Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43865   clinical trials with a EudraCT protocol, of which   7286   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta

    Summary
    EudraCT number
    		 2016-000378-38
    Trial protocol
    		 GB   HU   ES   CZ   NO   BE   NL   SI   IT   FI   FR  
    Global end of trial date
    12 Oct 2021

    Results information
    Results version number
    		 v2(current)
    This version publication date
    09 Jun 2023
    First version publication date
    16 Mar 2023
    Other versions
    		 v1
    Version creation reason
    • Correction of full data set
    No additional results have been added, the results for mean Lyso Gb3 and mean eGFR were removed, keeping median Lyso Gb3 and median eGFR

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    PB-102-F20
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02795676
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Protalix Ltd.
    Sponsor organisation address
    2 Snunit Street, Carmiel, Israel, 2161401
    Public contact
    Sari Alon, Protalix Ltd., +972 4-902-8100, sari@protalix.com
    Scientific contact
    Sari Alon, Protalix Ltd., +972 4-902-8100, sari@protalix.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
    22 Jul 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    12 Oct 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    12 Oct 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety, efficacy, and pharmacokinetics (PK) of PRX-102 (pegunigalsidase alfa) compared to agalsidase beta in adult Fabry disease patients with impaired renal function
    Protection of trial subjects
    The first infusions of agalsidase beta or PRX-102 were performed under controlled conditions at the investigation site. Patients were allowed to receive subsequent infusions at home if the Investigator and the sponsor’s Medical Monitor agreed that it was safe to do so, based on the patient’s clinical condition and on local practices and regulations. The administration of Agalsidase beta or PRX-102 was intravenously over 3 hours, every 2 weeks. After the first 3 months, infusion time was reduced gradually to 1.5 hours pending patient tolerability, per Principal Investigator (PI) evaluation, and Medical Monitor approval. All patients and study staff were blind to the treatment given throughout the whole study.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    22 Aug 2016
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy, Regulatory reason
    Long term follow-up duration
    		 60 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 52
    Country: Number of subjects enrolled
    Netherlands: 5
    Country: Number of subjects enrolled
    Norway: 2
    Country: Number of subjects enrolled
    Slovenia: 2
    Country: Number of subjects enrolled
    Spain: 3
    Country: Number of subjects enrolled
    United Kingdom: 5
    Country: Number of subjects enrolled
    Czechia: 1
    Country: Number of subjects enrolled
    Finland: 1
    Country: Number of subjects enrolled
    France: 3
    Country: Number of subjects enrolled
    Hungary: 1
    Country: Number of subjects enrolled
    Italy: 3
    Worldwide total number of subjects
    78
    EEA total number of subjects
    21
    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)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    78
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 Symptomatic adult Fabry patients who had been taking agalsidase beta for at least 1 year and on a stable dose for at least 6 months. No more than 50% could be female. Screening eGFR (CKD-EPI) 40 to 120 mL/min/1.73 m^2; Screening linear eGFR slope more negative than -2 mL/min/1.73 m^2/year based on at least 3 values over ~1 year.

    Pre-assignment
    Screening details
    		 Of the 78 randomized patients, 53 were assigned to the PRX-102 arm and 25 to the agalsidase beta arm. One PRX-102 patient withdrew consent before receiving the study product; accordingly, 77 patients were treated, 52 in PRX-102 arm and 25 in agalsidase beta arm.

    Pre-assignment period milestones
    Number of subjects started
    		 78
    Number of subjects completed
    		 77

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    		 Consent withdrawn by subject: 1
    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer
    Blinding implementation details
    The infusions were prepared by an unblinded pharmacist or nurse at the site or at a central pharmacy (for home care), resulting in identical infusion bag appearance and blinded labelling prior to administration. Both the patients and the staff members administering the treatments were blinded as to what the infusion bag contained.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Pegunigalsidase alfa ITT set
    Arm description
    		 Pegunigalsidase alfa administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Pegunigalsidase alfa
    Investigational medicinal product code
    Other name
    PRX-102
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    1 mg/kg administered as an intravenous infusion every 2 weeks for up to 24 months

    Arm title
    		 Agalsidase beta ITT set
    Arm description
    		 Agalsidase beta administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg
    Arm type
    		 Active comparator

    Investigational medicinal product name
    agalsidase beta
    Investigational medicinal product code
    Other name
    Fabrazyme
    Pharmaceutical forms
    Powder for concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    1 mg/kg administered as an intravenous infusion every 2 weeks for up to 24 months

    Number of subjects in period 1 [1]
    		Pegunigalsidase alfa ITT set Agalsidase beta ITT set
    Started
    52
    25
    Completed
    48
    24
    Not completed
    4
    1
         Consent withdrawn by subject
    2
    1
         Adverse event, non-fatal
    2
    -
    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 number of subjects reported in the baseline period (77) are not the same as the worldwide number enrolled (78), since one enrolled patient withdrew consent before receiving study drug, and therefore is not counted in the baseline period

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Pegunigalsidase alfa ITT set
    Reporting group description
    		 Pegunigalsidase alfa administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg

    Reporting group title
    Agalsidase beta ITT set
    Reporting group description
    		 Agalsidase beta administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg

    Reporting group values
    		 Pegunigalsidase alfa ITT set Agalsidase beta ITT set Total
    Number of subjects
    		 52 25 77
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 52 25 77
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 43.9 ( 10.2 ) 45.2 ( 9.6 ) -
    Gender categorical
    Units: Subjects
        Female
    		 23 7 30
        Male
    		 29 18 47

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Pegunigalsidase alfa ITT set
    Reporting group description
    		 Pegunigalsidase alfa administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg

    Reporting group title
    Agalsidase beta ITT set
    Reporting group description
    		 Agalsidase beta administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg

    Primary: Annualized change (slope) in estimated glomerular filtration rate (eGFR)

    		 Close Top of page
    End point title
    		 Annualized change (slope) in estimated glomerular filtration rate (eGFR)
    End point description
    The individual annualized mean change (slope) in eGFR (mL/min/1.73 m^2/year) is an estimate of the individual patient’s annualized change in eGFR, which is derived from the eGFR (by Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI formula, 2009] assessments over time, for up to 24 months.
    End point type
    Primary
    End point timeframe
    24 Months
    End point values
    		 Pegunigalsidase alfa ITT set Agalsidase beta ITT set
    Number of subjects analysed
    51
    25
    Units: mL/min/1.73 m^2/year
        median (confidence interval 95%)
    -2.514 (-3.788 to -1.240)
    -2.155 (-3.805 to -0.505)
    Statistical analysis title
    		 eGFR slope comparison
    Statistical analysis description
    The primary efficacy analysis compared eGFR slope between the treatment arms using a 2-stage model with quantile regression. At the 1st stage, the individual annualized change (slope) in eGFR was estimated for each patient using a linear regression model. At the 2nd stage, the annualized change (slope) of the eGFR between the two treatment arms were compared using quantile regression estimating the median slopes.
    Comparison groups
    Pegunigalsidase alfa ITT set v Agalsidase beta ITT set
    Number of subjects included in analysis
    76
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority [1]
    Method
    Parameter type
    		 Median difference (final values)
    Point estimate
    -0.359
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -2.444
         upper limit
    		 1.726
    Notes
    [1] - The dependent variable was the slope of each individual patient and the model included intercept and treatment arm. Non-inferiority was to be declared if the lower bound of the confidence interval for the treatment difference (PRX-102 minus agalsidase beta) was greater or equal to -3.0 mL/min/1.73 m^2/year.

