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    Clinical Trial Results:
    A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)

    Summary
    EudraCT number
    		 2020-002812-36
    Trial protocol
    		 DE   ES   FR   IT  
    Global end of trial date
    02 Oct 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    17 Apr 2024
    First version publication date
    17 Apr 2024
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    PTC743-NEU-003-FA
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04577352
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    PTC Therapeutics, Inc.
    Sponsor organisation address
    PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, United States, NJ 07080
    Public contact
    Medical Information, PTC Therapeutics International Limited, +353 19068700, medinfo@ptcbio.com
    Scientific contact
    PTC Therapeutics, Inc., Medical Information, +011 44 1-866-562-4620, medinfo@ptcbio.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001238-PIP03-21
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    02 Oct 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    02 Oct 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study was to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich Ataxia (FA).
    Protection of trial subjects
    This trial was designed and monitored in accordance with sponsor procedures, which comply with the ethical principles of Good Clinical Practice (GCP) as required by the major regulatory authorities, and in accordance with the Declaration of Helsinki.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    17 Dec 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
    Australia: 5
    Country: Number of subjects enrolled
    New Zealand: 3
    Country: Number of subjects enrolled
    France: 11
    Country: Number of subjects enrolled
    Germany: 5
    Country: Number of subjects enrolled
    Italy: 10
    Country: Number of subjects enrolled
    Spain: 9
    Country: Number of subjects enrolled
    Brazil: 16
    Country: Number of subjects enrolled
    Canada: 23
    Country: Number of subjects enrolled
    United States: 64
    Worldwide total number of subjects
    146
    EEA total number of subjects
    35
    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)
    31
    Adolescents (12-17 years)
    69
    Adults (18-64 years)
    44
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 The study included a double-blind, placebo-controlled phase and an open-label extension phase.

    Period 1
    Period 1 title
    Double-blind Phase (72 Weeks)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Vatiquinone
    Arm description
    		 Participants received vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if ˂12 years of age and weighing ˂25 kilograms (kg) or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Vatiquinone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Vatiquinone was administered per dose and schedule specified in the arm description.

    Arm title
    		 Placebo
    Arm description
    		 Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo matched to vatiquinone was administered per schedule specified in the arm description.

    Number of subjects in period 1
    		Vatiquinone Placebo
    Started
    73
    73
    Received at least 1 dose of study drug
    73
    73
    Completed
    63
    65
    Not completed
    10
    8
         Consent withdrawn by subject
    3
    2
         Adverse event, non-fatal
    6
    3
         Death
    -
    1
         Other than specified
    1
    1
         Protocol deviation
    -
    1
    Period 2
    Period 2 title
    Open-label Phase (24 Weeks)
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Vatiquinone
    Arm description
    		 Participants received vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if ˂12 years of age and weighing ˂25 kilograms (kg) or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Vatiquinone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Vatiquinone was administered per dose and schedule specified in the arm description.

    Arm title
    		 Placebo
    Arm description
    		 Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Vatiquinone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, Capsule
    Routes of administration
    Oral use, Oral use
    Dosage and administration details
    Vatiquinone was administered per dose and schedule specified in the arm description.

    Number of subjects in period 2
    		Vatiquinone Placebo
    Started
    63
    65
    Received at least 1 dose of study drug
    63
    65
    Completed
    63
    62
    Not completed
    0
    3
         Adverse event, non-fatal
    -
    2
         Other than specified
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Vatiquinone
    Reporting group description
    		 Participants received vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if ˂12 years of age and weighing ˂25 kilograms (kg) or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.

    Reporting group values
    		 Vatiquinone Placebo Total
    Number of subjects
    		 73 73 146
    Age Categorical
    Units: Subjects
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 18.9 ± 12.24 18.2 ± 11.27 -
    Gender Categorical
    Units: Subjects
        Female
    		 45 42 87
        Male
    		 28 31 59
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 10 9 19
        Not Hispanic or Latino
    		 63 63 126
        Not Reported
    		 0 1 1
    Race
    Units: Subjects
        White
    		 68 69 137
        Other
    		 5 4 9

    End points

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    End points reporting groups
    Reporting group title
    Vatiquinone
    Reporting group description
    		 Participants received vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if ˂12 years of age and weighing ˂25 kilograms (kg) or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.
    Reporting group title
    Vatiquinone
    Reporting group description
    		 Participants received vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if ˂12 years of age and weighing ˂25 kilograms (kg) or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase and for 24 weeks during the open-label phase.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.

    Primary: Change From Baseline in the mFARS Score at Week 72 - Modified Intent-to-treat (mITT) Analysis Set

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    End point title
    		 Change From Baseline in the mFARS Score at Week 72 - Modified Intent-to-treat (mITT) Analysis Set
    End point description
    mFARS is a 93-item scale; comprised of neurologic component of Friedreich Ataxia Rating Scale (FARS). For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. Total mFARS scores for each subscale: bulbar (0 to 5), upper limb coordination (0 to 36), lower limb coordination (0 to 16), and upright stability (0 to 36). mFARS total score was a composite score of all 4 subscales, ranging from 0 (normal) to 93 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. Least square (LS) mean and standard error (SE) was calculated using mixed-model repeated measures (MMRM). The mITT analysis set included all randomized participants, between age of 7 and 21 years who received at least 1 dose of study drug, had baseline and at least 1 postbaseline measurement of primary endpoint.
    End point type
    Primary
    End point timeframe
    Baseline, Week 72
    End point values
    		 Vatiquinone Placebo
    Number of subjects analysed
    61
    62
    Units: units on a scale
        least squares mean (standard error)
    1.218 ± 0.9652
    2.828 ± 0.9994
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Vatiquinone v Placebo
    Number of subjects included in analysis
    123
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.144
    Method
    		 Mixed models analysis
    Parameter type
    		 LS Mean Difference
    Point estimate
    -1.61
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -3.769
         upper limit
    		 0.55
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.1016

    Primary: Change From Baseline in the mFARS Score at Week 72 - Intent-to-treat (ITT) Analysis Set

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    End point title
    		 Change From Baseline in the mFARS Score at Week 72 - Intent-to-treat (ITT) Analysis Set
    End point description
    mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. Total mFARS scores for each subscale: bulbar (0 to 5), upper limb coordination (0 to 36), lower limb coordination (0 to 16), and upright stability (0 to 36). mFARS total score was a composite score of all 4 subscales, ranging from 0 (normal) to 93 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
    End point type
    Primary
    End point timeframe
    Baseline, Week 72
    End point values
    		 Vatiquinone Placebo
    Number of subjects analysed
    70
    73
    Units: units on a scale
        least squares mean (standard error)
    0.898 ± 0.8500
    2.555 ± 0.8770
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Vatiquinone v Placebo
    Number of subjects included in analysis
    143
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.0984
    Method
    		 Mixed models analysis
    Parameter type
    		 LS Mean Difference
    Point estimate
    -1.657
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -3.622
         upper limit
    		 0.308
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.0027

    Secondary: Change From Baseline in FARS-ADL Score at Week 72 - ITT Analysis Set

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    End point title
    		 Change From Baseline in FARS-ADL Score at Week 72 - ITT Analysis Set
    End point description
    The ADL component of the FARS includes 9 subscales: speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function. Each of these subscales is rated on a 5-point scale where 0=normal to 4=severe disability/inability to carry out activity independently for a total possible score of 0 to 36, with higher scores representing greater disability/dependency. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 72
    End point values
    		 Vatiquinone Placebo
    Number of subjects analysed
    70
    73
    Units: units on a scale
        least squares mean (standard error)
    0.707 ± 0.5219
    1.692 ± 0.5402
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Vatiquinone v Placebo
    Number of subjects included in analysis
    143
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.082
    Method
    		 Mixed models analysis
    Parameter type
    		 LS Mean Difference
    Point estimate
    -0.985
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -2.095
         upper limit
    		 0.125
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.5664

    Secondary: Change From Baseline in Friedreich Ataxia Rating Scale Activities of Daily Living (FARS-ADL) Score at Week 72 - mITT Analysis Set

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    End point title
    		 Change From Baseline in Friedreich Ataxia Rating Scale Activities of Daily Living (FARS-ADL) Score at Week 72 - mITT Analysis Set
    End point description
    The ADL component of the FARS includes 9 subscales: speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function. Each of these subscales is rated on a 5-point scale where 0=normal to 4=severe disability/inability to carry out activity independently for a total possible score of 0 to 36, with higher scores representing greater disability/dependency. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. The mITT analysis set included all randomized participants, between age of 7 and 21 years, inclusive, at Screening, had received at least 1 dose of study drug, had baseline and at least 1 post-baseline measurement of the primary endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 72
    End point values
    		 Vatiquinone Placebo
    Number of subjects analysed
    61
    62
    Units: units on a scale
        least squares mean (standard error)
    0.759 ± 0.5992
    1.677 ± 0.6202
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Vatiquinone v Placebo
    Number of subjects included in analysis
    123
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.1382
    Method
    		 Mixed models analysis
    Parameter type
    		 LS Mean Difference
    Point estimate
    -0.918
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -2.132
         upper limit
    		 0.296
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.6194

    Secondary: Change From Baseline in 1-Minute Walk Test (1MWT) at Week 72 - mITT Analysis Set

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    End point title
    		 Change From Baseline in 1-Minute Walk Test (1MWT) at Week 72 - mITT Analysis Set
    End point description
    The 1MWT is a timed performance test used to measure functional ability, walking endurance, balance, and muscle performance by measuring maximal walking speed in 1 minute. Participants were instructed to walk as quickly as possible for 1 minute without running. Maximal walking speed was measured upon completion of the walk and distance was recorded. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. The mITT analysis set included all randomized participants, between age of 7 and 21 years, inclusive, at Screening, had received at least 1 dose of study drug, had baseline and at least 1 post-baseline measurement of the primary endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 72
    End point values
    		 Vatiquinone Placebo
    Number of subjects analysed
    61
    62
    Units: meters
        least squares mean (standard error)
    -4.324 ± 2.5882
    -9.290 ± 2.6843
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Vatiquinone v Placebo
    Number of subjects included in analysis
    123
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.08
    Method
    		 Mixed models analysis
    Parameter type
    		 LS Mean Difference
    Point estimate
    4.966
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.593
         upper limit
    		 10.525
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.8363

    Secondary: Change From Baseline in 1MWT at Week 72 - ITT Analysis Set

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    End point title
    		 Change From Baseline in 1MWT at Week 72 - ITT Analysis Set
    End point description
    The 1MWT is a timed performance test used to measure functional ability, walking endurance, balance, and muscle performance by measuring maximal walking speed in 1 minute. Participants were instructed to walk as quickly as possible for 1 minute without running. Maximal walking speed was measured upon completion of the walk and distance was recorded. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 72
    End point values
    		 Vatiquinone Placebo
    Number of subjects analysed
    70
    73
    Units: meters
        least squares mean (standard error)
    -4.623 ± 2.2113
    -9.462 ± 2.2687
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Vatiquinone v Placebo
    Number of subjects included in analysis
    143
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.0551
    Method
    		 Mixed models analysis
    Parameter type
    		 LS Mean Difference
    Point estimate
    4.839
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.106
         upper limit
    		 9.784
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.5231

    Secondary: Number of Falls per 28 Days over Every 24-Week Period - mITT Analysis Set

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    End point title
    		 Number of Falls per 28 Days over Every 24-Week Period - mITT Analysis Set
    End point description
    Each participant was required to maintain a fall log, which included the date and time of each fall. Falls as defined by World Health Organization as "inadvertently coming to rest on the ground, floor or other lower level, excluding intentional change in position to rest in furniture, wall or other objects," were reported. Number of falls per 28 days during a time interval was calculated as the number of falls during the period divided by the number of days during the interval, and multiplied by 28. The falls that occurred on or after the first Loss of Ambulation visit were excluded from the analysis. The mITT analysis set included all randomized participants, between age of 7 and 21 years, inclusive, at Screening, had received at least 1 dose of study drug, had baseline and at least 1 post-baseline measurement of the primary endpoint. ‘Overall number of participants analyzed’ = participants evaluable for this endpoint. ‘n’ = participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Week 1-24, Week 25-48, and Week 49-72
    End point values
    		 Vatiquinone Placebo
    Number of subjects analysed
    60
    62
    Units: falls
    arithmetic mean (standard deviation)
        Week 1-24 (n = 60, 62)
    5.029 ± 14.4686
    3.999 ± 6.9569
        Week 25-48 (n = 56, 60)
    4.098 ± 11.5289
    3.900 ± 9.0212
        Week 49-72 (n = 54, 57)
    3.732 ± 10.0790
    4.462 ± 12.1785
    No statistical analyses for this end point

    Secondary: Number of Falls per 28 Days over Every 24-Week Period - ITT Analysis Set

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    End point title
    		 Number of Falls per 28 Days over Every 24-Week Period - ITT Analysis Set
    End point description
    Each participant was required to maintain a fall log, which included the date and time of each fall. Falls as defined by World Health Organization as "inadvertently coming to rest on the ground, floor or other lower level, excluding intentional change in position to rest in furniture, wall or other objects," were reported. Number of falls per 28 days during a time interval was calculated as the number of falls during the period divided by the number of days during the interval, and multiplied by 28. The falls that occurred on or after the first Loss of Ambulation visit were excluded from the analysis. The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint. ‘Overall number of participants analyzed’ = participants evaluable for this endpoint. ‘n’ = participants evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Week 1-24, Week 25-48, and Week 49-72
    End point values
    		 Vatiquinone Placebo
    Number of subjects analysed
    69
    73
    Units: falls
    arithmetic mean (standard deviation)
        Week 1-24 (n = 69, 73)
    4.464 ± 13.5618
    3.535 ± 6.5231
        Week 25-48 (n = 63, 69)
    3.920 ± 10.9850
    3.518 ± 8.4701
        Week 49-72 (n = 60, 65)
    3.407 ± 9.6043
    3.974 ± 11.4690
    No statistical analyses for this end point

    Other pre-specified: Change From Baseline in the Upright Stability Subscale of the mFARS at Week 72 - mITT Analysis Set

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    End point title
    		 Change From Baseline in the Upright Stability Subscale of the mFARS at Week 72 - mITT Analysis Set
    End point description
    mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. The upright stability subscale score ranges from 0 (normal) to 36 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. The mITT analysis set included all randomized participants, between age of 7 and 21 years who received at least 1 dose of study drug, had baseline and at least 1 postbaseline measurement of primary endpoint.
    End point type
    Other pre-specified
    End point timeframe
    Baseline, Week 72
    End point values
    		 Vatiquinone Placebo
    Number of subjects analysed
    61
    62
    Units: units on a scale
        least squares mean (standard error)
    1.734 ± 0.4911
    2.991 ± 0.5094
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Vatiquinone v Placebo
    Number of subjects included in analysis
    123
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.0212
    Method
    		 Mixed models analysis
    Parameter type
    		 LS Mean Difference
    Point estimate
    -1.257
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -2.325
         upper limit
    		 -0.188
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.5453

    Other pre-specified: Change From Baseline in the Upright Stability Subscale of the mFARS at Week 72 - ITT Analysis Set

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    End point title
    		 Change From Baseline in the Upright Stability Subscale of the mFARS at Week 72 - ITT Analysis Set
    End point description
    mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. The upright stability subscale score ranges from 0 (normal) to 36 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
    End point type
    Other pre-specified
    End point timeframe
    Baseline, Week 72
    End point values
    		 Vatiquinone Placebo
    Number of subjects analysed
    70
    73
    Units: units on a scale
        least squares mean (standard error)
    1.379 ± 0.4321
    2.489 ± 0.4468
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Vatiquinone v Placebo
    Number of subjects included in analysis
    143
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.0246
    Method
    		 Mixed models analysis
    Parameter type
    		 LS Mean Difference
    Point estimate
    -1.11
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -2.079
         upper limit
    		 -0.142
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.4941

    Other pre-specified: Change From Baseline in the Modified Fatigue Impact Scale (MFIS) Score at Week 72 - mITT Analysis Set

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    End point title
    		 Change From Baseline in the Modified Fatigue Impact Scale (MFIS) Score at Week 72 - mITT Analysis Set
    End point description
    The MFIS is a 21-item, reliable, validated instrument that has been utilized in many neurological disorders. It is a modified form of the Fatigue Impact Scale, a component of the Multiple Sclerosis Quality of Life Inventory. Each item was scored on a scale of 0 (never) to 4 (almost always). The total score was the sum of all item’s score and ranged from 0 (no fatigue impact) to 84 (almost always impacted by fatigue). Higher scores indicated greater impact of fatigue on participant function. The mITT analysis set included all randomized participants, between age of 7 and 21 years who received at least 1 dose of study drug, had baseline and at least 1 postbaseline measurement of primary endpoint.
    End point type
    Other pre-specified
    End point timeframe
    Baseline, Week 72
    End point values
    		 Vatiquinone Placebo
    Number of subjects analysed
    61
    62
    Units: units on a scale
        least squares mean (standard error)
    -0.756 ± 2.2197
    4.291 ± 2.3344
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Vatiquinone v Placebo
    Number of subjects included in analysis
    123
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.0252
    Method
    		 Mixed models analysis
    Parameter type
    		 LS Mean Difference
    Point estimate
    -5.048
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -9.468
         upper limit
    		 -0.628
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.255

    Other pre-specified: Change From Baseline in the MFIS Score at Week 72 - ITT Analysis Set

