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    Clinical Trial Results:
    Double-blind, randomized, placebo-controlled, Phase II/III trial on the efficacy and tolerability of treatment with budesonide oral suspension vs. placebo in children and adolescents with eosinophilic esophagitis

    Summary
    EudraCT number
    		 2017-003737-29
    Trial protocol
    		 DE   ES   PT   NL   GR   GB  
    Global end of trial date
    07 Aug 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    05 Apr 2025
    First version publication date
    05 Apr 2025
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    BUU-5/EEA
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 PEDEOS-1: Acronym
    Sponsors
    Sponsor organisation name
    Dr. Falk Pharma Gmbh
    Sponsor organisation address
    Leinenweberstr. 5, Freiburg Im Breisgau, Germany, 79100
    Public contact
    Dept. of Clinic. Res. & Development, Dr Falk Pharma GmbH, +49 76115140, zentrale@drfalkpharma.de, Dr Falk Pharma GmbH, 0049 76115140,
    Scientific contact
    Dept. of Clinic. Res. & Development, Dr Falk Pharma GmbH, +49 76115140, zentrale@drfalkpharma.de, Dr Falk Pharma GmbH, 0049 76115140,
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    18 Sep 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    30 Sep 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    07 Aug 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Double-blind phase: To prove superior efficacy of budesonide oral suspension compared to placebo in children and adolescents with eosinophilic esophagitis (EoE)
    Protection of trial subjects
    Prior to recruitment of patients, all relevant documents of the clinical study were submitted and approved by the Independent Ethics Committees (IECs) responsible for the participating investigators. Written consent documents embodied the elements of informed consent as described in the Declaration of Helsinki, the ICH Guidelines for Good Clinical Practice (GCP) and were in accordance with all applicable laws and regulations. The informed consent form and patient information sheet described the planned and permitted uses, transfers and disclosures of the patient’s personal data and personal health information for purposes of conducting the study. The informed consent form and the patient information sheet further explained the nature of the study, its objectives and potential risks and benefits as well as the date informed consent was given. Before being enrolled in the clinical trial, every patient was informed that participation in this trial was voluntary and that he/she could withdraw from the study at any time without giving a reason and without having to fear any loss in his/her medical care. The patient’s consent was obtained in writing before the start of the study. By signing the informed consent, the patient declared that he/she was participating voluntarily and intended to follow the study protocol instructions and the instructions of the investigator and to answer the questions asked during the course of the trial. For endoscopy and biopsy sampling to be performed for confirmation of diagnosis of eosinophilic esophagitis by the central pathologist, the patients received the standard preparation for sedation during the endoscopy as routinely performed at the study sites.
    Background therapy
    		 No concomitant background therapy, except stable diets and/or stable treatment with protonpumpinhibitors was allowed during the trial.
    Evidence for comparator
    		 Using a placebo arm in this clinical trial was ethically justified as there were compelling and scientifically sound methodological reasons for the use of a placebo control in this trial, since there were no comparator products with a marketing authorization for the treatment of EoE available. Moreover, the use of a placebo group was also justified, as it allowed to control for all other potential influences on the actual or apparent course of the disease other than those arising from the pharmacological action of budesonide (including but not limited to influences such as, spontaneous change in the disease, subject and investigator expectations, the effect of participating in this trial, or subjective elements of diagnosis or assessments), as stated in the “ICH Topic E10: Note for guidance on choice of control group in clinical trials” (CPMP/ICH/364/96).
    Actual start date of recruitment
    03 Sep 2019
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Netherlands: 8
    Country: Number of subjects enrolled
    Portugal: 12
    Country: Number of subjects enrolled
    Spain: 36
    Country: Number of subjects enrolled
    United Kingdom: 5
    Country: Number of subjects enrolled
    Greece: 8
    Country: Number of subjects enrolled
    Türkiye: 1
    Country: Number of subjects enrolled
    Australia: 4
    Country: Number of subjects enrolled
    Israel: 2
    Worldwide total number of subjects
    76
    EEA total number of subjects
    64
    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)
    35
    Adolescents (12-17 years)
    41
    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
    		 In total, 15 centers randomized patients: 1 center in Australia, 2 centers in Greece, 2 centers in Israel, 3 centers in Portugal, 3 centers in Spain, 2 centers in the Netherlands, 1 center in Turkey and 1 center in the United Kingdom.

    Pre-assignment
    Screening details
    		 105 patients were screened to assess the In-/Exclusion criteria. Of them, 76 patients were randomized and treated with budesonide or placebo.

    Period 1
    Period 1 title
    Double-blind treatment phase (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
    At the start of the DB treatment phase, patients were assigned to one of the treatment groups via a central stratified randomization procedure using a 1:1:1 balance. To guarantee the double-blinding, the study was conducted using placebo oral suspension identical in appearance and taste to active substance oral suspension. The patients’ allocation to one of the treatment groups by means of a computer-generated randomization list.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Test arm high dose
    Arm description
    		 Budesonide oral suspension 0.5 mg/1 mg BID (2-11 /12-17 years)
    Arm type
    		 Experimental

    Investigational medicinal product name
    Budesonide oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Budesonide oral suspension 0.5 mg/1 mg BID (2-11 /12-17 years)

    Arm title
    		 Test arm low dose
    Arm description
    		 Budesonide oral suspension 0.5 mg/1 mg OD (2-11 /12-17 years)
    Arm type
    		 Experimental

    Investigational medicinal product name
    Budesonide oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Budesonide oral suspension 0.5 mg/1 mg OD (2-11 /12-17 years)

    Arm title
    		 Placebo
    Arm description
    		 Placebo arm
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo oral suspension
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo oral suspension

    Number of subjects in period 1
    		Test arm high dose Test arm low dose Placebo
    Started
    26
    26
    24
    Completed
    26
    26
    23
    Not completed
    0
    0
    1
         Adverse event, non-fatal
    -
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Test arm high dose
    Reporting group description
    		 Budesonide oral suspension 0.5 mg/1 mg BID (2-11 /12-17 years)

    Reporting group title
    Test arm low dose
    Reporting group description
    		 Budesonide oral suspension 0.5 mg/1 mg OD (2-11 /12-17 years)

