Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43865   clinical trials with a EudraCT protocol, of which   7286   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    NASH EXploratory Single and COmbination Treatment (NEXSCOT): An open label, multicenter, platform study to evaluate the safety, tolerability, pharmacokinetics and efficacy of various single and combination treatments in patients with non-alcoholic fatty liver disease (NAFLD) who manifest a non-alcoholic steatohepatitis (NASH)-like biomarker phenotype.

    Summary
    EudraCT number
    		 2019-000440-10
    Trial protocol
    		 DE  
    Global end of trial date
    06 Jan 2022

    Results information
    Results version number
    		 v2(current)
    This version publication date
    14 Feb 2023
    First version publication date
    15 Dec 2022
    Other versions
    		 v1
    Version creation reason

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    CADPT02A12001
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04147195
    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
    06 Jan 2022
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    06 Jan 2022
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To determine the safety and tolerability of single or combination therapy during 12 weeks of treatment
    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
    04 Jun 2020
    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
    Argentina: 2
    Country: Number of subjects enrolled
    Germany: 3
    Country: Number of subjects enrolled
    United States: 36
    Worldwide total number of subjects
    41
    EEA total number of subjects
    3
    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)
    38
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 Participants were recruited from 10 sites in 3 countries.

    Pre-assignment
    Screening details
    		 Participants underwent a Screening period of up to 33 days followed by a Baseline assessment period of up to 27 days.

    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
    		 LYS006
    Arm description
    		 LYS006 20 mg was administered orally twice per day (b.i.d) for 12 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    LYS006
    Investigational medicinal product code
    LYS006
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    LYS006 20 mg was administered orally twice per day (b.i.d) for 12 weeks

    Arm title
    		 LYS006 + LJN452
    Arm description
    		 LYS006 20 mg was administered orally twice per day (b.i.d) in addition to LJN452 200ug administered orally once daily for 12 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    LJN452
    Investigational medicinal product code
    LJN452
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    LJN452 200ug administered orally once daily for 12 weeks

    Investigational medicinal product name
    LYS006
    Investigational medicinal product code
    LYS006
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    LYS006 20 mg was administered orally twice per day (b.i.d) for 12 weeks

    Number of subjects in period 1
    		LYS006 LYS006 + LJN452
    Started
    20
    21
    PD analysis set
    20
    17
    Completed
    16
    15
    Not completed
    4
    6
         Consent withdrawn by subject
    -
    1
         Adverse event, non-fatal
    -
    3
         Study Terminated by Sponsor
    4
    2

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    LYS006
    Reporting group description
    		 LYS006 20 mg was administered orally twice per day (b.i.d) for 12 weeks

    Reporting group title
    LYS006 + LJN452
    Reporting group description
    		 LYS006 20 mg was administered orally twice per day (b.i.d) in addition to LJN452 200ug administered orally once daily for 12 weeks

    Reporting group values
    		 LYS006 LYS006 + LJN452 Total
    Number of subjects
    		 20 21 41
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 19 19 38
        From 65-84 years
    		 1 2 3
        85 years and over
    		 0 0 0
    Age Continuous
    Units: Year
        arithmetic mean (standard deviation)
    		 52.0 ± 9.23 54.9 ± 8.36 -
    Sex: Female, Male
    Units: Participants
        Female
    		 11 11 22
        Male
    		 9 10 19
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 0 1 1
        Native Hawaiian or Other Pacific Islander
    		 0 1 1
        Black or African American
    		 1 1 2
        White
    		 19 17 36
        More than one race
    		 0 1 1
        Unknown or Not Reported
    		 0 0 0

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    LYS006
    Reporting group description
    		 LYS006 20 mg was administered orally twice per day (b.i.d) for 12 weeks

    Reporting group title
    LYS006 + LJN452
    Reporting group description
    		 LYS006 20 mg was administered orally twice per day (b.i.d) in addition to LJN452 200ug administered orally once daily for 12 weeks

    Primary: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

    		 Close Top of page
    End point title
    		 Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) [1]
    End point description
    Number of participants with AEs and SAEs including significant changes from baseline in vital signs, electrocardiograms and laboratory parameters qualifying and reported as AEs. The number of participants in each category is reported in the table.
    End point type
    Primary
    End point timeframe
    From the start of treatment to 28 days after end of treatment, assessed up to maximum duration of 113 Days
    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: No statistical analysis were planned for this endpoint.
    End point values
    		 LYS006 LYS006 + LJN452
    Number of subjects analysed
    20
    21
    Units: Participants
        AEs
    14
    17
        Treatment-related AEs
    2
    15
        SAEs
    0
    0
        AEs leading to discontinuation of study treatment
    0
    3
    No statistical analyses for this end point

    Secondary: Change From Baseline in Non-invasive Markers of Hepatic Fibrosis: Enhanced Liver Fibrosis Test (ELF) Score

    		 Close Top of page
    End point title
    		 Change From Baseline in Non-invasive Markers of Hepatic Fibrosis: Enhanced Liver Fibrosis Test (ELF) Score
    End point description
    The markers of fibrosis assessed in this test comprised hyaluronic acid (HA), tissue inhibitor of metalloproteinase (TIMP1) and procollagen III N-terminal peptide (PIIINP); these are components of the extracellular matrix and basement sinusoidal membrane of the liver and are elevated during fibrogenesis as a result of activation of the hepatic stellate cell. The ELF test is a composite score: < 7.7: no to mild fibrosis; ≥ 7.7 - < 9.8: Moderate fibrosis; ≥ 9.8 - < 11.3: Severe fibrosis; ≥ 11.3: Cirrhosis. Baseline is defined as the last non-missing measurement taken at the Screening and Baseline visits (prior to the first dose of study drug administered at the Day 1). A negative change from Baseline indicates decreased fibrosis.
    End point type
    Secondary
    End point timeframe
    Baseline and Days 57, 85 and EOS (Day 113)
    End point values
    		 LYS006 LYS006 + LJN452
    Number of subjects analysed
    20
    17
    Units: Scores on a scale
    arithmetic mean (standard deviation)
        Day 57
    -0.25 ± 0.564
    0.29 ± 0.286
        Day 85
    -0.12 ± 0.780
    0.18 ± 0.687
        EOS
    0.01 ± 0.713
    0.09 ± 0.461
    No statistical analyses for this end point

    Secondary: Change from Baseline in Cholesterol: fasting lipid profile endpoint

    		 Close Top of page
    End point title
    		 Change from Baseline in Cholesterol: fasting lipid profile endpoint
    End point description
    Fasting lipid profile (total cholesterol) was examined as a cardiometabolic risk parameter. Total cholesterol was measured on blood samples under fasted conditions and analyzed at a central laboratory. Baseline is defined as the last non-missing measurement taken at the Screening and Baseline visits (prior to the first dose of study drug administered at the Day 1). A negative change from Baseline indicates cardiovascular risk.
    End point type
    Secondary
    End point timeframe
    Baseline and Days 15, 29, 43, 57, 85 and EOS (Day 113)
    End point values
    		 LYS006 LYS006 + LJN452
    Number of subjects analysed
    20
    17
    Units: mmol / L
    arithmetic mean (standard deviation)
        Day 15
    -0.025 ± 0.5814
    0.086 ± 0.8825
        Day 29
    -0.058 ± 0.6058
    0.431 ± 1.0688
        Day 43
    -0.109 ± 0.6758
    0.474 ± 1.0364
        Day 57
    -0.246 ± 0.6341
    0.463 ± 1.3026
        Day 85
    0.001 ± 0.8558
    0.754 ± 0.9915
        EOS
    -0.283 ± 1.1182
    0.249 ± 0.5175
    No statistical analyses for this end point

    Secondary: Change from Baseline in percent liver fat at day 85

    		 Close Top of page
    End point title
    		 Change from Baseline in percent liver fat at day 85
    End point description
    Percent (%) Liver fat was measured by Magnetic Resonance Imaging Proton Density Liver Fat Fraction (MRIPDFF). Participants underwent magnetic resonance imaging twice during the course of the study (baseline and end of treatment) to quantitate liver fat. Baseline is defined as the last non-missing measurement taken at the Screening and Baseline visits (prior to the first dose of study drug administered at the Day 1). A negative change from Baseline indicates a reduction in a component of NAFLD.
    End point type
    Secondary
    End point timeframe
    Baseline and Day 85
    End point values
    		 LYS006 LYS006 + LJN452
    Number of subjects analysed
    14
    11
    Units: Percentage of Liver Fat
        arithmetic mean (standard deviation)
    -3.74 ± 3.470
    -7.52 ± 5.846
    No statistical analyses for this end point

    Secondary: Change From Baseline in Total Body Weight

    		 Close Top of page
    End point title
    		 Change From Baseline in Total Body Weight
    End point description
    Body weight (to the nearest 0.1 kilogram [kg] was measured on a calibrated scale. The measurement was performed with the study participant in underwear and without shoes; or while wearing minimal indoor clothing. Baseline is defined as the last non-missing measurement taken at the Screening and Baseline visits (prior to the first dose of study drug administered at the Day 1). A negative change from Baseline indicates improvement in obesity.
    End point type
    Secondary
    End point timeframe
    Baseline and Days 15, 29, 43, 57, 85 and EOS (Day 113)
    End point values
    		 LYS006 LYS006 + LJN452
    Number of subjects analysed
    20
    17
    Units: kg
    arithmetic mean (standard deviation)
        Day 15
    -0.21 ± 1.412
    -1.09 ± 2.030
        Day 29
    -0.27 ± 1.513
    -1.17 ± 2.455
        Day 43
    -0.24 ± 1.664
    -1.94 ± 2.780
        Day 57
    -0.48 ± 1.548
    -2.97 ± 3.144
        Day 85
    -0.54 ± 2.334
    -3.33 ± 2.892
        EOS
    0.17 ± 2.753
    -2.56 ± 2.789
    No statistical analyses for this end point

    Secondary: Change from Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) at Day 85

    		 Close Top of page
    End point title
    		 Change from Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) at Day 85
    End point description
    HOMA-IR is a test that uses a simultaneous fasting blood glucose test and fasting insulin test to accurately estimate the degree of insulin resistance (IR) and β-cell function (the cells of the pancreas that produce insulin). HOMA-IR scores are classified as follows: Insulin sensitive is considered less than 1.0, Healthy is considered 0.5-1.4, Above 1.8 is early insulin resistance and Above 2.7 is considered significant insulin resistance HOMA-IR= [Fasting glucose (mmol/L) x (fasting insulin (pmol/L)/6)] / 22.5 Baseline is defined as the last non-missing measurement taken at the Screening and Baseline visits (prior to the first dose of study drug administered at the Day 1). A negative change from Baseline indicates improvement in insulin sensitivity.
    End point type
    Secondary
    End point timeframe
    Baseline and Day 85
    End point values
    		 LYS006 LYS006 + LJN452
    Number of subjects analysed
    14
    9
    Units: HOMA-IR score
        arithmetic mean (standard deviation)
    -3.74 ± 9.865
    1.67 ± 7.741
    No statistical analyses for this end point

    Secondary: Change from baseline in Fasting Glucose

    		 Close Top of page
    End point title
    		 Change from baseline in Fasting Glucose
    End point description
    Fasting Glucose was examined as a cardiometabolic risk parameter. Total fasting glucose was measured on blood samples under fasted conditions and analyzed at a central laboratory. Baseline is defined as the last non-missing measurement taken at the Screening and Baseline visits (prior to the first dose of study drug administered at the Day 1). A negative change from Baseline indicates improvement in glycemic control.
    End point type
    Secondary
    End point timeframe
    Baseline and Days 15, 29, 43, 57, 85 and EOS (Day 113)
    End point values
    		 LYS006 LYS006 + LJN452
    Number of subjects analysed
    20
    17
    Units: mmol / L
    arithmetic mean (standard deviation)
        Day 15
    0.26 ± 2.609
    0.26 ± 2.402
        Day 29
    -0.04 ± 3.541
    0.61 ± 2.362
        Day 43
    -0.53 ± 3.655
    1.05 ± 2.449
        Day 57
    -0.82 ± 3.176
    0.84 ± 1.960
        Day 85
    -1.74 ± 3.810
    0.41 ± 2.023
        EOS
    -1.01 ± 3.627
    -0.40 ± 1.563
    No statistical analyses for this end point

    Secondary: Change from Baseline in Fasting Insulin at Day 85

    		 Close Top of page
    End point title
    		 Change from Baseline in Fasting Insulin at Day 85
    End point description
    Fasting insulin was examined as a cardiometabolic risk parameter. Total fasting insulin was measured on blood samples under fasted conditions and analyzed at a central laboratory. Baseline is defined as the last non-missing measurement taken at the Screening and Baseline visits (prior to the first dose of study drug administered at the Day 1). A negative change from Baseline indicates improvement in insulin sensitivity.
    End point type
    Secondary
    End point timeframe
    Baseline and Day 85
    End point values
    		 LYS006 LYS006 + LJN452
    Number of subjects analysed
    14
    9
    Units: pmol / L
        arithmetic mean (standard deviation)
    -28.36 ± 139.23
    14.23 ± 63.875
    No statistical analyses for this end point

    Secondary: Change from baseline in Hemoglobin A1c (HbA1c)

    		 Close Top of page
    End point title
    		 Change from baseline in Hemoglobin A1c (HbA1c)
    End point description
    HbA1c was examined as a cardiometabolic risk parameter. HbA1c was measured on blood samples under fasted conditions and analyzed at a central laboratory. Baseline is defined as the last non-missing measurement taken at the Screening and Baseline visits (prior to the first dose of study drug administered at the Day 1). A negative change from Baseline indicates improvement in glycemic control.
    End point type
    Secondary
    End point timeframe
    Baseline and Days 15, 29, 43, 57, 85 and EOS (Day 113)
    End point values
    		 LYS006 LYS006 + LJN452
    Number of subjects analysed
    20
    17
    Units: Percentage
    arithmetic mean (standard deviation)
        Day 15
    0.10 ± 0.194
    0.08 ± 0.338
        Day 29
    0.03 ± 0.431
    0.21 ± 0.487
        Day 43
    -0.02 ± 0.544
    0.31 ± 0.884
        Day 57
    -0.11 ± 0.730
    0.36 ± 0.680
        Day 85
    -0.48 ± 0.834
    -0.03 ± 0.863
        EOS
    -0.59 ± 0.961
    0.34 ± 0.359
    No statistical analyses for this end point

    Secondary: Change from Baseline in Alanine aminotransferase (ALT)

    		 Close Top of page
    End point title
    		 Change from Baseline in Alanine aminotransferase (ALT)
    End point description
    Alanine aminotransferase (ALT) is an enzyme found primarily in the liver. ALT is increased with liver damage. In this study, the blood levels of ALT was used to detect liver inflammation. Baseline is defined as the mean of the last 2 non-missing measurements taken at the Screening and Baseline visits (prior to the first dose of study drug administered at the Day 1). A negative change from Baseline indicates a reduction in liver inflammation.
    End point type
    Secondary
    End point timeframe
    Baseline and days 15, 29, 43, 57, 85 and EOS (Day 113)
    End point values
    		 LYS006 LYS006 + LJN452
    Number of subjects analysed
    20
    17
    Units: U / L
    arithmetic mean (standard deviation)
        Day 15
    0.97 ± 18.255
    -19.75 ± 25.617
        Day 29
    -6.92 ± 22.166
    -9.63 ± 13.774
        Day 43
    -11.13 ± 21.624
    -8.68 ± 14.573
        Day 57
    -12.09 ± 25.401
    -17.04 ± 12.841
        Day 85
    -7.21 ± 34.702
    -11.14 ± 26.318
        EOS
    -14.50 ± 29.619
    -8.05 ± 14.570
    No statistical analyses for this end point

    Secondary: Change from baseline in High-sensitivity C-reactive Protein (hsCRP)

    		 Close Top of page
    End point title
    		 Change from baseline in High-sensitivity C-reactive Protein (hsCRP)
    End point description
    High-sensitivity C-reactive protein is a blood test marker for inflammation in the body. HsCRP was measured from a blood sample and analyzed at a central laboratory. Baseline is defined as the last non-missing measurement taken at the Screening and Baseline visits (prior to the first dose of study drug administered at the Day 1). A negative change from Baseline indicates a reduction in liver inflammation.
    End point type
    Secondary
    End point timeframe
    Baseline and Days 57, 85 and EOS (Day 113)
    End point values
    		 LYS006 LYS006 + LJN452
    Number of subjects analysed
    20
    17
    Units: mg / L
    arithmetic mean (standard deviation)
        Day 57
    -0.32 ± 2.192
    -7.78 ± 27.500
        Day 85
    -0.62 ± 2.180
    0.24 ± 1.692
        EOS
    0.19 ± 3.432
    0.05 ± 1.015
    No statistical analyses for this end point

    Secondary: LYS006 plasma concentration

    		 Close Top of page
    End point title
    		 LYS006 plasma concentration
    End point description
    LYS006 plasma concentrations were determined by a validated liquid chromatography with tandem mass spectrometry (LC-MS/MS) method. No methods for imputation of missing data were used.
    End point type
    Secondary
    End point timeframe
    pre-dose at Days 1, 29, 57 and 85 and post-dose (1, 2, 3 and 4 hours) at Days 29 and 57
    End point values
    		 LYS006 LYS006 + LJN452
    Number of subjects analysed
    20
    21
    Units: ng / mL
    arithmetic mean (standard deviation)
        Day 1 (0 h)
    0.162 ± 0.648
    0.00 ± 0.00
        Day 29 (0 h)
    78.9 ± 74.0
    53.5 ± 74.9
        Day 29 (1 h)
    174 ± 89.6
    169 ± 105
        Day 29 (2 h)
    224 ± 113
    189 ± 105
        Day 29 (3 h)
    188 ± 89.8
    145 ± 54.9
        Day 29 (4 h)
    149 ± 73.6
    110 ± 31.8
        Day 57 (0 h)
    58.0 ± 57.2
    24.0 ± 21.4
        Day 57 (1 h)
    200 ± 118
    123 ± 123
        Day 57 (2 h)
    222 ± 80.3
    198 ± 88.7
        Day 57 (3 h)
    188 ± 74.3
    156 ± 59.2
        Day 57 (4 h)
    140 ± 83.3
    126 ± 54.1
        Day 85 (0 h)
    15.2 ± 17.6
    10.2 ± 18.7
    No statistical analyses for this end point

    Secondary: Maximum observed plasma concentration (Cmax) of LYS006

    		 Close Top of page
    End point title
    		 Maximum observed plasma concentration (Cmax) of LYS006
    End point description
    LYS006 plasma concentrations were determined by a validated liquid chromatography with tandem mass spectrometry (LC-MS/MS) method. Cmax of LYS006 was determined with Phoenix WinNonlin (Version 8.0 or higher). No methods for imputation of missing data were used.
    End point type
    Secondary
    End point timeframe
    pre-dose and post-dose (1, 2, 3 and 4 hours) at Days 29 and 57
    End point values
    		 LYS006 LYS006 + LJN452
    Number of subjects analysed
    20
    21
    Units: ng / mL
    arithmetic mean (standard deviation)
        Day 29
    264 ± 87.7
    215 ± 98.8
        Day 57
    271 ± 71.1
    228 ± 88.1
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Treatment-emergent adverse events
    Adverse event reporting additional description
    Any sign or symptom that occurs during the study treatment plus the 28 days post treatment
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.1
    Reporting groups
    Reporting group title
    LYS006
    Reporting group description
    		 LYS006 20 mg was administered orally twice per day (b.i.d) for 12 weeks

    Reporting group title
    Total
    Reporting group description
    		 Total

    Reporting group title
    LYS006 + LJN452
    Reporting group description
    		 LYS006 20 mg was administered orally twice per day (b.i.d) in addition to LJN452 200ug administered orally once daily for 12 weeks

    Serious adverse events
    		 LYS006 Total LYS006 + LJN452
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 41 (0.00%)
    0 / 21 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 LYS006 Total LYS006 + LJN452
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 20 (45.00%)
    24 / 41 (58.54%)
    15 / 21 (71.43%)
    Investigations
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 20 (0.00%)
    2 / 41 (4.88%)
    2 / 21 (9.52%)
         occurrences all number
    0
    3
    3
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 20 (0.00%)
    2 / 41 (4.88%)
    2 / 21 (9.52%)
         occurrences all number
    0
    2
    2
    Blood alkaline phosphatase increased
         subjects affected / exposed
    0 / 20 (0.00%)
    3 / 41 (7.32%)
    3 / 21 (14.29%)
         occurrences all number
    0
    3
    3
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    2 / 20 (10.00%)
    2 / 41 (4.88%)
    0 / 21 (0.00%)
         occurrences all number
    2
    2
    0
    Headache
         subjects affected / exposed
    4 / 20 (20.00%)
    8 / 41 (19.51%)
    4 / 21 (19.05%)
         occurrences all number
    4
    8
    4
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    1 / 20 (5.00%)
    3 / 41 (7.32%)
    2 / 21 (9.52%)
         occurrences all number
    1
    3
    2
    Diarrhoea
         subjects affected / exposed
    3 / 20 (15.00%)
    3 / 41 (7.32%)
    0 / 21 (0.00%)
         occurrences all number
    3
    3
    0
    Nausea
         subjects affected / exposed
    3 / 20 (15.00%)
    5 / 41 (12.20%)
    2 / 21 (9.52%)
         occurrences all number
    3
    5
    2
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    0 / 20 (0.00%)
    13 / 41 (31.71%)
    13 / 21 (61.90%)
         occurrences all number
    0
    14
    14
    Rash
         subjects affected / exposed
    0 / 20 (0.00%)
    2 / 41 (4.88%)
    2 / 21 (9.52%)
         occurrences all number
    0
    2
    2
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 20 (0.00%)
    2 / 41 (4.88%)
    2 / 21 (9.52%)
         occurrences all number
    0
    2
    2
    Metabolism and nutrition disorders
    Hyperglycaemia
         subjects affected / exposed
    1 / 20 (5.00%)
    4 / 41 (9.76%)
    3 / 21 (14.29%)
         occurrences all number
    1
    4
    3

    More information

    		 Close Top of page

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

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    10 Dec 2021
    The study was terminated based upon an ongoing review of the study data, which showed a low likelihood of achieving required efficacy in either treatment arm.
    -

    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.

    European Medicines Agency © 1995-Mon Apr 29 16:53:33 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA