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    Clinical Trial Results:
    A Phase 1, Prospective, Uncontrolled, Open-label, Multicenter, Dose-escalation Study Evaluating the Safety and Pharmacokinetics in Hereditary TTP (Thrombotic Thrombocytopenic Purpura)

    Summary
    EudraCT number
    		 2012-003221-19
    Trial protocol
    		 AT   DE   GB   PL   Outside EU/EEA  
    Global end of trial date
    22 Feb 2016

    Results information
    Results version number
    		 v1(current)
    This version publication date
    28 Aug 2016
    First version publication date
    28 Aug 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    281101
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02216084
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Baxalta Innovations GmbH
    Sponsor organisation address
    Industriestrasse 67, Vienna, Austria, 1220
    Public contact
    Clinical Trial Registries and Results Disclosure, Baxalta Innovations GmbH, ClinicalTrialsDisclosure@baxalta.com
    Scientific contact
    Clinical Trial Registries and Results Disclosure, Baxalta Innovations GmbH, ClinicalTrialsDisclosure@baxalta.com
    Sponsor organisation name
    Baxalta US Inc.
    Sponsor organisation address
    One Baxter Way, Westlake Village, United States, CA 91362-3811
    Public contact
    Clinical Trial Registries and Results Disclosure, Baxalta US Inc., ClinicalTrialsDisclosure@baxalta.com
    Scientific contact
    Clinical Trial Registries and Results Disclosure, Baxalta US Inc., ClinicalTrialsDisclosure@baxalta.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001160-PIP01-11
    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 Feb 2016
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    22 Feb 2016
    Global end of trial reached?
    Yes
    Global end of trial date
    22 Feb 2016
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety of BAX930 following single infusions at doses of 5, 20 and 40 U/kg body weight, including the occurrence of adverse events (serious and non-serious adverse events) and formation of binding and inhibitory antibodies to BAX930.
    Protection of trial subjects
    This study was conducted in accordance with the protocol, the International Conference of Harmonization Guideline for Good Clinical Practice E6 (ICH GCP, April 1996), Title 21 of the US code of Federal Regulations (US CFR), the European Clinical Trial Directive (2001/20/EC) and applicable national and local regulatory requirements. Subjects were to be recruited to the next dose level only after short-term safety had been demonstrated and reviewed by an independent Data Monitoring Committee at the preceding dose level
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    30 Sep 2014
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Austria: 3
    Country: Number of subjects enrolled
    Japan: 2
    Country: Number of subjects enrolled
    Germany: 3
    Country: Number of subjects enrolled
    Poland: 1
    Country: Number of subjects enrolled
    United Kingdom: 5
    Country: Number of subjects enrolled
    United States: 1
    Country: Number of subjects enrolled
    Switzerland: 1
    Worldwide total number of subjects
    16
    EEA total number of subjects
    12
    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)
    2
    Adults (18-64 years)
    14
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Nine sites participated in the study. All sites enrolled subjects. Subjects were to be recruited to the next dose level only after short-term safety had been demonstrated and reviewed by an independent DMC. Reenrollment of subjects completing treatment in cohort 1/2 into cohort 3 would have been possible but no subject was reenrolled across cohorts

    Pre-assignment
    Screening details
    		 A total of 16 subjects were enrolled in the study, accross 9 study sites, of which 15 subjects received the investigational product and completed the study. 1 subject discontinued the study before treatment due to Sponsor Decision, Patient not dosed during time period required by protocol.

