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    Clinical Trial Results:
    A Two-Part Phase 1/2a, Open-Label, Dose-Escalation Study to Evaluate the Tolerability and Preliminary Antitumour Activity of OPB-111001 in Patients with Advanced Cancers that are Poorly Responsive to Standard Anticancer Treatment

    Summary
    EudraCT number
    2013-001249-15
    Trial protocol
    GB  
    Global end of trial date
    08 Sep 2015

    Results information
    Results version number
    v1(current)
    This version publication date
    30 Sep 2016
    First version publication date
    30 Sep 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    314-12-401
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02042885
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Otsuka Novel Products GmbH
    Sponsor organisation address
    Erika-Mann-Str. 21, Munich, Germany, 80636
    Public contact
    Barbara Eschenbach, Otsuka Novel Products GmbH, +49 89-2060205-81, beschenbach@Otsuka-onpg.com
    Scientific contact
    Norbert Hittel, Otsuka Novel Products GmbH, +49 89-2060205-40, nhittel@Otsuka-onpg.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 Feb 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    03 Feb 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    08 Sep 2015
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To investigate the tolerability profile of OPB-111001 and to establish the maximum-tolerated dose (MTD), recommended phase 2 dose (RP2D), or both in patients with cancer of the prostate, ovary, cervix, endometrium, breast, or salivary gland.
    Protection of trial subjects
    This trial was conducted in compliance with International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines for conducting, recording, and reporting trials, as well as for archiving essential documents. Consistent with ethical principles for the protection of human research participants, no trial procedures were performed on trial participants until written consent had been obtained from them. The informed consent form (ICF), protocol, and amendments for this trial were submitted to and approved by the institutional review board (IRB) or independent ethics committee (IEC) for each respective trial site or country.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    22 Jan 2014
    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
    United Kingdom: 8
    Worldwide total number of subjects
    8
    EEA total number of subjects
    8
    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)
    2
    From 65 to 84 years
    6
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The trial was conducted in 8 participants at 2 trial sites in 1 country.

    Pre-assignment
    Screening details
    Participants had screening evaluations between Day -1 and -14 before entering the first 14-day treatment cycle.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    Not blinded
    Blinding implementation details
    This was an open-label study.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    IPDE Cohort 2 (OPB-111001 5mg)
    Arm description
    Participants in intra-patient dose escalation (IPDE) cohort 2 had received 5mg OPB-111001 orally per week.
    Arm type
    Experimental

    Investigational medicinal product name
    OPB-111001 5mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    During each cycle, participants received orally 5mg OPB-111001 per week.

    Arm title
    DE Cohort 2 (OPB-111001 10mg)
    Arm description
    Participants in dose escalation cohort 2 received 10mg OPB-111001 orally per week.
    Arm type
    Experimental

    Investigational medicinal product name
    OPB-111001 10mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    During each cycle, participants received orally 10mg OPB-111001 per week.

    Arm title
    IPDE Cohort 1 and DE Cohort 3 (OPB-111001 25 mg)
    Arm description
    Participants in IPDE cohort 1 and dose escalation Cohort 3 had received 25 mg OPB-111001 orally per week.
    Arm type
    Experimental

    Investigational medicinal product name
    OPB-111001 25mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    During each cycle, participants had received orally 25mg OPB-111001 per week.

    Number of subjects in period 1
    IPDE Cohort 2 (OPB-111001 5mg) DE Cohort 2 (OPB-111001 10mg) IPDE Cohort 1 and DE Cohort 3 (OPB-111001 25 mg)
    Started
    2
    3
    3
    Completed
    0
    0
    0
    Not completed
    2
    3
    3
         Terminated by Sponsor
    -
    1
    -
         Progressive Disease
    2
    2
    1
         Adverse Events
    -
    -
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    IPDE Cohort 2 (OPB-111001 5mg)
    Reporting group description
    Participants in intra-patient dose escalation (IPDE) cohort 2 had received 5mg OPB-111001 orally per week.

    Reporting group title
    DE Cohort 2 (OPB-111001 10mg)
    Reporting group description
    Participants in dose escalation cohort 2 received 10mg OPB-111001 orally per week.

    Reporting group title
    IPDE Cohort 1 and DE Cohort 3 (OPB-111001 25 mg)
    Reporting group description
    Participants in IPDE cohort 1 and dose escalation Cohort 3 had received 25 mg OPB-111001 orally per week.

    Reporting group values
    IPDE Cohort 2 (OPB-111001 5mg) DE Cohort 2 (OPB-111001 10mg) IPDE Cohort 1 and DE Cohort 3 (OPB-111001 25 mg) Total
    Number of subjects
    2 3 3 8
    Age categorical
    Units: Subjects
        In utero
    0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0 0 0
        Newborns (0-27 days)
    0 0 0 0
        Infants and toddlers (28 days-23 months)
    0 0 0 0
        Children (2-11 years)
    0 0 0 0
        Adolescents (12-17 years)
    0 0 0 0
        Adults (18-64 years)
    0 0 2 2
        From 65-84 years
    2 3 1 6
        85 years and over
    0 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    66.5 ± 0.7071 75.333 ± 2.0817 50.333 ± 20.2073 -
    Gender categorical
    Units: Subjects
        Female
    0 0 2 2
        Male
    2 3 1 6

    End points

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    End points reporting groups
    Reporting group title
    IPDE Cohort 2 (OPB-111001 5mg)
    Reporting group description
    Participants in intra-patient dose escalation (IPDE) cohort 2 had received 5mg OPB-111001 orally per week.

    Reporting group title
    DE Cohort 2 (OPB-111001 10mg)
    Reporting group description
    Participants in dose escalation cohort 2 received 10mg OPB-111001 orally per week.

    Reporting group title
    IPDE Cohort 1 and DE Cohort 3 (OPB-111001 25 mg)
    Reporting group description
    Participants in IPDE cohort 1 and dose escalation Cohort 3 had received 25 mg OPB-111001 orally per week.

    Primary: Determination of MTD or RP2D and Tolerability of OPB-111001 as defined by incidence of Adverse Events (AEs), changes from baseline in vital signs, clinical laboratory and electrocardiography assessments.

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    End point title
    Determination of MTD or RP2D and Tolerability of OPB-111001 as defined by incidence of Adverse Events (AEs), changes from baseline in vital signs, clinical laboratory and electrocardiography assessments. [1]
    End point description
    The primary safety endpoint was to determine the MTD or RP2D in the phase 1 with all safety data collected and in the phase 2a to evaluate the confirmatory tolerability of the MTD/RP2D of OPB-111001. All dose-limiting toxicity (DLTs) was entered as AEs. To support the determination of the MTD and/or RP2D adverse events analyses were presented that include only AEs. Treatment-emergent AEs (TEAEs) were defined as AEs occurring or worsening after the start of the first study treatment and up to 30 days after the last study treatment including all treatment cycles. AEs that started before start of study treatment were also considered TEAEs if any of the following conditions applied: Relationship of AE was reported as possibly related, probably related or related; The AE was serious; The AE led to discontinuation, interruption or dose reduction of study treatment; 4. the outcome of the AE was ‘Death’.
    End point type
    Primary
    End point timeframe
    MTD/RP2D - dose escalation part: After 1 or 3 cycles of treatment, determination of the MDR or RP2D and the tolerability of OPB-111001: continuously.
    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 inferential statistical analysis was performed for this primary safety endpoint.
    End point values
    IPDE Cohort 2 (OPB-111001 5mg) DE Cohort 2 (OPB-111001 10mg) IPDE Cohort 1 and DE Cohort 3 (OPB-111001 25 mg)
    Number of subjects analysed
    2
    3
    3
    Units: Number of participants
    number (not applicable)
        Any TEAE
    2
    3
    3
        TEAE related to OPB-111001
    1
    3
    3
        TEAE with an outcome of death
    0
    0
    0
        Serious TEAE
    1
    0
    2
        TEAE leading to discontinuation of study drug
    0
    0
    2
        DLT
    0
    0
    2
    No statistical analyses for this end point

    Secondary: Assessment of antitumor activity as defined by Response Evaluation Criteria in Solid Tumours (RECIST)

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    End point title
    Assessment of antitumor activity as defined by Response Evaluation Criteria in Solid Tumours (RECIST)
    End point description
    Assessment of antitumor activity as defined by RECIST, Version 1.1: complete response(CR), partial response (PR), stable disease (SD), objective response rate(ORR) (CR + PR). The objective tumor response was classified as CR, PR, SD, PD, Non-CR/Non-PD or NE according to RECIST 1.1 and was determined every 4 cycles.
    End point type
    Secondary
    End point timeframe
    Repeatedly every 8th week until end of study (average of 3 months approximately).
    End point values
    IPDE Cohort 2 (OPB-111001 5mg) DE Cohort 2 (OPB-111001 10mg) IPDE Cohort 1 and DE Cohort 3 (OPB-111001 25 mg)
    Number of subjects analysed
    1
    3
    1
    Units: Number of participants
    number (not applicable)
        Prostate (SD)
    0
    2
    0
        Prostate (non-CR/non-PD)
    1
    1
    0
        Cervical PD
    0
    0
    1
    No statistical analyses for this end point

    Secondary: Prostate-specific antigen (PSA) response in patients with prostate cancer.

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    End point title
    Prostate-specific antigen (PSA) response in patients with prostate cancer. [2]
    End point description
    PSA response in patients with prostate cancer: relative change determined according to Prostate Cancer Working Group 2 (PCWG2). For participants with prostate cancer, PSA values were measured as a tumor marker.
    End point type
    Secondary
    End point timeframe
    Repeatedly (Cycle 1 to 3 on Day 1, then every 4th week) until end of study (average of 3 months approximately).
    Notes
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Data for reporting arm IPDE Cohort 1 and DE Cohort 3 (25 mg) was not available due to very small sample size.
    End point values
    IPDE Cohort 2 (OPB-111001 5mg) DE Cohort 2 (OPB-111001 10mg)
    Number of subjects analysed
    2
    3
    Units: ng/mL
    median (full range (min-max))
        Cycle 2 Day 1 (N= 1, 3)
    7.4 (7.4 to 7.4)
    19 (5.7 to 22.8)
        Cycle 3 Day 1 (N= 1, 3)
    25.1 (25.1 to 25.1)
    -0.5 (-2.7 to 24)
        Cycle 5 Day 1 (N= 1, 3)
    42.1 (42.1 to 42.1)
    14.9 (3.8 to 78)
        Cycle 7 Day 1 (N= 1, 3)
    58.1 (58.1 to 58.1)
    55 (15.1 to 181)
        Cycle 9 Day 1 (N= 0, 1)
    0 (0 to 0)
    237 (237 to 237)
        1-week Follow-up (N= 1, 3)
    70.1 (70.1 to 70.1)
    184 (90 to 281)
        End of Study (N= 1, 2)
    77.1 (77.1 to 77.1)
    213 (101 to 325)
    No statistical analyses for this end point

    Secondary: Time to treatment failure (TTF)

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    End point title
    Time to treatment failure (TTF)
    End point description
    TTF was defined as the time from start of treatment with OPB-111001 to discontinuation of OPB-111001 for any reason, including disease progression, treatment toxicity, subject’s withdrawal and death. All participants who withdrew from the study because of any reason were analyzed as non-censored. Only participants who were still on treatment before the trial were stopped and discontinued treatment because of the stop of the trial were analyzed as censored at the date of discontinuation.
    End point type
    Secondary
    End point timeframe
    At end of study (after average of 3 months approximately).
    End point values
    IPDE Cohort 2 (OPB-111001 5mg) DE Cohort 2 (OPB-111001 10mg) IPDE Cohort 1 and DE Cohort 3 (OPB-111001 25 mg)
    Number of subjects analysed
    2
    3
    3
    Units: Days
    median (full range (min-max))
        Prostate (N= 1, 3, 0)
    106 (106 to 106)
    99 (93 to 127)
    0 (0 to 0)
        Cervical (N= 0, 0, 1)
    0 (0 to 0)
    0 (0 to 0)
    40 (40 to 40)
        Total (N= 1, 3, 1)
    106 (106 to 106)
    99 (93 to 127)
    40 (40 to 40)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    TEAEs were defined as AEs occurring or worsening after the start of the first study treatment and up to 30 days after the last treatment including all treatment cycles (an approximate duration of 8.6 weeks).
    Adverse event reporting additional description
    The safety population consisted of all enrolled participants who had received the study medication at least once.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    16.0
    Reporting groups
    Reporting group title
    IPDE Cohort 2 (OPB-111001 5mg)
    Reporting group description
    Participants in IPDE cohort 2 had received 5mg OPB-111001 orally per week.

    Reporting group title
    DE Cohort 2 (OPB-111001 10mg)
    Reporting group description
    Participants in dose escalation cohort 2 received 10mg OPB-111001 orally per week.

    Reporting group title
    IPDE Cohort 1 and DE Cohort 3 (OPB-111001 25 mg)
    Reporting group description
    Participants in IPDE cohort 1 and dose escalation Cohort 3 had received 25 mg OPB-111001 orally per week.

    Serious adverse events
    IPDE Cohort 2 (OPB-111001 5mg) DE Cohort 2 (OPB-111001 10mg) IPDE Cohort 1 and DE Cohort 3 (OPB-111001 25 mg)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    2 / 3 (66.67%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Vascular disorders
    Hypotension
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Investigations
    Troponin I increased
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Atrial flutter
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Neutropenia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vomiting
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    IPDE Cohort 2 (OPB-111001 5mg) DE Cohort 2 (OPB-111001 10mg) IPDE Cohort 1 and DE Cohort 3 (OPB-111001 25 mg)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 2 (100.00%)
    3 / 3 (100.00%)
    3 / 3 (100.00%)
    Vascular disorders
    Hypotension
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    2
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 2 (50.00%)
    2 / 3 (66.67%)
    1 / 3 (33.33%)
         occurrences all number
    2
    2
    1
    Oedema peripheral
         subjects affected / exposed
    2 / 2 (100.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    2
    0
    0
    Pyrexia
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    1
    0
    1
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Reproductive system and breast disorders
    Vaginal haemorrhage
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Investigations
    Blood creatinine increased
         subjects affected / exposed
    0 / 2 (0.00%)
    2 / 3 (66.67%)
    0 / 3 (0.00%)
         occurrences all number
    0
    2
    0
    Oxygen saturation decreased
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    2 / 3 (66.67%)
         occurrences all number
    0
    0
    2
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Neutrophil count decreased
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Troponin I increased
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    White blood cell count decreased
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    2
    0
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    1 / 3 (33.33%)
         occurrences all number
    0
    2
    3
    Tachycardia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    2 / 3 (66.67%)
         occurrences all number
    0
    0
    2
    Atrial flutter
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    2
    Bradyarrhythmia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Palpitations
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Blood and lymphatic system disorders
    Neutropenia
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    2 / 3 (66.67%)
         occurrences all number
    0
    4
    9
    Leukopenia
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    1
    Anaemia
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 2 (50.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    2
    1
    0
    Hiccups
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    2
    Pharyngeal oedema
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Pleural effusion
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    2
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    0 / 2 (0.00%)
    2 / 3 (66.67%)
    0 / 3 (0.00%)
         occurrences all number
    0
    2
    0
    Hypoaesthesia
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Paraesthesia
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Eye disorders
    Periorbital oedema
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    1 / 2 (50.00%)
    1 / 3 (33.33%)
    3 / 3 (100.00%)
         occurrences all number
    1
    1
    4
    Diarrhoea
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    3 / 3 (100.00%)
         occurrences all number
    0
    3
    6
    Vomiting
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    3 / 3 (100.00%)
         occurrences all number
    1
    0
    9
    Abdominal discomfort
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Abdominal pain
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Melaena
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Swollen tongue
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Constipation
         subjects affected / exposed
    1 / 2 (50.00%)
    3 / 3 (100.00%)
    2 / 3 (66.67%)
         occurrences all number
    1
    7
    3
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Nocturia
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Urinary incontinence
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Urinary retention
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Skin and subcutaneous tissue disorders
    Rash maculo-papular
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Urticaria
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    2
    0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    1
    Arthralgia
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Fluid overload
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Hypoalbuminaemia
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Hypokalaemia
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Infections and infestations
    Lung infection
         subjects affected / exposed
    1 / 2 (50.00%)
    2 / 3 (66.67%)
    0 / 3 (0.00%)
         occurrences all number
    1
    2
    0
    Urinary tract infection
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 3 (33.33%)
    1 / 3 (33.33%)
         occurrences all number
    0
    1
    1
    Lower respiratory tract infection
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Oral candidiasis
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Periorbital infection
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? No

    Interruptions (globally)

    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    08 Sep 2015
    During the escalation phase of Regimen A (1 dose per week) with which the study began, 2 DLTs occurred in the lowest dose level described in the protocol; therefore, the study was temporarily put on hold in Jan 2015. Based on Data Monitoring Committee recommendations and evaluation of data, the sponsor decided on 08 September 2015 to prematurely terminate the study. Data interpretation was limited by the very small sample size (enrollment 1/5 of planned sample size; not powered for formal statistical analysis) and the open-label observation.
    -

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    During the escalation phase of Regimen-A, 2 DLTs occurred in the lowest dose level therefore, the study was temporarily put on hold in January 2015. After further evaluation of data, the sponsor decided on 08 September 2015 not to restart the study.
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