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    Clinical Trial Results:
    Investigator- and subject-blinded, randomized, placebo-controlled study to evaluate safety, tolerability, pharmacokinetics and efficacy trial of CFZ533 in pediatric and young adult subjects with new onset type 1 diabetes (T1DM)

    Summary
    EudraCT number
    		 2018-004553-25
    Trial protocol
    		 BE   SI   DE   IT  
    Global end of trial date
    04 Jun 2024

    Results information
    Results version number
    		 v2(current)
    This version publication date
    02 Feb 2025
    First version publication date
    18 Dec 2024
    Other versions
    		 v1
    Version creation reason

    Trial information

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    Trial identification
    Sponsor protocol code
    CCFZ533X2207
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04129528
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharmaceuticals
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    04 Jun 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    04 Jun 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Primary objectives: • To evaluate effects of CFZ533 on pancreatic beta cell function in subjects with new-onset Type 1 Diabetes Mellitus (T1DM). • To evaluate the safety and tolerability of CFZ533 in subjects with new onset T1DM.
    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
    08 Nov 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
    Belgium: 13
    Country: Number of subjects enrolled
    Spain: 21
    Country: Number of subjects enrolled
    Slovenia: 1
    Country: Number of subjects enrolled
    Italy: 5
    Country: Number of subjects enrolled
    Germany: 2
    Country: Number of subjects enrolled
    United Kingdom: 2
    Worldwide total number of subjects
    44
    EEA total number of subjects
    42
    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)
    35
    Adults (18-64 years)
    9
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants took part in 12 investigative sites in 6 countries.

    Pre-assignment
    Screening details
    		 Enrolment was based on both screening and baseline results. The screening and baseline visit(s) may be conducted over 1 or more visits depending on the subject’s body weight and World Health Organization and European Medicines Agency (EMA) recommendations for trial related phlebotomy limits.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 CFZ533
    Arm description
    		 CFZ533 30 mg/kg i.v. dose on Day 1. From Day 8 up to Day 358 participants with body weight of ≥30 to <50 kg received 195 mg s.c., and participants with body weight of ≥ 50 kg to ≤125 kg received 300 mg s.c. on a weekly basis.
    Arm type
    		 Experimental

    Investigational medicinal product name
    CFZ533
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    CFZ533 30 mg/kg i.v. dose on Day 1.

    Investigational medicinal product name
    CFZ533
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    CFZ533 From Day 8 up to Day 358 participants with body weight of ≥30 to <50 kg received 195 mg s.c., and participants with body weight of ≥ 50 kg to ≤125 kg received 300 mg s.c. on a weekly basis.

    Arm title
    		 Placebo
    Arm description
    		 Placebo i.v. dose on Day 1. From Day 8 up to Day 358 participants with body weight of ≥30 to <50 kg and body weight of ≥ 50 kg to ≤125 kg received matching placebo s.c. on a weekly basis.
    Arm type
    		 Placebo

    Investigational medicinal product name
    CFZ533
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Placebo From Day 8 up to Day 358 participants with body weight of ≥30 to <50 kg and body weight of ≥ 50 kg to ≤125 kg received matching placebo s.c. on a weekly basis.

    Investigational medicinal product name
    CFZ533
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Placebo i.v. dose on Day 1.

    Number of subjects in period 1
    		CFZ533 Placebo
    Started
    29
    15
    Completed
    26
    13
    Not completed
    3
    2
         Physician decision
    1
    1
         Consent withdrawn by subject
    1
    1
         Adverse event
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    CFZ533
    Reporting group description
    		 CFZ533 30 mg/kg i.v. dose on Day 1. From Day 8 up to Day 358 participants with body weight of ≥30 to <50 kg received 195 mg s.c., and participants with body weight of ≥ 50 kg to ≤125 kg received 300 mg s.c. on a weekly basis.

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo i.v. dose on Day 1. From Day 8 up to Day 358 participants with body weight of ≥30 to <50 kg and body weight of ≥ 50 kg to ≤125 kg received matching placebo s.c. on a weekly basis.

    Reporting group values
    		 CFZ533 Placebo Total
    Number of subjects
    		 29 15 44
    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)
    		 23 12 35
        Adults (18-64 years)
    		 6 3 9
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 15.6 ( 2.81 ) 15.5 ( 3.07 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 10 5 15
        Male
    		 19 10 29
    Race/Ethnicity, Customized
    Units: Subjects
        White
    		 29 15 44

    End points

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    End points reporting groups
    Reporting group title
    CFZ533
    Reporting group description
    		 CFZ533 30 mg/kg i.v. dose on Day 1. From Day 8 up to Day 358 participants with body weight of ≥30 to <50 kg received 195 mg s.c., and participants with body weight of ≥ 50 kg to ≤125 kg received 300 mg s.c. on a weekly basis.

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo i.v. dose on Day 1. From Day 8 up to Day 358 participants with body weight of ≥30 to <50 kg and body weight of ≥ 50 kg to ≤125 kg received matching placebo s.c. on a weekly basis.

    Primary: Number of participants with treatment emergent adverse events (AEs) and serious adverse events (SAEs) during the on-treatment period

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    End point title
    		 Number of participants with treatment emergent adverse events (AEs) and serious adverse events (SAEs) during the on-treatment period [1]
    End point description
    Number of participants with treatment emergent AEs (any AE regardless of seriousness), AEs led to study treatment discontinuation, and SAEs. On-treatment period is defined as from date of first administration of study treatment to 98 days after date of last administration of study treatment (including start and stop date).
    End point type
    Primary
    End point timeframe
    Adverse events were reported from first dose of study treatment to 98 days after last dose, up to a maximum duration of approximately 65 weeks
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: only analyzed descriptively.
    End point values
    		 CFZ533 Placebo
    Number of subjects analysed
    29
    15
    Units: participants
        Adverse Events
    28
    13
        Serious Adverse Events
    4
    1
        AEs leading to discontinuation of study treatment
    4
    0
    No statistical analyses for this end point

    Primary: Normalized stimulated C-peptide area under the curve (AUC) at Week 52

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    End point title
    		 Normalized stimulated C-peptide area under the curve (AUC) at Week 52
    End point description
    The mixed meal tolerance test (MMTT) has appropriate sensitivity to detect residual insulin secretion and beta cell function. In the MMTT, following an 8-10 hour overnight fast, a weight-based liquid meal provided as 6 mL/kg (maximum 360 mL) of mixed meal, ingested over 5 min with timed blood samples for glucose and C peptide determination obtained 10 min prior to ingestion (t = −10), at baseline (t = 0), and at 15, 30, 60, 90, and 120 min after consumption of the liquid meal. The time collections for post load samples are based on the start time of the mixed meal. Stimulated C-peptide AUC by the standard MMTT, normalized by the duration of measurements, was analyzed with a mixed model repeated measures analysis.
    End point type
    Primary
    End point timeframe
    At Week 52, 10 min prior to ingestion, at start of ingestion, and at 15, 30, 60, 90, and 120 min after consumption of the liquid meal.
    End point values
    		 CFZ533 Placebo
    Number of subjects analysed
    29
    15
    Units: nmol/L
        geometric mean (confidence interval 80%)
    0.42 (0.36 to 0.49)
    0.36 (0.30 to 0.44)
    Statistical analysis title
    		 Normalized stimulated C-peptide AUC
    Comparison groups
    CFZ533 v Placebo
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.1817 [2]
    Method
    		 Mixed model repeated measure analysis
    Parameter type
    		 Ratio of geometric means CFZ533/placebo
    Point estimate
    1.173
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.94
         upper limit
    		 1.47
    Notes
    [2] - one-sided P-value

    Secondary: Maximum plasma concentration (Cmax) of CFZ533 after intravenous (IV) administration

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    End point title
    		 Maximum plasma concentration (Cmax) of CFZ533 after intravenous (IV) administration [3]
    End point description
    Cmax is defined as the maximum (peak) observed concentration following a dose. Free CFZ533 plasma concentrations were determined using a validated target-based sandwich enzyme-linked immunosorbent assay (ELISA) method.
    End point type
    Secondary
    End point timeframe
    Day 1: Pre-dose and 90 minutes after the start of the IV infusion (duration of the infusion is 30 minutes).
    Notes
    [3] - 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: PK Endpoint not analyzed for participants on placebo
    End point values
    		 CFZ533
    Number of subjects analysed
    29
    Units: ug/mL
        arithmetic mean (standard deviation)
    506 ( 124 )
    No statistical analyses for this end point

    Secondary: Trough plasma concentration (Ctrough) of CFZ533

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    End point title
    		 Trough plasma concentration (Ctrough) of CFZ533 [4]
    End point description
    Ctrough is the observed plasma concentration that is just prior to the beginning of, or at the end of a dosing interval. Free CFZ533 plasma concentrations were determined using a validated target-based sandwich ELISA method. The Number of Subjects Analyzed differs as stated on the category column, in case of difference from Number of subjects that started the Arm.
    End point type
    Secondary
    End point timeframe
    Pre-dose at: Day 1, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, Week 28, Week 32, Week 36, Week 40, Week 44, Week 48, Week 52, Week 56, Week 60, Week 64, Week 68, Week 72.
    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: PK Endpoint not analyzed for participants on placebo
    End point values
    		 CFZ533
    Number of subjects analysed
    29
    Units: ug/mL
    arithmetic mean (standard deviation)
        Day 1 (n=27)
    0.00 ( 0.00 )
        Week 4 (n=26)
    182 ( 40.9 )
        Week 8 (n=25)
    171 ( 45.8 )
        Week 12 (n=21)
    169 ( 43.1 )
        Week 16 (n=18)
    193 ( 55.2 )
        Week 20 (n=20)
    184 ( 68.1 )
        Week 24 (n=16)
    176 ( 65.2 )
        Week 28 (n=18)
    186 ( 61.4 )
        Week 32 (n=16)
    177 ( 65.4 )
        Week 36 (n=17)
    180 ( 86.7 )
        Week 40 (n=15)
    183 ( 80.0 )
        Week 44 (n=14)
    170 ( 88.7 )
        Week 48 (n=15)
    205 ( 101 )
        Week 52 (n=29)
    155 ( 111 )
        Week 56 (n=26)
    72.6 ( 53.3 )
        Week 60 (n=25)
    30.9 ( 24.9 )
        Week 64 (n=26)
    9.23 ( 11.7 )
        Week 68 (n=25)
    1.86 ( 3.15 )
        Week 72 (n=26)
    0.221 ( 0.499 )
    No statistical analyses for this end point

    Secondary: Time to reach maximum plasma concentration (Tmax) of CFZ533 after IV administration

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    End point title
    		 Time to reach maximum plasma concentration (Tmax) of CFZ533 after IV administration [5]
    End point description
    Tmax is the time to reach maximum (peak) drug concentration after single-dose administration (time). Free CFZ533 plasma concentrations were determined using a validated target-based sandwich ELISA method. Theoretical sampling time points were used to report Tmax.
    End point type
    Secondary
    End point timeframe
    Day 1: Pre-dose and 90 minutes after the start of the IV infusion (duration of the infusion is 30 minutes).
    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: PK Endpoint not analyzed for participants on placebo
    End point values
    		 CFZ533
    Number of subjects analysed
    29
    Units: hours
        median (full range (min-max))
    1.5 (1.5 to 1.5)
    No statistical analyses for this end point

    Secondary: Number of participants with full or partial remission

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    End point title
    		 Number of participants with full or partial remission
    End point description
    Full remission is defined by HbA1c ≤ 6.5% (48 mmol/mol) and no exogenous insulin use at Week 52. Partial remission 1 is defined by Insulin Dose Adjusted HbA1c (IDAA1c) ≤ 9.0 at Week 52. Partial remission 2 is defined by HbA1c < 7.0% (53 mmol/mol) and total daily insulin dose <0.5 units per kg per day at Week 52. Two different criteria for partial remission were considered, and patients were assessed separately according to each criterion.
    End point type
    Secondary
    End point timeframe
    Week 52
    End point values
    		 CFZ533 Placebo
    Number of subjects analysed
    29
    15
    Units: participants
        Full remission
    0
    0
        Partial remission 1
    20
    10
        Partial remission 2
    15
    6
    No statistical analyses for this end point

    Secondary: Normalized stimulated C-peptide area under the curve (AUC) at Week 72

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    End point title
    		 Normalized stimulated C-peptide area under the curve (AUC) at Week 72
    End point description
    The mixed meal tolerance test (MMTT) has appropriate sensitivity to detect residual insulin secretion and beta cell function. In the MMTT, following an 8-10 hour overnight fast, a weight-based liquid meal provided as 6 mL/kg (maximum 360 mL) of mixed meal, ingested over 5 min with timed blood samples for glucose and C peptide determination obtained 10 min prior to ingestion (t = −10), at baseline (t = 0), and at 15, 30, 60, 90, and 120 min after consumption of the liquid meal. The time collections for post load samples are based on the start time of the mixed meal. Stimulated C-peptide AUC by the standard MMTT, normalized by the duration of measurements, was analyzed with a mixed model repeated measures analysis.
    End point type
    Secondary
    End point timeframe
    At Week 72, 10 min prior to ingestion, at start of ingestion, and at 15, 30, 60, 90, and 120 min after consumption of the liquid meal.
    End point values
    		 CFZ533 Placebo
    Number of subjects analysed
    29
    15
    Units: nmol/L
        geometric mean (confidence interval 80%)
    0.40 (0.34 to 0.47)
    0.32 (0.26 to 0.39)
    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 follow up period, up to a maximum duration of approximately 72 weeks.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.0
    Reporting groups
    Reporting group title
    CFZ533
    Reporting group description
    		 CFZ533

    Reporting group title
    Total
    Reporting group description
    		 Total

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo

    Serious adverse events
    		 CFZ533 Total Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    6 / 29 (20.69%)
    7 / 44 (15.91%)
    1 / 15 (6.67%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Injury, poisoning and procedural complications
    Traumatic fracture
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 44 (2.27%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Tonic clonic movements
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 44 (2.27%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 44 (2.27%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 44 (2.27%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Large intestine infection
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 44 (2.27%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Mastoiditis
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 44 (2.27%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Diabetic metabolic decompensation
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 44 (2.27%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hypoglycaemia
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 44 (2.27%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 CFZ533 Total Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    27 / 29 (93.10%)
    41 / 44 (93.18%)
    14 / 15 (93.33%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Skin papilloma
         subjects affected / exposed
    2 / 29 (6.90%)
    2 / 44 (4.55%)
    0 / 15 (0.00%)
         occurrences all number
    2
    2
    0
    General disorders and administration site conditions
    Malaise
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Medical device pain
         subjects affected / exposed
    2 / 29 (6.90%)
    2 / 44 (4.55%)
    0 / 15 (0.00%)
         occurrences all number
    2
    2
    0
    Pyrexia
         subjects affected / exposed
    5 / 29 (17.24%)
    8 / 44 (18.18%)
    3 / 15 (20.00%)
         occurrences all number
    6
    10
    4
    Asthenia
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 44 (4.55%)
    1 / 15 (6.67%)
         occurrences all number
    2
    3
    1
    Injection site reaction
         subjects affected / exposed
    7 / 29 (24.14%)
    10 / 44 (22.73%)
    3 / 15 (20.00%)
         occurrences all number
    39
    114
    75
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 44 (4.55%)
    1 / 15 (6.67%)
         occurrences all number
    1
    2
    1
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    2 / 29 (6.90%)
    2 / 44 (4.55%)
    0 / 15 (0.00%)
         occurrences all number
    2
    2
    0
    Respiratory, thoracic and mediastinal disorders
    Productive cough
         subjects affected / exposed
    2 / 29 (6.90%)
    2 / 44 (4.55%)
    0 / 15 (0.00%)
         occurrences all number
    2
    2
    0
    Oropharyngeal pain
         subjects affected / exposed
    3 / 29 (10.34%)
    4 / 44 (9.09%)
    1 / 15 (6.67%)
         occurrences all number
    4
    5
    1
    Dyspnoea
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Cough
         subjects affected / exposed
    1 / 29 (3.45%)
    5 / 44 (11.36%)
    4 / 15 (26.67%)
         occurrences all number
    4
    8
    4
    Bronchial hyperreactivity
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Asthma
         subjects affected / exposed
    0 / 29 (0.00%)
    2 / 44 (4.55%)
    2 / 15 (13.33%)
         occurrences all number
    0
    2
    2
    Rhinorrhoea
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    5
    5
    Nasal congestion
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Blood creatine phosphokinase increased
         subjects affected / exposed
    1 / 29 (3.45%)
    3 / 44 (6.82%)
    2 / 15 (13.33%)
         occurrences all number
    1
    3
    2
    Lipids increased
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 44 (4.55%)
    1 / 15 (6.67%)
         occurrences all number
    1
    2
    1
    SARS-CoV-2 test positive
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Serology positive
         subjects affected / exposed
    2 / 29 (6.90%)
    2 / 44 (4.55%)
    0 / 15 (0.00%)
         occurrences all number
    2
    2
    0
    Urine analysis abnormal
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    2
    2
    Injury, poisoning and procedural complications
    Injection related reaction
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 44 (4.55%)
    1 / 15 (6.67%)
         occurrences all number
    2
    8
    6
    Limb injury
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 44 (4.55%)
    1 / 15 (6.67%)
         occurrences all number
    1
    2
    1
    Muscle strain
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Congenital, familial and genetic disorders
    Gilbert's syndrome
         subjects affected / exposed
    0 / 29 (0.00%)
    2 / 44 (4.55%)
    2 / 15 (13.33%)
         occurrences all number
    0
    2
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    7 / 29 (24.14%)
    9 / 44 (20.45%)
    2 / 15 (13.33%)
         occurrences all number
    14
    16
    2
    Blood and lymphatic system disorders
    Neutropenia
         subjects affected / exposed
    4 / 29 (13.79%)
    4 / 44 (9.09%)
    0 / 15 (0.00%)
         occurrences all number
    4
    4
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 44 (4.55%)
    1 / 15 (6.67%)
         occurrences all number
    1
    2
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    5 / 29 (17.24%)
    6 / 44 (13.64%)
    1 / 15 (6.67%)
         occurrences all number
    6
    7
    1
    Abdominal pain upper
         subjects affected / exposed
    2 / 29 (6.90%)
    4 / 44 (9.09%)
    2 / 15 (13.33%)
         occurrences all number
    2
    4
    2
    Vomiting
         subjects affected / exposed
    3 / 29 (10.34%)
    3 / 44 (6.82%)
    0 / 15 (0.00%)
         occurrences all number
    3
    3
    0
    Odynophagia
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 44 (4.55%)
    1 / 15 (6.67%)
         occurrences all number
    1
    4
    3
    Nausea
         subjects affected / exposed
    2 / 29 (6.90%)
    2 / 44 (4.55%)
    0 / 15 (0.00%)
         occurrences all number
    2
    2
    0
    Diarrhoea
         subjects affected / exposed
    4 / 29 (13.79%)
    4 / 44 (9.09%)
    0 / 15 (0.00%)
         occurrences all number
    9
    9
    0
    Hepatobiliary disorders
    Hyperbilirubinaemia
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Skin and subcutaneous tissue disorders
    Dermatitis contact
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Dermatitis atopic
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    2
    2
    Acne
         subjects affected / exposed
    2 / 29 (6.90%)
    2 / 44 (4.55%)
    0 / 15 (0.00%)
         occurrences all number
    2
    2
    0
    Ingrowing nail
         subjects affected / exposed
    2 / 29 (6.90%)
    2 / 44 (4.55%)
    0 / 15 (0.00%)
         occurrences all number
    2
    2
    0
    Endocrine disorders
    Hyperthyroidism
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    2 / 29 (6.90%)
    2 / 44 (4.55%)
    0 / 15 (0.00%)
         occurrences all number
    2
    2
    0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    7 / 29 (24.14%)
    9 / 44 (20.45%)
    2 / 15 (13.33%)
         occurrences all number
    7
    9
    2
    Chronic active Epstein-Barr virus infection
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Cytomegalovirus infection
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Epstein-Barr virus infection
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Gastroenteritis
         subjects affected / exposed
    3 / 29 (10.34%)
    5 / 44 (11.36%)
    2 / 15 (13.33%)
         occurrences all number
    3
    8
    5
    Gastroenteritis viral
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Influenza
         subjects affected / exposed
    3 / 29 (10.34%)
    6 / 44 (13.64%)
    3 / 15 (20.00%)
         occurrences all number
    4
    7
    3
    Nasopharyngitis
         subjects affected / exposed
    13 / 29 (44.83%)
    18 / 44 (40.91%)
    5 / 15 (33.33%)
         occurrences all number
    33
    42
    9
    Otitis externa
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 44 (4.55%)
    1 / 15 (6.67%)
         occurrences all number
    2
    3
    1
    Parvovirus B19 infection
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Pharyngitis
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 44 (4.55%)
    1 / 15 (6.67%)
         occurrences all number
    1
    3
    2
    Pharyngotonsillitis
         subjects affected / exposed
    0 / 29 (0.00%)
    2 / 44 (4.55%)
    2 / 15 (13.33%)
         occurrences all number
    0
    2
    2
    Respiratory tract infection
         subjects affected / exposed
    3 / 29 (10.34%)
    3 / 44 (6.82%)
    0 / 15 (0.00%)
         occurrences all number
    7
    7
    0
    Rhinitis
         subjects affected / exposed
    2 / 29 (6.90%)
    4 / 44 (9.09%)
    2 / 15 (13.33%)
         occurrences all number
    2
    5
    3
    Sinusitis
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 44 (2.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    Tinea pedis
         subjects affected / exposed
    2 / 29 (6.90%)
    2 / 44 (4.55%)
    0 / 15 (0.00%)
         occurrences all number
    2
    2
    0
    Tonsillitis
         subjects affected / exposed
    0 / 29 (0.00%)
    3 / 44 (6.82%)
    3 / 15 (20.00%)
         occurrences all number
    0
    4
    4
    Tooth abscess
         subjects affected / exposed
    0 / 29 (0.00%)
    2 / 44 (4.55%)
    2 / 15 (13.33%)
         occurrences all number
    0
    2
    2
    Upper respiratory tract infection
         subjects affected / exposed
    4 / 29 (13.79%)
    7 / 44 (15.91%)
    3 / 15 (20.00%)
         occurrences all number
    9
    12
    3
    Urinary tract infection
         subjects affected / exposed
    2 / 29 (6.90%)
    2 / 44 (4.55%)
    0 / 15 (0.00%)
         occurrences all number
    2
    2
    0
    Viral infection
         subjects affected / exposed
    2 / 29 (6.90%)
    2 / 44 (4.55%)
    0 / 15 (0.00%)
         occurrences all number
    2
    2
    0
    Metabolism and nutrition disorders
    Hyperglycaemia
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 44 (4.55%)
    1 / 15 (6.67%)
         occurrences all number
    1
    2
    1
    Hypoglycaemia
         subjects affected / exposed
    19 / 29 (65.52%)
    27 / 44 (61.36%)
    8 / 15 (53.33%)
         occurrences all number
    181
    285
    104

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    25 Jul 2019
    This protocol amendment addresses comments received from Federal Agency for Medicines and Health Products in the letter dated 08July2019.
    10 Dec 2019
    The purpose of this amendment was to include additional safety monitoring that has already been implemented via urgent safety measures (USMs) in response to a suspected unexpected serious adverse reaction that was reported in another CFZ533 study of lupus nephritis patients.
    30 Nov 2020
    The purpose of this amendment was to include additional safety monitoring in response to the newly prevalent community exposure to severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2).
    04 Jan 2021
    Based on comments by the MHRA, the age classifications in the Cohorts have been revised to be more in line with International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH) Topic E11 age classifications.
    17 Mar 2022
    The purpose of this amendment was to implement a Hybrid Trial Design which included a flexible model for incorporating both on-site (traditional on-site based) and off-site (also known as remote or decentralized) elements within the same study design.
    03 Apr 2023
    The purpose of this protocol amendment was primarily to reduce the sample size and duration of study conduct.

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    16 Mar 2020
    There was a temporary halt implemented for both recruitment and treatment of participants on 16-Mar-2020 due to the COVID-19 worldwide pandemic with the trial approved to restart on 27-Aug-2020 based on country and site feasibility coming out of COVID-19.
    27 Aug 2020

    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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