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    Clinical Trial Results:
    An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7

    Summary
    EudraCT number
    		 2013-001152-35
    Trial protocol
    		 GB   ES  
    Global end of trial date
    13 Jul 2016

    Results information
    Results version number
    		 v3(current)
    This version publication date
    31 Mar 2019
    First version publication date
    30 Aug 2017
    Other versions
    		 v1 , v2
    Version creation reason
    • New data added to full data set
    Update to subject disposition

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    UX003-CL201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01856218
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Ultragenyx Pharmaceutical Inc
    Sponsor organisation address
    60 Leveroni Court, Novato, United States, 94949
    Public contact
    Robert Hostutler, Sr. Clinical Program Manager, Clinical Operations, Ultragenyx Pharmaceutical Inc. , 1 4154838148, rhostutler@ultragenyx.com
    Scientific contact
    Christine Haller, MD, VP, Drug Safety and Pharmacovigilance, Ultragenyx Pharmaceutical Inc. , 1 4154838937, challer@ultragenyx.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001540-PIP01-13
    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
    13 Jul 2016
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    13 Jul 2016
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objectives of the study are to evaluate safety and tolerability of UX003 exposure and efficacy in subjects with mucopolysaccharidosis 7 (MPS VII, Sly syndrome) as determined by the reduction of total urinary glycosaminoglycan (uGAG) excretion.
    Protection of trial subjects
    The trial was designed, conducted, recorded, and reported in accordance with the principles established by the 18th World Medical Association General Assembly (Helsinki, 1964) and subsequent amendments and clarifications adopted by the General Assemblies. The investigators made every effort to ensure that the study was conducted in full conformance with Helsinki principles, International Council for Harmonization (ICH) Good Clinical Practice (GCP) guidelines, current Food and Drug Administration (FDA) regulations, EU Clinical Trial Directive 2001/20/EC, and local ethical and regulatory requirements. Each investigator was thoroughly familiar with the appropriate administration and potential risks of administration of the study drug, as described in the protocol and Investigator’s Brochure, prior to the initiation of the study. The method of obtaining and documenting informed consent and the contents of the informed consent form (ICF) complied with ICH GCP guidelines, the requirements of 21 CFR Part 50, “Protection of Human Subjects,” the Health Insurance Portability and Accountability Act regulations, and all other applicable regulatory requirements. Investigators were responsible for preparing the ICF and submitting it to the Sponsor for approval prior to submission to the Institutional Review Board (IRB). All ICFs were written in regional language and contained the minimum elements for consent as mandated by the ICH guidelines. An IRB-approved ICF was provided by the Sponsor prior to initiation of the study. Investigators obtained signed written informed consent from each potential study subject prior to the conduct of any study procedures and after the methods, objectives, requirements, and potential risks of the study were fully explained to each potential subject. Consent for participation could be withdrawn at any time for any reason by the subject.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    18 Nov 2013
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Regulatory reason
    Long term follow-up duration
    		 36 Months
    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)
    2
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    1
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 In this study, the 3 subjects who were enrolled at Site 111 in the United Kingdom (UK), were also enrolled at local sites in their home countries (1 in Spain and 2 in Turkey) for infusions and blood/urine sample collection. All subjects continued to attend study visits at the UK site for designated major efficacy assessment visits with infusions.

    Pre-assignment
    Screening details
    		 Subjects were evaluated for trial participation based on the protocol-specified inclusion and exclusion criteria. All 3 of the subjects who were screened also enrolled in the study.

    Period 1
    Period 1 title
    First Phase
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 UX003
    Arm description
    		 During the initial 14-week treatment period of the study, subjects received 2 mg/kg UX003 every other week (QOW) for 12 weeks. At Week 14, subjects continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: • 1 mg/kg UX003 for 8 weeks beginning on Week 14, then • 4 mg/kg UX003 for 8 weeks beginning on Week 22, then • 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, subjects who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Recombinant human beta-glucuronidase
    Investigational medicinal product code
    UX003
    Other name
    RHGUS, vestronidase alfa
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    UX003 requires weight-based dosing QOW intravenously by slow infusions over a period of approximately 4 hours. The dosage is designated per study period within first phase.

    Number of subjects in period 1
    		UX003
    Started
    3
    Completed
    3
    Period 2
    Period 2 title
    Long-Term Extension Phase
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 UX003
    Arm description
    		 After the first phase of the study, subjects who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Recombinant human beta-glucuronidase
    Investigational medicinal product code
    UX003
    Other name
    RHGUS, vestronidase alfa
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    UX003 requires weight-based dosing QOW intravenously by slow infusions over a period of approximately 4 hours. The dosage is designated per study period within first phase.

