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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01311687




Registration number
NCT01311687
Ethics application status
Date submitted
8/03/2011
Date registered
9/03/2011
Date last updated
24/10/2018

Titles & IDs
Public title
A Phase 3, Multicenter, Randomized, Open-Label Study to Compare the Efficacy and Safety of Pomalidomide in Combination With Low-Dose Dexamethasone Versus High-Dose Dexamethasone in Subjects With Refractory Multiple Myeloma or Relapsed and Refractory Multiple Myeloma and Companion Study
Scientific title
A Phase 3, Muticenter, Randomized, Open-label Study to Compare the Efficacy and Safety of Pomalidomide in Combination With Low-dose Dexamethasone Versus High-dose Dexamethasone in Subjects With Refractory or Relapsed and Refractory Multiple Myeloma
Secondary ID [1] 0 0
2010-019820-30
Secondary ID [2] 0 0
CC-4047-MM-003
Universal Trial Number (UTN)
Trial acronym
NIMBUS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Multiple Myeloma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Other cancer types

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - pomalidomide
Treatment: Drugs - Dexamethasone

Experimental: Pomalidomide + Low-Dose Dexamethasone - Participants received 4 mg pomalidomide administered by mouth on Days 1 to 21 of each 28-day treatment cycle and 40 mg dexamethasone (or 20 mg for participants > 75 years of age) administered by mouth once per day on Days 1, 8, 15, and 22 of each 28-day cycle until disease progression.

Active Comparator: High-Dose Dexamethasone - Participants received 40 mg dexamethasone (or 20 mg for participants > 75 years of age) administered by mouth once per day on Days 1 to 4, 9 to 12, and 17 to 20 of each 28-day treatment cycle until disease progression.


Treatment: Drugs: pomalidomide
4 mg pomalidomide capsules administered orally

Treatment: Drugs: Dexamethasone
40 mg dexamethasone (or 20 mg for participants > 75 years of age) tablets administered orally

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Progression-free Survival (PFS) - Primary Analysis - Progression-free survival was calculated as the time from randomization to disease progression as determined by the Independent Response Adjudication Committee based on the International Myeloma Working Group Uniform Response criteria (IMWG), or death on study, whichever occurred earlier. Progressive disease required 1 of the following: • Increase of = 25% from nadir in: o Serum M-component (absolute increase = 0.5 g/dl); o Urine M-component (absolute increase = 200 mg/24 hours); o Bone marrow plasma cell percentage (absolute % = 10%); • Development of new or increase in the size of existing bone lesions or soft tissue plasmacytomas; • Development of hypercalcemia (corrected serum calcium > 11.5 mg/dl) attributed solely to plasma cell proliferative disease.
Timepoint [1] 0 0
From randomization until the data cut-off date of 07 September 2012. Maximum duration of follow-up for PFS assessments was 57 weeks.
Primary outcome [2] 0 0
Progression-free Survival (PFS) With a Later Cut-off Date - Progression-free survival was calculated as the time from randomization to disease progression as determined by the Independent Response Adjudication Committee based on the International Myeloma Working Group Uniform Response criteria (IMWG), or death on study, whichever occurred earlier. Progressive disease requires 1 of the following: • Increase of = 25% from nadir in: o Serum M-component (absolute increase = 0.5 g/dl); o Urine M-component (absolute increase = 200 mg/24 hours); o Bone marrow plasma cell percentage (absolute % = 10%); • Development of new or increase in the size of existing bone lesions or soft tissue plasmacytomas; • Development of hypercalcemia (corrected serum calcium > 11.5 mg/dl) attributed solely to plasma cell proliferative disease.
Timepoint [2] 0 0
From randomization until the data cut-off date of 01 March 2013. Maximum duration of follow-up for PFS assessments was 74 weeks.
Secondary outcome [1] 0 0
Number of Participants With Adverse Events (AEs) - An adverse event is any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. A serious AE is any AE occurring at any dose that: • Results in death; • Is life-threatening; • Requires or prolongs existing inpatient hospitalization; • Results in persistent or significant disability/incapacity; • Is a congenital anomaly/birth defect; • Constitutes an important medical event. The Investigator assessed the relationship of each AE to study drug and graded the severity according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 4.0): Grade 1 = Mild (no limitation in activity or intervention required); Grade 2 = Moderate (some limitation in activity; no/minimal medical intervention required); Grade 3 = Severe (marked limitation in activity; medical intervention required, hospitalization possible); Grade 4 = Life-threatening; Grade 5 = Death.
Timepoint [1] 0 0
From first dose of study drug through to 30 days after the last dose as of the end of the study (29 August 2017); maximum time on treatment was 297, 269, and 239 weeks in the Pomalidomide + LD-Dex, HD-Dex, and cross-over groups respectively.
Secondary outcome [2] 0 0
Overall Survival - Primary Analysis - Overall survival is calculated as the time from randomization to death from any cause. Overall survival was censored at the last date that the participant was known to be alive for participants who were alive at the time of analysis and for participants who were lost to follow-up before death was documented.
Timepoint [2] 0 0
From randomization until the data cut-off date of 07 September 2012. Maximum time on follow-up for survival was 70 weeks.
Secondary outcome [3] 0 0
Overall Survival With a Later Cut-off Date - Overall survival is calculated as the time from randomization to death from any cause. Overall survival was censored at the last date that the participant was known to be alive for participants who were alive at the time of analysis and for participants who were lost to follow-up before death was documented.
Timepoint [3] 0 0
From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up for survival was 93 weeks.
Secondary outcome [4] 0 0
Overall Survival Based on the Final Dataset - Overall survival is calculated as the time from randomization to death from any cause. Overall survival was censored at the last date that the participant was known to be alive for participants who were alive at the time of analysis and for participants who were lost to follow-up before death was documented.
Timepoint [4] 0 0
From randomization until the data cut-off date of 29 August 2017. Maximum time on follow-up for survival was 324 weeks.
Secondary outcome [5] 0 0
Percentage of Participants With an Objective Response According to International Myeloma Working Group (IMWG) Uniform Response Criteria - Objective response is defined as a best overall response of stringent complete response (SCR), complete response (CR), very good partial response (VGPR) or partial response (PR) based on the Independent Response Adjudication Committee: SCR: CR and normal free light chain (FLC) ratio and no clonal cells in bone marrow; CR: Negative serum and urine on immunofixation, disappearance of any soft tissue plasmacytomas and = 5% plasma cells in bone marrow; VGPR: Serum and urine M-protein detectable by immunofixation but not on electrophoresis or = 90% reduction in serum M-protein and urine M-protein level < 100 mg/24 hours; PR: = 50% reduction of serum M-Protein and reduction in urinary M-protein by = 90% or to < 200 mg/24 hours. In addition to the above, if present at baseline a = 50% reduction in the size of soft tissue plasmacytomas is also required.
Timepoint [5] 0 0
From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.
Secondary outcome [6] 0 0
