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    Clinical Trial Results:
    A Multicenter, Multinational, Open-Label Study of Anti-Laronidase Antibody Formation and Urinary GAG Levels in Patients with Mucopolysaccaridosis I (MPS I) Being Treated with Aldurazyme® (laronidase)

    Summary
    EudraCT number
    2015-000585-61
    Trial protocol
    Outside EU/EEA  
    Global end of trial date
    25 May 2007

    Results information
    Results version number
    v1(current)
    This version publication date
    01 Apr 2016
    First version publication date
    25 Jul 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ALID02003
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT00144768
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Genzyme Corporation
    Sponsor organisation address
    500 Kendall Street, Cambridge, MA, United States, 02142
    Public contact
    Trial Transparency Team, Sanofi aventis recherche & développement , Contact-US@sanofi.com
    Scientific contact
    Trial Transparency Team, Sanofi aventis recherche & développement , Contact-US@sanofi.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
    05 May 2014
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    25 May 2007
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To determine whether the development of antibodies to laronidase in subjects with MPS I receiving Aldurazyme impairs the clearance of the glycosaminoglycan (GAG) substrate.
    Protection of trial subjects
    Paediatric subjects: The study was conducted by investigators experienced in the treatment of pediatric subjects. The parent(s) or guardian(s) as well as the children were fully informed of all pertinent aspects of the clinical trial as well as the possibility to discontinue at any time. In addition to the consent form for the parent(s)/guardian(s), an assent form in child-appropriate language was provided and explained to the child. Repeated invasive procedures were minimized. The number of blood samples as well as the amount of blood drawn were adjusted according to age and weight. A topical anesthesia may have been used to minimize distress and discomfort. Adult subject: Subjects were fully informed of all pertinent aspects of the clinical trial as well as the possibility to discontinue at any time in language and terms appropriate for the subject and considering the local culture. During the course of the trial, subjects were provided with individual subject cards indicating the nature of the trial the subject is participating, contact details and any information needed in the event of a medical emergency. Collected personal data and human biological samples were processed in compliance with the Sanofi-Aventis Group Personal Data Protection Charter ensuring that the Group abides by the laws governing personal data protection in force in all countries in which it operates.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    20 Jul 2004
    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
    United States: 6
    Worldwide total number of subjects
    6
    EEA total number of subjects
    0
    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)
    1
    Children (2-11 years)
    4
    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
    The study was conducted at 3 sites in the United States of America between 20 July 2004 and 25 May 2007.

    Pre-assignment
    Screening details
    A total of 6 subjects were enrolled in the study.

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

    Arms
    Arm title
    Laronidase
    Arm description
    Laronidase for 2.8 years.
    Arm type
    Experimental

    Investigational medicinal product name
    Laronidase
    Investigational medicinal product code
    Other name
    Aldurazyme ®
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Laronidase 0.58 mg/kg (100 U/kg) administered weekly.

    Number of subjects in period 1
    Laronidase
    Started
    6
    Completed
    0
    Not completed
    6
         Adverse event
             1
         Unspecified
             4
         Consent withdrawn by subject
             1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Laronidase
    Reporting group description
    Laronidase for 2.8 years.

    Reporting group values
    Laronidase Total
    Number of subjects
    6 6
    Age categorical
    Units: Subjects
        Infants and toddlers (28 days-23 months)
    1 1
        Children (2-11 years)
    4 4
        Adults (18-64 years)
    1 1
    Gender categorical
    Units: Subjects
        Female
    3 3
        Male
    3 3

    End points

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    End points reporting groups
    Reporting group title
    Laronidase
    Reporting group description
    Laronidase for 2.8 years.

