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    Clinical Trial Results:
    An open-label, multicenter, Post-Marketing Requirement study to investigate the safety and tolerability of octaplas™ in the management of pediatric patients who require therapeutic plasma exchange.

    Summary
    EudraCT number
    		 2020-000650-10
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    27 Jan 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    12 Mar 2020
    First version publication date
    12 Mar 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    LAS-213
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01938378
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Octapharma
    Sponsor organisation address
    121 River Street, Hoboken, United States, 07030
    Public contact
    Michael Eppolito, Octapharma, michael.eppolito@octapharma.com
    Scientific contact
    Michael Eppolito, Octapharma, michael.eppolito@octapharma.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
    27 Jan 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    27 Jan 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    27 Jan 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective is to assess the safety and tolerability of octaplas™ in the pediatric patients undergoing therapeutic plasma exchange (TPE) by monitoring ADRs, TEs, TEEs, and by measuring safety laboratory parameters.
    Protection of trial subjects
    This trial was conducted in accordance to the principles of GCP, ensuring that the rights, safety and well-being of patients are protected and in consistency with the Declaration of Helsinki. Inclusion and exclusion criteria were carefully defined in order to protect subjects from contraindications, interactions with other medication and risk factors associated with the investigational medicinal product. Throughout the study safety was assessed, such as occurrence of SAEs, ADRs, TEs, TEEs, and safety labs.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    30 Apr 2015
    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
    United States: 41
    Worldwide total number of subjects
    41
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    15
    Adolescents (12-17 years)
    17
    Adults (18-64 years)
    9
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Male or female patients aged 2 years through 20 years of age requiring therapeutic plasma exchange.

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

    Arms
    Arm title
    		 Octaplas
    Arm description
    		 Therapeutic plasma exchange with Octaplas
    Arm type
    		 Experimental

    Investigational medicinal product name
    Octaplas
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    The recommended dose is a total plasma volume between 40 to 60 mL/kg, as prescribed by the treating physician.

    Number of subjects in period 1
    		Octaplas
    Started
    41
    Completed
    41

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Trial (Overall Period)
    Reporting group description
    		 -

    Reporting group values
    		 Overall Trial (Overall Period) Total
    Number of subjects
    		 41 41
    Age categorical
    Units: Subjects
        Age Group 1 (2- <12)
    		 15 15
        Age Group 2 (12- <17)
    		 13 13
        Age Group 3 (≥17)
    		 13 13
    Age continuous
    Continuous age in years
    Units: years
        arithmetic mean (standard deviation)
    		 12.3 ( 5.40 ) -
    Gender categorical
    Gender of patients
    Units: Subjects
        Female
    		 23 23
        Male
    		 18 18
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 1 1
        Not Hispanic or Latino
    		 39 39
        Unknown or Not Reported
    		 1 1
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 2 2
        Asian
    		 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0
        Black or African American
    		 7 7
        White
    		 32 32
        More than one race
    		 0 0
        Unknown or Not Reported
    		 0 0
    Subject analysis sets

    Subject analysis set title
    Age Group 1 (2-<12)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Ages 2 to <12

    Subject analysis set title
    Age Group 2 (12-<17)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Ages 12 to <17

    Subject analysis set title
    Age Group 3 (≥17)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Ages ≥17

    Subject analysis sets values
    		 Age Group 1 (2-<12) Age Group 2 (12-<17) Age Group 3 (≥17)
    Number of subjects
    15
    13
    13
    Age categorical
    Units: Subjects
        Age Group 1 (2- <12)
    15
    0
    0
        Age Group 2 (12- <17)
    0
    13
    0
        Age Group 3 (≥17)
    0
    0
    13
    Age continuous
    Continuous age in years
    Units: years
        arithmetic mean (standard deviation)
    6.1 ( 2.34 )
    13.8 ( 1.52 )
    18.1 ( 1.04 )
    Gender categorical
    Gender of patients
    Units: Subjects
        Female
    12
    7
    4
        Male
    3
    6
    9
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    0
    0
    1
        Not Hispanic or Latino
    15
    12
    12
        Unknown or Not Reported
    0
    1
    0
    Race
    Units: Subjects
        American Indian or Alaska Native
    0
    2
    0
        Asian
    0
    0
    0
        Native Hawaiian or Other Pacific Islander
    0
    0
    0
        Black or African American
    2
    2
    3
        White
    13
    9
    10
        More than one race
    0
    0
    0
        Unknown or Not Reported
    0
    0
    0

    End points

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    End points reporting groups
    Reporting group title
    Octaplas
    Reporting group description
    		 Therapeutic plasma exchange with Octaplas

    Subject analysis set title
    Age Group 1 (2-<12)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Ages 2 to <12

    Subject analysis set title
    Age Group 2 (12-<17)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Ages 12 to <17

    Subject analysis set title
    Age Group 3 (≥17)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Ages ≥17

    Primary: Monitoring of Adverse Drug Reactions Caused by the Octaplas™Used for Plasma Exchange.

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    End point title
    		 Monitoring of Adverse Drug Reactions Caused by the Octaplas™Used for Plasma Exchange. [1]
    End point description
    End point type
    Primary
    End point timeframe
    up to 8 days including the 24 hour follow-up from treatment
    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: Count of Adverse Drug Reactions
    End point values
    		 Octaplas Age Group 1 (2-<12) Age Group 2 (12-<17) Age Group 3 (≥17)
    Number of subjects analysed
    41
    15
    13
    13
    Units: events
    8
    0
    5
    3
    No statistical analyses for this end point

    Primary: Monitoring of TEs and TEEs Caused by the Octaplas™Used for Plasma Exchange.

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    End point title
    		 Monitoring of TEs and TEEs Caused by the Octaplas™Used for Plasma Exchange. [2]
    End point description
    End point type
    Primary
    End point timeframe
    up to 8 days including the 24 hour follow-up from treatment
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Count of TEs and TEEs
    End point values
    		 Octaplas Age Group 1 (2-<12) Age Group 2 (12-<17) Age Group 3 (≥17)
    Number of subjects analysed
    41
    15
    13
    13
    Units: event
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Assessment of Blood Urea Nitrogen Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Blood Urea Nitrogen Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [3]
    Units: mg/dL
    arithmetic mean (standard deviation)
        TPE 1
    -1.4 ( 6.56 )
        TPE 2
    1.2 ( 4.69 )
        TPE 3
    1.1 ( 3.21 )
        TPE 4
    1.5 ( 2.67 )
        TPE 5
    -0.5 ( 4.36 )
        TPE 6
    -1.0 ( 0 )
    Notes
    [3] - TPE 1 34 subj. TPE 2 17 subj. TPE 3 11 subj. TPE 4 8 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Carbon Dioxide Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Carbon Dioxide Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [4]
    Units: mmol/L
    arithmetic mean (standard deviation)
        TPE 1
    2.2 ( 3.03 )
        TPE 2
    0.5 ( 2.79 )
        TPE 3
    1.2 ( 1.72 )
        TPE 4
    1.0 ( 2.98 )
        TPE 5
    0.3 ( 5.50 )
        TPE 6
    3.0 ( 0 )
    Notes
    [4] - TPE 1 34 subj. TPE 2 17 subj. TPE 3 11 subj. TPE 4 8 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Chloride Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Chloride Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre- TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [5]
    Units: mmol/L
    arithmetic mean (standard deviation)
        TPE 1
    0.5 ( 3.31 )
        TPE 2
    0.6 ( 2.72 )
        TPE 3
    -0.3 ( 2.37 )
        TPE 4
    -0.9 ( 2.59 )
        TPE 5
    1.0 ( 6.78 )
        TPE 6
    -3.0 ( 0 )
    Notes
    [5] - TPE 1 34 subj. TPE 2 17 subj. TPE 3 11 subj. TPE 4 8 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Creatinine Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Creatinine Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [6]
    Units: mg/dl
    arithmetic mean (standard deviation)
        TPE 1
    -0.4 ( 1.10 )
        TPE 2
    -0.1 ( 0.31 )
        TPE 3
    -0.1 ( 0.22 )
        TPE 4
    0.0 ( 0.09 )
        TPE 5
    0.1 ( 0.09 )
        TPE 6
    0.0 ( 0.0 )
    Notes
    [6] - TPE 1 34 subj. TPE 2 17 subj. TPE 3 11 subj. TPE 4 8 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Glucose Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Glucose Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [7]
    Units: mg/dl
    arithmetic mean (standard deviation)
        TPE 1
    -12.0 ( 65.83 )
        TPE 2
    12.5 ( 44.76 )
        TPE 3
    22.6 ( 45.69 )
        TPE 4
    -44.3 ( 158.61 )
        TPE 5
    15.8 ( 38.59 )
        TPE 6
    -2.0 ( 0 )
    Notes
    [7] - TPE 1 34 subj. TPE 2 17 subj. TPE 3 11 subj. TPE 4 8 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Potassium Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Potassium Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [8]
    Units: mmol/L
    arithmetic mean (standard deviation)
        TPE 1
    -0.1 ( 0.54 )
        TPE 2
    0.1 ( 0.26 )
        TPE 3
    0.1 ( 0.49 )
        TPE 4
    -0.2 ( 0.65 )
        TPE 5
    -0.1 ( 0.61 )
        TPE 6
    -0.7 ( 0 )
    Notes
    [8] - TPE 1 34 subj. TPE 2 17 subj. TPE 3 11 subj. TPE 4 8 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Sodium Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Sodium Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [9]
    Units: mmol/L
    arithmetic mean (standard deviation)
        TPE 1
    1.6 ( 2.41 )
        TPE 2
    1.5 ( 1.84 )
        TPE 3
    1.5 ( 2.84 )
        TPE 4
    0.8 ( 1.75 )
        TPE 5
    1.0 ( 2.16 )
        TPE 6
    2.0 ( 0 )
    Notes
    [9] - TPE 1 34 subj. TPE 2 17 subj. TPE 3 11 subj. TPE 4 8 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Leukocyte Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Leukocyte Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [10]
    Units: 10^9/L
    arithmetic mean (standard deviation)
        TPE 1
    0.9 ( 3.80 )
        TPE 2
    1.2 ( 3.93 )
        TPE 3
    -0.5 ( 2.44 )
        TPE 4
    1.2 ( 1.14 )
        TPE 5
    1.6 ( 1.55 )
        TPE 6
    3.3 ( 0 )
    Notes
    [10] - TPE 1 34 subj. TPE 2 18 subj. TPE 3 9 subj. TPE 4 7 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Erythrocyte Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Erythrocyte Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [11]
    Units: 10^12/L
    arithmetic mean (standard deviation)
        TPE 1
    0.1 ( 0.26 )
        TPE 2
    0.1 ( 0.27 )
        TPE 3
    0.1 ( 0.26 )
        TPE 4
    0.1 ( 0.17 )
        TPE 5
    -0.1 ( 0.49 )
        TPE 6
    -0.1 ( 0 )
    Notes
    [11] - TPE 1 34 subj. TPE 2 18 subj. TPE 3 9 subj. TPE 4 7 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Hemoglobin Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Hemoglobin Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [12]
    Units: g/dL
    arithmetic mean (standard deviation)
        TPE 1
    0.4 ( 0.74 )
        TPE 2
    0.3 ( 0.74 )
        TPE 3
    0.3 ( 0.70 )
        TPE 4
    -0.1 ( 0.67 )
        TPE 5
    -0.5 ( 1.44 )
        TPE 6
    -0.4 ( 0 )
    Notes
    [12] - TPE 1 34 subj. TPE 2 18 subj. TPE 3 9 subj. TPE 4 7 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Hematocrit Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Hematocrit Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [13]
    Units: % of the blood that is red blood cells
    arithmetic mean (standard deviation)
        TPE 1
    1.1 ( 2.12 )
        TPE 2
    0.8 ( 2.39 )
        TPE 3
    1.0 ( 2.37 )
        TPE 4
    0.6 ( 1.25 )
        TPE 5
    -1.2 ( 4.03 )
        TPE 6
    -1.6 ( 0 )
    Notes
    [13] - TPE 1 34 subj. TPE 2 18 subj. TPE 3 9 subj. TPE 4 7 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Mean Corpuscular Volume Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Mean Corpuscular Volume Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [14]
    Units: fL
    arithmetic mean (standard deviation)
        TPE 1
    0.2 ( 1.21 )
        TPE 2
    0.3 ( 1.55 )
        TPE 3
    -0.1 ( 1.55 )
        TPE 4
    -0.3 ( 2.01 )
        TPE 5
    -0.1 ( 2.13 )
        TPE 6
    -2.0 ( 0 )
    Notes
    [14] - TPE 1 34 subj. TPE 2 18 subj. TPE 3 9 subj. TPE 4 7 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Mean Corpuscular Hemoglobin Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Mean Corpuscular Hemoglobin Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [15]
    Units: pg
    arithmetic mean (standard deviation)
        TPE 1
    0.1 ( 0.70 )
        TPE 2
    0.1 ( 0.77 )
        TPE 3
    -0.2 ( 0.58 )
        TPE 4
    -0.8 ( 1.18 )
        TPE 5
    -0.4 ( 0.31 )
        TPE 6
    -0.2 ( 0 )
    Notes
    [15] - TPE 1 34 subj. TPE 2 18 subj. TPE 3 9 subj. TPE 4 7 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Mean Corpuscular Hemoglobin Concentration Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Mean Corpuscular Hemoglobin Concentration Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [16]
    Units: g/dL
    arithmetic mean (standard deviation)
        TPE 1
    0.1 ( 0.75 )
        TPE 2
    0 ( 0.97 )
        TPE 3
    -0.2 ( 0.91 )
        TPE 4
    -0.8 ( 1.15 )
        TPE 5
    -0.5 ( 0.62 )
        TPE 6
    0.4 ( 0 )
    Notes
    [16] - TPE 1 34 subj. TPE 2 18 subj. TPE 3 9 subj. TPE 4 7 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Mean Red Cell Distribution Width Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Mean Red Cell Distribution Width Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [17]
    Units: % red blood cell variation volume/size
    arithmetic mean (standard deviation)
        TPE 1
    0.0 ( 0.26 )
        TPE 2
    0.1 ( 0.37 )
        TPE 3
    -0.1 ( 0.33 )
        TPE 4
    0.0 ( 0.25 )
        TPE 5
    0.3 ( 0.52 )
        TPE 6
    -0.2 ( 0 )
    Notes
    [17] - TPE 1 34 subj. TPE 2 18 subj. TPE 3 9 subj. TPE 4 7 subj. TPE 5 4 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Assessment of Mean Ionized Calcium Levels from Pre-TPE to Post-TPE

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    End point title
    		 Assessment of Mean Ionized Calcium Levels from Pre-TPE to Post-TPE
    End point description
    Blood samples compare levels before each TPE (therapeutic plasma exchange) and after each TPE. Pre-TPE is within 24 hours before TPE start; Post-TPE is 30 minutes to 3 hours after TPE end. Differences in number of subjects analyzed for each TPE due to not all patients receiving multiple blood draws/TPEs.
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [18]
    Units: mmol/L
    arithmetic mean (standard deviation)
        TPE 1
    -0.030 ( 0.0927 )
        TPE 2
    0.026 ( 0.1203 )
        TPE 3
    1.026 ( 0.0684 )
        TPE 4
    0.011 ( 0.0995 )
        TPE 5
    -0.032 ( 0.1089 )
        TPE 6
    -0.075 ( 0 )
    Notes
    [18] - TPE 1 28 subj. TPE 2 14 subj. TPE 3 12 subj. TPE 4 8 subj. TPE 5 6 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Secondary: Investigator's Assessment of Overall Safety

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    End point title
    		 Investigator's Assessment of Overall Safety
    End point description
    Excellent: defined as the treatment was well tolerated by the patient; Moderate: defined as ADR(s) were observed, but easily resolved or not clinically significant; Poor: defined as ADR(s) were observed requiring significant medical intervention
    End point type
    Secondary
    End point timeframe
    up to 8 days including the 24 hour follow-up
    End point values
    		 Octaplas
    Number of subjects analysed
    41 [19]
    Units: participants
        Patients Who Underwent TPE 1 - Excellent
    37
        Patients Who Underwent TPE 1 - Moderate
    4
        Patients Who Underwent TPE 1 - Poor
    0
        Patients Who Underwent TPE 2 - Excellent
    25
        Patients Who Underwent TPE 2 - Moderate
    1
        Patients Who Underwent TPE 2 - Poor
    0
        Patients Who Underwent TPE 3 - Excellent
    18
        Patients Who Underwent TPE 3 - Moderate
    0
        Patients Who Underwent TPE 3 - Poor
    0
        Patients Who Underwent TPE 4 - Excellent
    9
        Patients Who Underwent TPE 4 - Moderate
    1
        Patients Who Underwent TPE 4 - Poor
    0
        Patients Who Underwent TPE 5 - Excellent
    6
        Patients Who Underwent TPE 5 - Moderate
    0
        Patients Who Underwent TPE 5 - Poor
    0
        Patients Who Underwent TPE 6 - Excellent
    1
        Patients Who Underwent TPE 6 - Moderate
    0
        Patients Who Underwent TPE 6 - Poor
    0
    Notes
    [19] - TPE 1 41 subj. TPE 2 26 subj. TPE 3 18 subj. TPE 4 10 subj. TPE 5 6 subj. TPE 6 1 subj.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 8 Days
    Adverse event reporting additional description
    ADRs will be elicited using a standard non-leading question such as “How have you been since the last evaluation?” If children or adolescent patients are unable to adequately understand and respond to this question, the information will be obtained from the patient’s guardian and/or Investigator or study nurse.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18
    Reporting groups
    Reporting group title
    Octaplas
    Reporting group description
    		 Therapeutic plasma exchange with Octaplas

    Reporting group title
    Pediatric Patients Undergoing TPE Age Group 1
    Reporting group description
    		 Age Group 1 (2-<12)

    Reporting group title
    Pediatric Patients Undergoing TPE Age Group 2
    Reporting group description
    		 Age Group 2 (12-<17)

    Reporting group title
    Pediatric Patients Undergoing TPE Age Group 3
    Reporting group description
    		 Age Group 3 (≥17)

    Serious adverse events
    		 Octaplas Pediatric Patients Undergoing TPE Age Group 1 Pediatric Patients Undergoing TPE Age Group 2 Pediatric Patients Undergoing TPE Age Group 3
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 15 (6.67%)
    0 / 13 (0.00%)
    0 / 13 (0.00%)
         number of deaths (all causes)
    1
    1
    0
    0
         number of deaths resulting from adverse events
    1
    1
    0
    0
    General disorders and administration site conditions
    Multi-organ Failure
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 15 (6.67%)
    0 / 13 (0.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Octaplas Pediatric Patients Undergoing TPE Age Group 1 Pediatric Patients Undergoing TPE Age Group 2 Pediatric Patients Undergoing TPE Age Group 3
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 41 (9.76%)
    0 / 15 (0.00%)
    3 / 13 (23.08%)
    1 / 13 (7.69%)
    Investigations
    Inflammatory marker increased
         subjects affected / exposed
    1 / 41 (2.44%)
    0 / 15 (0.00%)
    0 / 13 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    1
    0
    0
    1
    Injury, poisoning and procedural complications
    Citrate toxicity
         subjects affected / exposed
    2 / 41 (4.88%)
    0 / 15 (0.00%)
    2 / 13 (15.38%)
    0 / 13 (0.00%)
         occurrences all number
    2
    0
    2
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 41 (2.44%)
    0 / 15 (0.00%)
    1 / 13 (7.69%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    1
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 41 (2.44%)
    0 / 15 (0.00%)
    1 / 13 (7.69%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    1 / 41 (2.44%)
    0 / 15 (0.00%)
    0 / 13 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    1
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Urticaria
         subjects affected / exposed
    1 / 41 (2.44%)
    0 / 15 (0.00%)
    1 / 13 (7.69%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Musculoskeletal and connective tissue disorders
    Myalgia
         subjects affected / exposed
    1 / 41 (2.44%)
    0 / 15 (0.00%)
    0 / 13 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    1
    0
    0
    1

    More information

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    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
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