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo arm

    Reporting group values
    		 Test arm high dose Test arm low dose Placebo Total
    Number of subjects
    		 26 26 24 76
    Age categorical
    Stratum I: Age 2 to 11 years Stratum II: Age 12 to <18 years
    Units: Subjects
        Children (2-11 years)
    		 12 12 11 35
        Adolescents (12-17 years)
    		 14 14 13 41
    Age continuous
    Stratum I + II: 2 - < 18 years
    Units: years
        arithmetic mean (standard deviation)
    		 11.2 ( 4.5 ) 12.3 ( 3.8 ) 11.7 ( 3.8 ) -
    Gender categorical
    Units: Subjects
        Female
    		 3 8 6 17
        Male
    		 23 18 18 59
    Age categorial
    Units: Subjects
        Stratum I: age 2 to 11 years
    		 12 12 11 35
        Stratum II: age 12 to <18 years
    		 14 14 13 41
    Ethnic Group
    Units: Subjects
        White
    		 23 26 24 73
        Asian
    		 2 0 0 2
        Other
    		 1 0 0 1
    History of allergic disease
    Units: Subjects
        yes
    		 22 22 22 66
        no
    		 4 4 2 10
    Previous PPI EoE treatment
    Units: Subjects
        yes
    		 21 21 22 64
        no
    		 5 5 2 12
    Duration since first symptoms
    Units: years
        arithmetic mean (standard deviation)
    		 4.2 ( 3.67 ) 6.0 ( 3.80 ) 5.0 ( 2.51 ) -
    Duration since first diagnosis
    Units: years
        arithmetic mean (standard deviation)
    		 2.1 ( 2.45 ) 3.7 ( 2.83 ) 3.1 ( 2.87 ) -
    Overall peak eos/mm2 hpf
    Overall peak eosinophil count (eos)/mm2 high power field (hpf) derived from biopsies at screening (proximal, mid, and distal esophageal segment).
    Units: eos/mm2 hpf
        arithmetic mean (standard deviation)
    		 237.7 ( 123.1 ) 245.9 ( 108.7 ) 238.6 ( 122.1 ) -
    Total Modified Endoscopic Reference Score (EREFS; range: 0-9)
    Worst case assessment from all parts of the esophagus. Lower values reflect lower total endoscopic disease activity.
    Units: points
        arithmetic mean (standard deviation)
    		 3.2 ( 1.0 ) 3.6 ( 1.4 ) 3.2 ( 1.8 ) -
    'Inflammatory signs' subscore - Modified Endoscopic Reference Score (EREFS; range: 0-4)
    Worst case assessment from all parts of the esophagus. Lower values reflect lower endoscopic inflammatory disease activity.
    Units: points
        arithmetic mean (standard deviation)
    		 2.8 ( 1.1 ) 2.9 ( 1.0 ) 2.5 ( 1.1 ) -
    'Fibrotic signs' subscore - Modified Endoscopic Reference Score (EREFS, range: 0-4) at DB week 12
    Worst case assessment from all parts of the esophagus. Lower values reflect lower endoscopic fibrotic disease activity.
    Units: points
        arithmetic mean (standard deviation)
    		 0.3 ( 0.6 ) 0.6 ( 0.9 ) 0.5 ( 0.7 ) -
    Dysphagia Numerical Rating Scale [NRS] (0-10)
    0 = no troubles to swallow 10 = most severe troubles to swallow
    Units: points
        arithmetic mean (standard deviation)
    		 4.7 ( 2.5 ) 5.2 ( 2.4 ) 5.0 ( 2.2 ) -
    Pain during swallowing NRS (0-10)
    0 = no pain during swallowing 10 = most severe pain during swallowing
    Units: points
        arithmetic mean (standard deviation)
    		 3.0 ( 2.8 ) 3.1 ( 2.8 ) 2.0 ( 2.6 ) -
    Patient’s Global Assessment of EoE activity (NRS 0-10)
    0 = no symptoms 10 = most severe symptoms
    Units: points
        arithmetic mean (standard deviation)
    		 5.8 ( 1.3 ) 6.3 ( 1.4 ) 6.2 ( 1.4 ) -
    Physician’s Global Assessment of EoE activity (NRS 0-10)
    considered all findings concerning the severity of the patient’s EoE (clinical, endoscopic, histologic) 0 = inactive EoE 10 = most active EoE
    Units: points
        arithmetic mean (standard deviation)
    		 6.8 ( 1.2 ) 6.1 ( 1.3 ) 6.4 ( 1.4 ) -

    End points

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    End points reporting groups
    Reporting group title
    Test arm high dose
    Reporting group description
    		 Budesonide oral suspension 0.5 mg/1 mg BID (2-11 /12-17 years)

    Reporting group title
    Test arm low dose
    Reporting group description
    		 Budesonide oral suspension 0.5 mg/1 mg OD (2-11 /12-17 years)

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo arm

    Primary: Rate of patients with pathological remission and clinical response at DB week 12 (LOCF)

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    End point title
    		 Rate of patients with pathological remission and clinical response at DB week 12 (LOCF)
    End point description
    Rate of patients with pathological remission and clinical response at DB week 12 (LOCF) defined as fulfilling both criteria: - Histological remission, i.e., peak of <16 eos/mm2 hpf at DB week 12 (LOCF), AND - Clinical response defined as: Stratum I: Age 2 to 11 years at DB V1: ≥ 30% drop in the total score of PEESS Version 2.0 – parent report for children and teens (ages 2-18) from baseline to DB week 12 (LOCF), Stratum II: Age 12 to <18 years at DB V1: ≥ 30% drop in the total score of PEESS Version 2.0 – children and teens report (ages 8-18) from baseline to DB week 12 (LOCF).
    End point type
    Primary
    End point timeframe
    DB week 12
    End point values
    		 Test arm high dose Test arm low dose Placebo
    Number of subjects analysed
    26
    26
    24
    Units: Patients
        Yes
    18
    12
    0
        No
    8
    14
    23
    Statistical analysis title
    		 Primary: BUU-L vs. Placebo
    Statistical analysis description
    The rate of patients with histological remission (peak eos < 16 eos/mm2 hpf [i.e. <5 eos/hpf]) and clinical response (≥30% decrease in the total score of PEESS v2.0) at DB Week 12 (LOCF)
    Comparison groups
    Placebo v Test arm low dose
    Number of subjects included in analysis
    50
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 t-test, 1-sided
    Parameter type
    		 Risk difference (RD)
    Point estimate
    46.2
    Confidence interval
         level
    		 98.75%
         sides
    2-sided
         lower limit
    		 21.73
         upper limit
    		 70.57
    Statistical analysis title
    		 Primary: BUU-H vs. Placebo
    Comparison groups
    Test arm high dose v Placebo
    Number of subjects included in analysis
    50
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 t-test, 1-sided
    Parameter type
    		 Risk difference (RD)
    Point estimate
    69.2
    Confidence interval
         level
    		 98.75%
         sides
    2-sided
         lower limit
    		 46.62
         upper limit
    		 91.84

