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    Clinical Trial Results:
    A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease

    Summary
    EudraCT number
    		 2007-002840-21
    Trial protocol
    		 ES   GB   IT  
    Global end of trial date
    05 May 2009

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Sep 2018
    First version publication date
    31 May 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    HGT-GCB-039
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT00553631
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Shire
    Sponsor organisation address
    300 Shire Way, Lexington, Massachusetts, United States, 02421
    Public contact
    Tiffany Crump, Medical Communications Manager, Shire HGT, +1 484-595-8850, tcrump@shire.com
    Scientific contact
    Tiffany Crump, Medical Communications Manager, Shire HGT, +1 484-595-8850, tcrump@shire.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-000556-PIP01-09
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    05 May 2009
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    05 May 2009
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to compare the effects of velaglucerase alfa and imiglucerase on hemoglobin concentration in subjects with type 1 Gaucher disease.
    Protection of trial subjects
    This study was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki. This study was also conducted in accordance with local country regulations and International Conference on Harmonisation (ICH) Good Clinical Practice (GCP) E6 guidelines.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    29 Jan 2008
    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
    Spain: 3
    Country: Number of subjects enrolled
    United Kingdom: 1
    Country: Number of subjects enrolled
    Paraguay: 5
    Country: Number of subjects enrolled
    Argentina: 3
    Country: Number of subjects enrolled
    Israel: 4
    Country: Number of subjects enrolled
    Russian Federation: 4
    Country: Number of subjects enrolled
    Tunisia: 6
    Country: Number of subjects enrolled
    United States: 1
    Country: Number of subjects enrolled
    India: 8
    Worldwide total number of subjects
    35
    EEA total number of subjects
    4
    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)
    7
    Adolescents (12-17 years)
    2
    Adults (18-64 years)
    25
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The study was conducted in multiple sites from 29 January 2008 (first subject first enrolled) to 05 May 2009 (last subject completed).

    Pre-assignment
    Screening details
    		 Subjects at least 2 years of age with type 1 Gaucher disease.Gaucher-disease-related anemia and at least 1 of the following: moderate splenomegaly, Gaucher-disease-related thrombocytopenia, readily palpable enlarged liver. Subjects had not received treatment for Gaucher disease within 12 months prior to study entry.

    Period 1
    Period 1 title
    Overall study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Gene-Activated Human Glucocerebrosidase (GA-GCB)
    Arm description
    		 Velaglucerase alfa 60 unit per kilogram (U/kg) administered intravenously (IV) every other week for 39 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    velaglucerase alfa
    Investigational medicinal product code
    GA-GCB
    Other name
    VPRIV™, gene-activated human glucocerebrosidase
    Pharmaceutical forms
    Powder for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Velaglucerase alfa 60 U/kg administered IV every other week for 39 weeks.

    Arm title
    		 Imiglucerase
    Arm description
    		 Imiglucerase 60 U/kg administered IV every other week for 39 weeks.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Imiglucerase
    Investigational medicinal product code
    Other name
    Cerezyme
    Pharmaceutical forms
    Powder for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Imiglucerase 60 U/kg administered IV every other week for 39 weeks.

    Number of subjects in period 1 [1]
    		Gene-Activated Human Glucocerebrosidase (GA-GCB) Imiglucerase
    Started
    17
    17
    Completed
    16
    16
    Not completed
    1
    1
         Consent withdrawn by subject
    -
    1
         Lost to follow-up
    1
    -
    Notes
    [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: The baseline included only subjects who received treatment. Since 1 subject from the 35 randomized subjects did not receive treatment hence it was excluded.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Gene-Activated Human Glucocerebrosidase (GA-GCB)
    Reporting group description
    		 Velaglucerase alfa 60 unit per kilogram (U/kg) administered intravenously (IV) every other week for 39 weeks.

    Reporting group title
    Imiglucerase
    Reporting group description
    		 Imiglucerase 60 U/kg administered IV every other week for 39 weeks.

