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    Clinical Trial Results:
    A randomized, multi-center, subject and investigator blinded, placebo controlled, parallel group study to assess the efficacy, safety and tolerability of LYS006 in patients with mild to moderate ulcerative colitis

    Summary
    EudraCT number
    		 2019-003113-34
    Trial protocol
    		 DE   HU   NO   SK  
    Global end of trial date
    07 Nov 2022

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Nov 2023
    First version publication date
    04 Nov 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CLYS006X2202
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04074590
    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
    07 Nov 2022
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    07 Nov 2022
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of the trial was to assess the induction of clinical remission by LYS006 in patients with mild to moderate ulcerative colitis compared to placebo.
    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
    03 Feb 2020
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Bulgaria: 1
    Country: Number of subjects enrolled
    Czechia: 6
    Country: Number of subjects enrolled
    Germany: 2
    Country: Number of subjects enrolled
    Poland: 7
    Country: Number of subjects enrolled
    Russian Federation: 4
    Country: Number of subjects enrolled
    Slovakia: 3
    Worldwide total number of subjects
    23
    EEA total number of subjects
    19
    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)
    23
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants took part in 9 investigative sites in 6 countries.

    Pre-assignment
    Screening details
    		 After signing informed consent, screening evaluations took place from Day -28 to Day -1. During that period all assessments were performed to evaluate eligibility. Eligible patients returned for the Baseline visit on Day 1. Eligibility was confirmed prior to randomization and required baseline assessments were completed prior to dosing on Day 1.

    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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 LYS006 20mg
    Arm description
    		 LYS006 20mg oral dose, twice daily
    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 oral twice daily

    Arm title
    		 Placebo
    Arm description
    		 Placebo oral dose, twice daily
    Arm type
    		 Placebo

    Investigational medicinal product name
    LYS006
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo oral twice daily

    Number of subjects in period 1
    		LYS006 20mg Placebo
    Started
    16
    7
    Completed
    12
    7
    Not completed
    4
    0
         Physician decision
    1
    -
         Consent withdrawn by subject
    2
    -
         Adverse Event
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    LYS006 20mg
    Reporting group description
    		 LYS006 20mg oral dose, twice daily

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo oral dose, twice daily

    Reporting group values
    		 LYS006 20mg Placebo Total
    Number of subjects
    		 16 7 23
    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)
    		 16 7 23
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 38.3 ( 12.20 ) 45.7 ( 12.37 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 8 5 13
        Male
    		 8 2 10
    Race/Ethnicity, Customized
    Units: Subjects
        White
    		 16 7 23

    End points

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    End points reporting groups
    Reporting group title
    LYS006 20mg
    Reporting group description
    		 LYS006 20mg oral dose, twice daily

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo oral dose, twice daily

    Primary: Clinical remission rate at the End of the study treatment

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    End point title
    		 Clinical remission rate at the End of the study treatment
    End point description
    The Mayo score is an instrument designed to measure activity of ulcerative colitis. The Mayo score comprises of four sub scores: stool frequency, rectal bleeding, endoscopic findings and the Physician’s Global Assessment (PhGA). Each sub score is graded from 0 to 3 with higher scores indicating more severe disease. The full Mayo score is the sum of four sub scores, ranging from 0 to 12. Clinical remission is defined as a full Mayo score of 2 points or lower, with no individual subscore exceeding one point. The clinical remission rate is expressed as percentage of participants. The binary endpoint of clinical remission rate (Yes/No) at the EoT visit was modelled with binomial distribution and analyzed via the Bayesian approach with baseline total Mayo score and treatment group as explanatory variables, to compare the remission rates between the LYS006 and placebo groups.
    End point type
    Primary
    End point timeframe
    Week 8
    End point values
    		 LYS006 20mg Placebo
    Number of subjects analysed
    14
    7
    Units: Percentage of participants
        number (confidence interval 90%)
    8.95 (0.87 to 23.31)
    12.24 (5.67 to 24.12)
    Statistical analysis title
    		 Clinical remission rate
    Comparison groups
    LYS006 20mg v Placebo
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    		 [1]
    P-value
    		 = 0.037 [2]
    Method
    		 Bayesian analysis
    Parameter type
    		 Posterior estimate treatment difference
    Point estimate
    -3.29
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -18.02
         upper limit
    		 13.23
    Notes
    [1] - A Bayesian analysis of clinical remission rate (based on total Mayo score) with binomial distribution, was modelled with baseline total Mayo score and treatment group as explanatory variables, to compare the remission rates between LYS006 and placebo groups.
    [2] - Posterior probability that clinical remission rate >15% over placebo: Prob (diff>0.15)
    Statistical analysis title
    		 Clinical remission rate
    Comparison groups
    Placebo v LYS006 20mg
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    		 [3]
    P-value
    		 = 0.314 [4]
    Method
    		 Bayesian analysis
    Parameter type
    		 Posterior estimate treatment difference
    Point estimate
    -3.29
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -18.02
         upper limit
    		 13.23
    Notes
    [3] - A Bayesian analysis of clinical remission rate (based on total Mayo score) with binomial distribution, was modelled with baseline total Mayo score and treatment group as explanatory variables, to compare the remission rates between LYS006 and placebo groups.
    [4] - Posterior probability that clinical remission rate is better than placebo: Prob (diff>0)

    Secondary: Number of participants with treatment emergent adverse events (AEs) and serious adverse events (SAEs)

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    End point title
    		 Number of participants with treatment emergent adverse events (AEs) and serious adverse events (SAEs)
    End point description
    Number of participants with treatment emergent AEs, AEs led to study treatment discontinuation, SAEs and SAEs led to study treatment discontinuation.
    End point type
    Secondary
    End point timeframe
    AEs were reported from first dose until end of study treatment plus 30 days post treatment, up to a max. duration of approx. 115 days for participants treated for 12 weeks and up to a max. duration of approx. 87 days for participants treated for 8 weeks.
    End point values
    		 LYS006 20mg Placebo
    Number of subjects analysed
    16
    7
    Units: participants
        At least one AE
    7
    5
        At least one SAE
    0
    0
        AE leading to discontinuation
    2
    0
        SAE leading to discontinuation
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    AEs were reported from first dose until end of study treatment plus 30 days post treatment, up to a max. duration of approx. 115 days for participants treated for 12 weeks and up to a max. duration of approx. 87 days for participants treated for 8 weeks.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.1
    Reporting groups
    Reporting group title
    LYS006 20 mg BID
    Reporting group description
    		 LYS006 20 mg BID

    Reporting group title
    Total
    Reporting group description
    		 Total

    Reporting group title
    Placebo BID
    Reporting group description
    		 Placebo BID

    Serious adverse events
    		 LYS006 20 mg BID Total Placebo BID
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 23 (0.00%)
    0 / 7 (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: 0%
    Non-serious adverse events
    		 LYS006 20 mg BID Total Placebo BID
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 16 (43.75%)
    12 / 23 (52.17%)
    5 / 7 (71.43%)
    Investigations
    Urine protein/creatinine ratio increased
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 23 (4.35%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    1
    Faecal calprotectin increased
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 23 (4.35%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    1
    Cardiac disorders
    Atrioventricular block first degree
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 23 (4.35%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 23 (4.35%)
    0 / 7 (0.00%)
         occurrences all number
    1
    1
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 23 (4.35%)
    0 / 7 (0.00%)
         occurrences all number
    1
    1
    0
    Gastrointestinal disorders
    Colitis ulcerative
         subjects affected / exposed
    3 / 16 (18.75%)
    4 / 23 (17.39%)
    1 / 7 (14.29%)
         occurrences all number
    3
    4
    1
    Vomiting
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 23 (4.35%)
    0 / 7 (0.00%)
         occurrences all number
    1
    1
    0
    Hepatobiliary disorders
    Drug-induced liver injury
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 23 (4.35%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    1
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 23 (4.35%)
    0 / 7 (0.00%)
         occurrences all number
    1
    1
    0
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 23 (4.35%)
    0 / 7 (0.00%)
         occurrences all number
    1
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 23 (4.35%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    17 Jun 2020
    The purpose of this amendment is to address requests from Health Authorities, the Norwegian Health Authority (NOMA), the German Health Authority (BfArM), and the Czech Health Authority (SUKL). In addition, the following changes have been made to reduce patient burden and further align the study with the EMA Guideline (2018), ECCO guidelines (Harbord et al 2017) and ACG Clinical guideline (Rubin et al 2019). 1. Reduction of the treatment period from 12 to 8 weeks; 2. Replacement of colonoscopies with sigmoidoscopies; 3. Modifications to eligibility criteria; 4. Changes to biopsy sample collection and analysis.
    23 Mar 2021
    The protocol has been amended to adapt eligibility criteria and reduce patient burden that together, are anticipated to support recruitment activities. The following changes have been made: 1. Modifications to eligibility criteria; 2.Enriched PK profile at End of Treatment visit (EoT) is now optional, to reduce the patient burden and improve recruitment; 3. Reduction in the number of biopsies;4. Removed need to measure Body temperature orally; 5. Additional blood samples will be collected at the EoS visit; 6. Repeat of safety assessments at screening.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

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