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    Clinical Trial Results:
    Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome.

    Summary
    EudraCT number
    		 2020-004628-40
    Trial protocol
    		 BE   FR   PL  
    Global end of trial date
    17 Dec 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    17 Jul 2025
    First version publication date
    17 Jul 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    MRX-801
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04729751
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 IND: 119916, IND : 119917
    Sponsors
    Sponsor organisation name
    Mirum Pharmaceuticals Inc.
    Sponsor organisation address
    989 E Hillsdale Blvd. Suite 300, Foster City, United States, 94404
    Public contact
    Chief Medical Officer, Mirum Pharmaceuticals Inc., 1 6506674085, medinfo@mirumpharma.com
    Scientific contact
    Chief Medical Officer, Mirum Pharmaceuticals Inc., 1 6506674085, medinfo@mirumpharma.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001475-PIP02-13 EMEA-001475-PIP03-17
    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
    11 Feb 2025
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    17 Dec 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Dec 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of maralixibat in infant participants with cholestatic liver disease, Alagille syndrome (ALGS) or progressive familial intrahepatic cholestatic (PFIC).
    Protection of trial subjects
    All study participants (caregivers as applicable) were required to read and sign an Informed Consent Form (ICF). Participants were re-consented to the most current version of the ICF(s) during their participation in the study.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    09 Sep 2021
    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: 5
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    France: 3
    Country: Number of subjects enrolled
    Brazil: 4
    Country: Number of subjects enrolled
    United States: 7
    Country: Number of subjects enrolled
    Poland: 7
    Worldwide total number of subjects
    27
    EEA total number of subjects
    11
    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)
    27
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 27 participants were enrolled in the study, across 10 sites in 6 countries (Belgium, Brazil, France, Poland, United Kingdom, and United States), 17 patients in the ALGS Cohort and 10 in the PFIC Cohort.

    Pre-assignment
    Screening details
    		 The screening period starts when informed consent (by the legally authorized representative) is signed. The duration of the screening period is up to 4 weeks, during which all procedures listed for the screening visit in the schedule of assessment must be completed.

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

    Arms
    Arm title
    		 Maralixibat
    Arm description
    		 Open-label, multicenter, Phase 2 study to evaluate the safety and tolerability of maralixibat in the treatment of infants (<12 months of age) with cholestatic liver disease (ALGS or PFIC). Participants with ALGS received maralixibat doses in the ranges of 200 to 400 µg/kg QD. Participants with PFIC received maralixibat doses in the ranges of 300 QD to 600 µg/kg BID.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Maralixibat Chloride
    Investigational medicinal product code
    Other name
    Maralixibat
    Pharmaceutical forms
    Oral solution in bottle
    Routes of administration
    Oral use
    Dosage and administration details
    Four different strengths of the maralixibat oral solution (5, 10, 15, and 20 mg/mL) were used; dosing was based on participant weight.

    Number of subjects in period 1
    		Maralixibat
    Started
    27
    Completed
    25
    Not completed
    2
         Consent withdrawn by subject
    1
         Adverse event, non-fatal
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Maralixibat
    Reporting group description
    		 Open-label, multicenter, Phase 2 study to evaluate the safety and tolerability of maralixibat in the treatment of infants (<12 months of age) with cholestatic liver disease (ALGS or PFIC). Participants with ALGS received maralixibat doses in the ranges of 200 to 400 µg/kg QD. Participants with PFIC received maralixibat doses in the ranges of 300 QD to 600 µg/kg BID.

    Reporting group values
    		 Maralixibat 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)
    		 27 27
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 0 0
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age continuous
    Units: months
        least squares mean (standard deviation)
    		 7.1 ( 3.03 ) -
    Gender categorical
    Units: Subjects
        Female
    		 6 6
        Male
    		 21 21
    Subject analysis sets

    Subject analysis set title
    ALGS
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat.

    Subject analysis set title
    PFIC
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.

    Subject analysis sets values
    		 ALGS PFIC
    Number of subjects
    17
    10
    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)
    17
    10
        Children (2-11 years)
    0
    0
        Adolescents (12-17 years)
    0
    0
        Adults (18-64 years)
    0
    0
        From 65-84 years
    0
    0
        85 years and over
    0
    0
    Age continuous
    Units: months
        least squares mean (standard deviation)
    7.4 ( 2.48 )
    6.6 ( 3.89 )
    Gender categorical
    Units: Subjects
        Female
    3
    3
        Male
    14
    7

    End points

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    End points reporting groups
    Reporting group title
    Maralixibat
    Reporting group description
    		 Open-label, multicenter, Phase 2 study to evaluate the safety and tolerability of maralixibat in the treatment of infants (<12 months of age) with cholestatic liver disease (ALGS or PFIC). Participants with ALGS received maralixibat doses in the ranges of 200 to 400 µg/kg QD. Participants with PFIC received maralixibat doses in the ranges of 300 QD to 600 µg/kg BID.

    Subject analysis set title
    ALGS
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat.

    Subject analysis set title
    PFIC
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.

    Primary: Incidence of Treatment-Emergent Adverse Events (TEAEs)

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    End point title
    		 Incidence of Treatment-Emergent Adverse Events (TEAEs) [1]
    End point description
    TEAE = Treatment -emergent Adverse Event
    End point type
    Primary
    End point timeframe
    From Baseline through End of Treatment (up to approximately 13 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: High-level summary of treatment-emergent adverse events have been provided by the number and percentage of participants who experienced them. The summary includes the total number and percent of participants by cohort and overall maralixibat treated population.
    End point values
    		 Maralixibat ALGS PFIC
    Number of subjects analysed
    27
    17
    10
    Units: number of participants
        At least one TEAE
    26
    16
    10
        TEAE related to study drug
    7
    4
    3
        Grade ≥3 TEAE
    7
    6
    1
        Grade ≥3 TEAE related to study drug
    0
    0
    0
        Serious TEAE
    8
    6
    2
        Serious TEAE related to study drug
    0
    0
    0
        TEAE that led to study discontinuation
    1
    0
    1
        TEAE that led to death
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Change in fasting serum bile acid (sBA) levels

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    End point title
    		 Change in fasting serum bile acid (sBA) levels
    End point description
    End point type
    Secondary
    End point timeframe
    From Baseline through to Week 13, including the change from Baseline value.
    End point values
    		 Maralixibat ALGS PFIC
    Number of subjects analysed
    27
    17
    10
    Units: Serum bile acid levels (µmol/L)
    arithmetic mean (standard deviation)
        Baseline
    270.49 ( 187.898 )
    292.81 ( 218.756 )
    228.32 ( 107.525 )
        Week 13
    157.83 ( 102.890 )
    172.49 ( 109.924 )
    130.33 ( 88.204 )
        Change from Baseline
    -86.91 ( 124.960 )
    -75.68 ( 115.669 )
    -107.96 ( 146.766 )
    Statistical analysis title
    		 Descriptive Analysis of Serum Bile Acid Change
    Statistical analysis description
    Descriptive statistics were used to summarize the serum bile acid levels at Baseline and Week 13. Change from Baseline values were reported to assess long-term trends. No inferential statistical tests were applied.
    Comparison groups
    ALGS v PFIC
    Number of subjects included in analysis
    27
    Analysis specification
    Pre-specified
    Analysis type
    		 other [2]
    P-value
    		 = 0.05 [3]
    Method
    		 not applicable
    Parameter type
    		 not applicable
    Confidence interval
    Notes
    [2] - Descriptive statistics were used to summarize the serum bile acid levels at Baseline and Week 13. Change from Baseline values were reported to assess long-term trends. No inferential statistical tests were applied.
    [3] - Descriptive statistics were used to summarize the serum bile acid levels at Baseline and Week 13. Change from Baseline values were reported to assess long-term trends. No inferential statistical tests were applied.

