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    Clinical Trial Results:
    A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

    Summary
    EudraCT number
    2019-000884-26
    Trial protocol
    PL   BG   HU   BE   CZ   AT   ES   NL   GB   FR   GR   IT   HR   RO  
    Global end of trial date
    27 Apr 2022

    Results information
    Results version number
    v2(current)
    This version publication date
    21 Sep 2023
    First version publication date
    05 May 2023
    Other versions
    v1
    Version creation reason

    Trial information

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    Trial identification
    Sponsor protocol code
    TP0003
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04200456
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    UCB Biopharma SRL
    Sponsor organisation address
    Allée de la Recherche 60, Brussels, Belgium, 1070
    Public contact
    Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
    Scientific contact
    Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    05 Oct 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    21 Mar 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    27 Apr 2022
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    Demonstrate the clinical efficacy of rozanolixizumab in maintenance treatment in study participants with primary immune thrombocytopenia
    Protection of trial subjects
    During the conduct of the study all participants were closely monitored.
    Background therapy
    Background therapy as permitted in the protocol.
    Evidence for comparator
    Not applicable
    Actual start date of recruitment
    31 Jan 2020
    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
    France: 1
    Country: Number of subjects enrolled
    Georgia: 4
    Country: Number of subjects enrolled
    Hungary: 3
    Country: Number of subjects enrolled
    Italy: 1
    Country: Number of subjects enrolled
    Japan: 3
    Country: Number of subjects enrolled
    Korea, Republic of: 1
    Country: Number of subjects enrolled
    Moldova, Republic of: 2
    Country: Number of subjects enrolled
    Poland: 10
    Country: Number of subjects enrolled
    Russian Federation: 1
    Country: Number of subjects enrolled
    Taiwan: 1
    Country: Number of subjects enrolled
    Ukraine: 5
    Country: Number of subjects enrolled
    United Kingdom: 1
    Worldwide total number of subjects
    33
    EEA total number of subjects
    15
    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)
    29
    From 65 to 84 years
    4
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The study started to enroll study participants in Jan 2020 and terminated in April 2022.

    Pre-assignment
    Screening details
    Participant Flow refers to the Randomized Set.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Placebo
    Arm description
    Participants received a fixed-unit starting dose of placebo subcutaneous (sc) infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received placebo at prespecified time points.

    Arm title
    Rozanolixizumab
    Arm description
    Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.
    Arm type
    Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received rozanolixizumab at prespecified time points.

    Number of subjects in period 1
    Placebo Rozanolixizumab
    Started
    12
    21
    Completed
    9
    15
    Not completed
    3
    6
         Consent withdrawn by subject
    2
    2
         Physician decision
    -
    1
         Administration of Rescue and concern about IMP
    -
    1
         Adverse event, non-fatal
    -
    1
         Lack of efficacy
    1
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Participants received a fixed-unit starting dose of placebo subcutaneous (sc) infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.

    Reporting group title
    Rozanolixizumab
    Reporting group description
    Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.

    Reporting group values
    Placebo Rozanolixizumab Total
    Number of subjects
    12 21 33
    Age Categorical
    Units: participants
        <=18 years
    0 1 1
        Between 18 and 65 years
    10 18 28
        >=65 years
    2 2 4
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    51.4 ± 15.9 41.4 ± 12.8 -
    Sex: Female, Male
    Units: participants
        Female
    11 12 23
        Male
    1 9 10
    Platelet count
    Units: *10^9/L
        arithmetic mean (standard deviation)
    17.2 ± 11.3 17.0 ± 9.4 -

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Participants received a fixed-unit starting dose of placebo subcutaneous (sc) infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.

    Reporting group title
    Rozanolixizumab
    Reporting group description
    Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.

    Primary: Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50x10^9/L, for at least 8 out of 12 weeks during the last 12 weeks

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    End point title
    Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50x10^9/L, for at least 8 out of 12 weeks during the last 12 weeks [1]
    End point description
    Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10^9/L, for at least 8 out of 12 weeks during the last 12 weeks were reported. Randomized Set consisted of all enrolled study participants who were randomized. No formal analysis was carried out as the program was terminated.
    End point type
    Primary
    End point timeframe
    During the last 12 weeks (Week 13 to Week 25)
    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: No formal statistical hypothesis testing was planned for this study. Results were summarized in tables as descriptive statistics only.
    End point values
    Placebo Rozanolixizumab
    Number of subjects analysed
    12
    21
    Units: Percentage of participants
        number (not applicable)
    0
    19.0
    No statistical analyses for this end point

    Secondary: Cumulative number of weeks with Clinically Meaningful Platelet Response of ≥50×10^9/L over the 24-week Treatment Period

