64%)
    5 / 63 (7.94%)
    8 / 60 (13.33%)
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    0 / 56 (0.00%)
    1 / 54 (1.85%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Abdominal pain
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    1 / 61 (1.64%)
    1 / 63 (1.59%)
    4 / 60 (6.67%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Abdominal discomfort
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    1 / 61 (1.64%)
    1 / 63 (1.59%)
    3 / 60 (5.00%)
    1 / 28 (3.57%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    1 / 54 (1.85%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Pigmentation lip
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    3 / 63 (4.76%)
    1 / 60 (1.67%)
    1 / 28 (3.57%)
    1 / 28 (3.57%)
    1 / 56 (1.79%)
    1 / 54 (1.85%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Dyspepsia
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 63 (1.59%)
    2 / 60 (3.33%)
    1 / 28 (3.57%)
    1 / 28 (3.57%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Vomiting
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    2 / 61 (3.28%)
    1 / 63 (1.59%)
    2 / 60 (3.33%)
    1 / 28 (3.57%)
    1 / 28 (3.57%)
    0 / 56 (0.00%)
    2 / 54 (3.70%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Abdominal pain upper
    Additional description: Number of occurrences for each event were not reportedin this study.
         subjects affected / exposed
    1 / 61 (1.64%)
    0 / 63 (0.00%)
    2 / 60 (3.33%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 56 (1.79%)
    1 / 54 (1.85%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Frequent bowel movements
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    2 / 60 (3.33%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 63 (1.59%)
    2 / 60 (3.33%)
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    1 / 56 (1.79%)
    1 / 54 (1.85%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Skin and subcutaneous tissue disorders
    Skin discolouration
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    5 / 63 (7.94%)
    6 / 60 (10.00%)
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Skin hyperpigmentation
    Additional description: Number of occurrences for each event were not reportedin this study.
         subjects affected / exposed
    1 / 61 (1.64%)
    2 / 63 (3.17%)
    7 / 60 (11.67%)
    2 / 28 (7.14%)
    3 / 28 (10.71%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Ephelides
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    1 / 61 (1.64%)
    3 / 63 (4.76%)
    4 / 60 (6.67%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Acne
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 63 (1.59%)
    3 / 60 (5.00%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    1 / 56 (1.79%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Hair colour changes
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 63 (1.59%)
    3 / 60 (5.00%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Photosensitivity reaction
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    6 / 61 (9.84%)
    1 / 63 (1.59%)
    2 / 60 (3.33%)
    1 / 28 (3.57%)
    2 / 28 (7.14%)
    0 / 56 (0.00%)
    2 / 54 (3.70%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Solar lentigo
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 63 (1.59%)
    2 / 60 (3.33%)
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Pigmentation disorder
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    2 / 60 (3.33%)
    0 / 28 (0.00%)
    1 / 28 (3.57%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Back pain
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    1 / 61 (1.64%)
    3 / 63 (4.76%)
    0 / 60 (0.00%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Infections and infestations
    COVID-19
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    2 / 61 (3.28%)
    5 / 63 (7.94%)
    1 / 60 (1.67%)
    7 / 28 (25.00%)
    8 / 28 (28.57%)
    11 / 56 (19.64%)
    10 / 54 (18.52%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Upper respiratory tract infection
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    5 / 61 (8.20%)
    1 / 63 (1.59%)
    2 / 60 (3.33%)
    1 / 28 (3.57%)
    1 / 28 (3.57%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Nasopharyngitis
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    1 / 61 (1.64%)
    1 / 63 (1.59%)
    1 / 60 (1.67%)
    2 / 28 (7.14%)
    0 / 28 (0.00%)
    4 / 56 (7.14%)
    3 / 54 (5.56%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Metabolism and nutrition disorders
    Decreased appetite
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    2 / 60 (3.33%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    0 / 56 (0.00%)
    0 / 54 (0.00%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Vitamin D deficiency
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    0 / 63 (0.00%)
    0 / 60 (0.00%)
    3 / 28 (10.71%)
    1 / 28 (3.57%)
    3 / 56 (5.36%)
    2 / 54 (3.70%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0
    Iron deficiency
    Additional description: Number of occurrences for each event were not reported in this study.
         subjects affected / exposed
    0 / 61 (0.00%)
    1 / 63 (1.59%)
    0 / 60 (0.00%)
    0 / 28 (0.00%)
    0 / 28 (0.00%)
    4 / 56 (7.14%)
    2 / 54 (3.70%)
         occurrences all number
    0
    0
    0
    0
    0
    0
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Jul 2020
    Amendment 1 V2.0. The main changes were: -reflect inclusion of adolescents. - Sample size of the study was increased due to statistical considerations. - Secondary and Exploratory efficacy endpoints were amended. -Additional demographic information will be collected. -Description of the subgroup analyses was added. -Description of prohibited or precautionary concomitant medications was amended. -Approved prohibited concomitant medications were revised.
    08 Jun 2021
    Amendment 2 v3.0. The main changes were: - One exploratory objective to investigate the effect of mutations in MClR gene on efficacy was added. - Study methodology was updated to clarify the randomization rule for subjects with adolescents and better clarify the schedule of assessments. -The language in Number of planned subjects was revised correctly considering the stratification rule with subjects under 45 kg body weight. - Exclusion criteria 11 was revised to specify the criteria to exclude more than stage 3a Chronic Kidney Disease patients based on eGFR, and the equations for calculating eGFR for adolescents were specified. - The language in Risk: Benefit statement was updated to include the new findings of non-clinical findings (pre- and postnatal development study in rats) and COVID-19 pandemic.
    25 Oct 2021
    Amendment 3.0 v4.0. v4.0 was replaced by Amendment 3.1 v4.1 and approved as a part of v4.1 which was non-substatial amendment. The main changes were: - Sponsor’s UK representative address was added’ -Increased number of patients and sample size estimation. -Withdrawal of Individual Subjects section was updated. -QP certification revised to define UK and EU certification. -Adverse Events of Special Interest section was updated. -Management and Evaluation of Hepatic Adverse Events of Special Interest section was defined in more detail. -An external independent Data Monitoring Committee (iDMC) will be established.

    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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