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    Clinical Trial Results:
    A Multicenter, Randomized, Placebo-controlled Study of SBC-102 in Patients with Lysosomal Acid Lipase Deficiency (ARISE [Acid Lipase Replacement Investigating Safety and Efficacy])

    Due to a system error, the data reported in v1 is not correct and has been removed from public view.
    Summary
    EudraCT number
    2011-002750-31
    Trial protocol
    DE   GB   IT   ES   CZ   PL   GR   HR   FR  
    Global end of trial date

    Results information
    Results version number
    v2
    This version publication date
    20 Jul 2016
    First version publication date
    06 Aug 2015
    Other versions
    v1 (removed from public view) , v3 , v4
    Version creation reason
    • Correction of full data set
    Corrections are made in line with posting of the results of this trial to clinicaltrials.gov

    Trial information

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    Trial identification
    Sponsor protocol code
    LAL-CL02
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01757184
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals
    Sponsor organisation address
    352 Knotter Drive, Cheshire, United States, CT 06410
    Public contact
    Raquel Cerezo, Clinical Project Lead, Alexion Pharmaceuticals, +1 7814302475, CerezoR@alxn.com
    Scientific contact
    Mark Friedman, Medical Director, Alexion Pharmaceuticals, +1 7814302497, mark.friedman@alxn.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    EMEA-001331-PIP01-12
    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
    Interim
    Date of interim/final analysis
    04 Sep 2014
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    30 May 2014
    Global end of trial reached?
    No
    General information about the trial
    Main objective of the trial
    The primary objective was to demonstrate efficacy of sebelipase alfa relative to placebo, based on normalisation of alanine aminotransferase (ALT) in patients with Lysosomal Acid Lipase (LAL) Deficiency.
    Protection of trial subjects
    Subjects have the right to withdraw from the study at any time for any reason without prejudice to further treatment. The investigator and Sponsor also have the right to withdraw subjects from the study at any time. Specific reasons for discontinuation may include, but are not restricted to, the following: - intercurrent illness - medical-significant AEs, including AEs requiring emergency unblinding - pregnancy - protocol deviation or non-compliance - termination of the study by the sponsor
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    21 Jan 2013
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Efficacy, Safety
    Long term follow-up duration
    30 Months
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Poland: 7
    Country: Number of subjects enrolled
    Spain: 4
    Country: Number of subjects enrolled
    United Kingdom: 4
    Country: Number of subjects enrolled
    Croatia: 1
    Country: Number of subjects enrolled
    Czech Republic: 5
    Country: Number of subjects enrolled
    Germany: 2
    Country: Number of subjects enrolled
    Italy: 3
    Country: Number of subjects enrolled
    Argentina: 1
    Country: Number of subjects enrolled
    Australia: 4
    Country: Number of subjects enrolled
    France: 5
    Country: Number of subjects enrolled
    Japan: 2
    Country: Number of subjects enrolled
    Mexico: 4
    Country: Number of subjects enrolled
    Russian Federation: 4
    Country: Number of subjects enrolled
    Turkey: 4
    Country: Number of subjects enrolled
    United States: 16
    Worldwide total number of subjects
    66
    EEA total number of subjects
    31
    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)
    24
    Adolescents (12-17 years)
    23
    Adults (18-64 years)
    19
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    A total of 55 study centers located in 17 countries were initiated in this study including 49 during recruitment and 6 after to allow transfer of subjects for local treatment. Subjects were screened at 41 of 55 study centers in all countries except Greece.

    Pre-assignment
    Screening details
    To assess eligibility, subjects were screened for a period of up to 6 weeks prior to enrollment in the study. A total of 86 subjects were screened. Six of these subjects underwent re-screening (of which 2 were eligible for the study). In total, 66 subjects were eligible for the study and 20 subjects were screen failures.

