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    Clinical Trial Results:
    A Phase-3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study To Compare Efficacy and Safety of Pomalidomide in subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Red Blood Cell-Transfusion-Dependence

    Summary
    EudraCT number
    2010-018965-42
    Trial protocol
    GB   DE   NL   AT   IT   ES   BE   SE   PL  
    Global end of trial date
    14 May 2018

    Results information
    Results version number
    v2(current)
    This version publication date
    13 Jul 2019
    First version publication date
    31 May 2019
    Other versions
    v1
    Version creation reason

    Trial information

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    Trial identification
    Sponsor protocol code
    CC-4047-MF-002
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01178281
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Celgene Corporation
    Sponsor organisation address
    87 Morris Avenue, Summit, United States, 07901
    Public contact
    Clinical Trial Disclosure, Celgene Corporation, 01 888-260-1599, ClinicalTrialDisclosure@Celgene.com
    Scientific contact
    Robert Peter Gale, MD, PhD, Celgene Corporation, 01 908 656 0484, RGale@celgene.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
    14 May 2018
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    14 May 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The objective of this study is to determine whether pomalidomide is safe and effective in reversing red blood cell (RBC)-transfusion-dependence in persons with myeloproliferative neoplasm (MPN)-associated myelofibrosis (global study) and in reversing anemia in Chinese with MPN-associated myelofibrosis and severe anemia not receiving RBC-transfusions (China extension study only).
    Protection of trial subjects
    Protection of Patient Confidentiality, Archiving of Essential Documents
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    07 Sep 2010
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Safety, Efficacy
    Long term follow-up duration
    5 Years
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Australia: 3
    Country: Number of subjects enrolled
    Austria: 9
    Country: Number of subjects enrolled
    Belgium: 11
    Country: Number of subjects enrolled
    Canada: 8
    Country: Number of subjects enrolled
    China: 36
    Country: Number of subjects enrolled
    France: 21
    Country: Number of subjects enrolled
    Germany: 9
    Country: Number of subjects enrolled
    Italy: 36
    Country: Number of subjects enrolled
    Japan: 9
    Country: Number of subjects enrolled
    Netherlands: 7
    Country: Number of subjects enrolled
    Russian Federation: 13
    Country: Number of subjects enrolled
    Spain: 6
    Country: Number of subjects enrolled
    Sweden: 6
    Country: Number of subjects enrolled
    United Kingdom: 18
    Country: Number of subjects enrolled
    United States: 75
    Worldwide total number of subjects
    267
    EEA total number of subjects
    123
    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)
    91
    From 65 to 84 years
    172
    85 years and over
    4

    Subject disposition

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    Recruitment
    Recruitment details
    Participants in the global study were enrolled at 72 clinical centers in 15 countries. In addition, the China-specific extension study enrolled participants with myeloproliferative neoplasm (MPN)-associated myelofibrosis and severe anemia not receiving red blood cell (RBC)-transfusions at 5 sites in China.

    Pre-assignment
    Screening details
    Participants in the global study were randomized 2:1 to receive blinded pomalidomide or placebo. All participants in the China extension received open-label pomalidomide. Randomization was stratified by age (≤vs >65 years), white blood cells (< or ≥25 × 10⁹/L), and baseline transfusion requirement (≤vs >4 units RBC/28 days over the prior 84 days).

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Pomalidomide 0.5 mg
    Arm description
    Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit (defined as a reduction from Baseline of ≥ 50% in RBC-transfusion frequency during the prior 84-day interval) could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.
    Arm type
    Experimental

    Investigational medicinal product name
    Pomalidomide
    Investigational medicinal product code
    CC-4047
    Other name
    Imnovid, Pomalyst
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Pomalidomide 0.5 mg by mouth once daily

    Arm title
    Placebo
    Arm description
    Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo capsules by mouth once daily

    Arm title
    China Extension: Pomalidomide 0.5 mg
    Arm description
    Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion. Participants who experienced anemia response could continue treatment until the response was lost or other criteria for treatment discontinuation applied. Participants enrolled in the China extension study were not blinded.
    Arm type
    Experimental

    Investigational medicinal product name
    Pomalidomide
    Investigational medicinal product code
    CC-4047
    Other name
    Imnovid; Pomalyst
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Pomalidomide 0.5 mg by mouth once daily

    Number of subjects in period 1
    Pomalidomide 0.5 mg Placebo China Extension: Pomalidomide 0.5 mg
    Started
    168
    84
    15
    Received Study Drug
    167 [1]
    83 [2]
    15
    Completed
    168
    84
    15
    Notes
    [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: Completed indicates participants no longer on-study
    [2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: Completed indicates participants no longer on-study

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Pomalidomide 0.5 mg
    Reporting group description
    Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit (defined as a reduction from Baseline of ≥ 50% in RBC-transfusion frequency during the prior 84-day interval) could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.

    Reporting group title
    Placebo
    Reporting group description
    Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.

    Reporting group title
    China Extension: Pomalidomide 0.5 mg
    Reporting group description
    Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion. Participants who experienced anemia response could continue treatment until the response was lost or other criteria for treatment discontinuation applied. Participants enrolled in the China extension study were not blinded.

    Reporting group values
    Pomalidomide 0.5 mg Placebo China Extension: Pomalidomide 0.5 mg Total
    Number of subjects
    168 84 15 267
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    55 26 10 91
        From 65-84 years
    109 58 5 172
        85 years and over
    4 0 0 4
    Age Continuous
    Units: years
        median (full range (min-max))
    69.0 (40.0 to 90.0) 69.0 (44.0 to 81.0) 63.0 (41.0 to 76.0) -
    Gender, Male/Female
    Units: Subjects
        Female
    41 28 6 75
        Male
    127 56 9 192
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    3 1 0 4
        Not Hispanic or Latino
    144 79 15 238
        Unknown or Not Reported
    21 4 0 25
    Race/Ethnicity, Customized
    Units: Subjects
        American Indian or Alaskan Native
    1 0 0 1
        Asian
    20 11 15 46
        Black or African American
    2 3 0 5
        Native Hawaiian or Other Pacific Islanders
    1 0 0 1
        White
    122 66 0 188
        Other
    3 0 0 3
        Missing
    19 4 0 23
    Eastern Cooperative Oncology Group (ECOG) Performance Status
    ECOG performance status is used to describe a patient's level of functioning in terms of their ability to care for themselves, daily activity, and physical ability (walking, working, etc.). The scale ranges from 0 to 5: 0 = Fully active, no restrictions; 1= Restricted activity but ambulatory, able to carry out work of a light nature; 2= Ambulatory and capable of all self-care but unable to carry out work activities; 3= Limited self-care, confined to bed or chair more than 50% of waking hours; 4= Completely disabled, no self-care, confined to bed or chair; 5= Dead
    Units: Subjects
        0 (Fully active)
    53 22 5 80
        1 (Restrictive but ambulatory)
    85 47 9 141
        2 (Ambulatory but unable to work)
    30 15 1 46
        3 (Limited self care)
    0 0 0 0
        4 (Completely disabled)
    0 0 0 0
    Disease sub-type
    Units: Subjects
        Primary myelofibrosis
    127 65 10 202
        Post-polycythemia vera myelofibrosis
    17 8 2 27
        Post-essential thrombocythemia myelofibrosis
    23 11 3 37
        Missing
    1 0 0 1
    Baseline RBC Transfusion Burden
    Defined as the average number of RBC-transfusion-units per 28 days over the 84 days immediately prior to randomization.
    Units: units per 28 days
        median (full range (min-max))
    2.7 (1.3 to 13.3) 2.8 (1.3 to 10.0) 0.0 (0.0 to 0.0) -

    End points

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    End points reporting groups
    Reporting group title
    Pomalidomide 0.5 mg
    Reporting group description
    Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit (defined as a reduction from Baseline of ≥ 50% in RBC-transfusion frequency during the prior 84-day interval) could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.

    Reporting group title
    Placebo
    Reporting group description
    Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.

    Reporting group title
    China Extension: Pomalidomide 0.5 mg
    Reporting group description
    Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion. Participants who experienced anemia response could continue treatment until the response was lost or other criteria for treatment discontinuation applied. Participants enrolled in the China extension study were not blinded.

    Primary: China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days

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    End point title
    China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days [1] [2]
    End point description
    A response in the China extension study was defined as an increase in hemoglobin ≥ 15 g/L above baseline value (in the absence of RBC transfusion) for ≥ 84 consecutive days. The analysis was conducted in the China extension intent-to-treat (ITT) population which included all participants enrolled in the China extension study.
    End point type
    Primary
    End point timeframe
    From the first dose of study drug until treatment discontinuation; median treatment duration was 24.0 weeks.
    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: Endpoint was analyzed in the global study population only
    [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: Endpoint was analyzed in the global study population only
    End point values
    China Extension: Pomalidomide 0.5 mg
    Number of subjects analysed
    15
    Units: Participants
    1
    No statistical analyses for this end point

    Primary: Percentage of Participants who Achieved RBC-Transfusion Independence

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    End point title
    Percentage of Participants who Achieved RBC-Transfusion Independence [3]
    End point description
    RBC-transfusion independence was defined as the absence of RBC transfusions for any consecutive 84-day interval. The analysis was conducted in the global study intent-to-treat (ITT) population which included all randomized participants.
    End point type
    Primary
    End point timeframe
    168 days
    Notes
    [3] - 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: Endpoint was analyzed in the global study population only
    End point values
    Pomalidomide 0.5 mg Placebo
    Number of subjects analysed
    168
    84
    Units: percentage of participants
        number (confidence interval 95%)
    17.3 (11.88 to 23.84)
    16.7 (9.42 to 26.38)
    Statistical analysis title
    Statistical Analysis 1
    Comparison groups
    Pomalidomide 0.5 mg v Placebo
    Number of subjects included in analysis
    252
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 1
    Method
    Fisher exact
    Confidence interval