    Reporting group values
    		 Gene-Activated Human Glucocerebrosidase (GA-GCB) Imiglucerase Total
    Number of subjects
    		 17 17 34
    Age categorical
    Units: Subjects
        Less than equal to (<=)18 years
    		 4 5 9
        Between 18 and 65 years
    		 13 11 24
        Greater than equal to (>=) 65 years
    		 0 1 1
    Age continuous
    Age at the time of consent.
    Units: years
        median (full range (min-max))
    		 36 (7 to 60) 27 (3 to 37) -
    Gender categorical
    Units: Subjects
        Female
    		 9 9 18
        Male
    		 8 8 16

    End points

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    End points reporting groups
    Reporting group title
    Gene-Activated Human Glucocerebrosidase (GA-GCB)
    Reporting group description
    		 Velaglucerase alfa 60 unit per kilogram (U/kg) administered intravenously (IV) every other week for 39 weeks.

    Reporting group title
    Imiglucerase
    Reporting group description
    		 Imiglucerase 60 U/kg administered IV every other week for 39 weeks.

    Primary: Mean Change From Baseline to Month 9 in Hemoglobin (Hgb) Concentration for Each Treatment Group

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    End point title
    		 Mean Change From Baseline to Month 9 in Hemoglobin (Hgb) Concentration for Each Treatment Group [1]
    End point description
    Intent-to-treat (ITT) population comprised of all randomized subjects who received at least 1 full or partial dose of study drug.
    End point type
    Primary
    End point timeframe
    Baseline to Month 9
    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: Descriptive statistical analysis was only performed and inferential statistical analysis was not performed for this endpoint.
    End point values
    		 Gene-Activated Human Glucocerebrosidase (GA-GCB) Imiglucerase
    Number of subjects analysed
    17
    17
    Units: gram per deciliter (g/dl)
        arithmetic mean (standard error)
    1.624 ( 0.223 )
    1.488 ( 0.281 )
    No statistical analyses for this end point

    Secondary: Change From Baseline to Month 9 in Platelet Counts for Each Treatment Group

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    End point title
    		 Change From Baseline to Month 9 in Platelet Counts for Each Treatment Group
    End point description
    Values shown are observed change from Baseline to Month 9. ITT population
    End point type
    Secondary
    End point timeframe
    Baseline to Month 9
    End point values
    		 Gene-Activated Human Glucocerebrosidase (GA-GCB) Imiglucerase
    Number of subjects analysed
    17
    17
    Units: 10^9 per liter (10^9/L)
        arithmetic mean (standard error)
    110.41 ( 17.159 )
    144.38 ( 22.76 )
    No statistical analyses for this end point

    Secondary: Change From Baseline to Month 9 in Normalized Liver Volume (Percent (%) Body Weight) for Each Treatment Group

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    End point title
    		 Change From Baseline to Month 9 in Normalized Liver Volume (Percent (%) Body Weight) for Each Treatment Group
    End point description
    Values shown are observed change from Baseline to Month 9. Measured by Magnetic resonance imaging (MRI). Liver volume has been normalized for percent (%) body weight for each treatment arm. Liver size relative to body weight = (Liver volume [cubic centimeter (cc)]/Body weight [kg]*1000. ITT population.
    End point type
    Secondary
    End point timeframe
    Baseline to Month 9
    End point values
    		 Gene-Activated Human Glucocerebrosidase (GA-GCB) Imiglucerase
    Number of subjects analysed
    17
    17
    Units: cubic centimeter (cm^3)
        arithmetic mean (standard error)
    -1.31 ( 0.347 )
    -1.1 ( 0.182 )
    No statistical analyses for this end point

    Secondary: Change From Baseline to Month 9 in Normalized Spleen Volume (Percent (%) Body Weight) for Each Treatment Group

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    End point title
    		 Change From Baseline to Month 9 in Normalized Spleen Volume (Percent (%) Body Weight) for Each Treatment Group
    End point description
    Values shown are observed change from Baseline to month 9. Measured by Magnetic resonance imaging (MRI). Spleen volume was normalized for percent (%) of body weight for each treatment arm. Spleen size relative to body weight = (Spleen volume [cc]/Body weight [kg])*100. Ten subjects in each treatment group underwent splenectomy, and therefore, were excluded from the analysis. ITT population. Number of subjects analysed signifies subjects evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline to Month 9
    End point values
    		 Gene-Activated Human Glucocerebrosidase (GA-GCB) Imiglucerase
    Number of subjects analysed
    7
    7
    Units: cm^3
        arithmetic mean (standard error)
    -1.34 ( 0.424 )
    -2.46 ( 0.966 )
    No statistical analyses for this end point

    Secondary: Change From Baseline to Month 9 in Plasma Chitotriosidase for Each Treatment Group

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    End point title
    		 Change From Baseline to Month 9 in Plasma Chitotriosidase for Each Treatment Group
    End point description
    Values shown are observed change from Baseline to Month 9. Chitotriosidase levels were measured in 10 subjects in the velaglucerase alfa group and 11 subjects in the imiglucerase group. Units of measure is defined as nanomole per milliliter per hour. ITT population. Number of subjects analysed signifies subjects evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline to Month 9
    End point values
    		 Gene-Activated Human Glucocerebrosidase (GA-GCB) Imiglucerase
    Number of subjects analysed
    10
    11
    Units: nanomole/milliliter/hour (nmol/mL/h)
        arithmetic mean (standard error)
    -34711.9 ( 6887.77 )
    -35109.5 ( 7310.22 )
    No statistical analyses for this end point

    Secondary: Change From Baseline to Month 9 in Plasma Chemokine (C-C Motif) Ligand 18 (CCL18) for Each Treatment Group

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    End point title
    		 Change From Baseline to Month 9 in Plasma Chemokine (C-C Motif) Ligand 18 (CCL18) for Each Treatment Group
    End point description
    Values shown are observed change from Baseline to Month 9. ITT population.
    End point type
    Secondary
    End point timeframe
    Baseline to Month 9
    End point values
    		 Gene-Activated Human Glucocerebrosidase (GA-GCB) Imiglucerase
    Number of subjects analysed
    17
    17
    Units: nanogram per milliliter (ng/mL)
        arithmetic mean (standard error)
    -926.2 ( 113.29 )
    -1153.4 ( 269.63 )
    No statistical analyses for this end point

    Secondary: Number of Subjects Who Developed Antibody for Each Treatment Group

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    End point title
    		 Number of Subjects Who Developed Antibody for Each Treatment Group
    End point description
    Measure type is actual number of subjects who developed antibodies to treatment; GA-GCB or imiglucerase. Antibody detection was based upon serum samples collected at various time points throughout the study. Serum samples were screened using an enzyme-linked immunosorbent assay (ELISA) and positive antibody confirmation was determined using a radioimmunoprecipitation assay (RIP); positive samples were also tested for enzyme neutralizing activity. Subject samples were compared to internal assay controls (positive/negative), positive samples were determined based upon individual assay criteria. Safety population comprised of all randomized subjects who received at least 1 full or partial dose of study drug.
    End point type
    Secondary
    End point timeframe
    Baseline to Month 9
    End point values
    		 Gene-Activated Human Glucocerebrosidase (GA-GCB) Imiglucerase
    Number of subjects analysed
    17
    17
    Units: subjects
    0
    4
    No statistical analyses for this end point

    Secondary: Time to Response- Comparison of GA-GCB and Imiglucerase on the Earliest Time to Respond as Assessed Via Hemoglobin Concentration

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    End point title
    		 Time to Response- Comparison of GA-GCB and Imiglucerase on the Earliest Time to Respond as Assessed Via Hemoglobin Concentration
    End point description
    Time to response was defined as a ≥ 1 g/dL improvement in hemoglobin levels relative to Baseline. Units (%) correlates to the percentage of subjects who had a change of ≥ 1 g/dL improvement in hemoglobin levels relative to Baseline during their participation in the study. ITT population.
    End point type
    Secondary
    End point timeframe
    Response rate at Month 9 compared to Baseline
    End point values
    		 Gene-Activated Human Glucocerebrosidase (GA-GCB) Imiglucerase
    Number of subjects analysed
    17
    17
    Units: percentage of participants
        number (not applicable)
    92.9
    100
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events (AE) monitored from time informed consent/assent obtained through 30 days after the last infusion. For patients (pt) who completed this study and elected to enroll in the long-term extension study, AEs were monitored through the Week 41.
    Adverse event reporting additional description
    AE may have been discovered via observation, examination, questioning or complaint by subjects. Unexpected laboratory values that became significantly out of range and determined to be clinically significant by the investigator could have been reported as AEs. Other AE were determined to be possibly/probably related to GA-GCB by the investigator.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    9.0
    Reporting groups
    Reporting group title
    Gene-Activated Human Glucocerebrosidase (GA-GCB)
    Reporting group description
    		 Velaglucerase alfa 60 U/kg administered IV every other week for 39 weeks.

