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    Clinical Trial Results:
    A randomized, open-label, two arm, parallel group, proof-of-concept clinical trial to investigate the efficacy and safety of LNP023 compared with rituximab in the treatment of subjects with idiopathic membranous nephropathy

    Summary
    EudraCT number
    2019-001734-34
    Trial protocol
    GB   CZ   NL   ES   DE   FR  
    Global end of trial date
    20 Jan 2023

    Results information
    Results version number
    v1(current)
    This version publication date
    28 Jan 2024
    First version publication date
    28 Jan 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CLNP023D12201
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04154787
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    Novartis Campus, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.com
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.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
    20 Jan 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    20 Jan 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To assess the efficacy of LNP023 compared with rituximab.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    23 Nov 2019
    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
    Argentina: 5
    Country: Number of subjects enrolled
    China: 3
    Country: Number of subjects enrolled
    Czechia: 5
    Country: Number of subjects enrolled
    Germany: 8
    Country: Number of subjects enrolled
    United Kingdom: 5
    Country: Number of subjects enrolled
    India: 7
    Country: Number of subjects enrolled
    Netherlands: 1
    Country: Number of subjects enrolled
    Spain: 2
    Country: Number of subjects enrolled
    Taiwan: 1
    Worldwide total number of subjects
    37
    EEA total number of subjects
    16
    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
    3
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    Participants took part in 18 investigative sites in 9 countries/regions: Argentina (3), Czech Republic (1), Germany (4), India (2), Netherlands (1), Spain (2), United Kingdom (3), China (1) and Taiwan (1)

    Pre-assignment
    Screening details
    Overall, 37 (100%) subjects were randomized.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    LNP023 10/50 mg b.i.d.
    Arm description
    As per protocol V00, participants took LNP023 10 mg orally b.i.d. for 4 weeks followed by LNP023 50 mg orally b.i.d. for 20 weeks
    Arm type
    Experimental

    Investigational medicinal product name
    Iptacopan
    Investigational medicinal product code
    LNP023
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    LNP023 10/50 mg b.i.d.

    Arm title
    LNP023 200 mg b.i.d.
    Arm description
    Combination of the LNP023 25/200 mg b.i.d. and 50/200 mg b.i.d. groups. Under protocol V00, participants took LNP023 25 mg orally b.i.d. for 4 weeks followed by LNP023 200 mg orally b.i.d. for 20 weeks. Under protocol v01, participants took LNP023 50 mg orally b.i.d. for 4 weeks followed by LNP023 200 mg orally b.i.d. for 20 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Iptacopan
    Investigational medicinal product code
    LNP023
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    LNP023 25/200 mg b.i.d. and 50/200 mg b.i.d.

    Arm title
    Rituximab
    Arm description
    Rituximab 1 g i.v. at Day 1 and Day 15
    Arm type
    Active comparator

    Investigational medicinal product name
    Rituximab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Rituximab 1 g i.v. at Day 1 and Day 15

    Number of subjects in period 1
    LNP023 10/50 mg b.i.d. LNP023 200 mg b.i.d. Rituximab
    Started
    3
    19
    15
    Completed
    3
    9
    14
    Not completed
    0
    10
    1
         Patient Requires Other Treatment
    -
    1
    -
         Subject Decision
    -
    1
    -
         Adverse event, non-fatal
    -
    1
    -
         Study Terminated By Sponsor
    -
    6
    1
         Suspected Lack Of Efficacy
    -
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    LNP023 10/50 mg b.i.d.
    Reporting group description
    As per protocol V00, participants took LNP023 10 mg orally b.i.d. for 4 weeks followed by LNP023 50 mg orally b.i.d. for 20 weeks

    Reporting group title
    LNP023 200 mg b.i.d.
    Reporting group description
    Combination of the LNP023 25/200 mg b.i.d. and 50/200 mg b.i.d. groups. Under protocol V00, participants took LNP023 25 mg orally b.i.d. for 4 weeks followed by LNP023 200 mg orally b.i.d. for 20 weeks. Under protocol v01, participants took LNP023 50 mg orally b.i.d. for 4 weeks followed by LNP023 200 mg orally b.i.d. for 20 weeks.

