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

    Summary
    EudraCT number
    2015-001983-20
    Trial protocol
    GB  
    Global end of trial date
    18 Aug 2017

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

    Trial information

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    Trial identification
    Sponsor protocol code
    NGLU-CL01-T
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02618512
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals
    Sponsor organisation address
    100 College Street, New Haven, United States, 06510
    Public contact
    European Clinical Trial Information, Alexion Europe SAS, +33 1 47 10 06 06, ClinicalTrials.EU@alexion.com
    Scientific contact
    European Clinical Trial Information, Alexion Europe SAS, +33 1 47 10 06 06, ClinicalTrials.EU@alexion.com
    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
    31 Oct 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    18 Aug 2017
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Aug 2017
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of intravenous administration of SBC-103 in previously studied, SBC-103 treatment naïve subjects with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) who participated in the NGLU-CL01 study.
    Protection of trial subjects
    NOT APPLICABLE
    Background therapy
    NOT APPLICABLE
    Evidence for comparator
    Comparator was not used.
    Actual start date of recruitment
    17 Sep 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
    United Kingdom: 3
    Worldwide total number of subjects
    3
    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)
    3
    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
    This study (NGLU-CL01-T) was conducted in MPS IIIB patients who participated in the NGLU-CL01 study, a non-interventional study that evaluated structural brain abnormalities and BBB integrity by MRI and CSF-AI. All subjects enrolled in the NGLU-CL01 study were ≥ 5 years of age and had a definitive diagnosis of MPS IIIB.

    Pre-assignment
    Screening details
    Participants from NGU-CL01 Study were eligible for enrollment in NGLU-CL01-T. Subjects who met all of the inclusion criteria and none of the exclusion criteria were eligible to participate in this study

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

    Arms
    Arm title
    SBC-103
    Arm description
    Subjects were treated with 1 mg/kg IV every other week for a minimum of 12 weeks. After evaluation of 12 weeks of safety and tolerability data, subjects were dose-escalated to 3 mg/kg every other week.
    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
    Subjects were treated with 1 mg/kg IV for a minimum of 12 weeks. After evaluation of 12 weeks safety and tolerability data the individual subject dose were increased to 3 mg/kg every other week up to 3 years (156 weeks). The planned study duration was 156 weeks. Due to the early study termination, the actual study duration was 96 weeks.

    Number of subjects in period 1
    SBC-103
    Started
    3
    Completed
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Trial (overall period)
    Reporting group description
    -

    Reporting group values
    Overall Trial (overall period) Total
    Number of subjects
    3 3
    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)
    3 3
        Adolescents (12-17 years)
    0 0
        Adults (18-64 years)
    0 0
        From 65-84 years
    0 0
        85 years and over
    0 0
    Gender categorical
    Units: Subjects
        Female
    1 1
        Male
    2 2

    End points

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    End points reporting groups
    Reporting group title
    SBC-103
    Reporting group description
    Subjects were treated with 1 mg/kg IV every other week for a minimum of 12 weeks. After evaluation of 12 weeks of safety and tolerability data, subjects were dose-escalated to 3 mg/kg every other week.

    Primary: The primary endpoint of this study is safety and tolerability of SBC-103 in subjects with MPS IIIB

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    End point title
    The primary endpoint of this study is safety and tolerability of SBC-103 in subjects with MPS IIIB [1]
    End point description
    Measured by number of patients reporting adverse events.
    End point type
    Primary
    End point timeframe
    Baseline to 96 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 comparative analysis was done. 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.
    End point values
    SBC-103
    Number of subjects analysed
    3
    Units: patients
    3
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    2 Years
    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
    -

    Serious adverse events
    Overall Trial
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 3 (66.67%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Injury, poisoning and procedural complications
    Wound dehiscence
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Surgical and medical procedures
    Catheter placement
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin and subcutaneous tissue disorders
    Erythema multiforme
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Device related infection
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    Overall Trial
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 3 (100.00%)
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    5
    Catheter site pain
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Gait disturbance
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Pain
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Complication associated with device
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Excessive granulation tissue
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Fatigue
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Peripheral swelling
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Psychiatric disorders
    Restlessness
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    4
    Anxiety
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    3
    Sleep disorder
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2
    Abnormal behaviour
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Breath holding
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Emotional distress
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Premenstrual pain
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    3 / 3 (100.00%)
         occurrences all number
    8
    Procedural pain
         subjects affected / exposed
    3 / 3 (100.00%)
         occurrences all number
    4
    Contusion
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Post-traumatic pain
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Scratch
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Investigations
    Body temperature increased
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Cardiac disorders
    Bradycardia
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Epistaxis
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Choking
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Cough
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Rhinorrhoea
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2
    Psychomotor hyperactivity
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2
    Dyskinesia
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Seizure
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Motor dysfunction
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Petit mal epilepsy
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    3 / 3 (100.00%)
         occurrences all number
    6
    Diarrhoea
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    8
    Toothache
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    8
    Constipation
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2
    Salivary hypersecretion
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2
    Abdominal pain upper
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Abdominal discomfort
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Dysphagia
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Vomiting projectile
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Skin warm
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2
    Application site erythema
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Excessive granulation tissue
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Hair disorder
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Skin irritation
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Urticaria
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Musculoskeletal stiffness
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Pain in extremity
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Endocrine disorders
    Precocious puberty
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    3 / 3 (100.00%)
         occurrences all number
    11
    Staphylococcal infection
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2
    Candida infection
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Ear infection
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Urinary tract infection
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Viral upper respiratory tract infection
         subjects affected / exposed
    1 / 3 (33.33%)
         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
    07 Jul 2015
    Amendment# 1: The purpose of this amendment was to provide further clarification on: starting dose of study drug, contraceptive methods as it pertains to inclusion criteria #3 and the stopping rules for multiple subjects who experience SAEs possible related to study drug.

    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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