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    Clinical Trial Results:
    A Multicenter, Randomized, Subject-Blind, Investigator-Blind, Placebo-Controlled, Parallel-Group Study Evaluating the Efficacy, Safety, and Tolerability of Rozanolixizumab in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

    Summary
    EudraCT number
    2016-002411-17
    Trial protocol
    BE   DK   NL   DE   ES   FR   GB  
    Global end of trial date
    31 Mar 2021

    Results information
    Results version number
    v2(current)
    This version publication date
    13 May 2022
    First version publication date
    25 Mar 2022
    Other versions
    v1
    Version creation reason

    Trial information

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    Trial identification
    Sponsor protocol code
    CIDP01
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03861481
    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
    28 May 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    31 Mar 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    31 Mar 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the clinical efficacy of rozanolixizumab as a treatment for participants with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).
    Protection of trial subjects
    During the conduct of the study all participants were closely monitored. Immediate rescue therapy upon CIDP relapse
    Background therapy
    Background therapy as permitted in the protocol.
    Evidence for comparator
    -
    Actual start date of recruitment
    26 Mar 2019
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Ethical reason
    Long term follow-up duration
    18 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Belgium: 5
    Country: Number of subjects enrolled
    Denmark: 3
    Country: Number of subjects enrolled
    France: 3
    Country: Number of subjects enrolled
    Germany: 3
    Country: Number of subjects enrolled
    Netherlands: 2
    Country: Number of subjects enrolled
    Spain: 4
    Country: Number of subjects enrolled
    United Kingdom: 1
    Country: Number of subjects enrolled
    United States: 13
    Worldwide total number of subjects
    34
    EEA total number of subjects
    20
    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)
    28
    From 65 to 84 years
    6
    85 years and over
    0

    Subject disposition

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

    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 placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.
    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 matched to rozanolixizumab at prespecified time points.

    Arm title
    Rozanolixizumab
    Arm description
    Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.
    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
    Placebo Rozanolixizumab
    Started
    17
    17
    Completed
    11
    10
    Not completed
    6
    7
         COVID-19 pandemic circumstances
    -
    1
         Relapse
    1
    -
         Lack of efficacy
    4
    5
         Consent withdrawn by subject
    -
    1
         Participant unable to participate in study CIDP04
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.

    Reporting group title
    Rozanolixizumab
    Reporting group description
    Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.

    Reporting group values
    Placebo Rozanolixizumab Total
    Number of subjects
    17 17 34
    Age Categorical
    Units: participants
        <=18 years
    0 0 0
        Between 18 and 65 years
    16 12 28
        >=65 years
    1 5 6
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    56.4 ± 7.4 57.3 ± 13.3 -
    Sex: Female, Male
    Units: participants
        Female
    8 8 16
        Male
    9 9 18

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.

    Reporting group title
    Rozanolixizumab
    Reporting group description
    Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.

    Primary: Change from Baseline to Week 13 (Day 85) in inflammatory Rasch-built Overall Disability Scale (iRODS) score

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    End point title
    Change from Baseline to Week 13 (Day 85) in inflammatory Rasch-built Overall Disability Scale (iRODS) score
    End point description
    iRODS: linearly weighted patient-reported endpoint (questionnaire) that captures activity and social participation limitations in participants with CIDP. It consisted of 24 items and assesses participant’s ability to perform daily and social activities. Participants had 3 response options: 0=impossible to perform; 1=performed with difficulty; 2=easily performed, performed without difficulty. Raw sum scores of iRODS (range 0 to 48, where 0=worse and 48=best) were translated to log odds units (logits) scale, placing estimates on same logit scale which had a score range of -6.95 (most severe activity; social participation restrictions) to 8.11 (no activity; social participation limitations). A positive change is associated with better outcome of less disease activity and more social activity.Full Analysis Set: all participants who received at least 1 dose of treatment and had Baseline and post-Baseline iRODS measurement up to Week 13 (Day 85)/premature end of treatment (inclusively).
    End point type
    Primary
    End point timeframe
    From Baseline up to Week 13 (Day 85)
    End point values
    Placebo Rozanolixizumab
    Number of subjects analysed
    17
    16
    Units: score on a scale (logits)
        least squares mean (standard error)
    0.234 ± 0.379
    0.181 ± 0.468
    Statistical analysis title
    Statistical Analysis 1
    Comparison groups
    Rozanolixizumab v Placebo
    Number of subjects included in analysis
    33
    Analysis specification
    Pre-specified
    Analysis type
    [1]
    Method
    Parameter type
    LS Mean difference (Rozimab - Placebo)
    Point estimate
    -0.052
    Confidence interval
         level
    90%
         sides
    2-sided
         lower limit
    -0.892
         upper limit
    0.788
    Notes
    [1] - MMRM analysis (fixed effect terms: treatment, baseline iRODS score, prior Ig therapy administration route, assessment week, treatment by week interaction; random effect term: study participant). LS Mean Difference > 0 favours rozanolixizumab.

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Baseline until the Safety Follow-up Visit (up to Week 24)
    Adverse event reporting additional description
    A TEAE is defined as any event that was not present prior the first administration of investigational medicinal product (IMP) or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
    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 rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.

    Reporting group title
    Placebo
    Reporting group description
    Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.

    Serious adverse events
    Rozanolixizumab Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 17 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Nervous system disorders
    Chronic inflammatory demyelinating polyradiculoneuropathy
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 17 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    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
    15 / 17 (88.24%)
    14 / 17 (82.35%)
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    2
    Investigations
    Bacterial test positive
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 17 (0.00%)
         occurrences all number
    2
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    6 / 17 (35.29%)
    5 / 17 (29.41%)
         occurrences all number
    9
    10
    Chronic inflammatory demyelinating polyradiculoneuropathy
         subjects affected / exposed
    4 / 17 (23.53%)
    4 / 17 (23.53%)
         occurrences all number
    4
    4
    Hypoaesthesia
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    2
    General disorders and administration site conditions
    Infusion site erythema
         subjects affected / exposed
    3 / 17 (17.65%)
    0 / 17 (0.00%)
         occurrences all number
    4
    0
    Peripheral swelling
         subjects affected / exposed
    3 / 17 (17.65%)
    0 / 17 (0.00%)
         occurrences all number
    3
    0
    Fatigue
         subjects affected / exposed
    0 / 17 (0.00%)
    3 / 17 (17.65%)
         occurrences all number
    0
    3
    Pain
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    2
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    2 / 17 (11.76%)
    3 / 17 (17.65%)
         occurrences all number
    2
    5
    Nausea
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    6
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    3
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    3 / 17 (17.65%)
    0 / 17 (0.00%)
         occurrences all number
    3
    0
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    0 / 17 (0.00%)
    3 / 17 (17.65%)
         occurrences all number
    0
    4

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    15 Nov 2018
    After the protocol was completed at UCB, but before it was submitted to any regulatory authorities or IRBs/IECs, it was updated to better align it with the current understanding of available data from completed and ongoing studies with rozanolixizumab. The updated protocol was then submitted to regulatory authorities and the study began.
    09 Jul 2019
    The purpose of Amendment 2 was to provide clarification on the dose of IMP administered (including an allowance of ±10% compared with the target dose A), as well as a flexible infusion rate. Sensitivity analyses were introduced to account for deviations outside the ±10% target dosage; the descriptive analyses were to inform about actual doses administered to the study participants. The visit windows and the scheduling of the Randomization Visit in view of the Ig treatment at study start were clarified. The predominance of objective criteria over the investigator’s judgement was confirmed for the assessment of CIDP relapse. The timeframe of expected use of contraception poststudy completion was extended to 90 days in view of the probable half-life of rozanolixizumab. Exclusion Criterion No. 3 was extended to a prediabetic condition. The expectation with regards to the use of cannabidiols and medicinal marijuana was clarified in the concomitant medication section. This amendment confirmed the expectation of a single rater for the INCAT assessment to ensure consistency of the rating during the course of the study.

    Interruptions (globally)

    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    20 Mar 2020
    Recruitment was stopped from 20 Mar 20 due to Covid pandemic (participants in screening at the moment could not be randomized – participants under treatment continued in the study as judged appropriate by the investigators/participants).
    03 Jun 2020

    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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