Percentage of Participants With Objective Response According to European Group for Blood and Marrow Transplantation (EBMT) Criteria - Objective response defined as a best overall response of complete response (CR) or partial response (PR) based on the Independent Response Adjudication Committee: CR requires all of the following: - Absence of original monoclonal paraprotein in serum and urine by immunofixation maintained at least 42 days. - <5% plasma cell in bone marrow aspirate and on bone marrow biopsy, if performed. - No increase in size or number of lytic bone lesions. - Disappearance of soft tissue plasmacytomas. PR requires all of the following: - = 50% reduction in level of serum monoclonal paraprotein, maintained at least 42 days. - Reduction in 24-hour urinary light chain extraction by = 90% or to < 200 mg, maintained at least 42 days. - For patients with non-secretory myeloma, = 50% reduction in plasma cells in bone marrow aspirate and on biopsy, if performed, for at least 42 days. - = 50% reduction in the size of soft tissue plasmacytomas. - No increase in size or number of lytic bone lesions.
Timepoint [6] 0 0
From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.
Secondary outcome [7] 0 0
Time to Progression - Time to progression (TTP) is calculated as the time from randomization to the first documented progression confirmed by a blinded, independent Response Adjudication Committee and based on the International Myeloma Working Group Uniform Response criteria (IMWG). Progressive disease requires 1 of the following: • Increase of = 25% from nadir in: o Serum M-component (absolute increase = 0.5 g/dl); o Urine M-component (absolute increase = 200 mg/24 hours); o Bone marrow plasma cell percentage (absolute % = 10%); • Development of new or increase in the size of existing bone lesions or soft tissue plasmacytomas; • Development of hypercalcemia (corrected serum calcium > 11.5 mg/dl) attributed solely to plasma cell proliferative disease.
Timepoint [7] 0 0
From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.
Secondary outcome [8] 0 0
Time to Response - Time to response is calculated as the time from randomization to the initial documented response (partial response or better) based on IMWG criteria. SCR: CR and normal free light chain (FLC) ratio and no clonal cells in bone marrow; CR: Negative serum and urine on immunofixation, disappearance of any soft tissue plasmacytomas and = 5% plasma cells in bone marrow; VGPR: Serum and urine M-protein detectable by immunofixation but not on electrophoresis or = 90% reduction in serum M-protein and urine M-protein level < 100 mg/24 hours; PR: = 50% reduction of serum M-Protein and reduction in urinary M-protein by = 90% or to < 200 mg/24 hours. If present at baseline a = 50% reduction in size of soft tissue plasmacytomas is also required.
Timepoint [8] 0 0
From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.
Secondary outcome [9] 0 0
Duration of Response - Duration of response (calculated for responders only) is defined as time from the initial documented response (partial response or better) to confirmed disease progression, based on IMWG criteria assessed by the Independent Response Adjudication Committee.
Timepoint [9] 0 0
From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.
Secondary outcome [10] 0 0
Time to the First Hemoglobin Improvement - Time to increased hemoglobin, defined as the time from randomization to at least one category improvement from Baseline in common terminology criteria for adverse events (CTCAE) grade for hemoglobin level. Hemoglobin categories are: 1) Normal; 2) CTCAE Grade 1: < lower limit of normal (LLN) to 10.0 g/dL; 3) CTCAE Grade 2: < 10.0 to <8.0 g/dL. Participants with CTCAE Grade 3 anemia or worse at Baseline were excluded from the study.
Timepoint [10] 0 0
From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.
Secondary outcome [11] 0 0
Time to Improvement in Bone Pain - Time to improvement in bone pain is defined as the time from randomization to at least one category improvement from Baseline in bone pain category. Bone pain was categorized (from best to worst) according to answers to the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire for patients with Multiple Myeloma Module (QLQ-MY20), Question 1, "Have you had bone aches or pain?": 1) Not at all, 2) A little, 3) Quite a bit, or 4) Very much.
Timepoint [11] 0 0
From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.
Secondary outcome [12] 0 0
Time to Improvement in Renal Function - Time to improvement in renal function is defined as the time from randomization to at least one category improvement from Baseline in renal function. Renal Function was categorized as (from best to worst): - Normal: creatinine clearance =80 mL/min; - Grade 1: creatinine clearance =60 to <80 mL/min; - Grade 2 : creatinine clearance =45 to < 60 mL/min. Participants with creatinine clearance < 45 mL/min at baseline were excluded from the study.
Timepoint [12] 0 0
From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.
Secondary outcome [13] 0 0
Time to Improvement in Eastern Cooperative Oncology Group (ECOG) Performance Status - Time to improvement in ECOG performance status defined as the time from randomization until at least a one category improvement from Baseline in ECOG performance status score. The categories of the ECOG Performance Status Scale are as follows: -0: Fully active, able to carry on all pre-disease performance without restriction; -1: Restricted in physically strenuous activity but ambulatory and able to carry our work of a light or sedentary nature, e.g., light housework, office work; -2: Ambulatory and capable of all self-care but unable to carry out any work activities. Up and about more than 50% of waking hours. Patients with a score of 3, 4 or 5 were excluded from participating in the study.
Timepoint [13] 0 0
From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.
Secondary outcome [14] 0 0
Change From Baseline in the European Organization for Research and Treatment of Cancer Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Global Health Status Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Global Health Status/QOL scale is scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in QOL or functioning and positive values indicate improvement.
Timepoint [14] 0 0
Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6
Secondary outcome [15] 0 0
Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Physical Functioning Scale is scored between 0 and 100, with a high score indicating better functioning/support. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.
Timepoint [15] 0 0
Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6
Secondary outcome [16] 0 0
Change From Baseline in the EORTC QLQ-C30 Emotional Functioning Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Emotional Functioning Scale is scored between 0 and 100, with a high score indicating better functioning/support. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.
Timepoint [16] 0 0
Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6
Secondary outcome [17] 0 0
Change From Baseline in the EORTC QLQ-C30 Fatigue Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Fatigue Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reduction in fatigue (i.e. improvement in symptom) and positive values indicate increases in fatigue (i.e. worsening of symptom).
Timepoint [17] 0 0
Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6
Secondary outcome [18] 0 0
Change From Baseline in the EORTC QLQ-C30 Pain Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Pain Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reductions in pain (i.e. improvement in symptom) and positive values indicate increases in pain (i.e. worsening of symptom).
Timepoint [18] 0 0
Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6
Secondary outcome [19] 0 0
Change From Baseline in the European Organization for Research and Treatment of Cancer QoL Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) Disease Symptoms - The European Organization for Research and Treatment of Cancer QoL Questionnaire for Patients with Multiple Myeloma (EORTC QLQ-MY20) is a 20-question tool used in clinical research to assess health-related quality of life in multiple myeloma patients. The QLQ-MY20 includes four domains (Disease Symptoms, Side-Effects of Treatment, Body Image and Future Perspective). The EORTC QLQ-MY20 Disease Symptoms Scale is scored between 0 and 100, with a high score reflecting a higher level of symptoms. Negative change from Baseline values indicate reduction (i.e. improvement) in symptoms and positive values indicate increase (i.e. worsening) of symptoms.
Timepoint [19] 0 0
Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6
Secondary outcome [20] 0 0
Change From Baseline in the EORTC QLQ-MY20 Side Effects Domain - The European Organization for Research and Treatment of Cancer QoL Questionnaire for Patients with Multiple Myeloma (EORTC QLQ-MY20) is a 20-question tool used in clinical research to assess health-related quality of life in multiple myeloma patients. The QLQ-MY20 includes four domains (Disease Symptoms, Side-Effects of Treatment, Body Image and Future Perspective). The EORTC QLQ-MY20 Side Effects Scale is scored between 0 and 100, with a high score reflecting a higher level of symptoms. Negative change from Baseline values indicate reduction in side effects (i.e.improvement in symptom) and positive values indicate increase in side effects (i.e. worsening of symptom).
Timepoint [20] 0 0
Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6
Secondary outcome [21] 0 0
Change From Baseline in the European Quality of Life-5 Dimensions (EQ-5D) Utility Index Score - EQ-5D is a self-administered questionnaire that assesses health-related quality of life (QOL). The EQ-5D descriptive health profile comprises five dimensions of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Each dimension has 3 levels of response: No problem (1), some problems (2), and extreme problems (3). A unique EQ-5D health state is defined by combining one level from each of the five dimensions into a single utility index score. EQ-5D index values range from -0.59 to 1.00 where an EQ-5D score of 1.00 equals "perfect health", a score of 0 equals "death" and a score of -0.59 equals worst imaginable health state. A positive change from Baseline score indicates improvement in health status. A negative change from Baseline score indicates worsening in health status. Negative scores represent the possible though unlikely situation that a patient's QOL is worse than death, i.e. they would rather be dead than living with that QOL
Timepoint [21] 0 0
Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6
Secondary outcome [22] 0 0
Time to First Worsening of Quality of Life (QOL) Domains - Time to worsening in quality of life domains was calculated as the time from Baseline to the first worsened minimally important difference (MID), defined as the smallest change in a QOL score considered important to patients that would lead the patient or clinician to consider a change in therapy. MID thresholds were calculated in Standard Error of Measurement (SEM) units using the Baseline QOL data. Based on the MID, participants were classified as worsened according to the following: For the EORTC QLQ-C30 global health status and functional scales and the EQ-5D health utility score, participants were classified as worsened if their change from Baseline score was less than -1 SEM. For the EORTC QLQ-C30 symptom scores (fatigue and pain) and EORTC QLQ-MY20 disease symptoms and side effects scales, participants were classified as worsened if their change from Baseline score was greater than 1 SEM. See previous outcome measures for definitions of each scale.
Timepoint [22] 0 0
Assessed on Day 1 of the first 6 treatment cycles.

Eligibility
Key inclusion criteria
- Must be = 18 years of age

- Subjects must have documented diagnosis of multiple myeloma and have measurable
disease

- Subjects must have undergone prior treatment with = 2 treatment lines of anti-myeloma
therapy

- Subjects must have either refractory or relapsed and refractory disease defined as
documented disease progression during or within 60 days of completing their last
myeloma therapy

- All subjects must have received at least 2 consecutive cycles of prior treatment that
included lenalidomide and bortezomib

- All subjects must have failed treatment with both lenalidomide and bortezomib in one
of the following ways: 1) Documented progressive disease on or within 60 days of
completing treatment with lenalidomide and/or bortezomib, or 2) In case of prior
response [= partial response (PR)] to lenalidomide or bortezomib, subjects must have
relapsed within 6 months after stopping treatment with lenalidomide and/or
bortezomib-containing regimens, or 3) Subjects who have not had a = minimal response
(MR) and have developed intolerance/toxicity after a minimum of two cycles of
lenalidomide- and/or bortezomib-containing regimen

- Patients must have received adequate prior alkylator therapy

- Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2

- Females of childbearing potential (FCBP) must not become pregnant for 28 days prior to
initiation of study drug, during the study, and for 28 days after discontinuation

- Females must agree to abstain from breastfeeding during study participation and 28
days after study drug discontinuation

- Males must agree to use a latex condom during any sexual during the study and for 28
days following discontinuation from this study

- Males must also agree to refrain from donating semen or sperm while on pomalidomide
and for 28 days after discontinuation from this study
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Any of the following laboratory abnormalities:

- Absolute neutrophil count (ANC) < 1,000/µL

- Platelet count < 75,000/ µL for subjects in whom < 50% of bone marrow nucleated
cells are plasma cells

- Creatinine clearance < 45 mL/min

- Corrected serum calcium > 14 mg/dL

- Hemoglobin = 8 g/dL

- Serum glutamic oxaloacetic transaminase (SGOT)/ aspartate aminotransferase (AST)
or transaminase, serum glutamic pyruvic (SGPT)/ alanine aminotransferase (ALT) >
3.0 x upper limit of normal (ULN)

- Serum total bilirubin > 2.0 mg/dL

- Previous therapy with pomalidomide

- Hypersensitivity to thalidomide, lenalidomide, or dexamethasone

- Resistance to high-dose dexamethasone used in the last line of therapy

- Peripheral neuropathy = Grade 2

- Subjects who received an allogeneic bone marrow or allogeneic peripheral blood stem
cell transplant

- Subjects who are planning for or who are eligible for stem cell transplant

- Subjects with any one of the following: 1) Congestive heart failure, 2) Myocardial
infarction within 12 months prior to starting study treatment, 3) Unstable or poorly
controlled angina pectoris, including Prinzmetal variant angina pectoris

- Subjects who received any of the following within the last 14 days of initiation of
study treatment: 1) Plasmapheresis, 2) Major surgery, 3) Radiation therapy, 4) Use of
any anti-myeloma drug therapy

- Use of any investigational agents within 28 days or 5 half-lives (whichever is longer)
of treatment

- Subjects with conditions requiring chronic steroid or immunosuppressive treatment

- Any condition including the presence of laboratory abnormalities, which places the
subject at unacceptable risk if he/she were to participate in the study

- Incidence of gastrointestinal disease that may significantly alter the absorption of
pomalidomide

- Subjects unable or unwilling to undergo antithrombotic prophylactic treatment

- Any serious medical condition, laboratory abnormality, or psychiatric illness that
would prevent the subjects from signing the informed consent form

- Pregnant or breastfeeding females

- Known human immunodeficiency virus (HIV) positivity or active infectious hepatitis A,
B, or C

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA,VIC
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
Peter MacCallum Cancer Institute - East Melbourne
Recruitment hospital [3] 0 0
Frankston Hospital - Frankston
Recruitment hospital [4] 0 0
Princess Alexandra Hospital - Brisbane
Recruitment hospital [5] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [6] 0 0
Alfred hospital - Melbourne
Recruitment hospital [7] 0 0
Sir Charles Gairdner Hospital - Nedlands
Recruitment hospital [8] 0 0
Calvary Mater Hospital - Waratah
Recruitment hospital [9] 0 0
Border Medical Oncology - Wodonga
Recruitment hospital [10] 0 0
Wollongong Hospital - Wollongong
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3002 - East Melbourne
Recruitment postcode(s) [3] 0 0
3199 - Frankston
Recruitment postcode(s) [4] 0 0
QLD4102 - Brisbane
Recruitment postcode(s) [5] 0 0
2050 - Camperdown
Recruitment postcode(s) [6] 0 0
3004 - Melbourne
Recruitment postcode(s) [7] 0 0
6009 - Nedlands
Recruitment postcode(s) [8] 0 0
NSW 2298 - Waratah
Recruitment postcode(s) [9] 0 0
3690 - Wodonga
Recruitment postcode(s) [10] 0 0
2500 - Wollongong
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Minnesota
Country [2] 0 0
Belgium
State/province [2] 0 0
Gent
Country [3] 0 0
Belgium
State/province [3] 0 0
Leuven
Country [4] 0 0
Belgium
State/province [4] 0 0
Yvoir
Country [5] 0 0
Canada
State/province [5] 0 0
Alberta
Country [6] 0 0
Canada
State/province [6] 0 0
British Columbia
Country [7] 0 0
Canada
State/province [7] 0 0
Nova Scotia
Country [8] 0 0
Canada
State/province [8] 0 0
Ontario
Country [9] 0 0
Canada
State/province [9] 0 0
Quebec
Country [10] 0 0
Czechia
State/province [10] 0 0
Praha 2
Country [11] 0 0
Denmark
State/province [11] 0 0
Aalborg
Country [12] 0 0
Denmark
State/province [12] 0 0
Arhus C
Country [13] 0 0
Denmark
State/province [13] 0 0
Odense C
Country [14] 0 0
Denmark
State/province [14] 0 0
Vejle
Country [15] 0 0
France
State/province [15] 0 0
Angers Cedex 01
Country [16] 0 0
France
State/province [16] 0 0
Bayonne
Country [17] 0 0
France
State/province [17] 0 0
La Roche sur Yon
Country [18] 0 0
France
State/province [18] 0 0
Lille
Country [19] 0 0
France
State/province [19] 0 0
Marseille Cedex 9
Country [20] 0 0
France
State/province [20] 0 0
Nantes Cedex 1
Country [21] 0 0
France
State/province [21] 0 0
Paris, Cedex 10
Country [22] 0 0
France
State/province [22] 0 0
Paris
Country [23] 0 0
France
State/province [23] 0 0
Pessac
Country [24] 0 0
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Celgene
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to compare efficacy and safety of pomalidomide in combination
with low-dose dexamethasone versus high-dose dexamethasone in subjects with refractory or
relapsed and refractory multiple myeloma.
Trial website
https://clinicaltrials.gov/show/NCT01311687
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Lars Sternas, MD, PhD
Address 0 0
Celgene
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications