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    Clinical Trial Results:
    An Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

    Summary
    EudraCT number
    2019-000883-40
    Trial protocol
    HU   CZ   BG   PL   DE   GB   IT   RO  
    Global end of trial date
    21 Dec 2022

    Results information
    Results version number
    v2(current)
    This version publication date
    06 Mar 2024
    First version publication date
    22 Dec 2023
    Other versions
    v1
    Version creation reason

    Trial information

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    Trial identification
    Sponsor protocol code
    TP0004
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04596995
    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
    25 Apr 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    21 Dec 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    21 Dec 2022
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    Assess the long-term safety and tolerability of repeated treatment with rozanolixizumab
    Protection of trial subjects
    During the conduct of the study all participants were closely monitored.
    Background therapy
    Not applicable
    Evidence for comparator
    Not applicable
    Actual start date of recruitment
    06 Jan 2021
    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 Kingdom: 1
    Country: Number of subjects enrolled
    United States: 1
    Country: Number of subjects enrolled
    Germany: 1
    Country: Number of subjects enrolled
    Hungary: 2
    Country: Number of subjects enrolled
    Italy: 1
    Country: Number of subjects enrolled
    Poland: 11
    Country: Number of subjects enrolled
    Spain: 3
    Country: Number of subjects enrolled
    Moldova, Republic of: 1
    Country: Number of subjects enrolled
    Ukraine: 10
    Country: Number of subjects enrolled
    Taiwan: 2
    Country: Number of subjects enrolled
    China: 5
    Country: Number of subjects enrolled
    Georgia: 2
    Country: Number of subjects enrolled
    Japan: 1
    Country: Number of subjects enrolled
    Russian Federation: 2
    Worldwide total number of subjects
    43
    EEA total number of subjects
    18
    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)
    41
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The study started to enroll study participants in January 2021 and completed prematurely in December 2022. Study participants from TP0003 (NCT04200456) or TP0006 (NCT04224688) who had completed the 24-week Treatment Period (irrespective of rescue therapy) and met eligibility criteria for TP0004 were enrolled in this study.

    Pre-assignment
    Screening details
    Participant Flow refers to the Enrolled 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
    Arm title
    Rozanolixizumab
    Arm description
    In TP0004, participants received a fixed unit dose of rozanolixizumab subcutaneous (sc) infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers.
    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 as a weight-tiered fix dose by subcutaneous infusion during the Treatment Period.

    Number of subjects in period 1
    Rozanolixizumab
    Started
    43
    Completed
    14
    Not completed
    29
         Enrolled in managed access program
    3
         Consent withdrawn by subject
    12
         Lost to follow-up
    3
         Sponsor decision
    3
         Lack of efficacy
    8

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Rozanolixizumab
    Reporting group description
    In TP0004, participants received a fixed unit dose of rozanolixizumab subcutaneous (sc) infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers.

    Reporting group values
    Rozanolixizumab Total
    Number of subjects
    43 43
    Age Categorical
    Units: participants
        18 - <65 years
    41 41
        >=65 - <85 years
    2 2
        >=85 years
    0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    42.7 ± 13.7 -
    Sex: Female, Male
    Units: participants
        Female
    28 28
        Male
    15 15

    End points

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    End points reporting groups
    Reporting group title
    Rozanolixizumab
    Reporting group description
    In TP0004, participants received a fixed unit dose of rozanolixizumab subcutaneous (sc) infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers.

    Primary: 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) [1]
    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 study participants who received at least 1 dose of IMP (partial or full).
    End point type
    Primary
    End point timeframe
    From Baseline to end of Safety Follow-Up Period (up to Week 60)
    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
    Rozanolixizumab
    Number of subjects analysed
    43
    Units: percentage of Participants
        number (not applicable)
    90.7
    No statistical analyses for this end point

    Primary: Percentage of Participants With TEAEs leading to permanent withdrawal of rozanolixizumab (ie, study discontinuation)

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    End point title
    Percentage of Participants With TEAEs leading to permanent withdrawal of rozanolixizumab (ie, study discontinuation) [2]
    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 study participants who received at least 1 dose of IMP (partial or full).
    End point type
    Primary
    End point timeframe
    From Baseline to end of Safety Follow-Up Period (up to Week 60)
    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: No formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    Rozanolixizumab
    Number of subjects analysed
    43
    Units: percentage of Participants
        number (not applicable)
    0
    No statistical analyses for this end point

    Secondary: Percentage of Participants With Stable Clinically Meaningful Response without rescue therapy at ≥70% of the visits over the planned 52-week Treatment Period starting at Week 4

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    End point title
    Percentage of Participants With Stable Clinically Meaningful Response without rescue therapy at ≥70% of the visits over the planned 52-week Treatment Period starting at Week 4
    End point description
    Stable Clinically Meaningful Response was defined as Clinically Meaningful Response (ie, platelet count ≥50×10^9/L) without rescue therapy at ≥70% of the visits over the planned 52-week Treatment Period starting at Week 4. Safety Set included all study participants who received at least 1 dose of IMP (partial or full).
    End point type
    Secondary
    End point timeframe
    Over the 52-week Treatment Period (starting at Week 4)
    End point values
    Rozanolixizumab
    Number of subjects analysed
    43
    Units: percentage of participants
        number (not applicable)
    16.3
    No statistical analyses for this end point

    Secondary: Change from Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms domain score

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    End point title
    Change from Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms domain score
    End point description
    ITP-PAQ version 1 is a 44 item disease-specific Health-Related QOL 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) & Psychological (5 items); quality of life (QOL): Work QOL (4 items), Social QOL (4 items), Women’s Reproductive QOL (6 items) & Overall QOL (5 items).Each item is rated on a Likert-type scale having 4 to 7 responses.All item scores are transformed to a 0 to 100 continuum & are weighted equally to derive individual scale scores & total score(0-100) is calculated as:Sum of item scores within scale/raw sum range*100.Higher scores=better health status.Analysis set was Safety Set.Number of participants analyzed=participants evaluable for this endpoint & Number analyzed=participants evaluable at specified time points.Weeks 53 and 55 was used for participants who finished study on weekly & biweekly dosing respectively.
    End point type
    Secondary
    End point timeframe
    Weeks 53 or 55, compared to Baseline
    End point values
    Rozanolixizumab
    Number of subjects analysed
    12
    Units: score on a scale
    arithmetic mean (standard deviation)
        Week 53 (n=7)
    2.98 ± 11.21
        Week 55 (n=5)
    4.17 ± 12.50
    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 60)
    Adverse event reporting additional description
    Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (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
    In TP0004, participants received a fixed unit dose of rozanolixizumab sc infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers.

    Serious adverse events
    Rozanolixizumab
    Total subjects affected by serious adverse events
         subjects affected / exposed
    9 / 43 (20.93%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Leiomyoma
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Investigations
    Platelet count decreased
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Joint dislocation
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Radius fracture
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vascular disorders
    Haemorrhage
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Immune thrombocytopenia
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Reproductive system and breast disorders
    Uterine haemorrhage
         subjects affected / exposed
    2 / 43 (4.65%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Skin and subcutaneous tissue disorders
    Purpura
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin haemorrhage
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    2 / 43 (4.65%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Rozanolixizumab
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    31 / 43 (72.09%)
    Investigations
    Body temperature increased
         subjects affected / exposed
    5 / 43 (11.63%)
         occurrences all number
    8
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    3 / 43 (6.98%)
         occurrences all number
    3
    Nervous system disorders
    Headache
         subjects affected / exposed
    18 / 43 (41.86%)
         occurrences all number
    58
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    5 / 43 (11.63%)
         occurrences all number
    6
    Pyrexia
         subjects affected / exposed
    9 / 43 (20.93%)
         occurrences all number
    26
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    3 / 43 (6.98%)
         occurrences all number
    11
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    5 / 43 (11.63%)
         occurrences all number
    5
    Nausea
         subjects affected / exposed
    5 / 43 (11.63%)
         occurrences all number
    9
    Vomiting
         subjects affected / exposed
    3 / 43 (6.98%)
         occurrences all number
    7
    Skin and subcutaneous tissue disorders
    Petechiae
         subjects affected / exposed
    3 / 43 (6.98%)
         occurrences all number
    3
    Rash
         subjects affected / exposed
    3 / 43 (6.98%)
         occurrences all number
    4
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    3 / 43 (6.98%)
         occurrences all number
    3
    Infections and infestations
    COVID-19
         subjects affected / exposed
    7 / 43 (16.28%)
         occurrences all number
    8
    Pharyngitis
         subjects affected / exposed
    4 / 43 (9.30%)
         occurrences all number
    4
    Upper respiratory tract infection
         subjects affected / exposed
    3 / 43 (6.98%)
         occurrences all number
    4

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    11 May 2021
    The primary reason for this substantial protocol amendment (11 May 2021) was to incorporate changes in the objectives, endpoints, the statistical analysis section, as well as a specific study objective and endpoint on post-vaccination biomarkers in study participants who had received a coronavirus disease 2019 (COVID-19) vaccine. Other changes included incorporating local protocol amendments (0.1-Japan and 0.2-France only) into 1 global protocol. Additional updates were incorporated to provide further clarity on the protocol or to correct errors.
    03 Dec 2021
    The primary reason for this substantial protocol amendment (03 Dec 2021) was the recommendation of the external IDMC to modify the dosing regimen of the study. Ongoing study participants receiving the biweekly dosing regimen were required to be switched to the weekly dosing regimen according to Protocol Amendment 2. Note that Protocol Amendment 2 was not submitted to any regulatory authorities prior to issuance of Protocol Amendment 3. Therefore, the implementation of switching all ongoing study participants from biweekly to weekly dosing actually occurred following the approval of Protocol Amendment 3 at the respective study site. With Protocol Amendment 2, the exploratory arm intended to evaluate wider dosing intervals and reference to maintenance dosing was removed. Additional updates were incorporated to provide further clarity on the protocol or to correct errors.

    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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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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