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    Clinical Trial Results:
    An Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

    Summary
    EudraCT number
    2018-004392-12
    Trial protocol
    GB   DE   FR   BE   ES   NL   DK  
    Global end of trial date
    10 Nov 2021

    Results information
    Results version number
    v1(current)
    This version publication date
    22 Oct 2022
    First version publication date
    22 Oct 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CIDP04
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04051944
    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
    31 Jan 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    10 Nov 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    10 Nov 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Assess long-term safety and tolerability of rozanolixizumab in participants with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP)
    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
    21 Aug 2019
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Efficacy, Safety
    Long term follow-up duration
    19 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Belgium: 4
    Country: Number of subjects enrolled
    Denmark: 2
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    Germany: 2
    Country: Number of subjects enrolled
    Netherlands: 1
    Country: Number of subjects enrolled
    United Kingdom: 1
    Country: Number of subjects enrolled
    United States: 9
    Worldwide total number of subjects
    21
    EEA total number of subjects
    11
    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)
    14
    From 65 to 84 years
    7
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The study started to enroll study participants in Aug 2019 and concluded in Nov 2021.

    Pre-assignment
    Screening details
    Participant Flow refers to Enrolled Set. Participants from CIDP01 (NCT03861481) who had completed Treatment Period without a relapse of CIDP were directly enrolled into this study. Newly treated participants are participants treated with placebo in parent study. Previously treated participants are participants treated with RLZ in parent study.

    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
    Rozanolixizumab (Newly Treated)
    Arm description
    Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.
    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 Dose A at prespecified time points.

    Arm title
    Rozanolixizumab (Previously Treated)
    Arm description
    Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.
    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 Dose A at prespecified time points.

    Number of subjects in period 1
    Rozanolixizumab (Newly Treated) Rozanolixizumab (Previously Treated)
    Started
    11
    10
    Completed
    3
    6
    Not completed
    8
    4
         Study ending
    1
    1
         Lack of efficacy
    3
    -
         Consent withdrawn by subject
    2
    2
         Adverse event, non- fatal
    1
    1
         Early study termination by participant
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Rozanolixizumab (Newly Treated)
    Reporting group description
    Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.

    Reporting group title
    Rozanolixizumab (Previously Treated)
    Reporting group description
    Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.

    Reporting group values
    Rozanolixizumab (Newly Treated) Rozanolixizumab (Previously Treated) Total
    Number of subjects
    11 10 21
    Age Categorical
    Units: participants
        <=18 years
    0 0 0
        Between 18 and 65 years
    8 6 14
        >=65 years
    3 4 7
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    59.8 ± 5.1 59.1 ± 15.9 -
    Sex: Female, Male
    Units: participants
        Female
    4 6 10
        Male
    7 4 11

    End points

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    End points reporting groups
    Reporting group title
    Rozanolixizumab (Newly Treated)
    Reporting group description
    Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.

    Reporting group title
    Rozanolixizumab (Previously Treated)
    Reporting group description
    Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.

    Primary: Number of participants with treatment-emergent adverse event (TEAEs)

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    End point title
    Number of participants with treatment-emergent adverse event (TEAEs) [1]
    End point description
    An Adverse Event (AE) is any untoward medical occurrence in a participant or clinical investigation subject administered a pharmaceutical product, which does not necessarily had a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A TEAE was defined as any event that was not present prior the first administration of investigational medicinal product (IMP) in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study. The Safety Set (SS) consisted of all enrolled study participants who were administered at least one dose of rozanolixizumab in CIDP04.
    End point type
    Primary
    End point timeframe
    From Baseline until Follow-Up Visit (up to Week 84)
    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
    Rozanolixizumab (Newly Treated) Rozanolixizumab (Previously Treated)
    Number of subjects analysed
    11
    10
    Units: participants
    10
    10
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Baseline until Follow-Up Visit (up to Week 84)
    Adverse event reporting additional description
    A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Rozanolixizumab (Newly Treated)
    Reporting group description
    Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.

    Reporting group title
    Rozanolixizumab (Previously Treated)
    Reporting group description
    Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.

    Serious adverse events
    Rozanolixizumab (Newly Treated) Rozanolixizumab (Previously Treated)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 11 (18.18%)
    2 / 10 (20.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Malignant melanoma
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Chronic inflammatory demyelinating polyradiculoneuropathy
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sensory loss
         subjects affected / exposed
    1 / 11 (9.09%)
    0 / 10 (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
    Urinary tract infection
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 10 (10.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Rozanolixizumab (Newly Treated) Rozanolixizumab (Previously Treated)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    10 / 11 (90.91%)
    9 / 10 (90.00%)
    Injury, poisoning and procedural complications
    Muscle strain
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 10 (20.00%)
         occurrences all number
    1
    2
    Fall
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 10 (20.00%)
         occurrences all number
    0
    4
    Investigations
    Blood immunoglobulin G decreased
         subjects affected / exposed
    4 / 11 (36.36%)
    4 / 10 (40.00%)
         occurrences all number
    10
    8
    Respiratory, thoracic and mediastinal disorders
    Rhinorrhoea
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 10 (10.00%)
         occurrences all number
    1
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    4 / 11 (36.36%)
    1 / 10 (10.00%)
         occurrences all number
    7
    5
    Neuralgia
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 10 (20.00%)
         occurrences all number
    1
    2
    Sensory loss
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 10 (0.00%)
         occurrences all number
    3
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Fatigue
         subjects affected / exposed
    3 / 11 (27.27%)
    3 / 10 (30.00%)
         occurrences all number
    3
    4
    Peripheral swelling
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 10 (10.00%)
         occurrences all number
    1
    1
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 10 (20.00%)
         occurrences all number
    0
    2
    Diarrhoea
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 10 (10.00%)
         occurrences all number
    2
    1
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 10 (10.00%)
         occurrences all number
    1
    1
    Musculoskeletal and connective tissue disorders
    Joint swelling
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 10 (20.00%)
         occurrences all number
    1
    2
    Muscle spasms
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 10 (20.00%)
         occurrences all number
    0
    2
    Muscular weakness
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 10 (0.00%)
         occurrences all number
    4
    0
    Back pain
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 10 (10.00%)
         occurrences all number
    1
    1
    Infections and infestations
    Influenza
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Urinary tract infection
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 11 (18.18%)
    0 / 10 (0.00%)
         occurrences all number
    2
    0
    Nasopharyngitis
         subjects affected / exposed
    2 / 11 (18.18%)
    2 / 10 (20.00%)
         occurrences all number
    2
    2

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    12 Jul 2019
    The purpose of this substantial protocol amendment was to provide clarification on the dose of IMP administered (including an allowance of ±10% compared to the target Dose A arm), as well as a flexible infusion rate. Sensitivity analyses were introduced to account for deviation outside the 10% target dosage and the descriptive analyses will inform about actual doses administered to the study participants. Updates to the status of the other rozanolixizumab studies had been included. The predominance of objective criteria over the investigator’s judgment had been confirmed for the assessment of CIDP relapse. The timeframe of expected use of contraception poststudy completion had been extended to 90 days in view of the probable halflife of rozanolixizumab. Exclusion Criterion had been extended to prediabetic condition. The expectation with regards to the use of cannabidiols and medicinal marijuana had been clarified in the concomitant medication section. The protocol amendment confirmed the expectation of a single rater for the Inflammatory Neuropathy Cause and Treatment (INCAT) assessment to ensure consistency of the rating during the study. The exit interview assessment had been removed from the follow-up study; this allowed UCB to confirm the adequacy of this tool using data collected in CIDP01 before extending its use in another study. Safety reporting procedures had been updated to align with most current UCB practices.
    16 Mar 2020
    The purpose of this substantial protocol amendment was to assess the safety and key efficacy of rozanolixizumab during an optional additional treatment period (Treatment Period Part 2) of up to 52 weeks (or until an Access Program or equivalent was available for rozanolixizumab, whichever came first) on the basis of each study participant’s individual benefit-risk assessment at the end of the first 24-week Treatment Period. This amendment was the opportunity to update the new legal entity of UCB effective since Dec 2019. Additional wording pertaining study participants being able to self-administer IMP after appropriate training had been included. One exclusion criterion had been clarified. The management of hypogammaglobulinemia had been adjusted for better control. An additional interim analysis had been added at the time of lock of the parent study and another at the end of the first Treatment Period (24 weeks) matching the original timepoint of results availability.

    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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