    Secondary: To evaluate the effect on liver enzymes (ALT, AST) and bilirubin

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    End point title
    		 To evaluate the effect on liver enzymes (ALT, AST) and bilirubin
    End point description
    AST= aspartate aminotransferase, and ALT= alanine aminotransferase.
    End point type
    Secondary
    End point timeframe
    From Baseline through to Week 13
    End point values
    		 Maralixibat ALGS PFIC
    Number of subjects analysed
    27
    17
    10
    Units: ALT, AST and bilirubin levels (U/L)
    arithmetic mean (standard deviation)
        ALT, U/L Baseline
    160.56 ( 105.807 )
    167.35 ( 106.018 )
    149.00 ( 110.108 )
        ALT, U/L Week 13
    208.00 ( 182.954 )
    256.87 ( 190.732 )
    126.56 ( 143.630 )
        ALT, U/L Change from Baseline
    56.17 ( 161.298 )
    99.00 ( 190.981 )
    -15.22 ( 42.387 )
        AST, U/L Baseline
    174.85 ( 100.999 )
    184.12 ( 105.042 )
    159.10 ( 97.034 )
        AST, U/L Week 13
    196.87 ( 140.529 )
    236.87 ( 142.540 )
    121.88 ( 107.591 )
        AST, U/L Change from Baseline
    35.57 ( 102.183 )
    69.27 ( 136.827 )
    -27.63 ( 30.905 )
        Total bilirubin, µmol/L Baseline
    104.94 ( 95.564 )
    142.54 ( 102.323 )
    41.02 ( 23.651 )
        Total bilirubin, µmol/L Week 13
    79.51 ( 93.302 )
    114.89 ( 102.789 )
    20.53 ( 16.668 )
        Total bilirubin, µmol/L Change from Baseline
    -21.79 ( 37.737 )
    -22.85 ( 46.252 )
    -20.02 ( 18.575 )
    Statistical analysis title
    		 Descriptive Analysis of Liver enzymes
    Statistical analysis description
    Descriptive statistics were used to summarize the liver enzymes (ALT, AST and bilirubin) levels at Baseline and Week 13. Change from Baseline values were reported to assess long-term trends. No inferential statistical tests were applied.
    Comparison groups
    ALGS v PFIC
    Number of subjects included in analysis
    27
    Analysis specification
    Pre-specified
    Analysis type
    		 other [4]
    P-value
    		 = 0.05 [5]
    Method
    		 not applicable
    Parameter type
    		 not applicable
    Confidence interval
    Notes
    [4] - Descriptive statistics were used to summarize the liver enzymes (ALT, AST and bilirubin) levels at Baseline and Week 13. Change from Baseline values were reported to assess long-term trends. No inferential statistical tests were applied.
    [5] - Descriptive statistics were used to summarize the liver enzymes (ALT, AST and bilirubin) levels at Baseline and Week 13. Change from Baseline values were reported to assess long-term trends. No inferential statistical tests were applied.

    Secondary: To evaluate the effect on Lipid-Soluble Vitamins (LSVs)

