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    Clinical Trial Results:
    A Phase II multi-center, open label, randomized study to assess safety and efficacy of two different schedules of oral LDE225 in adult patients with relapsed/refractory or untreated elderly patients with acute leukemia

    Summary
    EudraCT number
    		 2012-004022-21
    Trial protocol
    		 AT   ES   GB   IE   SE   NO   BE   DE   FR   NL   HU   IT  
    Global end of trial date
    26 May 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    10 Jun 2016
    First version publication date
    10 Jun 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CLDE225X2203
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01826214
    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,
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111,
    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
    26 May 2015
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    26 May 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective was to evaluate the rate of complete remission (CR) and complete remission with incomplete blood count recovery (CRi) on two different dosing schedules of sonidegib in acute leukemia.
    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
    30 May 2013
    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
    Australia: 6
    Country: Number of subjects enrolled
    Austria: 4
    Country: Number of subjects enrolled
    Belgium: 7
    Country: Number of subjects enrolled
    Canada: 7
    Country: Number of subjects enrolled
    Germany: 15
    Country: Number of subjects enrolled
    Hungary: 1
    Country: Number of subjects enrolled
    Netherlands: 10
    Country: Number of subjects enrolled
    Norway: 4
    Country: Number of subjects enrolled
    Spain: 9
    Country: Number of subjects enrolled
    United Kingdom: 1
    Country: Number of subjects enrolled
    United States: 6
    Worldwide total number of subjects
    70
    EEA total number of subjects
    51
    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)
    19
    From 65 to 84 years
    50
    85 years and over
    1

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 35 patients were randomized but only 34 patients received at least one dose of study drug in the LDE225-800 (schedule B ) arm.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 LDE225-400
    Arm description
    		 Patients who were randomized to Schedule A, and received 400 mg LDE225 twice daily for the first two weeks only and after two weeks, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Sonidegib
    Investigational medicinal product code
    LDE225
    Other name
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    Sonidegib 400 mg 2x daily for 2 weeks, then 800mg once daily

    Arm title
    		 LDE225-800
    Arm description
    		 Patients who were randomized to Schedule B, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Sonidegib
    Investigational medicinal product code
    LDE225
    Other name
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    Sonidegib 800 mg taken orally one daily

    Number of subjects in period 1
    		LDE225-400 LDE225-800
    Started
    35
    35
    Treated
    35
    34
    Completed
    0
    0
    Not completed
    35
    35
         Adverse event, serious fatal
    3
    1
         Physician decision
    -
    2
         Study terminated by Sponsor
    -
    1
         not treated
    -
    1
         Adverse event, non-fatal
    7
    6
         Lost to follow-up
    1
    -
         Progressive disease
    22
    21
         Subject/guardian decision
    2
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    LDE225-400
    Reporting group description
    		 Patients who were randomized to Schedule A, and received 400 mg LDE225 twice daily for the first two weeks only and after two weeks, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study.

    Reporting group title
    LDE225-800
    Reporting group description
    		 Patients who were randomized to Schedule B, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study.

    Reporting group values
    		 LDE225-400 LDE225-800 Total
    Number of subjects
    		 35 35 70
    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)
    		 11 8 19
        From 65-84 years
    		 24 26 50
        85 years and over
    		 0 1 1
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 65.3 ( 12.31 ) 67.7 ( 11.65 ) -
    Gender, Male/Female
    Units: Participants
        Female
    		 18 13 31
        Male
    		 17 22 39

    End points

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    End points reporting groups
    Reporting group title
    LDE225-400
    Reporting group description
    		 Patients who were randomized to Schedule A, and received 400 mg LDE225 twice daily for the first two weeks only and after two weeks, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study.

    Reporting group title
    LDE225-800
    Reporting group description
    		 Patients who were randomized to Schedule B, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study.

    Primary: Rate of complete remission (CR)

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    End point title
    		 Rate of complete remission (CR) [1]
    End point description
    Complete Response (CR) was based on the International Working Group (IWG) criteria based on weekly peripheral blood count measurements and bone marrow biopsy/aspiration collection. Efficacy assessments were performed to determine CR. A treatment cycle was defined as 4 weeks. The outcome measure for the study is based on standardized response criteria as defined by the International Working Group (IWG) for AML. The IWG was established by a group of investigators interested in the design and conduct of clinical trials in acute myeloid leukemia (AML). The criteria established by this group (a set of recommendations for response assessment) are well established, endorsed by major institutions and Health Authorities, and are widely used in clinical trials. No statistical analysis was planned for this primary outcome.
    End point type
    Primary
    End point timeframe
    at screening, every week up to Week 9, every 2 weeks thereafter until CR, every 4 weeks after CR up to 24 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 planned for this primary outcome.
    End point values
    		 LDE225-400 LDE225-800
    Number of subjects analysed
    35
    35
    Units: Participants
    0
    0
    No statistical analyses for this end point

    Primary: Complete remission with incomplete blood count recovery (CRi)

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    End point title
    		 Complete remission with incomplete blood count recovery (CRi) [2]
    End point description
    The other primary efficacy endpoint was CRi based on the International Working Group (IWG) criteria based on weekly peripheral blood count measurements and bone marrow biopsy/aspiration collection. Efficacy assessments were performed to determine CRi. A treatment cycle was defined as 4 weeks. No statistical analysis was planned for this primary outcome.
    End point type
    Primary
    End point timeframe
    within 3 days after clearance of blasts from peripheral blood (PB), monthly thereafter until CR or reappearance of blasts in the PB, after CR every other month until discontination up to 24 months
    Notes
    [2] - 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 planned for this primary outcome.
    End point values
    		 LDE225-400 LDE225-800
    Number of subjects analysed
    35
    35
    Units: Participants
    1
    0
    No statistical analyses for this end point

    Secondary: Overall Response Rate (ORR)

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    End point title
    		 Overall Response Rate (ORR)
    End point description
    ORR was the rate of complete remission (CR), complete remission with incomplete blood count recovery (CRi) or partial response (PR) according to IWG criteria. CR, CRi or PR will be assessed through bone marrow biopsy/aspirate and peripheral blood blasts counts.
    End point type
    Secondary
    End point timeframe
    Every 8 weeks for the first 6 months and every 12 weeks until 53 weeks after the last patient is enrolled or until relapse up to 24 months
    End point values
    		 LDE225-400 LDE225-800
    Number of subjects analysed
    35
    35
    Units: Participants
    1
    0
    No statistical analyses for this end point

    Secondary: Parmacokintics (PK) parameter: Cmax

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    End point title
    		 Parmacokintics (PK) parameter: Cmax
    End point description
    Cmax is the maximum observed plasma concentration after drug administration.The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the pharmacokineticist. Cmax was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters.
    End point type
    Secondary
    End point timeframe
    Week 1 Day 1, Week 9 Day 1
    End point values
    		 LDE225-400 LDE225-800
    Number of subjects analysed
    29
    33
    Units: ng/mL
    arithmetic mean (standard deviation)
        Week 1 Day 1
    237 ( 158 )
    343 ( 275 )
        Week 9 Day 1(n: 7, 7)
    1640 ( 612 )
    1500 ( 874 )
    No statistical analyses for this end point

    Secondary: Parmacokintics (PK) parameter: Tmax

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    End point title
    		 Parmacokintics (PK) parameter: Tmax
    End point description
    Tmax is the time to reach Cmax. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. Tmax was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters.
    End point type
    Secondary
    End point timeframe
    Week 1 Day 1,Week 9 Day 1
    End point values
    		 LDE225-400 LDE225-800
    Number of subjects analysed
    29
    33
    Units: hr
    median (inter-quartile range (Q1-Q3))
        Week 1 Day 1
    2.13 (1 to 8.08)
    2.12 (1 to 8)
        Week 9 Day 1 (n: 7, 7)
    1.88 (1.12 to 8.13)
    2.02 (0 to 23.3)
    No statistical analyses for this end point

    Secondary: Parmacokintics (PK) parameter: AUC0-8h

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    End point title
    		 Parmacokintics (PK) parameter: AUC0-8h
    End point description
    AUC0-8h is the area under the concentration-time curve from time zero to 8 hours. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. AUC0-8h was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters.
    End point type
    Secondary
    End point timeframe
    Week 1 Day 1,Week 9 Day 1
    End point values
    		 LDE225-400 LDE225-800
    Number of subjects analysed
    27
    33
    Units: ng*hr/mL
    arithmetic mean (standard deviation)
        Week 1 Day1
    988 ( 542 )
    1560 ( 1230 )
        Week 9 Day1 (n: 7, 7)
    9750 ( 2830 )
    7910 ( 5090 )
    No statistical analyses for this end point

    Secondary: Parmacokintics (PK) parameter: AUC0-24h

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    End point title
    		 Parmacokintics (PK) parameter: AUC0-24h
    End point description
    AUC0-8h is the area under the concentration-time curve from time zero to 8 hours. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. AUC0-8h was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters.
    End point type
    Secondary
    End point timeframe
    Week 1 Day 1,Week 9 Day 1
    End point values
    		 LDE225-400 LDE225-800
    Number of subjects analysed
    7
    32
    Units: ng*hr/mL
    arithmetic mean (standard deviation)
        Week 1 Day 1
    0 ( 0 )
    3110 ( 2620 )
        Week 9 Day 1 (n: 7, 6)
    26500 ( 6650 )
    24000 ( 11500 )
    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 L:ast Patient Last Visit (LPLV). All Adverse Events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit
    Adverse event reporting additional description
    Consistent with EudraCT disclosure specifications, Novartis has reported under the Serious adverse events field “number of deaths resulting from adverse events” all those deaths, resulting from serious adverse events that are deemed to be causally related to treatment by the investigator.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.0
    Reporting groups
    Reporting group title
    LDE225 800 mg QD
    Reporting group description
    		 LDE225 800 mg QD

    Reporting group title
    LDE225 400 mg BID in the first two weeks, then 800 QD
    Reporting group description
    		 LDE225 400 mg BID in the first two weeks, then 800 QD

    Serious adverse events
    		 LDE225 800 mg QD LDE225 400 mg BID in the first two weeks, then 800 QD
    Total subjects affected by serious adverse events
         subjects affected / exposed
    25 / 34 (73.53%)
    25 / 35 (71.43%)
         number of deaths (all causes)
    16
    13
         number of deaths resulting from adverse events
    0
    0
    Investigations
    BLOOD CREATINE PHOSPHOKINASE INCREASED
         subjects affected / exposed
    2 / 34 (5.88%)
    2 / 35 (5.71%)
         occurrences causally related to treatment / all
    2 / 2
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    MYOGLOBIN BLOOD INCREASED
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    CANCER PAIN
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    LEUKAEMIC INFILTRATION
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Injury, poisoning and procedural complications
    FALL
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    SUBDURAL HAEMORRHAGE
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vascular disorders
    CIRCULATORY COLLAPSE
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    CEREBRAL HAEMORRHAGE
         subjects affected / exposed
    1 / 34 (2.94%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    0 / 1
    DYSGEUSIA
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    HAEMORRHAGE INTRACRANIAL
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    QUADRIPLEGIA
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    ANAEMIA
         subjects affected / exposed
    2 / 34 (5.88%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    FEBRILE NEUTROPENIA
         subjects affected / exposed
    7 / 34 (20.59%)
    9 / 35 (25.71%)
         occurrences causally related to treatment / all
    0 / 13
    0 / 10
         deaths causally related to treatment / all
    0 / 2
    0 / 1
    LEUKOCYTOSIS
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    PANCYTOPENIA
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    ASTHENIA
         subjects affected / exposed
    1 / 34 (2.94%)
    2 / 35 (5.71%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    FATIGUE
         subjects affected / exposed
    1 / 34 (2.94%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    GENERAL PHYSICAL HEALTH DETERIORATION
         subjects affected / exposed
    1 / 34 (2.94%)
    4 / 35 (11.43%)
         occurrences causally related to treatment / all
    0 / 1
    1 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    MULTI-ORGAN FAILURE
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    PYREXIA
         subjects affected / exposed
    4 / 34 (11.76%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    ABDOMINAL PAIN
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    ABDOMINAL PAIN UPPER
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    COLITIS
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    CONSTIPATION
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    DIARRHOEA
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    GASTRIC HAEMORRHAGE
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    GASTROINTESTINAL HAEMORRHAGE
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    GINGIVAL HYPERTROPHY
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    NAUSEA
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    OESOPHAGEAL ULCER
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    STOMATITIS
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    VOMITING
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    EPISTAXIS
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    IDIOPATHIC PNEUMONIA SYNDROME
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    LARYNGEAL INFLAMMATION
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    PLEURAL EFFUSION
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    RESPIRATORY FAILURE
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Musculoskeletal and connective tissue disorders
    BACK PAIN
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 35 (5.71%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    MUSCLE SPASMS
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    MYALGIA
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    MYOPATHY
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    OSTEONECROSIS OF JAW
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    ANAL ABSCESS
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    ARTHRITIS INFECTIVE
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    BACTERAEMIA
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    CLOSTRIDIAL INFECTION
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    DEVICE RELATED INFECTION
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    ENTEROCOCCAL BACTERAEMIA
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    EPIGLOTTITIS
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    INFECTION
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    INFLUENZA
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    LUNG INFECTION
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 35 (5.71%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    NEUTROPENIC INFECTION
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    PNEUMONIA
         subjects affected / exposed
    3 / 34 (8.82%)
    3 / 35 (8.57%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 3
         deaths causally related to treatment / all
    0 / 1
    0 / 1
    SEPSIS
         subjects affected / exposed
    4 / 34 (11.76%)
    2 / 35 (5.71%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 2
         deaths causally related to treatment / all
    0 / 3
    0 / 1
    STAPHYLOCOCCAL SEPSIS
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    URINARY TRACT INFECTION
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    DEHYDRATION
         subjects affected / exposed
    0 / 34 (0.00%)
    1 / 35 (2.86%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    FLUID OVERLOAD
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    GOUT
         subjects affected / exposed
    1 / 34 (2.94%)
    0 / 35 (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
    		 LDE225 800 mg QD LDE225 400 mg BID in the first two weeks, then 800 QD
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    34 / 34 (100.00%)
    35 / 35 (100.00%)
    Vascular disorders
    HAEMATOMA
         subjects affected / exposed
    2 / 34 (5.88%)
    0 / 35 (0.00%)
         occurrences all number
    2
    0
    HYPERTENSION
         subjects affected / exposed
    2 / 34 (5.88%)
    0 / 35 (0.00%)
         occurrences all number
    2
    0
    ORTHOSTATIC HYPOTENSION
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 35 (5.71%)
         occurrences all number
    0
    2
    General disorders and administration site conditions
    ASTHENIA
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 35 (2.86%)
         occurrences all number
    2
    1
    CHILLS
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 35 (5.71%)
         occurrences all number
    0
    2
    FATIGUE
         subjects affected / exposed
    10 / 34 (29.41%)
    8 / 35 (22.86%)
         occurrences all number
    15
    9
    FEELING COLD
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 35 (5.71%)
         occurrences all number
    0
    2
    GENERAL PHYSICAL HEALTH DETERIORATION
         subjects affected / exposed
    3 / 34 (8.82%)
    1 / 35 (2.86%)
         occurrences all number
    3
    1
    MALAISE
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 35 (2.86%)
         occurrences all number
    2
    1
    NON-CARDIAC CHEST PAIN
         subjects affected / exposed
    2 / 34 (5.88%)
    0 / 35 (0.00%)
         occurrences all number
    2
    0
    OEDEMA PERIPHERAL
         subjects affected / exposed
    5 / 34 (14.71%)
    4 / 35 (11.43%)
         occurrences all number
    5
    4
    PAIN
         subjects affected / exposed
    3 / 34 (8.82%)
    0 / 35 (0.00%)
         occurrences all number
    3
    0
    PYREXIA
         subjects affected / exposed
    10 / 34 (29.41%)
    7 / 35 (20.00%)
         occurrences all number
    11
    9
    Respiratory, thoracic and mediastinal disorders
    COUGH
         subjects affected / exposed
    4 / 34 (11.76%)
    3 / 35 (8.57%)
         occurrences all number
    6
    3
    DYSPNOEA
         subjects affected / exposed
    6 / 34 (17.65%)
    7 / 35 (20.00%)
         occurrences all number
    8
    9
    EPISTAXIS
         subjects affected / exposed
    3 / 34 (8.82%)
    4 / 35 (11.43%)
         occurrences all number
    3
    4
    OROPHARYNGEAL PAIN
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 35 (2.86%)
         occurrences all number
    3
    1
    PRODUCTIVE COUGH
         subjects affected / exposed
    3 / 34 (8.82%)
    0 / 35 (0.00%)
         occurrences all number
    4
    0
    Psychiatric disorders
    ANXIETY
         subjects affected / exposed
    0 / 34 (0.00%)
    4 / 35 (11.43%)
         occurrences all number
    0
    4
    CONFUSIONAL STATE
         subjects affected / exposed
    0 / 34 (0.00%)
    4 / 35 (11.43%)
         occurrences all number
    0
    4
    INSOMNIA
         subjects affected / exposed
    2 / 34 (5.88%)
    2 / 35 (5.71%)
         occurrences all number
    2
    2
    Investigations
    ASPARTATE AMINOTRANSFERASE INCREASED
         subjects affected / exposed
    1 / 34 (2.94%)
    2 / 35 (5.71%)
         occurrences all number
    1
    2
    BLOOD CREATINE PHOSPHOKINASE INCREASED
         subjects affected / exposed
    7 / 34 (20.59%)
    8 / 35 (22.86%)
         occurrences all number
    9
    9
    BLOOD CREATININE INCREASED
         subjects affected / exposed
    5 / 34 (14.71%)
    1 / 35 (2.86%)
         occurrences all number
    6
    1
    BLOOD MAGNESIUM DECREASED
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 35 (2.86%)
         occurrences all number
    2
    1
    MYOGLOBIN BLOOD INCREASED
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 35 (5.71%)
         occurrences all number
    0
    2
    NEUTROPHIL COUNT DECREASED
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 35 (2.86%)
         occurrences all number
    2
    1
    PLATELET COUNT DECREASED
         subjects affected / exposed
    2 / 34 (5.88%)
    2 / 35 (5.71%)
         occurrences all number
    5
    3
    WEIGHT DECREASED
         subjects affected / exposed
    2 / 34 (5.88%)
    2 / 35 (5.71%)
         occurrences all number
    2
    2
    WHITE BLOOD CELL COUNT DECREASED
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 35 (2.86%)
         occurrences all number
    2
    1
    Injury, poisoning and procedural complications
    FALL
         subjects affected / exposed
    2 / 34 (5.88%)
    0 / 35 (0.00%)
         occurrences all number
    2
    0
    Cardiac disorders
    ATRIAL FIBRILLATION
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 35 (2.86%)
         occurrences all number
    2
    1
    Nervous system disorders
    DIZZINESS
         subjects affected / exposed
    5 / 34 (14.71%)
    2 / 35 (5.71%)
         occurrences all number
    7
    2
    DYSGEUSIA
         subjects affected / exposed
    5 / 34 (14.71%)
    3 / 35 (8.57%)
         occurrences all number
    5
    3
    HEADACHE
         subjects affected / exposed
    5 / 34 (14.71%)
    3 / 35 (8.57%)
         occurrences all number
    9
    3
    Blood and lymphatic system disorders
    ANAEMIA
         subjects affected / exposed
    6 / 34 (17.65%)
    5 / 35 (14.29%)
         occurrences all number
    8
    6
    FEBRILE NEUTROPENIA
         subjects affected / exposed
    2 / 34 (5.88%)
    3 / 35 (8.57%)
         occurrences all number
    2
    3
    LEUKOCYTOSIS
         subjects affected / exposed
    4 / 34 (11.76%)
    1 / 35 (2.86%)
         occurrences all number
    5
    1
    LEUKOPENIA
         subjects affected / exposed
    3 / 34 (8.82%)
    2 / 35 (5.71%)
         occurrences all number
    4
    4
    NEUTROPENIA
         subjects affected / exposed
    2 / 34 (5.88%)
    3 / 35 (8.57%)
         occurrences all number
    2
    4
    THROMBOCYTOPENIA
         subjects affected / exposed
    5 / 34 (14.71%)
    7 / 35 (20.00%)
         occurrences all number
    7
    7
    Gastrointestinal disorders
    ABDOMINAL PAIN
         subjects affected / exposed
    3 / 34 (8.82%)
    4 / 35 (11.43%)
         occurrences all number
    3
    5
    ABDOMINAL PAIN UPPER
         subjects affected / exposed
    1 / 34 (2.94%)
    2 / 35 (5.71%)
         occurrences all number
    1
    2
    APHTHOUS STOMATITIS
         subjects affected / exposed
    2 / 34 (5.88%)
    0 / 35 (0.00%)
         occurrences all number
    2
    0
    CONSTIPATION
         subjects affected / exposed
    8 / 34 (23.53%)
    6 / 35 (17.14%)
         occurrences all number
    9
    6
    DIARRHOEA
         subjects affected / exposed
    8 / 34 (23.53%)
    7 / 35 (20.00%)
         occurrences all number
    11
    8
    DYSPEPSIA
         subjects affected / exposed
    3 / 34 (8.82%)
    3 / 35 (8.57%)
         occurrences all number
    3
    3
    DYSPHAGIA
         subjects affected / exposed
    2 / 34 (5.88%)
    0 / 35 (0.00%)
         occurrences all number
    2
    0
    GINGIVAL BLEEDING
         subjects affected / exposed
    3 / 34 (8.82%)
    0 / 35 (0.00%)
         occurrences all number
    3
    0
    HAEMORRHOIDS
         subjects affected / exposed
    1 / 34 (2.94%)
    2 / 35 (5.71%)
         occurrences all number
    1
    2
    MOUTH HAEMORRHAGE
         subjects affected / exposed
    3 / 34 (8.82%)
    0 / 35 (0.00%)
         occurrences all number
    3
    0
    MOUTH ULCERATION
         subjects affected / exposed
    1 / 34 (2.94%)
    2 / 35 (5.71%)
         occurrences all number
    1
    2
    NAUSEA
         subjects affected / exposed
    10 / 34 (29.41%)
    11 / 35 (31.43%)
         occurrences all number
    12
    12
    ORAL DISORDER
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 35 (5.71%)
         occurrences all number
    0
    2
    ORAL PAIN
         subjects affected / exposed
    3 / 34 (8.82%)
    1 / 35 (2.86%)
         occurrences all number
    3
    1
    VOMITING
         subjects affected / exposed
    12 / 34 (35.29%)
    7 / 35 (20.00%)
         occurrences all number
    13
    8
    Skin and subcutaneous tissue disorders
    ALOPECIA
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 35 (5.71%)
         occurrences all number
    0
    2
    NIGHT SWEATS
         subjects affected / exposed
    2 / 34 (5.88%)
    0 / 35 (0.00%)
         occurrences all number
    3
    0
    PETECHIAE
         subjects affected / exposed
    6 / 34 (17.65%)
    1 / 35 (2.86%)
         occurrences all number
    8
    1
    RASH
         subjects affected / exposed
    2 / 34 (5.88%)
    4 / 35 (11.43%)
         occurrences all number
    3
    4
    SKIN LESION
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 35 (5.71%)
         occurrences all number
    0
    2
    Renal and urinary disorders
    DYSURIA
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 35 (5.71%)
         occurrences all number
    0
    2
    Musculoskeletal and connective tissue disorders
    BACK PAIN
         subjects affected / exposed
    2 / 34 (5.88%)
    3 / 35 (8.57%)
         occurrences all number
    3
    3
    BONE PAIN
         subjects affected / exposed
    2 / 34 (5.88%)
    2 / 35 (5.71%)
         occurrences all number
    2
    2
    MUSCLE SPASMS
         subjects affected / exposed
    5 / 34 (14.71%)
    8 / 35 (22.86%)
         occurrences all number
    5
    9
    MUSCULAR WEAKNESS
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 35 (2.86%)
         occurrences all number
    2
    1
    MUSCULOSKELETAL PAIN
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 35 (2.86%)
         occurrences all number
    3
    1
    MYALGIA
         subjects affected / exposed
    8 / 34 (23.53%)
    4 / 35 (11.43%)
         occurrences all number
    9
    5
    MYOPATHY
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 35 (5.71%)
         occurrences all number
    0
    2
    PAIN IN EXTREMITY
         subjects affected / exposed
    4 / 34 (11.76%)
    1 / 35 (2.86%)
         occurrences all number
    4
    1
    Infections and infestations
    INFECTION
         subjects affected / exposed
    2 / 34 (5.88%)
    0 / 35 (0.00%)
         occurrences all number
    2
    0
    NASOPHARYNGITIS
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 35 (2.86%)
         occurrences all number
    2
    1
    ORAL HERPES
         subjects affected / exposed
    1 / 34 (2.94%)
    2 / 35 (5.71%)
         occurrences all number
    1
    2
    SKIN INFECTION
         subjects affected / exposed
    1 / 34 (2.94%)
    2 / 35 (5.71%)
         occurrences all number
    1
    2
    UPPER RESPIRATORY TRACT INFECTION
         subjects affected / exposed
    3 / 34 (8.82%)
    0 / 35 (0.00%)
         occurrences all number
    3
    0
    URINARY TRACT INFECTION
         subjects affected / exposed
    2 / 34 (5.88%)
    1 / 35 (2.86%)
         occurrences all number
    2
    2
    Metabolism and nutrition disorders
    DECREASED APPETITE
         subjects affected / exposed
    5 / 34 (14.71%)
    7 / 35 (20.00%)
         occurrences all number
    7
    7
    HYPERKALAEMIA
         subjects affected / exposed
    3 / 34 (8.82%)
    0 / 35 (0.00%)
         occurrences all number
    3
    0
    HYPOALBUMINAEMIA
         subjects affected / exposed
    0 / 34 (0.00%)
    2 / 35 (5.71%)
         occurrences all number
    0
    2
    HYPOCALCAEMIA
         subjects affected / exposed
    0 / 34 (0.00%)
    3 / 35 (8.57%)
         occurrences all number
    0
    4
    HYPOKALAEMIA
         subjects affected / exposed
    6 / 34 (17.65%)
    4 / 35 (11.43%)
         occurrences all number
    7
    4
    HYPOMAGNESAEMIA
         subjects affected / exposed
    2 / 34 (5.88%)
    0 / 35 (0.00%)
         occurrences all number
    3
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    02 Dec 2013
    This amendment included the following changes in the protocol: Updated the eligibility criteria to add the exclusion of patients who are potentially eligible; for salvage allogenic stem cell transplant; a provision to stop recruitment in the study during the conduct of IA was added; a provision to keep the clinical trial team blinded was removed; PFS was replaced with event-free survival to be consistent with the International Working Group for AML.
    07 Jul 2014
    This amendment included the following changes in the protocol: Deletion of the post-treatment follow-up, and survival follow-up from the study design; addition that bone marrow assessment for patients, who remain in the study after IA, were performed every other month until the discontinuation of sonidegib; revision that the final analysis of the study will be performed after all patients who are on the study have either been placed in a roll-over protocol or discontinued treatment due to death, disease progression/treatment failure, withdrawal of consent or lost to follow-up.

    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.
    Only 34 of the 35 patients in the LDE225 800 mg once daily arm received at least dose of study drug were analyzed for safety.
    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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