    Number of subjects in period 2
    		UX003
    Started
    3
    Completed > 118 weeks on treatment
    3
    Completed
    0
    Not completed
    3
         Transitioned to compassionate use
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    First Phase
    Reporting group description
    		 -

    Reporting group values
    		 First Phase Total
    Number of subjects
    		 3 3
    Age categorical
    Units: Subjects
        Children (2-11 years)
    		 2 2
        Adults (18-64 years)
    		 1 1
    Gender categorical
    Units: Subjects
        Female
    		 1 1
        Male
    		 2 2

    End points

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    End points reporting groups
    Reporting group title
    UX003
    Reporting group description
    		 During the initial 14-week treatment period of the study, subjects received 2 mg/kg UX003 every other week (QOW) for 12 weeks. At Week 14, subjects continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: • 1 mg/kg UX003 for 8 weeks beginning on Week 14, then • 4 mg/kg UX003 for 8 weeks beginning on Week 22, then • 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, subjects who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks.
    Reporting group title
    UX003
    Reporting group description
    		 After the first phase of the study, subjects who elected to continue drug treatment were transitioned to the long-term extension phase, where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.

    Subject analysis set title
    UX003
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    During the initial 14-week treatment period of the study, subjects received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, subjects continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: • 1 mg/kg UX003 for 8 weeks beginning on Week 14, then • 4 mg/kg UX003 for 8 weeks beginning on Week 22, then • 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, subjects who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the continuation period, subjects who elected to continue drug treatment were transitioned to the long-term extension period of the study where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.

    Primary: Percentage Change From Baseline in uGAG Dermatan Sulfate

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    End point title
    		 Percentage Change From Baseline in uGAG Dermatan Sulfate [1]
    End point description
    Percentage change from baseline in the concentration of uGAGs normalized to the urinary creatinine concentration as measured by liquid chromatography-mass spectrometry/mass spectrometry-dermatan sulfate.
    End point type
    Primary
    End point timeframe
    Week 14, Week 22, Week 30, Week 38, Week 72, and end of study (up to Week 132)
    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: Mean (SD) statistics are presented, per protocol.
    End point values
    		 UX003
    Number of subjects analysed
    3
    Units: percentage change
    arithmetic mean (standard deviation)
        Initial treatment-Week 14
    -50.41 ( 7.895 )
        Forced dose titration-Week 22
    -38.94 ( 7.137 )
        Forced dose titration-Week 30
    -63.49 ( 6.889 )
        Forced dose titration-Week 38
    -51.82 ( 9.033 )
        Continuation-Week 72
    -54.2 ( 8.442 )
        Long term extension-end of study
    -34.44 ( 48.56 )
    No statistical analyses for this end point

    Primary: Percentage Change From Baseline in uGAG Chondroitin Sulfate

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    End point title
    		 Percentage Change From Baseline in uGAG Chondroitin Sulfate [2]
    End point description
    Percentage change from baseline in the concentration of uGAGs normalized to the urinary creatinine concentration as measured by liquid chromatography-mass spectrometry/mass spectrometry-chondroitin sulfate.
    End point type
    Primary
    End point timeframe
    Week 14, Week 22, Week 30, Week 38, Week 72, and end of study (up to Week 132)
    Notes
    [2] - 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: Mean (SD) statistics are presented, per protocol.
    End point values
    		 UX003
    Number of subjects analysed
    3
    Units: percentage change
    arithmetic mean (standard deviation)
        Initial treatment-Week 14
    -52.5 ( 13.726 )
        Forced dose titration-Week 22
    -47.58 ( 3.958 )
        Forced dose titration-Week 30
    -60.78 ( 7.207 )
        Forced dose titration-Week 38
    -52.08 ( 13.298 )
        Continuation-Week 72
    -56.96 ( 13.379 )
        Long term extension-end of study
    -30.83 ( 40.794 )
    No statistical analyses for this end point

    Primary: Number of Subjects With Any ≥ 50% Decrease in uGAG

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    End point title
    		 Number of Subjects With Any ≥ 50% Decrease in uGAG [3]
    End point description
    Subjects with a ≥ 50% decrease in the concentration of uGAGs normalized to the urinary creatinine concentration as measured by liquid chromatography-mass spectrometry/mass spectrometry-dermatan sulfate or chondroitin sulfate.
    End point type
    Primary
    End point timeframe
    up to Week 132
    Notes
    [3] - 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: Descriptive statistics are presented, per protocol.
    End point values
    		 UX003
    Number of subjects analysed
    3
    Units: subjects
    3
    No statistical analyses for this end point

    Primary: Number of Subjects with Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and Study/Treatment Discontinuations

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    End point title
    		 Number of Subjects with Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and Study/Treatment Discontinuations [4]
    End point description
    Adverse Event (AE): any untoward medical occurrence in a subject, whether or not considered drug related. SAE: an AE or suspected adverse reaction that at any dose results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; a congenital anomaly/birth defect. Other important medical events may also, in the opinion of the Investigator, be considered SAEs. An AE was considered a TEAE if it occurred on or after the first dose, and was not present prior to the first dose, or it was present at the first dose but increased in severity during the study. Events recorded as either possibly, probably, or definitely related to treatment were categorized as related. AE severity was graded using the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.03.
    End point type
    Primary
    End point timeframe
    Up to 242 weeks + 30 days. SAEs were recorded beginning at the time the subject signed the informed consent form through 30 days following the last study visit. Non-serious AEs were recorded from the time of signing the ICF through the last study visit.
    Notes
    [4] - 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: Descriptive statistics are presented, per protocol.
    End point values
    		 UX003
    Number of subjects analysed
    3
    Units: subjects
        Any TEAE
    3
        Treatment-related TEAE
    2
        SAE
    2
        Grade 3 or 4 TEAE
    2
        TEAE leading to treatment discontinuation
    1
        TEAE leading to study discontinuation
    0
        Fatal TEAE
    0
    No statistical analyses for this end point

    Secondary: Dose Selection for the Long-term Extension Phase

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    End point title
    		 Dose Selection for the Long-term Extension Phase
    End point description
    The choice of the dose of UX003 QOW for the Long-Term Extension Phase was based on a preliminary efficacy analysis at Week 36 prior to all 3 subjects completing the Forced-dose Titration Period of the First Phase of the study.
    End point type
    Secondary
    End point timeframe
    Week 36
    End point values
    		 UX003
    Number of subjects analysed
    3
    Units: mg/kg
        number (not applicable)
    4
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 242 weeks + 30 days. SAEs were recorded beginning at the time the subject signed the informed consent form through 30 days following the last study visit. Non-serious AEs were recorded from the time of signing the ICF through the last study visit.
    Adverse event reporting additional description
    TEAEs are presented. An AE was considered a TEAE if it occurred on or after the first dose, and was not present prior to the first dose, or it was present at the first dose but increased in severity during the study.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    UX003
    Reporting group description
    		 During the initial 14-week treatment period of the study, subjects received 2 mg/kg UX003 QOW for 12 weeks. At Week 14, subjects continued on UX003 therapy and began a forced dose titration period for an additional 24 weeks at the dose sequence of 1, 4, and 2 mg/kg UX003 QOW as follows: • 1 mg/kg UX003 for 8 weeks beginning on Week 14, then • 4 mg/kg UX003 for 8 weeks beginning on Week 22, then • 2 mg/kg UX003 for 8 weeks beginning on Week 30. Following the 24 week forced dose titration period, subjects who continued on treatment (continuation period) received 2 mg/kg UX003 QOW beginning at Week 38 for up to an additional 36 weeks. After the continuation period, subjects who elected to continue drug treatment were transitioned to the long-term extension period of the study where they were treated with UX003 at 4 mg/kg beginning at Week 74, for up to an additional 168 weeks.

    Serious adverse events
    		 UX003
    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
    Surgical and medical procedures
    Inguinal hernia repair
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Spinal cord compression
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Cerebral ventricle dilatation
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Oedema peripheral
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Incarcerated inguinal hernia
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Osteoarthritis
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 UX003
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 3 (100.00%)
    Investigations
    Body temperature increased
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Weight increased
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Papilloma
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Ligament sprain
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Skin abrasion
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Nystagmus
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Reflexes abnormal
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Visual field defect
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    General disorders and administration site conditions
    Discomfort
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Gait disturbance
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Infusion site extravasation
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    3
    Oedema peripheral
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Pyrexia
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    3
    Generalised Oedema
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Diarrhoea
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2
    Oral mucosal erythema
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Toothache
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    3
    Respiratory, thoracic and mediastinal disorders
    Bronchospasm
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Cough
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2
    Increased viscosity of bronchial secretion
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Nasal congestion
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Oropharyngeal pain
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    4
    Respiratory failure
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    3
    Rhinitis allergic
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2
    Rhinorrhoea
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Skin and subcutaneous tissue disorders
    Decubitus ulcer
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Erythema
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Pruritus
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Rash
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Rash macular
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Seborrhoeic dermatitis
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    3 / 3 (100.00%)
         occurrences all number
    6
    Groin pain
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Musculoskeletal 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
    Myalgia
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Infections and infestations
    Acute sinusitis
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Dermatitis infected
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Ear infection
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Molluscum contagiosum
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Nasopharyngitis
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Nosocomial infection
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Otitis media
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    2
    Pharyngitis
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    3
    Respiratory tract infection
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Tonsillitis
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Tooth abscess
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 3 (66.67%)
         occurrences all number
    7
    Urinary tract infection
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    2
    Viral rash
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Increased appetite
         subjects affected / exposed
    1 / 3 (33.33%)
         occurrences all number
    1

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    22 Nov 2013
    The most important changes included the change from a single-center study to a global multi-center study, a clarification that the normal uGAG reference range was the age-specific normal mean. The definition of relatedness for AEs was updated, and pregnancy language was updated to include acceptable forms of contraception and the required length of time after the last dose of study drug that subjects should continue to use an acceptable form of contraception.
    01 Dec 2014
    The addition of the Long-term Extension Phase with selection of 4 mg/kg dose to the study design, and addition of PK sampling time points.

    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.
    This study was terminated due to business considerations unrelated to safety or efficacy concerns.
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    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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