CompletedNCT01324947Updated Nov 19, 2019Results posted

Study to Evaluate the Safety and Efficacy of Pomalidomide Monotherapy in Subjects With Refractory or Relapsed Refractory Multiple Myeloma

A Phase 3 interventional study of pomalidomide in Multiple Myeloma, sponsored by Celgene. Completed at 91 sites in 15 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2019-11-19.

Sponsored by Celgene (part of Bristol-Myers Squibb) · Phase 3, Interventional, and Treatment

Phase: Phase 3
Study type: Interventional
Enrollment: 74
Allocation: Not applicable
Ages: 18 Years and older
Sex: All

Study summary

The purpose of this study is to evaluate the efficacy and safety of pomalidomide monotherapy in subjects with refractory or relapsed and refractory multiple myeloma who were enrolled in study CC-4047-MM-003 (NCT01311687) and discontinued treatment with high-dose dexamethasone due to disease progression.

Conditions studied

Multiple Myeloma

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Keywords

Myeloma
Multiple Myeloma
Relapsed Multiple Myeloma
Relapsed and Refractory Multiple Myeloma
Refractory Myeloma
Resistant Multiple Myeloma
Treatment-resistant Multiple Myeloma
Pomalidomide
Lenalidomide-resistant
Bortezomib-resistant
Companion

In context

Multiple Myeloma

3,712 studies on the registry are indexed under Multiple Myeloma; 752 are open to participants now.

This study's enrollment of 74 is above the median of 41 across 2,965 interventional studies indexed under Multiple Myeloma.

Browse Multiple Myeloma studies →

Lead sponsor

Celgene is the lead sponsor of 428 studies on the registry; 12 are open to participants now.

Of its 104 completed or terminated interventional studies of FDA-regulated products, 30 (29%) have results posted.

First submitted before Celgene became part of Bristol-Myers Squibb.

Browse Celgene studies →All Bristol-Myers Squibb studies →

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

Subjects with refractory or relapsed and refractory multiple myeloma who were enrolled in Study CC-4047-MM-003 and discontinued study therapy with dexamethasone alone (Treatment Arm B) after at least starting the second cycle of dexamethasone treatment and due to development of documented disease progression according to the International Myeloma Working Group (IMWG) criteria and as decided by an Independent Review Adjudication Committee (IRAC).
Must be ≥ 18 years at the time of signing the informed consent form.
The subject must understand and voluntarily sign an informed consent document prior to any study related assessments/procedures being conducted. The only exception is if a skeletal survey was performed within 90 days prior to the start of Cycle 1, then a new survey will not be required.
Must be able to adhere to the study visit schedule and other protocol requirements.
Subjects must have documented diagnosis of multiple myeloma and have measurable disease (serum M-protein ≥ 0.5g/dL or urine M-protein ≥ 200 mg/24 hours).
Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2.
Females of childbearing potential (FCBP†) must agree to utilize two reliable forms of contraception simultaneously or practice true abstinence [when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post ovulation methods) and withdrawal are not acceptable methods of contraception]from heterosexual contact for at least 28 days before starting study drug, while participating in the study (including dose interruptions), and for at least 28 days after study treatment discontinuation and must agree to regular pregnancy testing during this timeframe.
Females must agree to abstain from breastfeeding during study participation and 28 days after study discontinuation.
Males must agree to either use a latex condom during any sexual contact with FCBP or practice true abstinence [when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g. calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception] while participating in the study and for 28 days following discontinuation from this study, even if he has undergone a successful vasectomy. .
Males must also agree to refrain from donating semen or sperm while on pomalidomide and for 28 days after discontinuation from this study treatment.
All subjects must agree to refrain from donating blood while on study drug and for 28 days after discontinuation from this study treatment.
All subjects must agree not to share study medication

