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    Clinical Trial Results:
    A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy

    Summary
    EudraCT number
    2018-000464-29
    Trial protocol
    DE   SE   IE  
    Global end of trial date
    22 Sep 2020

    Results information
    Results version number
    v1(current)
    This version publication date
    03 Feb 2022
    First version publication date
    03 Feb 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CAT-1004-301
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03703882
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Astria Therapeutics, Inc
    Sponsor organisation address
    100 High Street, 28th Floor, Boston, MA, United States, 02110
    Public contact
    Andrew Nichols, PhD - Chief Scientific Officer, Astria Therapeutics, Inc, 001 617-349-1971, anichols@astriatx.com
    Scientific contact
    Andrew Nichols, PhD - Chief Scientific Officer, Astria Therapeutics, Inc, 001 617-349-1971, anichols@astriatx.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    13 Oct 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    22 Sep 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    22 Sep 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the efficacy of edasalonexent as measured by change from Baseline (CFB) on North Star Ambulatory Assessment (NSAA) Total Score in pediatric patients with Duchenne muscular dystrophy (DMD)
    Protection of trial subjects
    This study was conducted in accordance with the protocol, Health Insurance Portability and Accountability Act (HIPAA) regulations, Food and Drug Administration (FDA) GCP Regulations (21 CFR Parts 50, 56, and 312) and ICH guidelines for GCP (E6) and clinical safety data management (E2A), and the ethical principles that had their origin in the Declaration of Helsinki. The study was conducted in accordance with applicable local law(s) and regulation(s).
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    02 Oct 2018
    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
    Sweden: 4
    Country: Number of subjects enrolled
    Germany: 8
    Country: Number of subjects enrolled
    Ireland: 4
    Country: Number of subjects enrolled
    United States: 84
    Country: Number of subjects enrolled
    Canada: 10
    Country: Number of subjects enrolled
    United Kingdom: 14
    Country: Number of subjects enrolled
    Israel: 1
    Country: Number of subjects enrolled
    Australia: 6
    Worldwide total number of subjects
    131
    EEA total number of subjects
    16
    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)
    131
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    This was a multi-center study conducted by 37 principal investigators at 37 study centers in 8 countries (United States, Canada, United Kingdom, Germany, Ireland, Israel, Sweden, and Australia).

    Pre-assignment
    Screening details
    A total of 151 patients were screened of which 20 failed screening. 131 patients who participated in the study included 126 randomized patients and 5 participants who were dosed siblings of previously randomized patients.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Monitor, Data analyst, Carer, Assessor
    Blinding implementation details
    Patients were randomized to receive edasalonexent 100 mg/kg/day (administered as approximately 33 mg/kg TID) or placebo in double-blind fashion daily for 52 weeks. Patients, investigators, study staff, and the Sponsor were blinded to study drug assignment. Prior to scheduled unblinding, if an emergency required the identity of the IP to be known by the Investigator in order to provide appropriate medical treatment, the Investigator was allowed to unblind using the Interactive Web Response System

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Dose 1
    Arm description
    Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.
    Arm type
    Experimental

    Investigational medicinal product name
    Edasalonexent
    Investigational medicinal product code
    Other name
    Edasa, CAT-1004
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day for 52 weeks.

    Arm title
    Placebo
    Arm description
    Matching placebo
    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
    Subjects received a placebo capsules taken by mouth three times per day for 52 weeks.

    Number of subjects in period 1
    Dose 1 Placebo
    Started
    88
    43
    Completed
    85
    37
    Not completed
    3
    6
         Consent withdrawn by subject
    1
    -
         Physician decision
    -
    1
         Adverse event, non-fatal
    1
    -
         Starting another treatment
    -
    1
         Progressive Disease
    1
    -
         Non-compliance with study drug
    -
    1
         Lost to follow-up
    -
    2
         Non-compliance with study procedure
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Dose 1
    Reporting group description
    Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.

    Reporting group title
    Placebo
    Reporting group description
    Matching placebo

    Reporting group values
    Dose 1 Placebo Total
    Number of subjects
    88 43 131
    Age categorical
    Safety Population: All patients who received at least 1 dose of study drug, with patients analyzed based on the actual study treatment received. This included the set of patients who were assigned the same treatment as their randomized sibling.
    Units: Subjects
        Children (2-11 years)
    88 43 131
    Age continuous
    Safety Population: All patients who received at least 1 dose of study drug, with patients analyzed based on the actual study treatment received. This included the set of patients who were assigned the same treatment as their randomized sibling.
    Units: years
        arithmetic mean (standard deviation)
    5.65 ( 1.048 ) 5.77 ( 0.995 ) -
    Gender categorical
    Safety Population: All patients who received at least 1 dose of study drug, with patients analyzed based on the actual study treatment received. This included the set of patients who were assigned the same treatment as their randomized sibling.
    Units: Subjects
        Female
    0 0 0
        Male
    88 43 131
    Ethnicity
    Safety Population: All patients who received at least 1 dose of study drug, with patients analyzed based on the actual study treatment received. This included the set of patients who were assigned the same treatment as their randomized sibling.
    Units: Subjects
        Hispanic or Latino
    14 6 20
        Not Hispanic or Latino
    69 35 104
        Unknown or Not Reported
    5 2 7
    Race
    Safety Population: All patients who received at least 1 dose of study drug, with patients analyzed based on the actual study treatment received. This included the set of patients who were assigned the same treatment as their randomized sibling. A patient who reported more than 1 race was categorized as multiracial.
    Units: Subjects
        American Indian or Alaska Native
    0 0 0
        Asian
    3 2 5
        Native Hawaiian or Other Pacific Islander
    1 0 1
        Black or African American
    4 1 5
        White
    74 38 112
        More than one race
    3 1 4
        Unknown or Not Reported
    2 1 3
        Missing-Race
    1 0 1

    End points

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    End points reporting groups
    Reporting group title
    Dose 1
    Reporting group description
    Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.

    Reporting group title
    Placebo
    Reporting group description
    Matching placebo

    Primary: Change From Baseline in North Star Ambulatory Assessment (NSAA)

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    End point title
    Change From Baseline in North Star Ambulatory Assessment (NSAA)
    End point description
    To assess change from baseline in NSAA Total Score at Week 52. NSAA is a clinician-reported outcome instrument designed to measure ambulatory function in males with DMD. Patients asked to perform 17 different functional activities, including a 10MWT, rising from sit to stand, standing on one leg, climbing and descending a step, stand from supine, lifting the head, standing on heels, and jumping. Each function activity will be scored as 0=(unable to achieve independently), scored as 1=(modified method but achieves goal independent of physical assistance from another), or scored as 2 =(no obvious modification of activity) or “Not Scored”. If the NSAA test was performed and any of the individual items are scored as “not scored” (i.e., for reasons unrelated to the patients physical capabilities), corresponding total score will be set to missing. sum of 17 scores will be used to form an ordinal total score (range 0 – 34). Full Analysis population
    End point type
    Primary
    End point timeframe
    Baseline (Day 1) to Week 52
    End point values
    Dose 1 Placebo
    Number of subjects analysed
    81
    38
    Units: score on a scale
        arithmetic mean (standard deviation)
    -1.5 ( 4.41 )
    -1.8 ( 3.81 )
    Statistical analysis title
    Change From Baseline in NSAA
    Statistical analysis description
    Full Analysis population
    Comparison groups
    Dose 1 v Placebo
    Number of subjects included in analysis
    119
    Analysis specification
    Pre-specified
    Analysis type
    superiority [1]
    P-value
    = 0.6705 [2]
    Method
    ANCOVA
    Confidence interval
    Notes
    [1] - The model includes change from baseline in the NSAA Total Score as the dependent variable, treatment, visit, and treatment-by-visit interaction as fixed effects, with baseline age, time to stand from supine, region (North America vs. Europe/Asia/Australia), and baseline NSAA score as covariates (including a by-visit interaction term for each covariate), and patient as a random effect.
    [2] - Least Squares means, p-values from mixed-model repeated-measures(MMRM) ANCOVA.

    Secondary: Change From Baseline in 10-meter Walk/Run Test

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    End point title
    Change From Baseline in 10-meter Walk/Run Test
    End point description
    To assess the changes from baseline to Week 52 on the 10-meter walk/run test (10MWT). For timed function tests (TFTs), the time will be set to 12 seconds and the speed to 0 if the TFT assessment meets the following TFT grading criteria. Grade of 1 or 2 (from a 6-point scale). 1=Unable to walk independently 2=Unable to walk independently but can walk with knee-ankle foot orthoses or support from a person 3=Highly adapted wide based lordotic gait. Cannot increase walking speed 4=Moderately adapted gait. Can pick up speed but cannot run 5=Able to pick up speed, but runs with a double stance phase, i.e. cannot achieve both feet off the ground 6=Runs and gets both feet off the ground (with no double stance phase) Full Analysis population: All patients in the Randomized Population who received at least 1 dose of study drug and provided at least 1 valid post Baseline NSAA efficacy assessment.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 52
    End point values
    Dose 1 Placebo
    Number of subjects analysed
    81
    38
    Units: score on a scale
        arithmetic mean (standard deviation)
    -0.0058 ( 0.03010 )
    -0.0093 ( 0.02538 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Time to Stand From Supine

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    End point title
    Change From Baseline in Time to Stand From Supine
    End point description
    To assess the change from baseline in the stand from supine speed at Week 52. For timed function tests (TFTs) , the time will be set to 12 seconds and the speed to 0 if the TFT assessment meets the following TFT grading criteria. Grade of 1 or 2 (from a 6-point scale). 1 = Unable to stand from supine, even with use of a chair, 2 = Assisted Gowers -requires furniture for assist in arising from supine to full upright posture (no time to be recorded) 3=Rolls over, stands up with both hands “climbing up” the legs to achieve full upright posture 4=Rolls over, stands up with 1 hand support on leg 5=Rolls to the side and stands up with one or both hands on the floor to start to rise but does not touch legs 6=Stands up without rolling over or using hands on legs or floor Full Analysis population: All patients in the Randomized Population who received at least 1 dose of study drug and provided at least 1 valid post Baseline NSAA efficacy assessment.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 52
    End point values
    Dose 1 Placebo
    Number of subjects analysed
    81
    38
    Units: score on a scale
    arithmetic mean (standard deviation)
        Change From Baseline in Time to Stand From Supine
    -0.0389 ( 0.06728 )
    0.0459 ( 0.06171 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in 4-stair Climb

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    End point title
    Change From Baseline in 4-stair Climb
    End point description
    To assess the change from baseline to Week 52 on the 4-Stair Climb. For timed function tests (TFTs) , the time will be set to 12 seconds and the speed to 0 if the TFT assessment meets the following TFT grading criteria. Grade of 1(from a 6-point scale)1=Unable to climb 4 standard stairs(no time recorded) 2=Climbs 4 standard stairs “marking time”(climbs one foot at a time, with both feet on a step before moving to next step), uses both arms on one or both handrails or uses 1 handrail and the other arm pushes on the leg 3=Climbs 4 standard stairs “marking time”, using one arm on one handrail or one hand pushing on leg or body 4=Climbs 4 standard stairs “marking time”, not needing handrail and not using hands to push on leg 5=Climbs 4 standard stairs alternating feet, needs handrail/s for support or uses arms to push on the leg or body 6=Climbs 4 standard stairs alternating feet, not needing handrail support or using arm to push on the leg Full Analysis population
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 52
    End point values
    Dose 1 Placebo
    Number of subjects analysed
    81
    38
    Units: score on a scale
        arithmetic mean (standard deviation)
    -0.0220 ( 0.08920 )
    -0.0392 ( 0.07352 )
    No statistical analyses for this end point

    Secondary: Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse

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    End point title
    Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse
    End point description
    Safety population: All patients who received at least 1 dose of study drug, with patients analyzed based on the actual study treatment received. This included the set of patients who were assigned the same treatment as their randomized sibling. Adverse events that occurred from the time of the administration of the first dose of investigational product (IP) through the end of the safety follow-up were considered treatment-emergent AEs (TEAEs). Treatment-Related TEAEs (TRT-Related TATEs)
    End point type
    Secondary
    End point timeframe
    Up to Week 52
    End point values
    Dose 1 Placebo
    Number of subjects analysed
    88
    43
    Units: participants
        Treatment Emergent Adverse Event (TEAEs)
    85
    41
        Related Treatment Emergent Adverse Event (TEAEs)
    61
    14
        Serious TEAE
    1
    1
        Serious Related TEAEs
    0
    0
        TEAEs Leading to Study Discontinuation
    1
    0
        TRT-Related TEAEs Leading to Study
    1
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to Week 52
    Adverse event reporting additional description
    A treatment-emergent Adverse Events (TEAE) is any Adverse Events that newly appeared, increased in frequency or worsened in severity following initiation of study drug administration. Subjects with more than one AE of the same system organ class (SOC) / preferred term (PT) were counted only once for that SOC / PT. Safety population
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    21
    Reporting groups
    Reporting group title
    Dose 1
    Reporting group description
    Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day.

    Reporting group title
    Placebo
    Reporting group description
    Matching placebo

    Serious adverse events
    Dose 1 Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 88 (1.14%)
    1 / 43 (2.33%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Infections and infestations
    Bronchiolitis
         subjects affected / exposed
    0 / 88 (0.00%)
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastroenteritis norovirus A
         subjects affected / exposed
    1 / 88 (1.14%)
    0 / 43 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 2%
    Non-serious adverse events
    Dose 1 Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    85 / 88 (96.59%)
    41 / 43 (95.35%)
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    6 / 88 (6.82%)
    0 / 43 (0.00%)
         occurrences all number
    6
    0
    Influenza like illness A
         subjects affected / exposed
    0 / 88 (0.00%)
    5 / 43 (11.63%)
         occurrences all number
    0
    6
    Non-cardiac chest pain
         subjects affected / exposed
    3 / 88 (3.41%)
    0 / 43 (0.00%)
         occurrences all number
    3
    0
    Pyrexia
         subjects affected / exposed
    17 / 88 (19.32%)
    9 / 43 (20.93%)
         occurrences all number
    18
    14
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    13 / 88 (14.77%)
    11 / 43 (25.58%)
         occurrences all number
    19
    15
    Rhinorrhoea
         subjects affected / exposed
    3 / 88 (3.41%)
    5 / 43 (11.63%)
         occurrences all number
    4
    8
    Epistaxis
         subjects affected / exposed
    6 / 88 (6.82%)
    1 / 43 (2.33%)
         occurrences all number
    10
    3
    Oropharyngeal pain
         subjects affected / exposed
    4 / 88 (4.55%)
    1 / 43 (2.33%)
         occurrences all number
    5
    1
    Sinus congestion
         subjects affected / exposed
    3 / 88 (3.41%)
    0 / 43 (0.00%)
         occurrences all number
    3
    0
    Psychiatric disorders
    Attention deficit hyperactivity disorder
         subjects affected / exposed
    2 / 88 (2.27%)
    1 / 43 (2.33%)
         occurrences all number
    2
    1
    Investigations
    Weight increased
         subjects affected / exposed
    1 / 88 (1.14%)
    2 / 43 (4.65%)
         occurrences all number
    1
    2
    Injury, poisoning and procedural complications
    Arthropod bite
         subjects affected / exposed
    2 / 88 (2.27%)
    1 / 43 (2.33%)
         occurrences all number
    2
    1
    Contusion
         subjects affected / exposed
    6 / 88 (6.82%)
    2 / 43 (4.65%)
         occurrences all number
    11
    2
    Fall
         subjects affected / exposed
    14 / 88 (15.91%)
    4 / 43 (9.30%)
         occurrences all number
    20
    5
    Ligament sprain
         subjects affected / exposed
    3 / 88 (3.41%)
    1 / 43 (2.33%)
         occurrences all number
    3
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    3 / 88 (3.41%)
    1 / 43 (2.33%)
         occurrences all number
    3
    1
    Headache
         subjects affected / exposed
    12 / 88 (13.64%)
    8 / 43 (18.60%)
         occurrences all number
    15
    10
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    3 / 88 (3.41%)
    0 / 43 (0.00%)
         occurrences all number
    3
    0
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    4 / 88 (4.55%)
    1 / 43 (2.33%)
         occurrences all number
    4
    1
    Abdominal pain
         subjects affected / exposed
    7 / 88 (7.95%)
    1 / 43 (2.33%)
         occurrences all number
    14
    1
    Abdominal pain upper
         subjects affected / exposed
    15 / 88 (17.05%)
    9 / 43 (20.93%)
         occurrences all number
    22
    15
    Constipation
         subjects affected / exposed
    6 / 88 (6.82%)
    3 / 43 (6.98%)
         occurrences all number
    7
    3
    Dental caries
         subjects affected / exposed
    2 / 88 (2.27%)
    1 / 43 (2.33%)
         occurrences all number
    2
    1
    Diarrhoea
         subjects affected / exposed
    54 / 88 (61.36%)
    12 / 43 (27.91%)
         occurrences all number
    94
    20
    Faeces soft
         subjects affected / exposed
    2 / 88 (2.27%)
    1 / 43 (2.33%)
         occurrences all number
    3
    1
    Nausea
         subjects affected / exposed
    7 / 88 (7.95%)
    5 / 43 (11.63%)
         occurrences all number
    8
    5
    Toothache