    Reporting group title
    Imiglucerase
    Reporting group description
    		 Imiglucerase 60 U/kg administered IV every other week for 39 weeks.

    Serious adverse events
    		 Gene-Activated Human Glucocerebrosidase (GA-GCB) Imiglucerase
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 17 (17.65%)
    0 / 17 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Nervous system disorders
    Convulsion
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Thrombocytopenia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Dermatitis allergic
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Gene-Activated Human Glucocerebrosidase (GA-GCB) Imiglucerase
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    16 / 17 (94.12%)
    16 / 17 (94.12%)
    Vascular disorders
    Flushing
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    2
    Hypertension
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Hypotension
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 17 (5.88%)
         occurrences all number
    1
    1
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Axillary pain
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Chills
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    2
    Face oedema
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Fatigue
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Feeling abnormal
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Feeling cold
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    3
    Feeling hot
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    2
    Hunger
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Inflammatory pain
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Influenza like illness
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    4
    Injection site haemorrhage
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 17 (5.88%)
         occurrences all number
    1
    1
    Oedema peripheral
         subjects affected / exposed
    3 / 17 (17.65%)
    0 / 17 (0.00%)
         occurrences all number
    3
    0
    Pain
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Pyrexia
         subjects affected / exposed
    4 / 17 (23.53%)
    2 / 17 (11.76%)
         occurrences all number
    4
    3
    Immune system disorders
    Food allergy
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Hypersensitivity
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 17 (0.00%)
         occurrences all number
    2
    0
    Reproductive system and breast disorders
    Pelvic pain
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Vaginal discharge
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 17 (11.76%)
    2 / 17 (11.76%)
         occurrences all number
    2
    2
    Dyspnoea
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Epistaxis
         subjects affected / exposed
    2 / 17 (11.76%)
    2 / 17 (11.76%)
         occurrences all number
    2
    7
    Pharyngolaryngeal pain
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 17 (5.88%)
         occurrences all number
    1
    1
    Respiratory disorder
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Rhinorrhoea
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 17 (5.88%)
         occurrences all number
    1
    1
    Stridor
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Investigations
    Blood potassium increased
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Activated partial thromboplastin time prolonged
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Blood pressure systolic increased
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    2
    Electrocardiogram abnormal
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Laboratory test abnormal
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 17 (5.88%)
         occurrences all number
    1
    1
    Oxygen saturation decreased
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Prothrombin time prolonged
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Serum ferritin increased
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Injury, poisoning and procedural complications
    Injury
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Post-Traumatic pain
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Tongue injury
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Cardiac disorders
    Sinus bradycardia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Sinus tachycardia
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Nervous system disorders
    Carpal tunnel syndrome
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Convulsion
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Dizziness
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 17 (11.76%)
         occurrences all number
    1
    3
    Headache
         subjects affected / exposed
    3 / 17 (17.65%)
    3 / 17 (17.65%)
         occurrences all number
    7
    6
    Hemiparesis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Migraine
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    2
    Paraesthesia
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 17 (5.88%)
         occurrences all number
    2
    1
    Tremor
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Spontaneous haematoma
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Thrombocythaemia
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 17 (5.88%)
         occurrences all number
    1
    1
    Thrombocytopenia
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Eye disorders
    Conjunctivitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Dry eye
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Abdominal pain
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 17 (11.76%)
         occurrences all number
    2
    3
    Abdominal pain upper
         subjects affected / exposed
    1 / 17 (5.88%)
    3 / 17 (17.65%)
         occurrences all number
    1
    4
    Aphthous stomatitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Constipation
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Diarrhoea
         subjects affected / exposed
    3 / 17 (17.65%)
    1 / 17 (5.88%)
         occurrences all number
    4
    1
    Food poisoning
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorder
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Gingival bleeding
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Nausea
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    3
    0
    Odynophagia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Tooth loss
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Toothache
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Vomiting
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 17 (11.76%)
         occurrences all number
    2
    2
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 17 (5.88%)
         occurrences all number
    1
    1
    Dermatitis allergic
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Lichen planus
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Pruritus
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 17 (0.00%)
         occurrences all number
    3
    0
    Rash
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Rash pruritic
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Swelling face
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    2
    0
    Urticaria
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 17 (5.88%)
         occurrences all number
    2
    2
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 17 (5.88%)
         occurrences all number
    1
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    4 / 17 (23.53%)
    3 / 17 (17.65%)
         occurrences all number
    11
    21
    Arthritis
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Arthropathy
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Back pain
         subjects affected / exposed
    2 / 17 (11.76%)
    2 / 17 (11.76%)
         occurrences all number
    2
    3
    Bone pain
         subjects affected / exposed
    2 / 17 (11.76%)
    3 / 17 (17.65%)
         occurrences all number
    4
    3
    Muscle spasms
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 17 (11.76%)
         occurrences all number
    1
    2
    Myalgia
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 17 (5.88%)
         occurrences all number
    2
    1
    Neck pain
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 17 (5.88%)
         occurrences all number
    2
    1
    Pain in extremity
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 17 (5.88%)
         occurrences all number
    1
    3
    Shoulder pain
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 17 (11.76%)
         occurrences all number
    1
    2
    Bronchitis acute
         subjects affected / exposed
    1 / 17 (5.88%)
    1 / 17 (5.88%)
         occurrences all number
    2
    1
    Cystitis
         subjects affected / exposed
    2 / 17 (11.76%)
    1 / 17 (5.88%)
         occurrences all number
    2
    1
    Cervicitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Ear infection
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Gastroenteritis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Helminthic infection
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Herpes simplex
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Infection parasitic
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Influenza
         subjects affected / exposed
    3 / 17 (17.65%)
    4 / 17 (23.53%)
         occurrences all number
    4
    6
    Nasopharyngitis
         subjects affected / exposed
    3 / 17 (17.65%)
    3 / 17 (17.65%)
         occurrences all number
    3
    3
    Otitis externa
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Paronychia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Pulpitis dental
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Respiratory tract infection
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Rhinitis
         subjects affected / exposed
    3 / 17 (17.65%)
    1 / 17 (5.88%)
         occurrences all number
    3
    1
    Sinusitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    Skin bacterial infection
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Tinea versicolour
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 17 (0.00%)
         occurrences all number
    2
    0
    Staphylococcal infection
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Tonsillitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 17 (0.00%)
         occurrences all number
    2
    0
    Tooth infection
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    Urinary tract infection
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 17 (0.00%)
         occurrences all number
    2
    0
    Viral infection
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    13 Feb 2007
    • Change the cytokine assessment used from Macrophage Colony Stimulating Factor (MCSF) to Granulocyte Macrophage Colony Stimulating Factor (GM-CSF). • The requirement for pregnancy testing was changed from a urine and a serum test at all visits during the treatment phase to a urine test followed by a serum test only if the urine test was positive. • Change collection of adverse event information from time of first infusion to time of informed consent.
    10 Jun 2008
    • The language describing the primary endpoint was modified to: The primary endpoint of this study is to measure the mean change from Baseline to Week 41/End of Study (EOS) in hemoglobin concentration between the two treatment groups. Previously it was not specified that the change was to Week 41. • The language describing the tertiary endpoint of evaluating cytokine parameters was changed to indicate that this would only occur in subjects who were ≥ 18 years of age. • The inclusion criteria defining Gaucher-disease-related anemia, was altered by removing the definition of “being at least 0.5 g/dL” below the lower limit of normal for age and gender. • The inclusion criterion regarding the need for contraception during study participation had the text added for clarification regarding contraception requirements for men in the study to specify that male subjects must use a medically acceptable method of birth control throughout their participation in the study and must report pregnancy of a partner. • The text in Exclusion Criterion 2 has had text added for clarification regarding immunogenic reactions. In addition to being antibody positive or experiencing an anaphylactic reaction, it was specified that a subject could not be anaphylactoid. • Clarifying text was added to Exclusion Criterion 4. The example of erythropoietin for a red blood cell growth factor was added and it was specified that the use of inhaled corticosteroid therapy and intermittent corticosteroids was permitted in certain circumstances. • An additional exclusion criterion was added, specifying that pregnant or lactating women were excluded. • Additional blood sampling for subjects ≥18 years of age was added for immune and inflammatory response testing. • Frequency of chitotriosidase and CCL18 measurement was changed from every study visit (excluding Week 1 and Week 41) to every other week (including Week 1 and Week 41)

    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.
    Subjects aged 2-4 years: 4 subjects (23.5%) in the imiglucerase group and 0 subjects in the GA-GCB group.
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    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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