    Secondary: Rate of patients with histological remission, defined as a peak of <16 eos/mm2 hpf at DB week 12 (LOCF),

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    End point title
    		 Rate of patients with histological remission, defined as a peak of <16 eos/mm2 hpf at DB week 12 (LOCF),
    End point description
    End point type
    Secondary
    End point timeframe
    DB week 12 (LOCF)
    End point values
    		 Test arm high dose Test arm low dose Placebo
    Number of subjects analysed
    26
    26
    24
    Units: Patients
        Yes
    23
    16
    0
        No
    3
    10
    24
    Statistical analysis title
    		 Primary secondary: BUU-L vs. Placebo
    Comparison groups
    Test arm low dose v Placebo
    Number of subjects included in analysis
    50
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 t-test, 1-sided
    Parameter type
    		 Risk difference (RD)
    Point estimate
    61.5
    Confidence interval
         level
    		 98.75%
         sides
    2-sided
         lower limit
    		 37.7
         upper limit
    		 85.4
    Statistical analysis title
    		 Primary secondary: BUU-H vs. Placebo
    Comparison groups
    Test arm high dose v Placebo
    Number of subjects included in analysis
    50
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 t-test, 1-sided
    Parameter type
    		 Risk difference (RD)
    Point estimate
    72.8
    Confidence interval
         level
    		 98.75%
         sides
    2-sided
         lower limit
    		 72.8
         upper limit
    		 100

    Secondary: Change in the peak eos/mm2 hpf from screening to DB week 12 (LOCF)

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    End point title
    		 Change in the peak eos/mm2 hpf from screening to DB week 12 (LOCF)
    End point description
    End point type
    Secondary
    End point timeframe
    From Screening to DB week 12 (LOCF)
    End point values
    		 Test arm high dose Test arm low dose Placebo
    Number of subjects analysed
    25
    26
    22
    Units: eos/mm2
        least squares mean (standard deviation)
    -230.3 ( 25.5 )
    -165.7 ( 25 )
    -11.4 ( 27.2 )
    Statistical analysis title
    		 Secondary Second endpoint: BUU-L vs. Placebo
    Comparison groups
    Test arm low dose v Placebo
    Number of subjects included in analysis
    48
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 t-test, 1-sided
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -154.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -228
         upper limit
    		 -80.6
    Variability estimate
    Standard deviation
    Dispersion value
    36.9
    Statistical analysis title
    		 Secondary Second endpoint: BUU-H vs. Placebo
    Comparison groups
    Test arm high dose v Placebo
    Number of subjects included in analysis
    47
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 t-test, 1-sided
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -218.9
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -293.3
         upper limit
    		 -144.5
    Variability estimate
    Standard deviation
    Dispersion value
    37.3

