Clinical Trial Page

Clinical Trial Results:
A Phase 2, Prospective, Randomized, Multicenter, Double-blind, Active-control, Parallel-group Study to Determine the Safety of and to Select a Treatment Regimen of CC-4047 (Pomalidomide) Either as Single-agent or in Combination With Prednisone to Study Further in Subjects With Myelofibrosis With Myeloid Metaplasia

Summary
EudraCT number
 2006-004553-17
Trial protocol
 GB   ES   AT   IT   DE  
Global end of trial date
24 Sep 2013

Results information
Results version number
 v1(current)
This version publication date
14 Jul 2016
First version publication date
07 Aug 2015
Other versions

Trial information

 Close Top of page
Trial identification
Sponsor protocol code
CC-4047-MMM-001
Additional study identifiers
ISRCTN number
 -
US NCT number
 NCT00463385
WHO universal trial number (UTN)
 -
Sponsors
Sponsor organisation name
Celgene Corporation
Sponsor organisation address
86 Morris Avenue, Summit, NJ, United States, 07901
Public contact
Clinical Trial Disclosure 86 Morris Avenue Summit, NJ 07901, Celgene Corporation 86 Morris Avenue Summit, NJ 07901, 1 866-260-1599, ClinicalTrialDisclosure@Celgene.com
Scientific contact
Robert Gale, MD 86 Morris Avenue Summit, NJ 07901, Robert Gale, MD Celgene Corporation 86 Morris Avenue Summit, NJ 07901, 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
13 Nov 2014
Is this the analysis of the primary completion data?
Yes
Primary completion date
24 Sep 2013
Global end of trial reached?
Yes
Global end of trial date
24 Sep 2013
Was the trial ended prematurely?
No
General information about the trial
Main objective of the trial
To select a treatment regimen of CC-4047 either as single-agent or in combination with prednisone to study further in subjects with myelofibrosis with myeloid metaplasia (MMM).
Protection of trial subjects
Protection of Subjects by Institutional Review Board/Independent Ethics Committee Review and Approval; Protection of Patient Confidentiality
Background therapy
 -
Evidence for comparator
 This study in MMM was designed to determine the appropriate CC-4047 dose and regimen as a monotherapy or in combination with prednisone. The comparator (prednisone monotherapy) facilitates the description of the Adverse Event (AE) profile of CC-4047. The prednisone control arm also allowed for a comparison in response rates of the CC-4047 mono- and combination therapies; thus providing a reasonable basis of information for designing further studies should the outcomes be in favor of a CC-4047 arm.
Actual start date of recruitment
19 Apr 2007
Long term follow-up planned
No
Independent data monitoring committee (IDMC) involvement?
Yes
Population of trial subjects
Number of subjects enrolled per country
Country: Number of subjects enrolled
Spain: 3
Country: Number of subjects enrolled
Austria: 5
Country: Number of subjects enrolled
Italy: 28
Country: Number of subjects enrolled
United States: 52
Worldwide total number of subjects
88
EEA total number of subjects
36
Number of subjects enrolled per age group
In utero
0
Preterm newborn - gestational age
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)
35
From 65 to 84 years
52
85 years and over
1

Subject disposition

 Close Top of page
Recruitment
Recruitment details
 -

Pre-assignment
Screening details
 Participants were entered into the Pre-randomization phase and were evaluated for the inclusion and exclusion criteria for the Double-Blind Treatment Phase of the study. The Pre-Randomization Phase did not last more than 28 days. However, the bone marrow histology for diagnosis may have preceded this period.

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, Monitor
Blinding implementation details
A double-blind technique was used and was chosen to minimize bias on the part of participants, investigators, and the sponsor. Identical placebo capsules were supplied to match the 0.5 mg and 1.0 mg pomalidomide capsules. Placebo to match prednisone was also provided. The blind was not to be broken during the Double-Blind Treatment Phase unless in the opinion of the investigator it was absolutely needed to safely treat the subject. The medical monitor was to be contacted prior to unblinding.

Arms
Are arms mutually exclusive
Yes

Arm title
 Prednisone
Arm description
 Participants received oral prednisone from Day 1-28 of each 28-day cycle for up to 3 cycles (84 days), 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day, and pomalidomide placebo tablets on Days 1-28 for up to 12 cycles in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, participants were discontinued from the study.
Arm type
 Active comparator

Investigational medicinal product name
Prednisone
Investigational medicinal product code
H02 AB07
Other name
Deltasone; Orasone
Pharmaceutical forms
Capsule, hard
Routes of administration
Oral use
Dosage and administration details
Participants received oral prednisone from Day 1-28 of each 28-day cycle for up to 3 cycles (84 days), 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day, and pomalidomide placebo tablets on Days 1-28 for up to 12 cycles in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, participants were discontinued from the study.

Investigational medicinal product name
Pomalidomide Placebo
Investigational medicinal product code
Other name
Placebo
Pharmaceutical forms
Capsule, hard
Routes of administration
Oral use
Dosage and administration details
Participants received oral prednisone from Day 1-28 of each 28-day cycle for up to 3 cycles (84 days), 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day, and pomalidomide placebo tablets on Days 1-28 for up to 12 cycles in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, participants were discontinued from the study.

Arm title
 Pomalidomide 2 mg
Arm description
 Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and prednisone placebo tablets on Days 1-28 for the first 3 cycles in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.
Arm type
 Experimental

Investigational medicinal product name
Pomalidomide
Investigational medicinal product code
CC-4047
Other name
Imnovid; Pomalyst
Pharmaceutical forms
Capsule, hard
Routes of administration
Oral use
Dosage and administration details
Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and prednisone placebo tablets on Days 1-28 for the first 3 cycles in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal

Investigational medicinal product name
Prednisone Placebo
Investigational medicinal product code
Other name
Placebo
Pharmaceutical forms
Capsule, hard
Routes of administration
Oral use
Dosage and administration details
Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and prednisone placebo tablets on Days 1-28 for the first 3 cycles in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Arm title
 Pomalidomide 2 mg + Prednisone
Arm description
 Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.
Arm type
 Experimental

Investigational medicinal product name
Pomalidomide
Investigational medicinal product code
CC-4047
Other name
Imnovid; Pomalyst
Pharmaceutical forms
Capsule, hard
Routes of administration
Oral use
Dosage and administration details
Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal. Period Title: Overall Study

Investigational medicinal product name
Prednisone
Investigational medicinal product code
H02 AB07
Other name
Pharmaceutical forms
Tablet
Routes of administration
Oral use
Dosage and administration details
Oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal

Arm title
 Pomalidomide 0.5 mg + Prednisone
Arm description
 Participants received 0.5 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 0.5 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.
Arm type
 Experimental

Investigational medicinal product name
Pomalidomide
Investigational medicinal product code
CC-4047
Other name
Imnovid
Pharmaceutical forms
Capsule, hard
Routes of administration
Oral use
Dosage and administration details
Participants received 0.5 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 0.5 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal. Period Title: Overall Study

Investigational medicinal product name
Predisone
Investigational medicinal product code
H02 AB07
Other name
Pharmaceutical forms
Tablet
Routes of administration
Oral use
Dosage and administration details
Participants received 0.5 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 0.5 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Number of subjects in period 1
Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Started
22
22
22
22
Treated
22
22
19
22
Completed
0
0
0
0
Not completed
22
22
22
22
     Disease Progression
6
6
7
9
     Death
2
1
2
-
     Missing
2
-
-
2
     1 participant received commercial drug
-
1
-
-
     Adverse event, non-fatal
5
8
4
2
     Consent withdrawn by subject
3
3
3
4
     Unspecified
4
3
6
4
     Lost to follow-up
-
-
-
1

Baseline characteristics

 Close Top of page
Baseline characteristics reporting groups
Reporting group title
Prednisone
Reporting group description
 Participants received oral prednisone from Day 1-28 of each 28-day cycle for up to 3 cycles (84 days), 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day, and pomalidomide placebo tablets on Days 1-28 for up to 12 cycles in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, participants were discontinued from the study.

Reporting group title
Pomalidomide 2 mg
Reporting group description
 Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and prednisone placebo tablets on Days 1-28 for the first 3 cycles in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Reporting group title
Pomalidomide 2 mg + Prednisone
Reporting group description
 Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Reporting group title
Pomalidomide 0.5 mg + Prednisone
Reporting group description
 Participants received 0.5 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 0.5 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Reporting group values
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone Total
Number of subjects
 22 22 22 22 88
Age categorical
Units: Subjects
    Adults (18-64 years)
 10 8 8 9 35
    From 65-84 years
 12 14 14 12 52
    85 years and over
 0 0 0 1 1
Age continuous
Units: years
    median (full range (min-max))
 66 (44 to 80) 68 (50 to 83) 67.5 (36 to 82) 69.5 (43 to 86) -
Gender categorical
Units: Subjects
    Female
 8 5 7 9 29
    Male
 14 17 15 13 59
Race/Ethnicity
Units: Subjects
    White
 21 22 21 22 86
    Black
 0 0 1 0 1
    Hispanic
 1 0 0 0 1
Janus kinase 2 (JAK2) Mutation
JAK2^V617F mutation result based on quantitative polymerase chain reaction (PCR) analysis in neutrophil preparation.
Units: Subjects
    Negative
 6 7 8 8 29
    Positive
 13 11 10 9 43
    Missing
 3 4 4 5 16
Eastern Cooperative Oncology Group (ECOG) Performance Status
ECOG performance status scale and criteria used to assess disease progression, how the disease affects the daily living abilities of the patient, and determine appropriate treatment: 0= Fully active, able to carry on all pre-disease activities; 1= Restricted in physically strenuous activity; ambulatory and able to carry out light work; 2= Ambulatory and capable of all selfcare; unable to perform work activities. Up and about > 50% of waking hours; 3= Capable of only limited selfcare, confined to bed/chair > 50% of waking hours; 4= Completely disabled. Confined to bed or chair; 5= Dead
Units: Subjects
    Grade 0
 12 11 6 14 43
    Grade 1
 8 9 10 6 33
    Garde 2
 2 2 5 2 11
    Missing
 0 0 1 0 1
Myelofibrosis with myeloid metaplasia Subtype
Units: Subjects
    Agnogenic Myeloid Metaplasia (AMM)
 16 16 16 13 61
    Postpolycythemic Myeloid Metaplasia (PPMM)
 3 4 4 2 13
    Postthromocythemic Myeloid Metaplasia (PTMM)
 3 2 2 7 14
Red Blood Cell (RBC) Transfusion Dependence [1]
A patient who receives at least a total of two units of RBC transfusion within 28 days on or prior to the first study drug dosing date is an RBC-transfusion-dependent patient. Otherwise a patient is an RBC-transfusion-independent patient.
Units: Subjects
    Yes
 12 10 9 12 43
    No
 10 12 13 10 45
Time Since Myelofibrosis Diagnosis
Units: Years
    median (full range (min-max))
 1.5 (0 to 14.3) 0.6 (0 to 6.3) 1.1 (0 to 13) 1.7 (0 to 10.9) -
RBC Transfusion Burden
Units: units/28 days
    median (full range (min-max))
 2 (0 to 7) 1 (0 to 6) 0 (0 to 6) 1 (0 to 7) -

End points

 Close Top of page
End points reporting groups
Reporting group title
Prednisone
Reporting group description
 Participants received oral prednisone from Day 1-28 of each 28-day cycle for up to 3 cycles (84 days), 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day, and pomalidomide placebo tablets on Days 1-28 for up to 12 cycles in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, participants were discontinued from the study.

Reporting group title
Pomalidomide 2 mg
Reporting group description
 Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and prednisone placebo tablets on Days 1-28 for the first 3 cycles in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Reporting group title
Pomalidomide 2 mg + Prednisone
Reporting group description
 Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Reporting group title
Pomalidomide 0.5 mg + Prednisone
Reporting group description
 Participants received 0.5 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 0.5 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Primary: Percentage of Participants With a Clinical Response Within the First 6 Cycles of Treatment

 Close Top of page
End point title
 Percentage of Participants With a Clinical Response Within the First 6 Cycles of Treatment
End point description
A clinical responder was defined as either: a. A baseline red blood cell (RBC)-transfusion-dependent participant with a ≥ 56 consecutive day RBC transfusion-free period after the first dose of study drug, or b. A baseline RBC-transfusion-independent participant with an increase in hemoglobin of 2.0 g/dL or more from baseline for ≥ 56 consecutive days in the absence of RBC transfusions, or c. A participant with either a ≥ 50% reduction in palpable splenomegaly of a spleen that was ≥ 10 cm at baseline or a spleen that was palpable at > 5 cm and became not palpable. Participants who discontinued the study early without achieving clinical response were counted as non-responders. Modified intent-to-treat (MITT), defined as the patients who had a confirmed diagnosis of Myelofibrosis with myeloid metaplasia (MMM), received at least one dose of study drug, and participated in the study for at least 56 days.
End point type
Primary
End point timeframe
Up to 168 days
End point values
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Number of subjects analysed
20
17
19
21
Units: percentage of participants
    number (confidence interval 95%)
55 (31.53 to 76.94)
23.5 (6.81 to 49.9)
21.1 (6.05 to 45.57)
47.6 (25.71 to 70.22)
Statistical analysis title
 Clinical Response
Statistical analysis description
Statistical Analysis 1 for Percentage of Participants With a Clinical Response Within the First 6 Cycles of Treatment
Comparison groups
Prednisone v Pomalidomide 2 mg
Number of subjects included in analysis
37
Analysis specification
Pre-specified
Analysis type
 superiority
P-value
 = 0.092
Method
 Fisher exact
Confidence interval
Statistical analysis title
 Clinical Response
Statistical analysis description
Participants With a Clinical Response Within the First 6 Cycles of Treatment
Comparison groups
Prednisone v Pomalidomide 2 mg + Prednisone
Number of subjects included in analysis
39
Analysis specification
Pre-specified
Analysis type
 superiority
P-value
 = 0.048
Method
 Fisher exact
Confidence interval
Statistical analysis title
 Clinical Response
Statistical analysis description
Participants With a Clinical Response Within the First 6 Cycles of Treatment
Comparison groups
Prednisone v Pomalidomide 0.5 mg + Prednisone
Number of subjects included in analysis
41
Analysis specification
Pre-specified
Analysis type
 superiority
P-value
 = 0.758
Method
 Fisher exact
Confidence interval

Secondary: Percentage of Participants With a Clinical Response Within the First 12 Cycles of Treatment

 Close Top of page
End point title
 Percentage of Participants With a Clinical Response Within the First 12 Cycles of Treatment
End point description
A clinical responder was defined as either: a. A baseline red blood cell (RBC)-transfusion-dependent participant with a ≥ 56 consecutive day RBC transfusion-free period after the first dose of study drug, or b. A baseline RBC-transfusion-independent participant with an increase in hemoglobin of 2.0 g/dL or more from baseline for ≥ 56 consecutive days in the absence of RBC transfusions, or c. A participant with either a ≥ 50% reduction in palpable splenomegaly of a spleen that was ≥ 10 cm at baseline or a spleen that was palpable at > 5 cm and became not palpable. Participants who discontinued the study early without achieving clinical response were counted as non-responders. Intent-to-treat (ITT), defined as all patients who were randomized, independent of whether they received study treatment or not.
End point type
Secondary
End point timeframe
Up to 336 days
End point values
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Number of subjects analysed
22
22
22
22
Units: Percentage of participants
    number (confidence interval 95%)
50 (28.22 to 71.78)
18.2 (5.19 to 40.28)
18.2 (5.19 to 40.28)
45.5 (24.39 to 67.79)
No statistical analyses for this end point

Secondary: Time to the First Clinical Response

 Close Top of page
End point title
 Time to the First Clinical Response
End point description
Title: Time to the First Clinical Response Description: The time to the first clinical response achieved within 168 days after the first study drug dosing date was calculated for participants who achieved a clinical response as: Start date of the first clinical response - the first study drug date +1. A clinical responder was defined as either: a. A baseline red blood cell (RBC)-transfusion-dependent participant with a ≥ 56 consecutive day RBC transfusion-free period after the first dose of study drug, or b. A baseline RBC-transfusion-independent participant with an increase in hemoglobin of 2.0 g/dL or more from baseline for ≥ 56 consecutive days in the absence of RBC transfusions, or c. A participant with either a ≥ 50% reduction in palpable splenomegaly of a spleen that was ≥ 10 cm at baseline or a spleen that was palpable at > 5 cm and became not palpable. Intent-to-treat population with a clinical response
End point type
Secondary
End point timeframe
Up to 168 days
End point values
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Number of subjects analysed
11
4
4
10
Units: weeks
    median (full range (min-max))
0.3 (0.1 to 15.6)
8 (2.6 to 17.3)
10.1 (0.1 to 20)
1.2 (0.1 to 16.6)
No statistical analyses for this end point

Secondary: Duration of First Clinical Response

 Close Top of page
End point title
 Duration of First Clinical Response
End point description
For RBC-transfusion-dependent patients, duration of response was calculated as the last day of response - first day of response +1, where the last day of response was the date of the first RBC-transfusion administered at or more than 56 days after the response started. For patients who did not receive a subsequent transfusion after the response started, the end date of response was censored at the day of last hemoglobin assessment. For RBC-transfusion-independent patients, the duration of response was calculated as the last day of response - first day of response +1, where the last day of response was the earlier of the date of a hemoglobin increase of < 2.0 g/dL and the date of a RBC transfusion at ≥ 56 days after the response started. For patients whose hemoglobin measurements were always ≥ 2.0 g/dL and never received a RBC transfusion after response started, the end date of the response was censored at the date of last hemoglobin measurement. Kaplan-Meier methodology was used.
End point type
Secondary
End point timeframe
Up to 40 months
End point values
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Number of subjects analysed
11
4 [1]
4
10
Units: months
    median (confidence interval 95%)
3.7 (3 to 6.6)
9999 (4.7 to 9999)
6 (2.3 to 9.8)
10.6 (2.8 to 16.1)
Notes
[1] - Median not estimable as only 1 patient progressed in this group as defined by 9999
No statistical analyses for this end point

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

 Close Top of page
End point title
 Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Subscale and Total Scores
End point description
The FACT-An comprises the four subscales of the 27-item FACT-General Scale (FACT-G), Physical Well-being, Social/Family Well-being, Emotion Well-being, Functional Well-Being, and the Additional Concerns Anemia subscale. Questions are rated on a scale from 0 to 4, where higher scores indicate more impact on quality of life. • Physical Well-being consists of 7 questions, the subscale score ranges from 0-28; • Social/Family Well-being consists of 7 questions, the subscale score ranges from 0-28; • Emotion Well-being consists of 6 questions, the subscale score ranges from 0-24; • Functional Well-Being consists of 7 questions, the subscale score ranges from 0-28; • Anemia subscale consists of 20 questions, the subscale score ranges from 0-80; • Total FACT-An score ranges from 0-188. Intent-to-treat patients with available data.
End point type
Secondary
End point timeframe
Baseline and Cycle 6 (168 days).
End point values
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Number of subjects analysed
9
7
3
12
Units: units on a scale
arithmetic mean (standard deviation)
    Physical Well-Being subscale
0.6 ± 1.5
0.4 ± 6.42
5.3 ± 4.04
2.3 ± 2.26
    Social/Family Well-Being subscale
1.9 ± 3.08
-1.9 ± 2.59
1.7 ± 3.79
0.9 ± 6.84
    Emotional Well-Being subscale
1.3 ± 3.32
0 ± 4.76
-0.3 ± 2.7
1.7 ± 3.47
    Functional Well-Being subscale
0.9 ± 4.14
-2.1 ± 8.99
2.7 ± 3.06
2.5 ± 6.5
    Anemia subscale
1.2 ± 9.47
2.3 ± 21.34
19.3 ± 18.93
5.8 ± 8.85
    total Fact-Anemia Score
2.3 ± 12.42
1.6 ± 36.51
27.3 ± 25.74
11.4 ± 13.51
No statistical analyses for this end point

Secondary: Change From Baseline in Hemoglobin Concentration for Responders

 Close Top of page
End point title
 Change From Baseline in Hemoglobin Concentration for Responders
End point description
Change from Baseline in hemoglobin for participants with a clinical response within the first 6 cycles of treatment. Intent-to-treat participants with a clinical response and available hemoglobin values at each time point.
End point type
Secondary
End point timeframe
Baseline, Cycle 6 (168 days)
End point values
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Number of subjects analysed
6
2
0 [2]
8
Units: g/dL
    median (full range (min-max))
1.4 (-0.5 to 4.1)
2 (0.7 to 3.2)
( to )
-0.1 (-1.9 to 3.9)
Notes
[2] - No participants with a hemoglobin response in this group
No statistical analyses for this end point

Secondary: Change From Baseline in Hemoglobin Concentration for Non-Responders

 Close Top of page
End point title
 Change From Baseline in Hemoglobin Concentration for Non-Responders
End point description
Change from Baseline in hemoglobin for participants without a clinical response within the first 6 cycles of treatment. Intent-to-treat participants with no clinical response and available hemoglobin values at each time point.
End point type
Secondary
End point timeframe
Baseline, Cycle 6 (168 days)
End point values
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Number of subjects analysed
3
5
5
6
Units: g/dL
    median (full range (min-max))
1.2 (-0.2 to 2.7)
0.1 (-0.8 to 1.3)
-0.8 (-2 to 1.9)
0.5 (-0.3 to 1)
No statistical analyses for this end point

Secondary: Change From Baseline in Likert Abdominal Pain Scale

 Close Top of page
End point title
 Change From Baseline in Likert Abdominal Pain Scale
End point description
Participants rated abdominal discomfort or pain over the previous week on a scale from zero to ten, where zero is no discomfort or pain and ten is the worst pain imaginable. Intent-to-treat patients with available data.
End point type
Secondary
End point timeframe
Baseline and Cycle 6 (168 days)
End point values
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Number of subjects analysed
10
7
3
12
Units: units on a scale
    arithmetic mean (standard deviation)
0.3 ± 1.83
-1 ± 3.11
0.3 ± 1.15
-0.1 ± 1.68
No statistical analyses for this end point

Secondary: Number of Participants With Adverse Events (AEs)

 Close Top of page
End point title
 Number of Participants With Adverse Events (AEs)
End point description
A serious AE (SAE) was defined as any AE which resulted in death or was life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or constituted an important medical event (events that may have jeopardized the patient or required intervention to prevent one of the outcomes listed above). The severity of AEs were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 3.0) or according to the following scale: Grade 1 = Mild; Grade 2 = Moderate; Grade 3 = Severe; Grade 4 = Life-threatening; Grade 5 = Death. The Investigator determined the relationship between study drug and the occurrence of an AE as "Not Related" or "Related" (since the study was double-blinded, a patient receiving only prednisone could have an AE that was judged as related to pomalidomide, and vice-versa). Safety population (all treated patients).
End point type
Secondary
End point timeframe
From date of the first dose of the study drug until discontinuation or the data cut-off date (up to approximately 45 months).
End point values
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Number of subjects analysed
22
22
19
22
Units: Participants
number (not applicable)
    At least one AE
20
21
18
21
    At least one AE related to pomalidomide
15
17
16
15
    At least one AE related to prednisone
10
10
11
5
    At least one Grade 3-4 AE
10
14
13
15
    At least one Grade 3-4 AE related to pomalidomide
6
7
11
6
    At least one Grade 3-4 AE related to prednisone
5
2
6
3
    At least one SAE
6
10
11
8
    At least one SAE related to pomalidomide
4
6
8
3
    At least one SAE related to prednisone
4
3
5
3
    AE leading to discontinuation of pomalidomide
7
11
5
6
    AE leading to discontinuation of prednisone
5
7
2
1
    AE leading to a dose reduction of pomalidomide
0
2
1
1
    AE leading to a dose interruption of pomalidomide
5
9
9
7
    AE leading to a dose interruption of prednisone
2
8
6
3
No statistical analyses for this end point

Secondary: Percentage of Participants With Clinical Response by Baseline JAK2 Positive Assessment

 Close Top of page
End point title
 Percentage of Participants With Clinical Response by Baseline JAK2 Positive Assessment
End point description
Percentage of participants who achieved a clinical response, presented by participants with positive janus kinase 2 (JAK2) V617F mutation results at Baseline. Includes Intent-to-treat population with non-missing JAK2 Baseline assessment results. The number of participants analyzed indicates the number of participants with a positive JAK2 result for each treatment group respectively.
End point type
Secondary
End point timeframe
Up to 336 days
End point values
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Number of subjects analysed
13 [3]
11 [4]
10 [5]
9 [6]
Units: Percentage of participants
    number (not applicable)
46.2
27.3
30
66.7
Notes
[3] - Includes those who were positive for the janus kinase 2 (JAK2) V617F results.
[4] - Includes those who were positive for the janus kinase 2 (JAK2) V617F results.
[5] - Includes those who were positive for the janus kinase 2 (JAK2) V617F results.
[6] - Includes those who were positive for the janus kinase 2 (JAK2) V617F results.
No statistical analyses for this end point

Secondary: Percentage of Participants With Clinical Response by Baseline JAK2 Negative Assessment

 Close Top of page
End point title
 Percentage of Participants With Clinical Response by Baseline JAK2 Negative Assessment
End point description
Percentage of participants who achieved a clinical response, presented by participants with negative janus kinase 2 (JAK2) V617F mutation results at Baseline. Includes Intent-to-treat population with non-missing JAK2 Baseline assessment results. The number of participants analyzed indicates the number of participants with a negative JAK2 result for each treatment group respectively.
End point type
Secondary
End point timeframe
Up to 336 days
End point values
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Number of subjects analysed
6 [7]
7 [8]
8 [9]
8 [10]
Units: Percentage of participants who achieved
    number (not applicable)
50
28.6
12.5
25
Notes
[7] - Includes those who were negative for the janus kinase 2 (JAK2) V617F results.
[8] - Includes those who were negative for the janus kinase 2 (JAK2) V617F results.
[9] - Includes those who were negative for the janus kinase 2 (JAK2) V617F results.
[10] - Includes those who were negative for the janus kinase 2 (JAK2) V617F results.
No statistical analyses for this end point

Adverse events

 Close Top of page
Adverse events information
Timeframe for reporting adverse events
From the first dose of the study drug through to 30 days after the last dose; up to the data cut-off date of 18 December 2013; up to 81 months
Assessment type
 Systematic
Dictionary used for adverse event reporting
Dictionary name
 MedDRA
Dictionary version
15.0
Reporting groups
Reporting group title
Prednisone
Reporting group description
 Participants received oral prednisone from Day 1-28 of each 28-day cycle for up to 3 cycles (84 days), 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day, and pomalidomide placebo tablets on Days 1-28 for up to 12 cycles in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, participants were discontinued from the study

Reporting group title
Pomalidomide 2 mg
Reporting group description
 Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and prednisone placebo tablets on Days 1-28 for the first 3 cycles in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal. Period Title: Overall Study

Reporting group title
Pomalidomide 2 mg + Prednisone
Reporting group description
 Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone capsules on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Reporting group title
Pomalidomide 0.5 mg + Prednisone
Reporting group description
 Participants received 0.5 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, eligible participants continued to receive oral pomalidomide 0.5 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Serious adverse events
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Total subjects affected by serious adverse events
     subjects affected / exposed
6 / 22 (27.27%)
10 / 22 (45.45%)
11 / 19 (57.89%)
8 / 22 (36.36%)
     number of deaths (all causes)
2
3
4
1
     number of deaths resulting from adverse events
2
3
3
0
Vascular disorders
Deep Vein Thrombosis
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
1 / 1
1 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Hypotension
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
General disorders and administration site conditions
Asthenia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Chills
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
2 / 2
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Disease Progression
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 1
0 / 0
0 / 0
Non-Cardiac Chest Pain
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Pyrexia
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
2 / 3
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Psychiatric disorders
Mental Status Changes
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Injury, poisoning and procedural complications
Thoracic vertebral fracture
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Craniocerebral injury
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 1
0 / 0
0 / 0
Investigations
International Normalised Ratio Increased
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Cardiac disorders
Atrial Fibrillation
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Atrial Flutter
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
1 / 1
0 / 0
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Bradycardia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
1 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Cardiac Failure
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 1
0 / 0
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Cardiac Failure Acute
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Cardiac failure congestive
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 3
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Left ventricular dysfunction
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Myocardial Infarction
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 1
0 / 0
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Right ventricular failure
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Respiratory, thoracic and mediastinal disorders
Hypoxia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
1 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Lung infiltration
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
1 / 1
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Pleural effusion
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
1 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Pulmonary Embolism
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
1 / 1
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Pulmonary hypertension
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Respiratory Failure
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
2 / 19 (10.53%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
2 / 2
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 1
1 / 1
0 / 0
Blood and lymphatic system disorders
Acquired Von Willebrand Disease
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
1 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Anaemia
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
1 / 19 (5.26%)
2 / 22 (9.09%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
0 / 1
0 / 2
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Eosinophilia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
1 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Febrile Neutropenia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
1 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Haemolytic Anaemia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Leukocytosis
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
1 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Neutropenia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
1 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Nervous system disorders
Cerebral haemorrhage
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
1 / 1
0 / 0
0 / 0
0 / 0
     deaths causally related to treatment / all
1 / 1
0 / 0
0 / 0
0 / 0
Cerebrovascular Accident
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
1 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Cognitive disorder
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Hepatic encephalopathy
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 1
0 / 0
Memory Impairment
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
1 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Gastrointestinal disorders
Abdominal pain
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
1 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Ascites
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 2
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Colitis
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Constipation
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
1 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Diarrhoea
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
1 / 1
0 / 0
1 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Gastrointestinal haemorrhage
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 2
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Lower gastrointestinal haemorrhage
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Varices oesophageal
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Renal and urinary disorders
Renal Failure
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Renal Failure Acute
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
2 / 2
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Renal Failure Chronic
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Musculoskeletal and connective tissue disorders
Lung infection pseudomonal
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Endocrine disorders
Hyperthyroidism
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Metabolism and nutrition disorders
Cachexia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 2
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Dehydration
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 1
0 / 0
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Failure to thrive
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
1 / 1
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Fluid retention
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
1 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Hyperglycaemia
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
1 / 1
0 / 0
1 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Hyperuricaemia
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 1
0 / 0
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Infections and infestations
Bronchitis
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
0 / 0
1 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Cellulitis
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Diverticulitis
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Lobar Pneumonia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Perirectal abscess
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
0 / 0
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Pneumonia
     subjects affected / exposed
1 / 22 (4.55%)
3 / 22 (13.64%)
3 / 19 (15.79%)
2 / 22 (9.09%)
     occurrences causally related to treatment / all
1 / 1
1 / 5
1 / 4
2 / 5
     deaths causally related to treatment / all
0 / 0
0 / 1
0 / 0
0 / 0
Respiratory tract infection
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Septic shock
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
1 / 1
0 / 0
0 / 0
0 / 0
     deaths causally related to treatment / all
1 / 1
0 / 0
0 / 0
0 / 0
Urinary tract infection enterococcal
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences causally related to treatment / all
0 / 0
0 / 0
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
0 / 0
0 / 0
Frequency threshold for reporting non-serious adverse events: 5%
Non-serious adverse events
 Prednisone Pomalidomide 2 mg Pomalidomide 2 mg + Prednisone Pomalidomide 0.5 mg + Prednisone
Total subjects affected by non serious adverse events
     subjects affected / exposed
20 / 22 (90.91%)
20 / 22 (90.91%)
17 / 19 (89.47%)
21 / 22 (95.45%)
Vascular disorders
Haematoma
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
2 / 22 (9.09%)
     occurrences all number
0
0
0
3
Hypertension
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
1
0
1
0
Hypotension
     subjects affected / exposed
1 / 22 (4.55%)
2 / 22 (9.09%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences all number
1
3
0
0
Immune system disorders
Seasonal Allergy
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
1
0
1
0
General disorders and administration site conditions
Asthenia
     subjects affected / exposed
1 / 22 (4.55%)
3 / 22 (13.64%)
1 / 19 (5.26%)
4 / 22 (18.18%)
     occurrences all number
1
4
1
7
Chills
     subjects affected / exposed
2 / 22 (9.09%)
2 / 22 (9.09%)
1 / 19 (5.26%)
4 / 22 (18.18%)
     occurrences all number
4
2
2
5
Fatigue
     subjects affected / exposed
6 / 22 (27.27%)
2 / 22 (9.09%)
6 / 19 (31.58%)
7 / 22 (31.82%)
     occurrences all number
17
3
12
27
Feeling jittery
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Gait Disturbance
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences all number
0
8
0
0
Oedema
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
0 / 19 (0.00%)
2 / 22 (9.09%)
     occurrences all number
0
2
0
2
Oedema peripheral
     subjects affected / exposed
6 / 22 (27.27%)
8 / 22 (36.36%)
10 / 19 (52.63%)
10 / 22 (45.45%)
     occurrences all number
7
8
24
20
Pain
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
0 / 19 (0.00%)
2 / 22 (9.09%)
     occurrences all number
0
2
0
3
Psychiatric disorders
Confusional State
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
2 / 19 (10.53%)
0 / 22 (0.00%)
     occurrences all number
0
1
2
0
Depression
     subjects affected / exposed
1 / 22 (4.55%)
2 / 22 (9.09%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
1
2
1
0
Insomnia
     subjects affected / exposed
4 / 22 (18.18%)
2 / 22 (9.09%)
2 / 19 (10.53%)
3 / 22 (13.64%)
     occurrences all number
6
2
2
5
Mental Status Changes
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
1
1
0
Personality Change
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Reproductive system and breast disorders
Benign Prostatic Hyperplasia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Injury, poisoning and procedural complications
Contusion
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
2 / 19 (10.53%)
1 / 22 (4.55%)
     occurrences all number
0
2
2
1
Excoriation
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Laceration
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Investigations
Cardiac Murmur
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
0 / 19 (0.00%)
2 / 22 (9.09%)
     occurrences all number
0
2
0
2
Heart Rate Increased
     subjects affected / exposed
2 / 22 (9.09%)
0 / 22 (0.00%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences all number
3
0
0
0
Weight decreased
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
0 / 19 (0.00%)
2 / 22 (9.09%)
     occurrences all number
1
0
0
3
Cardiac disorders
Arrhythmia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Palpitations
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
2 / 19 (10.53%)
0 / 22 (0.00%)
     occurrences all number
1
0
2
0
Sinus Bradycardia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Tachycardia
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences all number
0
2
0
1
Respiratory, thoracic and mediastinal disorders
Cough
     subjects affected / exposed
6 / 22 (27.27%)
6 / 22 (27.27%)
4 / 19 (21.05%)
8 / 22 (36.36%)
     occurrences all number
6
6
4
12
Dysphonia
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
2 / 19 (10.53%)
2 / 22 (9.09%)
     occurrences all number
0
1
4
2
Dyspnoea
     subjects affected / exposed
7 / 22 (31.82%)
7 / 22 (31.82%)
3 / 19 (15.79%)
6 / 22 (27.27%)
     occurrences all number
7
9
6
24
Dyspnoea Exertional
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences all number
0
3
0
0
Epistaxis
     subjects affected / exposed
1 / 22 (4.55%)
2 / 22 (9.09%)
5 / 19 (26.32%)
3 / 22 (13.64%)
     occurrences all number
1
2
6
5
Nasal Congestion
     subjects affected / exposed
3 / 22 (13.64%)
0 / 22 (0.00%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences all number
3
0
0
0
Oropharyngeal Pain
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
3 / 22 (13.64%)
     occurrences all number
0
0
1
4
Pleural Effusion
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
2
1
0
Pleuritic Pain
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Pulmonary Hypertension
     subjects affected / exposed
1 / 22 (4.55%)
2 / 22 (9.09%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences all number
1
2
0
0
Blood and lymphatic system disorders
Anaemia
     subjects affected / exposed
2 / 22 (9.09%)
5 / 22 (22.73%)
3 / 19 (15.79%)
5 / 22 (22.73%)
     occurrences all number
2
7
4
12
Leukopenia
     subjects affected / exposed
0 / 22 (0.00%)
3 / 22 (13.64%)
0 / 19 (0.00%)
2 / 22 (9.09%)
     occurrences all number
0
4
0
5
Neutropenia
     subjects affected / exposed
1 / 22 (4.55%)
5 / 22 (22.73%)
3 / 19 (15.79%)
3 / 22 (13.64%)
     occurrences all number
1
21
13
6
Thrombocytopenia
     subjects affected / exposed
2 / 22 (9.09%)
5 / 22 (22.73%)
3 / 19 (15.79%)
3 / 22 (13.64%)
     occurrences all number
2
10
9
3
Thrombocytosis
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
1 / 19 (5.26%)
1 / 22 (4.55%)
     occurrences all number
0
1
1
2
Nervous system disorders
Aphonia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Balance Disorder
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
2 / 22 (9.09%)
     occurrences all number
0
0
0
2
Burning Sensation
     subjects affected / exposed
3 / 22 (13.64%)
0 / 22 (0.00%)
0 / 19 (0.00%)
2 / 22 (9.09%)
     occurrences all number
3
0
0
2
Dementia Alzheimer's type
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Dizziness
     subjects affected / exposed
4 / 22 (18.18%)
2 / 22 (9.09%)
6 / 19 (31.58%)
8 / 22 (36.36%)
     occurrences all number
4
2
11
37
Dysgeusia
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
1
0
1
0
Headache
     subjects affected / exposed
2 / 22 (9.09%)
2 / 22 (9.09%)
0 / 19 (0.00%)
4 / 22 (18.18%)
     occurrences all number
2
2
0
5
Hypoaesthesia
     subjects affected / exposed
1 / 22 (4.55%)
1 / 22 (4.55%)
1 / 19 (5.26%)
2 / 22 (9.09%)
     occurrences all number
2
2
2
12
Memory Impairment
     subjects affected / exposed
1 / 22 (4.55%)
1 / 22 (4.55%)
2 / 19 (10.53%)
0 / 22 (0.00%)
     occurrences all number
1
3
3
0
Migraine
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Paraesthesia
     subjects affected / exposed
4 / 22 (18.18%)
2 / 22 (9.09%)
3 / 19 (15.79%)
4 / 22 (18.18%)
     occurrences all number
5
2
4
15
Tremor
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences all number
0
3
0
0
Eye disorders
Eye Irritation
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
2 / 19 (10.53%)
1 / 22 (4.55%)
     occurrences all number
0
1
2
10
Lacrimation Increased
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
2 / 19 (10.53%)
0 / 22 (0.00%)
     occurrences all number
0
0
2
0
Periorbital Oedema
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
1 / 22 (4.55%)
     occurrences all number
0
0
1
10
Vision blurred
     subjects affected / exposed
3 / 22 (13.64%)
2 / 22 (9.09%)
4 / 19 (21.05%)
1 / 22 (4.55%)
     occurrences all number
3
6
4
1
Ear and labyrinth disorders
Ear congestion
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Tinnitus
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
1
1
0
Gastrointestinal disorders
Abdominal distension
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
3 / 22 (13.64%)
     occurrences all number
0
1
0
3
Abdominal Pain
     subjects affected / exposed
4 / 22 (18.18%)
3 / 22 (13.64%)
1 / 19 (5.26%)
5 / 22 (22.73%)
     occurrences all number
5
4
2
12
Abdominal Pain Upper
     subjects affected / exposed
2 / 22 (9.09%)
2 / 22 (9.09%)
1 / 19 (5.26%)
3 / 22 (13.64%)
     occurrences all number
2
2
1
3
Ascites
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
4
0
Constipation
     subjects affected / exposed
2 / 22 (9.09%)
2 / 22 (9.09%)
5 / 19 (26.32%)
4 / 22 (18.18%)
     occurrences all number
2
6
6
19
Diarrhoea
     subjects affected / exposed
6 / 22 (27.27%)
6 / 22 (27.27%)
8 / 19 (42.11%)
1 / 22 (4.55%)
     occurrences all number
7
10
13
6
Dyspepsia
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
5 / 19 (26.32%)
3 / 22 (13.64%)
     occurrences all number
0
2
5
6
Flatulence
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
0 / 19 (0.00%)
2 / 22 (9.09%)
     occurrences all number
1
0
0
2
Gastrooesophageal Reflux Disease
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
1
0
1
0
Gingival bleeding
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Haematochezia
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
2 / 19 (10.53%)
1 / 22 (4.55%)
     occurrences all number
0
1
2
2
Nausea
     subjects affected / exposed
4 / 22 (18.18%)
4 / 22 (18.18%)
3 / 19 (15.79%)
1 / 22 (4.55%)
     occurrences all number
5
5
7
1
Oral pain
     subjects affected / exposed
2 / 22 (9.09%)
0 / 22 (0.00%)
0 / 19 (0.00%)
1 / 22 (4.55%)
     occurrences all number
2
0
0
1
Tongue ulceration
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Vomiting
     subjects affected / exposed
2 / 22 (9.09%)
3 / 22 (13.64%)
1 / 19 (5.26%)
3 / 22 (13.64%)
     occurrences all number
2
3
1
8
Renal and urinary disorders
Haematuria
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
1
1
0
Pollakiuria
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
2 / 19 (10.53%)
0 / 22 (0.00%)
     occurrences all number
1
0
2
0
Urinary Incontinence
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Hepatobiliary disorders
Hyperbilirubinaemia
     subjects affected / exposed
1 / 22 (4.55%)
0 / 22 (0.00%)
2 / 19 (10.53%)
0 / 22 (0.00%)
     occurrences all number
1
0
3
0
Skin and subcutaneous tissue disorders
Dry Skin
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences all number
0
2
0
0
Ecchymosis
     subjects affected / exposed
1 / 22 (4.55%)
3 / 22 (13.64%)
1 / 19 (5.26%)
1 / 22 (4.55%)
     occurrences all number
1
3
1
1
Erythema
     subjects affected / exposed
0 / 22 (0.00%)
2 / 22 (9.09%)
0 / 19 (0.00%)
4 / 22 (18.18%)
     occurrences all number
0
2
0
5
Increased Tendency To Bruise
     subjects affected / exposed
2 / 22 (9.09%)
0 / 22 (0.00%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences all number
2
0
0
0
Night Sweats
     subjects affected / exposed
2 / 22 (9.09%)
2 / 22 (9.09%)
1 / 19 (5.26%)
5 / 22 (22.73%)
     occurrences all number
3
2
1
8
Pruritus
     subjects affected / exposed
1 / 22 (4.55%)
1 / 22 (4.55%)
2 / 19 (10.53%)
3 / 22 (13.64%)
     occurrences all number
1
2
2
6
Rash
     subjects affected / exposed
1 / 22 (4.55%)
8 / 22 (36.36%)
3 / 19 (15.79%)
3 / 22 (13.64%)
     occurrences all number
1
14
3
6
Rash Pruritic
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Skin Odour Abnormal
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Musculoskeletal and connective tissue disorders
Arthralgia
     subjects affected / exposed
3 / 22 (13.64%)
3 / 22 (13.64%)
1 / 19 (5.26%)
2 / 22 (9.09%)
     occurrences all number
5
3
1
5
Back Pain
     subjects affected / exposed
1 / 22 (4.55%)
2 / 22 (9.09%)
2 / 19 (10.53%)
1 / 22 (4.55%)
     occurrences all number
1
3
2
1
Bone Pain
     subjects affected / exposed
1 / 22 (4.55%)
3 / 22 (13.64%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences all number
1
3
0
0
Muscle Spasms
     subjects affected / exposed
3 / 22 (13.64%)
1 / 22 (4.55%)
6 / 19 (31.58%)
5 / 22 (22.73%)
     occurrences all number
4
1
6
6
Muscular Weakness
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
0 / 19 (0.00%)
3 / 22 (13.64%)
     occurrences all number
0
1
0
4
Musculoskeletal Chest Pain
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
2 / 19 (10.53%)
0 / 22 (0.00%)
     occurrences all number
0
0
2
0
Myalgia
     subjects affected / exposed
1 / 22 (4.55%)
2 / 22 (9.09%)
1 / 19 (5.26%)
4 / 22 (18.18%)
     occurrences all number
7
7
1
24
Osteoarthritis
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Pain In Extremity
     subjects affected / exposed
2 / 22 (9.09%)
4 / 22 (18.18%)
2 / 19 (10.53%)
3 / 22 (13.64%)
     occurrences all number
2
5
3
3
Endocrine disorders
Hypothyroidism
     subjects affected / exposed
0 / 22 (0.00%)
3 / 22 (13.64%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences all number
0
3
0
0
Metabolism and nutrition disorders
Decreased Appetite
     subjects affected / exposed
1 / 22 (4.55%)
1 / 22 (4.55%)
0 / 19 (0.00%)
3 / 22 (13.64%)
     occurrences all number
1
1
0
4
Gout
     subjects affected / exposed
0 / 22 (0.00%)
1 / 22 (4.55%)
1 / 19 (5.26%)
1 / 22 (4.55%)
     occurrences all number
0
1
1
1
Hyperglycaemia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
2 / 22 (9.09%)
     occurrences all number
0
0
0
2
Hyperuricaemia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
3 / 22 (13.64%)
     occurrences all number
0
0
1
3
Hypokalaemia
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
2 / 22 (9.09%)
     occurrences all number
0
0
1
2
Infections and infestations
Cystitis
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
1 / 22 (4.55%)
     occurrences all number
0
0
1
1
Enterococcal Sepsis
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Eye infection
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
0 / 22 (0.00%)
     occurrences all number
0
0
1
0
Influenza
     subjects affected / exposed
1 / 22 (4.55%)
1 / 22 (4.55%)
2 / 19 (10.53%)
2 / 22 (9.09%)
     occurrences all number
2
3
2
3
Nasopharyngitis
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
3 / 22 (13.64%)
     occurrences all number
0
0
0
7
Pneumonia
     subjects affected / exposed
2 / 22 (9.09%)
2 / 22 (9.09%)
0 / 19 (0.00%)
0 / 22 (0.00%)
     occurrences all number
2
5
0
0
Respiratory tract infection
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
0 / 19 (0.00%)
2 / 22 (9.09%)
     occurrences all number
0
0
0
2
Upper Respiratory Tract Infection
     subjects affected / exposed
3 / 22 (13.64%)
2 / 22 (9.09%)
1 / 19 (5.26%)
2 / 22 (9.09%)
     occurrences all number
3
3
1
3
Urinary tract infection
     subjects affected / exposed
0 / 22 (0.00%)
0 / 22 (0.00%)
1 / 19 (5.26%)
1 / 22 (4.55%)
     occurrences all number
0
0
2
1

More information

 Close Top of page

Substantial protocol amendments (globally)
Were there any global substantial amendments to the protocol? Yes
Date
Amendment
08 Jan 2008
For the European Union only and the following included: 1. The inclusion of new study personnel and central lab 2. The pomalidomide warning label and handling instructions 4. Allowing for additional visits, and minor administrative wording changes
13 Aug 2008
The protocol was amended (for all countries) to add the extension phase and for miscellaneous minor changes
13 Feb 2009
The protocol was amended (for all countries) to update information on: 1. An update to the study drug packaging and storage 2. A revision to the extension phase from 12 cycles to open-ended for responders 3. Minor grammatical revisions.

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

Sponsors and Collaborators

Medical Conditions

Drug Interventions

3
Subscribe