    Secondary: Estimated Glomerular Filtration rate (eGFR)

    		 Close Top of page
    End point title
    		 Estimated Glomerular Filtration rate (eGFR)
    End point description
    eGFR was calculated based on measured serum creatinine levels according to the CKD-EPI formula.
    End point type
    Secondary
    End point timeframe
    24 months
    End point values
    		 Pegunigalsidase alfa ITT set Agalsidase beta ITT set
    Number of subjects analysed
    52
    25
    Units: mL/min/1.73 m^2
    median (full range (min-max))
        Baseline
    73.45 (30.2 to 125.9)
    74.85 (34.1 to 107.6)
        Month 24
    69.35 (27.6 to 113.7)
    74.48 (24.4 to 114.8)
        Change from Baseline to Month 24
    -2.39 (-36.9 to 21.8)
    -3.20 (-18.0 to 16.8)
    No statistical analyses for this end point

    Secondary: Plasma lyso-Gb3

    		 Close Top of page
    End point title
    		 Plasma lyso-Gb3
    End point description
    Globotriaosylsphingosine (Lyso-Gb3) is Fabry disease specific biomarker measured in the plasma (nanomole/liter, nM).
    End point type
    Secondary
    End point timeframe
    24 Months
    End point values
    		 Pegunigalsidase alfa ITT set Agalsidase beta ITT set
    Number of subjects analysed
    52
    25
    Units: nM
    median (full range (min-max))
        Baseline
    15.20 (0.8 to 143.9)
    17.60 (2.1 to 142.0)
        Month 24
    18.80 (2.4 to 139.4)
    15.30 (1.5 to 71.2)
        Change from Baseline to Month 24
    1.15 (-32.2 to 32.7)
    -1.50 (-102.3 to 2.4)
    No statistical analyses for this end point

    Secondary: Short Form Brief Pain Inventory (BPI)

    		 Close Top of page
    End point title
    		 Short Form Brief Pain Inventory (BPI)
    End point description
    The Short Form Brief Pain Inventory (BPI) questioner is self-completed by patients regarding pain severity and interference. Descriptive statistics summarizes the findings for the change from baseline at Week 104 for “Pain at Its Worst in Last 24 Hours” . The severity of various aspects of pain scored on a scale of 0 to 10 (no pain / pain as bad as you can imagine).
    End point type
    Secondary
    End point timeframe
    24 Month
    End point values
    		 Pegunigalsidase alfa ITT set Agalsidase beta ITT set
    Number of subjects analysed
    52
    25
    Units: score
    arithmetic mean (standard error)
        Baseline
    3.5 ( 0.4 )
    2.6 ( 0.6 )
        Month 24
    3.3 ( 0.5 )
    3.0 ( 0.7 )
        Change from Baseline to Month 24
    -0.1 ( 0.5 )
    0.6 ( 0.6 )
    No statistical analyses for this end point

    Secondary: Mainz Severity Score Index (MSSI)

    		 Close Top of page
    End point title
    		 Mainz Severity Score Index (MSSI)
    End point description
    The Mainz Severity Score Index (MSSI), is an instrument that is specifically designed to measure the severity of Fabry disease signs/symptoms and to monitor the clinical course of the disease. MSSI is administered by the investigator, yields scores for general, neurological, cardiovascular, renal, and overall assessments. An overall score of less than 20 points is considered mild, 20 to 40 is considered moderate, and greater than 40 is considered severe signs and symptoms of Fabry disease.
    End point type
    Secondary
    End point timeframe
    24 Month
    End point values
    		 Pegunigalsidase alfa ITT set Agalsidase beta ITT set
    Number of subjects analysed
    52
    25
    Units: score
    arithmetic mean (standard error)
        Baseline
    23.18 ( 1.42 )
    25.16 ( 2.14 )
        Month 24
    22.11 ( 1.80 )
    27.09 ( 2.30 )
        Change from Baseline to Month 24
    -2.07 ( 0.77 )
    2.04 ( 1.10 )
    No statistical analyses for this end point

    Secondary: Urine Protein/Creatinine Ratio (UPCR)

    		 Close Top of page
    End point title
    		 Urine Protein/Creatinine Ratio (UPCR)
    End point description
    The UPCR provides an estimate of protein excretion in urine, is used as an indicator of the extent of chronic kidney disease, and was classified into three categories: 1) UPCR ≤ 0.5 gr/gr, 2) 0.5gr/gr < UPCR < 1gr/gr, 3) 1gr/gr ≤ UPCR. Presented as the percent of patients (%) in each category at baseline and Month 24.
    End point type
    Secondary
    End point timeframe
    24 months
    End point values
    		 Pegunigalsidase alfa ITT set Agalsidase beta ITT set
    Number of subjects analysed
    52
    25
    Units: percent of patients (%)
        Baseline: UPCR ≤ 0.5 gr/gr
    69
    80
        Month 24: UPCR ≤ 0.5 gr/gr
    76
    75
        Baseline: 0.5 < UPCR < 1 gr/gr
    17
    8
        Month 24: 0.5 < UPCR < 1 gr/gr
    11
    8
        Baseline: UPCR ≥ 1 gr/gr
    14
    12
        Month 24: UPCR ≥ 1 gr/gr
    13
    17
    No statistical analyses for this end point

    Secondary: Left Ventricular Mass Index (LVMI) with hypertrophy at baseline

    		 Close Top of page
    End point title
    		 Left Ventricular Mass Index (LVMI) with hypertrophy at baseline
    End point description
    Left Ventricular Mass Index (LVMI) based on cardiac MRI for patients with hypertrophy at baseline (for males hypertrophy is above 91 g/m^2 and for females above 77 g/m^2).
    End point type
    Secondary
    End point timeframe
    24 Months
    End point values
    		 Pegunigalsidase alfa ITT set Agalsidase beta ITT set
    Number of subjects analysed
    12
    9
    Units: g/m^2
    median (full range (min-max))
        Baseline
    108.005 (81.77 to 168.42)
    103.030 (78.91 to 147.33)
        Month 24
    118.130 (87.78 to 150.67)
    121.380 (63.78 to 187.23)
        Change from Baseline to Month 24
    -4.790 (-24.42 to 21.55)
    4.120 (-28.41 to 41.10)
    No statistical analyses for this end point

    Secondary: Left Ventricular Mass Index (LVMI) without hypertrophy at baseline

    		 Close Top of page
    End point title
    		 Left Ventricular Mass Index (LVMI) without hypertrophy at baseline
    End point description
    Left Ventricular Mass Index (LVMI) based on cardiac MRI for patients without hypertrophy at baseline (for males hypertrophy is above 91 g/m^2 and for females above 77 g/m^2).
    End point type
    Secondary
    End point timeframe
    24 Months
    End point values
    		 Pegunigalsidase alfa ITT set Agalsidase beta ITT set
    Number of subjects analysed
    28
    13
    Units: g/m^2
    median (full range (min-max))
        Baseline
    55.555 (33.81 to 89.24)
    66.040 (35.74 to 86.92)
        Month 24
    52.160 (35.80 to 100.01)
    62.520 (35.38 to 88.48)
        Change from Baseline to Month 24
    1.990 (-29.37 to 18.37)
    0.515 (-13.69 to 11.15)
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Adverse events (AEs) were collected at every visit.
    Adverse event reporting additional description
    A treatment-emergent adverse event (TEAE) is defined as any AE occurring after the start of the first infusion.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19
    Reporting groups
    Reporting group title
    Patients in the pegunigalsidase alfa arm
    Reporting group description
    		 Events occurring from the start of the study treatment to the final dose were defined as treatment-emergent adverse events (TEAEs)

    Reporting group title
    Patients in the agalsidase beta arm
    Reporting group description
    		 Events occurring from the start of the study treatment to the final dose were defined as treatment-emergent adverse events (TEAEs)

    Serious adverse events
    		 Patients in the pegunigalsidase alfa arm Patients in the agalsidase beta arm
    Total subjects affected by serious adverse events
         subjects affected / exposed
    8 / 52 (15.38%)
    6 / 25 (24.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Vascular disorders
    Aortic stenosis
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Venous thrombosis limb
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Surgical and medical procedures
    Medical device battery replacement
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nephrectomy
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Hypothermia
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Chest pain
         subjects affected / exposed
    0 / 52 (0.00%)
    2 / 25 (8.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Psychiatric disorders
    Suicidal ideation
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Investigations
    Hepatic enzyme increased
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Femur fracture
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Atrioventricular block second degree
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Atrial fibrillation
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tachycardia
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Ventricular tachycardia
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Altered state of consciousness
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Protein-losing gastroenteropathy
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    0 / 52 (0.00%)
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    1 / 52 (1.92%)
    0 / 25 (0.00%)
         occurrences causally related to treatment / all
    0 / 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
    		 Patients in the pegunigalsidase alfa arm Patients in the agalsidase beta arm
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    47 / 52 (90.38%)
    24 / 25 (96.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    3 / 52 (5.77%)
    1 / 25 (4.00%)
         occurrences all number
    4
    1
    Hypotension
         subjects affected / exposed
    0 / 52 (0.00%)
    2 / 25 (8.00%)
         occurrences all number
    0
    2
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    9 / 52 (17.31%)
    4 / 25 (16.00%)
         occurrences all number
    10
    6
    Pyrexia
         subjects affected / exposed
    5 / 52 (9.62%)
    3 / 25 (12.00%)
         occurrences all number
    5
    4
    Oedema peripheral
         subjects affected / exposed
    4 / 52 (7.69%)
    3 / 25 (12.00%)
         occurrences all number
    9
    3
    Infusion site extravasation
         subjects affected / exposed
    3 / 52 (5.77%)
    0 / 25 (0.00%)
         occurrences all number
    4
    0
    Pain
         subjects affected / exposed
    2 / 52 (3.85%)
    3 / 25 (12.00%)
         occurrences all number
    3
    5
    Chest pain
         subjects affected / exposed
    1 / 52 (1.92%)
    3 / 25 (12.00%)
         occurrences all number
    1
    3
    Influenza like illness
         subjects affected / exposed
    1 / 52 (1.92%)
    3 / 25 (12.00%)
         occurrences all number
    1
    4
    Malaise
         subjects affected / exposed
    1 / 52 (1.92%)
    2 / 25 (8.00%)
         occurrences all number
    1
    3
    Chest discomfort
         subjects affected / exposed
    0 / 52 (0.00%)
    2 / 25 (8.00%)
         occurrences all number
    0
    3
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    4 / 52 (7.69%)
    1 / 25 (4.00%)
         occurrences all number
    5
    2
    Hypersensitivity
         subjects affected / exposed
    2 / 52 (3.85%)
    2 / 25 (8.00%)
         occurrences all number
    2
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    6 / 52 (11.54%)
    5 / 25 (20.00%)
         occurrences all number
    7
    7
    Upper respiratory tract congestion
         subjects affected / exposed
    4 / 52 (7.69%)
    0 / 25 (0.00%)
         occurrences all number
    6
    0
    Nasal congestion
         subjects affected / exposed
    3 / 52 (5.77%)
    1 / 25 (4.00%)
         occurrences all number
    3
    1
    Oropharyngeal pain
         subjects affected / exposed
    3 / 52 (5.77%)
    3 / 25 (12.00%)
         occurrences all number
    4
    3
    Rhinorrhoea
         subjects affected / exposed
    1 / 52 (1.92%)
    3 / 25 (12.00%)
         occurrences all number
    1
    3
    Product issues
    Device occlusion
         subjects affected / exposed
    0 / 52 (0.00%)
    2 / 25 (8.00%)
         occurrences all number
    0
    2
    Investigations
    Urine protein/creatinine ratio increased
         subjects affected / exposed
    3 / 52 (5.77%)
    0 / 25 (0.00%)
         occurrences all number
    5
    0
    Blood creatine increased
         subjects affected / exposed
    2 / 52 (3.85%)
    4 / 25 (16.00%)
         occurrences all number
    5
    5
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 52 (1.92%)
    2 / 25 (8.00%)
         occurrences all number
    1
    3
    Fall
         subjects affected / exposed
    1 / 52 (1.92%)
    3 / 25 (12.00%)
         occurrences all number
    1
    4
    Thermal burn
         subjects affected / exposed
    1 / 52 (1.92%)
    2 / 25 (8.00%)
         occurrences all number
    1
    4
    Wound
         subjects affected / exposed
    0 / 52 (0.00%)
    2 / 25 (8.00%)
         occurrences all number
    0
    2
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    4 / 52 (7.69%)
    1 / 25 (4.00%)
         occurrences all number
    5
    3
    Palpitations
         subjects affected / exposed
    3 / 52 (5.77%)
    2 / 25 (8.00%)
         occurrences all number
    4
    2
    Cardiomyopathy
         subjects affected / exposed
    0 / 52 (0.00%)
    2 / 25 (8.00%)
         occurrences all number
    0
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    11 / 52 (21.15%)
    5 / 25 (20.00%)
         occurrences all number
    19
    9
    Dizziness
         subjects affected / exposed
    6 / 52 (11.54%)
    2 / 25 (8.00%)
         occurrences all number
    8
    2
    Neuralgia
         subjects affected / exposed
    4 / 52 (7.69%)
    0 / 25 (0.00%)
         occurrences all number
    5
    0
    Neuropathy peripheral
         subjects affected / exposed
    3 / 52 (5.77%)
    0 / 25 (0.00%)
         occurrences all number
    3
    0
    Sciatica
         subjects affected / exposed
    3 / 52 (5.77%)
    0 / 25 (0.00%)
         occurrences all number
    5
    0
    Paraesthesia
         subjects affected / exposed
    2 / 52 (3.85%)
    4 / 25 (16.00%)
         occurrences all number
    2
    8
    Cerebral infarction
         subjects affected / exposed
    0 / 52 (0.00%)
    2 / 25 (8.00%)
         occurrences all number
    0
    2
    Migraine
         subjects affected / exposed
    0 / 52 (0.00%)
    2 / 25 (8.00%)
         occurrences all number
    0
    3
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    4 / 52 (7.69%)
    2 / 25 (8.00%)
         occurrences all number
    4
    2
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    3 / 52 (5.77%)
    1 / 25 (4.00%)
         occurrences all number
    4
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    10 / 52 (19.23%)
    6 / 25 (24.00%)
         occurrences all number
    15
    10
    Nausea
         subjects affected / exposed
    9 / 52 (17.31%)
    3 / 25 (12.00%)
         occurrences all number
    10
    3
    Abdominal pain
         subjects affected / exposed
    6 / 52 (11.54%)
    0 / 25 (0.00%)
         occurrences all number
    6
    0
    Vomiting
         subjects affected / exposed
    6 / 52 (11.54%)
    3 / 25 (12.00%)
         occurrences all number
    8
    8
    Gastrooesophageal reflux disease
         subjects affected / exposed
    3 / 52 (5.77%)
    1 / 25 (4.00%)
         occurrences all number
    3
    1
    Abdominal pain upper
         subjects affected / exposed
    2 / 52 (3.85%)
    4 / 25 (16.00%)
         occurrences all number
    2
    7
    Abdominal discomfort
         subjects affected / exposed
    1 / 52 (1.92%)
    3 / 25 (12.00%)
         occurrences all number
    1
    3
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    5 / 52 (9.62%)
    2 / 25 (8.00%)
         occurrences all number
    5
    3
    dermatitis contact
         subjects affected / exposed
    1 / 52 (1.92%)
    2 / 25 (8.00%)
         occurrences all number
    1
    3
    Erythema
         subjects affected / exposed
    1 / 52 (1.92%)
    2 / 25 (8.00%)
         occurrences all number
    1
    6
    Pruritus
         subjects affected / exposed
    0 / 52 (0.00%)
    3 / 25 (12.00%)
         occurrences all number
    0
    23
    Urticaria
         subjects affected / exposed
    0 / 52 (0.00%)
    2 / 25 (8.00%)
         occurrences all number
    0
    2
    Renal and urinary disorders
    Proteinuria
         subjects affected / exposed
    6 / 52 (11.54%)
    0 / 25 (0.00%)
         occurrences all number
    7
    0
    Haematuria
         subjects affected / exposed
    3 / 52 (5.77%)
    0 / 25 (0.00%)
         occurrences all number
    4
    0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    8 / 52 (15.38%)
    5 / 25 (20.00%)
         occurrences all number
    12
    6
    Pain in extremity
         subjects affected / exposed
    8 / 52 (15.38%)
    4 / 25 (16.00%)
         occurrences all number
    15
    5
    Muscle spasms
         subjects affected / exposed
    5 / 52 (9.62%)
    3 / 25 (12.00%)
         occurrences all number
    6
    3
    Arthralgia
         subjects affected / exposed
    4 / 52 (7.69%)
    2 / 25 (8.00%)
         occurrences all number
    4
    4
    Musculoskeletal pain
         subjects affected / exposed
    3 / 52 (5.77%)
    2 / 25 (8.00%)
         occurrences all number
    3
    2
    Arthritis
         subjects affected / exposed
    1 / 52 (1.92%)
    2 / 25 (8.00%)
         occurrences all number
    1
    2
    Joint swelling
         subjects affected / exposed
    0 / 52 (0.00%)
    2 / 25 (8.00%)
         occurrences all number
    0
    3
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    11 / 52 (21.15%)
    4 / 25 (16.00%)
         occurrences all number
    21
    6
    Sinusitis
         subjects affected / exposed
    8 / 52 (15.38%)
    3 / 25 (12.00%)
         occurrences all number
    9
    5
    Upper respiratory tract infection
         subjects affected / exposed
    6 / 52 (11.54%)
    4 / 25 (16.00%)
         occurrences all number
    12
    7
    Urinary tract infection
         subjects affected / exposed
    6 / 52 (11.54%)
    3 / 25 (12.00%)
         occurrences all number
    6
    4
    Bronchitis
         subjects affected / exposed
    5 / 52 (9.62%)
    5 / 25 (20.00%)
         occurrences all number
    6
    7
    Respiratory tract infection
         subjects affected / exposed
    3 / 52 (5.77%)
    1 / 25 (4.00%)
         occurrences all number
    4
    3
    Viral infection
         subjects affected / exposed
    3 / 52 (5.77%)
    3 / 25 (12.00%)
         occurrences all number
    3
    5
    Pneumonia
         subjects affected / exposed
    2 / 52 (3.85%)
    2 / 25 (8.00%)
         occurrences all number
    2
    2
    Viral upper respiratory tract infection
         subjects affected / exposed
    2 / 52 (3.85%)
    2 / 25 (8.00%)
         occurrences all number
    3
    2
    Gastrointestinal viral infection
         subjects affected / exposed
    1 / 52 (1.92%)
    2 / 25 (8.00%)
         occurrences all number
    1
    4
    Pharyngitis
         subjects affected / exposed
    1 / 52 (1.92%)
    4 / 25 (16.00%)
         occurrences all number
    1
    4
    Gastroenteritis
         subjects affected / exposed
    0 / 52 (0.00%)
    3 / 25 (12.00%)
         occurrences all number
    0
    3

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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
    For support, Contact us.
    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.

    European Medicines Agency © 1995-Sat Apr 27 21:24:16 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA