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    Clinical Trial Results:
    Randomized, cohort study of standardized reduction of subcutaneous immunoglobulin treatment in patients with chronic inflammatory demyelinating polyneuropathy

    Summary
    EudraCT number
    		 2017-002024-24
    Trial protocol
    		 DK  
    Global end of trial date
    11 Jun 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Dec 2021
    First version publication date
    04 Dec 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    AUH-2016-100
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Aarhus University Hospital
    Sponsor organisation address
    Palle Juul-Jensens Boulevard 165, Aarhus N, Denmark, 8200
    Public contact
    Lars Markvardsen, Aarhus University Hospital, 0045 78450000, larsmark@rm.dk
    Scientific contact
    Lars Markvardsen, Aarhus University Hospital, 0045 78450000, larsmark@rm.dk
    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 Oct 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    11 Jun 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To identify the lowest effective dosage of subcutaneous immunoglobulin in maintenance treatment of CIDP
    Protection of trial subjects
    All participants had possibility to contact study nurses and physicians during the study period in case of adverse events.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Oct 2017
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Efficacy
    Long term follow-up duration
    		 2 Years
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Denmark: 55
    Worldwide total number of subjects
    55
    EEA total number of subjects
    55
    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)
    34
    From 65 to 84 years
    21
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants were recruited from outpatient clinics at the neurological departments in Denmark, who are responsible for treatment and follow-up of patients in subcutaneous immunoglobulin for chronic inflammatory demyelinating polyneuropathy (CIDP)

    Pre-assignment
    Screening details
    		 Inclusion criteria: Fulfilling CIDP criteria for propable, possible or definite CIDP (including subtypes) Stable treament with SCIG (no change in dosage >3 months prior to inclusion) Screened: n=81 Excluded: n=26; unstable SCIG (n=10), concomittant treatment (n=3), discontinue SCIG (n=2), decline to participate (n=11 )

    Period 1
    Period 1 title
    Intervention (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 6 week evaluation
    Arm description
    		 Standardized, tapering off regimen with clinical evaluation every 12th week
    Arm type
    		 Experimental

    Investigational medicinal product name
    Human immunoglobulin for subcutaneous administration
    Investigational medicinal product code
    Other name
    Gammanorm, Hizentra
    Pharmaceutical forms
    Solution for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Treatment with SCIG (in percent of individual, pre-study dosage): 90%, 75%, 50%, 25%, 0% (12 weeks treatment for each step)

    Arm title
    		 12 week evaluation
    Arm description
    		 Standardized, tapering off regimen with clinical evaluation every 12th week
    Arm type
    		 Experimental

    Investigational medicinal product name
    Human immunoglobulin for subcutaneous administration
    Investigational medicinal product code
    Other name
    Gammanorm, Hizentra
    Pharmaceutical forms
    Solution for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Treatment with SCIG (in percent of individual, pre-study dosage): 90%, 75%, 50%, 25%, 0% (12 weeks treatment for each step)

    Number of subjects in period 1
    		6 week evaluation 12 week evaluation
    Started
    27
    28
    Completed
    11
    9
    Not completed
    16
    19
         Lack of efficacy
    16
    19

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    6 week evaluation
    Reporting group description
    		 Standardized, tapering off regimen with clinical evaluation every 12th week

    Reporting group title
    12 week evaluation
    Reporting group description
    		 Standardized, tapering off regimen with clinical evaluation every 12th week

    Reporting group values
    		 6 week evaluation 12 week evaluation Total
    Number of subjects
    		 27 28 55
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 19 14 33
        From 65-84 years
    		 8 14 22
        85 years and over
    		 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 9 7 16
        Male
    		 18 21 39
    Subject analysis sets

    Subject analysis set title
    Relapse versus remission
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Analysis of the number of participants dropping out (remission) or excluded (relapse) during stepwise reduction of SCIG dosage indicating active disease

    Subject analysis set title
    Frequent versus rare evaluation
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Evalaution of frequent versus rare clinical evaluation to detect clinical meaningful deterioration

    Subject analysis sets values
    		 Relapse versus remission Frequent versus rare evaluation
    Number of subjects
    55
    35
    Age categorical
    Units: Subjects
        In utero
    0
    0
        Preterm newborn infants (gestational age < 37 wks)
    0
    0
        Newborns (0-27 days)
    0
    0
        Infants and toddlers (28 days-23 months)
    0
    0
        Children (2-11 years)
    0
    0
        Adolescents (12-17 years)
    0
    0
        Adults (18-64 years)
    33
    17
        From 65-84 years
    22
    18
        85 years and over
    0
    0
    Age continuous
    Units:
        
    ( )
    ( )
    Gender categorical
    Units: Subjects
        Female
        Male

    End points

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    End points reporting groups
    Reporting group title
    6 week evaluation
    Reporting group description
    		 Standardized, tapering off regimen with clinical evaluation every 12th week

    Reporting group title
    12 week evaluation
    Reporting group description
    		 Standardized, tapering off regimen with clinical evaluation every 12th week

    Subject analysis set title
    Relapse versus remission
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Analysis of the number of participants dropping out (remission) or excluded (relapse) during stepwise reduction of SCIG dosage indicating active disease

    Subject analysis set title
    Frequent versus rare evaluation
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Evalaution of frequent versus rare clinical evaluation to detect clinical meaningful deterioration

    Primary: Number of patients in remission

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    End point title
    		 Number of patients in remission
    End point description
    All patients followed the same dose reduction regimen with the following dosages (pct of SCIG dose at inclusion) Week 0-12: 90% Week 12-24: 75% Week 24-36: 50% Week 36-48: 25% Week 48-60: 0%
    End point type
    Primary
    End point timeframe
    From inclusion (week 0) to end-of-study (week 60)
    End point values
    		 6 week evaluation 12 week evaluation Relapse versus remission
    Number of subjects analysed
    27
    28
    55
    Units: number
    11
    9
    20
    Statistical analysis title
    		 Fishers exact test
    Comparison groups
    6 week evaluation v 12 week evaluation
    Number of subjects included in analysis
    55
    Analysis specification
    Pre-specified
    Analysis type
    		 equivalence
    P-value
    		 = 0.58
    Method
    		 Fisher exact
    Parameter type
    		 Risk ratio (RR)
    Point estimate
    0.83
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.49
         upper limit
    		 1.47

    Primary: Total reduction of SCIG dosage

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    End point title
    		 Total reduction of SCIG dosage
    End point description
    All patients followed the same dose reduction regimen with the following dosages (pct of SCIG dose at inclusion) Week 0-12: 90% Week 12-24: 75% Week 24-36: 50% Week 36-48: 25% Week 48-60: 0%
    End point type
    Primary
    End point timeframe
    From inclusion (week 0) to end-of-study (week 60)
    End point values
    		 6 week evaluation 12 week evaluation
    Number of subjects analysed
    27
    28
    Units: percent
        median (full range (min-max))
    25 (0 to 100)
    17.5 (0 to 100)
    Statistical analysis title
    		 Overall dosage reduction
    Comparison groups
    6 week evaluation v 12 week evaluation
    Number of subjects included in analysis
    55
    Analysis specification
    Pre-specified
    Analysis type
    		 equivalence
    P-value
    		 = 0.77
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Secondary: Self-registration versus registration at clinic

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    End point title
    		 Self-registration versus registration at clinic
    End point description
    Number of patients with relapse registered due to a planned clinical evaluation
    End point type
    Secondary
    End point timeframe
    From inclusion (week 0) up to end-of-study (week 60)
    End point values
    		 6 week evaluation 12 week evaluation Frequent versus rare evaluation
    Number of subjects analysed
    27
    28
    35
    Units: numbers
    13
    11
    24
    Statistical analysis title
    		 Fisher´s test of relative risk
    Statistical analysis description
    Ability to detect clinical deterioration at clinical visit versus self-registration
    Comparison groups
    6 week evaluation v 12 week evaluation
    Number of subjects included in analysis
    55
    Analysis specification
    Pre-specified
    Analysis type
    		 equivalence
    P-value
    		 = 0.17
    Method
    		 Fisher exact
    Parameter type
    		 Risk ratio (RR)
    Point estimate
    0.5
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.2
         upper limit
    		 1.2

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Inclusion (week 0) to end-of-study (week 60)
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 Self-reporting
    Dictionary version
    1
    Reporting groups
    Reporting group title
    Entire cohort
    Reporting group description
    		 All participants went through the same intervention, only follow regimen was different.

    Serious adverse events
    		 Entire cohort
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 55 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Entire cohort
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    28 / 55 (50.91%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Breast inflammation
         subjects affected / exposed
    1 / 55 (1.82%)
         occurrences all number
    1
    Nervous system disorders
    Numbness, Muscle fatigue
    Additional description: General signs from the nervous system according to the diagnosis CIDP (including numbness, palsies, walking difficulties)
         subjects affected / exposed
    6 / 55 (10.91%)
         occurrences all number
    6
    General disorders and administration site conditions
    Dizziness
         subjects affected / exposed
    3 / 55 (5.45%)
         occurrences all number
    4
    Headache
         subjects affected / exposed
    1 / 55 (1.82%)
         occurrences all number
    1
    Gastrointestinal disorders
    Nausea, Diarrhoea, Abdominal discomfort
         subjects affected / exposed
    8 / 55 (14.55%)
         occurrences all number
    8
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    4 / 55 (7.27%)
         occurrences all number
    5
    Renal and urinary disorders
    Urinary tract infection
         subjects affected / exposed
    3 / 55 (5.45%)
         occurrences all number
    3
    Musculoskeletal and connective tissue disorders
    Arthropathy
         subjects affected / exposed
    1 / 55 (1.82%)
         occurrences all number
    1
    Infections and infestations
    Respiratory tract infection
         subjects affected / exposed
    1 / 55 (1.82%)
         occurrences all number
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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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