Exclusion criteria

Exclusion Criteria

The presence of any of the following will exclude a subject from enrollment:
1. Subjects with multiple myeloma who were not treated as a part of Study CC-4047-MM-003 (Arm B).
2. Subjects who received any anti-myeloma or anti-cancer therapies within the last 14 days of wash-out period before initiation of study treatment.
3. Subjects who discontinued CC-4047-MM-003 study ≥120 days.
4. Subjects who initiate another anti-myeloma therapy from the time of disease progression on study CC-4047-MM-003 to the time of treatment initiation in the companion study.
5. 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; or a platelet count \< 30,000/µL for subjects in whom ≥ 50% of bone marrow nucleated cells are plasma cells
  - Creatinine Clearance \< 45 mL/min according to Cockcroft-Gault formula (If creatinine clearance calculated from the 24-hour urine sample is ≥45 ml/min, patient will qualify for the trial)
  - Corrected serum calcium > 14 mg/dL (> 3.5 mmol/L);
  - Hemoglobin \< 8 g/dL (\< 4.9 mmol/L; prior RBC transfusion or recombinant human erythropoietin use is permitted)
  - Serum SGOT/AST or SGPT/ALT > 3.0 x upper limit of normal (ULN)
  - Serum total bilirubin > 2.0 mg/dL (34.2 μmol/L); or > 3.0 x ULN for subjects with hereditary benign hyperbilirubinaemia
6. Prior history of malignancies, other than Multiple Myeloma (MM), unless the subject has been free of the disease for ≥ 5 years. Exceptions include the following:
  - Basal or Squamous cell carcinoma of the skin
  - Carcinoma in situ of the cervix or breast
  - Incidental histologic finding of prostate cancer (TNM stage of T1a or T1b)
7. Hypersensitivity to thalidomide or lenalidomide. (This includes ≥ Grade 3 rash during prior thalidomide or lenalidomide therapy).
8. Peripheral neuropathy ≥ Grade 2.
9. Subjects who received an allogeneic bone marrow or allogeneic peripheral blood stem cell transplant less than 12 months prior to initiation of study treatment and who have not discontinued immunosuppressive treatment for at least 4 weeks prior to initiation of study treatment and are currently dependent on such treatment.
10. Subjects who are planning for or who are eligible for stem cell transplant.
11. Subjects with any one of the following:
  - Congestive heart failure (NY Heart Association Class III or IV)
  - Myocardial infarction within 12 months prior to starting study treatment
  - Unstable or poorly controlled angina pectoris, including Prinzmetal variant angina pectoris
12. Subjects who received any of the following within the last 14 days of initiation of study treatment:
  - Plasmapheresis
  - Major surgery (kyphoplasty is not considered major surgery)
  - Radiation therapy
13. Use of any investigational agents within 28 days or 5 half lives (whichever is longer) of treatment.
14. Subjects with chronic conditions such as rheumatoid arthritis, multiple sclerosis and lupus, which likely need additional steroid or immunosuppressive treatments in addition to the study treatment.
15. Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study.
16. Incidence of gastrointestinal disease that may significantly alter the absorption of pomalidomide.
17. Subjects unable or unwilling to undergo antithrombotic prophylactic treatment.
18. Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form.
19. Pregnant or breastfeeding females.
20. Known human immunodeficiency virus (HIV) positivity or active infectious hepatitis A, B or C.

Study design

Phase: Phase 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 74 participants (actual)

Study arms

Experimental
Pomalidomide
Oral pomalidomide 4 mg on Days 1-21 of 28-day cycle until progressive disease (PD) or unacceptable toxicity
Drug: pomalidomide

Interventions

Drugpomalidomide
Oral pomalidomide 4 mg on Days 1-21 of 28-day cycle until progressive disease (PD) or unacceptable toxicity
Also known as: CC-4047

What researchers measure

Primary outcomes

Percentage of Participants With an Objective Response According to International Myeloma Working Group (IMWG) Uniform Response Criteria Based on Investigator Assessment
Objective response 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 Investigator Assessment. 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. A ≥50% decrease in the difference between involved and uninvolved FLC levels in place of the M-protein criteria or a ≥50% reduction in plasma cells in place of M-protein if baseline was ≥30%. If present at baseline a ≥50% reduction in size of soft tissue plasmacytomas.
Time frame: From randomization through the study follow-up phase; up to the data cut-off of 31 July 2014; Maximum time on follow-up was 141.1 weeks.