    Secondary: Rate of patients with clinico-pathological remission

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    End point title
    		 Rate of patients with clinico-pathological remission
    End point description
    Rate of patients with clinico-pathological remission defined as: o Histological remission, i.e., peak of <16 eos/mm2 hpf at DB week 12 (LOCF), and o Clinical remission (i.e., no or only minimal problems) defined as clinical response (≥ 30% drop in the total score of PEESS Version 2.0 from baseline to DB week 12 [LOCF]) AND Stratum I: Age 2 to 11 years at DB V1: PEESS Version 2.0 – parent report for children and teens (ages 2-18) • ≤4 points in each of the subdomains GERD/nausea-vomiting/pain, and • ≤7 points in the subdomain dysphagia, or • ≤5 points in the total score and ≥2 subdomains with a drop of at least 50% compared to baseline at DB week 12 (LOCF), Stratum II: Age 12 to <18 years at DB V1: PEESS Version 2.0 – children and teens report (ages 8-18) • ≤4 points in each of the subdomains GERD/nausea-vomiting/pain, and • ≤7 points in the subdomain dysphagia, or • ≤5 points in the total score and ≥2 subdomains with a drop of at least 50% compared to baseline
    End point type
    Secondary
    End point timeframe
    DB weel 12 (LOCF).
    End point values
    		 Test arm high dose Test arm low dose Placebo
    Number of subjects analysed
    26
    26
    24
    Units: Patients
        Yes
    9
    7
    0
        No
    17
    19
    24
    Statistical analysis title
    		 Third secondary endpoint: Primary: BUU-L vs. Place
    Comparison groups
    Test arm low dose v Placebo
    Number of subjects included in analysis
    50
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0066
    Method
    		 t-test, 1-sided
    Parameter type
    		 Risk difference (RD)
    Point estimate
    26.9
    Confidence interval
         level
    		 98.75%
         sides
    2-sided
         lower limit
    		 5.2
         upper limit
    		 48.7
    Statistical analysis title
    		 Third secondary endpoint: Primary: BUU-H vs. Place
    Comparison groups
    Test arm high dose v Placebo
    Number of subjects included in analysis
    50
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0012
    Method
    		 t-test, 1-sided
    Parameter type
    		 Risk difference (RD)
    Point estimate
    34.6
    Confidence interval
         level
    		 98.75%
         sides
    2-sided
         lower limit
    		 11.3
         upper limit
    		 57.9

    Secondary: Rate of patients with clinical remission (i.e., no or only minimal problems) defined as above (clinical remission component of the endpoint clinico-pathological remission) at DB week 12 (LOCF)

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    End point title
    		 Rate of patients with clinical remission (i.e., no or only minimal problems) defined as above (clinical remission component of the endpoint clinico-pathological remission) at DB week 12 (LOCF)
    End point description
    End point type
    Secondary
    End point timeframe
    DB week 12
    End point values
    		 Test arm high dose Test arm low dose Placebo
    Number of subjects analysed
    26
    26
    24
    Units: Patients
        Yes
    10
    11
    12
        No
    16
    15
    12
    Statistical analysis title
    		 Fourth secondary endpoint: BUU-L vs. Placebo
    Comparison groups
    Test arm low dose v Placebo
    Number of subjects included in analysis
    50
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.7964
    Method
    		 t-test, 1-sided
    Parameter type
    		 Risk difference (RD)
    Point estimate
    -7.7
    Confidence interval
         level
    		 98.75%
         sides
    2-sided
         lower limit
    		 -42.8
         upper limit
    		 27.5
    Statistical analysis title
    		 Fourth secondary endpoint: BUU-H vs. Placebo
    Comparison groups
    Test arm high dose v Placebo
    Number of subjects included in analysis
    50
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.8657
    Method
    		 t-test, 1-sided
    Parameter type
    		 Risk difference (RD)
    Point estimate
    -11.5
    Confidence interval
         level
    		 98.75%
         sides
    2-sided
         lower limit
    		 -46.4
         upper limit
    		 23.4

    Secondary: Rate of patients with clinical response at DB week 12 (LOCF)

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    End point title
    		 Rate of patients with clinical response at DB week 12 (LOCF)
    End point description
    Rate of patients with clinical response at DB week 12 (LOCF), defined as Stratum I: Age 2 to 11 years at DB V1: ≥ 30% drop in the total score of PEESS Version 2.0 – parent report for children and teens (ages 2-18) from baseline to DB week 12 (LOCF), Stratum II: Age 12 to <18 years at DB V1: ≥ 30% drop in the total score of PEESS Version 2.0 – children and teens report (ages 8-18) from baseline to DB week 12 (LOCF) • In the subgroup of patients with ≥ 4 points in NRS for dysphagia on the day of the baseline visit (only patients of Stratum II: Age 12 to <18 years at DB V1), the rate of patients with resolution of dysphagia symptom (i.e., no or only minimal problems). Resolution of dysphagia symptom is defined as a severity of ≤2 points on 0 to 10-point (0-10) NRS on each day in the week prior to DB week 12 (LOCF).
    End point type
    Secondary
    End point timeframe
    DB week 12
    End point values
    		 Test arm high dose Test arm low dose Placebo
    Number of subjects analysed
    26
    26
    24
    Units: Patients
        Yes
    20
    18
    17
        No
    6
    8
    7
    Statistical analysis title
    		 Fifth secondary endpoint: BUU-L vs. Placebo
    Comparison groups
    Test arm low dose v Placebo
    Number of subjects included in analysis
    50
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.6663
    Method
    		 t-test, 1-sided
    Parameter type
    		 Risk difference (RD)
    Point estimate
    -1.6
    Confidence interval
         level
    		 98.75%
         sides
    2-sided
         lower limit
    		 -34
         upper limit
    		 30.8
    Statistical analysis title
    		 Fifth secondary endpoint: BUU-H vs. Placebo
    Comparison groups
    Test arm high dose v Placebo
    Number of subjects included in analysis
    50
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.4328
    Method
    		 t-test, 1-sided
    Parameter type
    		 Risk difference (RD)
    Point estimate
    6.1
    Confidence interval
         level
    		 98.75%
         sides
    2-sided
         lower limit
    		 -24.9
         upper limit
    		 37.1

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    12 weeks
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    Test arm high dose
    Reporting group description
    		 Budesonide oral suspension 0.5 mg/1 mg BID (2-11 /12-17 years)

    Reporting group title
    Test arm low dose
    Reporting group description
    		 Budesonide oral suspension 0.5 mg/1 mg OD (2-11 /12-17 years)

