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    Clinical Trial Results:
    Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

    Summary
    EudraCT number
    		 2021-005314-34
    Trial protocol
    		 BE   DE   FR   ES   NL   IT   PL  
    Global end of trial date
    18 Dec 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    03 Jan 2026
    First version publication date
    03 Jan 2026
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    SRK-015-003
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05156320
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 IND: 136872
    Sponsors
    Sponsor organisation name
    Scholar Rock, Inc.
    Sponsor organisation address
    301 Binney Street, 3rd Floor, Cambridge, MA, United States, 02142
    Public contact
    Clinical Information Desk, Scholar Rock, Inc., +1 857259 3860 , clinicaltrials@scholarrock.com
    Scientific contact
    Clinical Information Desk, Scholar Rock, Inc., +1 857259 3860 , clinicaltrials@scholarrock.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-002951-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
    18 Dec 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Dec 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study was to assess the efficacy of apitegromab (SRK-015) compared with placebo using the Hammersmith Functional Motor Scale Expanded (HFMSE) in subjects 2 through 12 years old.
    Protection of trial subjects
    This study was designed and monitored in accordance with Sponsor procedures, which comply with the ethical principles of the International Council for Harmonisation (ICH) on GCP guidance and in accordance with the Declaration of Helsinki (Version 2013). The study was also conducted in accordance with national and local legal requirements and in accordance with United States (US) Investigational New Drug regulations (21 Code of Federal Regulations [CFR] 56) and the European Union (EU)’s Commission Directive 2005/28/EC of 8 April 2005.
    Background therapy
    		 Nusinersen or Risdiplam.
    Evidence for comparator
    		 -
    Actual start date of recruitment
    12 Feb 2022
    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
    United Kingdom: 8
    Country: Number of subjects enrolled
    Netherlands: 6
    Country: Number of subjects enrolled
    Poland: 16
    Country: Number of subjects enrolled
    Spain: 15
    Country: Number of subjects enrolled
    Belgium: 9
    Country: Number of subjects enrolled
    France: 16
    Country: Number of subjects enrolled
    Germany: 16
    Country: Number of subjects enrolled
    Italy: 31
    Country: Number of subjects enrolled
    United States: 71
    Worldwide total number of subjects
    188
    EEA total number of subjects
    109
    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)
    148
    Adolescents (12-17 years)
    33
    Adults (18-64 years)
    7
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Subjects took part in this multicenter study in Belgium, France, Germany, Italy, Netherland, Spain, Poland, United Kingdom and the United States from 28 March 2022 to 18 December 2024. Subjects with Type 2 and Type 3 spinal muscular atrophy (SMA) were enrolled into either the Main Efficacy Population (MEP) or the Exploratory Subpopulation (EXP).

    Pre-assignment
    Screening details
    		 Study included a Screening Period, a Treatment Period, and a Follow-Up Period. A total of 216 subjects were screened of which 188 subjects (156 subjects in the MEP, and 32 subjects in the EXP) were randomized.

    Period 1
    Period 1 title
    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
    The sponsor, subjects, parents or caregivers, Investigators, and site personnel, with the exception of the designated unblinded personnel (eg, site Pharmacist), were blinded to treatment assignments.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Main Efficacy Population: Placebo
    Arm description
    		 Subjects aged 2-12 years received apitegromab matching placebo, IV infusion, Q4W, over a period of 1-2 hours during the 52-week Treatment Period.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection/infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Apitegromab matching placebo, IV infusion, Q4W, over a period of 1-2 hours.

    Arm title
    		 Main Efficacy Population: Apitegromab 10 mg/kg
    Arm description
    		 Subjects aged 2-12 years received apitegromab 10 mg/kg, IV infusion, Q4W over a period of 1-2 hours during the 52-week Treatment Period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Apitegromab
    Investigational medicinal product code
    Other name
    SRK-015
    Pharmaceutical forms
    Injection/infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Apitegromab 10 mg/kg, IV infusion, Q4W, over a period of 1-2 hours.

