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    Clinical Trial Results:
    A Two-Part Phase 1/2 Study to Determine Safety, Tolerability, Pharmacokinetics, and Activity of K0706, a Novel Tyrosine Kinase Inhibitor (TKI), in Healthy Subjects and in Subjects with Chronic Myeloid Leukemia (CML) or Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ ALL)

    Summary
    EudraCT number
    		 2016-001754-18
    Trial protocol
    		 CZ   GB   BE   PL   ES   HU   RO  
    Global end of trial date
    03 Jan 2025

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

    Trial information

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    Trial identification
    Sponsor protocol code
    CLR_15_03
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02629692
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 IND Number: 127347
    Sponsors
    Sponsor organisation name
    Sun Pharma Advanced Research Company (SPARC) Limited
    Sponsor organisation address
    17/B Mahal Industrial Estate, Mahakali Caves Road Andheri (E), Mumbai , India, 400093
    Public contact
    Sponsor representative - Dr. Sandeep Inamdar, Sun Pharma Advanced Research Company (SPARC) Limited, 022 66455876, clinical.trials@sparcmail.com
    Scientific contact
    Sponsor representative - Dr. Sandeep Inamdar, Sun Pharma Advanced Research Company (SPARC) Limited, 022 66455876, clinical.trials@sparcmail.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
    03 Jan 2025
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    03 Jan 2025
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of Part A of the study is to: • Examine the safety and tolerability of single oral doses of K0706 in healthy male subjects The primary objectives of Part B of the study are to: • Determine the MTD (or RP2D) of K0706 administered orally in subjects with the selected BCR::ABL1-associated hematologic malignancies who have resistance or intolerance to the local Standard of Care CML therapy • Evaluate the safety of K0706 in subjects with BCR::ABL1-associated hematologic malignancies listed in the entry criteria The primary objectives of Part C of the study are to: • Evaluate the anti-leukemic efficacy of K0706 in subjects with CML-CP by cytogenetic outcomes and in subjects with CML-AP & BP by hematologic outcomes who have failed ≥ 3 TKIs, one of which includes ponatinib
    Protection of trial subjects
    The trial and site activities were monitored according to the ICH-GCP guidelines considering every aspect of the trial, ensuring that the rights, safety and well-being of patients are protected and consistent with the Declaration of Helsinki.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    27 Jun 2016
    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
    Romania: 5
    Country: Number of subjects enrolled
    Spain: 4
    Country: Number of subjects enrolled
    United Kingdom: 6
    Country: Number of subjects enrolled
    Belgium: 2
    Country: Number of subjects enrolled
    France: 13
    Country: Number of subjects enrolled
    Hungary: 2
    Country: Number of subjects enrolled
    Italy: 3
    Country: Number of subjects enrolled
    India: 20
    Country: Number of subjects enrolled
    Korea, Democratic People's Republic of: 15
    Country: Number of subjects enrolled
    United States: 54
    Worldwide total number of subjects
    124
    EEA total number of subjects
    29
    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)
    98
    From 65 to 84 years
    26
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Part A: The screening must be completed within 28 days prior to the first dose of the study drug. Part B: The screening must be performed within 21 days prior to the first dose of the study drug (i.e. Cycle 1 Day 1). Part C: The screening must be performed within 24 days prior to first dose of the study drug (i.e.: Cycle 1 Day 1).

    Period 1
    Period 1 title
    K0706 (Vodobatinib) (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 K0706 Part B+C
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Vodobatinib
    Investigational medicinal product code
    K0706
    Other name
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    Part B: K0706 capsules will be self-administered. The initial administered dose will be the dose from Part A where acceptable safety was observed. Part C: K0706 capsules will be self-administered once daily at dose of 174 mg.

    Arm title
    		 K0706 Part A
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Vodobatinib
    Investigational medicinal product code
    K0706
    Other name
    Vodobatinib
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    Part A: K0706 capsules were administered orally (in an upright position) after an overnight fast of at least 8 hours. For the food effect study, the 6 mg and 24 mg doses were evaluated.

    Number of subjects in period 1
    		K0706 Part B+C K0706 Part A
    Started
    84
    40
    Completed
    44
    39
    Not completed
    40
    1
         Increased worsening of memory impairment grade 2
    1
    -
         Adverse event, non-fatal
    13
    -
         Death
    1
    -
         Patient chose to come off trial
    1
    -
         Subject change treatment plan
    1
    -
         Progression of disease
    18
    -
         Lost to follow-up
    1
    1
         Missing
    1
    -
         Transitioning to alternate therapy
    1
    -
         Consent withdrawal and lack of compliance
    1
    -
         Lack of efficacy
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    K0706 Part B+C
    Reporting group description
    		 -

    Reporting group title
    K0706 Part A
    Reporting group description
    		 -

    Reporting group values
    		 K0706 Part B+C K0706 Part A Total
    Number of subjects
    		 84 40 124
    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)
    		 58 40 98
        From 65-84 years
    		 26 0 26
        85 years and over
    		 0 0 0
    Gender categorical
    Units: Subjects
        Male
    		 46 40 86
        Female
    		 38 0 38

    End points

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    End points reporting groups
    Reporting group title
    K0706 Part B+C
    Reporting group description
    		 -

    Reporting group title
    K0706 Part A
    Reporting group description
    		 -

    Primary: To Determine the Maximum Tolerated Dose (MTD) as Determined by Frequency of Dose Limiting Toxicities

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    End point title
    		 To Determine the Maximum Tolerated Dose (MTD) as Determined by Frequency of Dose Limiting Toxicities [1]
    End point description
    Part B
    End point type
    Primary
    End point timeframe
    Dose Limiting toxicities observed over a 4 week period
    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: Analysis Population Description: Safety Analysis Set
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    61
    0 [2]
    Units: Count of participants
    4
    Notes
    [2] - This parameter is only applicable to Part B
    No statistical analyses for this end point

    Primary: Incidence and severity of treatment emergent AEs (PART B)

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    End point title
    		 Incidence and severity of treatment emergent AEs (PART B) [3]
    End point description
    Part B
    End point type
    Primary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    Notes
    [3] - 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: Analysis Population Description: Safety Analysis Set
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    61
    0 [4]
    Units: Number of participants
    57
    Notes
    [4] - This parameter is only applicable to Part B
    No statistical analyses for this end point

    Primary: For CML subjects in CP at study entry

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    End point title
    		 For CML subjects in CP at study entry [5]
    End point description
    PART C: Proportion of subjects achieving Major Cytogenetic Response [ defined as complete cytogenetic response (CCyR; 0% Ph+metaphases) or partial cytogenetic response (PCyR; 1-35% Ph+ metaphases)] as assessed by conventional Karyotyping of Bone marrow aspirate
    End point type
    Primary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    Notes
    [5] - 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: Analysis Population Description: Efficacy Analysis Set
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    18
    0 [6]
    Units: Count of participants
    13
    Notes
    [6] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Primary: For CML subjects in AP at study entry

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    End point title
    		 For CML subjects in AP at study entry [7]
    End point description
    PART C: Proportion of subjects achieving Major Hematologic Response [ defined as complete hematologic response (CHR) or no evidence of leukemia (NEL)] as assessed by complete blood count of peripheral blood sample
    End point type
    Primary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    Notes
    [7] - 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: Analysis Population Description: Efficacy Analysis Set
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    5
    0 [8]
    Units: Number of participants
    1
    Notes
    [8] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Primary: For CML subjects in BP at study entry

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    End point title
    		 For CML subjects in BP at study entry [9]
    End point description
    PART C: Proportion of subjects achieving Major Hematologic Response [defined as complete hematologic response (CHR) or no evidence of leukemia (NEL)] as assessed by complete blood count of peripheral blood sample
    End point type
    Primary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    Notes
    [9] - 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: Analysis Population Description: Efficacy Analysis Set
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    0 [10]
    0 [11]
    Units: Count of participants
    Notes
    [10] - No subjects in this group
    [11] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Primary: Examination of the Safety and Tolerability of Single Oral Doses of K0706

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    End point title
    		 Examination of the Safety and Tolerability of Single Oral Doses of K0706 [12]
    End point description
    Part A
    End point type
    Primary
    End point timeframe
    Approximately 56 ± 2 days
    Notes
    [12] - 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: Analysis Population Description: Safety Analysis Set
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    0 [13]
    40
    Units: Number of participants
    40
    Notes
    [13] - This end point is only applicable to Part A
    No statistical analyses for this end point

    Secondary: Pharmacokinetic Profile of K0706 - Cmax [The Maximum (Peak) Observed Drug Concentration After Dose Administration]

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    End point title
    		 Pharmacokinetic Profile of K0706 - Cmax [The Maximum (Peak) Observed Drug Concentration After Dose Administration]
    End point description
    Part B and Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed for up to approximately 60 months after the first dose of Vodobatinib (K0706)
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    48
    0 [14]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    3217.7 ( 83.5 )
    ( )
    Notes
    [14] - This parameter is only applicable to Parts B and C
    No statistical analyses for this end point

    Secondary: Pharmacokinetic Profile of Vodobatinib (K0706) - Tmax [The Time to Reach Maximum (Peak) Drug Concentration After Dose Administration]

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    End point title
    		 Pharmacokinetic Profile of Vodobatinib (K0706) - Tmax [The Time to Reach Maximum (Peak) Drug Concentration After Dose Administration]
    End point description
    Part B and Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed for up to approximately 60 months after the first dose of Vodobatinib (K0706)
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    48
    0 [15]
    Units: hours
        median (full range (min-max))
    2 (0 to 8)
    ( to )
    Notes
    [15] - This parameter is only applicable to Parts B and C
    No statistical analyses for this end point

    Secondary: Pharmacokinetic Profile of Vodobatinib (K0706) - Cmin [ Minimum Observed Drug Concentration After Dose Administration]

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    End point title
    		 Pharmacokinetic Profile of Vodobatinib (K0706) - Cmin [ Minimum Observed Drug Concentration After Dose Administration]
    End point description
    Part B and Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed for up to approximately 60 months after the first dose of Vodobatinib (K0706)
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    48
    0 [16]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    720.1 ( 133.6 )
    ( )
    Notes
    [16] - This parameter is only applicable to Parts B and C
    No statistical analyses for this end point

    Secondary: In Subjects With CML- CP:Proportion of Subjects Achieving Complete Hematological Response as Assessed by Complete Blood Count of Peripheral Blood Sample

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    End point title
    		 In Subjects With CML- CP:Proportion of Subjects Achieving Complete Hematological Response as Assessed by Complete Blood Count of Peripheral Blood Sample
    End point description
    Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    18
    0 [17]
    Units: NA
    4
    Notes
    [17] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Secondary: In Subjects With CML- CP:Proportion of Subjects Achieving Complete Cytogenetic Response as Assessed by Conventional Karyotyping of Bone Marrow Aspirate

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    End point title
    		 In Subjects With CML- CP:Proportion of Subjects Achieving Complete Cytogenetic Response as Assessed by Conventional Karyotyping of Bone Marrow Aspirate
    End point description
    Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    18
    0 [18]
    Units: NA
    10
    Notes
    [18] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Secondary: In Subjects With CML- CP:Proportion of Subjects Achieving Major Molecular Response as Assessed by BCR-ABL Transcript Levels (BCR-ABL1 Ratio of ≤ 0.1%) in Peripheral Blood Using PCR (Polymerase Chain Reaction)

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    End point title
    		 In Subjects With CML- CP:Proportion of Subjects Achieving Major Molecular Response as Assessed by BCR-ABL Transcript Levels (BCR-ABL1 Ratio of ≤ 0.1%) in Peripheral Blood Using PCR (Polymerase Chain Reaction)
    End point description
    Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    18
    0 [19]
    Units: NA
    7
    Notes
    [19] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Secondary: In Subjects With CML-AP & BP: Proportion of Subjects Achieving Complete Cytogenetic Response as Assessed by Conventional Karyotyping of Bone Marrow Aspirate

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    End point title
    		 In Subjects With CML-AP & BP: Proportion of Subjects Achieving Complete Cytogenetic Response as Assessed by Conventional Karyotyping of Bone Marrow Aspirate
    End point description
    Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    5
    0 [20]
    Units: NA
    2
    Notes
    [20] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Secondary: In Subjects With CML-AP & BP: Proportion of Subjects Achieving Partial Cytogenetic Response (PCyR) as Assessed by Conventional Karyotyping of Bone Marrow Aspirate

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    End point title
    		 In Subjects With CML-AP & BP: Proportion of Subjects Achieving Partial Cytogenetic Response (PCyR) as Assessed by Conventional Karyotyping of Bone Marrow Aspirate
    End point description
    Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of K0706
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    5
    0 [21]
    Units: NA
    0
    Notes
    [21] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Secondary: In Subjects With CML-AP & BP: Proportion of Subjects Achieving Major Molecular Response as Assessed by BCR-ABL Transcript Levels (BCR-ABL1 Ratio of ≤ 0.1%) in Peripheral Blood Using PCR (Polymerase Chain Reaction)

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    End point title
    		 In Subjects With CML-AP & BP: Proportion of Subjects Achieving Major Molecular Response as Assessed by BCR-ABL Transcript Levels (BCR-ABL1 Ratio of ≤ 0.1%) in Peripheral Blood Using PCR (Polymerase Chain Reaction)
    End point description
    Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    5
    0 [22]
    Units: NA
    1
    Notes
    [22] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Secondary: Time to Major Cytogenetic Response (MCyR): Time to MCyR is the Time From First Dose to First MCyR (0-35% Ph+ Metaphases) ; Computed Only for Subjects Who Achieved MCyR

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    End point title
    		 Time to Major Cytogenetic Response (MCyR): Time to MCyR is the Time From First Dose to First MCyR (0-35% Ph+ Metaphases) ; Computed Only for Subjects Who Achieved MCyR
    End point description
    Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    5
    0 [23]
    Units: months
        arithmetic mean (standard deviation)
    3.9 ( 2.53 )
    ( )
    Notes
    [23] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Secondary: Time to Major Molecular Response : Time to MMR is the Time From First Dose to First MMR (BCR-ABL1 Ratio of ≤ 0.1%) Computed Only for Subjects Who Achieved MMR

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    End point title
    		 Time to Major Molecular Response : Time to MMR is the Time From First Dose to First MMR (BCR-ABL1 Ratio of ≤ 0.1%) Computed Only for Subjects Who Achieved MMR
    End point description
    Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    7
    0 [24]
    Units: months
        arithmetic mean (standard deviation)
    5.0 ( 4.71 )
    ( )
    Notes
    [24] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Secondary: In All Subjects Progression Free Survival (PFS)

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    End point title
    		 In All Subjects Progression Free Survival (PFS)
    End point description
    Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    23 [25]
    0 [26]
    Units: Months
        number (confidence interval 95%)
    81.2 (57.2 to 92.5)
    ( to )
    Notes
    [25] - Timepoint: At 12 months
    [26] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Secondary: In All Subjects Overall Survival (OS)

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    End point title
    		 In All Subjects Overall Survival (OS)
    End point description
    Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    23 [27]
    0 [28]
    Units: Months
        number (confidence interval 95%)
    95.7 (72.9 to 99.4)
    ( to )
    Notes
    [27] - Timepoint: At 12 months
    [28] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Secondary: Incidence and Severity of Treatment Emergent AEs (PART C)

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    End point title
    		 Incidence and Severity of Treatment Emergent AEs (PART C)
    End point description
    Part C
    End point type
    Secondary
    End point timeframe
    All subjects will be followed up for 60 months from the first dose of Vodobatinib (K0706)
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    23
    0 [29]
    Units: NA
    21
    Notes
    [29] - This parameter is only applicable to Part C
    No statistical analyses for this end point

    Secondary: To Characterize the Pharmacokinetics (Cmax) of K0706 After Single Oral Doses in Healthy Male Subjects

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    End point title
    		 To Characterize the Pharmacokinetics (Cmax) of K0706 After Single Oral Doses in Healthy Male Subjects
    End point description
    Part A
    End point type
    Secondary
    End point timeframe
    Approximately 28 ± 2 days
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    0 [30]
    6 [31]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    ( )
    866.5 ( 35.9 )
    Notes
    [30] - This end point is only applicable to Part A
    [31] - PK Analysis Set (SAD study; 24 mg cohort)
    No statistical analyses for this end point

    Secondary: To Characterize the Pharmacokinetics (Cmax) of K0706 After Single Oral Doses in Fasted and Fed State in Healthy Male Subjects

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    End point title
    		 To Characterize the Pharmacokinetics (Cmax) of K0706 After Single Oral Doses in Fasted and Fed State in Healthy Male Subjects
    End point description
    Part A
    End point type
    Secondary
    End point timeframe
    Approximately 28 ± 2 days
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    0 [32]
    8 [33]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    ( )
    642 ( 54.6 )
    Notes
    [32] - This endpoint is only applicable to Part A
    [33] - PK Analysis Set (24 mg cohort); Cmax (fasted state)
    No statistical analyses for this end point

    Secondary: To Characterize the Pharmacokinetics (AUC(0-inf)) of K0706 After Single Oral Doses in Healthy Male Subjects

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    End point title
    		 To Characterize the Pharmacokinetics (AUC(0-inf)) of K0706 After Single Oral Doses in Healthy Male Subjects
    End point description
    Part A
    End point type
    Secondary
    End point timeframe
    Approximately 28 ± 2 days
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    0 [34]
    6 [35]
    Units: ng*h/mL
        geometric mean (geometric coefficient of variation)
    ( )
    6322 ( 32.6 )
    Notes
    [34] - This parameter is only applicable to Part A
    [35] - PK Analysis Set (SAD study; 24 mg cohort)
    No statistical analyses for this end point

    Secondary: To Characterize the Pharmacokinetics (Cmax) of K0706 After Single Oral Doses in Fasted and Fed State in Healthy Male Subjects

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    End point title
    		 To Characterize the Pharmacokinetics (Cmax) of K0706 After Single Oral Doses in Fasted and Fed State in Healthy Male Subjects
    End point description
    Part A
    End point type
    Secondary
    End point timeframe
    Approximately 28 ± 2 days
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    0 [36]
    8 [37]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    ( )
    323.7 ( 26.3 )
    Notes
    [36] - This end point is only applicable to Part A
    [37] - PK Analysis Set (24 mg cohort); Cmax (fed state)
    No statistical analyses for this end point

    Secondary: To Characterize the Pharmacokinetics of K0706 (AUC(0-inf)) After Single Oral Doses in Fasted and Fed State in Healthy Male Subjects

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    End point title
    		 To Characterize the Pharmacokinetics of K0706 (AUC(0-inf)) After Single Oral Doses in Fasted and Fed State in Healthy Male Subjects
    End point description
    Part A
    End point type
    Secondary
    End point timeframe
    Approximately 28 ± 2 days
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    0 [38]
    8 [39]
    Units: ng*h/mL
        geometric mean (geometric coefficient of variation)
    ( )
    5739 ( 26.2 )
    Notes
    [38] - This end point is only applicable to Part A
    [39] - PK Analysis Set (24 mg cohort); AUC(0-inf) (fasted state)
    No statistical analyses for this end point

    Secondary: To Characterize the Pharmacokinetics of K0706 (AUC(0-inf)) After Single Oral Doses in Fasted and Fed State in Healthy Male Subjects

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    End point title
    		 To Characterize the Pharmacokinetics of K0706 (AUC(0-inf)) After Single Oral Doses in Fasted and Fed State in Healthy Male Subjects
    End point description
    Part A
    End point type
    Secondary
    End point timeframe
    Approximately 28 ± 2 days
    End point values
    		 K0706 Part B+C K0706 Part A
    Number of subjects analysed
    0 [40]
    8 [41]
    Units: ng*h/mL
        geometric mean (geometric coefficient of variation)
    ( )
    4521 ( 28.8 )
    Notes
    [40] - This end point is only applicable to Part A
    [41] - PK Analysis Set (24 mg cohort); AUC(0-inf) (fed state)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    AE reporting period for safety surveillance begins after subject is randomized into the study and receives at least 1 dose of drug for treatment emergent adverse events (TEAEs) and continues until 30 days from end of treatment visit.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    K0706
    Reporting group description
    		 Parts B+C

    Serious adverse events
    		 K0706
    Total subjects affected by serious adverse events
         subjects affected / exposed
    27 / 124 (21.77%)
         number of deaths (all causes)
    8
         number of deaths resulting from adverse events
    7
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Chronic myeloid leukaemia
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Lung neoplasm malignant
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Lung adenocarcinoma
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Septic shock
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Deep vein thrombosis
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Disease progression
         subjects affected / exposed
    2 / 124 (1.61%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 1
    Pyrexia
         subjects affected / exposed
    2 / 124 (1.61%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Sudden death
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Immune system disorders
    Swollen tongue
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    2 / 124 (1.61%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Pulmonary toxicity
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Skull fracture
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Spinal fracture
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Pericardial effusion
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Acute myocardial infarction
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Amnesia
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Cerebral haemorrhage
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cerebrovascular accident
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Dementia
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Haemorrhage intracranial
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    1 / 1
    Hypoxic-ischaemic encephalopathy
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Transient ischaemic attack
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    2 / 124 (1.61%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Febrile neutropenia
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Leukocytosis
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Nausea
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Varices oesophageal
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Cholelithiasis
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cholecystitis acute
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Haematuria
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Arthritis
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Back pain
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Bone pain
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Muscular weakness
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal chest pain
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    3 / 124 (2.42%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    2 / 124 (1.61%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    COVID-19
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia fungal
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Pneumonia viral
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin infection
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Suspected COVID-19
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Viral upper respiratory tract infection
         subjects affected / exposed
    1 / 124 (0.81%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 K0706
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    93 / 124 (75.00%)
    Investigations
    Lipase increased
         subjects affected / exposed
    12 / 124 (9.68%)
         occurrences all number
    12
    Amylase increased
         subjects affected / exposed
    7 / 124 (5.65%)
         occurrences all number
    7
    Vascular disorders
    Hypertension
         subjects affected / exposed
    11 / 124 (8.87%)
         occurrences all number
    11
    Nervous system disorders
    Headache
         subjects affected / exposed
    15 / 124 (12.10%)
         occurrences all number
    15
    Dizziness
         subjects affected / exposed
    8 / 124 (6.45%)
         occurrences all number
    8
    Blood and lymphatic system disorders
    Thrombocytopenia
         subjects affected / exposed
    28 / 124 (22.58%)
         occurrences all number
    28
    Anaemia
         subjects affected / exposed
    16 / 124 (12.90%)
         occurrences all number
    16
    Neutropenia
         subjects affected / exposed
    12 / 124 (9.68%)
         occurrences all number
    12
    Leukopenia
         subjects affected / exposed
    7 / 124 (5.65%)
         occurrences all number
    7
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    16 / 124 (12.90%)
         occurrences all number
    16
    Pyrexia
         subjects affected / exposed
    9 / 124 (7.26%)
         occurrences all number
    9
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    19 / 124 (15.32%)
         occurrences all number
    19
    Constipation
         subjects affected / exposed
    15 / 124 (12.10%)
         occurrences all number
    15
    Nausea
         subjects affected / exposed
    13 / 124 (10.48%)
         occurrences all number
    13
    Vomiting
         subjects affected / exposed
    10 / 124 (8.06%)
         occurrences all number
    10
    Abdominal pain
         subjects affected / exposed
    10 / 124 (8.06%)
         occurrences all number
    10
    Abdominal pain upper
         subjects affected / exposed
    8 / 124 (6.45%)
         occurrences all number
    8
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    16 / 124 (12.90%)
         occurrences all number
    16
    Dyspnoea
         subjects affected / exposed
    12 / 124 (9.68%)
         occurrences all number
    12
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    13 / 124 (10.48%)
         occurrences all number
    13
    Dry skin
         subjects affected / exposed
    10 / 124 (8.06%)
         occurrences all number
    10
    Pruritus
         subjects affected / exposed
    8 / 124 (6.45%)
         occurrences all number
    8
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    10 / 124 (8.06%)
         occurrences all number
    10
    Back pain
         subjects affected / exposed
    10 / 124 (8.06%)
         occurrences all number
    10
    Myalgia
         subjects affected / exposed
    10 / 124 (8.06%)
         occurrences all number
    10
    Pain in extremity
         subjects affected / exposed
    9 / 124 (7.26%)
         occurrences all number
    9
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    12 / 124 (9.68%)
         occurrences all number
    12
    Upper respiratory tract infection
         subjects affected / exposed
    11 / 124 (8.87%)
         occurrences all number
    11
    COVID-19
         subjects affected / exposed
    10 / 124 (8.06%)
         occurrences all number
    10
    Rhinitis
         subjects affected / exposed
    7 / 124 (5.65%)
         occurrences all number
    7
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    8 / 124 (6.45%)
         occurrences all number
    8

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    12 Sep 2019
    The total number of sites participating in Part C was changed from 80 to 90. The study population was specified and Ph+ ALL was excluded from the pivotal study after consideration of FDA comments and the lack of sufficient data in Ph+ALL of Part B. Additional secondary objectives were added in alignment with the endpoints. The objectives were modified in alignment with endpoints. The duration of study treatment was extended to 60 months as per FDA’s recommendations. Primary endpoints changed as per FDA recommendation to not consider maintenance of response as an efficacy endpoint. Inclusion and Exclusion criteria updated.

    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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    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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