    Primary: Percentage Change from Baseline in Urinary Glycosaminoglycan (GAG) Levels

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    End point title
    Percentage Change from Baseline in Urinary Glycosaminoglycan (GAG) Levels [1]
    End point description
    Number of subjects analysed = all enrolled subjects who received at least one study infusion of Aldurazyme and data available for urinary GAG. Here, 'n' signifies the number of subjects with data available at the study time points.
    End point type
    Primary
    End point timeframe
    Baseline, Week 26, 52
    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: Only descriptive data was planned for this outcome measure.
    End point values
    Laronidase
    Number of subjects analysed
    3
    Units: percent change
    median (full range (min-max))
        Week 26 (n=2)
    -51.11 (-55.97 to -46.24)
        Week 52 (n=3)
    -50 (-56.95 to -43.31)
    No statistical analyses for this end point

    Primary: Immunogenicity

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    End point title
    Immunogenicity [2]
    End point description
    End point type
    Primary
    End point timeframe
    Up to Week 52
    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: As this endpoint was not analysed, so no statistical analysis was provided.
    End point values
    Laronidase
    Number of subjects analysed
    0 [3]
    Units: titre
        arithmetic mean (standard deviation)
    ±
    Notes
    [3] - Endpoint was not analysed due to insufficient data.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All Adverse Events (AE) were collected from signature of the informed consent form up to the final visit (up to 145 Weeks) regardless of seriousness or relationship to investigational product.
    Adverse event reporting additional description
    Reported adverse events are treatment-emergent adverse events that is AEs that developed/worsened during the ‘on treatment period’ (from the first infusion of study drug upto last infusion of study drug). Analysis was performed on safety population included all enrolled subjects who received at least one infusion of study drug.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    10.1
    Reporting groups
    Reporting group title
    Laronidase
    Reporting group description
    Laronidase for 2.8 years.