    Arm title
    		 Main Efficacy Population: Apitegromab 20 mg/kg
    Arm description
    		 Subjects aged 2-12 years received apitegromab 20 mg/kg, IV infusion, Q4W over a period of 1-2 hours during the 52-week Treatment Period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Apitegromab
    Investigational medicinal product code
    Other name
    SRK-015
    Pharmaceutical forms
    Injection/infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Apitegromab 20 mg/kg, IV infusion, Q4W, over a period of 1-2 hours.

    Arm title
    		 Exploratory Subpopulation: Placebo
    Arm description
    		 Subjects aged 13-21 years received apitegromab matching placebo, IV infusion, Q4W over a period of 1-2 hours during the 52-week Treatment Period.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection/infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Apitegromab matching placebo, IV infusion, Q4W, over a period of 1-2 hours.

    Arm title
    		 Exploratory Subpopulation: Apitegromab 20 mg/kg
    Arm description
    		 Subjects aged 13-21 years received apitegromab 20 mg/kg, IV infusion, Q4W over a period of 1-2 hours during the 52-week Treatment Period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Apitegromab
    Investigational medicinal product code
    Other name
    SRK-015
    Pharmaceutical forms
    Injection/infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Apitegromab 20 mg/kg, IV infusion, Q4W, over a period of 1-2 hours.

    Number of subjects in period 1
    		Main Efficacy Population: Placebo Main Efficacy Population: Apitegromab 10 mg/kg Main Efficacy Population: Apitegromab 20 mg/kg Exploratory Subpopulation: Placebo Exploratory Subpopulation: Apitegromab 20 mg/kg
    Started
    50
    53
    53
    10
    22
    Completed
    50
    53
    52
    10
    21
    Not completed
    0
    0
    1
    0
    1
         Consent withdrawn by subject
    -
    -
    -
    -
    1
         Other
    -
    -
    1
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Main Efficacy Population: Placebo
    Reporting group description
    		 Subjects aged 2-12 years received apitegromab matching placebo, IV infusion, Q4W, over a period of 1-2 hours during the 52-week Treatment Period.

    Reporting group title
    Main Efficacy Population: Apitegromab 10 mg/kg
    Reporting group description
    		 Subjects aged 2-12 years received apitegromab 10 mg/kg, IV infusion, Q4W over a period of 1-2 hours during the 52-week Treatment Period.

    Reporting group title
    Main Efficacy Population: Apitegromab 20 mg/kg
    Reporting group description
    		 Subjects aged 2-12 years received apitegromab 20 mg/kg, IV infusion, Q4W over a period of 1-2 hours during the 52-week Treatment Period.

    Reporting group title
    Exploratory Subpopulation: Placebo
    Reporting group description
    		 Subjects aged 13-21 years received apitegromab matching placebo, IV infusion, Q4W over a period of 1-2 hours during the 52-week Treatment Period.

    Reporting group title
    Exploratory Subpopulation: Apitegromab 20 mg/kg
    Reporting group description
    		 Subjects aged 13-21 years received apitegromab 20 mg/kg, IV infusion, Q4W over a period of 1-2 hours during the 52-week Treatment Period.

    Reporting group values
    		 Main Efficacy Population: Placebo Main Efficacy Population: Apitegromab 10 mg/kg Main Efficacy Population: Apitegromab 20 mg/kg Exploratory Subpopulation: Placebo Exploratory Subpopulation: Apitegromab 20 mg/kg Total
    Number of subjects
    		 50 53 53 10 22 188
    Age categorical
    Units: Subjects
        2 years to 18 years
    		 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 8.1 ( 2.46 ) 7.4 ( 2.57 ) 7.9 ( 2.46 ) 15.2 ( 1.75 ) 16.1 ( 2.59 ) -
    Gender categorical
    Units: Subjects
        Female
    		 25 23 26 5 15 94
        Male
    		 25 30 27 5 7 94
    Race
    Units: Subjects
        Asian
    		 2 2 4 0 0 8
        Black or African American
    		 1 1 0 0 2 4
        White
    		 41 35 40 6 14 136
        Other
    		 1 3 0 0 0 4
        Not Reported or Unknown
    		 5 12 9 4 6 36

    End points

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    End points reporting groups
    Reporting group title
    Main Efficacy Population: Placebo
    Reporting group description
    		 Subjects aged 2-12 years received apitegromab matching placebo, IV infusion, Q4W, over a period of 1-2 hours during the 52-week Treatment Period.

    Reporting group title
    Main Efficacy Population: Apitegromab 10 mg/kg
    Reporting group description
    		 Subjects aged 2-12 years received apitegromab 10 mg/kg, IV infusion, Q4W over a period of 1-2 hours during the 52-week Treatment Period.

    Reporting group title
    Main Efficacy Population: Apitegromab 20 mg/kg
    Reporting group description
    		 Subjects aged 2-12 years received apitegromab 20 mg/kg, IV infusion, Q4W over a period of 1-2 hours during the 52-week Treatment Period.

    Reporting group title
    Exploratory Subpopulation: Placebo
    Reporting group description
    		 Subjects aged 13-21 years received apitegromab matching placebo, IV infusion, Q4W over a period of 1-2 hours during the 52-week Treatment Period.

    Reporting group title
    Exploratory Subpopulation: Apitegromab 20 mg/kg
    Reporting group description
    		 Subjects aged 13-21 years received apitegromab 20 mg/kg, IV infusion, Q4W over a period of 1-2 hours during the 52-week Treatment Period.

    Subject analysis set title
    Main Efficacy Population: Apitegromab 10 mg/kg + 20 mg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All subjects aged 2-12 years who received apitegromab 10 mg/kg or 20 mg/kg, IV infusion, Q4W, over a period of 1-2 hours during the 52-week Treatment Period.

    Primary: Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score at Week 52

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    End point title
    		 Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score at Week 52 [1]
    End point description
    The HFMSE assesses the physical abilities of patients with Type 2 and Type 3 SMA. It comprises of 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. Modified Intention-to-Treat (MITT) Set, defined as all subjects who received at least 1 dose of study drug and had at least 1 postbaseline evaluable HFMSE assessment.
    End point type
    Primary
    End point timeframe
    Baseline, Week 52
    Notes
    [1] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Applicable treatment arms were assessed for analysis.
    End point values
    		 Main Efficacy Population: Placebo Main Efficacy Population: Apitegromab 10 mg/kg Main Efficacy Population: Apitegromab 20 mg/kg Main Efficacy Population: Apitegromab 10 mg/kg + 20 mg/kg
    Number of subjects analysed
    50
    53
    53
    106
    Units: score on a scale
        least squares mean (standard error)
    -1.2 ( 0.66 )
    1.0 ( 0.64 )
    0.2 ( 0.64 )
    0.6 ( 0.48 )
    Statistical analysis title
    		 Statistical Analysis 1
    Statistical analysis description
    MMRM includes the fixed effects of treatment group, visit, treatment group-by-visit interaction, baseline HFMSE total score, baseline HFMSE total score-by-visit interaction, type of SMN Therapy (i.e., nusinersen or risdiplam), and age at initiation of SMN Therapy (>=5 and <5 years). Comparison was presented by the difference between apitegromab group versus placebo.
    Comparison groups
    Main Efficacy Population: Placebo v Main Efficacy Population: Apitegromab 10 mg/kg + 20 mg/kg
    Number of subjects included in analysis
    156
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0192
    Method
    		 Mixed models analysis
    Parameter type
    		 LS Mean Difference
    Point estimate
    1.8
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.3
         upper limit
    		 3.32
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.76
    Statistical analysis title
    		 Statistical Analysis 2
    Statistical analysis description
    MMRM includes the fixed effects of treatment group, visit, treatment group-by-visit interaction, baseline HFMSE total score, baseline HFMSE total score-by-visit interaction, type of SMN Therapy (i.e., nusinersen or risdiplam), and age at initiation of SMN Therapy (>=5 and <5 years).
    Comparison groups
    Main Efficacy Population: Placebo v Main Efficacy Population: Apitegromab 20 mg/kg
    Number of subjects included in analysis
    103
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1149
    Method
    		 Mixed models analysis
    Parameter type
    		 LS Mean Difference
    Point estimate
    1.4
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.34
         upper limit
    		 3.13
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.88

    Secondary: Change From Baseline in Revised Upper Limb Module (RULM) Total Score at Week 52

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    End point title
    		 Change From Baseline in Revised Upper Limb Module (RULM) Total Score at Week 52 [2]
    End point description
    The RULM is an assessment of upper limb function in nonambulatory patients with SMA that was performed for patients who were 30 months of age or older at baseline. The 19 scored items assess functions that relate to everyday life, such as placing hands from lap, pressing a button, and picking up a token. With the exception of 1 activity with a binary score, these items are scored 0, 1, or 2, where 0 denotes unable, 1 denotes able with modification, and 2 denotes able with no modification. The maximum score achievable is 37. MITT Set, defined as all subjects who received at least 1 dose of study drug and had at least 1 postbaseline evaluable HFMSE assessment.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    Notes
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Applicable treatment arms were assessed for analysis.
    End point values
    		 Main Efficacy Population: Placebo Main Efficacy Population: Apitegromab 10 mg/kg Main Efficacy Population: Apitegromab 20 mg/kg
    Number of subjects analysed
    50
    53
    53
    Units: score on a scale
        least squares mean (standard error)
    0.1 ( 0.40 )
    0.7 ( 0.39 )
    0.8 ( 0.38 )
    No statistical analyses for this end point

    Secondary: Percentage of Subjects With >=3-Point Change From Baseline in the HFMSE Total Score at Week 52

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    End point title
    		 Percentage of Subjects With >=3-Point Change From Baseline in the HFMSE Total Score at Week 52 [3]
    End point description
    Percentage of subjects treated with apitegromab who achieved a specific number of points of HFMSE improvement from baseline were reported. The HFMSE assesses the physical abilities of patients with Type 2 and Type 3 SMA. It comprises of 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. MITT Set, defined as all subjects who received at least 1 dose of study drug and had at least 1 postbaseline evaluable HFMSE assessment.
    End point type
    Secondary
    End point timeframe
    Week 52
    Notes
    [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Applicable treatment arms were assessed for analysis.
    End point values
    		 Main Efficacy Population: Placebo Main Efficacy Population: Apitegromab 10 mg/kg Main Efficacy Population: Apitegromab 20 mg/kg
    Number of subjects analysed
    50
    53
    53
    Units: percentage of subjects
        number (not applicable)
    13.5
    34.2
    25.0
    No statistical analyses for this end point

    Secondary: Change From Baseline in Number of World Health Organization (WHO) Motor Development Milestones Attained at Week 52

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    End point title
    		 Change From Baseline in Number of World Health Organization (WHO) Motor Development Milestones Attained at Week 52 [4]
    End point description
    The WHO Multicenter Growth Reference Study performance criteria was utilized to assess the WHO motor development milestones of subjects with Type 2 and nonambulatory Type 3 SMA enrolled in Cohort 2 and Cohort 3 relative to baseline. The WHO motor development milestones are a set of 6 distinct gross motor milestones that are considered to be universal and fundamental to acquiring the ability to walk independently. They include 1) sitting without support, 2) hands and knees crawling, 3) standing with assistance, 4) walking with assistance, 5) standing alone, and 6) walking without assistance. Each item is recorded as 1 (unable), 2 (refusal), 3 (Yes) or 9 (did not test). The number of 3s was counted as the final score. The minimum was 0, which means no motor milestones were achieved; the maximum was 6, which means all 6 milestones were achieved. MITT Set, defined as all subjects who received at least 1 dose of study drug and had at least 1 postbaseline evaluable HFMSE assessment.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    Notes
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Applicable treatment arms were assessed for analysis.
    End point values
    		 Main Efficacy Population: Placebo Main Efficacy Population: Apitegromab 10 mg/kg Main Efficacy Population: Apitegromab 20 mg/kg
    Number of subjects analysed
    50
    53
    53
    Units: motor milestone
        least squares mean (standard error)
    0.0 ( 0.10 )
    0.1 ( 0.09 )
    0.1 ( 0.09 )
    No statistical analyses for this end point

    Secondary: Incidence of Treatment Emergent Adverse Events (TEAEs) and Severe Adverse Events (SAEs)

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    End point title
    		 Incidence of Treatment Emergent Adverse Events (TEAEs) and Severe Adverse Events (SAEs)
    End point description
    A Treatment Emergent Adverse Events (TEAE) is defined as an adverse event that started or worsened in severity after the start of the first dose of study drug. The Safety Set is defined as all randomized subjects who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    From start of study up to end of study (Day 505)
    End point values
    		 Main Efficacy Population: Placebo Main Efficacy Population: Apitegromab 10 mg/kg Main Efficacy Population: Apitegromab 20 mg/kg Exploratory Subpopulation: Placebo Exploratory Subpopulation: Apitegromab 20 mg/kg
    Number of subjects analysed
    50
    53
    53
    10
    22
    Units: subjects
        TEAEs
    43
    51
    46
    9
    19
        SAEs
    5
    9
    12
    1
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From start of study up to end of study (Day 505)
    Adverse event reporting additional description
    The Safety Set included all randomized subjects who received at least 1 dose of study drug.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.1
    Reporting groups
    Reporting group title
    Pooled Population (Apitegromab 10 mg/kg + 20 mg/kg Combined)
    Reporting group description
    		 Type 2 SMA and Nonambulatory Type 3 SMA, ages 2 through 21 years old at Screening. Participants were randomized to receive apitegromab 10 mg/kg or 20 mg/kg for up to 52 weeks.

    Reporting group title
    Pooled Population (Apitegromab 10 mg/kg)
    Reporting group description
    		 Type 2 SMA and Nonambulatory Type 3 SMA, ages 2 through 21 years old at Screening. Participants were randomized to receive apitegromab 10 mg/kg for up to 52 weeks.

    Reporting group title
    Pooled Population (Apitegromab 20 mg/kg)
    Reporting group description
    		 Type 2 SMA and Nonambulatory Type 3 SMA, ages 2 through 21 years old at Screening. Participants were randomized to receive apitegromab 20 mg/kg for up to 52 weeks.

    Reporting group title
    Comparator: Comparator: Pooled Population (Placebo)
    Reporting group description
    		 Type 2 SMA and Nonambulatory Type 3 SMA, ages 2 through 21 years old at Screening. Participants were randomized to receive Placebo for up to 52 weeks.

    Serious adverse events
    		 Pooled Population (Apitegromab 10 mg/kg + 20 mg/kg Combined) Pooled Population (Apitegromab 10 mg/kg) Pooled Population (Apitegromab 20 mg/kg) Comparator: Comparator: Pooled Population (Placebo)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    21 / 128 (16.41%)
    9 / 53 (16.98%)
    12 / 75 (16.00%)
    6 / 60 (10.00%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 128 (0.78%)
    0 / 53 (0.00%)
    1 / 75 (1.33%)
    0 / 60 (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
    Blood and lymphatic system disorders
    Immune thrombocytopenia
         subjects affected / exposed
    1 / 128 (0.78%)
    1 / 53 (1.89%)
    0 / 75 (0.00%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    2 / 128 (1.56%)
    1 / 53 (1.89%)
    1 / 75 (1.33%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Vomiting
         subjects affected / exposed
    1 / 128 (0.78%)
    0 / 53 (0.00%)
    1 / 75 (1.33%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 0
    0 / 4
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure
         subjects affected / exposed
    2 / 128 (1.56%)
    2 / 53 (3.77%)
    0 / 75 (0.00%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Atelectasis
         subjects affected / exposed
    1 / 128 (0.78%)
    1 / 53 (1.89%)
    0 / 75 (0.00%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Dyspnoea
         subjects affected / exposed
    1 / 128 (0.78%)
    0 / 53 (0.00%)
    1 / 75 (1.33%)
    0 / 60 (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
    Epistaxis
         subjects affected / exposed
    1 / 128 (0.78%)
    0 / 53 (0.00%)
    1 / 75 (1.33%)
    0 / 60 (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
    Skin and subcutaneous tissue disorders
    Urticaria
         subjects affected / exposed
    0 / 128 (0.00%)
    0 / 53 (0.00%)
    0 / 75 (0.00%)
    1 / 60 (1.67%)
         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
    Musculoskeletal and connective tissue disorders
    Scoliosis
         subjects affected / exposed
    2 / 128 (1.56%)
    1 / 53 (1.89%)
    1 / 75 (1.33%)
    2 / 60 (3.33%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
    0 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Muscle contracture
         subjects affected / exposed
    1 / 128 (0.78%)
    0 / 53 (0.00%)
    1 / 75 (1.33%)
    0 / 60 (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
    Pelvic misalignment
         subjects affected / exposed
    0 / 128 (0.00%)
    0 / 53 (0.00%)
    0 / 75 (0.00%)
    1 / 60 (1.67%)
         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
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    7 / 128 (5.47%)
    3 / 53 (5.66%)
    4 / 75 (5.33%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 8
    0 / 3
    0 / 5
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Adenovirus infection
         subjects affected / exposed
    2 / 128 (1.56%)
    0 / 53 (0.00%)
    2 / 75 (2.67%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    2 / 128 (1.56%)
    1 / 53 (1.89%)
    1 / 75 (1.33%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Appendicitis
         subjects affected / exposed
    1 / 128 (0.78%)
    1 / 53 (1.89%)
    0 / 75 (0.00%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Enterovirus infection
         subjects affected / exposed
    1 / 128 (0.78%)
    0 / 53 (0.00%)
    1 / 75 (1.33%)
    0 / 60 (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
    Gastrointestinal infection
         subjects affected / exposed
    1 / 128 (0.78%)
    1 / 53 (1.89%)
    0 / 75 (0.00%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metapneumovirus infection
         subjects affected / exposed
    1 / 128 (0.78%)
    1 / 53 (1.89%)
    0 / 75 (0.00%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonia viral
         subjects affected / exposed
    1 / 128 (0.78%)
    1 / 53 (1.89%)
    0 / 75 (0.00%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory syncytial virus infection
         subjects affected / exposed
    1 / 128 (0.78%)
    1 / 53 (1.89%)
    0 / 75 (0.00%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Rhinovirus infection
         subjects affected / exposed
    1 / 128 (0.78%)
    0 / 53 (0.00%)
    1 / 75 (1.33%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 2
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Viral infection
         subjects affected / exposed
    1 / 128 (0.78%)
    1 / 53 (1.89%)
    0 / 75 (0.00%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory syncytial virus bronchiolitis
         subjects affected / exposed
    0 / 128 (0.00%)
    0 / 53 (0.00%)
    0 / 75 (0.00%)
    1 / 60 (1.67%)
         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
    Respiratory tract infection viral
         subjects affected / exposed
    0 / 128 (0.00%)
    0 / 53 (0.00%)
    0 / 75 (0.00%)
    1 / 60 (1.67%)
         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
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    3 / 128 (2.34%)
    2 / 53 (3.77%)
    1 / 75 (1.33%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 2
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metabolic acidosis
         subjects affected / exposed
    1 / 128 (0.78%)
    1 / 53 (1.89%)
    0 / 75 (0.00%)
    0 / 60 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
         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
    		 Pooled Population (Apitegromab 10 mg/kg + 20 mg/kg Combined) Pooled Population (Apitegromab 10 mg/kg) Pooled Population (Apitegromab 20 mg/kg) Comparator: Comparator: Pooled Population (Placebo)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    115 / 128 (89.84%)
    51 / 53 (96.23%)
    64 / 75 (85.33%)
    51 / 60 (85.00%)
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    8 / 128 (6.25%)
    6 / 53 (11.32%)
    2 / 75 (2.67%)
    4 / 60 (6.67%)
         occurrences all number
    10
    8
    2
    4
    Fall
         subjects affected / exposed
    8 / 128 (6.25%)
    6 / 53 (11.32%)
    2 / 75 (2.67%)
    4 / 60 (6.67%)
         occurrences all number
    10
    8
    2
    5
    Vascular disorders
    Haematoma
         subjects affected / exposed
    4 / 128 (3.13%)
    3 / 53 (5.66%)
    1 / 75 (1.33%)
    0 / 60 (0.00%)
         occurrences all number
    5
    4
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    27 / 128 (21.09%)
    12 / 53 (22.64%)
    15 / 75 (20.00%)
    12 / 60 (20.00%)
         occurrences all number
    45
    16
    29
    35
    Dizziness
         subjects affected / exposed
    4 / 128 (3.13%)
    3 / 53 (5.66%)
    1 / 75 (1.33%)
    3 / 60 (5.00%)
         occurrences all number
    6
    3
    3
    4
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    33 / 128 (25.78%)
    18 / 53 (33.96%)
    15 / 75 (20.00%)
    17 / 60 (28.33%)
         occurrences all number
    60
    37
    23
    18
    Fatigue
         subjects affected / exposed
    8 / 128 (6.25%)
    3 / 53 (5.66%)
    5 / 75 (6.67%)
    2 / 60 (3.33%)
         occurrences all number
    16
    7
    9
    2
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    4 / 128 (3.13%)
    4 / 53 (7.55%)
    0 / 75 (0.00%)
    5 / 60 (8.33%)
         occurrences all number
    4
    4
    0
    6
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    29 / 128 (22.66%)
    16 / 53 (30.19%)
    13 / 75 (17.33%)
    10 / 60 (16.67%)
         occurrences all number
    42
    23
    19
    14
    Diarrhoea
         subjects affected / exposed
    13 / 128 (10.16%)
    9 / 53 (16.98%)
    4 / 75 (5.33%)
    1 / 60 (1.67%)
         occurrences all number
    19
    15
    4
    1
    Abdominal pain
         subjects affected / exposed
    10 / 128 (7.81%)
    5 / 53 (9.43%)
    5 / 75 (6.67%)
    2 / 60 (3.33%)
         occurrences all number
    14
    5
    9
    3
    Nausea
         subjects affected / exposed
    7 / 128 (5.47%)
    4 / 53 (7.55%)
    3 / 75 (4.00%)
    7 / 60 (11.67%)
         occurrences all number
    11
    7
    4
    8
    Abdominal pain upper
         subjects affected / exposed
    6 / 128 (4.69%)
    3 / 53 (5.66%)
    3 / 75 (4.00%)
    3 / 60 (5.00%)
         occurrences all number
    7
    4
    3
    4
    Constipation
         subjects affected / exposed
    5 / 128 (3.91%)
    1 / 53 (1.89%)
    4 / 75 (5.33%)
    1 / 60 (1.67%)
         occurrences all number
    5
    1
    4
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    31 / 128 (24.22%)
    16 / 53 (30.19%)
    15 / 75 (20.00%)
    12 / 60 (20.00%)
         occurrences all number
    42
    25
    17
    14
    Rhinorrhoea
         subjects affected / exposed
    12 / 128 (9.38%)
    6 / 53 (11.32%)
    6 / 75 (8.00%)
    2 / 60 (3.33%)
         occurrences all number
    14
    8
    6
    3
    Oropharyngeal pain
         subjects affected / exposed
    10 / 128 (7.81%)
    6 / 53 (11.32%)
    4 / 75 (5.33%)
    7 / 60 (11.67%)
         occurrences all number
    11
    6
    5
    8
    Nasal congestion
         subjects affected / exposed
    7 / 128 (5.47%)
    2 / 53 (3.77%)
    5 / 75 (6.67%)
    2 / 60 (3.33%)
         occurrences all number
    9
    3
    6
    2
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    15 / 128 (11.72%)
    10 / 53 (18.87%)
    5 / 75 (6.67%)
    5 / 60 (8.33%)
         occurrences all number
    26
    14
    12
    8
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    11 / 128 (8.59%)
    7 / 53 (13.21%)
    4 / 75 (5.33%)
    3 / 60 (5.00%)
         occurrences all number
    13
    9
    4
    3
    Myalgia
         subjects affected / exposed
    8 / 128 (6.25%)
    3 / 53 (5.66%)
    5 / 75 (6.67%)
    1 / 60 (1.67%)
         occurrences all number
    11
    3
    8
    1
    Pain in extremity
         subjects affected / exposed
    7 / 128 (5.47%)
    5 / 53 (9.43%)
    2 / 75 (2.67%)
    2 / 60 (3.33%)
         occurrences all number
    8
    6
    2
    2
    Scoliosis
         subjects affected / exposed
    6 / 128 (4.69%)
    4 / 53 (7.55%)
    2 / 75 (2.67%)
    4 / 60 (6.67%)
         occurrences all number
    6
    4
    2
    4
    Muscle contracture
         subjects affected / exposed
    5 / 128 (3.91%)
    1 / 53 (1.89%)
    4 / 75 (5.33%)
    1 / 60 (1.67%)
         occurrences all number
    6
    1
    5
    1
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    32 / 128 (25.00%)
    15 / 53 (28.30%)
    17 / 75 (22.67%)
    14 / 60 (23.33%)
         occurrences all number
    50
    21
    29
    19
    Upper respiratory tract infection
         subjects affected / exposed
    28 / 128 (21.88%)
    12 / 53 (22.64%)
    16 / 75 (21.33%)
    18 / 60 (30.00%)
         occurrences all number
    40
    16
    24
    33
    Rhinitis
         subjects affected / exposed
    16 / 128 (12.50%)
    8 / 53 (15.09%)
    8 / 75 (10.67%)
    6 / 60 (10.00%)
         occurrences all number
    17
    8
    9
    13
    Ear infection
         subjects affected / exposed
    13 / 128 (10.16%)
    5 / 53 (9.43%)
    8 / 75 (10.67%)
    4 / 60 (6.67%)
         occurrences all number
    18
    8
    10
    4
    Gastroenteritis
         subjects affected / exposed
    12 / 128 (9.38%)
    7 / 53 (13.21%)
    5 / 75 (6.67%)
    3 / 60 (5.00%)
         occurrences all number
    16
    8
    8
    4
    COVID-19
         subjects affected / exposed
    9 / 128 (7.03%)
    5 / 53 (9.43%)
    4 / 75 (5.33%)
    4 / 60 (6.67%)
         occurrences all number
    9
    5
    4
    5
    Pharyngitis streptococcal
         subjects affected / exposed
    6 / 128 (4.69%)
    4 / 53 (7.55%)
    2 / 75 (2.67%)
    3 / 60 (5.00%)
         occurrences all number
    8
    6
    2
    3
    Influenza
         subjects affected / exposed
    5 / 128 (3.91%)
    3 / 53 (5.66%)
    2 / 75 (2.67%)
    4 / 60 (6.67%)
         occurrences all number
    6
    4
    2
    4
    Urinary tract infection
         subjects affected / exposed
    4 / 128 (3.13%)
    0 / 53 (0.00%)
    4 / 75 (5.33%)
    1 / 60 (1.67%)
         occurrences all number
    5
    0
    5
    1
    Respiratory syncytial virus infection
         subjects affected / exposed
    3 / 128 (2.34%)
    3 / 53 (5.66%)
    0 / 75 (0.00%)
    0 / 60 (0.00%)
         occurrences all number
    3
    3
    0
    0
    Bronchitis
         subjects affected / exposed
    2 / 128 (1.56%)
    2 / 53 (3.77%)
    0 / 75 (0.00%)
    6 / 60 (10.00%)
         occurrences all number
    2
    2
    0
    6
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    4 / 128 (3.13%)
    3 / 53 (5.66%)
    1 / 75 (1.33%)
    1 / 60 (1.67%)
         occurrences all number
    4
    3
    1
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    03 Aug 2022
    The primary reasons for amending the SRK-015-003 protocol were to add collection of antidrug antibodies at various visits, align the timing of vital sign and hypersensitivity monitoring, Change the version of the Columbia-Suicide Severity Rating Scale (C SSRS) to the Children’s “baseline/screening” and the Children’s “since last visit,” which are more appropriate for clinical trials, continue blinding of treatment assignment for patients, Investigators, and site personnel until the completion of the extension trial to reduce the chances of introducing bias in measures assessed in the extension trial, clarify that the interim analysis and stopping the trial for early efficacy are optional and to clarify the time allowed between apitegromab doses, the required timing for pharmacokinetic (PK)-matched electrocardiograms (ECGs), eligibility for the extension trial, the end of study visit for patients who enroll in the extension trial, and overdose language/reporting procedures.

    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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