    Pre-assignment period milestones
    Number of subjects started
    		 16
    Number of subjects completed
    		 15

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    		 Patient not dosed during time period per protocol: 1
    Period 1
    Period 1 title
    Overall Trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Dose Cohort 1 (5 U/kg)
    Arm description
    		 Subjects will receive a single pharmacokinetic infusion of BAX930 with dosage of 5 U/kg bodyweight
    Arm type
    		 Experimental

    Investigational medicinal product name
    BAX930
    Investigational medicinal product code
    Other name
    rADAMTS13
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Intravenous bolus use
    Dosage and administration details
    5 U/kg bodyweight

    Arm title
    		 Dose Cohort 2 (20 U/kg)
    Arm description
    		 Subjects will receive a single pharmacokinetic infusion of BAX930 with dosage of 20 U/kg bodyweight
    Arm type
    		 Experimental

    Investigational medicinal product name
    BAX930
    Investigational medicinal product code
    Other name
    rADAMTS13
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Intravenous bolus use
    Dosage and administration details
    20 U/kg bodyweight

    Arm title
    		 Dose Cohort 3 (40 U/kg)
    Arm description
    		 Subjects will receive a single pharmacokinetic infusion of BAX930 with dosage of 40 U/kg bodyweight
    Arm type
    		 Experimental

    Investigational medicinal product name
    BAX930
    Investigational medicinal product code
    Other name
    rADAMTS13
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Intravenous bolus use
    Dosage and administration details
    40 U/kg bodyweight

    Number of subjects in period 1 [1]
    		Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Started
    3
    3
    9
    Completed
    3
    3
    9
    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: A total of 16 subjects were enrolled in the study, across 9 study sites, of which 15 subjects received the investigational product and completed the study. 1 subject discontinued the study before treatment due to 'Sponsor Decision, Patient not dosed during time period required by protocol'.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Dose Cohort 1 (5 U/kg)
    Reporting group description
    		 Subjects will receive a single pharmacokinetic infusion of BAX930 with dosage of 5 U/kg bodyweight

    Reporting group title
    Dose Cohort 2 (20 U/kg)
    Reporting group description
    		 Subjects will receive a single pharmacokinetic infusion of BAX930 with dosage of 20 U/kg bodyweight

    Reporting group title
    Dose Cohort 3 (40 U/kg)
    Reporting group description
    		 Subjects will receive a single pharmacokinetic infusion of BAX930 with dosage of 40 U/kg bodyweight

    Reporting group values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg) Total
    Number of subjects
    		 3 3 9 15
    Age categorical
    Units: Subjects
        Adults (18-65 years)
    		 3 3 7 13
        Adolescents (12-17 years)
    		 0 0 2 2
    Gender categorical
    Units:
        Male
    		 1 1 5 7
        Female
    		 2 2 4 8
    Subject analysis sets

    Subject analysis set title
    Pharmacokinetic full analysis set (PK-FAS)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All subjects who received at least one dose of the investigational product and provided at least 4 valid data points from scheduled PK timepoints after start of dosing with BAX 930.

    Subject analysis set title
    Pharmacokinetic per protocol set (PK-PPS)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All subjects in the PK-FAS with no major protocol deviations or events that could have affected the integrity of the PK data.

    Subject analysis set title
    Safety analyis set (SAF)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All subjects who received at least one dose of the investigational product.

    Subject analysis sets values
    		 Pharmacokinetic full analysis set (PK-FAS) Pharmacokinetic per protocol set (PK-PPS) Safety analyis set (SAF)
    Number of subjects
    15
    15
    15
    Age categorical
    Units: Subjects
        Adults (18-65 years)
    13
    13
    13
        Adolescents (12-17 years)
    2
    2
    2
    Age continuous
    Units: years
        log mean
    ( )
    ( )
    ( )
    Gender categorical
    Units:
        Male
    7
    7
    7
        Female
    8
    8
    8

    End points

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    End points reporting groups
    Reporting group title
    Dose Cohort 1 (5 U/kg)
    Reporting group description
    		 Subjects will receive a single pharmacokinetic infusion of BAX930 with dosage of 5 U/kg bodyweight

    Reporting group title
    Dose Cohort 2 (20 U/kg)
    Reporting group description
    		 Subjects will receive a single pharmacokinetic infusion of BAX930 with dosage of 20 U/kg bodyweight

    Reporting group title
    Dose Cohort 3 (40 U/kg)
    Reporting group description
    		 Subjects will receive a single pharmacokinetic infusion of BAX930 with dosage of 40 U/kg bodyweight

    Subject analysis set title
    Pharmacokinetic full analysis set (PK-FAS)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All subjects who received at least one dose of the investigational product and provided at least 4 valid data points from scheduled PK timepoints after start of dosing with BAX 930.

    Subject analysis set title
    Pharmacokinetic per protocol set (PK-PPS)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All subjects in the PK-FAS with no major protocol deviations or events that could have affected the integrity of the PK data.

    Subject analysis set title
    Safety analyis set (SAF)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All subjects who received at least one dose of the investigational product.

    Primary: Occurrence of Adverse Events (serious and non-serious AEs) and formation of binding and inhibitory antibodies to BAX 930

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    End point title
    		 Occurrence of Adverse Events (serious and non-serious AEs) and formation of binding and inhibitory antibodies to BAX 930 [1]
    End point description
    To evaluate the safety of BAX930 following single infusion at doses 5, 20 and 40 U/kg bodyweight.
    End point type
    Primary
    End point timeframe
    Up to 28 (+/- 3) days after investigational product infusion.
    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: Per protocol, only descriptive statistics were collected for this endpoint.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg) Safety analyis set (SAF)
    Number of subjects analysed
    3
    3
    9
    15
    Units: Number of events
        Treatment Emergent Adverse Events (TEAEs)
    17
    9
    13
    39
        Severe TEAEs
    0
    0
    0
    0
        TEAEs Related to Investigational Product
    0
    0
    5
    5
        TEAEs Related to Study Procedure
    0
    1
    3
    4
        Treatment Emergent Serious Adverse Events (TESAEs)
    0
    0
    0
    0
        TEAEs Leading to Discontinuation of Study
    0
    0
    0
    0
        TEAEs Leading to Discont./Interrup. of IP
    0
    0
    0
    0
        TEAEs Leading to Death
    0
    0
    0
    0
        Breaktrough TEAEs
    0
    0
    0
    0
        TESAEs Related to Investigational Product
    0
    0
    0
    0
        Positive Anti-ADAMTS13-antibodies (neutralizing)
    0
    0
    0
    0
        Positive Anti-BAX930-antibodies (binding)
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: PK Parameter: Incremental Recovery (IR) for ADAMTS13 activity

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    End point title
    		 PK Parameter: Incremental Recovery (IR) for ADAMTS13 activity
    End point description
    PK parameter were evaluated after a single infusion of BAX930 in Cohorts 1, 2 and 3. The population consisted of adult subjects only (2 adolescent subjects are not included due to sparse PK sampling scheme).
    End point type
    Secondary
    End point timeframe
    Pharmacokinetic samples were taken within 1 hour pre-infusion and up to 288 (+/- 4) hours post-infusion.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Number of subjects analysed
    3
    3
    7
    Units: U/mL*kg/U
    geometric mean (geometric coefficient of variation)
        Technozym Assay
    0.0169 ( 5.5 )
    0.0176 ( 33.7 )
    0.0205 ( 22.7 )
        FRETS-VWF73 Assay
    0.0153 ( 20.1 )
    0.02 ( 34.1 )
    0.0232 ( 14.9 )
    No statistical analyses for this end point

    Secondary: PK Parameter: Incremental Recovery (IR) for ADAMTS13 antigen

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    End point title
    		 PK Parameter: Incremental Recovery (IR) for ADAMTS13 antigen
    End point description
    PK parameter were evaluated after a single infusion of BAX930 in Cohorts 1, 2 and 3. The population consisted of adult subjects only (2 adolescent subjects are not included due to sparse PK sampling scheme).
    End point type
    Secondary
    End point timeframe
    Pharmacokinetic samples were taken within 1 hour pre-infusion and up to 288 (+/- 4) hours post-infusion.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Number of subjects analysed
    3
    3
    7
    Units: µg/mL*kg/µg
        geometric mean (geometric coefficient of variation)
    0.018 ( 6.5 )
    0.0241 ( 35.8 )
    0.0244 ( 16.6 )
    No statistical analyses for this end point

    Secondary: PK Parameter: Maximum concentration following infusion (Cmax) for ADAMTS13 activity

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    End point title
    		 PK Parameter: Maximum concentration following infusion (Cmax) for ADAMTS13 activity
    End point description
    PK parameter were evaluated after a single infusion of BAX930 in Cohorts 1, 2 and 3. The population consisted of adult subjects only (2 adolescent subjects are not included due to sparse PK sampling scheme).
    End point type
    Secondary
    End point timeframe
    Pharmacokinetic samples were taken within 1 hour pre-infusion and up to 288 (+/- 4) hours post-infusion.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Number of subjects analysed
    3
    3
    7
    Units: U/mL
    geometric mean (geometric coefficient of variation)
        Technozym Assay
    0.087 ( 4.6 )
    0.35 ( 34.5 )
    0.837 ( 20.5 )
        FRETS-VWF73 Assay
    0.079 ( 22.2 )
    0.398 ( 35.3 )
    0.948 ( 15.1 )
    No statistical analyses for this end point

    Secondary: PK Parameter: Maximum concentration following infusion (Cmax) for ADAMTS13 antigen

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    End point title
    		 PK Parameter: Maximum concentration following infusion (Cmax) for ADAMTS13 antigen
    End point description
    PK parameter were evaluated after a single infusion of BAX930 in Cohorts 1, 2 and 3. The population consisted of adult subjects only (2 adolescent subjects are not included due to sparse PK sampling scheme).
    End point type
    Secondary
    End point timeframe
    Pharmacokinetic samples were taken within 1 hour pre-infusion and up to 288 (+/- 4) hours post-infusion.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Number of subjects analysed
    3
    3
    7
    Units: µg/mL
        geometric mean (geometric coefficient of variation)
    0.065 ( 3.6 )
    0.323 ( 37.1 )
    0.672 ( 14.7 )
    No statistical analyses for this end point

    Secondary: PK Parameter: Maximum time to reach Cmax (tmax)

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    End point title
    		 PK Parameter: Maximum time to reach Cmax (tmax)
    End point description
    PK parameter were evaluated after a single infusion of BAX930 in Cohorts 1, 2 and 3. The population consisted of adult subjects only (2 adolescent subjects are not included due to sparse PK sampling scheme).
    End point type
    Secondary
    End point timeframe
    Pharmacokinetic samples were taken within 1 hour pre-infusion and up to 288 (+/- 4) hours post-infusion.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Number of subjects analysed
    3
    3
    7
    Units: hours
    median (full range (min-max))
        ADAMTS13 Technozym
    0.25 (0.25 to 0.52)
    0.55 (0.28 to 0.97)
    0.3 (0.25 to 0.58)
        ADAMTS13 FRETS-VWF73
    1 (0.52 to 1)
    0.33 (0.25 to 0.53)
    0.37 (0.22 to 0.58)
        ADAMTS13:Ag
    0.25 (0.25 to 0.52)
    0.55 (0.53 to 0.97)
    0.3 (0.22 to 1.12)
    No statistical analyses for this end point

    Secondary: PK Parameter: Terminal or disposition half-life (t1/2)

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    End point title
    		 PK Parameter: Terminal or disposition half-life (t1/2)
    End point description
    PK parameter were evaluated after a single infusion of BAX930 in Cohorts 1, 2 and 3. The population consisted of adult subjects only (2 adolescent subjects are not included due to sparse PK sampling scheme).
    End point type
    Secondary
    End point timeframe
    Pharmacokinetic samples were taken within 1 hour pre-infusion and up to 288 (+/- 4) hours post-infusion.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Number of subjects analysed
    3 [2]
    3
    7
    Units: hours
    geometric mean (geometric coefficient of variation)
        ADAMTS13 Technozym
    88.6 ( 108 )
    59.6 ( 21.2 )
    60.7 ( 19.6 )
        ADAMTS13 FRETS-VWF73
    999999 ( 999999 )
    42.1 ( 46.7 )
    59.2 ( 23.6 )
        ADAMTS13:Ag
    86.3 ( 40.9 )
    57.1 ( 32.6 )
    66.2 ( 20.5 )
    Notes
    [2] - For ADAMTS13 FRETS-VWF73 n=1. PK Parameter estimation not possible. 999999 was entered.
    No statistical analyses for this end point

    Secondary: PK Parameter: Mean residence time (MRT(0-inf))

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    End point title
    		 PK Parameter: Mean residence time (MRT(0-inf))
    End point description
    PK parameter were evaluated after a single infusion of BAX930 in Cohorts 1, 2 and 3. The population consisted of adult subjects only (2 adolescent subjects are not included due to sparse PK sampling scheme).
    End point type
    Secondary
    End point timeframe
    Pharmacokinetic samples were taken within 1 hour pre-infusion and up to 288 (+/- 4) hours post-infusion.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Number of subjects analysed
    3 [3]
    3
    7
    Units: hours
    geometric mean (geometric coefficient of variation)
        ADAMTS13 Technozym
    133.992 ( 115.4 )
    87.849 ( 28.7 )
    83.936 ( 24.8 )
        ADAMTS13 FRETS-VWF73
    999999 ( 999999 )
    58.464 ( 43.2 )
    84.178 ( 31.9 )
        ADAMTS13:Ag
    123.954 ( 49.7 )
    81.415 ( 41.5 )
    86.27 ( 23.7 )
    Notes
    [3] - For ADAMTS13 FRETS-VWF73 n=1. PK Parameter estimation not possible. 999999 was entered.
    No statistical analyses for this end point

    Secondary: PK Parameter: Systemic clearance (CL)

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    End point title
    		 PK Parameter: Systemic clearance (CL)
    End point description
    PK parameter were evaluated after a single infusion of BAX930 in Cohorts 1, 2 and 3. The population consisted of adult subjects only (2 adolescent subjects are not included due to sparse PK sampling scheme).
    End point type
    Secondary
    End point timeframe
    Pharmacokinetic samples were taken within 1 hour pre-infusion and up to 288 (+/- 4) hours post-infusion.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Number of subjects analysed
    3 [4]
    3
    7
    Units: mL/h
    geometric mean (geometric coefficient of variation)
        ADAMTS13 Technozym
    39.4 ( 126.9 )
    61.6 ( 42.4 )
    67.4 ( 28.2 )
        ADAMTS13 FRETS-VWF73
    999999 ( 999999 )
    70.2 ( 33.2 )
    62 ( 33.6 )
        ADAMTS13:Ag
    46.9 ( 50 )
    49.2 ( 48.3 )
    61.5 ( 32.9 )
    Notes
    [4] - For ADAMTS13 FRETS-VWF73 n=1. PK Parameter estimation not possible. 999999 was entered.
    No statistical analyses for this end point

    Secondary: PK Parameter: Steady state volume of distribution(Vss)

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    End point title
    		 PK Parameter: Steady state volume of distribution(Vss)
    End point description
    PK parameter were evaluated after a single infusion of BAX930 in Cohorts 1, 2 and 3. The population consisted of adult subjects only (2 adolescent subjects are not included due to sparse PK sampling scheme).
    End point type
    Secondary
    End point timeframe
    Pharmacokinetic samples were taken within 1 hour pre-infusion and up to 288 (+/- 4) hours post-infusion.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Number of subjects analysed
    3 [5]
    3
    7
    Units: mL
    geometric mean (geometric coefficient of variation)
        ADAMTS13 Technozym
    5280 ( 14.2 )
    5410 ( 26.7 )
    5650 ( 33.5 )
        ADAMTS13 FRETS-VWF73
    999999 ( 999999 )
    4110 ( 33.3 )
    5220 ( 19.3 )
        ADAMTS13:Ag
    5810 ( 35.2 )
    4010 ( 36 )
    5300 ( 30.5 )
    Notes
    [5] - For ADAMTS13 FRETS-VWF73 n=1. PK Parameter estimation not possible. 999999 was entered.
    No statistical analyses for this end point

    Secondary: PK Parameter: Area under the plasma-time concentration curve from zero to infinity (AUC(0-inf)) for ADAMTS13 activity

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    End point title
    		 PK Parameter: Area under the plasma-time concentration curve from zero to infinity (AUC(0-inf)) for ADAMTS13 activity
    End point description
    PK parameter were evaluated after a single infusion of BAX930 in Cohorts 1, 2 and 3. The population consisted of adult subjects only (2 adolescent subjects are not included due to sparse PK sampling scheme).
    End point type
    Secondary
    End point timeframe
    Pharmacokinetic samples were taken within 1 hour pre-infusion and up to 288 (+/- 4) hours post-infusion.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Number of subjects analysed
    3 [6]
    3
    7
    Units: U*h/mL
    geometric mean (geometric coefficient of variation)
        ADAMTS13 Technozym
    7.94 ( 81.1 )
    21.7 ( 17.4 )
    48.9 ( 28.5 )
        ADAMTS13 FRETS-VWF73
    999999 ( 999999 )
    19.1 ( 24.8 )
    53.1 ( 24.6 )
    Notes
    [6] - For ADAMTS13 FRETS-VWF73 n=1. PK Parameter estimation not possible. 999999 was entered.
    No statistical analyses for this end point

    Secondary: PK Parameter: Area under the plasma-time concentration curve from zero to infinity (AUC(0-inf)) for ADAMTS13 antigen

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    End point title
    		 PK Parameter: Area under the plasma-time concentration curve from zero to infinity (AUC(0-inf)) for ADAMTS13 antigen
    End point description
    PK parameter were evaluated after a single infusion of BAX930 in Cohorts 1, 2 and 3. The population consisted of adult subjects only (2 adolescent subjects are not included due to sparse PK sampling scheme).
    End point type
    Secondary
    End point timeframe
    Pharmacokinetic samples were taken within 1 hour pre-infusion and up to 288 (+/- 4) hours post-infusion.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Number of subjects analysed
    3
    3
    7
    Units: µg*h/mL
        geometric mean (geometric coefficient of variation)
    4.66 ( 20.7 )
    18.3 ( 9.4 )
    36 ( 29.5 )
    No statistical analyses for this end point

    Secondary: PK Parameter: Area under the plasma-time concentration curve from zero to the last measured timepoint (AUC(0-t)) for ADAMTS13 activity

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    End point title
    		 PK Parameter: Area under the plasma-time concentration curve from zero to the last measured timepoint (AUC(0-t)) for ADAMTS13 activity
    End point description
    PK parameter were evaluated after a single infusion of BAX930 in Cohorts 1, 2 and 3. The population consisted of adult subjects only (2 adolescent subjects are not included due to sparse PK sampling scheme).
    End point type
    Secondary
    End point timeframe
    Pharmacokinetic samples were taken within 1 hour pre-infusion and up to 288 (+/- 4) hours post-infusion.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Number of subjects analysed
    3
    3
    7
    Units: U*h/mL
    geometric mean (geometric coefficient of variation)
        ADAMTS13 Technozym
    6.12 ( 35.4 )
    20.3 ( 23.6 )
    46.6 ( 26.4 )
        ADAMTS13 FRETS-VWF73
    0.325 ( 440.6 )
    15.3 ( 31.3 )
    47.8 ( 25.4 )
    No statistical analyses for this end point

    Secondary: PK Parameter: Area under the plasma-time concentration curve from zero to the last measured timepoint (AUC(0-t)) for ADAMTS13 antigen