    Serious adverse events
    Laronidase
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 6 (83.33%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Collapse Of Lung
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Shunt Occlusion
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Congenital, familial and genetic disorders
    Foramen Magnum Stenosis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Atelectasis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Diaphragmatic Hernia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Wheezing
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory Failure
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Immune system disorders
    Drug Hypersensitivity
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Cervical Myelopathy
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Headache
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Intracranial Pressure Increased
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Local Swelling
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Abdominal Pain
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Intestinal Obstruction
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Swollen Tongue
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Vomiting
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin and subcutaneous tissue disorders
    Idiopathic Urticaria
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Swelling Face
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Urticaria
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Pain In Extremity
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Sacroiliitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Bacterial Tracheitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Central Line Infection
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Influenza
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Lobar Pneumonia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Osteomyelitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Staphylococcal Infection
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Tracheitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Laronidase
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 6 (100.00%)
    Vascular disorders
    Flushing
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Pallor
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    General disorders and administration site conditions
    Catheter Site Pain
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Catheter Site Erythema
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Disease Progression
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Chills
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    3
    Fatigue
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Infusion Site Swelling
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Non-Cardiac Chest Pain
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Irritability
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Injection Site Reaction
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Pyrexia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    3
    Oedema Peripheral
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Psychiatric disorders
    Abnormal Behaviour
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Agitation
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Restlessness
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Reproductive system and breast disorders
    Testicular Pain
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Incision Site Pain
         subjects affected / exposed
    3 / 6 (50.00%)
         occurrences all number
    8
    Procedural Nausea
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Shunt Occlusion
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Investigations
    Blood Iron Decreased
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Blood Urine Present
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Cardiac Murmur
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Coagulation Time Prolonged
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Oxygen Saturation Decreased
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Weight Decreased
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    3
    Cardiac disorders
    Cyanosis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Cardiomegaly
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Congenital, familial and genetic disorders
    Foramen Magnum Stenosis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Choking
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Cough
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Atelectasis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Dyspnoea
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Diaphragmatic Hernia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Hypoventilation
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Increased Upper Airway Secretion
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Lung Disorder
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Hypoxia
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Nasal Congestion
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Pharyngolaryngeal Pain
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Rhinitis Allergic
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Rhinorrhoea
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    3
    Throat Irritation
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Sleep Apnoea Syndrome
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Nervous system disorders
    Cervical Myelopathy
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Headache
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    10
    Convulsion
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Hypoaesthesia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Hyperreflexia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Intracranial Pressure Increased
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Neurological Symptom
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Spastic Paraplegia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Unresponsive To Stimuli
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    3
    Eye disorders
    Cataract
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Eye Swelling
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    3
    Conjunctivitis
         subjects affected / exposed
    3 / 6 (50.00%)
         occurrences all number
    3
    Eye Discharge
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Ear and labyrinth disorders
    Conductive Deafness
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Otorrhoea
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Hearing Impaired
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Gastrointestinal disorders
    Abdominal Pain
         subjects affected / exposed
    3 / 6 (50.00%)
         occurrences all number
    3
    Abdominal Distension
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Dry Mouth
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Diarrhoea
         subjects affected / exposed
    3 / 6 (50.00%)
         occurrences all number
    5
    Gastrooesophageal Reflux Disease
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Faecal Incontinence
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Haematochezia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Gingivitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Nausea
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    4
    Reflux Oesophagitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Umbilical Hernia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    5 / 6 (83.33%)
         occurrences all number
    10
    Renal and urinary disorders
    Neurogenic Bladder
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Dysuria
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Hepatobiliary disorders
    Hepatosplenomegaly
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Dermatitis Contact
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Alopecia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Dermatitis Diaper
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    4
    Erythema Multiforme
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Idiopathic Urticaria
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Petechiae
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Pruritus Allergic
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Pruritus
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Urticaria
         subjects affected / exposed
    3 / 6 (50.00%)
         occurrences all number
    34
    Rash Generalised
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Back Pain
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Kyphosis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Joint Contracture
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Muscle Atrophy
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Muscle Spasms
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Osteoporosis
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2
    Pain In Extremity
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    3
    Sacroiliitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    3
    Infections and infestations
    Bacteraemia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Cellulitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Candida Nappy Rash
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Ear Infection
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Folliculitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Incision Site Cellulitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Gastroenteritis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Influenza
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Osteomyelitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Nasopharyngitis
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    4
    Otitis Externa
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Otitis Media Acute
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Otitis Media
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    5
    Otitis Media Bacterial
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Otitis Media Chronic
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    3
    Pneumonia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Respiratory Tract Infection
         subjects affected / exposed
    3 / 6 (50.00%)
         occurrences all number
    5
    Rhinitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    2
    Sinusitis
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    4
    Tracheitis
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    3
    Upper Respiratory Tract Infection
         subjects affected / exposed
    5 / 6 (83.33%)
         occurrences all number
    10
    Viral Upper Respiratory Tract Infection
         subjects affected / exposed
    2 / 6 (33.33%)
         occurrences all number
    2

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    13 Jan 2004
    Amendment included follwoing statements: Text was added to clarify that testing for immunoglobulin G (IgG) antibodies would be performed at baseline in addition to quarterly during the study. Repeat testing of urinary GAG levels was changed from quarterly to within 1 month after any urinary GAG value that was >= 15% higher than a previous value. The criterion exempting subjects from neutralizing antibody testing if they had undergone a recent surgical procedure was removed. Text was added to clarify collection of urine samples for urinary GAG measurement.
    17 Feb 2005
    -The number of subjects to be enrolled was reduced from 50 to 25, due to a lower than anticipated subjects accrual rate. -The criterion for testing neutralizing antibodies at Week 12 was clarified as being done if the value was <35% of the baseline value. -The window for baseline urinary GAG, safety laboratory, urinalysis, and IgG evaluations for subjects <5 years of age were shortened to within 3 months prior to enrollment.
    28 Dec 2005
    The purpose of this amendment was to clarify sample collection procedures for subjects who had been treated with Aldurazyme prior to study entry. Wording was added to state that testing of neutralizing antibodies in subjects who did not enter the study as treatment naïve could be achieved by using archived samples taken prior to study entry, if available.

    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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