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    Clinical Trial Results:
    A randomized, double blind, placebo-controlled, multicenter, Phase III study investigating the efficacy and safety of ruxolitinib in early myelofibrosis patients with high molecular risk mutations (ReTHINK) Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www. novctrd.com/CtrdWeb/home.novfor complete trial results

    Summary
    EudraCT number
    		 2014-004928-21
    Trial protocol
    		 ES   SE   GB   AT   BE   HU   GR   FR   PT   FI   DK   PL   IT  
    Global end of trial date
    23 Oct 2017

    Results information
    Results version number
    		 v1(current)
    This version publication date
    08 Nov 2018
    First version publication date
    08 Nov 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CINC424A2353
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02598297
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma, AG
    Sponsor organisation address
    CH-4002, 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
    23 Oct 2017
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Oct 2017
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the effect of ruxolitinib in delaying progression of MF from early disease to advanced disease.
    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 2016
    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: 1
    Country: Number of subjects enrolled
    Belgium: 4
    Country: Number of subjects enrolled
    Canada: 1
    Country: Number of subjects enrolled
    Denmark: 1
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    Germany: 4
    Country: Number of subjects enrolled
    United Kingdom: 1
    Country: Number of subjects enrolled
    Greece: 5
    Country: Number of subjects enrolled
    Hong Kong: 1
    Country: Number of subjects enrolled
    Israel: 6
    Country: Number of subjects enrolled
    Italy: 4
    Country: Number of subjects enrolled
    Japan: 4
    Country: Number of subjects enrolled
    Poland: 3
    Country: Number of subjects enrolled
    Russian Federation: 2
    Country: Number of subjects enrolled
    Spain: 3
    Country: Number of subjects enrolled
    Sweden: 3
    Country: Number of subjects enrolled
    Turkey: 4
    Worldwide total number of subjects
    49
    EEA total number of subjects
    31
    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)
    24
    From 65 to 84 years
    25
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Approximately 320 male or female adults (age 18 or over) with a confirmed diagnosis of MF were planned to be enrolled. The target population was not met due to early study termination. A total of 49 subjects were enrolled in the study, 25 in the ruxolitinib arm and 24 in the placebo arm.

    Pre-assignment
    Screening details
    		 Approximately 320 male or female adults (age 18 or over) with a confirmed diagnosis of MF were planned to be enrolled. The target population was not met due to early study termination. A total of 49 subjects were enrolled in the study, 25 in the ruxolitinib arm and 24 in the placebo arm.

    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
    		 Investigator, Carer, Assessor, Subject

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Ruxolitinib (INC424)
    Arm description
    		 Two tablets of ruxolitinib 5 mg were administered orally twice per day
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ruxolitinib
    Investigational medicinal product code
    INC424
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Treatment period 1: Two tablets of ruxolitinib 5 mg were administered orally twice per day. Treatment period 2: tablets of either 5, 15, 20 mg twice orally per day based on platelet counts

    Arm title
    		 Ruxolitinib Placebo
    Arm description
    		 Two tablets of 5mg placebo were administered orally twice per day
    Arm type
    		 Placebo

    Investigational medicinal product name
    Ruxolitinib Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Treatment period 1: Two tablets of placebo 5 mg were administered orally twice per day. Treatment period 2: tablets of either 5, 15, 20 mg twice orally per day based on platelet counts

    Number of subjects in period 1
    		Ruxolitinib (INC424) Ruxolitinib Placebo
    Started
    25
    24
    Not treted withstudy drug
    1
    0
    Subjects followed for survival
    1
    0
    Completed
    0
    0
    Not completed
    25
    24
         Physician decision
    1
    -
         Study terminated by Sponsor
    21
    23
         Adverse event, non-fatal
    1
    1
         Followed for survival
    1
    -
         Subject/guardian decision
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Ruxolitinib (INC424)
    Reporting group description
    		 Two tablets of ruxolitinib 5 mg were administered orally twice per day

    Reporting group title
    Ruxolitinib Placebo
    Reporting group description
    		 Two tablets of 5mg placebo were administered orally twice per day

    Reporting group values
    		 Ruxolitinib (INC424) Ruxolitinib Placebo Total
    Number of subjects
    		 25 24 49
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 14 10 24
        From 65-84 years
    		 11 14 25
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 59.0 ( 14.23 ) 67.4 ( 7.72 ) -
    Sex: Female, Male
    Units: Subjects
        Female
    		 12 7 19
        Male
    		 13 17 30
    Race/Ethnicity, Customized
    Units: Subjects
        Caucasian|
    		 19 23 42
        Asian|
    		 4 1 5
        Other|
    		 1 0 1
        Missing|
    		 1 0 1

    End points

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    End points reporting groups
    Reporting group title
    Ruxolitinib (INC424)
    Reporting group description
    		 Two tablets of ruxolitinib 5 mg were administered orally twice per day

    Reporting group title
    Ruxolitinib Placebo
    Reporting group description
    		 Two tablets of 5mg placebo were administered orally twice per day

    Primary: Progression free survival (PFS-1)

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    End point title
    		 Progression free survival (PFS-1) [1]
    End point description
    Progression free survival (PFS-1) from date of randomization until the occurrence of any of the criteria for disease progression: ● Progressive splenomegaly ● Circulating peripheral blast counts > 10% ● Leukemic transformation ● Hb < 10g/dl with absolute decrease of at least 3 g/dl from baseline ● White blood cell (WBC) counts > 25 x 103/ μL ● MF-7 score ≥ 30 ● Death from any cause
    End point type
    Primary
    End point timeframe
    From randomization till disease progression (estimated to be assessed up 48 months)
    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 was performed for this endpoint as the study terminated early.
    End point values
    		 Ruxolitinib (INC424) Ruxolitinib Placebo
    Number of subjects analysed
    25
    24
    Units: Participants
    0
    0
    No statistical analyses for this end point

    Secondary: Time to primary progression (TTP)

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    End point title
    		 Time to primary progression (TTP)
    End point description
    TTP is defined as time from randomization until disease progression as defined for PFS-1 excluding death as an event.
    End point type
    Secondary
    End point timeframe
    From randomization till progression (estimated to be assessed up to 48 months)
    End point values
    		 Ruxolitinib (INC424) Ruxolitinib Placebo
    Number of subjects analysed
    25
    24
    Units: Months
    0
    0
    No statistical analyses for this end point

    Secondary: Percentage Change in spleen volume from baseline

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    End point title
    		 Percentage Change in spleen volume from baseline
    End point description
    Change in spleen volume (by MRI/CT) from baseline
    End point type
    Secondary
    End point timeframe
    From baseline and assessed on 12 week intervals until end of treatment (EOT)
    End point values
    		 Ruxolitinib (INC424) Ruxolitinib Placebo
    Number of subjects analysed
    25
    24
    Units: Percentage change from baseline
    arithmetic mean (standard deviation)
        Week 12 (N = 19, 19)|
    -10.8 ( 24.45 )
    9.1 ( 12.34 )
        Week 24 (N = 13, 8)|
    -17.8 ( 18.83 )
    17.6 ( 17.78 )
        Week 36 (N = 7, 2)|
    -18.4 ( 31.62 )
    32.5 ( 18.81 )
        Week 48 ( N = 2, 0)|
    -23.5 ( 10.57 )
    999 ( 999 )
        End of Treatment (EOT) (N = 10, 9)|
    -11.6 ( 8.08 )
    18.1 ( 18.58 )
    No statistical analyses for this end point

    Secondary: Percentage change in symptoms from baseline using MF-7

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    End point title
    		 Percentage change in symptoms from baseline using MF-7
    End point description
    Percentage change from Baseline in MF-7 total symptom score and 7 individual symptoms at each visit was summarized with descriptive statistics. For this scale, symptoms range from 0 to 10 for the severity experienced within the past 24 hours, with 0 being for absence of symptoms and 10 for worst imaginable symptoms.
    End point type
    Secondary
    End point timeframe
    From Baseline and assessed every 4 weeks until end of treatment
    End point values
    		 Ruxolitinib (INC424) Ruxolitinib Placebo
    Number of subjects analysed
    25 [2]
    24 [3]
    Units: Percentage change in scores
    arithmetic mean (standard deviation)
        TSD change from baseline @ W12|
    -0.3 ( 4.45 )
    0.5 ( 6.08 )
        TSD change from baseline @ W24|
    0.5 ( 3.71 )
    -0.8 ( 6.44 )
        TSD change from baseline @ W48|
    -0.3 ( 3.06 )
    999 ( 999 )
        TSD change from BL @ EOT|
    1.1 ( 4.40 )
    3.3 ( 9.53 )
        Tiredness: Baseline (BL)|
    1.8 ( 1.45 )
    1.5 ( 1.53 )
        Tiredness: change from baseline @ W12|
    0.1 ( 1.47 )
    0.4 ( 1.27 )
        Tiredness: change from baseline @ W24|
    -0.1 ( 0.76 )
    1.1 ( 2.52 )
        Tiredness: change from baseline @ W48|
    -0.3 ( 0.58 )
    999 ( 999 )
        Tiredness change from BL @ EOT|
    0.8 ( 1.32 )
    1.1 ( 2.28 )
        Filling up quickly when you eat (FUQWYE):BL|
    1.0 ( 1.41 )
    0.8 ( 1.27 )
        FUQWYE: change from BL @W12|
    0.1 ( 1.49 )
    -0.1 ( 2.09 )
        FUQWYE: change from BL @W24|
    -0.2 ( 0.99 )
    0.1 ( 1.17 )
        FUQWYE: change from BL @W48|
    0.7 ( 1.15 )
    999 ( 999 )
        FUQWYE: change from BL @ EOT|
    0.2 ( 1.08 )
    0.3 ( 2.29 )
        Abdominal discomfort (AD): BL|
    0.7 ( 1.43 )
    0.6 ( 1.10 )
        Abdominal discomfort change from BL @W12|
    -0.1 ( 1.66 )
    -0.1 ( 0.85 )
        Abdominal discomfort change from BL @W24|
    0.0 ( 1.15 )
    -0.3 ( 0.71 )
        Abdominal discomfort change from BL @W48|
    0.0 ( 0.00 )
    999 ( 999 )
        AD: change from BL @ EOT|
    0.1 ( 0.92 )
    0.3 ( 1.40 )
        Night sweat (NS): Baseline (BL)|
    0.7 ( 1.07 )
    1.1 ( 1.45 )
        Night sweat change from BL @W12|
    -0.1 ( 0.71 )
    -0.3 ( 1.29 )
        Night sweat change from BL @W24|
    0.5 ( 1.45 )
    -0.7 ( 1.41 )
        Night sweat change from BL @W48|
    -0.3 ( 0.58 )
    999 ( 999 )
        Night Sweat: change from BL @ EOT|
    0.0 ( 0.85 )
    0.3 ( 2.12 )
        Itching (Pruritus):BL|
    0.9 ( 1.42 )
    0.6 ( 0.93 )
        Itching (Pruritus): change from BL @W12|
    -0.3 ( 0.67 )
    0.1 ( 1.45 )
        Itching (Pruritus): change from BL @W24|
    0.4 ( 0.77 )
    -0.3 ( 1.22 )
        Itching (Pruritus): change from BL @W48|
    -1.0 ( 1.73 )
    999 ( 999 )
        Itching: change from BL @ EOT|
    0.1 ( 1.03 )
    0.4 ( 1.75 )
        Bone Pain: BL|
    0.9 ( 1.35 )
    0.9 ( 1.33 )
        Bone Pain: change from BL @W12|
    -0.4 ( 1.01 )
    0.3 ( 1.56 )
        Bone Pain: change from BL @W24|
    -0.2 ( 1.17 )
    -0.8 ( 1.79 )
        Bone Pain: change from BL @W48|
    0.7 ( 1.15 )
    999 ( 999 )
        Bone Pain: change from BL @ EOT|
    0.1 ( 0.96 )
    0.6 ( 1.50 )
        Pain under ribs on left side (PUROLS): BL|
    0.7 ( 1.18 )
    0.4 ( 0.82 )
        PUROLS: change from BL @W12|
    0.4 ( 1.01 )
    0.1 ( 0.76 )
        PUROLS: change from BL @W24|
    0.0 ( 0.41 )
    0.1 ( 1.17 )
        PUROLS: change from BL @W48|
    0.0 ( 0.00 )
    999 ( 999 )
        PUROLS: change from BL @ EOT|
    -0.2 ( 0.68 )
    0.3 ( 1.00 )
    Notes
    [2] - Baseline n = 25 Week 12 (n= 19) Week 24 (n = 13) Week 48 (n = 3) EOT (n = 15)
    [3] - Baseline n = 24 Week 12 (n= 20) Week 24 (n = 9) Week 48 (n = 0) EOT (n = 16)
    No statistical analyses for this end point

    Secondary: Percentage change in symptoms from baseline using EQ-5D

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    End point title
    		 Percentage change in symptoms from baseline using EQ-5D
    End point description
    EQ-5D profiles were tabulated at baseline and each scheduled assessment. EQ visual analogue scale values were summarized descriptively by arm for each scheduled visit. The 5 scores for mobility, self-care, usual activities, pain/discomfort and anxiety/depression are all self-explanatory (eg “I have no problems walking” to “I am unable to walk”), except for the following overall health check, where 100 is the best of health, and 0 is the worst health, Only the categories with non-zero counts are presented with these results.
    End point type
    Secondary
    End point timeframe
    From Baseline and assessed every 4 weeks until end of treatment
    End point values
    		 Ruxolitinib (INC424) Ruxolitinib Placebo
    Number of subjects analysed
    25
    24
    Units: Participants
        Mobility- Baseline: No problem|
    17
    18
        Mobility- Baseline: Slight problems|
    4
    5
        Mobility- Baseline: Moderate problems|
    1
    1
        Mobility- Baseline: Extreme problems|
    1
    0
        Mobility - Week 4:No prob.|
    17
    14
        Mobility - Week 4: Slight problems|
    4
    7
        Mobility - Week 4: Moderate problems|
    1
    2
        Mobility - Week 8: No problem|
    17
    15
        Mobility - Week 8: Slight problems|
    3
    6
        Mobility - Week 8: Moderate problems|
    0
    1
        Mobility - Week 12: No problem|
    15
    12
        Mobility - Week 12: Slight problems|
    4
    6
        Mobility - Week 12: Moderate problems|
    0
    2
        Mobility - Week 16: No problem|
    12
    10
        Mobility - Week 16: Slight problems|
    2
    7
        Mobility - Week 16: Moderate problems|
    1
    0
        Mobility - Week 20: No problem|
    12
    5
        Mobility - Week 20: Slight problems|
    2
    6
        Mobility - Week 24: No problem|
    12
    5
        Mobility - Week 24: Slight problems|
    1
    3
        Mobility - Week 24: Moderate problems|
    0
    1
        Mobility - Week 32: No problem|
    8
    1
        Mobility - Week 32: Slight problems|
    0
    1
        Mobility - Week 32: Moderate problems|
    1
    0
        Mobility - Week 40: No problem|
    5
    0
        Mobility - Week 40: Slight problems|
    1
    0
        Mobility - Week 48: No problem|
    2
    0
        Mobility - Week 48: Slight problems|
    1
    0
        Mobility - EOT: No problem|
    11
    12
        Mobility - EOT: Slight problems|
    4
    3
        Mobility - EOT: Moderate problems|
    0
    1
        Mobility- 30 day safety FU: No problem|
    2
    3
        Mobility- 30 day safety FU: Slight problems|
    1
    0
        Self-care - Baseline: No problem|
    23
    22
        Self-care - Baseline: Slight problems|
    0
    2
        Self-care - Week 4: No problem|
    21
    20
        Self-care - Week 4: Slight problems|
    0
    3
        Self-care - Week 4: Moderate problems|
    1
    0
        Self-care - Week 8: No problem|
    19
    21
        Self-care - Week 8: Slight problems|
    1
    1
        Self-care - Week 12: No problem|
    17
    19
        Self-care - Week 12: Slight problems|
    1
    1
        Self-care - Week 12: Moderate problems|
    1
    0
        Self-care - Week 16: No problem|
    15
    16
        Self-care - Week 16: Slight problems|
    0
    1
        Self-care - Week 20: No problem|
    14
    11
        Self-care - Week 24: No problem|
    12
    9
        Self-care - Week 24: Slight problems|
    1
    0
        Self-care - Week 32: No problem|
    8
    2
        Self-care - Week 32: Slight problems|
    1
    0
        Self-care - Week 40: No problem|
    5
    0
        Self-care - Week 40: Slight problems|
    1
    0
        Self-care - Week 48: No problem|
    3
    0
        Self-care - EOT: No problem|
    14
    15
        Self-care - EOT: Slight problems|
    1
    1
        Self-care - 30 day Safety FU: No problem|
    3
    3
        Usual activities -Baseline: No problem|
    18
    21
        Usual activities -Baseline: Slight problems|
    4
    3
        Usual activities - Baseline: Moderate problems|
    1
    0
        Usual activities - Week 4: No problem|
    20
    18
        Usual activities - Week 4: Slight problems|
    1
    4
        Usual activities - Week 4: Moderate problems|
    1
    0
        Usual activities - Week 4: Severe problems|
    0
    1
        Usual activities - Week 8: No problem|
    20
    18
        Usual activities - Week 8: Slight problems|
    0
    3
        Usual activities - Week 8: Moderate problems|
    0
    1
        Usual activities - Week 12: No problem|
    16
    16
        Usual activities - Week 12: Slight problems|
    3
    3
        Usual activities - Week 12: Moderate problems|
    0
    1
        Usual activities - Week 16: No problem|
    13
    14
        Usual activities - Week 16: Slight problems|
    2
    3
        Usual activities - Week 20: No problem|
    13
    9
        Usual activities - Week 20: Slight problems|
    1
    2
        Usual activities - Week 24: No problem|
    12
    9
        Usual activities - Week 24: Slight problems|
    1
    0
        Usual activities - Week 32: No problem|
    8
    2
        Usual activities - Week 32: Slight problems|
    1
    0
        Usual activities - Week 40: No problem|
    5
    0
        Usual activities - Week 40: Slight problems|
    1
    0
        Usual activities - Week 48: No problem|
    3
    0
        Usual activities - EOT: No problem|
    12
    13
        Usual activities -EOT: Slight problems|
    3
    3
        Usual activities - 30 day Safety FU: No problem|
    2
    3
        Usual activities - 30 say Safety FU: Slight probs|
    1
    0
        Pain/Discomfort - Baseline: No problem|
    13
    11
        Pain/Discomfort - Baseline: Slight problems|
    8
    12
        Pain/Discomfort - Baseline: Moderate problems|
    2
    1
        Pain/Discomfort - Week 4: No problem|
    11
    15
        Pain/Discomfort - Week 4: Slight problems|
    10
    6
        Pain/Discomfort - Week 4: Moderate problems|
    1
    2
        Pain/Discomfort - Week 8: No problem|
    14
    13
        Pain/Discomfort - Week 8: Slight problems|
    5
    7
        Pain/Discomfort - Week 8: Moderate problems|
    1
    2
        Pain/Discomfort - Week 12: No problem|
    12
    10
        Pain/Discomfort - Week 12: Slight problems|
    7
    6
        Pain/Discomfort - Week 12: Moderate problems|
    0
    4
        Pain/Discomfort - Week 16: No problem|
    9
    11
        Pain/Discomfort - WK16: Slight problems|
    6
    3
        Pain/Discomfort - WK16: Moderate problems|
    0
    3
        Pain/Discomfort - WK20: No problem|
    8
    8
        Pain/Discomfort - WK20: Slight problems|
    6
    3
        Pain/Discomfort - WK24: No problem|
    9
    7
        Pain/Discomfort - WK24: Slight problems|
    4
    2
        Pain/Discomfort - WK32: No problem|
    7
    2
        Pain/Discomfort - WK32: Slight problems|
    2
    0
        Pain/Discomfort - WK40: No problem|
    4
    0
        Pain/Discomfort - WK40: Slight problems|
    2
    0
        Pain/Discomfort - WK48: No problem|
    3
    0
        Pain/Discomfort - EOT: No problem|
    8
    10
        Pain/Discomfort - EOT: Slight problems|
    7
    3
        Pain/Discomfort - EOT: Moderate problems|
    0
    3
        Pain/Discomfort - 30 day Safety FU: No problem|
    1
    2
        Pain/Discomfort - 30 day safety FU: Slight probs|
    2
    0
        Pain/Discomfort - 30 day Safety FU: Moderate probs
    0
    1
        Anxiety/Depression - Baseline: No problem
    14
    18
        Anxiety/Depression - Baseline: Slight problems
    6
    3
        Anxiety/Depression - Baseline: Moderate problems
    3
    3
        Anxiety/Depression - WK4: No problem
    13
    15
        Anxiety/Depression - WK4: Slight problems|
    7
    6
        Anxiety/Depression - WK4: Moderate problems|
    2
    2
        Anxiety/Depression - WK8: No problem|
    11
    16
        Anxiety/Depression - WK8: Slight problems|
    8
    2
        Anxiety/Depression - WK8: Moderate problems|
    1
    4
        Anxiety/Depression - WK12: No problem|
    8
    13
        Anxiety/Depression - WK12: Slight problems|
    10
    3
        Anxiety/Depression -WK12: Moderate problems|
    1
    4
        Anxiety/Depression - WK16: No problem|
    10
    10
        Anxiety/Depression - WK16: Slight problems|
    5
    5
        Anxiety/Depression - WK16: Moderate problems|
    0
    2
        Anxiety/Depression -WK20: No problem|
    10
    8
        Anxiety/Depression - WK20: Slight problems|
    4
    3
        Anxiety/Depression - WK24: No problem|
    11
    7
        Anxiety/Depression - WK24: Slight problems|
    2
    2
        Anxiety/Depression - WK32: No problem|
    7
    2
        Anxiety/Depression - WK32: Slight problems|
    2
    0
        Anxiety/Depression - WK40: No problem|
    5
    0
        Anxiety/Depression - WK40: Slight problems
    1
    0
        Anxiety/Depression - WK48: No problem
    3
    0
        Anxiety/Depression - EOT: No problem
    11
    12
        Anxiety/Depression - EOT: Slight probs
    4
    3
        Anxiety/Depression - EOT: Moderate probs
    0
    1
        Anxiety/Depression - 30 day Safety FU: No probs
    2
    2
        Anxiety/Depression-30 day Safety FU: Slight probs
    1
    0
        Anxiety/Depression-30 day Safety FU: Severe probs|
    0
    1
    No statistical analyses for this end point

    Secondary: Overall survival

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    End point title
    		 Overall survival
    End point description
    To evaluate the effect of ruxolitinib on overall survival
    End point type
    Secondary
    End point timeframe
    Time from randomization to date of death due to any cause (estimated to be assessed up to 48 months).
    End point values
    		 Ruxolitinib (INC424) Ruxolitinib Placebo
    Number of subjects analysed
    25
    24
    Units: Participants
    0
    0
    No statistical analyses for this end point

    Secondary: Plasma ruxolitinib concentrations

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    End point title
    		 Plasma ruxolitinib concentrations
    End point description
    Characterize pharmacokinetics (PK)by utilizing a population PK approach.
    End point type
    Secondary
    End point timeframe
    Week 12, Wk 48
    End point values
    		 Ruxolitinib (INC424) Ruxolitinib Placebo
    Number of subjects analysed
    25
    24
    Units: Participants
    0
    0
    No statistical analyses for this end point

    Secondary: Progression free survival (PFS-2)

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    End point title
    		 Progression free survival (PFS-2)
    End point description
    PFS-2 assessed by 25% increase over new baseline of PFS-1 in any of the following: ● Progressive splenomegaly ● 25 % increase in MF-7 score with absolute score ≥ 30
    End point type
    Secondary
    End point timeframe
    From date of randomization until second disease progression or death, whichever comes first (estimated to be assessed up to 72 months)
    End point values
    		 Ruxolitinib (INC424) Ruxolitinib Placebo
    Number of subjects analysed
    25
    24
    Units: Participants
    0
    0
    No statistical analyses for this end point

    Secondary: Quality-adjusted life years from baseline

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    End point title
    		 Quality-adjusted life years from baseline
    End point description
    EQ-5D-5L (EuroQol-5D-5L, is a standardized instrument for measuring health outcomes, is consists of a descriptive system and a visual analogue scale - scores can be summarized into a single index score that provides a simple measure of health for clinical and economic appraisal ) The EQ-5D-5L health states will be converted into index values (utilities) from which the QALY (Quality - adjusted life years) will be calculated. QALY will be summarized descriptively by treatment arm.
    End point type
    Secondary
    End point timeframe
    Change from Baseline compared with schduled study visits at the following intervals every 4 weeks up to week 24, every 8 weeks up to Week 48, every 12 weeks past Wk 48 until End of treatment and 30 day follow up visit
    End point values
    		 Ruxolitinib (INC424) Ruxolitinib Placebo
    Number of subjects analysed
    25
    24
    Units: Participants
    0
    0
    No statistical analyses for this end point

    Secondary: Time to first progressive splenomegaly (TTPS)

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    End point title
    		 Time to first progressive splenomegaly (TTPS)
    End point description
    Time to first progressive splenomegaly as determined by spleen volume (by Magnetic Resonance Imaging (MRI)/Computed Tomography (CT).
    End point type
    Secondary
    End point timeframe
    From randomization until earliest time to progressive splenomegaly (estimated to be assesed up to 48 months)
    End point values
    		 Ruxolitinib (INC424) Ruxolitinib Placebo
    Number of subjects analysed
    25
    24
    Units: Months
    0
    0
    No statistical analyses for this end point

    Secondary: Time to first symptomatic progression (TTSP)

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    End point title
    		 Time to first symptomatic progression (TTSP)
    End point description
    Time to first symptomatic progression as determined by Myelofibrosis 7 Item Symptom Scale (MF-7)
    End point type
    Secondary
    End point timeframe
    From randomization until symptomatic progression (MF-7)(estimated to be assesed up to 48 months)
    End point values
    		 Ruxolitinib (INC424) Ruxolitinib Placebo
    Number of subjects analysed
    25
    24
    Units: Months
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse Events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All Adverse Events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit, up to about 48 months.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20.1
    Reporting groups
    Reporting group title
    PLACEBO
    Reporting group description
    		 PLACEBO

    Reporting group title
    INC424
    Reporting group description
    		 INC424

    Serious adverse events
    		 PLACEBO INC424
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 24 (4.17%)
    2 / 24 (8.33%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Gastric cancer
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Tendon rupture
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pneumonitis
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 24 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 PLACEBO INC424
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    11 / 24 (45.83%)
    17 / 24 (70.83%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    3 / 24 (12.50%)
    3 / 24 (12.50%)
         occurrences all number
    3
    0
    Blood and lymphatic system disorders
    Thrombocytopenia
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 24 (0.00%)
         occurrences all number
    2
    0
    Anaemia
         subjects affected / exposed
    6 / 24 (25.00%)
    7 / 24 (29.17%)
         occurrences all number
    6
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    4 / 24 (16.67%)
    3 / 24 (12.50%)
         occurrences all number
    4
    0
    Pyrexia
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 24 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    0
    0
    Skin and subcutaneous tissue disorders
    Night sweats
         subjects affected / exposed
    2 / 24 (8.33%)
    1 / 24 (4.17%)
         occurrences all number
    2
    0
    Pruritus
         subjects affected / exposed
    3 / 24 (12.50%)
    0 / 24 (0.00%)
         occurrences all number
    3
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    0 / 24 (0.00%)
    4 / 24 (16.67%)
         occurrences all number
    0
    0
    Respiratory tract infection
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    0
    Urinary tract infection
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 24 (8.33%)
         occurrences all number
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 24 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    0
    0

    More information

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

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

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd.com/CtrdWeb/home.novfor complete trial results
    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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