    Reporting group title
    Rituximab
    Reporting group description
    Rituximab 1 g i.v. at Day 1 and Day 15

    Reporting group values
    LNP023 10/50 mg b.i.d. LNP023 200 mg b.i.d. Rituximab Total
    Number of subjects
    3 19 15 37
    Age categorical
    Units: Subjects
        In utero
    0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0 0 0
        Newborns (0-27 days)
    0 0 0 0
        Infants and toddlers (28 days-23 months)
    0 0 0 0
        Children (2-11 years)
    0 0 0 0
        Adolescents (12-17 years)
    0 0 0 0
        Adults (18-64 years)
    2 19 13 34
        From 65-84 years
    1 0 2 3
        85 years and over
    0 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    55.0 ( 18.52 ) 48.9 ( 8.84 ) 46.7 ( 15.33 ) -
    Sex: Female, Male
    Units: participants
        Female
    1 4 1 6
        Male
    2 15 14 31
    Race/Ethnicity, Customized
    Units: Subjects
        Asian
    1 5 4 10
        Black Or African American
    0 0 1 1
        Unknown
    0 0 1 1
        White
    2 14 9 25

    End points

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    End points reporting groups
    Reporting group title
    LNP023 10/50 mg b.i.d.
    Reporting group description
    As per protocol V00, participants took LNP023 10 mg orally b.i.d. for 4 weeks followed by LNP023 50 mg orally b.i.d. for 20 weeks

    Reporting group title
    LNP023 200 mg b.i.d.
    Reporting group description
    Combination of the LNP023 25/200 mg b.i.d. and 50/200 mg b.i.d. groups. Under protocol V00, participants took LNP023 25 mg orally b.i.d. for 4 weeks followed by LNP023 200 mg orally b.i.d. for 20 weeks. Under protocol v01, participants took LNP023 50 mg orally b.i.d. for 4 weeks followed by LNP023 200 mg orally b.i.d. for 20 weeks.

    Reporting group title
    Rituximab
    Reporting group description
    Rituximab 1 g i.v. at Day 1 and Day 15

    Subject analysis set title
    LNP023 10 mg b.i.d.
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Low dose of LNP023 under Protocol V00, LNP023 10mg taken orally b.i.d. during the first 4 weeks of treatment. PK (pharmacokinetic) analysis set included all participants with at least one available valid PK concentration measurement.

    Subject analysis set title
    LNP023 50 mg b.i.d. (20-week administration)
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Under Protocol V00, LNP023 50mg taken orally b.i.d. during the last 20 weeks of treatment. PK (pharmacokinetic) analysis set included all participants with at least one available valid PK concentration measurement.

    Subject analysis set title
    LNP023 25 mg b.i.d.
    Subject analysis set type
    Per protocol
    Subject analysis set description
    High dose of LNP023 under Protocol V00, LNP023 25mg taken orally b.i.d. during the first 4 weeks of treatment. PK (pharmacokinetic) analysis set included all participants with at least one available valid PK concentration measurement.

    Subject analysis set title
    LNP023 50 mg b.i.d. (4-week administration)
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Under Protocol V01, LNP023 50mg taken orally b.i.d. during the first 4 weeks of treatment. PK (pharmacokinetic) analysis set included all participants with at least one available valid PK concentration measurement.

    Subject analysis set title
    LNP023 200 mg b.i.d.
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Under protocol V00, participants took LNP023 25 mg orally b.i.d. for 4 weeks followed by LNP023 200 mg orally b.i.d. for 20 weeks. Under protocol v01, participants took LNP023 50 mg orally b.i.d. for 4 weeks followed by LNP023 200 mg orally b.i.d. for 20 weeks. PK (pharmacokinetic) analysis set included all participants with at least one available valid PK concentration measurement.

    Primary: Ratio between baseline Urine Protein Creatinine Ratio (UPCR) and Urine Protein Creatinine Ratio at 24 weeks of treatment (from 24h urine collection)

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    End point title
    Ratio between baseline Urine Protein Creatinine Ratio (UPCR) and Urine Protein Creatinine Ratio at 24 weeks of treatment (from 24h urine collection) [1]
    End point description
    The primary endpoint of this study is the ratio between UPCR at 24 weeks of treatment measured in 24h urine and baseline UPCR . To assess the primary objective, the log-transformed ratio to baseline in UPCR was analyzed using a mixed model for repeated measures (MMRM). The results were back transformed and presented on the original scale.
    End point type
    Primary
    End point timeframe
    Baseline, Day 113, Day 169
    Notes
    [1] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analysis was planned for this primary outcome.
    End point values
    LNP023 200 mg b.i.d. Rituximab
    Number of subjects analysed
    10
    13
    Units: ratio to baseline
    geometric mean (confidence interval 95%)
        Day 113 (n=10, 13)
    0.94 (0.74 to 1.20)
    0.89 (0.71 to 1.10)
        Day 169 (n=9, 9)
    0.88 (0.65 to 1.19)
    0.64 (0.48 to 0.86)
    Statistical analysis title
    UPCR ratio
    Comparison groups
    Rituximab v LNP023 200 mg b.i.d.
    Number of subjects included in analysis
    23
    Analysis specification
    Pre-specified
    Analysis type
    [2]
    P-value
    = 0.267 [3]
    Method
    Mixed models analysis
    Parameter type
    Adjusted geometric mean ratio
    Point estimate
    1.37
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.9
         upper limit
    2.08
    Notes
    [2] - Comparison of adjusted geometric mean ratios on Day 169
    [3] - Calculated at one-sided 10% level from a lower-tailed test

    Secondary: Change from baseline in plasma levels of circulating fragment of factor B (Bb)

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    End point title
    Change from baseline in plasma levels of circulating fragment of factor B (Bb) [4]
    End point description
    The drug (LNP023) is expected to block the complement alternative pathway dysregulation and thereby should normalize complement biomarker levels in serum. Bb is a biomarker that accurately reflects the level of complement Alternative Pathway activation. Baseline is defined as the last non-missing measurement prior to randomization. Measurements for LNP023 group were done pre-dose.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 15, Day 29, Day 57, Day 113 and Day 169
    Notes
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analysis was planned for this outcome.
    End point values
    LNP023 200 mg b.i.d. Rituximab
    Number of subjects analysed
    17
    13
    Units: ng/mL
    arithmetic mean (standard deviation)
        Day 15 (n=17, 13)
    1520.59 ( 9370.086 )
    -417.69 ( 701.667 )
        Day 29 (n=15, 13)
    -732.00 ( 1412.633 )
    -318.46 ( 604.440 )
        Day 57 (n=14, 13)
    -545.00 ( 1379.201 )
    -75.38 ( 787.545 )
        Day 113 (n=11, 12)
    -877.27 ( 1562.716 )
    -219.17 ( 703.568 )
        Day 169 (n=10, 9)
    -984.00 ( 1794.419 )
    -570.00 ( 611.167 )
    No statistical analyses for this end point

    Secondary: Change from baseline in plasma levels of sC5b-9

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    End point title
    Change from baseline in plasma levels of sC5b-9 [5]
    End point description
    Soluble C5b-9 (sC5b-9) is a biomarker of the complement pathway activity that correlate with disease progression. Baseline is defined as the last non-missing measurement prior to randomization. Measurements for LNP023 group were done pre-dose.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 15, Day 29, Day 57, Day 113, Day 169
    Notes
    [5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analysis was planned for this outcome.
    End point values
    LNP023 200 mg b.i.d. Rituximab
    Number of subjects analysed
    17
    13
    Units: ng/mL
    arithmetic mean (standard deviation)
        Day 15 (n=17, 13)
    -80.40 ( 98.229 )
    -38.49 ( 52.258 )
        Day 29 (n=15, 13)
    -82.80 ( 118.338 )
    -24.51 ( 94.826 )
        Day 57 (n=14, 13)
    -90.62 ( 108.483 )
    -14.02 ( 107.651 )
        Day 113 (n=11, 12)
    -113.32 ( 108.118 )
    -24.95 ( 128.721 )
        Day 169 (n=10, 9)
    -84.07 ( 96.045 )
    -21.76 ( 122.654 )
    No statistical analyses for this end point

    Secondary: Ratio to baseline of urine protein creatinine ratio (UPCR) measured in first morning void

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    End point title
    Ratio to baseline of urine protein creatinine ratio (UPCR) measured in first morning void [6]
    End point description
    Adjusted geometric mean ratio to baseline of Urine Protein Creatinine Ratio (UPCR) measured in first morning void. First morning void urine sample was collected in the morning of the day before the visit and kept in the fridge.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 15, Day 29, Day 57, Day 85, Day 113, Day 141, Day 169, Day 266 and Day 378
    Notes
    [6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analysis was planned for this outcome.
    End point values
    LNP023 200 mg b.i.d. Rituximab
    Number of subjects analysed
    18
    13
    Units: ratio to baseline
    geometric mean (confidence interval 95%)
        Day 15 (n=15, 11)
    0.94 (0.65 to 1.34)
    1.15 (0.75 to 1.76)
        Day 29 (n=18, 12)
    0.98 (0.70 to 1.36)
    0.90 (0.60 to 1.35)
        Day 57 (n=14, 12)
    1.02 (0.71 to 1.48)
    0.77 (0.51 to 1.16)
        Day 85 (n=11, 13)
    1.03 (0.68 to 1.56)
    0.75 (0.50 to 1.13)
        Day 113 (n=10, 11)
    1.05 (0.68 to 1.63)
    0.92 (0.60 to 1.41)
        Day 141 (n=10, 9)
    0.75 (0.48 to 1.19)
    0.77 (0.48 to 1.23)
        Day 169 (n=9, 9)
    0.72 (0.44 to 1.16)
    0.55 (0.34 to 0.89)
        Day 266 (n=6, 7)
    0.57 (0.33 to 0.99)
    0.34 (0.20 to 0.57)
        Day 378 (End Of Study) (n=8, 8)
    0.37 (0.22 to 0.63)
    0.46 (0.27 to 0.76)
    Statistical analysis title
    Ratio at Day 169
    Statistical analysis description
    Day 169
    Comparison groups
    LNP023 200 mg b.i.d. v Rituximab
    Number of subjects included in analysis
    31
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    = 0.4528 [7]
    Method
    Mixed Model for Repeated Measures
    Parameter type
    Geometric mean ratios
    Point estimate
    1.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.65
         upper limit
    2.61
    Notes
    [7] - Calculated from a two-sided test at the 0.05 significance level
    Statistical analysis title
    Ratio at Day 15
    Statistical analysis description
    Day 15
    Comparison groups
    LNP023 200 mg b.i.d. v Rituximab
    Number of subjects included in analysis
    31
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    = 0.4598 [8]
    Method
    Mixed Model for Repeated Measures
    Parameter type
    Adjusted geometric mean ratios
    Point estimate
    0.81
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.47
         upper limit
    1.42
    Notes
    [8] - Calculated from a two-sided test at the 0.05 significance level

    Secondary: Proportion of participants by treatment response at 24 weeks of treatment

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    End point title
    Proportion of participants by treatment response at 24 weeks of treatment [9]
    End point description
    Participants were considered complete responders if at 24 weeks of treatment, they showed complete remission of proteinuria (i.e., Urine Protein (UP) ≤ 0.3 g/24h), partial responders if they showed partial remission (i.e., UP > 0.3g/24h and ≤ 3.5 g/24h and a reduction of UP by >50% from baseline), and non-responders if UP >3.5g/24h and/or reduction of UP from baseline <50%.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 169
    Notes
    [9] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analysis was planned for this outcome.
    End point values
    LNP023 200 mg b.i.d. Rituximab
    Number of subjects analysed
    9
    9
    Units: participants
        Complete
    0
    0
        Partial
    2
    2
        No response
    7
    7
    No statistical analyses for this end point

    Secondary: Change from baseline in (eGFR) estimated Glomerular Filtration Rate over time

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    End point title
    Change from baseline in (eGFR) estimated Glomerular Filtration Rate over time [10]
    End point description
    Changes in renal function were assessed via estimated glomerular filtration rate (eGFR). Change in eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 15, Day 29, Day 57, Day 85, Day 113, Day 141, Day 169
    Notes
    [10] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analysis was planned for this outcome.
    End point values
    LNP023 200 mg b.i.d. Rituximab
    Number of subjects analysed
    19
    13
    Units: mL/min/1.73 m^2
    arithmetic mean (standard deviation)
        Day 15 (n=18, 13)
    2.3 ( 12.07 )
    0.3 ( 8.33 )
        Day 29 (n=19, 13)
    -0.5 ( 8.53 )
    6.1 ( 10.44 )
        Day 57 (n=14, 13)
    2.0 ( 11.10 )
    9.2 ( 21.05 )
        Day 85 (n=12, 13)
    -1.6 ( 11.53 )
    10.2 ( 16.40 )
        Day 113 (n=11, 11)
    1.2 ( 8.64 )
    10.8 ( 15.41 )
        Day 141 (n=10, 9)
    -1.8 ( 9.10 )
    7.1 ( 9.29 )
        Day 169 (n=10, 9)
    -1.3 ( 7.36 )
    3.1 ( 13.21 )
    No statistical analyses for this end point

    Secondary: Pharmacokinetic parameter Cmax in plasma

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    End point title
    Pharmacokinetic parameter Cmax in plasma [11]
    End point description
    Pharmacokinetics of LNP023: Cmax is the maximum (peak) observed plasma drug concentration after dose administration (mass x volume-1)
    End point type
    Secondary
    End point timeframe
    Day 29 and Day 113 (pre-dose and 0.25 hours, 0.5 hours, 1 hour 2 hours, 4 hours and 6 hours post dose)
    Notes
    [11] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analysis was planned for this outcome.
    End point values
    LNP023 200 mg b.i.d. LNP023 10 mg b.i.d. LNP023 50 mg b.i.d. (20-week administration) LNP023 25 mg b.i.d. LNP023 50 mg b.i.d. (4-week administration)
    Number of subjects analysed
    7
    2
    2
    3
    10
    Units: ng/mL
    arithmetic mean (standard deviation)
        Day 29 (n= 0, 2, 3, 10, 0 )
    999 ( 999 )
    1200 ( 799 )
    999 ( 999 )
    2070 ( 938 )
    2140 ( 917 )
        Day 113 (n= 7, 0, 0, 0, 2 )
    4810 ( 2850 )
    999 ( 999 )
    1220 ( 304 )
    999 ( 999 )
    999 ( 999 )
    No statistical analyses for this end point

    Secondary: Pharmacokinetic parameter Tmax in plasma

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    End point title
    Pharmacokinetic parameter Tmax in plasma [12]
    End point description
    Pharmacokinetics of LNP023 : Tmax is the time to reach maximum (peak) plasma drug concentration after dose administration (time). Actual sampling time points were considered for the calculation of PK parameters.
    End point type
    Secondary
    End point timeframe
    Day 29 and Day 113 (pre-dose and 0.25 hours, 0.5 hours, 1 hour 2 hours, 4 hours and 6 hours post dose)
    Notes
    [12] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analysis was planned for this outcome.
    End point values
    LNP023 200 mg b.i.d. LNP023 10 mg b.i.d. LNP023 50 mg b.i.d. (20-week administration) LNP023 25 mg b.i.d. LNP023 50 mg b.i.d. (4-week administration)
    Number of subjects analysed
    7
    2
    2
    3
    10
    Units: hours
    median (full range (min-max))
        Day 29 (n= 0, 2, 3, 10, 0 )
    999 (999 to 999)
    2.04 (2.00 to 2.08)
    999 (999 to 999)
    2.00 (1.00 to 4.00)
    2.03 (1.00 to 4.38)
        Day 113 (n= 7, 0, 0, 0, 2 )
    1.00 (0.250 to 6.00)
    999 (999 to 999)
    2.05 (2.00 to 2.10)
    999 (999 to 999)
    999 (999 to 999)
    No statistical analyses for this end point

    Secondary: Pharmacokinetic parameter AUClast in plasma

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    End point title
    Pharmacokinetic parameter AUClast in plasma [13]
    End point description
    Pharmacokinetics of LNP023: AUClast is the AUC from time zero to the last measurable concentration sampling time (tlast) (mass x time x volume-1)
    End point type
    Secondary
    End point timeframe
    Day 29 and Day 113 (pre-dose and 0.25 hours, 0.5 hours, 1 hour 2 hours, 4 hours and 6 hours post dose)
    Notes
    [13] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analysis was planned for this outcome.
    End point values
    LNP023 200 mg b.i.d. LNP023 10 mg b.i.d. LNP023 50 mg b.i.d. (20-week administration) LNP023 25 mg b.i.d. LNP023 50 mg b.i.d. (4-week administration)
    Number of subjects analysed
    7
    2
    2
    3
    10
    Units: hr*ng/mL
    arithmetic mean (standard deviation)
        Day 29 (n= 0, 2, 3, 10, 0 )
    999 ( 999 )
    5970 ( 4150 )
    999 ( 999 )
    8140 ( 3080 )
    9920 ( 3960 )
        Day 113 (n= 7, 0, 0, 0, 2 )
    22200 ( 15600 )
    999 ( 999 )
    5940 ( 989 )
    999 ( 999 )
    999 ( 999 )
    No statistical analyses for this end point

    Secondary: Pharmacokinetic parameter AUCtau in plasma

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    End point title
    Pharmacokinetic parameter AUCtau in plasma [14]
    End point description
    Pharmacokinetics of LNP023 : AUCtau is the AUC calculated to the end of a dosing interval (tau) at steady-state (amount x time x volume-1)
    End point type
    Secondary
    End point timeframe
    Day 29 and Day 113 (pre-dose and 0.25 hours, 0.5 hours, 1 hour 2 hours, 4 hours and 6 hours post dose)
    Notes
    [14] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analysis was planned for this outcome.
    End point values
    LNP023 200 mg b.i.d. LNP023 10 mg b.i.d. LNP023 50 mg b.i.d. (20-week administration) LNP023 25 mg b.i.d. LNP023 50 mg b.i.d. (4-week administration)
    Number of subjects analysed
    7
    2
    2
    3
    10
    Units: hr*ng/mL
    arithmetic mean (standard deviation)
        Day 29 (n= 0, 2, 3, 10, 0 )
    999 ( 999 )
    9850 ( 5790 )
    999 ( 999 )
    12800 ( 3490 )
    16500 ( 5920 )
        Day 113 (n= 7, 0, 0, 0, 2 )
    36800 ( 26100 )
    999 ( 999 )
    10700 ( 1640 )
    999 ( 999 )
    999 ( 999 )
    No statistical analyses for this end point

    Secondary: Pharmacokinetics in urine: renal plasma clearance derived from 24 hour urine sample

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    End point title
    Pharmacokinetics in urine: renal plasma clearance derived from 24 hour urine sample [15]
    End point description
    Pharmacokinetics of LNP023 in urine: Renal plasma clearance derived from 24 hour urine sample
    End point type
    Secondary
    End point timeframe
    Day 113
    Notes
    [15] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No statistical analysis was planned for this outcome.
    End point values
    LNP023 200 mg b.i.d.
    Number of subjects analysed
    7
    Units: L/hr
        arithmetic mean (standard deviation)
    1.19 ( 0.668 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events were reported from first dose of study treatment until end of study treatment plus 29 weeks post treatment, up to maximum duration of 53 weeks.
    Adverse event reporting additional description
    Any sign or symptom that occurs during the conduct of the trial and the safety follow-up.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    25.1
    Reporting groups
    Reporting group title
    LNP023 10/50 mg b.i.d.
    Reporting group description
    As per protocol V00, participants took LNP023 10 mg orally b.i.d. for 4 weeks followed by LNP023 50 mg orally b.i.d. for 20 weeks

    Reporting group title
    LNP023 200 mg b.i.d. (combination of 25/200 mg, 50/200 mg)
    Reporting group description
    Combination of the LNP023 25/200 mg b.i.d. and 50/200 mg b.i.d. groups. Under protocol V00, participants took LNP023 25 mg orally b.i.d. for 4 weeks followed by LNP023 200 mg orally b.i.d. for 20 weeks. Under protocol v01, participants took LNP023 50 mg orally b.i.d. for 4 weeks followed by LNP023 200 mg orally b.i.d. for 20 weeks.

    Reporting group title
    Pooled LNP023
    Reporting group description
    Combination of the LNP023 10/50 mg b.i.d., 25/200 mg b.i.d. and 50/200 mg b.i.d. groups.

    Reporting group title
    Rituximab 1 g i.v.
    Reporting group description
    Rituximab 1 g i.v. at Day 1 and Day 15

    Reporting group title
    Total
    Reporting group description
    Total

    Serious adverse events
    LNP023 10/50 mg b.i.d. LNP023 200 mg b.i.d. (combination of 25/200 mg, 50/200 mg) Pooled LNP023 Rituximab 1 g i.v. Total
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 3 (0.00%)
    3 / 19 (15.79%)
    3 / 22 (13.64%)
    3 / 15 (20.00%)
    6 / 37 (16.22%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary embolism
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    1 / 15 (6.67%)
    2 / 37 (5.41%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Erythema
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19 pneumonia
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Lower respiratory tract infection
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
    0 / 1
         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
    LNP023 10/50 mg b.i.d. LNP023 200 mg b.i.d. (combination of 25/200 mg, 50/200 mg) Pooled LNP023 Rituximab 1 g i.v. Total
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 3 (66.67%)
    13 / 19 (68.42%)
    15 / 22 (68.18%)
    7 / 15 (46.67%)
    22 / 37 (59.46%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    1
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 19 (10.53%)
    2 / 22 (9.09%)
    0 / 15 (0.00%)
    2 / 37 (5.41%)
         occurrences all number
    0
    4
    4
    0
    4
    Influenza like illness
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Pyrexia
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Oedema peripheral
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 19 (10.53%)
    2 / 22 (9.09%)
    0 / 15 (0.00%)
    2 / 37 (5.41%)
         occurrences all number
    0
    2
    2
    0
    2
    Oedema
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Reproductive system and breast disorders
    Epididymal cyst
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 19 (0.00%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    1
    0
    1
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Dysphonia
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Dyspnoea
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    2
    2
    Epistaxis
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    1
    1
    Psychiatric disorders
    Sleep disorder
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Investigations
    Liver function test increased
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    1
    1
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    1
    1
    Injury, poisoning and procedural complications
    Limb injury
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    1
    1
    Infusion related reaction
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    1
    1
    Head injury
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 19 (0.00%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    1
    0
    1
    0
    1
    Fall
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    1
    1
    Post-traumatic pain
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    1
    1
    Nervous system disorders
    Syncope
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Hypersomnia
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Dizziness
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 19 (10.53%)
    2 / 22 (9.09%)
    0 / 15 (0.00%)
    2 / 37 (5.41%)
         occurrences all number
    0
    2
    2
    0
    2
    Headache
         subjects affected / exposed
    0 / 3 (0.00%)
    3 / 19 (15.79%)
    3 / 22 (13.64%)
    0 / 15 (0.00%)
    3 / 37 (8.11%)
         occurrences all number
    0
    3
    3
    0
    3
    Blood and lymphatic system disorders
    Lymphopenia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    1
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Dyspepsia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 19 (0.00%)
    1 / 22 (4.55%)
    1 / 15 (6.67%)
    2 / 37 (5.41%)
         occurrences all number
    1
    0
    1
    1
    2
    Abdominal pain lower
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Inguinal hernia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    1
    1
    Epigastric discomfort
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    1
    1
    Rash maculo-papular
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Renal and urinary disorders
    Nephrotic syndrome
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    1
    1
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Back pain
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 19 (10.53%)
    2 / 22 (9.09%)
    0 / 15 (0.00%)
    2 / 37 (5.41%)
         occurrences all number
    0
    3
    3
    0
    3
    Bone hypertrophy
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Muscle spasms
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 19 (5.26%)
    2 / 22 (9.09%)
    1 / 15 (6.67%)
    3 / 37 (8.11%)
         occurrences all number
    1
    1
    2
    2
    4
    Infections and infestations
    COVID-19
         subjects affected / exposed
    0 / 3 (0.00%)
    3 / 19 (15.79%)
    3 / 22 (13.64%)
    1 / 15 (6.67%)
    4 / 37 (10.81%)
         occurrences all number
    0
    3
    3
    1
    4
    Lower respiratory tract infection
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Influenza
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Ear infection
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Coronavirus infection
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    1 / 15 (6.67%)
    2 / 37 (5.41%)
         occurrences all number
    0
    1
    1
    1
    2
    Nasopharyngitis
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    1 / 15 (6.67%)
    2 / 37 (5.41%)
         occurrences all number
    0
    1
    1
    1
    2
    Respiratory tract infection viral
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Pneumonia
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Metabolism and nutrition disorders
    Hyperuricaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1
    Hypertriglyceridaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    1 / 15 (6.67%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    0
    1
    1
    Gout
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 19 (0.00%)
    0 / 22 (0.00%)
    2 / 15 (13.33%)
    2 / 37 (5.41%)
         occurrences all number
    0
    0
    0
    2
    2
    Hypokalaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    2 / 19 (10.53%)
    2 / 22 (9.09%)
    1 / 15 (6.67%)
    3 / 37 (8.11%)
         occurrences all number
    0
    2
    2
    1
    3
    Vitamin B12 deficiency
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 19 (5.26%)
    1 / 22 (4.55%)
    0 / 15 (0.00%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    1
    0
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    04 Mar 2021
    The main purpose of the global amendment was to align with study results obtained from ongoing trials with LNP023 in other indications which support best efficacy results with LNP023 at dose levels of 200mg b.i.d. LNP023 low dose arm (10/50mg) was removed from this study and the first portion of the high dose arm was modified from 25 to 50mg b.i.d for 4 weeks.

    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.
    Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd.com/CtrdWeb/home.nov for complete trial results.
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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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