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo arm

    Serious adverse events
    		 Test arm high dose Test arm low dose Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 26 (7.69%)
    1 / 26 (3.85%)
    1 / 24 (4.17%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Surgical and medical procedures
    Arthroscopy
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 26 (3.85%)
    0 / 26 (0.00%)
    0 / 24 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Seizure
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 26 (3.85%)
    0 / 24 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    1 / 26 (3.85%)
    0 / 26 (0.00%)
    0 / 24 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Neck mass
    alternative assessment type: Non-systematic
         subjects affected / exposed
    0 / 26 (0.00%)
    0 / 26 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Test arm high dose Test arm low dose Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    22 / 26 (84.62%)
    19 / 26 (73.08%)
    20 / 24 (83.33%)
    Vascular disorders
    Vascular disorders
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 26 (3.85%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    General disorders and administration site conditions
    General disorders and administration site conditions
         subjects affected / exposed
    5 / 26 (19.23%)
    4 / 26 (15.38%)
    4 / 24 (16.67%)
         occurrences all number
    6
    7
    6
    Immune system disorders
    Immune system disorder
         subjects affected / exposed
    1 / 26 (3.85%)
    1 / 26 (3.85%)
    1 / 24 (4.17%)
         occurrences all number
    1
    1
    1
    Reproductive system and breast disorders
    Reproductive system and breast disorders
         subjects affected / exposed
    1 / 26 (3.85%)
    2 / 26 (7.69%)
    0 / 24 (0.00%)
         occurrences all number
    1
    4
    0
    Respiratory, thoracic and mediastinal disorders
    Respiratory, thoracic and mediastinal disorders
         subjects affected / exposed
    7 / 26 (26.92%)
    4 / 26 (15.38%)
    4 / 24 (16.67%)
         occurrences all number
    9
    5
    8
    Psychiatric disorders
    Psychiatric disorder
         subjects affected / exposed
    2 / 26 (7.69%)
    0 / 26 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    2
    0
    0
    Investigations
    Investigations
         subjects affected / exposed
    2 / 26 (7.69%)
    1 / 26 (3.85%)
    1 / 24 (4.17%)
         occurrences all number
    3
    1
    2
    Injury, poisoning and procedural complications
    Injury, poisoning and procedural complications
         subjects affected / exposed
    4 / 26 (15.38%)
    2 / 26 (7.69%)
    1 / 24 (4.17%)
         occurrences all number
    7
    3
    2
    Nervous system disorders
    Nervous system disorders
         subjects affected / exposed
    5 / 26 (19.23%)
    6 / 26 (23.08%)
    4 / 24 (16.67%)
         occurrences all number
    18
    22
    7
    Ear and labyrinth disorders
    Ear and labyrinth disorders
         subjects affected / exposed
    1 / 26 (3.85%)
    0 / 26 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    1
    Eye disorders
    Eye disorders
         subjects affected / exposed
    0 / 26 (0.00%)
    1 / 26 (3.85%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    Gastrointestinal disorders
    Gastrointestinal disorder
         subjects affected / exposed
    11 / 26 (42.31%)
    10 / 26 (38.46%)
    10 / 24 (41.67%)
         occurrences all number
    22
    21
    15
    Skin and subcutaneous tissue disorders
    Skin and subcutaneous tissue disorders
         subjects affected / exposed
    2 / 26 (7.69%)
    3 / 26 (11.54%)
    1 / 24 (4.17%)
         occurrences all number
    2
    3
    1
    Renal and urinary disorders
    Renal and urinary disorders
         subjects affected / exposed
    1 / 26 (3.85%)
    0 / 26 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Musculoskeletal and connective tissue disorders
         subjects affected / exposed
    2 / 26 (7.69%)
    1 / 26 (3.85%)
    3 / 24 (12.50%)
         occurrences all number
    2
    1
    3
    Infections and infestations
    Infections and infestations
         subjects affected / exposed
    12 / 26 (46.15%)
    9 / 26 (34.62%)
    10 / 24 (41.67%)
         occurrences all number
    20
    14
    19
    Metabolism and nutrition disorders
    Metabolism and nutrition disorders
         subjects affected / exposed
    3 / 26 (11.54%)
    3 / 26 (11.54%)
    0 / 24 (0.00%)
         occurrences all number
    3
    3
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    16 Apr 2019
    1) It was specified that patients with a Helicobacter pylori associated gastritis shall not be randomized into the study. 2 ) An ACTH test has been integrated as mandatory test to be performed at screening, and of double-blind phase and end of treatment (end of tapering phase). 3) To assess patient’s acceptance of the study drug, an objective has been added, relating to analysis of newly added questionnaire asking for patient’s acceptance of study medication related to taste and handling. 4) To incorporate assessment and analyses of height, head circumference and pubertal stage the assessments, an additional safety endpoint was added. Additionally growth retardation has been specified as adverse event of special interest. 5) To specify the study population regarding steroid treatment versus potential PPI or dietary treatment, inclusion and exclusion criteria have been modified and added. 6) To specify the study population regarding severity of disease and immediate treatment need, inclusion and exclusion criteria have been modified and added.
    22 Jul 2019
    1) To specify the study population regarding steroid treatment versus potential PPI or dietary treatment, inclusion and exclusion criteria have been modified and added. 2) Inclusion criteria no. 10 has been amended concerning pregnancy and birth control measures. 3) It was specified that patients with a Helicobacter pylori associated gastritis shall not be randomized into the study. 4) To specify the study population regarding severity of disease and immediate treatment need, exclusion criteria have been modified and added. 5) An ACTH test has been integrated as mandatory test to be performed at screening, end of double-blind phase and end of treatment (end of tapering phase). 6) To assess patient’s acceptance of the study drug, an objective has been added, relating to analysis of newly added questionnaire asking for patient’s acceptance of study medication related to taste and handling. 7) To incorporate assessment and analyses of height, head circumference and pubertal stage the assessments, an additional safety endpoint was added. Additionally growth retardation has been specified as adverse event of special interest.

    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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