Secondary outcomes

Percentage of Participants With Objective Response According to European Group for Blood and Marrow Transplantation (EBMT) Criteria Based on Investigator Assessment
Objective response defined as a best overall response of complete response (CR) or partial response (PR) based on the CR and 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.
Time frame: From randomization through the study follow-up phase; up to the data cut-off of 31 July 2014; Maximum time on follow-up was 141.1 weeks.
Number of Participants With Adverse Events and Type of Adverse Events
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
Time frame: From first dose of study drug through to 30 days after the last dose, until the data cut-off date of 31 July 2014. Maximum time on treatment was 94.1 weeks.
Kaplan Meier Estimates for Progression Free Survival (PFS) by Investigator Based on IMWG
Progression-free survival was calculated as the time from randomization to disease progression as determined by the Investigator 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: * Serum M-component (absolute increase ≥ 0.5 g/dl) * Urine M-component (absolute increase ≥ 200 mg/24 hours) * In patients without measurable serum and urine M-protein levels the difference between involved and uninvolved free light chain (FLC) levels (absolute increase \> 100 mg/dl) * Bone marrow plasma cell percentage (absolute % ≥ 10%) * Development of new or increase in the size of existing bone lesions or soft tissue plasmacytomas.
Time frame: From randomization through the follow-up phase; Maximum duration of follow-up for PFS was 90.3 weeks.
Kaplan-Meier Estimate for Time to Progression (TTP) Based on Investigator Assessment Using IMWG Criteria
Time to progression (TTP) was calculated as the time from randomization to the first documented progression confirmed by the investigator 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: * Serum M-component (absolute increase ≥ 0.5 g/dl) * Urine M-component (absolute increase ≥ 200 mg/24 hours) * In patients without measurable serum and urine M-protein levels the difference between involved and uninvolved free light chain (FLC) levels (absolute increase \> 100 mg/dl) * 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.
Time frame: From randomization through the follow-up phase; up to the data-cut off of 31 July 2014; Maximum time to progression follow-up was 90.3 weeks.
Kaplan-Meier Estimate Duration of Response Based on Investigator Assessment Using IMWG Criteria
Duration of Response (calculated for responders only) is defined as the time from the initial documented response (partial response or better) to confirmed disease progression by the investigator based on IMWG criteria.
Time frame: From randomization through the study follow-up phase; up to the data cut-off of 31 July 2014; Maximum duration of response follow-up was 90.3 weeks.
Kaplan-Meier Estimate for Overall Survival
Overall survival was 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.
Time frame: From randomization through the follow-up phase; Maximum time on follow-up was 141.1 weeks.
Time to Response Based on IMWG and Assessed by the Investigator
Time to Response was calculated as the time from enrollment to the initial response (PR or better) based on IMWG and assessed by the investigator.
Time frame: From randomization through the follow-up phase; up to the data-cut off of 31 July 2014; Maximum time to response was 23.1 weeks

Results

Posted Jul 30, 2015

Participant flow

Participant flow — Overall Study
Milestone	Pomalidomide
Started	74
Safety population	73
Efficacy evaluable population (eep)	64
Completed	0
Not completed	74
Withdrew: Adverse event	7
Withdrew: Death	10
Withdrew: Other	3
Withdrew: Progressive disease	50
Withdrew: Withdrawal by subject	3
Withdrew: 1 discontinued before starting drug	1

Outcome measures

PrimaryPercentage of Participants With an Objective Response According to International Myeloma Working Group (IMWG) Uniform Response Criteria Based on Investigator Assessment

Objective response 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 Investigator Assessment. 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. A ≥50% decrease in the difference between involved and uninvolved FLC levels in place of the M-protein criteria or a ≥50% reduction in plasma cells in place of M-protein if baseline was ≥30%. If present at baseline a ≥50% reduction in size of soft tissue plasmacytomas.