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    End point title
    		 PK Parameter: Area under the plasma-time concentration curve from zero to the last measured timepoint (AUC(0-t)) for ADAMTS13 antigen
    End point description
    PK parameter were evaluated after a single infusion of BAX930 in Cohorts 1, 2 and 3. The population consisted of adult subjects only (2 adolescent subjects are not included due to sparse PK sampling scheme).
    End point type
    Secondary
    End point timeframe
    Pharmacokinetic samples were taken within 1 hour pre-infusion and up to 288 (+/- 4) hours post-infusion.
    End point values
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 2 (20 U/kg) Dose Cohort 3 (40 U/kg)
    Number of subjects analysed
    3
    3
    7
    Units: µg*h/mL
        geometric mean (geometric coefficient of variation)
    4.03 ( 11.2 )
    17.2 ( 12.7 )
    34.1 ( 27.2 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    For each subject from first exposure of BAX930 until completion visit (28 +/- 3 days). Overall study period (all subjects) was 1 year and 5 months.
    Adverse event reporting additional description
    The population consisted of subjects who received at least one dose of BAX930.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.1
    Reporting groups
    Reporting group title
    Dose Cohort 1 (5 U/kg)
    Reporting group description
    		 Subjects will receive a single pharmacokinetic infusion of BAX930 with dosage of 5 U/kg bodyweight

    Reporting group title
    Dose Cohort 3 (40 U/kg)
    Reporting group description
    		 Subjects will receive a single pharmacokinetic infusion of BAX930 with dosage of 40 U/kg bodyweight

    Reporting group title
    Dose Cohort 2 (20 U/kg)
    Reporting group description
    		 Subjects will receive a single pharmacokinetic infusion of BAX930 with dosage of 20 U/kg bodyweight

    Serious adverse events
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 3 (40 U/kg) Dose Cohort 2 (20 U/kg)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    0 / 3 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Dose Cohort 1 (5 U/kg) Dose Cohort 3 (40 U/kg) Dose Cohort 2 (20 U/kg)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 3 (100.00%)
    7 / 9 (77.78%)
    2 / 3 (66.67%)
    Investigations
    Hemoglobin Decreased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Von Willebrand's Factor Activity Decreased
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Von Willebrand's Factor Antigen Decreased
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    6
    0
    3
    Dizziness
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 9 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    1
    1
    0
    Paraesthesia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    4
    0
    0
    Facial Paresis
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Migraine
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Visual Field Defect
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Feeling Hot
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Oedema Peripheral
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    2
    0
    Abdominal Pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Flatulence
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Gastrooesophageal Reflux Disease
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 9 (11.11%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Vomiting
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal Pain
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Urticaria
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Muscle Tightness
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 9 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    1 / 3 (33.33%)
    2 / 9 (22.22%)
    0 / 3 (0.00%)
         occurrences all number
    1
    2
    0
    Pharyngitis
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 9 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    30 Jul 2015
    Exclusion criterion 5 corrected to reflect autoimmune disorders instead of hematological disorders; Excl. 6 clarified which subjects with neurological events may be included in the study; Excl. 12 updated to clarify the use of hydrocortisone administration; Excl. 17 updated to clarify the reference was to mental disorder and not any disorder; section 6.2 Clinical condition/indication updated; 6.5 Evaluation of anticipated risks and benefits of the IP to human subjects updated; 8.6 Study Stopping rules - stopping rule was added; added that IgE antibodies to be performed for any subject experiencing allergic reactions; under medications text was added to clarify the FFP administration timepoints; added that at screening CD4 levels were to be tested for all subjects; clarify the start of recording of AEs and which AEs and SAEs were not to be included in the analysis; clarified timepoints for weight/height measurements; clarified periods during which FFP and ADAMTS13 containing products were to be withheld; Cardiac troponin I biomarker added to flowdiagram; Bicarbonate, protein, albumin added to assessments; updates according new protocol template for text in sections: withdrawal and completion/discontinuation, SAEs, urgent safety measures and nonmedical complaints

    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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