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    Clinical Trial Results:
    Multi-Center Phase II Study with Pomalidomide in Patients with Myeloproliferative Neoplasms in Fibrotic Stage

    Summary
    EudraCT number
    2009-010738-23
    Trial protocol
    DE  
    Global end of trial date
    14 Sep 2016

    Results information
    Results version number
    v1(current)
    This version publication date
    04 Mar 2021
    First version publication date
    04 Mar 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    MPN-SG 01-09
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT00949364
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Universitätsklinikum Ulm
    Sponsor organisation address
    Albert-Einstein-Allee 23, Ulm, Germany, 89081
    Public contact
    Innere Medizin III, Studienzentrale, Universitätsklinikum Ulm, +49 731500 45901, frank.stegelmann@uniklinik-ulm.de
    Scientific contact
    Innere Medizin III, Studienzentrale, Universitätsklinikum Ulm, +49 731500 45901, frank.stegelmann@uniklinik-ulm.de
    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
    13 May 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    14 Sep 2016
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Sep 2016
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    • To evaluate clinical efficacy (disease response) of pomalidomide in MF patients based on the consensus criteria of the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT), extended by the criterion RBC-transfusion independence (TI)
    Protection of trial subjects
    In this study, safety was assessed by evaluating the following: reported adverse events, clinical laboratory test results, vital signs measurements, ECG findings, chest X-ray, sonographic assessment of the Spleen, physical examination findings, monitoring of concomitant therapy. For each safety parameter, all findings (whether normal or abnormal) were recorded in the CRF.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    03 Dec 2009
    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
    Germany: 103
    Worldwide total number of subjects
    103
    EEA total number of subjects
    103
    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)
    16
    From 65 to 84 years
    86
    85 years and over
    1

    Subject disposition

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    Recruitment
    Recruitment details
    First Patient in: 03.12.2009 Last Patient in: 23.04.2013 Last Patient out: 14.09.2016 After the enrolment and Treatment of 38 patients recruitment was interrupted in September 2010 until approval of teh amended protocol Version 2.0 (26.06.2011). Recruitment was restarted in October 2011.

    Pre-assignment
    Screening details
    Diagnosis of myeloproliferative neoplasms (de novo, secondary, or unclassifiable) with biopsy proven MF; Anemia with hemoglobin <10g/dl or Transfusion-dependent anemia or thrombocytopenia <50 G/l or Transfusion-dependent thrombocytopenia

    Pre-assignment period milestones
    Number of subjects started
    103
    Number of subjects completed
    96

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    Protocol deviation: 6
    Reason: Number of subjects
    Consent withdrawn by subject: 1
    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Cohort 1
    Arm description
    Treatment started with a Phase of pomalidomide with 2 mg per day. Individual dose reduction as outlined in the safety section was allowed. If no Response was achieved after 3 months, prednisolone was added in a starting dose of 30 mg per day. In the Absence of progressive disease, at least 6 months of Treatment with pomalidomide was intended. In patietns without disease Progression after 6 months and those with Response to Treatment were intended to receive pomalidomide for at least 12 months.
    Arm type
    Experimental

    Investigational medicinal product name
    Pomalidomide
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    1 Capsule of 2 mg per day for 12 months

    Investigational medicinal product name
    Prednisolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    If no complete Response, partial Response or clinical improvement occurred after 3 cycles, prednisolone is added to pomalidomide in a starting dose of 30 mg/day for 28 days followed by 15 mg/day and 10 mg/day for 28 days, respectively, consequently thereafter. In case of progressive disease Treatment ist stopped. Otherwise, continous Treatment at least until end of cycle 12 is intended. For patients responding to the combination Treatment a concomitant Treatment after cycle 6 with prednisolone in doses equal or below 7.5 mg/day are allowed.

    Arm title
    Cohort 2: 3-month prednisolone
    Arm description
    Treatment for all patients starts with pomalidomide as single Agent at a dose of 0.5 mg/day. The Addition of prednisolone will be initiated as randomized at start of cycle 4 (starting dose 30 mg/day). In the Absence of progressive disease, at least 12 cycles of Treatment with pomalidomide are intended.
    Arm type
    Experimental

    Investigational medicinal product name
    Pomalidomide
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    1 Capsule of 0.5 mg per day for 12 months

    Investigational medicinal product name
    Prednisolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Treatment starts with pomalidomide as a single agent therapy with 0.5 mg/day. Prednisolone will be started in the absence of progressive disease as randomized as start of cycle 4 in a starting dose of 30 mg/day for 28 days followed by 15 mg/day and 10 mg/day for 28 days, respectively, consequently thereafter, if no response was achieved. In case of progressive disease Treatment ist stopped. Otherwise, continuous Treatment at least until end of cycle 12 is intended. For patients responding to the combination Treatment a concomitant Treatment after cycle 6 with prednisolone in doses equal or below 7.5 mg/day are allowed.

    Arm title
    Cohort 2: 6-month prednisolone
    Arm description
    Treatment for all patients starts with pomalidomide as single Agent at a dose of 0.5 mg/day. The Addition of prednisolone will be initiated as randomized at start of cycle 7 (starting dose 30 mg/day). In the Absence of progressive disease, at least 12 cycles of Treatment with pomalidomide are intended
    Arm type
    Experimental

    Investigational medicinal product name
    Pomalidomide
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    1 Capsule of 0.5 mg per day for 12 months

    Investigational medicinal product name
    Prednisolone
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Treatment starts with pomalidomide as a single agent therapy with 0.5 mg/day. Prednisolone will be started in the absence of progressive disease as randomized at start of cycle 7 in a starting dose of 30 mg/day for 28 days followed by 15 mg/day and 10 mg/day for 28 days, respectively, consequently thereafter, if no response was achieved. In case of progressive disease Treatment ist stopped. Otherwise, continuous Treatment at least until end of cycle 12 is intended. For patients responding to the combination Treatment a concomitant Treatment after cycle 9 with prednisolone in doses equal or below 7.5 mg/day are allowed.

    Number of subjects in period 1 [1]
    Cohort 1 Cohort 2: 3-month prednisolone Cohort 2: 6-month prednisolone
    Started
    38
    27
    31
    Completed
    19
    7
    7
    Not completed
    19
    20
    24
         Lack of efficacy
    3
    7
    5
         Non-compliance
    1
    -
    -
         Adverse event, serious fatal
    -
    4
    3
         Adverse event, non-fatal
    14
    1
    9
         Consent withdrawn by subject
    1
    8
    5
         allo-HSCT
    -
    -
    2
    Notes
    [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: 103 patients were registered into the study. Cohort1: 3 patients were not eligible and did not enter the Treatment Phase. Cohort2: 3 Patient were not eligible and 1 patient withdrew his consent and did not enter the Treatment Phase. This was before the randomization to "Cohort2: 3-month prednisolone" and "Cohort2: 6-month prednisolone" happened.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    Treatment started with a Phase of pomalidomide with 2 mg per day. Individual dose reduction as outlined in the safety section was allowed. If no Response was achieved after 3 months, prednisolone was added in a starting dose of 30 mg per day. In the Absence of progressive disease, at least 6 months of Treatment with pomalidomide was intended. In patietns without disease Progression after 6 months and those with Response to Treatment were intended to receive pomalidomide for at least 12 months.

    Reporting group title
    Cohort 2: 3-month prednisolone
    Reporting group description
    Treatment for all patients starts with pomalidomide as single Agent at a dose of 0.5 mg/day. The Addition of prednisolone will be initiated as randomized at start of cycle 4 (starting dose 30 mg/day). In the Absence of progressive disease, at least 12 cycles of Treatment with pomalidomide are intended.

    Reporting group title
    Cohort 2: 6-month prednisolone
    Reporting group description
    Treatment for all patients starts with pomalidomide as single Agent at a dose of 0.5 mg/day. The Addition of prednisolone will be initiated as randomized at start of cycle 7 (starting dose 30 mg/day). In the Absence of progressive disease, at least 12 cycles of Treatment with pomalidomide are intended

    Reporting group values
    Cohort 1 Cohort 2: 3-month prednisolone Cohort 2: 6-month prednisolone Total
    Number of subjects
    38 27 31 96
    Age categorical
    Units: Subjects
        In utero
    0
        Preterm newborn infants (gestational age < 37 wks)
    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)
    0
        From 65-84 years
    0
        85 years and over
    0
    Age continuous
    Units: years
        median (full range (min-max))
    70.6 (50 to 82) 73 (49 to 85) 71.4 (58 to 80) -
    Gender categorical
    Units: Subjects
        Female
    13 10 10 33
        Male
    25 17 21 63
    Type of Myelofibrosis
    Units: Subjects
        PMF
    26 21 23 70
        post ET-MF
    3 2 2 7
        post PV-MF
    8 2 5 15
        unclassified MF
    1 2 1 4
    DIPPS
    Units: Subjects
        High
    10 4 9 23
        Intermediate-2
    23 19 20 62
        Intermediate-1
    5 4 2 11
    Constitutional symptoms
    defined as fever, sweating or weight loss
    Units: Subjects
        present
    8 8 10 26
        not present
    30 19 21 70
    ECOG
    Units: Subjects
        ECOG 0
    17 12 7 36
        ECOG 1
    20 13 22 55
        ECOG 2
    1 2 2 5
    RBC transfusion dependent
    Units: Subjects
        yes
    28 22 23 73
        no
    10 5 8 23
    Platelet transfusion dependent
    Units: Subjects
        yes
    7 5 6 18
        no
    31 22 25 78
    Cytogenetics
    Units: Subjects
        Low-Risk
    24 12 19 55
        High-Risk
    9 4 4 17
        Missing
    5 11 8 24
    JAK2V617F Mutation
    Is a Jak2V617F Mutation available?
    Units: Subjects
        Yes
    1 14 19 34
        No
    37 13 12 62
    CALR mutations
    Is a CALR Mutation available?
    Units: Subjects
        Yes
    5 6 7 18
        No
    33 21 24 78
    MPLW515L Mutation
    Is a MPLW515L Mutation available?
    Units: Subjects
        Yes
    5 1 0 6
        No
    33 26 31 90
    ASXL1 mutation
    Is a ASXL1 Mutation available?
    Units: Subjects
        Yes
    13 6 10 29
        No
    25 21 21 67
    DNMT2A mutation
    Is a DNMT2A Mutation available?
    Units: Subjects
        Yes
    2 3 6 11
        No
    36 24 25 85
    TET2 mutation
    Is a TET2 Mutation available?
    Units: Subjects
        Yes
    4 2 4 10
        No
    34 25 27 86
    EZH2 mutation
    Is a EZH2 Mutation available?
    Units: Subjects
        Yes
    3 1 1 5
        No
    35 26 30 91
    IDH 1/2 mutation
    Is a IDH 1/2 Mutation available?
    Units: Subjects
        Yes
    1 0 0 1
        No
    37 27 31 95
    SRSF2 mutation
    IS a SRSF2 Mutation available?
    Units: Subjects
        Yes
    5 2 3 10
        No
    33 25 28 86
    TP53 mutation
    Is a TP53 Mutation available?
    Units: Subjects
        Yes
    1 0 0 1
        No
    37 27 31 95
    Splenomegaly
    Units: cm
        median (full range (min-max))
    19.6 (10 to 32) 19 (10 to 32) 17.5 (12 to 43) -
    LDH
    Units: U/l
        median (full range (min-max))
    490 (200 to 2376) 612 (156 to 1599) 544 (153 to 1136) -
    Hemoglobin
    Units: g/dl
        median (full range (min-max))
    9.4 (4.8 to 10.9) 8.5 (5.8 to 13.7) 9.0 (5.5 to 12.8) -
    Platelets
    Units: G/l
        median (full range (min-max))
    122.5 (11 to 1400) 139 (3 to 716) 91 (12 to 394) -
    Subject analysis sets

    Subject analysis set title
    Full analysis
    Subject analysis set type
    Intention-to-treat
    Subject analysis set description
    Includes all patients who received the study medication at least once

    Subject analysis sets values
    Full analysis
    Number of subjects
    96
    Age categorical
    Units: Subjects
        In utero
        Preterm newborn infants (gestational age < 37 wks)
        Newborns (0-27 days)
        Infants and toddlers (28 days-23 months)
        Children (2-11 years)
        Adolescents (12-17 years)
        Adults (18-64 years)
        From 65-84 years
        85 years and over
    Age continuous
    Units: years
        median (full range (min-max))
    71.5 (49 to 85)
    Gender categorical
    Units: Subjects
        Female
    33
        Male
    63
    Type of Myelofibrosis
    Units: Subjects
        PMF
    70
        post ET-MF
    7
        post PV-MF
    15
        unclassified MF
    4
    DIPPS
    Units: Subjects
        High
    23
        Intermediate-2
    62
        Intermediate-1
    11
    Constitutional symptoms
    defined as fever, sweating or weight loss
    Units: Subjects
        present
    26
        not present
    70
    ECOG
    Units: Subjects
        ECOG 0
    36
        ECOG 1
    55
        ECOG 2
    5
    RBC transfusion dependent
    Units: Subjects
        yes
    73
        no
    23
    Platelet transfusion dependent
    Units: Subjects
        yes
    18
        no
    78
    Cytogenetics
    Units: Subjects
        Low-Risk
    55
        High-Risk
    17
        Missing
    24
    JAK2V617F Mutation
    Is a Jak2V617F Mutation available?
    Units: Subjects
        Yes
    34
        No
    62
    CALR mutations
    Is a CALR Mutation available?
    Units: Subjects
        Yes
    18
        No
    78
    MPLW515L Mutation
    Is a MPLW515L Mutation available?
    Units: Subjects
        Yes
    6
        No
    90
    ASXL1 mutation
    Is a ASXL1 Mutation available?
    Units: Subjects
        Yes
    29
        No
    67
    DNMT2A mutation
    Is a DNMT2A Mutation available?
    Units: Subjects
        Yes
    11
        No
    85
    TET2 mutation
    Is a TET2 Mutation available?
    Units: Subjects
        Yes
    10
        No
    86
    EZH2 mutation
    Is a EZH2 Mutation available?
    Units: Subjects
        Yes
    5
        No
    91
    IDH 1/2 mutation
    Is a IDH 1/2 Mutation available?
    Units: Subjects
        Yes
    1
        No
    95
    SRSF2 mutation
    IS a SRSF2 Mutation available?
    Units: Subjects
        Yes
    10
        No
    86
    TP53 mutation
    Is a TP53 Mutation available?
    Units: Subjects
        Yes
    1
        No
    95
    Splenomegaly
    Units: cm
        median (full range (min-max))
    18.6 (10 to 43)
    LDH
    Units: U/l
        median (full range (min-max))
    542 (153 to 2376)
    Hemoglobin
    Units: g/dl
        median (full range (min-max))
    9.05 (4.8 to 13.7)
    Platelets
    Units: G/l
        median (full range (min-max))
    103.5 (3 to 1400)

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    Treatment started with a Phase of pomalidomide with 2 mg per day. Individual dose reduction as outlined in the safety section was allowed. If no Response was achieved after 3 months, prednisolone was added in a starting dose of 30 mg per day. In the Absence of progressive disease, at least 6 months of Treatment with pomalidomide was intended. In patietns without disease Progression after 6 months and those with Response to Treatment were intended to receive pomalidomide for at least 12 months.

    Reporting group title
    Cohort 2: 3-month prednisolone
    Reporting group description
    Treatment for all patients starts with pomalidomide as single Agent at a dose of 0.5 mg/day. The Addition of prednisolone will be initiated as randomized at start of cycle 4 (starting dose 30 mg/day). In the Absence of progressive disease, at least 12 cycles of Treatment with pomalidomide are intended.

    Reporting group title
    Cohort 2: 6-month prednisolone
    Reporting group description
    Treatment for all patients starts with pomalidomide as single Agent at a dose of 0.5 mg/day. The Addition of prednisolone will be initiated as randomized at start of cycle 7 (starting dose 30 mg/day). In the Absence of progressive disease, at least 12 cycles of Treatment with pomalidomide are intended

    Subject analysis set title
    Full analysis
    Subject analysis set type
    Intention-to-treat
    Subject analysis set description
    Includes all patients who received the study medication at least once

    Primary: Disease response

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    End point title
    Disease response
    End point description
    Objective disease response as defined by the IWG-MRT for response in MF patients, extended by the criterion RBC-Transfusion dependence (TI).
    End point type
    Primary
    End point timeframe
    The end point disease response was examined at the end of each cycle until at least cycle 12.
    End point values
    Cohort 1 Cohort 2: 3-month prednisolone Cohort 2: 6-month prednisolone
    Number of subjects analysed
    38
    26
    30
    Units: Number of subjects
        Complete remission
    0
    0
    0
        Partial remission
    1
    1
    2
        Clinical improvement
    11
    2
    2
        RBC Transfusion dependence
    3
    4
    3
        No change
    23
    19
    23
    Statistical analysis title
    Logistic regression model
    Comparison groups
    Cohort 1 v Cohort 2: 3-month prednisolone v Cohort 2: 6-month prednisolone
    Number of subjects included in analysis
    94
    Analysis specification
    Pre-specified
    Analysis type
    superiority [1]
    P-value
    = 0.05
    Method
    Regression, Cox
    Parameter type
    Odds ratio (OR)
    Point estimate
    2.62
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1
         upper limit
    6.87
    Variability estimate
    Standard deviation
    Notes
    [1] - The superiority was supported by the results of a logistic regression model, which revealed that a higher dose of pomaldomide of 2.0 mg/day instead of 0.5 mg/day as significantly associated with a higher Response rate (OR, 2.62; 95% CI, 1.00 - 6.87).

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Timeframe for reporting adverse events: Adverse Events were reported from Informed Consent signature up to 28 days after last study drug administration or until all drug-related toxicities had been resolved, whichever was later.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    CTCAE
    Dictionary version
    3.0
    Reporting groups
    Reporting group title
    Study population
    Reporting group description
    Includes cohort 1 and cohort 2

    Serious adverse events
    Study population
    Total subjects affected by serious adverse events
         subjects affected / exposed
    75 / 96 (78.13%)
         number of deaths (all causes)
    19
         number of deaths resulting from adverse events
    19
    Vascular disorders
    Syncope
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Epistaxis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hypertensive crisis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pulmonary embolism
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Deep vein thrombosis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    arterial stenosis limb
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Haematoma
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Surgical and medical procedures
    Transurethral prostatectomy
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Prostatic operation
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Arterial bypass operation
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Toe amputation
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Acute myeloid leukaemia
         subjects affected / exposed
    12 / 96 (12.50%)
         occurrences causally related to treatment / all
    0 / 12
         deaths causally related to treatment / all
    0 / 3
    Bladder cancer
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Bronchial carcinoma
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Breast cancer female
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Chloroma
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal cell carcinoma
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Langerhans' cell histiocytosis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Immune system disorders
    Drug hypersensitivity
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    Asthenia
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General physical health deterioration
         subjects affected / exposed
    3 / 96 (3.13%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Condition aggravated
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Wound hemorrhage
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Investigations
    Blood glucose increased
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Cardiac failure
         subjects affected / exposed
    4 / 96 (4.17%)
         occurrences causally related to treatment / all
    1 / 5
         deaths causally related to treatment / all
    0 / 0
    Supraventricular tachycardia
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Atrial fibrillation
         subjects affected / exposed
    5 / 96 (5.21%)
         occurrences causally related to treatment / all
    0 / 6
         deaths causally related to treatment / all
    0 / 0
    Arrhythmia
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Bradyarrhythmia
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Left ventricular dysfunction
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Myocardial infarction
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Tachyarrhythmia
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pericardial effusion
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Lung infiltration
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Bronchitis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Pulmonary hypertension
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pleural effusion
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pulmonary embolism
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Acute myeloid leukaemia
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 1
    Anaemia
         subjects affected / exposed
    4 / 96 (4.17%)
         occurrences causally related to treatment / all
    1 / 4
         deaths causally related to treatment / all
    0 / 0
    Febrile neutropenia
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Leukopenia
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Neutropenia
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Haemolysis
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Polycythaemia
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    3 / 96 (3.13%)
         occurrences causally related to treatment / all
    1 / 3
         deaths causally related to treatment / all
    0 / 0
    Transfusion reaction
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Cerebral haemorrhage
         subjects affected / exposed
    3 / 96 (3.13%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 2
    Gastrointestinal disorders
    Ascites
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Enterocolitis haemorrhagic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Gastritis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Inguinal hernia
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal pain
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Diverticular perforation
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal perforation
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Dysphagia
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Renal disorder
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Oedema due to renal disease
         subjects affected / exposed
    3 / 96 (3.13%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Renal failure
         subjects affected / exposed
    3 / 96 (3.13%)
         occurrences causally related to treatment / all
    1 / 3
         deaths causally related to treatment / all
    0 / 1
    Renal colic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin and subcutaneous tissue disorders
    Erysipelas
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    rash
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Intervertebral disc protrusion
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Myalgia
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Hepatic steatosis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hypokalaemia
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    1 / 1
    Infections and infestations
    Bacterial prostatitis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Bronchopneumonia
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Chronic sinusitis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Clostridium colitis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cystitis
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Erysipelas
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Febrile infection
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Gastroenteritis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infection
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal infection
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Lung infection
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Otitis media acute
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pleural Infection
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Paronychia
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    10 / 96 (10.42%)
         occurrences causally related to treatment / all
    1 / 12
         deaths causally related to treatment / all
    0 / 3
    Sinusitis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pulmonary sepsis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Sepsis
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 2
    Septic shock
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Urosepsis
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Study population
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    94 / 96 (97.92%)
    Vascular disorders
    Hematoma
         subjects affected / exposed
    9 / 96 (9.38%)
         occurrences all number
    9
    Hemorrhage pulmonary - Select
         subjects affected / exposed
    13 / 96 (13.54%)
         occurrences all number
    13
    Petechiae
         subjects affected / exposed
    7 / 96 (7.29%)
         occurrences all number
    8
    Edema: limb
         subjects affected / exposed
    12 / 96 (12.50%)
         occurrences all number
    13
    Investigations
    Bilirubin
         subjects affected / exposed
    5 / 96 (5.21%)
         occurrences all number
    5
    Hyperuricemia
         subjects affected / exposed
    7 / 96 (7.29%)
         occurrences all number
    7
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    6 / 96 (6.25%)
         occurrences all number
    6
    Hypertension
         subjects affected / exposed
    11 / 96 (11.46%)
         occurrences all number
    11
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    15 / 96 (15.63%)
         occurrences all number
    18
    Dyspnea
         subjects affected / exposed
    30 / 96 (31.25%)
         occurrences all number
    32
    Pulmonary - Other
         subjects affected / exposed
    6 / 96 (6.25%)
         occurrences all number
    6
    Blood and lymphatic system disorders
    Blood - Other
         subjects affected / exposed
    8 / 96 (8.33%)
         occurrences all number
    9
    Hemoglobin
         subjects affected / exposed
    25 / 96 (26.04%)
         occurrences all number
    25
    Leukocytes
         subjects affected / exposed
    8 / 96 (8.33%)
         occurrences all number
    8
    Neutrophils
         subjects affected / exposed
    13 / 96 (13.54%)
         occurrences all number
    13
    Platelets
         subjects affected / exposed
    26 / 96 (27.08%)
         occurrences all number
    27
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    18 / 96 (18.75%)
         occurrences all number
    20
    Neurology - Other
         subjects affected / exposed
    9 / 96 (9.38%)
         occurrences all number
    9
    Neuropathy - sensory
         subjects affected / exposed
    5 / 96 (5.21%)
         occurrences all number
    5
    General disorders and administration site conditions
    Constitutional symptoms - Other
         subjects affected / exposed
    15 / 96 (15.63%)
         occurrences all number
    15
    Fatigue
         subjects affected / exposed
    40 / 96 (41.67%)
         occurrences all number
    44
    Fever
         subjects affected / exposed
    10 / 96 (10.42%)
         occurrences all number
    10
    Insomnia
         subjects affected / exposed
    8 / 96 (8.33%)
         occurrences all number
    8
    Sweating
         subjects affected / exposed
    15 / 96 (15.63%)
         occurrences all number
    15
    Weight loss
         subjects affected / exposed
    7 / 96 (7.29%)
         occurrences all number
    7
    Pain Gastrointestinal: Abdomen NOS
         subjects affected / exposed
    18 / 96 (18.75%)
         occurrences all number
    23
    Pain Musculoskeletal: Bone
         subjects affected / exposed
    5 / 96 (5.21%)
         occurrences all number
    5
    Pain Musculoskeletal: Extremity-limb
         subjects affected / exposed
    6 / 96 (6.25%)
         occurrences all number
    6
    Pain Musculoskeletal: Joint
         subjects affected / exposed
    7 / 96 (7.29%)
         occurrences all number
    7
    Pain Musculoskeletal: Muscle
         subjects affected / exposed
    12 / 96 (12.50%)
         occurrences all number
    15
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    13 / 96 (13.54%)
         occurrences all number
    15
    Diarrhea
         subjects affected / exposed
    19 / 96 (19.79%)
         occurrences all number
    22
    Nausea
         subjects affected / exposed
    10 / 96 (10.42%)
         occurrences all number
    10
    Renal and urinary disorders
    Renal - Other
         subjects affected / exposed
    26 / 96 (27.08%)
         occurrences all number
    27
    Skin and subcutaneous tissue disorders
    Dermatology - Other
         subjects affected / exposed
    19 / 96 (19.79%)
         occurrences all number
    23
    Pruritus
         subjects affected / exposed
    11 / 96 (11.46%)
         occurrences all number
    11
    Rash
         subjects affected / exposed
    7 / 96 (7.29%)
         occurrences all number
    7
    Infections and infestations
    Infection - Other
         subjects affected / exposed
    17 / 96 (17.71%)
         occurrences all number
    20
    Infection with unknown ANC
         subjects affected / exposed
    5 / 96 (5.21%)
         occurrences all number
    5
    Infection Pulmonary/Upper respiratory: Upper airway NOS
         subjects affected / exposed
    10 / 96 (10.42%)
         occurrences all number
    10

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    02 Aug 2011
    Amendment 1 (dated 02 August 2011) was issued after enrolment of n = 38 patients. The following Major procedural changes were made to the protocol: - Dose reduction of Pomalidomide to 0,5 mg once daily. - Additionally, randomisation into either additional prednisolone after 3 months or 6 months when patients have a stable disease. - Change of sample size to 95 patients

    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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