    Secondary: Overall Survival

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    End point title
    Overall Survival [4]
    End point description
    The time from randomization to the death or to the latest date when participants were known to be alive. Overall survival was analyzed in the global study intent-to-treat population using the Kaplan-Meier method; participants who were alive or lost to follow-up were censored at the latest date they were known to be alive.
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to end of study; median follow-up time was 19.1 months in the pomalidomide 0.5 mg arm and 17.6 months in the placebo arm.
    Notes
    [4] - 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: Endpoint was analyzed in the global study population only
    End point values
    Pomalidomide 0.5 mg Placebo
    Number of subjects analysed
    168
    84
    Units: months
        median (confidence interval 95%)
    24.2 (19.5 to 33.5)
    26.2 (18.0 to 32.6)
    Statistical analysis title
    Statistical Analysis 1
    Comparison groups
    Pomalidomide 0.5 mg v Placebo
    Number of subjects included in analysis
    252
    Analysis specification
    Pre-specified
    Analysis type
    other
    P-value
    = 0.929
    Method
    Logrank
    Confidence interval

    Secondary: Duration of RBC-Transfusion Independence

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    End point title
    Duration of RBC-Transfusion Independence [5]
    End point description
    The duration of RBC-transfusion independence is the time from the date at which the first RBC-transfusion independence started to the date of another RBC-transfusion given at least 84 days after the time the transfusion independence started. The duration of RBC-transfusion independence was analyzed in the global study intent-to-treat population who had an RBC-transfusion independence response using the Kaplan-Meier method. Data were censored at the end of the treatment phase for participants who had not received another RBC-transfusion after the start of transfusion independence by the end of treatment phase. "99999" indicates data that were not estimable.
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to 28 days after last dose, as of the data cut-off date of 16 Jan 2013; Median treatment duration was 23.6 weeks in the pomalidomide arm and 23.9 weeks in the placebo arm.
    Notes
    [5] - 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: Endpoint was analyzed in the global study population only
    End point values
    Pomalidomide 0.5 mg Placebo
    Number of subjects analysed
    29 [6]
    14 [7]
    Units: months
        median (confidence interval 95%)
    99999 (4.8 to 99999)
    5.8 (3.0 to 99999)
    Notes
    [6] - Participants with an RBC-transfusion independence response
    [7] - Participants with an RBC-transfusion independence response
    No statistical analyses for this end point

    Secondary: Time to RBC-Transfusion Independence

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    End point title
    Time to RBC-Transfusion Independence [8]
    End point description
    Time to response was measured from first dose of study drug to the start of the first response. The start date of the response was defined as one day after the last date of an RBC-transfusion for participants who received a transfusion after the first dose, and as the date of the first dose of study drug for participants who received no transfusions during the 84 days after the first dose of study drug.
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to 28 days after last dose, as of the data cut-off date of 16 Jan 2013; median treatment duration was 23.6 weeks in the pomalidomide arm and 23.9 weeks in the placebo arm.
    Notes
    [8] - 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: Endpoint was analyzed in the global study population only
    End point values
    Pomalidomide 0.5 mg Placebo
    Number of subjects analysed
    29 [9]
    14 [10]
    Units: weeks
        median (full range (min-max))
    6.9 (0.1 to 20.1)
    2.4 (0.1 to 15.4)
    Notes
    [9] - Participants with an RBC-transfusion independence response
    [10] - Participants with an RBC-transfusion independence response
    No statistical analyses for this end point

    Secondary: Number of Participants with Treatment-emergent Adverse Events

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    End point title
    Number of Participants with Treatment-emergent Adverse Events
    End point description
    A TEAE is an adverse event (AE) that starts on or after the first dose of study drug. The severity of each AE was graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE),Version 4.0 and according to the following scale: Grade 1 = Mild (transient or mild discomfort; no limitation in activity; no medical intervention/therapy required); Grade 2 = Moderate (mild to moderate limitation in activity, some assistance may be needed; minimal medical intervention/therapy required); Grade 3 = Severe (marked limitation in activity, assistance usually required; medical intervention/therapy required, hospitalization possible); Grade 4 = Life-threatening (extreme limitation in activity, significant assistance or medical intervention/therapy required, hospitalization or hospice care probable); Grade 5 = Death Drug-related (related) AEs are those suspected by the Investigator as being related to administration of study drug.
    End point type
    Secondary
    End point timeframe
    From the first dose of study drug until 28 days after last dose; median treatment duration was 23.7 weeks in the pomalidomide arm, 23.9 weeks in the placebo arm, and 24.0 weeks in the China extension pomalidomide arm.
    End point values
    Pomalidomide 0.5 mg Placebo China Extension: Pomalidomide 0.5 mg
    Number of subjects analysed
    167
    83
    15
    Units: Participants
        Any adverse event (AE)
    164
    81
    12
        Adverse event suspected as related to study drug
    90
    32
    3
        Adverse event leading to dose interruption
    48
    17
    2
        Drug-related AE leading to dose interruption
    26
    6
    1
        AE leading to discontinuation of study drug
    53
    14
    0
        Related AE leading to study drug discontinuation
    21
    8
    0
        Grade 3/4 adverse event
    100
    44
    4
        Grade 3/4 AE related to study drug
    45
    13
    0
        Grade 3/4 AE leading to study drug discontinuation
    33
    9
    0
        Grade 3/4 AE leading to dose interruption
    36
    14
    1
        Grade 5 adverse event
    17
    10
    0
        Grade 5 AE related to study drug
    1
    3
    0
        Serious adverse event (SAE)
    76
    29
    0
        SAE related to study drug
    24
    7
    0
        SAE leading to discontinuation of study drug
    31
    8
    0
        SAE leading to dose interruption
    22
    7
    0
    No statistical analyses for this end point

    Secondary: Healthcare Resource Utilization

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    End point title
    Healthcare Resource Utilization [11]
    End point description
    Characterization of medical resource utilization among participants treated with pomalidomide as compared to participants receiving placebo treatment.
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to 28 days after last dose, as of the data cut-off date of 16 Jan 2013; Median treatment duration was 23.6 weeks in the pomalidomide arm and 23.9 weeks in the placebo arm.
    Notes
    [11] - 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: There was no data collected for healthcare resource utilization
    End point values
    Pomalidomide 0.5 mg Placebo
    Number of subjects analysed
    0 [12]
    0 [13]
    Units: Participants
    Notes
    [12] - Healthcare resource utilization data were not collected during the study.
    [13] - Healthcare resource utilization data were not collected during the study.
    No statistical analyses for this end point

    Secondary: Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score

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    End point title
    Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score [14]
    End point description
    EQ-5D is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D includes 2 components: the EQ-5D health state profile (descriptive system) and the EQ-5D visual analog scale (VAS). For the health state profile participants rate their perceived health state today on 5 dimensions: mobility, selfcare, usual activities, pain/discomfort, and anxiety/depression on a Likert-type scale from 1 to 3, where 1 = “no problems,” 2 = “some problems,” and 3 = “extreme problems.” The EQ-5D Health Utility Index (HUI) was generated from the five health state domain scores, and ranges from -0.594 (worst) and 1 (best) imaginable health state, with -0.594 representing an “unconscious” health state.
    End point type
    Secondary
    End point timeframe
    Baseline and Days 85 and 169
    Notes
    [14] - 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: Endpoint was analyzed in the global study population only
    End point values
    Pomalidomide 0.5 mg Placebo
    Number of subjects analysed
    162 [15]
    82 [16]
    Units: score on a scale
    arithmetic mean (standard deviation)
        Day 85
    -0.0385 ± 0.2480
    -0.0298 ± 0.2129
        Day 169
    -0.0202 ± 0.1666
    0.0766 ± 0.1546
    Notes
    [15] - Day 85 N = 112 Day 169 N = 41
    [16] - Day 85, N = 56 Day 169 N = 13
    No statistical analyses for this end point

    Secondary: Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale

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    End point title
    Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale [17]
    End point description
    EQ-5D is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D includes 2 components: the EQ-5D health state profile (descriptive system) and the EQ-5D visual analog scale (VAS). On the VAS the participant rates his/her health state on a line from 0 (worst imaginable health) to 100 (best imaginable health).
    End point type
    Secondary
    End point timeframe
    Baseline and Days 85 and 169
    Notes
    [17] - 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: Endpoint was analyzed in the global study population only
    End point values
    Pomalidomide 0.5 mg Placebo
    Number of subjects analysed
    161 [18]
    81 [19]
    Units: units on a scale
    arithmetic mean (standard deviation)
        Day 85
    2.0 ± 20.78
    -1.4 ± 14.07
        Day 169
    2.9 ± 22.85
    0.3 ± 24.25
    Notes
    [18] - Day 85 N = 111 Day 169 N = 41
    [19] - Day 85 N = 55 Day 169 N = 13
    No statistical analyses for this end point

    Secondary: Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score

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    End point title
    Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score [20]
    End point description
    The FACT-An is a 47-item, cancer-specific questionnaire consisting of a core 27-item general questionnaire measuring the four general domains of QoL (physical, social/family, emotional and functional well-being), and an additional 20-item anemia questionnaire (FACT-An Anemia subscale) that measures 13 fatigue-associated items (FACT-F Fatigue subscale) and seven non-fatigue-related items. Each item is scored using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a bit; and 4 = Very much). FACT-An total score is calculated by adding all the FACT-An subscales together. The total score ranges from 0-188 with higher scores representing better QOL.
    End point type
    Secondary
    End point timeframe
    Baseline and Days 85 and 169
    Notes
    [20] - 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: Endpoint was analyzed in the global study population only
    End point values
    Pomalidomide 0.5 mg Placebo
    Number of subjects analysed
    159 [21]
    81 [22]
    Units: units on a scale
    arithmetic mean (standard deviation)
        Day 85
    -2.1 ± 20.32
    4.3 ± 18.73
        Day 169
    6.2 ± 20.49
    11.9 ± 18.60
    Notes
    [21] - Day 85 N = 109 Day 169 N = 41
    [22] - Day 85 N = 56 Day 169 N = 13
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Deaths are from first dose of study drug up to end of study, maximum time on-study was 85 months. AEs are from the first dose of study drug until 28 days after last dose; median treatment duration was 23.7, 23.9, and 24.0 weeks in each arm respectively.
    Adverse event reporting additional description
    The safety population includes all participants who received at least one dose of study drug; one participant randomized to pomalidomide and one participant randomized to placebo in the global study did not receive treatment and are excluded from the safety population.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    15.1
    Reporting groups
    Reporting group title
    Global Study: Pomalidomide 0.5 mg
    Reporting group description
    Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.

    Reporting group title
    Global Study: Placebo
    Reporting group description
    Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.

    Reporting group title
    China Extension: Pomalidomide 0.5 mg
    Reporting group description
    Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion. Participants who experienced anemia response could continue treatment until the response was lost or other criteria for treatment discontinuation applied.

    Serious adverse events
    Global Study: Pomalidomide 0.5 mg Global Study: Placebo China Extension: Pomalidomide 0.5 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    76 / 167 (45.51%)
    29 / 83 (34.94%)
    0 / 15 (0.00%)
         number of deaths (all causes)
    115
    54
    8
         number of deaths resulting from adverse events
    Vascular disorders
    AXILLARY VEIN THROMBOSIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    DEEP VEIN THROMBOSIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    HYPOTENSION
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    INTERMITTENT CLAUDICATION
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    JUGULAR VEIN THROMBOSIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    PHLEBITIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    B-CELL LYMPHOMA
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    BASAL CELL CARCINOMA
         subjects affected / exposed
    1 / 167 (0.60%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    HISTIOCYTOSIS HAEMATOPHAGIC
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    MALIGNANT MELANOMA
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    MYELOFIBROSIS
         subjects affected / exposed
    9 / 167 (5.39%)
    3 / 83 (3.61%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 9
    3 / 5
    0 / 0
         deaths causally related to treatment / all
    0 / 3
    1 / 3
    0 / 0
    OESOPHAGEAL ADENOCARCINOMA
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    ASTHENIA
         subjects affected / exposed
    2 / 167 (1.20%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    FATIGUE
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    GENERALISED OEDEMA
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    INFLUENZA LIKE ILLNESS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    MULTI-ORGAN FAILURE
         subjects affected / exposed
    2 / 167 (1.20%)
    2 / 83 (2.41%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    1 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    1 / 2
    0 / 0
    NON-CARDIAC CHEST PAIN
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    OEDEMA PERIPHERAL
         subjects affected / exposed
    4 / 167 (2.40%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 4
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    PYREXIA
         subjects affected / exposed
    5 / 167 (2.99%)
    3 / 83 (3.61%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    2 / 5
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    SUDDEN CARDIAC DEATH
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    SUDDEN DEATH
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    Psychiatric disorders
    ABNORMAL BEHAVIOUR
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    FALL
         subjects affected / exposed
    1 / 167 (0.60%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    INJURY
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    SUBDURAL HAEMATOMA
         subjects affected / exposed
    1 / 167 (0.60%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    SUBDURAL HAEMORRHAGE
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    TRANSFUSION-RELATED CIRCULATORY OVERLOAD
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    Investigations
    ASPARTATE AMINOTRANSFERASE INCREASED
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    PLATELET COUNT INCREASED
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    WHITE BLOOD CELL COUNT INCREASED
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    ACUTE CORONARY SYNDROME
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    ACUTE MYOCARDIAL INFARCTION
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    ATRIAL FIBRILLATION
         subjects affected / exposed
    2 / 167 (1.20%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    CARDIAC ARREST
         subjects affected / exposed
    2 / 167 (1.20%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    CARDIAC FAILURE
         subjects affected / exposed
    5 / 167 (2.99%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    4 / 9
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
    CARDIAC FAILURE ACUTE
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    CARDIAC FAILURE CONGESTIVE
         subjects affected / exposed
    3 / 167 (1.80%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    CONDUCTION DISORDER
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    CORONARY ARTERY DISEASE
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    Respiratory, thoracic and mediastinal disorders
    DYSPNOEA
         subjects affected / exposed
    1 / 167 (0.60%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    EPISTAXIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    HYPOXIA
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    INTERSTITIAL LUNG DISEASE
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    PNEUMONITIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    PULMONARY EMBOLISM
         subjects affected / exposed
    2 / 167 (1.20%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    PULMONARY HYPERTENSION
         subjects affected / exposed
    2 / 167 (1.20%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    RESPIRATORY FAILURE
         subjects affected / exposed
    2 / 167 (1.20%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    ANAEMIA
         subjects affected / exposed
    10 / 167 (5.99%)
    3 / 83 (3.61%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    3 / 14
    1 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    DISSEMINATED INTRAVASCULAR COAGULATION
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    FEBRILE NEUTROPENIA
         subjects affected / exposed
    3 / 167 (1.80%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 3
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    HAEMOLYSIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    HAEMOLYTIC ANAEMIA
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    NEUTROPENIA
         subjects affected / exposed
    1 / 167 (0.60%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    PANCYTOPENIA
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    SPLENIC EMBOLISM
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    SPLENIC INFARCTION
         subjects affected / exposed
    1 / 167 (0.60%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    SPLENOMEGALY
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    THROMBOCYTOPENIA
         subjects affected / exposed
    3 / 167 (1.80%)
    2 / 83 (2.41%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 3
    1 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    CEREBROVASCULAR ACCIDENT
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    CONVULSION
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    LOSS OF CONSCIOUSNESS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    SYNCOPE
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    VIITH NERVE PARALYSIS
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    ABDOMINAL DISTENSION
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    ASCITES
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    DIARRHOEA
         subjects affected / exposed
    0 / 167 (0.00%)
    2 / 83 (2.41%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    ENTEROCOLITIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    GASTRIC HAEMORRHAGE
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    GASTROINTESTINAL HAEMORRHAGE
         subjects affected / exposed
    2 / 167 (1.20%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    HAEMORRHOIDAL HAEMORRHAGE
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    HAEMORRHOIDS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    MELAENA
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    VOMITING
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    CALCULUS BLADDER
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    CALCULUS URETERIC
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    NEPHROLITHIASIS
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    NEPHROTIC SYNDROME
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    RENAL COLIC
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    RENAL FAILURE
         subjects affected / exposed
    3 / 167 (1.80%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    RENAL FAILURE ACUTE
         subjects affected / exposed
    1 / 167 (0.60%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    RENAL FAILURE CHRONIC
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    URINARY RETENTION
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    URINARY TRACT OBSTRUCTION
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    CHOLECYSTITIS
         subjects affected / exposed
    1 / 167 (0.60%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    Skin and subcutaneous tissue disorders
    ACUTE FEBRILE NEUTROPHILIC DERMATOSIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    DRUG ERUPTION
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    LEUKOCYTOCLASTIC VASCULITIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    PETECHIAE
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    RASH MACULO-PAPULAR
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    SKIN ULCER
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    Musculoskeletal and connective tissue disorders
    BACK PAIN
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    DECREASED APPETITE
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    DEHYDRATION
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    DIABETIC KETOACIDOSIS
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    HYPOGLYCAEMIA
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    Infections and infestations
    ATYPICAL PNEUMONIA
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    BACTEROIDES BACTERAEMIA
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    BRONCHITIS
         subjects affected / exposed
    2 / 167 (1.20%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    BRONCHOPULMONARY ASPERGILLOSIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    CELLULITIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    DEVICE RELATED INFECTION
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    ENTEROCOLITIS INFECTIOUS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    ERYSIPELAS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    GASTROINTESTINAL CANDIDIASIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    INFECTIOUS PLEURAL EFFUSION
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    LUNG INFECTION
         subjects affected / exposed
    3 / 167 (1.80%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    LYMPHADENITIS BACTERIAL
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    NECROTISING FASCIITIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    PARONYCHIA
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    PNEUMOCYSTIS JIROVECI PNEUMONIA
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    PNEUMONIA
         subjects affected / exposed
    6 / 167 (3.59%)
    4 / 83 (4.82%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 7
    0 / 4
    0 / 0
         deaths causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    SEPSIS
         subjects affected / exposed
    1 / 167 (0.60%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    SEPTIC SHOCK
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    SIALOADENITIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    STAPHYLOCOCCAL SEPSIS
         subjects affected / exposed
    2 / 167 (1.20%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    URINARY TRACT INFECTION
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    UROSEPSIS
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    WOUND INFECTION
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    0 / 15 (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
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Global Study: Pomalidomide 0.5 mg Global Study: Placebo China Extension: Pomalidomide 0.5 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    155 / 167 (92.81%)
    76 / 83 (91.57%)
    12 / 15 (80.00%)
    General disorders and administration site conditions
    ASTHENIA
         subjects affected / exposed
    23 / 167 (13.77%)
    8 / 83 (9.64%)
    0 / 15 (0.00%)
         occurrences all number
    31
    9
    0
    FATIGUE
         subjects affected / exposed
    35 / 167 (20.96%)
    17 / 83 (20.48%)
    3 / 15 (20.00%)
         occurrences all number
    51
    23
    4
    OEDEMA PERIPHERAL
         subjects affected / exposed
    53 / 167 (31.74%)
    14 / 83 (16.87%)
    1 / 15 (6.67%)
         occurrences all number
    83
    18
    1
    PYREXIA
         subjects affected / exposed
    30 / 167 (17.96%)
    9 / 83 (10.84%)
    2 / 15 (13.33%)
         occurrences all number
    51
    13
    2
    CHEST PAIN
         subjects affected / exposed
    2 / 167 (1.20%)
    0 / 83 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    2
    OEDEMA
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Injury, poisoning and procedural complications
    FALL
         subjects affected / exposed
    7 / 167 (4.19%)
    5 / 83 (6.02%)
    0 / 15 (0.00%)
         occurrences all number
    9
    7
    0
    LIGAMENT SPRAIN
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    Investigations
    WEIGHT DECREASED
         subjects affected / exposed
    11 / 167 (6.59%)
    3 / 83 (3.61%)
    1 / 15 (6.67%)
         occurrences all number
    12
    3
    1
    WHITE BLOOD CELL COUNT DECREASED
         subjects affected / exposed
    2 / 167 (1.20%)
    0 / 83 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    5
    0
    2
    WHITE BLOOD CELL COUNT INCREASED
         subjects affected / exposed
    0 / 167 (0.00%)
    1 / 83 (1.20%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    2
    Cardiac disorders
    ARRHYTHMIA
         subjects affected / exposed
    2 / 167 (1.20%)
    1 / 83 (1.20%)
    1 / 15 (6.67%)
         occurrences all number
    4
    1
    1
    Blood and lymphatic system disorders
    ANAEMIA
         subjects affected / exposed
    12 / 167 (7.19%)
    5 / 83 (6.02%)
    2 / 15 (13.33%)
         occurrences all number
    16
    9
    2
    NEUTROPENIA
         subjects affected / exposed
    25 / 167 (14.97%)
    4 / 83 (4.82%)
    0 / 15 (0.00%)
         occurrences all number
    73
    9
    0
    THROMBOCYTOPENIA
         subjects affected / exposed
    21 / 167 (12.57%)
    12 / 83 (14.46%)
    1 / 15 (6.67%)
         occurrences all number
    41
    22
    1
    Respiratory, thoracic and mediastinal disorders
    COUGH
         subjects affected / exposed
    24 / 167 (14.37%)
    9 / 83 (10.84%)
    1 / 15 (6.67%)
         occurrences all number
    31
    10
    1
    DYSPNOEA
         subjects affected / exposed
    28 / 167 (16.77%)
    8 / 83 (9.64%)
    1 / 15 (6.67%)
         occurrences all number
    44
    8
    1
    DYSPNOEA EXERTIONAL
         subjects affected / exposed
    10 / 167 (5.99%)
    3 / 83 (3.61%)
    0 / 15 (0.00%)
         occurrences all number
    12
    5
    0
    EPISTAXIS
         subjects affected / exposed
    8 / 167 (4.79%)
    5 / 83 (6.02%)
    0 / 15 (0.00%)
         occurrences all number
    15
    10
    0
    PLEURAL EFFUSION
         subjects affected / exposed
    5 / 167 (2.99%)
    2 / 83 (2.41%)
    1 / 15 (6.67%)
         occurrences all number
    5
    2
    1
    Nervous system disorders
    DIZZINESS
         subjects affected / exposed
    17 / 167 (10.18%)
    12 / 83 (14.46%)
    0 / 15 (0.00%)
         occurrences all number
    25
    16
    0
    SOMNOLENCE
         subjects affected / exposed
    5 / 167 (2.99%)
    5 / 83 (6.02%)
    0 / 15 (0.00%)
         occurrences all number
    5
    7
    0
    Gastrointestinal disorders
    ABDOMINAL DISTENSION
         subjects affected / exposed
    11 / 167 (6.59%)
    1 / 83 (1.20%)
    0 / 15 (0.00%)
         occurrences all number
    12
    1
    0
    ABDOMINAL PAIN
         subjects affected / exposed
    22 / 167 (13.17%)
    11 / 83 (13.25%)
    0 / 15 (0.00%)
         occurrences all number
    27
    11
    0
    ABDOMINAL PAIN UPPER
         subjects affected / exposed
    10 / 167 (5.99%)
    3 / 83 (3.61%)
    0 / 15 (0.00%)
         occurrences all number
    12
    3
    0
    CONSTIPATION
         subjects affected / exposed
    24 / 167 (14.37%)
    9 / 83 (10.84%)
    0 / 15 (0.00%)
         occurrences all number
    28
    9
    0
    DIARRHOEA
         subjects affected / exposed
    35 / 167 (20.96%)
    17 / 83 (20.48%)
    2 / 15 (13.33%)
         occurrences all number
    53
    20
    3
    NAUSEA
         subjects affected / exposed
    21 / 167 (12.57%)
    16 / 83 (19.28%)
    0 / 15 (0.00%)
         occurrences all number
    25
    17
    0
    VOMITING
         subjects affected / exposed
    12 / 167 (7.19%)
    7 / 83 (8.43%)
    0 / 15 (0.00%)
         occurrences all number
    18
    9
    0
    Renal and urinary disorders
    CALCULUS URETERIC
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    1
    NEPHROLITHIASIS
         subjects affected / exposed
    3 / 167 (1.80%)
    0 / 83 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    4
    0
    1
    Hepatobiliary disorders
    HEPATIC CYST
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    HEPATIC FUNCTION ABNORMAL
         subjects affected / exposed
    1 / 167 (0.60%)
    0 / 83 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    1
    Skin and subcutaneous tissue disorders
    NIGHT SWEATS
         subjects affected / exposed
    10 / 167 (5.99%)
    3 / 83 (3.61%)
    0 / 15 (0.00%)
         occurrences all number
    12
    3
    0
    PRURITUS
         subjects affected / exposed
    12 / 167 (7.19%)
    5 / 83 (6.02%)
    0 / 15 (0.00%)
         occurrences all number
    14
    6
    0
    RASH
         subjects affected / exposed
    11 / 167 (6.59%)
    2 / 83 (2.41%)
    0 / 15 (0.00%)
         occurrences all number
    19
    2
    0
    Musculoskeletal and connective tissue disorders
    ARTHRALGIA
         subjects affected / exposed
    6 / 167 (3.59%)
    8 / 83 (9.64%)
    0 / 15 (0.00%)
         occurrences all number
    9
    10
    0
    MUSCLE SPASMS
         subjects affected / exposed
    9 / 167 (5.39%)
    5 / 83 (6.02%)
    0 / 15 (0.00%)
         occurrences all number
    12
    6
    0
    PAIN IN EXTREMITY
         subjects affected / exposed
    11 / 167 (6.59%)
    6 / 83 (7.23%)
    0 / 15 (0.00%)
         occurrences all number
    13
    9
    0
    Metabolism and nutrition disorders
    DECREASED APPETITE
         subjects affected / exposed
    20 / 167 (11.98%)
    7 / 83 (8.43%)
    0 / 15 (0.00%)
         occurrences all number
    22
    7
    0
    HYPERURICAEMIA
         subjects affected / exposed
    7 / 167 (4.19%)
    5 / 83 (6.02%)
    0 / 15 (0.00%)
         occurrences all number
    8
    5
    0
    HYPERGLYCAEMIA
         subjects affected / exposed
    3 / 167 (1.80%)
    2 / 83 (2.41%)
    1 / 15 (6.67%)
         occurrences all number
    3
    2
    1
    Infections and infestations
    BRONCHITIS
         subjects affected / exposed
    9 / 167 (5.39%)
    4 / 83 (4.82%)
    2 / 15 (13.33%)
         occurrences all number
    13
    5
    2
    NASOPHARYNGITIS
         subjects affected / exposed
    13 / 167 (7.78%)
    0 / 83 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    22
    0
    0
    PNEUMONIA
         subjects affected / exposed
    10 / 167 (5.99%)
    2 / 83 (2.41%)
    1 / 15 (6.67%)
         occurrences all number
    10
    2
    1
    UPPER RESPIRATORY TRACT INFECTION
         subjects affected / exposed
    10 / 167 (5.99%)
    5 / 83 (6.02%)
    1 / 15 (6.67%)
         occurrences all number
    11
    5
    2
    URINARY TRACT INFECTION
         subjects affected / exposed
    12 / 167 (7.19%)
    2 / 83 (2.41%)
    0 / 15 (0.00%)
         occurrences all number
    15
    3
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    28 Sep 2010
    Significant changes included: - The concept of Clinical Benefit was introduced to enable subjects with a 50% decrease in RBC-transfusion frequency to continue receiving study-medication even if RBC-transfusion independence was not achieved. This applied to subjects achieving RBC-transfusion independence if RBC transfusion independence ended due to an unrelated event. - Blood sampling was done in a subset of subjects to explore the pharmacokinetics of pomalidomide in this population. - RBC transfusions used to determine eligibility required a pre RBC-transfusion hemoglobin level ≤ 90 g/L. In subjects where a hemoglobin level < 90 g/L is dangerous, entry into the study with pre RBC-transfusion hemoglobin level > 90 g/L was allowed as long as a 6-month RBC-transfusion history was available to confirm eligibility. - If a subject could not be randomized within 28 days of signing Informed Consent due to logistical reasons, the medical monitor could approve randomization as soon as possible thereafter to eliminate the need to repeat screening procedures when not otherwise necessary. - A hemoglobin level measured > 72 hours before a RBC-transfusion could be used to determine eligibility if there were no intervening RBC-transfusions. - The medical monitor could approve the inclusion of a subject based on a bone marrow biopsy done > 6 months pre-screening. - In North America and the European Union a unit of RBC is approximately 240 mL and the average weight of a 50-year-old male is 90 kg. The protocol definition of ≥ 2 U RBC/28 days is equivalent to 5 mL/kg/28 days. In Japan and China a unit of RBCs is about 125 mL and the average weight of a 50-year-old male is 60 kg. The equivalent RBC-transfusion dose is 2.5 U/28 days. - Subjects receiving anti-coagulants other than low-dose aspirin were not excluded - Criteria for determination of blastic transformation based on blasts in blood was clarified. - 5-year follow-up for collection of new cancers was added

    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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