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    Clinical Trial Results:
    A MULTICENTER, OPEN-LABEL, MULTIPLE-DOSE STUDY TO EVALUATE THE SAFETY, TOLERABILITY, AND EFFICACY OF UCB7665 IN SUBJECTS WITH PRIMARY IMMUNE THROMBOCYTOPENIA

    Summary
    EudraCT number
    2015-003984-12
    Trial protocol
    DE   CZ   ES   BE   PL   LT   BG   IT  
    Global end of trial date
    04 Feb 2019

    Results information
    Results version number
    v1(current)
    This version publication date
    19 Feb 2020
    First version publication date
    19 Feb 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    TP0001
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02718716
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    UCB Biopharma SPRL
    Sponsor organisation address
    Allée de la Recherche 60, Brussels, Belgium, B-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 Mar 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    04 Feb 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    04 Feb 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of UCB7665 administered by subcutaneous (sc) infusion in patients with immune thrombocytopenia (ITP)
    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
    02 Mar 2016
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Bulgaria: 1
    Country: Number of subjects enrolled
    Czech Republic: 2
    Country: Number of subjects enrolled
    Georgia: 6
    Country: Number of subjects enrolled
    Germany: 5
    Country: Number of subjects enrolled
    Italy: 4
    Country: Number of subjects enrolled
    Moldova, Republic of: 8
    Country: Number of subjects enrolled
    Poland: 27
    Country: Number of subjects enrolled
    Romania: 5
    Country: Number of subjects enrolled
    Spain: 5
    Country: Number of subjects enrolled
    United Kingdom: 3
    Worldwide total number of subjects
    66
    EEA total number of subjects
    52
    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)
    49
    From 65 to 84 years
    16
    85 years and over
    1

    Subject disposition

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    Recruitment
    Recruitment details
    The study started to enroll patients in March 2016 and concluded in February 2019.

    Pre-assignment
    Screening details
    The study included a Screening Period (1 to 28 days), a Dosing Period of 1 to 4 weeks, and an Observation Period of 8 weeks. Participant Flow refers to the Safety Set.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    UCB7665 dose 1
    Arm description
    Participants in this arm received 5 subcutaneous (sc) doses of UCB7665 dose 1 at 1-week intervals.
    Arm type
    Experimental

    Investigational medicinal product name
    UCB7665
    Investigational medicinal product code
    UCB7665
    Other name
    Rozanolixizumab
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 100 mg/mL was administered as a subcutaneous (sc) infusion using an infusion pump which was programmed at a constant flow rate.

    Arm title
    UCB7665 dose 2
    Arm description
    Participants in this arm received 3 subcutaneous (sc) doses of UCB7665 dose 2 at 1-week intervals.
    Arm type
    Experimental

    Investigational medicinal product name
    UCB7665
    Investigational medicinal product code
    UCB7665
    Other name
    Rozanolixizumab
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 100 mg/mL was administered as a subcutaneous (sc) infusion using an infusion pump which was programmed at a constant flow rate.

    Arm title
    UCB7665 dose 3
    Arm description
    Participants in this arm received 2 subcutaneous (sc) doses of UCB7665 dose 3 at 1-week intervals.
    Arm type
    Experimental

    Investigational medicinal product name
    UCB7665
    Investigational medicinal product code
    UCB7665
    Other name
    Rozanolixizumab
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 100 mg/mL was administered as a subcutaneous (sc) infusion using an infusion pump which was programmed at a constant flow rate.

    Arm title
    UCB7665 dose 4
    Arm description
    Participants in this arm received 1 subcutaneous (sc) dose of UCB7665 dose 4.
    Arm type
    Experimental

    Investigational medicinal product name
    UCB7665
    Investigational medicinal product code
    UCB7665
    Other name
    Rozanolixizumab
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 100 mg/mL was administered as a subcutaneous (sc) infusion using an infusion pump which was programmed at a constant flow rate.

    Arm title
    UCB7665 dose 5
    Arm description
    Participants in this arm received 1 subcutaneous (sc) dose of UCB7665 dose 5.
    Arm type
    Experimental

    Investigational medicinal product name
    UCB7665
    Investigational medicinal product code
    UCB7665
    Other name
    Rozanolixizumab
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    UCB7665 100 mg/mL was administered as a subcutaneous (sc) infusion using an infusion pump which was programmed at a constant flow rate.

    Number of subjects in period 1
    UCB7665 dose 1 UCB7665 dose 2 UCB7665 dose 3 UCB7665 dose 4 UCB7665 dose 5
    Started
    15
    15
    12
    12
    12
    Completed
    14
    15
    12
    12
    12
    Not completed
    1
    0
    0
    0
    0
         Lack of efficacy
    1
    -
    -
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    UCB7665 dose 1
    Reporting group description
    Participants in this arm received 5 subcutaneous (sc) doses of UCB7665 dose 1 at 1-week intervals.

    Reporting group title
    UCB7665 dose 2
    Reporting group description
    Participants in this arm received 3 subcutaneous (sc) doses of UCB7665 dose 2 at 1-week intervals.

    Reporting group title
    UCB7665 dose 3
    Reporting group description
    Participants in this arm received 2 subcutaneous (sc) doses of UCB7665 dose 3 at 1-week intervals.

    Reporting group title
    UCB7665 dose 4
    Reporting group description
    Participants in this arm received 1 subcutaneous (sc) dose of UCB7665 dose 4.

    Reporting group title
    UCB7665 dose 5
    Reporting group description
    Participants in this arm received 1 subcutaneous (sc) dose of UCB7665 dose 5.

    Reporting group values
    UCB7665 dose 1 UCB7665 dose 2 UCB7665 dose 3 UCB7665 dose 4 UCB7665 dose 5 Total
    Number of subjects
    15 15 12 12 12 66
    Age categorical
    Units: Subjects
        <=18 years
    0 0 0 0 0 0
        Between 18 and 65 years
    7 14 10 11 7 49
        >=65 years
    8 1 2 1 5 17
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    59.1 ± 18.4 46.0 ± 15.9 46.3 ± 16.8 45.8 ± 14.6 56.1 ± 18.2 -
    Gender categorical
    Units: Subjects
        Male
    7 4 5 5 3 24
        Female
    8 11 7 7 9 42

    End points

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    End points reporting groups
    Reporting group title
    UCB7665 dose 1
    Reporting group description
    Participants in this arm received 5 subcutaneous (sc) doses of UCB7665 dose 1 at 1-week intervals.

    Reporting group title
    UCB7665 dose 2
    Reporting group description
    Participants in this arm received 3 subcutaneous (sc) doses of UCB7665 dose 2 at 1-week intervals.

    Reporting group title
    UCB7665 dose 3
    Reporting group description
    Participants in this arm received 2 subcutaneous (sc) doses of UCB7665 dose 3 at 1-week intervals.

    Reporting group title
    UCB7665 dose 4
    Reporting group description
    Participants in this arm received 1 subcutaneous (sc) dose of UCB7665 dose 4.

    Reporting group title
    UCB7665 dose 5
    Reporting group description
    Participants in this arm received 1 subcutaneous (sc) dose of UCB7665 dose 5.

    Subject analysis set title
    UCB7665 dose 1 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants in this arm received 5 subcutaneous (sc) doses of UCB7665 dose 1 at 1-week intervals. Participants formed the Safety Set (SS).

    Subject analysis set title
    UCB7665 dose 2 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants in this arm received 3 subcutaneous (sc) doses of UCB7665 dose 2 at 1-week intervals. Participants formed the SS.

    Subject analysis set title
    UCB7665 dose 3 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants in this arm received 2 subcutaneous (sc) doses of UCB7665 dose 3 at 1-week intervals. Participants formed the SS.

    Subject analysis set title
    UCB7665 dose 4 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants in this arm received 1 subcutaneous (sc) dose of UCB7665 dose 4. Participants formed the SS.

    Subject analysis set title
    UCB7665 dose 5 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants in this arm received 1 subcutaneous (sc) dose of UCB7665 dose 5. Participants formed the SS.

    Primary: Percentage of participants experiencing at least one Treatment Emergent Adverse Event (TEAE) during the study

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    End point title
    Percentage of participants experiencing at least one Treatment Emergent Adverse Event (TEAE) during the study [1]
    End point description
    TEAEs were defined as Adverse Events starting after the time of first Investigational Medicinal Product (IMP) administration up to and including 8 weeks after the final dose.
    End point type
    Primary
    End point timeframe
    From Visit 2 (Week 1) until End of Study Visit or Early Termination (up to 12 weeks after the first investigational medicinal product (IMP) administration)
    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 as descriptive statistics only.
    End point values
    UCB7665 dose 1 (SS) UCB7665 dose 2 (SS) UCB7665 dose 3 (SS) UCB7665 dose 4 (SS) UCB7665 dose 5 (SS)
    Number of subjects analysed
    15
    15
    12
    12
    12
    Units: percentage of participants
        number (not applicable)
    80.0
    60.0
    58.3
    91.7
    100
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events were collected from Week 1 until the End-of-Study Visit (8 weeks following the final dose)
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    21.1
    Reporting groups
    Reporting group title
    UCB7665 dose 1 (SS)
    Reporting group description
    Participants in this arm received 5 subcutaneous (sc) doses of UCB7665 dose 1 at 1-week intervals. Participants formed the Safety Set (SS).

    Reporting group title
    UCB7665 dose 2 (SS)
    Reporting group description
    Participants in this arm received 3 subcutaneous (sc) doses of UCB7665 dose 2 at 1-week intervals. Participants formed the SS.

    Reporting group title
    UCB7665 dose 3 (SS)
    Reporting group description
    Participants in this arm received 2 subcutaneous (sc) doses of UCB7665 dose 3 at 1-week intervals. Participants formed the SS.

    Reporting group title
    UCB7665 dose 4 (SS)
    Reporting group description
    Participants in this arm received 1 subcutaneous (sc) dose of UCB7665 dose 4. Participants formed the SS.

    Reporting group title
    UCB7665 dose 5 (SS)
    Reporting group description
    Participants in this arm received 1 subcutaneous (sc) dose of UCB7665 dose 5. Participants formed the SS.

    Serious adverse events
    UCB7665 dose 1 (SS) UCB7665 dose 2 (SS) UCB7665 dose 3 (SS) UCB7665 dose 4 (SS) UCB7665 dose 5 (SS)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    1 / 12 (8.33%)
    2 / 12 (16.67%)
    0 / 12 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    Investigations
    Platelet count decreased
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Thrombocytopenia
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 12 (8.33%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Genital haemorrhage
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    UCB7665 dose 1 (SS) UCB7665 dose 2 (SS) UCB7665 dose 3 (SS) UCB7665 dose 4 (SS) UCB7665 dose 5 (SS)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    11 / 15 (73.33%)
    9 / 15 (60.00%)
    7 / 12 (58.33%)
    10 / 12 (83.33%)
    12 / 12 (100.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Hypotension
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 12 (8.33%)
    1 / 12 (8.33%)
    3 / 12 (25.00%)
         occurrences all number
    0
    0
    1
    1
    3
    Asthenia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    1
    0
    Influenza like illness
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    0
    0
    0
    Chest discomfort
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Fatigue
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Infusion site oedema
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    1
    Injection site reaction
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Oedema peripheral
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    0
    0
    0
    1
    Reproductive system and breast disorders
    Menorrhagia
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    2 / 12 (16.67%)
         occurrences all number
    0
    0
    0
    1
    4
    Arthropod bite
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Fall
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Traumatic haematoma
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Investigations
    Neutrophil count decreased
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    3
    Blood creatinine increased
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Blood pressure increased
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Rhinorrhoea
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    0
    0
    0
    Cough
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    1
    Dyspnoea
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Epistaxis
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Rhinitis allergic
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    0
    1
    0
    Immune thrombocytopenic purpura
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 15 (20.00%)
    6 / 15 (40.00%)
    3 / 12 (25.00%)
    5 / 12 (41.67%)
    9 / 12 (75.00%)
         occurrences all number
    6
    7
    4
    6
    9
    Dizziness
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    1
    Eye disorders
    Visual impairment
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    Keratitis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    Ear pain
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Tinnitus
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 15 (6.67%)
    2 / 15 (13.33%)
    1 / 12 (8.33%)
    2 / 12 (16.67%)
    2 / 12 (16.67%)
         occurrences all number
    1
    2
    1
    2
    2
    Vomiting
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    2 / 12 (16.67%)
    4 / 12 (33.33%)
         occurrences all number
    0
    0
    0
    2
    4
    Nausea
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    1 / 12 (8.33%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    1
    1
    0
    Mouth haemorrhage
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    1
    1
    Toothache
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    Abdominal pain
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Abdominal pain upper
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Angina bullosa haemorrhagica
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    1
    Constipation
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Dyspepsia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Gastritis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Haematuria
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Urinary incontinence
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    1
    0
    Skin reaction
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    Alopecia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Joint swelling
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    0
    0
    0
    2
    Arthralgia
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    1
    Myalgia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    Pain in extremity
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    0
    0
    1
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    1 / 12 (8.33%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    1
    1
    1
    1
    0
    Influenza
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    2
    1
    0
    0
    0
    Viral infection
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Respiratory tract infection
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Rhinitis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Urinary tract infection
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 12 (8.33%)
    0 / 12 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    19 May 2016
    The primary purpose of this substantial amendment (dated 19 May 2016) was to include additional laboratory tests (serology test) at the Screening Visit in order to exclude study participants with chronic and ongoing infections with human immunodeficiency virus (HIV), hepatitis B, and hepatitis C. Exclusion criteria related to medical history were updated to define the criteria precisely and increase clarity. Withdrawal criteria for potential drug-induced liver injury (PDILI) and evaluation of PDILI were also updated to enable the effective management and assessment of any PDILI cases as outlined in the Food and Drug Administration Guidance for Industry, Drug-Induced Liver Injury: Premarketing Clinical Evaluation (Jul 2009). UCB has developed prespecified criteria for managing any PDILI events and discontinuing investigational medicinal product (IMP). In this amendment, a more conservative approach compared with the previous version was included. However, there were no changes in the potential risk of PDILI with rozanolixizumab since the previous version. In addition, height was added in order to be able to calculate body mass index (BMI), the serious adverse event (SAE) reporting details were updated, and several additional exploratory biomarkers were added as immunological variables.
    21 Oct 2016
    This substantial amendment (dated 21 Oct 2016) was written to introduce a third cohort of 4 mg/kg subcutaneous (sc) twice weekly. However, based on preliminary emerging data, it was expected that the planned inclusion of a third dose arm with 4 mg/kg of subcutaneous (sc) rozanolixizumab given twice per week would not create additional insight regarding the safety and the tolerability of rozanolixizumab in study participants with immune thrombocytopenia (ITP). Therefore, the planned implementation of Global Protocol Amendment 2.0 was canceled.
    15 Feb 2017
    This substantial amendment (dated 15 Feb 2017) was written to further explore the safety, tolerability, and pharmacodynamic (PD) effect of the same cumulative dose of rozanolixizumab administered with higher doses given in fewer sc infusions by integrating 3 new dose arms into the study. The primary purpose of this amendment was to include 3 additional cohorts (Dose Arms 3, 4, and 5) and a nonmandatory genomic substudy. In addition, the required period for contraceptive use and pregnancy testing was reduced from 3 months to 2 months after the final dose in female study participants of childbearing potential.

    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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