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    Clinical Trial Results:
    A randomized, subject and investigator blinded, placebo-controlled and multi-center platform study, to assess efficacy and safety of different investigational drugs in patients with moderate to severe hidradenitis suppurativa

    Summary
    EudraCT number
    		 2018-002757-30
    Trial protocol
    		 FR   HU   DK   NL   IS   CZ   BE   ES  
    Global end of trial date

    Results information
    Results version number
    		 v1(current)
    This version publication date
    11 Dec 2025
    First version publication date
    11 Dec 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CCFZ533H12201BC
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03827798
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    Lichtstrasse 35, Basel, Switzerland, 4056
    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
    Interim
    Date of interim/final analysis
    04 Dec 2024
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    04 Dec 2024
    Global end of trial reached?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to assess the efficacy of the investigational treatments compared to the pooled placebo group from Cohorts A, B, C, and D, in moderate to severe inflammatory hidradenitis suppurativa (HS) patients by comparing the proportion of participants achieving clinical response defined by the simplified Hidradenitis Suppurativa Clinical Response (sHiSCR) after 16 weeks of treatment. This study has a cohort E which is ongoing and will be reported in CTIS.
    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
    27 Feb 2019
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Austria: 3
    Country: Number of subjects enrolled
    Belgium: 13
    Country: Number of subjects enrolled
    Czechia: 12
    Country: Number of subjects enrolled
    Denmark: 5
    Country: Number of subjects enrolled
    France: 50
    Country: Number of subjects enrolled
    Germany: 28
    Country: Number of subjects enrolled
    Hungary: 19
    Country: Number of subjects enrolled
    Iceland: 1
    Country: Number of subjects enrolled
    Netherlands: 14
    Country: Number of subjects enrolled
    Spain: 29
    Country: Number of subjects enrolled
    United States: 31
    Worldwide total number of subjects
    205
    EEA total number of subjects
    174
    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)
    205
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants took part in 36 investigative sites in 11 countries.

    Pre-assignment
    Screening details
    		 The study consisted of a screening period of up to 35 days.

    Period 1
    Period 1 title
    Treatment Epoch (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Investigator, Carer, Assessor, Subject

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Cohort A - CFZ533 600 mg
    Arm description
    		 CFZ533 600 mg administered subcutaneous (s.c) weekly for 4 weeks, followed by bi-weekly until Week 15.
    Arm type
    		 Experimental

    Investigational medicinal product name
    CFZ533
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    CFZ533 600 mg administered subcutaneous (s.c) weekly for 4 weeks, followed by bi-weekly until Week 15.

    Arm title
    		 Cohort A - Placebo to CFZ533
    Arm description
    		 Placebo administered subcutaneous (s.c) weekly for 4 weeks, followed by bi-weekly until Week 15.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Placebo administered subcutaneous (s.c) weekly for 4 weeks, followed by bi-weekly until Week 15.

    Arm title
    		 Cohort B - LYS006 20 mg
    Arm description
    		 LYS006 20 mg administered orally twice per day until Week 16.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LYS006
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    LYS006 20 mg administered orally twice per day until Week 16.

    Arm title
    		 Cohort B - Placebo to LYS006
    Arm description
    		 Placebo administered orally twice per day until Week 16.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo administered orally twice per day until Week 16.

    Arm title
    		 Cohort C - MAS825 300 mg
    Arm description
    		 MAS825 300 mg administered s.c. bi-weekly for 4 weeks, followed by monthly until Week 13.
    Arm type
    		 Experimental

    Investigational medicinal product name
    MAS825
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    MAS825 300 mg administered s.c. bi-weekly for 4 weeks, followed by monthly until Week 13.

    Arm title
    		 Cohort C - Placebo to MAS825
    Arm description
    		 Placebo administered s.c. bi-weekly for 4 weeks, followed by monthly until Week 13.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Placebo administered s.c. bi-weekly for 4 weeks, followed by monthly until Week 13.

    Arm title
    		 Cohort D - LOU064 25 mg
    Arm description
    		 LOU064 25 mg administered orally twice per day until Week 16.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LOU064
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    LOU064 25 mg administered orally twice per day until Week 16.

    Arm title
    		 Cohort D - LOU064 100 mg
    Arm description
    		 LOU064 100 mg administered orally twice per day until Week 16.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LOU064
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    LOU064 100 mg administered orally twice per day until Week 16.

    Arm title
    		 Cohort D - Placebo to LOU064
    Arm description
    		 Placebo administered orally twice per day until Week 16.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo administered orally twice per day until Week 16.

    Number of subjects in period 1
    		Cohort A - CFZ533 600 mg Cohort A - Placebo to CFZ533 Cohort B - LYS006 20 mg Cohort B - Placebo to LYS006 Cohort C - MAS825 300 mg Cohort C - Placebo to MAS825 Cohort D - LOU064 25 mg Cohort D - LOU064 100 mg Cohort D - Placebo to LOU064
    Started
    29
    16
    27
    13
    33
    10
    33
    33
    11
    Completed
    25
    12
    20
    9
    29
    9
    29
    26
    8
    Not completed
    4
    4
    7
    4
    4
    1
    4
    7
    3
         Physician decision
    1
    -
    1
    2
    1
    -
    -
    -
    -
         Consent withdrawn by subject
    2
    3
    4
    1
    2
    1
    4
    3
    2
         Protocol Deviation
    -
    -
    1
    -
    -
    -
    -
    -
    -
         Adverse event
    1
    1
    1
    1
    1
    -
    -
    3
    -
         Lost to follow-up
    -
    -
    -
    -
    -
    -
    -
    1
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort A - CFZ533 600 mg
    Reporting group description
    		 CFZ533 600 mg administered subcutaneous (s.c) weekly for 4 weeks, followed by bi-weekly until Week 15.

    Reporting group title
    Cohort A - Placebo to CFZ533
    Reporting group description
    		 Placebo administered subcutaneous (s.c) weekly for 4 weeks, followed by bi-weekly until Week 15.

    Reporting group title
    Cohort B - LYS006 20 mg
    Reporting group description
    		 LYS006 20 mg administered orally twice per day until Week 16.

    Reporting group title
    Cohort B - Placebo to LYS006
    Reporting group description
    		 Placebo administered orally twice per day until Week 16.

    Reporting group title
    Cohort C - MAS825 300 mg
    Reporting group description
    		 MAS825 300 mg administered s.c. bi-weekly for 4 weeks, followed by monthly until Week 13.

    Reporting group title
    Cohort C - Placebo to MAS825
    Reporting group description
    		 Placebo administered s.c. bi-weekly for 4 weeks, followed by monthly until Week 13.

    Reporting group title
    Cohort D - LOU064 25 mg
    Reporting group description
    		 LOU064 25 mg administered orally twice per day until Week 16.

    Reporting group title
    Cohort D - LOU064 100 mg
    Reporting group description
    		 LOU064 100 mg administered orally twice per day until Week 16.

    Reporting group title
    Cohort D - Placebo to LOU064
    Reporting group description
    		 Placebo administered orally twice per day until Week 16.

    Reporting group values
    		 Cohort A - CFZ533 600 mg Cohort A - Placebo to CFZ533 Cohort B - LYS006 20 mg Cohort B - Placebo to LYS006 Cohort C - MAS825 300 mg Cohort C - Placebo to MAS825 Cohort D - LOU064 25 mg Cohort D - LOU064 100 mg Cohort D - Placebo to LOU064 Total
    Number of subjects
    		 29 16 27 13 33 10 33 33 11 205
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0 0 0 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0 0 0 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0 0 0 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0 0 0 0 0 0 0
        Children (2-11 years)
    		 0 0 0 0 0 0 0 0 0 0
        Adolescents (12-17 years)
    		 0 0 0 0 0 0 0 0 0 0
        Adults (18-64 years)
    		 29 16 27 13 33 10 33 33 11 205
        From 65-84 years
    		 0 0 0 0 0 0 0 0 0 0
        85 years and over
    		 0 0 0 0 0 0 0 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 38.0 ( 8.68 ) 41.4 ( 9.54 ) 36.4 ( 9.62 ) 36.5 ( 13.29 ) 39.5 ( 8.22 ) 36.9 ( 10.26 ) 35.6 ( 10.84 ) 35.3 ( 10.99 ) 44.1 ( 7.83 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 16 10 17 7 19 3 18 20 3 113
        Male
    		 13 6 10 6 14 7 15 13 8 92
    Race/Ethnicity, Customized
    Units: Subjects
        Asian
    		 1 0 1 0 0 0 0 1 0 3
        Black Or African American
    		 1 1 1 0 1 0 1 1 1 7
        Other
    		 1 0 1 0 5 0 2 0 0 9
        Unknown
    		 0 0 0 0 1 0 0 0 0 1
        White
    		 26 15 24 13 26 10 30 31 10 185

    End points

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    End points reporting groups
    Reporting group title
    Cohort A - CFZ533 600 mg
    Reporting group description
    		 CFZ533 600 mg administered subcutaneous (s.c) weekly for 4 weeks, followed by bi-weekly until Week 15.

    Reporting group title
    Cohort A - Placebo to CFZ533
    Reporting group description
    		 Placebo administered subcutaneous (s.c) weekly for 4 weeks, followed by bi-weekly until Week 15.

    Reporting group title
    Cohort B - LYS006 20 mg
    Reporting group description
    		 LYS006 20 mg administered orally twice per day until Week 16.

    Reporting group title
    Cohort B - Placebo to LYS006
    Reporting group description
    		 Placebo administered orally twice per day until Week 16.

    Reporting group title
    Cohort C - MAS825 300 mg
    Reporting group description
    		 MAS825 300 mg administered s.c. bi-weekly for 4 weeks, followed by monthly until Week 13.

    Reporting group title
    Cohort C - Placebo to MAS825
    Reporting group description
    		 Placebo administered s.c. bi-weekly for 4 weeks, followed by monthly until Week 13.

    Reporting group title
    Cohort D - LOU064 25 mg
    Reporting group description
    		 LOU064 25 mg administered orally twice per day until Week 16.

    Reporting group title
    Cohort D - LOU064 100 mg
    Reporting group description
    		 LOU064 100 mg administered orally twice per day until Week 16.

    Reporting group title
    Cohort D - Placebo to LOU064
    Reporting group description
    		 Placebo administered orally twice per day until Week 16.

    Subject analysis set title
    Pooled Placebo (Cohorts A, B and C)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Placebo to CFZ533, LYS006 and MAS825.

    Subject analysis set title
    Pooled Placebo (Cohorts A, B, C and D)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Placebo to CFZ533, LYS006, MAS825 and LOU064.

    Primary: Percentage of participants achieving Clinical Response measured by simplified Hidradenitis Suppurativa (sHiSCR)

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    End point title
    		 Percentage of participants achieving Clinical Response measured by simplified Hidradenitis Suppurativa (sHiSCR) [1]
    End point description
    sHiSCR was defined as at least a 50 percent (%) reduction in abscess and inflammatory nodule (AN) counts, and no increase in draining fistula count related to baseline. The primary variable was modeled with the binomial distribution. A neutral non-informative Beta (1/3, 1/3) distribution was used as the prior for the response rate for all treatment groups. Based on the priors and the observed primary outcome, posterior distributions for the response rate for the investigational treatment and pooled placebo groups were computed respectively. At the time of the statistical comparison for cohorts A, B, and C, the placebo data for cohorts D and E were incomplete and therefore excluded. Similarly, during the comparison for cohort D, the placebo data for cohort E was still pending and was not included.
    End point type
    Primary
    End point timeframe
    Baseline, Week 16
    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: The end point is specific for the arms presented.
    End point values
    		 Cohort A - CFZ533 600 mg Cohort B - LYS006 20 mg Cohort C - MAS825 300 mg Cohort D - LOU064 25 mg Cohort D - LOU064 100 mg Pooled Placebo (Cohorts A, B and C) Pooled Placebo (Cohorts A, B, C and D)
    Number of subjects analysed
    29
    25
    32
    33
    33
    39
    49
    Units: percentage of participants
        number (not applicable)
    58.62
    32.00
    46.88
    72.73
    48.48
    35.9
    34.69
    Statistical analysis title
    		 Clinical Response
    Comparison groups
    Cohort A - CFZ533 600 mg v Pooled Placebo (Cohorts A, B and C)
    Number of subjects included in analysis
    68
    Analysis specification
    Pre-specified
    Analysis type
    		 [2]
    Method
    Parameter type
    		 Posterior estimate treatment difference
    Point estimate
    22.1
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 2.5
         upper limit
    		 41
    Notes
    [2] - Posterior estimate treatment difference for percentage of responders
    Statistical analysis title
    		 Clinical Response
    Comparison groups
    Cohort B - LYS006 20 mg v Pooled Placebo (Cohorts A, B and C)
    Number of subjects included in analysis
    64
    Analysis specification
    Pre-specified
    Analysis type
    		 [3]
    Method
    Parameter type
    		 Posterior estimate treatment difference
    Point estimate
    -3.3
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -21.9
         upper limit
    		 15.9
    Notes
    [3] - Posterior estimate treatment difference for percentage of responders
    Statistical analysis title
    		 Clinical Response
    Comparison groups
    Cohort C - MAS825 300 mg v Pooled Placebo (Cohorts A, B and C)
    Number of subjects included in analysis
    71
    Analysis specification
    Pre-specified
    Analysis type
    		 [4]
    Method
    Parameter type
    		 Posterior estimate treatment difference
    Point estimate
    12.2
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -7.5
         upper limit
    		 31.6
    Notes
    [4] - Posterior estimate treatment difference for percentage of responders
    Statistical analysis title
    		 Clinical Response
    Comparison groups
    Cohort D - LOU064 25 mg v Pooled Placebo (Cohorts A, B, C and D)
    Number of subjects included in analysis
    82
    Analysis specification
    Pre-specified
    Analysis type
    		 [5]
    Method
    Parameter type
    		 Posterior estimate treatment difference
    Point estimate
    37.2
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 19.7
         upper limit
    		 53
    Notes
    [5] - Posterior estimate treatment difference for percentage of responders
    Statistical analysis title
    		 Clinical Response
    Comparison groups
    Cohort D - LOU064 100 mg v Pooled Placebo (Cohorts A, B, C and D)
    Number of subjects included in analysis
    82
    Analysis specification
    Pre-specified
    Analysis type
    		 [6]
    Method
    Parameter type
    		 Posterior estimate treatment difference
    Point estimate
    13.9
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -4.2
         upper limit
    		 31.9
    Notes
    [6] - Posterior estimate treatment difference for percentage of responders

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Cohorts A, B and C: 28 weeks including 12 weeks follow up period. Cohort D: 20 weeks including 4 weeks follow up period.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.1
    Reporting groups
    Reporting group title
    Cohort A-CFZ533 600 mg
    Reporting group description
    		 CFZ533 600 mg

    Reporting group title
    Cohort C-MAS825 300 mg
    Reporting group description
    		 MAS825 300 mg

    Reporting group title
    Cohort B-LYS006 20 mg
    Reporting group description
    		 LYS006 20 mg

    Reporting group title
    Cohort D-LOU064 25 mg
    Reporting group description
    		 LOU064 25 mg

    Reporting group title
    Cohort D-LOU064 100 mg
    Reporting group description
    		 LOU064 100 mg

    Reporting group title
    Pooled Placebo
    Reporting group description
    		 Pooled Placebo (Cohorts A, B, C and D)

    Reporting group title
    Total
    Reporting group description
    		 Total

    Serious adverse events
    		 Cohort A-CFZ533 600 mg Cohort C-MAS825 300 mg Cohort B-LYS006 20 mg Cohort D-LOU064 25 mg Cohort D-LOU064 100 mg Pooled Placebo Total
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 29 (13.79%)
    1 / 33 (3.03%)
    2 / 27 (7.41%)
    1 / 33 (3.03%)
    1 / 33 (3.03%)
    1 / 50 (2.00%)
    10 / 205 (4.88%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    0
    Investigations
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    1 / 27 (3.70%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Hypertensive crisis
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    0 / 33 (0.00%)
    1 / 33 (3.03%)
    0 / 50 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Pancreatitis acute
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    1 / 33 (3.03%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Ovarian cyst
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Testicular torsion
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Alcohol withdrawal syndrome
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    1 / 27 (3.70%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Depression
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 33 (3.03%)
    0 / 27 (0.00%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Abscess limb
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Genital abscess
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Testicular abscess
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    1 / 50 (2.00%)
    1 / 205 (0.49%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    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
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Cohort A-CFZ533 600 mg Cohort C-MAS825 300 mg Cohort B-LYS006 20 mg Cohort D-LOU064 25 mg Cohort D-LOU064 100 mg Pooled Placebo Total
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    23 / 29 (79.31%)
    22 / 33 (66.67%)
    19 / 27 (70.37%)
    26 / 33 (78.79%)
    15 / 33 (45.45%)
    33 / 50 (66.00%)
    138 / 205 (67.32%)
    Investigations
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    2 / 33 (6.06%)
    0 / 33 (0.00%)
    1 / 50 (2.00%)
    3 / 205 (1.46%)
         occurrences all number
    0
    0
    0
    2
    0
    1
    3
    Bacterial test positive
         subjects affected / exposed
    2 / 29 (6.90%)
    1 / 33 (3.03%)
    1 / 27 (3.70%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    4 / 205 (1.95%)
         occurrences all number
    2
    1
    1
    0
    0
    0
    4
    Blood glucose increased
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    2 / 33 (6.06%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    2 / 205 (0.98%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    2
    Blood urine present
         subjects affected / exposed
    2 / 29 (6.90%)
    1 / 33 (3.03%)
    0 / 27 (0.00%)
    2 / 33 (6.06%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    5 / 205 (2.44%)
         occurrences all number
    2
    1
    0
    2
    0
    0
    5
    Nitrite urine present
         subjects affected / exposed
    2 / 29 (6.90%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    2 / 205 (0.98%)
         occurrences all number
    3
    0
    0
    0
    0
    0
    3
    Crystal urine present
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    1 / 27 (3.70%)
    0 / 33 (0.00%)
    1 / 33 (3.03%)
    5 / 50 (10.00%)
    7 / 205 (3.41%)
         occurrences all number
    0
    0
    1
    0
    1
    6
    8
    C-reactive protein increased
         subjects affected / exposed
    0 / 29 (0.00%)
    2 / 33 (6.06%)
    0 / 27 (0.00%)
    2 / 33 (6.06%)
    0 / 33 (0.00%)
    3 / 50 (6.00%)
    7 / 205 (3.41%)
         occurrences all number
    0
    2
    0
    2
    0
    4
    8
    Urine albumin/creatinine ratio increased
         subjects affected / exposed
    2 / 29 (6.90%)
    1 / 33 (3.03%)
    3 / 27 (11.11%)
    1 / 33 (3.03%)
    1 / 33 (3.03%)
    2 / 50 (4.00%)
    10 / 205 (4.88%)
         occurrences all number
    3
    2
    3
    1
    1
    2
    12
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 33 (3.03%)
    1 / 27 (3.70%)
    2 / 33 (6.06%)
    0 / 33 (0.00%)
    4 / 50 (8.00%)
    9 / 205 (4.39%)
         occurrences all number
    1
    1
    1
    3
    0
    4
    10
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 29 (6.90%)
    5 / 33 (15.15%)
    4 / 27 (14.81%)
    4 / 33 (12.12%)
    0 / 33 (0.00%)
    6 / 50 (12.00%)
    21 / 205 (10.24%)
         occurrences all number
    4
    7
    4
    9
    0
    8
    32
    Dizziness
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 33 (0.00%)
    2 / 27 (7.41%)
    2 / 33 (6.06%)
    1 / 33 (3.03%)
    1 / 50 (2.00%)
    7 / 205 (3.41%)
         occurrences all number
    1
    0
    3
    2
    1
    1
    8
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 29 (3.45%)
    3 / 33 (9.09%)
    4 / 27 (14.81%)
    3 / 33 (9.09%)
    1 / 33 (3.03%)
    4 / 50 (8.00%)
    16 / 205 (7.80%)
         occurrences all number
    1
    3
    4
    3
    1
    4
    16
    Asthenia
         subjects affected / exposed
    3 / 29 (10.34%)
    2 / 33 (6.06%)
    0 / 27 (0.00%)
    2 / 33 (6.06%)
    0 / 33 (0.00%)
    3 / 50 (6.00%)
    10 / 205 (4.88%)
         occurrences all number
    3
    2
    0
    2
    0
    3
    10
    Pyrexia
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 33 (3.03%)
    3 / 27 (11.11%)
    2 / 33 (6.06%)
    0 / 33 (0.00%)
    2 / 50 (4.00%)
    9 / 205 (4.39%)
         occurrences all number
    1
    2
    3
    2
    0
    2
    10
    Blood and lymphatic system disorders
    Increased tendency to bruise
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    0 / 33 (0.00%)
    2 / 33 (6.06%)
    0 / 50 (0.00%)
    2 / 205 (0.98%)
         occurrences all number
    0
    0
    0
    0
    2
    0
    2
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 33 (6.06%)
    1 / 27 (3.70%)
    2 / 33 (6.06%)
    3 / 33 (9.09%)
    5 / 50 (10.00%)
    14 / 205 (6.83%)
         occurrences all number
    1
    2
    1
    2
    3
    5
    14
    Constipation
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    1 / 33 (3.03%)
    2 / 33 (6.06%)
    1 / 50 (2.00%)
    4 / 205 (1.95%)
         occurrences all number
    0
    0
    0
    1
    2
    1
    4
    Nausea
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 33 (0.00%)
    1 / 27 (3.70%)
    2 / 33 (6.06%)
    0 / 33 (0.00%)
    2 / 50 (4.00%)
    6 / 205 (2.93%)
         occurrences all number
    1
    0
    1
    2
    0
    3
    7
    Flatulence
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    2 / 27 (7.41%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    2 / 205 (0.98%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    2
    Abdominal pain
         subjects affected / exposed
    2 / 29 (6.90%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    1 / 33 (3.03%)
    1 / 33 (3.03%)
    2 / 50 (4.00%)
    6 / 205 (2.93%)
         occurrences all number
    2
    0
    0
    1
    1
    2
    6
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 33 (3.03%)
    2 / 27 (7.41%)
    2 / 33 (6.06%)
    1 / 33 (3.03%)
    0 / 50 (0.00%)
    7 / 205 (3.41%)
         occurrences all number
    1
    1
    2
    2
    1
    0
    7
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    2 / 33 (6.06%)
    1 / 33 (3.03%)
    2 / 50 (4.00%)
    6 / 205 (2.93%)
         occurrences all number
    1
    0
    0
    2
    1
    2
    6
    Pain of skin
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    2 / 33 (6.06%)
    1 / 33 (3.03%)
    0 / 50 (0.00%)
    3 / 205 (1.46%)
         occurrences all number
    0
    0
    0
    2
    1
    0
    3
    Hidradenitis
         subjects affected / exposed
    0 / 29 (0.00%)
    5 / 33 (15.15%)
    2 / 27 (7.41%)
    1 / 33 (3.03%)
    0 / 33 (0.00%)
    5 / 50 (10.00%)
    13 / 205 (6.34%)
         occurrences all number
    0
    6
    3
    1
    0
    5
    15
    Erythema
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 33 (3.03%)
    0 / 27 (0.00%)
    2 / 33 (6.06%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    4 / 205 (1.95%)
         occurrences all number
    1
    1
    0
    2
    0
    0
    4
    Renal and urinary disorders
    Proteinuria
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 33 (0.00%)
    1 / 27 (3.70%)
    2 / 33 (6.06%)
    0 / 33 (0.00%)
    2 / 50 (4.00%)
    6 / 205 (2.93%)
         occurrences all number
    1
    0
    1
    2
    0
    2
    6
    Psychiatric disorders
    Depression
         subjects affected / exposed
    0 / 29 (0.00%)
    2 / 33 (6.06%)
    1 / 27 (3.70%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    3 / 205 (1.46%)
         occurrences all number
    0
    2
    1
    0
    0
    0
    3
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    2 / 29 (6.90%)
    1 / 33 (3.03%)
    1 / 27 (3.70%)
    1 / 33 (3.03%)
    0 / 33 (0.00%)
    1 / 50 (2.00%)
    6 / 205 (2.93%)
         occurrences all number
    2
    1
    1
    1
    0
    1
    6
    Myalgia
         subjects affected / exposed
    2 / 29 (6.90%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    2 / 205 (0.98%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    2
    Back pain
         subjects affected / exposed
    2 / 29 (6.90%)
    1 / 33 (3.03%)
    1 / 27 (3.70%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    1 / 50 (2.00%)
    5 / 205 (2.44%)
         occurrences all number
    2
    1
    2
    0
    0
    1
    6
    Arthralgia
         subjects affected / exposed
    3 / 29 (10.34%)
    0 / 33 (0.00%)
    1 / 27 (3.70%)
    1 / 33 (3.03%)
    0 / 33 (0.00%)
    2 / 50 (4.00%)
    7 / 205 (3.41%)
         occurrences all number
    3
    0
    1
    1
    0
    4
    9
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    1 / 29 (3.45%)
    3 / 33 (9.09%)
    4 / 27 (14.81%)
    7 / 33 (21.21%)
    2 / 33 (6.06%)
    1 / 50 (2.00%)
    18 / 205 (8.78%)
         occurrences all number
    2
    3
    4
    7
    2
    1
    19
    Influenza
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    1 / 33 (3.03%)
    0 / 33 (0.00%)
    3 / 50 (6.00%)
    4 / 205 (1.95%)
         occurrences all number
    0
    0
    0
    1
    0
    3
    4
    Ear infection
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    2 / 27 (7.41%)
    2 / 33 (6.06%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    4 / 205 (1.95%)
         occurrences all number
    0
    0
    2
    2
    0
    0
    4
    COVID-19
         subjects affected / exposed
    3 / 29 (10.34%)
    8 / 33 (24.24%)
    1 / 27 (3.70%)
    2 / 33 (6.06%)
    2 / 33 (6.06%)
    3 / 50 (6.00%)
    19 / 205 (9.27%)
         occurrences all number
    3
    8
    1
    2
    2
    3
    19
    Bacteriuria
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 33 (3.03%)
    0 / 27 (0.00%)
    2 / 33 (6.06%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    3 / 205 (1.46%)
         occurrences all number
    0
    1
    0
    2
    0
    0
    3
    Sinusitis
         subjects affected / exposed
    2 / 29 (6.90%)
    3 / 33 (9.09%)
    0 / 27 (0.00%)
    0 / 33 (0.00%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    5 / 205 (2.44%)
         occurrences all number
    2
    3
    0
    0
    0
    0
    5
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    2 / 33 (6.06%)
    1 / 33 (3.03%)
    2 / 50 (4.00%)
    6 / 205 (2.93%)
         occurrences all number
    1
    0
    0
    2
    2
    2
    7
    Tonsillitis
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 33 (0.00%)
    0 / 27 (0.00%)
    2 / 33 (6.06%)
    0 / 33 (0.00%)
    0 / 50 (0.00%)
    2 / 205 (0.98%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    24 Jul 2019
    The main purpose of this amendment was to remove the requirement for male contraception in the LYS006 cohort given that completed preclinical safety studies with LYS006 showed there was no teratogenic or genotoxic potential observed with LYS006
    19 Jun 2020
    The main purpose of this amendment was to address requests from Health Authorities and Ethics Committees, as well as to correct some inconsistencies in the protocol discovered during implementation.
    26 Feb 2021
    The main purpose of this amendment was to introduce an additional cohort (Cohort C) into this platform study.
    03 Dec 2021
    The main purpose of this amendment was to introduce an additional cohort (Cohort D) into this platform study.
    03 Feb 2022
    The purpose of this amendment was to add a blood sample for coagulation parameters to Cohort D, for safety monitoring reasons.
    03 Jun 2022
    The purpose of this amendment was to address comments raised by the Health Authority.
    14 Mar 2023
    The main purpose of this amendment was to introduce an additional cohort (Cohort E) into this platform study.
    12 Jul 2023
    The main purpose of this amendment was to update the dosage form for Cohort E.
    29 Nov 2023
    The main purpose of this amendment was to revise the exclusion criteria specific to Cohort E.
    24 May 2024
    The main purpose of this amendment was to introduce the Auxiliary Medicinal Products (AxMP) definition and related safety reporting rules, to comply with EU Clinical Trial Regulation 536/2014 (EU CTR).

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    24 May 2023
    Novartis filed a study Temporary Halt in May 2023 to maintain the study active as Cohort D was about to be completed and the submission of Cohort E-related protocol amendment (Amendment No. 8) was in process of preparation.
    09 Jan 2024

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    For support, Contact us.
    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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