Time frame:: From randomization through the study follow-up phase; up to the data cut-off of 31 July 2014; Maximum time on follow-up was 141.1 weeks.
Reported as:: Number · percentage of participants

Percentage of Participants With an Objective Response According to International Myeloma Working Group (IMWG) Uniform Response Criteria Based on Investigator Assessment
percentage of participants	Pomalidomide
Percentage of Participants With an Objective Response According to International Myeloma Working Group (IMWG) Uniform Response Criteria Based on Investigator Assessment	23.0

SecondaryPercentage of Participants With Objective Response According to European Group for Blood and Marrow Transplantation (EBMT) Criteria Based on Investigator Assessment

Objective response defined as a best overall response of complete response (CR) or partial response (PR) based on the CR and 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.

Time frame:: From randomization through the study follow-up phase; up to the data cut-off of 31 July 2014; Maximum time on follow-up was 141.1 weeks.
Reported as:: Number · percentage of participants

Percentage of Participants With Objective Response According to European Group for Blood and Marrow Transplantation (EBMT) Criteria Based on Investigator Assessment
percentage of participants	Pomalidomide
Percentage of Participants With Objective Response According to European Group for Blood and Marrow Transplantation (EBMT) Criteria Based on Investigator Assessment	20.3

SecondaryNumber of Participants With Adverse Events and Type of Adverse Events

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

Time frame:: From first dose of study drug through to 30 days after the last dose, until the data cut-off date of 31 July 2014. Maximum time on treatment was 94.1 weeks.
Reported as:: Number · participants

Number of Participants With Adverse Events and Type of Adverse Events
participants	Pomalidomide
Any adverse event	73
Grade 3-4 adverse event	64
AE related to pomalidomide	51
Grade 3-4 AE related to pomalidomide	33
Grade 5 AE	19
≥1 Serious AE (SAE)	52
≥1 SAE related to pomalidomide	15
≥1 SAE leading to stopping pomalidomide	6
≥AE leading to discontinuation of pomalidomide	8
≥1 study drug related AE leading to stopping POM	1
≥1 AE leading to reduction of pomalidomide	11
≥1 study drug related AE leading to reducing POM	9
≥1 AE leading to interruption of pomalidomide	41
≥ 1 study drug related interruption of POM	25

SecondaryKaplan Meier Estimates for Progression Free Survival (PFS) by Investigator Based on IMWG

Progression-free survival was calculated as the time from randomization to disease progression as determined by the Investigator 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: * Serum M-component (absolute increase ≥ 0.5 g/dl) * Urine M-component (absolute increase ≥ 200 mg/24 hours) * In patients without measurable serum and urine M-protein levels the difference between involved and uninvolved free light chain (FLC) levels (absolute increase \> 100 mg/dl) * Bone marrow plasma cell percentage (absolute % ≥ 10%) * Development of new or increase in the size of existing bone lesions or soft tissue plasmacytomas.

Time frame:: From randomization through the follow-up phase; Maximum duration of follow-up for PFS was 90.3 weeks.
Reported as:: Median · weeks

Kaplan Meier Estimates for Progression Free Survival (PFS) by Investigator Based on IMWG
weeks	Pomalidomide
Kaplan Meier Estimates for Progression Free Survival (PFS) by Investigator Based on IMWG	16.0 (11.6 to 19.7)

SecondaryKaplan-Meier Estimate for Time to Progression (TTP) Based on Investigator Assessment Using IMWG Criteria

Time to progression (TTP) was calculated as the time from randomization to the first documented progression confirmed by the investigator 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: * Serum M-component (absolute increase ≥ 0.5 g/dl) * Urine M-component (absolute increase ≥ 200 mg/24 hours) * In patients without measurable serum and urine M-protein levels the difference between involved and uninvolved free light chain (FLC) levels (absolute increase \> 100 mg/dl) * 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.

Time frame:: From randomization through the follow-up phase; up to the data-cut off of 31 July 2014; Maximum time to progression follow-up was 90.3 weeks.
Reported as:: Median · weeks

Kaplan-Meier Estimate for Time to Progression (TTP) Based on Investigator Assessment Using IMWG Criteria
weeks	Pomalidomide
Kaplan-Meier Estimate for Time to Progression (TTP) Based on Investigator Assessment Using IMWG Criteria	19.0 (14.1 to 27.4)

SecondaryKaplan-Meier Estimate Duration of Response Based on Investigator Assessment Using IMWG Criteria

Duration of Response (calculated for responders only) is defined as the time from the initial documented response (partial response or better) to confirmed disease progression by the investigator based on IMWG criteria.

Time frame:: From randomization through the study follow-up phase; up to the data cut-off of 31 July 2014; Maximum duration of response follow-up was 90.3 weeks.
Reported as:: Median · weeks

Kaplan-Meier Estimate Duration of Response Based on Investigator Assessment Using IMWG Criteria
weeks	Pomalidomide
Kaplan-Meier Estimate Duration of Response Based on Investigator Assessment Using IMWG Criteria	28.3 (8.1 to 53.4)

SecondaryKaplan-Meier Estimate for Overall Survival

Overall survival was 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.

Time frame:: From randomization through the follow-up phase; Maximum time on follow-up was 141.1 weeks.
Reported as:: Median · weeks

Kaplan-Meier Estimate for Overall Survival
weeks	Pomalidomide
Kaplan-Meier Estimate for Overall Survival	33.6 (26.4 to 66.3)

SecondaryTime to Response Based on IMWG and Assessed by the Investigator

Time to Response was calculated as the time from enrollment to the initial response (PR or better) based on IMWG and assessed by the investigator.

Time frame:: From randomization through the follow-up phase; up to the data-cut off of 31 July 2014; Maximum time to response was 23.1 weeks
Reported as:: Median · weeks

Time to Response Based on IMWG and Assessed by the Investigator
weeks	Pomalidomide
Time to Response Based on IMWG and Assessed by the Investigator	8.3 (4.1 to 23.1)

Adverse events

Collected over From first dose of study drug through to 30 days after the last dose, until the data cut-off date of 31 July 2014. Maximum time on treatment was 94.1 weeks.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Pomalidomide	—	52/73 (71.2%)	68/73 (93.2%)

Most frequent serious events

Showing 10 of 62

Most frequent serious events
Event	Pomalidomide
PneumoniaInfections and infestations	11/73
AnaemiaBlood and lymphatic system disorders	10/73
General physical health deteriorationGeneral disorders	8/73
Pulmonary embolismRespiratory, thoracic and mediastinal disorders	4/73
HypercalcaemiaMetabolism and nutrition disorders	4/73
DiarrhoeaGastrointestinal disorders	4/73
Renal failure acuteRenal and urinary disorders	4/73
NeutropeniaBlood and lymphatic system disorders	3/73
Renal failureRenal and urinary disorders	3/73
Febrile neutropeniaBlood and lymphatic system disorders	2/73

Most frequent other events

Showing 10 of 32

Most frequent other events
Event	Pomalidomide
AnaemiaBlood and lymphatic system disorders	36/73
NeutropeniaBlood and lymphatic system disorders	35/73
ThrombocytopeniaBlood and lymphatic system disorders	16/73
FatigueGeneral disorders	16/73
ConstipationGastrointestinal disorders	16/73
DyspnoeaRespiratory, thoracic and mediastinal disorders	15/73
PyrexiaGeneral disorders	13/73
Back painMusculoskeletal and connective tissue disorders	13/73
HypokalaemiaMetabolism and nutrition disorders	13/73
Bone painMusculoskeletal and connective tissue disorders	12/73

Baseline characteristics

The intent-to-treat (ITT) population was defined as all enrolled participants.

Age, Continuous
Age, Continuous(years)	Pomalidomide
Mean	64.9 ± 9.25

Sex: Female, Male
Sex: Female, Male(Participants)	Pomalidomide
Female	32
Male	42

Race
Race(participants)	Pomalidomide
American Indian or Alaska Native	0
Asian	0
Black or African American	1
Native Hawaiian or Other Pacific Islanders	0
White	52
Other	2
Not Collected	19

Ethnicity
Ethnicity(participants)	Pomalidomide
Hispanic or Latino	6
Not Hispanic or Latino	49
Not Collected	19

Participants with Prior Anti-Myeloma (MM) Therapies
Participants with Prior Anti-Myeloma (MM) Therapies(participants)	Pomalidomide
Number	74

Durie-Salmon Multiple Myeloma Stage before Study Entry
Durie-Salmon Multiple Myeloma Stage before Study Entry(participants)	Pomalidomide
Stage I	8
Stage II	15
Stage III	50
Missing	1

Time From First Pathologic Diagnosis
Time From First Pathologic Diagnosis(years)	Pomalidomide
Mean	7.6 ± 3.72

Study locations

91 sites

Royal Adelaide Hospital - SA Pathology Haematology
Adelaide, 5000, Australia
Princess Alexandra Hospital - Haematology
Brisbane, 4102, Australia
Royal Prince Alfred Hospital - Institute of Haematology
Camperdown, 2050, Australia
Peter McCallum Cancer Institute - Directorate of Cancer Medicine
East Melbourne, 3002, Australia
Frankston Hospital-Peninsula Health - Oncology Day Unit
Frankston, 3199, Australia
The Alfred Hospital - Malignant Haematology & Stem Cell Transplantation
Melbourne, 3004, Australia
Calvary Mater Newcastle - Haematology
Waratah, 2298, Australia
Border Medical Oncology
Wodonga, 3690, Australia
Wollongong Hospital - Haematology
Wollongong, 2500, Australia
UZ Gent - Hematology
Ghent, 9000, Belgium
University Hospital Leuven - Hematology
Leuven, 3000, Belgium
Cliniques Universitaires ULC de Mont-Godinne - Hematology
Yvoir, 5530, Belgium
Tom Baker Cancer Center
Calgary, Alberta T2N 4N2, Canada
Cross Cancer Institute
Edmonton, Alberta T6G 1Z2, Canada
British Columbia Cancer Agency, Vancouver Centre
Vancouver, British Columbia V5Z 1M9, Canada
Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia B3H 2Y9, Canada
London Health Sciences Centre
London, Ontario N6A 5W9, Canada
Princess Margaret Hospital, University Health Network
Toronto, Ontario M5G 2M9, Canada
Maisonneuve-Rosemont Hospital
Montreal, Quebec H1T 2M4, Canada
Royal Victoria Hospital
Montreal, Quebec H3A1 A1, Canada
Charles University Hospital - Internal Medicine
Prague, 12808, Czechia
Aalborg Sygemus - Haematology
Aalborg, 9000, Denmark
Aarhus University Hospital
Aarhus, 8000, Denmark
Odense University Hospital
Odense, 5000, Denmark
Vejle Hospital - Hematology
Vejle, 7100, Denmark
CHU Angers - Service des maladies du sang
Angers, 49033, France
Centre Hospitalier de la côte basque - Hematologie
Bayonne, 64019, France
Centre Hospitalier Départemental Vendée - Onco-hematologie
La Roche, 85925, France
CHRU de Lille - Service des maladies du sang
Lille, 59037, France
Institut Paoli Calmette - Hematology 1
Marseille, 13009, France
CHU Hôtel-Dieu - Hematologie
Nantes, 44093, France
Hôpital Saint Louis - Immuno-hematologie
Paris, 75010, France
CHU Saint Antoine - Service des maladies du sang
Paris, 75012, France
CHRU - Hôpital du Haut Lévêque - Centre François Magendie Service des maladies du sang
Pessac, 33604, France
Centre Hospitalier Lyon sud - Hematologie
Pierre-Bénite, 69495, France
CHRU Hôpital Purpan - Hematologie
Toulouse, 31059, France
Hôpital Bretonneau - Hématologie & Thérapie cellulaire
Tours, 37044, France
CHU Nancy - Hematologie
Vandœuvre-lès-Nancy, 54511, France
Universitatsklinikum Carl Gustav Carus-Medizinische Klinik und Poliklinik I
Dresden, 01307, Germany
Universitätsklinikum Essen, Klinik für Hämatologie Westdeutsches Tumorzentrum
Essen, 45122, Germany
Askepios Klinik Altona-Abteilung Hamatologie und Internistische Onkologie
Hamburg, 22763, Germany
Universitätsklinikum Heidelberg - Medizinische Klinik und Poliklinik V
Heidelberg, 69120, Germany
Universitätsklinikum Jena - Klinik fur Innere Medizin II-Hamatologie/Onkologie
Jena, 07740, Germany
Universitätsklinikum Leipzig - Medizinische Klinik und Poliklinik II
Leipzig, 04103, Germany
Universitätsklinikum Münster - Medizinische Klinik und Poliklinik A
Münster, 48149, Germany
Universitätsklinikum Tübingen - Medizinische Klinik und Poliklinik - Abteilung II
Tübingen, 72076, Germany
Universitätsklinikum Ulm - Klinik fur Innere Medizin III
Ulm, 89081, Germany
Universitätsklinikum Würzburg - Medizinische Klinik und Poliklinik II
Würzburg, 97080, Germany
University of Athens - Alexandra Hospital
Athens, 14572, Greece
Università degli Studi di Bologna - Policlinico S. Orsola - Hematology
Bologna, 40138, Italy
AO Universitaria San Martino - hematooncology
Genova, 16132, Italy
Fondazione "G. Pascale" - Hematology
Naples, 80131, Italy
Ospedale San Luigi AO Luigi Gonzaga - Hematology
Orbassano, 10043, Italy
Universita degli Studi di Padova - Clinical & Experimental Medicine
Padova, 35128, Italy
Ospedale Guglielmo da Saliceto - hematooncology
Piacenza, 29100, Italy
Unità di Ematologia Arcispedale S. Maria Nuova - Haematology
Reggio Emilia, 42100, Italy
Policlinico Umberto I, Università "La Sapienza" di Roma - Hematology
Roma, 00161, Italy
A.O.U. San Giovanni Battista - Hematology
Torino, 10126, Italy
VUMC - Hematology
Amsterdam, 1081 HV, Netherlands
Erasmus Medical Center - Hematology
Rotterdam, 3015 CE, Netherlands
University Medical Center - Hematology
Utrecht, 3584 CX, Netherlands
Hematological Research Center under the Russian Academy of Medical Sciences - Hematology & BMT
Moscow, 125167, Russia
Moscow State Medical Institution Municipal Clinical Hospital n.a. S.P. Botkin - Hematology
Moscow, 125284, Russia
Russian Research Institute of Hematology and Blood Transfusion - Hematology
Saint Petersburg, 191024, Russia
State Higher Educational Institution St. Petersburg State Medical University - Onco-hematology
Saint Petersburg, 197341, Russia
Hospital Germans Trias i Pujol - Hematology
Badalona, 08916, Spain
Hospital Clinic i Provincial de Barcelona - Hematology
Barcelona, 08036, Spain
Hospital de Donostia - Hematology
Guipúzcoa, 20014, Spain
Hospital de La Princesa - Hematology
Madrid, 28006, Spain
Hospital 12 de Octubre - Hematology
Madrid, 28041, Spain
Hospital de Salamanca - Hematology
Salamanca, 37007, Spain
Hospital Universitario Marqués de Valdecilla - Hematology
Santander, 39008, Spain
Hospital La Fe - Hematology
Valencia, 46009, Spain
Sahlgrenska Hospital, University of Goteborg - Hematology
Gothenburg, S-41345, Sweden
Karolinska University Hospital Huddinge - Center of hematology
Stockholm, 14152, Sweden
Karolinska University Hospital-medicine
Stockholm, 14186, Sweden
Karolinska University Hospital Solna- medicine
Stockholm, 17176, Sweden
Overlakare Medocomcentrum - Hematology
Uppsala, 75185, Sweden
Inselspital, Institut für Medizinische Onkologie
Bern, 3010, Switzerland
Hôpitaux Universitaire de Genève - Oncologie
Geneva, 1211, Switzerland
Klinik und Poliklinik für Onkologie - UniversitätsSpital Zürich
Zurich, 8091, Switzerland
Royal Bournemouth Hospital - Haematology
Bournemouth, BH7 7DW, United Kingdom
St James's University Hospital - Haematology
Leeds, LS9 7TF, United Kingdom
St Bartholomew's Hospital - Medical Oncology
London, EC1A 7BE, United Kingdom
King's College Hospital - Haematology Clinical Trials
London, SE5 9RS, United Kingdom
Freeman Hospital - Northern Centre for Cancer Care
Newcastle upon Tyne, NE7 7DN, United Kingdom
Nottingham City Hospital - Centre for Clinical Haematology
Nottingham, NG5 1PB, United Kingdom
Derriford Hospital - Haematology
Plymouth, PL6 8DH, United Kingdom
Royal hallamshire Hospital - Haematology
Sheffield, S10 2JF, United Kingdom
Royal Marsden NHS Foundation Trust - Haematology
Surrey, SM2 5PT, United Kingdom
Royal Wolverhampton Hospitals Trust - Research and Development
Wolverhampton, WV10 OQP, United Kingdom