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    End point title
    		 To evaluate the effect on Lipid-Soluble Vitamins (LSVs)
    End point description
    The values marked with 0 reported for Vitamin K values for PFIC Week 13 and Change from Baseline indicates that the values were not calculated.
    End point type
    Secondary
    End point timeframe
    Change from baseline to Week 13 in vitamins A, D, E, and K
    End point values
    		 Maralixibat ALGS PFIC
    Number of subjects analysed
    27
    17
    10
    Units: serum levels
    arithmetic mean (standard deviation)
        Vitamin A umol/L Baseline
    1.68 ( 1.108 )
    1.78 ( 1.189 )
    1.50 ( 0.991 )
        Vitamin A umol/L Week 13
    2.16 ( 0.969 )
    2.08 ( 1.031 )
    2.32 ( 0.884 )
        Vitamin A umol/L Change from Baseline
    0.51 ( 0.732 )
    0.45 ( 0.678 )
    0.61 ( 0.878 )
        Vitamin D nmol/L Baseline
    62.73 ( 58.027 )
    62.64 ( 58.563 )
    62.87 ( 60.308 )
        Vitamin D nmol/L Week 13
    84.47 ( 57.160 )
    78.42 ( 58.952 )
    94.84 ( 56.849 )
        Vitamin D nmol/L Change from Baseline
    12.42 ( 67.451 )
    7.63 ( 79.383 )
    20.65 ( 44.394 )
        Vitamin E mg/L Baseline
    5.28 ( 4.833 )
    6.54 ( 5.351 )
    2.78 ( 2.149 )
        Vitamin E mg/L Week 13
    5.39 ( 4.701 )
    5.99 ( 5.084 )
    3.98 ( 3.658 )
        Vitamin E mg/L Change from Baseline
    -0.04 ( 2.074 )
    -0.46 ( 2.214 )
    0.92 ( 1.422 )
        Vitamin K nmol/L Baseline
    21.68 ( 70.436 )
    31.22 ( 84.315 )
    0.21 ( 0.133 )
        Vitamin K nmol/L Week 13
    14.64 ( 0 )
    14.64 ( 0 )
    0 ( 0 )
        Vitamin K nmol/L Change from Baseline
    -241.14 ( 0 )
    -241.14 ( 0 )
    0 ( 0 )
    Statistical analysis title
    		 Descriptive Analysis of Lipid-Soluble Vitamins
    Statistical analysis description
    Descriptive statistics were used to summarize the Lipid-Soluble Vitamins levels at Baseline and Week 13. Change from Baseline values were reported to assess long-term trends. No inferential statistical tests were applied.
    Comparison groups
    ALGS v PFIC
    Number of subjects included in analysis
    27
    Analysis specification
    Pre-specified
    Analysis type
    		 other [6]
    P-value
    		 = 0.05 [7]
    Method
    		 not applicable
    Parameter type
    		 not applicable
    Confidence interval
    Notes
    [6] - Descriptive statistics were used to summarize the Lipid-Soluble Vitamins levels at Baseline and Week 13. Change from Baseline values were reported to assess long-term trends. No inferential statistical tests were applied.
    [7] - Descriptive statistics were used to summarize the Lipid-Soluble Vitamins levels at Baseline and Week 13. Change from Baseline values were reported to assess long-term trends. No inferential statistical tests were applied.

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline to end of treatment.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.1
    Reporting groups
    Reporting group title
    ALGS
    Reporting group description
    		 All participants in the ALGS arm who received maralixibat and were included in the AE analysis.

    Reporting group title
    PFIC
    Reporting group description
    		 All participants in the PFIC arm who received maralixibat and were included in the AE analysis.

    Serious adverse events
    		 ALGS PFIC
    Total subjects affected by serious adverse events
         subjects affected / exposed
    6 / 17 (35.29%)
    2 / 10 (20.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    General disorders and administration site conditions
    Crying
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pyrexia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infantile colic
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Intestinal obstruction
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Abdominal sepsis
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Adenovirus infection
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Appendicitis perforated
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Corona virus infection
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastroenteritis adenovirus
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Otitis media acute
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Varicella
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Viral infection
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    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
    		 ALGS PFIC
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    16 / 17 (94.12%)
    10 / 10 (100.00%)
    Investigations
    Investigations
         subjects affected / exposed
    3 / 17 (17.65%)
    3 / 10 (30.00%)
         occurrences all number
    5
    6
    Injury, poisoning and procedural complications
    Injury, poisoning and procedural complications
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 10 (20.00%)
         occurrences all number
    1
    4
    Nervous system disorders
    Nervous system disorders
         subjects affected / exposed
    2 / 17 (11.76%)
    2 / 10 (20.00%)
         occurrences all number
    2
    2
    Blood and lymphatic system disorders
    Coagulopathy
         subjects affected / exposed
    0 / 17 (0.00%)
    3 / 10 (30.00%)
         occurrences all number
    0
    6
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    3 / 17 (17.65%)
    2 / 10 (20.00%)
         occurrences all number
    8
    2
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    7 / 17 (41.18%)
    4 / 10 (40.00%)
         occurrences all number
    15
    8
    Vomiting
         subjects affected / exposed
    2 / 17 (11.76%)
    5 / 10 (50.00%)
         occurrences all number
    5
    8
    Teething
         subjects affected / exposed
    3 / 17 (17.65%)
    2 / 10 (20.00%)
         occurrences all number
    7
    3
    Abdominal pain
         subjects affected / exposed
    3 / 17 (17.65%)
    1 / 10 (10.00%)
         occurrences all number
    4
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    4 / 17 (23.53%)
    2 / 10 (20.00%)
         occurrences all number
    5
    2
    Rhinorrhoea
         subjects affected / exposed
    2 / 17 (11.76%)
    3 / 10 (30.00%)
         occurrences all number
    3
    4
    Nasal congestion
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Skin and subcutaneous tissue disorders
    Skin and subcutaneous tissue disorders
         subjects affected / exposed
    4 / 17 (23.53%)
    2 / 10 (20.00%)
         occurrences all number
    5
    2
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    6 / 17 (35.29%)
    4 / 10 (40.00%)
         occurrences all number
    9
    6
    Upper respiratory tract infection
         subjects affected / exposed
    4 / 17 (23.53%)
    4 / 10 (40.00%)
         occurrences all number
    12
    5
    Viral infection
         subjects affected / exposed
    4 / 17 (23.53%)
    4 / 10 (40.00%)
         occurrences all number
    4
    7
    Corona virus infection
         subjects affected / exposed
    3 / 17 (17.65%)
    2 / 10 (20.00%)
         occurrences all number
    3
    2
    Rhinitis
         subjects affected / exposed
    3 / 17 (17.65%)
    2 / 10 (20.00%)
         occurrences all number
    3
    2
    Ear infection
         subjects affected / exposed
    4 / 17 (23.53%)
    0 / 10 (0.00%)
         occurrences all number
    8
    0
    Gastroenteritis
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 10 (10.00%)
         occurrences all number
    2
    1
    Influenza
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 10 (20.00%)
         occurrences all number
    1
    1
    Conjunctivitis
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Otitis media
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Respiratory tract infection viral
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Metabolism and nutrition disorders
    Hypervitaminosis D
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    26 Apr 2021
    Protocol version 2 (Global) released on 26 April 2021. Key Changes made: - Change in inclusion criteria (lower age limit removed) - Inclusion of 75mcg/kg dose for patients <1month - Clarifications/corrections from the previous version in SOA, participant compliance, neurodevelopmental assessments, laboratory evaluations - Addition of Adverse Event of Special Interest (AESI) - Change in the dose continuation/stopping rules - Removed the requirement for dose escalation in the presence of an investigator or trained study staff
    01 Jun 2021
    Protocol version 3 (Global) released on 01 June 2021. Key Changes made: - The Schedule of Assessments, Table 1, has been revised to reduce the number of site visits to decrease participant and caregiver burden - Inclusion of participants with gestational age <36 weeks, with approval by the medical monitor - Update of blood volumes to be drawn from participants during the study - Clarification of timepoints for PK assessments
    14 Jan 2022
    Protocol version 5 (Global) released on 14 January 2022. Key changes made: - Added LTE 16 week repeating cycle period, LTE final study visit, and safety follow-up contact. - Revised to remove the option for participants to roll over into Study 800 and to instead add the Study 801 LTE. Participants will no longer roll over into Study 800; they will remain within Study 801 LTE or transition to commercially available drug or an expanded access program, as applicable - Endpoints of assessments using the Itch Reported Outcome Observer (ItchRO[Obs]) instrument - Clarification regarding the inclusion of preterm-born infants. - Details for procedures related to study medication interruption or participant withdrawal due to AEs or disease progression. - Disease-related questionnaire to the long-term follow up period - Updated blood volume requirements and priority of sample collection

    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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