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    End point title
    Cumulative number of weeks with Clinically Meaningful Platelet Response of ≥50×10^9/L over the 24-week Treatment Period
    End point description
    Total number of weeks with platelet counts ≥50×10^9/L over the 24-week Treatment Period of the study (Week 1 to Week 25) were reported. Randomized Set consisted of all enrolled study participants who were randomized. No formal analysis was carried out as the program was terminated.
    End point type
    Secondary
    End point timeframe
    Week 1 up to Week 25
    End point values
    Placebo Rozanolixizumab
    Number of subjects analysed
    12
    21
    Units: Weeks
        median (full range (min-max))
    0.0 (0 to 7)
    3.0 (0 to 24)
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8

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    End point title
    Percentage of Participants with Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8
    End point description
    Clinically meaningful platelet response was defined as platelet count of ≥50×10^9/L. Randomized Set consisted of all enrolled study participants who were randomized. No formal analysis was carried out as the program was terminated.
    End point type
    Secondary
    End point timeframe
    Baseline to Day 8
    End point values
    Placebo Rozanolixizumab
    Number of subjects analysed
    12
    21
    Units: Percentage of participants
        number (not applicable)
    16.7
    52.4
    No statistical analyses for this end point

    Secondary: Time to first Clinically Meaningful Platelet Response (CMPR) of ≥50×10^9/L: time from starting treatment to achievement of first response of ≥50×10^9/L

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    End point title
    Time to first Clinically Meaningful Platelet Response (CMPR) of ≥50×10^9/L: time from starting treatment to achievement of first response of ≥50×10^9/L
    End point description
    Time from starting treatment to achievement of first Clinically Meaningful Platelet Response of ≥50×10^9/L was defined as date of first clinically meaningful response - date of first treatment + 1. Median was calculated based upon the Kaplan-Meier estimate. Randomized Set consisted of all enrolled study participants who were randomized. 999 for placebo arm signifies that upper confidence limit for placebo is not provided for the 95% CI of the median time to first CMPR as there is no time at which the upper bound of the CI for the Kaplan-Meier estimator is less than or equal to 0.5 and 999 for rozanolixizumab arm signifies that upper confidence limit for rozanolixizumab is not provided for the 95% CI of the median time to first CMPR as there is no time at which the upper bound of the CI for the Kaplan-Meier estimator is less than or equal to 0.5.
    End point type
    Secondary
    End point timeframe
    Time from starting treatment to achievement of first response of ≥50×10^9/L (up to Week 25)
    End point values
    Placebo Rozanolixizumab
    Number of subjects analysed
    12
    21
    Units: days
        median (confidence interval 95%)
    44.0 (6.0 to 999)
    8.0 (6.0 to 999)
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Response defined as platelet count ≥30*10^9/L and at least doubling of baseline, at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding

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    End point title
    Percentage of Participants with Response defined as platelet count ≥30*10^9/L and at least doubling of baseline, at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding
    End point description
    Response was defined as platelet count ≥30×10^9/L and at least doubling of baseline, at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding. Randomized Set consisted of all enrolled study participants who were randomized. No formal analysis was carried out as the program was terminated.
    End point type
    Secondary
    End point timeframe
    From Baseline during Treatment Period (up to Week 25)
    End point values
    Placebo Rozanolixizumab
    Number of subjects analysed
    12
    21
    Units: Percentage of participants
        number (not applicable)
    8.3
    33.3
    No statistical analyses for this end point

    Secondary: Time to first rescue therapy

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    End point title
    Time to first rescue therapy
    End point description
    Time to first rescue therapy was defined as date of first rescue therapy use - date of first treatment + 1. Median was calculated based upon Kaplan-Meier estimate. The probability of requiring rescue medication did not reach 0.5 so KM median in the rozanolixizumab arm could not be estimated. Randomized Set consisted of all enrolled study participants who were randomized. 999 for placebo arm signifies that upper confidence limit for placebo is not provided for 95% CI of median time to rescue therapy as there is no time at which upper bound of CI for Kaplan-Meier estimator is less than or equal to 0.5. 999 for median signifies that probability of participants requiring rescue medication did not reach 0.5 so the Kaplan-Meier median could not be estimated. 999 signifies that upper confidence limit for rozanolixizumab is not provided for the 95% CI of median time to rescue therapy as there is no time at which upper bound of CI for the Kaplan-Meier estimator is less than or equal to 0.5.
    End point type
    Secondary
    End point timeframe
    From Baseline to first rescue therapy (up to Week 25)
    End point values
    Placebo Rozanolixizumab
    Number of subjects analysed
    12
    21
    Units: Days
        median (confidence interval 95%)
    34.5 (4.0 to 999)
    999 (23.0 to 999)
    No statistical analyses for this end point

    Secondary: Change from Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score

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    End point title
    Change from Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score
    End point description
    ITP-PAQ version 1 is a 44 item disease-specific Health-Related Quality of Life questionnaire developed for use in adults with chronic ITP. It includes 10 scales as physical health: Symptoms (6 items), Bother (3 items), Fatigue (4 items), Activity (2 items); emotional health: Fear (5 items) and Psychological (5 items); quality of life (QOL): Work QOL (4 items), Social QOL (4 items), Women’s Reproductive QOL (6 items) and Overall QOL (5 items). Each item is rated on a Likert-type scale containing 4 to 7 responses. All item scores are transformed to a 0 to 100 continuum and are weighted equally to derive individual scale scores and total score (0-100) is calculated as per formula: Sum of item scores within the scale/raw sum range*100. Higher scores indicate better health status. Randomized Set: enrolled study participants who were randomized. Number of Participants analyzed signifies participants evaluable for this endpoint. No formal analysis was carried out as program was terminated.
    End point type
    Secondary
    End point timeframe
    From Baseline during Treatment Period (up to Week 25)
    End point values
    Placebo Rozanolixizumab
    Number of subjects analysed
    9
    16
    Units: units on a scale
        arithmetic mean (standard deviation)
    6.9 ± 13.8
    5.5 ± 9.2
    No statistical analyses for this end point

    Secondary: Percentage of Participants With treatment-emergent adverse events (TEAEs)

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    End point title
    Percentage of Participants With treatment-emergent adverse events (TEAEs)
    End point description
    An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose. Safety set included all randomized study participants who received at least one dose of IMP.
    End point type
    Secondary
    End point timeframe
    From Baseline to end of Safety Follow-Up Period (up to Week 31)
    End point values
    Placebo Rozanolixizumab
    Number of subjects analysed
    12
    21
    Units: Percentage of participants
        number (not applicable)
    75.0
    85.7
    No statistical analyses for this end point

    Secondary: Percentage of Participants With TEAEs leading to withdrawal of investigational medicinal product (ie, study discontinuation)

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    End point title
    Percentage of Participants With TEAEs leading to withdrawal of investigational medicinal product (ie, study discontinuation)
    End point description
    An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of IMP, whether or not considered related to the IMP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose. Safety set included all randomized study participants who received at least one dose of IMP.
    End point type
    Secondary
    End point timeframe
    From Baseline to end of Safety Follow-Up Period (up to Week 31)
    End point values
    Placebo Rozanolixizumab
    Number of subjects analysed
    12
    21
    Units: Percentage of participants
        number (not applicable)
    0
    4.8
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Baseline to end of Safety Follow-Up Period (up to Week 31)
    Adverse event reporting additional description
    TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Rozanolixizumab
    Reporting group description
    Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.

    Reporting group title
    Placebo
    Reporting group description
    Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.

    Serious adverse events
    Rozanolixizumab Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 21 (9.52%)
    1 / 12 (8.33%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Vascular disorders
    Haemorrhage
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Immune thrombocytopenia
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 12 (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
    Urethritis
         subjects affected / exposed
    1 / 21 (4.76%)
    0 / 12 (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
    Rozanolixizumab Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    16 / 21 (76.19%)
    9 / 12 (75.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Fatigue
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Pyrexia
         subjects affected / exposed
    9 / 21 (42.86%)
    0 / 12 (0.00%)
         occurrences all number
    34
    0
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    0 / 21 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    3
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Investigations
    Body temperature increased
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    14 / 21 (66.67%)
    5 / 12 (41.67%)
         occurrences all number
    48
    8
    Dizziness
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    3 / 21 (14.29%)
    2 / 12 (16.67%)
         occurrences all number
    12
    2
    Ear and labyrinth disorders
    Tinnitus
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    5 / 21 (23.81%)
    1 / 12 (8.33%)
         occurrences all number
    6
    1
    Vomiting
         subjects affected / exposed
    4 / 21 (19.05%)
    0 / 12 (0.00%)
         occurrences all number
    5
    0
    Constipation
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Abdominal pain
         subjects affected / exposed
    1 / 21 (4.76%)
    1 / 12 (8.33%)
         occurrences all number
    1
    1
    Diarrhoea
         subjects affected / exposed
    2 / 21 (9.52%)
    2 / 12 (16.67%)
         occurrences all number
    9
    2
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    Rash macular
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Infections and infestations
    Rhinitis
         subjects affected / exposed
    2 / 21 (9.52%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    COVID-19
         subjects affected / exposed
    1 / 21 (4.76%)
    2 / 12 (16.67%)
         occurrences all number
    1
    2
    Cystitis
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Enterocolitis infectious
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Respiratory tract infection viral
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Bronchitis
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Metabolism and nutrition disorders
    Hyperglycaemia
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Type 2 diabetes mellitus
         subjects affected / exposed
    0 / 21 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    21 Nov 2019
    The primary purpose of this substantial amendment (21 Nov 2019) was to incorporate the feedback from the United States Food and Drug Administration (FDA) received on 23 October 2019. In addition to updates to provide clarity and consistency within the protocol and administrative revisions, the following modifications were made: • Added details on study stopping rules • Added ADA postdose samples to facilitate clinical validation of drug tolerance in the ADA assay • Eligibility criteria were modified to exclude participants with undiagnosed IgA deficiency and to include patients with moderate renal impairment. • Updated rescue therapy to include any systemic increase in corticosteroids dose above the Baseline dose • Included new wording specific to the predefined order of formal hypotheses testing and the sequence in which testing would be performed • Added an additional estimand for a secondary endpoint • Provided timing for obtaining IgG samples due to interference of other tests • Country-specific requirements: − Information specific to Moldova was no longer applicable to the study. − Updates to Poland were made to align with Polish Health Authority’s and Clinical Trial Facilitation Group recommendations regarding pregnancy testing. • Added new wording that local guidelines should be followed regarding antibiotic prophylaxis in asplenic participants to remind investigators about the importance of antibiotic therapy in management of infections in splenectomized participants.
    29 Sep 2020
    The primary reason for this substantial amendment (29 Sep 2020) was to incorporate changes in the endpoints and the statistical analysis section, and to incorporate agency-required local protocol amendments into 1 global protocol. The country-specific changes were incorporated in Protocol Amendment 2 Appendix. In addition to updates to provide clarity and consistency within the protocol and administrative revisions, the following modifications were made: • Changed the number of additional participants that could be recruited into the study from 75 to 60 (maximum total sample size was changed from 105 to 90) based on revised sample size calculation method and assumptions • Primary analysis (previously incorporated by local Protocol Amendments 1.1, 1.2, and 1.3) − Removed all reference to the Fisher’s Exact test and included as a separate supplemental estimand − Added more details regarding the Cochran-Mantel-Haenszel test • Added details to explain that the interim analysis was to have been conducted on combined data from TP0003 and TP0006, including amendment of the futility stopping rule • Modified study criteria to include study participants who had failed or were intolerant to 2 or more prior ITP therapies per global implementation of an ANSM request and implement feedback received from the FD • Deleted or moved to “other efficacy endpoints” endpoints that did not measure different manifestation of the disease and provided redundant information • Included additional “other” efficacy endpoints • Provided additional wording for clarification on the action taken for study participants on the lowest dose level with a platelet count between >200x109/L and <400x109/L (previously incorporated by local Protocol Amendments 1.2 and 1.3).
    29 Sep 2020
    This includes the continued information from Protocol Amendment 2 • Added that an independent Quantitative Clinical Pharmacologist/Modeling and Simulation Scientist may have access to the randomization code to review unblinded PK, platelet and serum IgG data to allow modelling activities to be started by an independent scientist • Explained that contingency measures during a pandemic and other exceptional circumstances had been included • Increased the number of sites from 50 to 70 • Country-specific requirements: − United States and Canada only: Updated and clarified study stopping rule per FDA request to change the study stopping rule − Japan only (previously incorporated by local Protocol Amendment 1.2): ▪ Added instructions for SAE reporting (investigational device) and device deficiency reporting specific for Japan, in accordance with local regulations in Japan ▪ Added chest X-ray assessment to early withdrawal (EW) visit and EOS visit to confirm safety at study termination ▪ Added the T-SPOT test as a recommended IGRA test in addition to the QuantiFERON test ▪ Included details on the consent requirements for participants aged <20 years of age ▪ Added exclusion criterion relative to partial splenic artery embolization as this procedure might have been used for treatment of ITP in Japan ▪ Removed wording on use of cannabinoids and medicinal marijuana because these drugs are prohibited by law in Japan.
    03 Dec 2021
    The primary reason for this substantial amendment (03 Dec 2021) was to modify the dosing regimen of the study on the recommendation of the IDMC. Only 1 study participant was enrolled under Protocol Amendment 3, and this participant was not treated prior to study termination. Thus, this aCSR, including analysis of the data for this study, was based on the protocol under Amendment 2.

    Interruptions (globally)

    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    24 Mar 2020
    From 24 March 2020 through 04 June 2020, enrollment into the study was temporarily on hold due to the coronavirus disease 2019 (COVID-19) pandemic outbreak.
    05 Jun 2020
    19 Nov 2021
    From 19 Nov 2021, enrollment into the study was temporarily suspended to allow for the development of a protocol amendment (#3) to change the dosing frequency from biweekly to weekly. Reactivation commenced on 19 March 2022 with first screening after the re-start occuring 06 April 2022.
    06 Apr 2022

    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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