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    Clinical Trial Results:
    Phase IIIb, open-label, single-arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101 administered intrathecally (1.2 x 10^14 vector genomes) to participants 2 to <18 years of age with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen (Spinraza®) or risdiplam (Evrysdi®)

    Summary
    EudraCT number
    		 2021-006709-31
    Trial protocol
    		 ES   FR   DE   BE   NL   IT  
    Global end of trial date
    29 Nov 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    31 May 2025
    First version publication date
    31 May 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    COAV101B12302
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05386680
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    Novartis Campus, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.com
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-002168-PIP01-17
    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
    29 Nov 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    29 Nov 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective was to characterize the safety and tolerability of OAV101B (administered by lumbar intrathecal injection) over a 52-week period in participants with SMA aged 2 to <18 years who had discontinued treatment with nusinersen or risdiplam.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    12 Jan 2023
    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: 1
    Country: Number of subjects enrolled
    Belgium: 2
    Country: Number of subjects enrolled
    Canada: 1
    Country: Number of subjects enrolled
    France: 5
    Country: Number of subjects enrolled
    Italy: 1
    Country: Number of subjects enrolled
    Japan: 4
    Country: Number of subjects enrolled
    Netherlands: 6
    Country: Number of subjects enrolled
    Spain: 4
    Country: Number of subjects enrolled
    United States: 3
    Worldwide total number of subjects
    27
    EEA total number of subjects
    18
    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)
    25
    Adolescents (12-17 years)
    2
    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
    		 -

    Pre-assignment
    Screening details
    		 The study aimed to enroll approximately 28 participants across each of 2 age brackets (2 to <6 years, and 6 to <18 years).

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 OAV101 1.2x10^14 vg - All participants
    Arm description
    		 Intrathecal administration of OAV101 at a dose of 1.2 x 10^14 vector genomes, one time dose
    Arm type
    		 Experimental

    Investigational medicinal product name
    onasemnogene abeparvovec-xioi
    Investigational medicinal product code
    OAV101
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    administered at a dose 1.2 x 10^14 vector genomes as a one-time injection

    Number of subjects in period 1
    		OAV101 1.2x10^14 vg - All participants
    Started
    27
    Completed
    25
    Not completed
    2
         Guardian decision
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    OAV101 1.2x10^14 vg - All participants
    Reporting group description
    		 Intrathecal administration of OAV101 at a dose of 1.2 x 10^14 vector genomes, one time dose

    Reporting group values
    		 OAV101 1.2x10^14 vg - All participants Total
    Number of subjects
    		 27 27
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 25 25
        Adolescents (12-17 years)
    		 2 2
        Adults (18-64 years)
    		 0 0
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 7.40 ( 3.348 ) -
    Sex: Female, Male
    Units: Participants
        Female
    		 12 12
        Male
    		 15 15
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0
        Asian
    		 6 6
        Native Hawaiian or Other Pacific Islander
    		 0 0
        Black or African American
    		 0 0
        White
    		 13 13
        More than one race
    		 1 1
        Unknown or Not Reported
    		 7 7

    End points

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    End points reporting groups
    Reporting group title
    OAV101 1.2x10^14 vg - All participants
    Reporting group description
    		 Intrathecal administration of OAV101 at a dose of 1.2 x 10^14 vector genomes, one time dose

    Primary: Overview of treatment-emergent adverse events by age subgroup

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    End point title
    		 Overview of treatment-emergent adverse events by age subgroup [1]
    End point description
    An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. The occurrence of AEs must be sought by non-directive questioning of the participant at each visit during the study. Adverse events also may be detected when they are volunteered by the participant during or between visits or through physical examination findings, laboratory test findings, or other assessments. disc. = discontinuation
    End point type
    Primary
    End point timeframe
    Adverse events were reported from single dose of study treatment plus 52 weeks, up to a maximum time period of 52 weeks.
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: not applicable for single arm study
    End point values
    		 OAV101 1.2x10^14 vg - All participants
    Number of subjects analysed
    27
    Units: Participants
        Any treatment-emergent adverse event (TEAE)
    27
        Any TEAE related to study treatment
    13
        Any serious TEAE
    4
        Any serious TEAE related to study treatment
    0
        Any severe TEAE
    1
        Any TEAE leading to study disc.
    0
        Any TEAE leading to death
    0
        Any TEAE of special interest
    13
    No statistical analyses for this end point

    Primary: Treatment-emergent adverse events related to treatment by system organ class, preferred term, age subgroup (>= 10%)

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    End point title
    		 Treatment-emergent adverse events related to treatment by system organ class, preferred term, age subgroup (>= 10%) [2]
    End point description
    An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. The occurrence of AEs must be sought by non-directive questioning of the participant at each visit during the study. Adverse events also may be detected when they are volunteered by the participant during or between visits or through physical examination findings, laboratory test findings, or other assessments. adm. = administration
    End point type
    Primary
    End point timeframe
    Adverse events were reported from single dose of study treatment plus 52 weeks, up to a maximum time period of 52 weeks.
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: not applicable for single arm study
    End point values
    		 OAV101 1.2x10^14 vg - All participants
    Number of subjects analysed
    27
    Units: Participants
        Number of participants with at least one event
    13
        Gastrointestinal disorders
    7
        -Vomiting
    6
        General disorders and adm. site conditions
    5
        -Pyrexia
    3
        Nervous system disorders
    6
        -Headache
    4
    No statistical analyses for this end point

    Primary: Adverse events of special interest by system organ class, preferred term, age subgroup

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    End point title
    		 Adverse events of special interest by system organ class, preferred term, age subgroup [3]
    End point description
    An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. The occurrence of AEs must be sought by non-directive questioning of the participant at each visit during the study. Adverse events also may be detected when they are volunteered by the participant during or between visits or through physical examination findings, laboratory test findings, or other assessments. An adverse event of special interest (AESI) is primarily defined by using standard Medical Dictionary for Regulatory Activities (MedDRA) queries, and identified as follows: Hepatotoxicity, Transient thrombocytopenia, Thrombotic microangiopathy, Cardiac adverse events, signs and symptoms that may be suggestive dorsal root ganglia toxicity, and new malignancies.
    End point type
    Primary
    End point timeframe
    Adverse events were reported from single dose of study treatment plus 52 weeks, up to a maximum time period of 52 weeks.
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: not applicable for single arm study
    End point values
    		 OAV101 1.2x10^14 vg - All participants
    Number of subjects analysed
    27
    Units: Participants
        Hepatotoxicity
    4
        -Hepatic enzyme increased
    3
        -Hypertransaminasaemia
    1
        Transient thrombocytopenia -
    8
        -Epistaxis
    3
        -Contusion
    2
        -Bone contusion
    1
        -Gastric haemorrhage
    1
        -Lower gastrointestinal haemorrhage
    1
        -Petechiae
    1
        Signs of dorsal root ganglia toxicity -
    2
        -Paraesthesia
    1
        -Sensory disturbance
    1
        Thrombotic microangiopathy -
    0
        Cardiac adverse events
    0
        New malignancies
    0
    No statistical analyses for this end point

    Secondary: Change from baseline at Week 52 visit in the HFMSE total score - Mean (SD)

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    End point title
    		 Change from baseline at Week 52 visit in the HFMSE total score - Mean (SD)
    End point description
    The Hammersmith Functional Motor Scale Expanded (HFMSE) is a SMA-specific 33-item assessment that is administered by qualified clinical evaluators. Each motor skill item is scored on a 3-point Likert scale from 0 (no response) to 2 (full response), with a total score range of 0 to 66. A higher score indicates a higher ability level.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 OAV101 1.2x10^14 vg - All participants
    Number of subjects analysed
    21
    Units: scores on a scale
        arithmetic mean (standard deviation)
    0.17 ( 2.878 )
    No statistical analyses for this end point

    Secondary: Change from baseline at Week 52 visit in the RULM total Score - Mean (SD)

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    End point title
    		 Change from baseline at Week 52 visit in the RULM total Score - Mean (SD)
    End point description
    The Revised Upper Limb Model (RULM) is a validated, SMA-specific assessment that measures motor performance in the upper limbs from childhood through adulthood in ambulatory and never ambulatory individuals with SMA. The revised version of the test consists of 19 scorable items: 18 items scored on a 0 (unable) to 2 (full achievement) scale, and one item that is scored from 0 (unable) to 1 (able). These item scores are summed to give a total score ranging from 0 to 37 points with lower scores reflecting poorer ability.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 OAV101 1.2x10^14 vg - All participants
    Number of subjects analysed
    21
    Units: scores on a scale
        arithmetic mean (standard deviation)
    0.29 ( 2.849 )
    No statistical analyses for this end point

    Secondary: Change from baseline at Week 52 visit in Assessment of Caregiver Experience in ACEND instrument score - Mean (SD)

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    End point title
    		 Change from baseline at Week 52 visit in Assessment of Caregiver Experience in ACEND instrument score - Mean (SD)
    End point description
    The Assessment of Caregiver Experience in Neuromuscular Disease (ACEND) instrument quantifies the caregiver impact experienced by parents/caregivers of children affected with severe neuromuscular diseases, including children with SMA. The total score is on a scale of 0 to 100 with a higher score indicating that caregivers experienced less intense caregiving impact.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 OAV101 1.2x10^14 vg - All participants
    Number of subjects analysed
    24
    Units: scores on a scale
        arithmetic mean (standard deviation)
    1.43 ( 9.318 )
    No statistical analyses for this end point

    Secondary: Change from baseline at Week 52 visit in the HFMSE total score - LS Means

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    End point title
    		 Change from baseline at Week 52 visit in the HFMSE total score - LS Means
    End point description
    The Hammersmith Functional Motor Scale Expanded (HFMSE) is a SMA-specific 33-item assessment that is administered by qualified clinical evaluators. Each motor skill item is scored on a 3-point Likert scale from 0 (no response) to 2 (full response), with a total score range of 0 to 66. A higher score indicates a higher ability level.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 OAV101 1.2x10^14 vg - All participants
    Number of subjects analysed
    21
    Units: scores on a scale
        least squares mean (confidence interval 95%)
    1.05 (-0.21 to 2.32)
    No statistical analyses for this end point

    Secondary: Change from baseline at Week 52 visit in the RULM total Score - LS Means

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    End point title
    		 Change from baseline at Week 52 visit in the RULM total Score - LS Means
    End point description
    The Revised Upper Limb Model (RULM) is a validated, SMA-specific assessment that measures motor performance in the upper limbs from childhood through adulthood in ambulatory and never ambulatory individuals with SMA. The revised version of the test consists of 19 scorable items: 18 items scored on a 0 (unable) to 2 (full achievement) scale, and one item that is scored from 0 (unable) to 1 (able). These item scores are summed to give a total score ranging from 0 to 37 points with lower scores reflecting poorer ability.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 OAV101 1.2x10^14 vg - All participants
    Number of subjects analysed
    21
    Units: scores on a scale
        least squares mean (confidence interval 95%)
    0.59 (-0.56 to 1.73)
    No statistical analyses for this end point

    Secondary: Change from baseline at Week 52 visit in Assessment of Caregiver Experience in ACEND instrument score - LS Means

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    End point title
    		 Change from baseline at Week 52 visit in Assessment of Caregiver Experience in ACEND instrument score - LS Means
    End point description
    The Assessment of Caregiver Experience in Neuromuscular Disease (ACEND) instrument quantifies the caregiver impact experienced by parents/caregivers of children affected with severe neuromuscular diseases, including children with SMA. The total score is on a scale of 0 to 100 with a higher score indicating that caregivers experienced less intense caregiving impact.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 OAV101 1.2x10^14 vg - All participants
    Number of subjects analysed
    24
    Units: scores on a scale
        least squares mean (confidence interval 95%)
    1.06 (-2.90 to 5.02)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events were reported from single dose of study treatment plus 52 weeks, up to a maximum time period of 52 weeks.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.1
    Reporting groups
    Reporting group title
    Overall
    Reporting group description
    		 Overall

    Serious adverse events
    		 Overall
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 27 (14.81%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Investigations
    Influenza virus test positive
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Cyclic vomiting syndrome
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 5
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Respiratory distress
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Lung disorder
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Nasal congestion
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Bronchitis viral
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    COVID-19
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia aspiration
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 27 (3.70%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Overall
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    27 / 27 (100.00%)
    Investigations
    Hepatic enzyme increased
         subjects affected / exposed
    3 / 27 (11.11%)
         occurrences all number
    3
    Weight increased
         subjects affected / exposed
    5 / 27 (18.52%)
         occurrences all number
    5
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    3 / 27 (11.11%)
         occurrences all number
    6
    Contusion
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    8 / 27 (29.63%)
         occurrences all number
    18
    Tremor
         subjects affected / exposed
    4 / 27 (14.81%)
         occurrences all number
    5
    Blood and lymphatic system disorders
    Iron deficiency anaemia
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    2
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    4 / 27 (14.81%)
         occurrences all number
    5
    Pyrexia
         subjects affected / exposed
    13 / 27 (48.15%)
         occurrences all number
    26
    Swelling face
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    2
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    3
    Constipation
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    2
    Abdominal pain upper
         subjects affected / exposed
    6 / 27 (22.22%)
         occurrences all number
    7
    Abdominal pain
         subjects affected / exposed
    4 / 27 (14.81%)
         occurrences all number
    6
    Dysphagia
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    4
    Nausea
         subjects affected / exposed
    3 / 27 (11.11%)
         occurrences all number
    4
    Toothache
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    2
    Vomiting
         subjects affected / exposed
    13 / 27 (48.15%)
         occurrences all number
    21
    Respiratory, thoracic and mediastinal disorders
    Rhinorrhoea
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    3
    Oropharyngeal pain
         subjects affected / exposed
    3 / 27 (11.11%)
         occurrences all number
    3
    Nasal congestion
         subjects affected / exposed
    3 / 27 (11.11%)
         occurrences all number
    3
    Epistaxis
         subjects affected / exposed
    3 / 27 (11.11%)
         occurrences all number
    5
    Cough
         subjects affected / exposed
    5 / 27 (18.52%)
         occurrences all number
    8
    Productive cough
         subjects affected / exposed
    5 / 27 (18.52%)
         occurrences all number
    5
    Psychiatric disorders
    Affect lability
         subjects affected / exposed
    3 / 27 (11.11%)
         occurrences all number
    3
    Initial insomnia
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    2
    Irritability
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    2
    Musculoskeletal and connective tissue disorders
    Myalgia
         subjects affected / exposed
    3 / 27 (11.11%)
         occurrences all number
    3
    Muscular weakness
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    2
    Back pain
         subjects affected / exposed
    4 / 27 (14.81%)
         occurrences all number
    6
    Pain in extremity
         subjects affected / exposed
    5 / 27 (18.52%)
         occurrences all number
    8
    Scoliosis
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    2
    Infections and infestations
    COVID-19
         subjects affected / exposed
    4 / 27 (14.81%)
         occurrences all number
    5
    Ear infection
         subjects affected / exposed
    4 / 27 (14.81%)
         occurrences all number
    4
    Upper respiratory tract infection
         subjects affected / exposed
    3 / 27 (11.11%)
         occurrences all number
    6
    Nasopharyngitis
         subjects affected / exposed
    15 / 27 (55.56%)
         occurrences all number
    25
    Influenza
         subjects affected / exposed
    3 / 27 (11.11%)
         occurrences all number
    3
    Metabolism and nutrition disorders
    Increased appetite
         subjects affected / exposed
    5 / 27 (18.52%)
         occurrences all number
    5
    Decreased appetite
         subjects affected / exposed
    2 / 27 (7.41%)
         occurrences all number
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    17 May 2023
    The purpose of this amendment was to expand the age group for inclusion in the study from 2 to 12 years of age, to 2 to <18 years of age to cover the full pediatric age range and for consistency with the study population age range of Novartis Phase III study (COAV101B2301) in treatment naïve participants aged 2 to <18 years. In addition, the risk language has been revised to include the theoretical risk of tumorigenicity due to the very low potential incorporation of AAV vector DNA into chromosomal DNA that has been noted based on published literature for AAV-based therapies; also, the liver safety monitoring for elevated levels has been updated to apply more stringent monitoring to ensure a timely follow-up and to align with FDA guidance for drug induced liver toxicity.

    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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