    Period 1
    Period 1 title
    Double-blind followed by Open-Label (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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Double-blind Sebelipase Alfa
    Arm description
    Double-blind period: IV infusions
    Arm type
    Experimental

    Investigational medicinal product name
    Sebelipase alfa
    Investigational medicinal product code
    SBC-102
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Every other week IV infusions of SBC-102 at a dose of 1 mg/kg

    Arm title
    Double-blind Placebo
    Arm description
    Double-blind period: IV infusions
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Every other week infusions of placebo

    Number of subjects in period 1
    Double-blind Sebelipase Alfa Double-blind Placebo
    Started
    36
    30
    Completed
    35
    30
    Not completed
    1
    0
         Adverse event, non-fatal
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Double-blind Sebelipase Alfa
    Reporting group description
    Double-blind period: IV infusions

    Reporting group title
    Double-blind Placebo
    Reporting group description
    Double-blind period: IV infusions

    Reporting group values
    Double-blind Sebelipase Alfa Double-blind Placebo Total
    Number of subjects
    36 30 66
    Age categorical
    Units: Subjects
        Children (2-11 years)
    14 10 24
        Adolescents (12-17 years)
    9 14 23
        Adults (18-64 years)
    13 6 19
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    16.9 ± 11.6 15.2 ± 10.2 -
    Gender categorical
    Units: Subjects
        Female
    18 15 33
        Male
    18 15 33
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    6 4 10
        Not Hispanic or Latino
    30 26 56
    Race
    Units: Subjects
        Asian
    1 0 1
        Japanese
    2 0 2
        Black or African American
    1 0 1
        White
    27 28 55
        Other
    5 2 7
    Subject analysis sets

    Subject analysis set title
    FAS
    Subject analysis set type
    Modified intention-to-treat
    Subject analysis set description
    The FAS comprised subjects in the Consented Set who, in addition, were randomised and received at least 1 dose of sebelipase alfa or placebo. The FAS was a modified intention-to-treat (ITT) dataset.

    Subject analysis set title
    Consented Set
    Subject analysis set type
    Intention-to-treat
    Subject analysis set description
    The Consented Subject Set (Consented Set) was comprised of all subjects who signed informed consent.

    Subject analysis set title
    Per Protocol Set
    Subject analysis set type
    Per protocol
    Subject analysis set description
    The Per Protocol Set (PP Set) comprised subjects in the FAS who, in addition, (1) received at least 9 complete infusions of study drug during the double-blind treatment period; (2) had measurements of ALT at both baseline and Week 20; (3) had Week 20 assessments within 12 to 21 days of the preceding (Week 18) infusion; (4) did not change their LLM; and (5) did not have any other major protocol deviation that would affect interpretation of results for serum transaminases or serum lipids.

    Subject analysis set title
    Extension Analysis Set
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Extension Analysis Set (EAS), comprised of subjects in the Consented Set who, in addition, were randomised to treatment and received at least 1 dose (or any portion of a dose) of sebelipase alfa. For subjects who were originally randomised to sebelipase alfa and received at least 1 dose of sebelipase alfa (SA/SA), all assessments from both the double-blind and the open-label period were included in the EAS. This included subjects who were dosed in the double-blind phase with sebelipase alfa, but did not initiate open-label sebelipase alfa. For subjects who were originally randomised to placebo and received at least 1 dose of sebelipase alfa in the open-label period (PBO/SA), only assessments from the open-label period were included in the EAS.

    Subject analysis sets values
    FAS Consented Set Per Protocol Set Extension Analysis Set
    Number of subjects
    66
    66
    63
    66
    Age categorical
    Units: Subjects
        Children (2-11 years)
    24
    24
    23
    24
        Adolescents (12-17 years)
    23
    23
    22
    23
        Adults (18-64 years)
    19
    19
    18
    19
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    16.1 ± 10.9
    16.1 ± 10.9
    16.3 ± 11.1
    16.1 ± 10.9
    Gender categorical
    Units: Subjects
        Female
    33
    33
    33
    33
        Male
    33
    33
    30
    33
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    10
    10
    9
    10
        Not Hispanic or Latino
    56
    56
    54
    56
    Race
    Units: Subjects
        Asian
    1
    1
    1
    1
        Japanese
    2
    2
    1
    2
        Black or African American
    1
    1
    1
    1
        White
    55
    55
    53
    55
        Other
    7
    7
    7
    7

    End points

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    End points reporting groups
    Reporting group title
    Double-blind Sebelipase Alfa
    Reporting group description
    Double-blind period: IV infusions

    Reporting group title
    Double-blind Placebo
    Reporting group description
    Double-blind period: IV infusions

    Subject analysis set title
    FAS
    Subject analysis set type
    Modified intention-to-treat
    Subject analysis set description
    The FAS comprised subjects in the Consented Set who, in addition, were randomised and received at least 1 dose of sebelipase alfa or placebo. The FAS was a modified intention-to-treat (ITT) dataset.

    Subject analysis set title
    Consented Set
    Subject analysis set type
    Intention-to-treat
    Subject analysis set description
    The Consented Subject Set (Consented Set) was comprised of all subjects who signed informed consent.

    Subject analysis set title
    Per Protocol Set
    Subject analysis set type
    Per protocol
    Subject analysis set description
    The Per Protocol Set (PP Set) comprised subjects in the FAS who, in addition, (1) received at least 9 complete infusions of study drug during the double-blind treatment period; (2) had measurements of ALT at both baseline and Week 20; (3) had Week 20 assessments within 12 to 21 days of the preceding (Week 18) infusion; (4) did not change their LLM; and (5) did not have any other major protocol deviation that would affect interpretation of results for serum transaminases or serum lipids.

    Subject analysis set title
    Extension Analysis Set
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Extension Analysis Set (EAS), comprised of subjects in the Consented Set who, in addition, were randomised to treatment and received at least 1 dose (or any portion of a dose) of sebelipase alfa. For subjects who were originally randomised to sebelipase alfa and received at least 1 dose of sebelipase alfa (SA/SA), all assessments from both the double-blind and the open-label period were included in the EAS. This included subjects who were dosed in the double-blind phase with sebelipase alfa, but did not initiate open-label sebelipase alfa. For subjects who were originally randomised to placebo and received at least 1 dose of sebelipase alfa in the open-label period (PBO/SA), only assessments from the open-label period were included in the EAS.

    Primary: ALT normalisation

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    End point title
    ALT normalisation
    End point description
    The primary efficacy endpoint was the proportion of subjects who achieved ALT (alanine aminotransferase) normalisation (i.e., ALT below the age-and gender-specific ULN provided by the central laboratory performing this assay) at the end of the double-blind treatment period (i.e. the last double-blind assessment), relative to placebo.
    End point type
    Primary
    End point timeframe
    At the end of double-blind period (week 20)
    End point values
    Double-blind Sebelipase Alfa Double-blind Placebo
    Number of subjects analysed
    36
    30
    Units: number of subjects with normalized ALT
    11
    2
    Statistical analysis title
    Primary Analysis
    Comparison groups
    Double-blind Sebelipase Alfa v Double-blind Placebo
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0271
    Method
    Fisher exact
    Parameter type
    Risk difference (RD)
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -
         upper limit
    -
    Variability estimate
    Standard deviation

    Secondary: LDL-c Reduction

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    End point title
    LDL-c Reduction
    End point description
    Relative reduction (percentage change from baseline) in LDL-c at the end of the double-blind period
    End point type
    Secondary
    End point timeframe
    At end of double-blind period (week 20)
    End point values
    Double-blind Sebelipase Alfa Double-blind Placebo
    Number of subjects analysed
    36
    30
    Units: mean percent reduction
        arithmetic mean (standard deviation)
    -28.42 ± 22.304
    -6.25 ± 13.015
    Statistical analysis title
    Secondary efficacy endpoint
    Comparison groups
    Double-blind Sebelipase Alfa v Double-blind Placebo
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001
    Method
    Wilcoxon (Mann-Whitney)
    Parameter type
    Risk difference (RD)
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -
         upper limit
    -
    Variability estimate
    Standard deviation

    Secondary: non-HDL-c Reduction

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    End point title
    non-HDL-c Reduction
    End point description
    Relative reduction (percentage change from baseline) in non-high-density lipoprotein-cholesterol (non-HDL-c) at the end of the double-blind period
    End point type
    Secondary
    End point timeframe
    At the end of the double-blind period (week 20)
    End point values
    Double-blind Sebelipase Alfa Double-blind Placebo
    Number of subjects analysed
    36
    30
    Units: Percent reduction
        arithmetic mean (standard deviation)
    -27.97 ± 18.612
    -6.94 ± 10.922
    Statistical analysis title
    Secondary efficacy endpoint
    Comparison groups
    Double-blind Sebelipase Alfa v Double-blind Placebo
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001
    Method
    Wilcoxon (Mann-Whitney)
    Parameter type
    Risk difference (RD)
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -
         upper limit
    -
    Variability estimate
    Standard deviation

    Secondary: Triglycerides Reduction

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    End point title
    Triglycerides Reduction
    End point description
    Relative reduction (percentage change from baseline) in triglycerides (TG) at the end of the double-blind period
    End point type
    Secondary
    End point timeframe
    At the end of double-blind period (week 20)
    End point values
    Double-blind Sebelipase Alfa Double-blind Placebo
    Number of subjects analysed
    36
    30
    Units: percent decrease from baseline
        arithmetic mean (standard deviation)
    -25.45 ± 29.411
    -11.14 ± 28.827
    Statistical analysis title
    Secondary efficacy endpoint
    Comparison groups
    Double-blind Sebelipase Alfa v Double-blind Placebo
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0375
    Method
    Wilcoxon (Mann-Whitney)
    Parameter type
    Risk difference (RD)
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -
         upper limit
    -

    Secondary: HDL-c Increase

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    End point title
    HDL-c Increase
    End point description
    Relative increase (percentage change from baseline) in high-density lipoprotein-cholesterol (HDL-c) at the end of the double-blind period
    End point type
    Secondary
    End point timeframe
    At the end of the double-blind period (week 20)
    End point values
    Double-blind Sebelipase Alfa Double-blind Placebo
    Number of subjects analysed
    36
    30
    Units: percent increase from baseline
        arithmetic mean (standard deviation)
    19.57 ± 16.833
    -0.29 ± 12.36
    Statistical analysis title
    Secondary efficacy endpoint
    Comparison groups
    Double-blind Sebelipase Alfa v Double-blind Placebo
    Number of subjects included in analysis
    66
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001
    Method
    Wilcoxon (Mann-Whitney)
    Parameter type
    Risk difference (RD)
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -
         upper limit
    -
    Variability estimate
    Standard deviation

    Secondary: Liver Fat Content Reduction

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    End point title
    Liver Fat Content Reduction
    End point description
    Decrease in liver fat content, as assessed by magnetic resonance imaging (MRI) (in the subset of subjects for whom imaging was performed)
    End point type
    Secondary
    End point timeframe
    At end of the double-blind period (week 20)
    End point values
    Double-blind Sebelipase Alfa Double-blind Placebo
    Number of subjects analysed
    32
    25
    Units: percent decrease from baseline
        arithmetic mean (standard deviation)
    -31.98 ± 26.763
    -4.21 ± 15.559
    Statistical analysis title
    Secondary efficacy endpoint
    Comparison groups
    Double-blind Sebelipase Alfa v Double-blind Placebo
    Number of subjects included in analysis
    57
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.0001
    Method
    Wilcoxon (Mann-Whitney)
    Parameter type
    Risk difference (RD)
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -
         upper limit
    -
    Variability estimate
    Standard deviation

    Secondary: Liver Histology Improvement

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    End point title
    Liver Histology Improvement
    End point description
    Improvement in hepatic histology (in the subset of subjects for whom liver biopsy was performed, as determined by blinded central review)
    End point type
    Secondary
    End point timeframe
    At the end of double-blind period (week 20)
    End point values
    Double-blind Sebelipase Alfa Double-blind Placebo
    Number of subjects analysed
    16
    10
    Units: # subjects with improved liver histology
    10
    4
    Statistical analysis title
    Secondary efficacy endpoint
    Comparison groups
    Double-blind Sebelipase Alfa v Double-blind Placebo
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.4216
    Method
    Fisher exact
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -
         upper limit
    -

    Secondary: Liver Volume Reduction

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    End point title
    Liver Volume Reduction
    End point description
    Relative reduction (percentage change from baseline) in liver volume, as assessed by magnetic resonance imaging, in the subset of subjects for whom imaging was performed.
    End point type
    Secondary
    End point timeframe
    At the end of the double-blind period (week 20)
    End point values
    Double-blind Sebelipase Alfa Double-blind Placebo
    Number of subjects analysed
    33
    27
    Units: percent from baseline
        arithmetic mean (standard deviation)
    -10.28 ± 10.51
    -2.66 ± 10.107
    Statistical analysis title
    Secondary efficacy endpoint
    Comparison groups
    Double-blind Sebelipase Alfa v Double-blind Placebo
    Number of subjects included in analysis
    60
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0068
    Method
    Wilcoxon (Mann-Whitney)
    Parameter type
    Risk difference (RD)
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -
         upper limit
    -
    Variability estimate
    Standard deviation

    Secondary: AST Normalisation

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    End point title
    AST Normalisation
    End point description
    The proportion of subjects with an abnormal baseline aspartate aminotransferase (AST; i.e., > ULN) who achieved AST normalisation, based on age- and gender-specific normal ranges provided by the central laboratory performing this assay
    End point type
    Secondary
    End point timeframe
    At end of double-blind period (week 20)
    End point values
    Double-blind Sebelipase Alfa Double-blind Placebo
    Number of subjects analysed
    36
    29
    Units: participants
    15
    1
    Statistical analysis title
    Secondary efficacy endpoint
    Comparison groups
    Double-blind Sebelipase Alfa v Double-blind Placebo
    Number of subjects included in analysis
    65
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0003
    Method
    Fisher exact
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -
         upper limit
    -

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    20 week double-blind treatment period
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    15.1
    Reporting groups
    Reporting group title
    Sebelipase Alfa group
    Reporting group description
    Subjects receiving receive sebelipase alfa 1 mg/kg via IV infusion qow

    Reporting group title
    Placebo group
    Reporting group description
    Subjects received matched placebo via IV infusion qow.

    Serious adverse events
    Sebelipase Alfa group Placebo group
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 36 (5.56%)
    1 / 30 (3.33%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Immune system disorders
    Infusion related reaction
         subjects affected / exposed
    1 / 36 (2.78%)
    0 / 30 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Road traffic accident
         subjects affected / exposed
    0 / 36 (0.00%)
    1 / 30 (3.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Gastritis
         subjects affected / exposed
    1 / 36 (2.78%)
    0 / 30 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    1 / 36 (2.78%)
    0 / 30 (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: 5%
    Non-serious adverse events
    Sebelipase Alfa group Placebo group
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    31 / 36 (86.11%)
    28 / 30 (93.33%)
    Injury, poisoning and procedural complications
    Procedural pain
    Additional description: Procedural pain after liver biopsy unrelated to study drug
         subjects affected / exposed
    1 / 36 (2.78%)
    3 / 30 (10.00%)
         occurrences all number
    1
    3
    Investigations
    Body temperature increased
         subjects affected / exposed
    2 / 36 (5.56%)
    1 / 30 (3.33%)
         occurrences all number
    4
    1
    Cardiac disorders
    Arrhythmia
         subjects affected / exposed
    0 / 36 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    0
    2
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    6 / 36 (16.67%)
    1 / 30 (3.33%)
         occurrences all number
    8
    1
    Epistaxis
         subjects affected / exposed
    4 / 36 (11.11%)
    6 / 30 (20.00%)
         occurrences all number
    8
    7
    Cough
         subjects affected / exposed
    3 / 36 (8.33%)
    3 / 30 (10.00%)
         occurrences all number
    3
    4
    Rhinorrhoea
         subjects affected / exposed
    2 / 36 (5.56%)
    1 / 30 (3.33%)
         occurrences all number
    2
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    10 / 36 (27.78%)
    6 / 30 (20.00%)
         occurrences all number
    17
    7
    Syncope
         subjects affected / exposed
    2 / 36 (5.56%)
    0 / 30 (0.00%)
         occurrences all number
    2
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    7 / 36 (19.44%)
    6 / 30 (20.00%)
         occurrences all number
    7
    6
    Asthenia
         subjects affected / exposed
    3 / 36 (8.33%)
    1 / 30 (3.33%)
         occurrences all number
    3
    1
    Fatigue
         subjects affected / exposed
    0 / 36 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    0
    2
    Vaccination site pain
         subjects affected / exposed
    0 / 36 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    0
    2
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    2 / 36 (5.56%)
    0 / 30 (0.00%)
         occurrences all number
    2
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    6 / 36 (16.67%)
    5 / 30 (16.67%)
         occurrences all number
    6
    6
    Abdominal pain
         subjects affected / exposed
    3 / 36 (8.33%)
    1 / 30 (3.33%)
         occurrences all number
    4
    1
    Constipation
         subjects affected / exposed
    3 / 36 (8.33%)
    1 / 30 (3.33%)
         occurrences all number
    3
    1
    Nausea
         subjects affected / exposed
    3 / 36 (8.33%)
    2 / 30 (6.67%)
         occurrences all number
    3
    2
    Vomiting
         subjects affected / exposed
    3 / 36 (8.33%)
    3 / 30 (10.00%)
         occurrences all number
    4
    6
    Abdominal pain upper
         subjects affected / exposed
    2 / 36 (5.56%)
    2 / 30 (6.67%)
         occurrences all number
    3
    2
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    1 / 36 (2.78%)
    3 / 30 (10.00%)
         occurrences all number
    3
    4
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 36 (5.56%)
    1 / 30 (3.33%)
         occurrences all number
    2
    1
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    6 / 36 (16.67%)
    6 / 30 (20.00%)
         occurrences all number
    8
    7
    Nasopharyngitis
         subjects affected / exposed
    4 / 36 (11.11%)
    3 / 30 (10.00%)
         occurrences all number
    4
    3
    Sinusitis
    Additional description: Non-serious sinusitis unrelated to study drug
         subjects affected / exposed
    2 / 36 (5.56%)
    0 / 30 (0.00%)
         occurrences all number
    2
    0
    Rhinitis
         subjects affected / exposed
    2 / 36 (5.56%)
    3 / 30 (10.00%)
         occurrences all number
    2
    3
    Tonsillitis
         subjects affected / exposed
    2 / 36 (5.56%)
    4 / 30 (13.33%)
         occurrences all number
    2
    4
    Pharyngitis
         subjects affected / exposed
    0 / 36 (0.00%)
    5 / 30 (16.67%)
         occurrences all number
    0
    5
    Respiratory tract infection viral
         subjects affected / exposed
    0 / 36 (0.00%)
    2 / 30 (6.67%)
         occurrences all number
    0
    2
    Varicella
         subjects affected / exposed
    0 / 36 (0.00%)
    3 / 30 (10.00%)
         occurrences all number
    0
    2

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? No

    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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