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    Clinical Trial Results:
    A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously.

    Summary
    EudraCT number
    2013-003400-39
    Trial protocol
    GB   ES  
    Global end of trial date
    16 Oct 2017

    Results information
    Results version number
    v1(current)
    This version publication date
    02 May 2018
    First version publication date
    02 May 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    NGLU-CL02
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02324049
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals, Inc.
    Sponsor organisation address
    100 College Street, New Haven, United States, 06510
    Public contact
    European Clinical Trial Information, Alexion Europe SAS, +33 147100606, clinicaltrials.eu@alexion.com
    Scientific contact
    European Clinical Trial Information, Alexion Europe SAS, +33 147100606, clinicaltrials.eu@alexion.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    EMEA-001653-PIP01-14
    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
    03 Nov 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    16 Oct 2017
    Global end of trial reached?
    Yes
    Global end of trial date
    16 Oct 2017
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of the IV administration of SBC-103 in subjects with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) with evaluable signs or symptoms of developmental delay.
    Protection of trial subjects
    This study was conducted in accordance with International Conference on Harmonisation (ICH) Good Clinical Practice, and the principles of the Declaration of Helsinki, in addition to following the laws and regulations of the country or countries in which a study is conducted.
    Background therapy
    No background therapy used for this study.
    Evidence for comparator
    No comparator used.
    Actual start date of recruitment
    22 Jan 2015
    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
    Spain: 1
    Country: Number of subjects enrolled
    United Kingdom: 2
    Country: Number of subjects enrolled
    United States: 8
    Worldwide total number of subjects
    11
    EEA total number of subjects
    3
    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)
    11
    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
    Pediatric MPS IIIB patients, aged ≥ 2 to < 12 years old, were eligible for the study.

    Pre-assignment
    Screening details
    A definitive diagnosis of MPS IIIB, as determined by either documented deficiency in NAGLU enzyme activity ≤10% of the mean value in normal individuals at Screening OR documented functionally-relevant mutations in both alleles of the NAGLU gene based on historical or Screening laboratory results.

    Period 1
    Period 1 title
    Overall (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Not blinded
    Blinding implementation details
    Not a blinded study.

    Arms
    Arm title
    SBC-103
    Arm description
    Part A (Initial therapy): Patients were enrolled in 1 of 3 different dosing cohorts (0.3, 1.0, or 3.0 mg/kg), with patients enrolling in the lowest dosage first. Dosing occurred every other week, [QOW] for 24 weeks, followed by a ≥ 4 week treatment break. Part B: Dosing was escalated to the next higher dose that was considered safe (1.0 or 3.0 mg/kg QOW) for ≥ 8 weeks. Patients who received doses of 0.3 mg/kg in Part A were considered for a second dose escalation to 3.0 mg/kg at any time during Part B provided that they tolerated at least 2 doses of 1.0 mg/kg in Part B. Patients who received and tolerated at least 4 doses of SBC-103 QOW at 3.0 mg/kg were considered for participation in Part C. Part C: Were randomized, open-label assessment of SBC-103 at doses of 5.0 or 10.0 mg/kg administered IV. Dosing in Part C began at the 5.0 mg/kg dose level. The decision to began dosing in the first patient at 10.0 mg/kg was based on review of safety data at 5.0 mg/kg.
    Arm type
    Experimental

    Investigational medicinal product name
    SBC-103
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Intravenous SBC-103 (0.3, 1.0, 3.0, 5.0, 10.0 mg/kg).

    Number of subjects in period 1
    SBC-103
    Started
    11
    Completed
    11

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall
    Reporting group description
    -

    Reporting group values
    Overall Total
    Number of subjects
    11 11
    Age categorical
    Units: Subjects
        In utero
    0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0
        Newborns (0-27 days)
    0 0
        Infants and toddlers (28 days-23 months)
    0 0
        Children (2-11 years)
    11 11
        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
    The overall age at study entry ranged from 25 to 123 months.
    Units: months
        arithmetic mean (standard deviation)
    72.6 ± 38.39 -
    Gender categorical
    Units: Subjects
        Female
    4 4
        Male
    7 7
    Subject analysis sets

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    The Safety Analysis Set, defined as all patients for whom informed consent had been obtained, who have a confirmed diagnosis of MPS IIIB, and who have received any amount of SBC-103, was used to summarize all safety and tolerability data.

    Subject analysis sets values
    Safety Analysis Set
    Number of subjects
    11
    Age categorical
    Units: Subjects
        In utero
    0
        Preterm newborn infants (gestational age < 37 wks)
    0
        Newborns (0-27 days)
    0
        Infants and toddlers (28 days-23 months)
    0
        Children (2-11 years)
    11
        Adolescents (12-17 years)
    0
        Adults (18-64 years)
    0
        From 65-84 years
    0
        85 years and over
    0
    Age continuous
    The overall age at study entry ranged from 25 to 123 months.
    Units: months
        arithmetic mean (standard deviation)
    72.6 ± 38.39
    Gender categorical
    Units: Subjects
        Female
    4
        Male
    7

    End points

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    End points reporting groups
    Reporting group title
    SBC-103
    Reporting group description
    Part A (Initial therapy): Patients were enrolled in 1 of 3 different dosing cohorts (0.3, 1.0, or 3.0 mg/kg), with patients enrolling in the lowest dosage first. Dosing occurred every other week, [QOW] for 24 weeks, followed by a ≥ 4 week treatment break. Part B: Dosing was escalated to the next higher dose that was considered safe (1.0 or 3.0 mg/kg QOW) for ≥ 8 weeks. Patients who received doses of 0.3 mg/kg in Part A were considered for a second dose escalation to 3.0 mg/kg at any time during Part B provided that they tolerated at least 2 doses of 1.0 mg/kg in Part B. Patients who received and tolerated at least 4 doses of SBC-103 QOW at 3.0 mg/kg were considered for participation in Part C. Part C: Were randomized, open-label assessment of SBC-103 at doses of 5.0 or 10.0 mg/kg administered IV. Dosing in Part C began at the 5.0 mg/kg dose level. The decision to began dosing in the first patient at 10.0 mg/kg was based on review of safety data at 5.0 mg/kg.

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    The Safety Analysis Set, defined as all patients for whom informed consent had been obtained, who have a confirmed diagnosis of MPS IIIB, and who have received any amount of SBC-103, was used to summarize all safety and tolerability data.

    Primary: The primary objective of the study was to evaluate the safety and tolerability of intravenous (IV) administration of SBC-103 in subjectswith MPS III

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    End point title
    The primary objective of the study was to evaluate the safety and tolerability of intravenous (IV) administration of SBC-103 in subjectswith MPS III [1]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline to 142 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: No statistical analyses have been specified for this primary endpoint.
    End point values
    SBC-103
    Number of subjects analysed
    11
    Units: patients
    11
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline to week 142
    Adverse event reporting additional description
    Baseline to 142 weeks. The planned duration of treatment was approximately 164 weeks. Due to early termination of Study NGLU-CL02, the actual study duration was 142weeks.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    19
    Reporting groups
    Reporting group title
    Overall Trial
    Reporting group description
    11 patients enrolled across 4 clinical sites in 3 countries, 5 patients were randomly assigned to the SBC-103 5.0mg/kg – every other week (QOW) dose and 6 patients were assigned to the SBC-103 10.0 mg/kg – QOW dose. All 11 patients had completed Part A and Part B of the study.

    Serious adverse events
    Overall Trial
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 11 (27.27%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Vascular disorders
    Flushing
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    3 / 3
         deaths causally related to treatment / all
    0 / 0
    Investigations
    Blood pressure increased
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Cyanosis
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Hypoxia
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin and subcutaneous tissue disorders
    Urticaria
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    Swelling face
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Bacteraemia
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    1 / 3
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Staphylococcal bacteraemia
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Overall Trial
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    11 / 11 (100.00%)
    Vascular disorders
    Flushing
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    6
    Hyperaemia
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    2
    Hypertension
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    2
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    9 / 11 (81.82%)
         occurrences all number
    21
    Catheter site erythema
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    2
    Catheter site rash
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Catheter site swelling
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Influenza like illness
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Local swelling
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Pain
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Psychiatric disorders
    Hypervigilance
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    2
    Insomnia
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    2
    Aggression
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Agitation
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Anxiety
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Head injury
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    3
    Fall
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    2
    Infusion related reaction
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    2
    Anaesthetic complication neurological
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Contusion
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Eye contusion
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Eyelid injury
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Foreign body
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Lip injury
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Mouth injury
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Periorbital haemorrhage
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Investigations
    Cardiac murmur
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    2
    Body temperature increased
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    CSF protein increased
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Electrocardiogram Q wave abnormal
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Liver palpable
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Mean platelet volume increased
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Oxygen saturation decreased
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Serum ferritin decreased
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Cardiac disorders
    Left ventricular hypertrophy
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Sinus bradycardia
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Tachycardia
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Congenital, familial and genetic disorders
    Dysmorphism
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    5 / 11 (45.45%)
         occurrences all number
    10
    Rhinorrhoea
         subjects affected / exposed
    3 / 11 (27.27%)
         occurrences all number
    4
    Nasal congestion
         subjects affected / exposed
    3 / 11 (27.27%)
         occurrences all number
    3
    Wheezing
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    3
    Nasal obstruction
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    2
    Asthma
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Bronchial secretion retention
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Oropharyngeal pain
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    3
    Lymphadenopathy
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Hyperreflexia
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Hypertonia
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Lethargy
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Psychomotor hyperactivity
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Seizure
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Speech disorder
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Syncope
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Eye disorders
    Mydriasis
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    2
    Eye swelling
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Ocular hyperaemia
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Ear and labyrinth disorders
    Deafness
         subjects affected / exposed
    4 / 11 (36.36%)
         occurrences all number
    4
    Ear pain
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    4
    Deafness bilateral
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Hypoacusis
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Middle ear effusion
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    7 / 11 (63.64%)
         occurrences all number
    26
    Diarrhoea
         subjects affected / exposed
    6 / 11 (54.55%)
         occurrences all number
    15
    Salivary hypersecretion
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    2
    Constipation
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    2
    Breath odour
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Eructation
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Lip swelling
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Nausea
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Pancreatic enlargement
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Swollen tongue
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Renal and urinary disorders
    Proteinuria
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    2
    Product issues
    Device occlusion
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    4 / 11 (36.36%)
         occurrences all number
    6
    Dermatitis diaper
         subjects affected / exposed
    4 / 11 (36.36%)
         occurrences all number
    4
    Erythema
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    2
    Rash erythematous
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    2
    Dermatitis allergic
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Dermatitis contact
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Dry skin
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Eczema
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Miliaria
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Seborrhoeic dermatitis
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Skin discolouration
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Skin plaque
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Swelling face
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Papule
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Coccydynia
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Muscle mass
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Musculoskeletal pain
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Scoliosis
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Endocrine disorders
    Precocious puberty
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Iron deficiency
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    5 / 11 (45.45%)
         occurrences all number
    16
    Upper respiratory tract infection
         subjects affected / exposed
    4 / 11 (36.36%)
         occurrences all number
    4
    Lower respiratory tract infection
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    6
    Rhinitis
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    3
    Gastroenteritis viral
         subjects affected / exposed
    2 / 11 (18.18%)
         occurrences all number
    2
    Ear infection
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    2
    Fungal skin infection
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    2
    Hordeolum
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    2
    Pharyngitis streptococcal
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    2
    Candida nappy rash
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Conjunctivitis
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Eye infection
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Eye infection bacterial
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Folliculitis
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Fungal infection
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Influenza
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Localised infection
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Oral candidiasis
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Oral herpes
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Otitis externa
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Otitis media
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Paronychia
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Respiratory tract infection viral
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Rotavirus infection
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Tonsillitis
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1
    Viral infection
         subjects affected / exposed
    1 / 11 (9.09%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    10 Apr 2014
    Amendment #1 (10Apr2014): Revised inclusion/exclusion criteria for further clarification on assessment questionnaires, adjustments to blood samples obtained.
    05 Jun 2014
    Amendment # 2 (05Jun2014):Added clarification to the stopping rules and included language for patients to receive central line placement for general anesthesia for specified protocol assessments.
    04 Nov 2014
    Amendment #3 (04Nov2014): Changes to the study design include: removal of PET imaging, inclusion of additional nonclinical data to support data design, study drug administration regimen changed, and age range of patients changed.
    26 Dec 2014
    Amendment #4 (26Dec2014): Added additional safety monitoring measurements, revised the required quality of life assessments, clarification of the types of lab assessments required and added dose pausing rules.
    15 Feb 2015
    Amendment #5 (15Feb2015): Additional subject enrollment into a cohort-based upon screening, and refined the timing of multiple assessments.
    11 Dec 2015
    Amendment #6 (11Dec2015): Addition of Part C which included 2 additional doses.

    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.
    Study NGLU-CL02 was terminated early, as part of the early termination of the SBC-103 program. The Sponsor’s decision to terminate the SBC-103 program was reached after review of the data from all interventional clinical studies of SBC-103.
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