         subjects affected / exposed
    3 / 88 (3.41%)
    0 / 43 (0.00%)
         occurrences all number
    3
    0
    Vomiting
         subjects affected / exposed
    29 / 88 (32.95%)
    11 / 43 (25.58%)
         occurrences all number
    57
    18
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    20 / 88 (22.73%)
    2 / 43 (4.65%)
         occurrences all number
    25
    2
    Renal and urinary disorders
    Chromaturia
         subjects affected / exposed
    2 / 88 (2.27%)
    2 / 43 (4.65%)
         occurrences all number
    2
    4
    Pollakiuria
         subjects affected / exposed
    3 / 88 (3.41%)
    0 / 43 (0.00%)
         occurrences all number
    3
    0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 88 (1.14%)
    2 / 43 (4.65%)
         occurrences all number
    1
    2
    Muscle spasms
         subjects affected / exposed
    10 / 88 (11.36%)
    1 / 43 (2.33%)
         occurrences all number
    11
    1
    Pain in extremity
         subjects affected / exposed
    6 / 88 (6.82%)
    5 / 43 (11.63%)
         occurrences all number
    11
    7
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    18 / 88 (20.45%)
    5 / 43 (11.63%)
         occurrences all number
    26
    5
    Conjunctivitis
         subjects affected / exposed
    2 / 88 (2.27%)
    1 / 43 (2.33%)
         occurrences all number
    2
    1
    Ear infection
         subjects affected / exposed
    7 / 88 (7.95%)
    5 / 43 (11.63%)
         occurrences all number
    9
    5
    Gastroenteritis
         subjects affected / exposed
    3 / 88 (3.41%)
    2 / 43 (4.65%)
         occurrences all number
    3
    2
    Gastroenteritis viral
         subjects affected / exposed
    3 / 88 (3.41%)
    2 / 43 (4.65%)
         occurrences all number
    3
    3
    Impetigo
         subjects affected / exposed
    3 / 88 (3.41%)
    0 / 43 (0.00%)
         occurrences all number
    3
    0
    Influenza
         subjects affected / exposed
    10 / 88 (11.36%)
    2 / 43 (4.65%)
         occurrences all number
    10
    3
    Lower respiratory tract infection
         subjects affected / exposed
    2 / 88 (2.27%)
    2 / 43 (4.65%)
         occurrences all number
    2
    2
    Nasopharyngitis
         subjects affected / exposed
    19 / 88 (21.59%)
    9 / 43 (20.93%)
         occurrences all number
    28
    13
    Pharyngitis streptococcal
         subjects affected / exposed
    4 / 88 (4.55%)
    4 / 43 (9.30%)
         occurrences all number
    4
    4
    Tonsillitis
         subjects affected / exposed
    3 / 88 (3.41%)
    0 / 43 (0.00%)
         occurrences all number
    3
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    6 / 88 (6.82%)
    2 / 43 (4.65%)
         occurrences all number
    7
    2

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    16 Apr 2020
    The amendment included the following key changes in response to the COVID-19 pandemic: • As patients may not have been able to come on-site for visits or for a reduced amount of time, modifications were made to the original study procedures and schedule of assessments to reduce the patient’s risk or exposure to COVID-19 for Week 39 and Week 52 study visits. The modifications included: - Allowance for the Week 52 visit to be conducted out of the visit window (but in no case should the projected target date have been changed by more than 2 months). - Ability to perform study procedures either on site or remotely by using alternative approaches to collect study assessments. - Remote assessment for AEs and concomitant medications, as the top priority was safety. - Ability to defer other safety assessments, including physical examinations, chemistry and hematology labs and urinalysis for 6-month intervals if not feasible, based on the safety profile of edasalonexent to date and the consideration that all patients in this study had been on study drug for 6 months. - Allowance for drug dispensation to take place according to local site procedures, including secured, temperature-controlled shipments to the patients’ homes • A prioritized list of assessments has been included in Appendix of the protocol. • Clarification that post the COVID-19 pandemic, clinical monitoring may resume at amore intense pace to clean all data accumulated.

    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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