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    End point title
    		 Change From Baseline in the MFIS Score at Week 72 - ITT Analysis Set
    End point description
    The MFIS is a 21-item, reliable, validated instrument that has been utilized in many neurological disorders. It is a modified form of the Fatigue Impact Scale, a component of the Multiple Sclerosis Quality of Life Inventory. Each item was scored on a scale of 0 (never) to 4 (almost always). The total score was the sum of all item’s score and ranged from 0 (no fatigue impact) to 84 (almost always impacted by fatigue). Higher scores indicated greater impact of fatigue on participant function. The ITT analysis set included all randomized participants who had received at least 1 dose of study drug (vatiquinone or placebo), and had baseline and at least 1 postbaseline measurement of the primary endpoint.
    End point type
    Other pre-specified
    End point timeframe
    Baseline, Week 72
    End point values
    		 Vatiquinone Placebo
    Number of subjects analysed
    70
    73
    Units: units on a scale
        least squares mean (standard error)
    -1.200 ± 1.9897
    4.354 ± 2.0758
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    Vatiquinone v Placebo
    Number of subjects included in analysis
    143
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.0095
    Method
    		 Mixed models analysis
    Parameter type
    		 LS Mean Difference
    Point estimate
    -5.554
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -9.75
         upper limit
    		 -1.358
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.1409

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to Week 100
    Adverse event reporting additional description
    Double-blind phase: The safety analysis set included all participants who received at least 1 dose of study drug. On-Vatiquinone period: On-Vatiquinone safety analysis set included all randomized participants who received at least 1 dose of Vatiquinone anytime during the study.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    Double-blind Phase: Vatiquinone
    Reporting group description
    		 Participants received vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the double-blind phase.

    Reporting group title
    On-Vatiquinone Period: Placebo/Vatiquinone
    Reporting group description
    		 Participants who received placebo for 72 weeks in the double-blind phase, received vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.

    Reporting group title
    On-Vatiquinone Period: Vatiquinone/Vatiquinone
    Reporting group description
    		 Participants who received vatiquinone for 72 weeks in the double-blind phase continued to receive vatiquinone capsule at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label phase.

    Reporting group title
    Double-blind Phase: Placebo
    Reporting group description
    		 Participants received placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the double-blind phase.

    Serious adverse events
    		 Double-blind Phase: Vatiquinone On-Vatiquinone Period: Placebo/Vatiquinone On-Vatiquinone Period: Vatiquinone/Vatiquinone Double-blind Phase: Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    8 / 73 (10.96%)
    2 / 65 (3.08%)
    10 / 73 (13.70%)
    8 / 73 (10.96%)
         number of deaths (all causes)
    1
    0
    1
    1
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Intentional product misuse
         subjects affected / exposed
    1 / 73 (1.37%)
    0 / 65 (0.00%)
    1 / 73 (1.37%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    0 / 73 (0.00%)
    0 / 65 (0.00%)
    0 / 73 (0.00%)
    1 / 73 (1.37%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Myocarditis
         subjects affected / exposed
    1 / 73 (1.37%)
    0 / 65 (0.00%)
    1 / 73 (1.37%)
    1 / 73 (1.37%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac failure
         subjects affected / exposed
    1 / 73 (1.37%)
    0 / 65 (0.00%)
    1 / 73 (1.37%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
    0 / 0
    Nervous system disorders
    Seizure
         subjects affected / exposed
    0 / 73 (0.00%)
    0 / 65 (0.00%)
    0 / 73 (0.00%)
    1 / 73 (1.37%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Presyncope
         subjects affected / exposed
    0 / 73 (0.00%)
    1 / 65 (1.54%)
    0 / 73 (0.00%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Non-cardiac chest pain
         subjects affected / exposed
    0 / 73 (0.00%)
    0 / 65 (0.00%)
    0 / 73 (0.00%)
    1 / 73 (1.37%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Immune system disorders
    Multisystem inflammatory syndrome in children
         subjects affected / exposed
    0 / 73 (0.00%)
    0 / 65 (0.00%)
    0 / 73 (0.00%)
    1 / 73 (1.37%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 73 (0.00%)
    0 / 65 (0.00%)
    1 / 73 (1.37%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Vomiting
         subjects affected / exposed
    0 / 73 (0.00%)
    1 / 65 (1.54%)
    0 / 73 (0.00%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nausea
         subjects affected / exposed
    0 / 73 (0.00%)
    1 / 65 (1.54%)
    0 / 73 (0.00%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Impaired gastric emptying
         subjects affected / exposed
    0 / 73 (0.00%)
    1 / 65 (1.54%)
    0 / 73 (0.00%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Suicide attempt
         subjects affected / exposed
    1 / 73 (1.37%)
    0 / 65 (0.00%)
    2 / 73 (2.74%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Depression
         subjects affected / exposed
    1 / 73 (1.37%)
    0 / 65 (0.00%)
    1 / 73 (1.37%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Completed suicide
         subjects affected / exposed
    0 / 73 (0.00%)
    0 / 65 (0.00%)
    0 / 73 (0.00%)
    1 / 73 (1.37%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    Anxiety
         subjects affected / exposed
    1 / 73 (1.37%)
    0 / 65 (0.00%)
    1 / 73 (1.37%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Myalgia
         subjects affected / exposed
    1 / 73 (1.37%)
    0 / 65 (0.00%)
    1 / 73 (1.37%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Gastroenteritis viral
         subjects affected / exposed
    1 / 73 (1.37%)
    0 / 65 (0.00%)
    1 / 73 (1.37%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    1 / 73 (1.37%)
    0 / 65 (0.00%)
    1 / 73 (1.37%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Appendicitis perforated
         subjects affected / exposed
    1 / 73 (1.37%)
    0 / 65 (0.00%)
    1 / 73 (1.37%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    0 / 73 (0.00%)
    0 / 65 (0.00%)
    1 / 73 (1.37%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    0 / 73 (0.00%)
    1 / 65 (1.54%)
    0 / 73 (0.00%)
    0 / 73 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 73 (0.00%)
    0 / 65 (0.00%)
    0 / 73 (0.00%)
    1 / 73 (1.37%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Type 2 diabetes mellitus
         subjects affected / exposed
    0 / 73 (0.00%)
    0 / 65 (0.00%)
    0 / 73 (0.00%)
    1 / 73 (1.37%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Double-blind Phase: Vatiquinone On-Vatiquinone Period: Placebo/Vatiquinone On-Vatiquinone Period: Vatiquinone/Vatiquinone Double-blind Phase: Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    71 / 73 (97.26%)
    53 / 65 (81.54%)
    71 / 73 (97.26%)
    73 / 73 (100.00%)
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    4 / 73 (5.48%)
    2 / 65 (3.08%)
    5 / 73 (6.85%)
    9 / 73 (12.33%)
         occurrences all number
    4
    2
    5
    14
    Fatigue
         subjects affected / exposed
    9 / 73 (12.33%)
    3 / 65 (4.62%)
    11 / 73 (15.07%)
    13 / 73 (17.81%)
         occurrences all number
    12
    3
    14
    21
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    4 / 73 (5.48%)
    0 / 65 (0.00%)
    4 / 73 (5.48%)
    3 / 73 (4.11%)
         occurrences all number
    9
    0
    9
    8
    Respiratory, thoracic and mediastinal disorders
    Epistaxis
         subjects affected / exposed
    6 / 73 (8.22%)
    0 / 65 (0.00%)
    8 / 73 (10.96%)
    3 / 73 (4.11%)
         occurrences all number
    8
    0
    10
    4
    Oropharyngeal pain
         subjects affected / exposed
    8 / 73 (10.96%)
    3 / 65 (4.62%)
    8 / 73 (10.96%)
    8 / 73 (10.96%)
         occurrences all number
    11
    4
    11
    11
    Cough
         subjects affected / exposed
    5 / 73 (6.85%)
    1 / 65 (1.54%)
    5 / 73 (6.85%)
    6 / 73 (8.22%)
         occurrences all number
    5
    1
    5
    6
    Rhinorrhoea
         subjects affected / exposed
    4 / 73 (5.48%)
    1 / 65 (1.54%)
    4 / 73 (5.48%)
    2 / 73 (2.74%)
         occurrences all number
    4
    1
    4
    2
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    5 / 73 (6.85%)
    1 / 65 (1.54%)
    6 / 73 (8.22%)
    3 / 73 (4.11%)
         occurrences all number
    5
    1
    6
    5
    Investigations
    Weight increased
         subjects affected / exposed
    0 / 73 (0.00%)
    1 / 65 (1.54%)
    1 / 73 (1.37%)
    6 / 73 (8.22%)
         occurrences all number
    0
    1
    1
    6
    International normalised ratio increased
         subjects affected / exposed
    3 / 73 (4.11%)
    0 / 65 (0.00%)
    4 / 73 (5.48%)
    0 / 73 (0.00%)
         occurrences all number
    3
    0
    4
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    2 / 73 (2.74%)
    1 / 65 (1.54%)
    2 / 73 (2.74%)
    6 / 73 (8.22%)
         occurrences all number
    2
    1
    2
    6
    Fall
         subjects affected / exposed
    58 / 73 (79.45%)
    34 / 65 (52.31%)
    59 / 73 (80.82%)
    61 / 73 (83.56%)
         occurrences all number
    363
    89
    468
    441
    Ligament sprain
         subjects affected / exposed
    4 / 73 (5.48%)
    0 / 65 (0.00%)
    4 / 73 (5.48%)
    5 / 73 (6.85%)
         occurrences all number
    4
    0
    4
    5
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    1 / 73 (1.37%)
    0 / 65 (0.00%)
    1 / 73 (1.37%)
    4 / 73 (5.48%)
         occurrences all number
    1
    0
    1
    6
    Nervous system disorders
    Balance disorder
         subjects affected / exposed
    2 / 73 (2.74%)
    0 / 65 (0.00%)
    2 / 73 (2.74%)
    4 / 73 (5.48%)
         occurrences all number
    2
    0
    2
    4
    Dizziness
         subjects affected / exposed
    11 / 73 (15.07%)
    7 / 65 (10.77%)
    12 / 73 (16.44%)
    8 / 73 (10.96%)
         occurrences all number
    15
    8
    18
    8
    Headache
         subjects affected / exposed
    22 / 73 (30.14%)
    7 / 65 (10.77%)
    25 / 73 (34.25%)
    26 / 73 (35.62%)
         occurrences all number
    37
    9
    43
    53
    Syncope
         subjects affected / exposed
    4 / 73 (5.48%)
    1 / 65 (1.54%)
    4 / 73 (5.48%)
    1 / 73 (1.37%)
         occurrences all number
    4
    1
    4
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    19 / 73 (26.03%)
    5 / 65 (7.69%)
    21 / 73 (28.77%)
    10 / 73 (13.70%)
         occurrences all number
    28
    5
    31
    12
    Dyspepsia
         subjects affected / exposed
    4 / 73 (5.48%)
    1 / 65 (1.54%)
    5 / 73 (6.85%)
    3 / 73 (4.11%)
         occurrences all number
    8
    1
    10
    3
    Nausea
         subjects affected / exposed
    13 / 73 (17.81%)
    5 / 65 (7.69%)
    15 / 73 (20.55%)
    10 / 73 (13.70%)
         occurrences all number
    16
    8
    19
    16
    Abdominal pain
         subjects affected / exposed
    10 / 73 (13.70%)
    1 / 65 (1.54%)
    10 / 73 (13.70%)
    6 / 73 (8.22%)
         occurrences all number
    15
    1
    15
    7
    Abdominal pain upper
         subjects affected / exposed
    7 / 73 (9.59%)
    8 / 65 (12.31%)
    8 / 73 (10.96%)
    10 / 73 (13.70%)
         occurrences all number
    9
    14
    11
    17
    Odynophagia
         subjects affected / exposed
    2 / 73 (2.74%)
    0 / 65 (0.00%)
    2 / 73 (2.74%)
    4 / 73 (5.48%)
         occurrences all number
    2
    0
    2
    5
    Vomiting
         subjects affected / exposed
    14 / 73 (19.18%)
    7 / 65 (10.77%)
    17 / 73 (23.29%)
    9 / 73 (12.33%)
         occurrences all number
    17
    10
    21
    13
    Skin and subcutaneous tissue disorders
    Eczema
         subjects affected / exposed
    3 / 73 (4.11%)
    2 / 65 (3.08%)
    4 / 73 (5.48%)
    3 / 73 (4.11%)
         occurrences all number
    4
    2
    6
    3
    Pruritus
         subjects affected / exposed
    3 / 73 (4.11%)
    0 / 65 (0.00%)
    5 / 73 (6.85%)
    1 / 73 (1.37%)
         occurrences all number
    3
    0
    5
    1
    Rash
         subjects affected / exposed
    4 / 73 (5.48%)
    0 / 65 (0.00%)
    5 / 73 (6.85%)
    5 / 73 (6.85%)
         occurrences all number
    4
    0
    7
    6
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    6 / 73 (8.22%)
    1 / 65 (1.54%)
    7 / 73 (9.59%)
    1 / 73 (1.37%)
         occurrences all number
    7
    1
    9
    1
    Back pain
         subjects affected / exposed
    7 / 73 (9.59%)
    1 / 65 (1.54%)
    9 / 73 (12.33%)
    6 / 73 (8.22%)
         occurrences all number
    8
    1
    14
    8
    Muscle spasms
         subjects affected / exposed
    3 / 73 (4.11%)
    2 / 65 (3.08%)
    4 / 73 (5.48%)
    6 / 73 (8.22%)
         occurrences all number
    3
    2
    4
    7
    Myalgia
         subjects affected / exposed
    3 / 73 (4.11%)
    0 / 65 (0.00%)
    3 / 73 (4.11%)
    4 / 73 (5.48%)
         occurrences all number
    3
    0
    3
    4
    Pain in extremity
         subjects affected / exposed
    9 / 73 (12.33%)
    1 / 65 (1.54%)
    9 / 73 (12.33%)
    7 / 73 (9.59%)
         occurrences all number
    11
    1
    11
    10
    Infections and infestations
    Gastroenteritis viral
         subjects affected / exposed
    5 / 73 (6.85%)
    1 / 65 (1.54%)
    5 / 73 (6.85%)
    4 / 73 (5.48%)
         occurrences all number
    5
    1
    5
    5
    Influenza
         subjects affected / exposed
    8 / 73 (10.96%)
    2 / 65 (3.08%)
    9 / 73 (12.33%)
    11 / 73 (15.07%)
         occurrences all number
    9
    2
    10
    13
    Nasopharyngitis
         subjects affected / exposed
    12 / 73 (16.44%)
    6 / 65 (9.23%)
    12 / 73 (16.44%)
    17 / 73 (23.29%)
         occurrences all number
    19
    9
    20
    34
    Gastroenteritis
         subjects affected / exposed
    3 / 73 (4.11%)
    1 / 65 (1.54%)
    4 / 73 (5.48%)
    4 / 73 (5.48%)
         occurrences all number
    3
    1
    4
    4
    COVID-19
         subjects affected / exposed
    32 / 73 (43.84%)
    4 / 65 (6.15%)
    36 / 73 (49.32%)
    24 / 73 (32.88%)
         occurrences all number
    36
    5
    41
    26
    Sinusitis
         subjects affected / exposed
    4 / 73 (5.48%)
    0 / 65 (0.00%)
    4 / 73 (5.48%)
    2 / 73 (2.74%)
         occurrences all number
    5
    0
    5
    2
    Viral upper respiratory tract infection
         subjects affected / exposed
    4 / 73 (5.48%)
    0 / 65 (0.00%)
    4 / 73 (5.48%)
    2 / 73 (2.74%)
         occurrences all number
    4
    0
    4
    2
    Viral rhinitis
         subjects affected / exposed
    8 / 73 (10.96%)
    1 / 65 (1.54%)
    11 / 73 (15.07%)
    3 / 73 (4.11%)
         occurrences all number
    18
    1
    24
    4
    Viral infection
         subjects affected / exposed
    3 / 73 (4.11%)
    2 / 65 (3.08%)
    4 / 73 (5.48%)
    1 / 73 (1.37%)
         occurrences all number
    3
    2
    4
    1
    Urinary tract infection
         subjects affected / exposed
    3 / 73 (4.11%)
    1 / 65 (1.54%)
    6 / 73 (8.22%)
    2 / 73 (2.74%)
         occurrences all number
    3
    1
    6
    4
    Upper respiratory tract infection
         subjects affected / exposed
    9 / 73 (12.33%)
    8 / 65 (12.31%)
    12 / 73 (16.44%)
    8 / 73 (10.96%)
         occurrences all number
    17
    9
    24
    10
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    5 / 73 (6.85%)
    1 / 65 (1.54%)
    5 / 73 (6.85%)
    3 / 73 (4.11%)
         occurrences all number
    6
    1
    6
    3

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    28 Aug 2020
    The overall reasons of this amendment was to revise the length of the placebo-controlled portion of the trial from 48 weeks to 72 weeks and the open-label portion from 48 weeks to 24 weeks.
    03 Mar 2021
    The overall reasons of this amendment was to clarify unblinding procedures, clarify disease worsening as a reason for discontinuation from study treatment, add a table of prohibited medications and add text clarifying the volume of blood that will be drawn during the study.
    15 Apr 2021
    The overall reasons of this amendment was to add risk/benefit language with regard to the pediatric population, revise the timing of the follow up visit from 10 to 30 days to approximately 30 days, and add details regarding maintaining the blind.
    21 May 2021
    The overall reason of this amendment was to clarify concomitant medication use and add exclusion of illicit drug use.

    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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