References and documents

Publications

Moreau P, Weisel KC, Song KW, Gibson CJ, Saunders O, Sternas LA, Hong K, Zaki MH, Dimopoulos MA. Relationship of response and survival in patients with relapsed and refractory multiple myeloma treated with pomalidomide plus low-dose dexamethasone in the MM-003 trial randomized phase III trial (NIMBUS). Leuk Lymphoma. 2016 Dec;57(12):2839-2847. doi: 10.1080/10428194.2016.1180685. Epub 2016 May 13. PubMed 27173785 ↗
Miguel JS, Weisel K, Moreau P, Lacy M, Song K, Delforge M, Karlin L, Goldschmidt H, Banos A, Oriol A, Alegre A, Chen C, Cavo M, Garderet L, Ivanova V, Martinez-Lopez J, Belch A, Palumbo A, Schey S, Sonneveld P, Yu X, Sternas L, Jacques C, Zaki M, Dimopoulos M. Pomalidomide plus low-dose dexamethasone versus high-dose dexamethasone alone for patients with relapsed and refractory multiple myeloma (MM-003): a randomised, open-label, phase 3 trial. Lancet Oncol. 2013 Oct;14(11):1055-1066. doi: 10.1016/S1470-2045(13)70380-2. Epub 2013 Sep 3. PubMed 24007748 ↗
Morgan G, Palumbo A, Dhanasiri S, Lee D, Weisel K, Facon T, Delforge M, Oriol A, Zaki M, Yu X, Sternas L, Jacques C, Akehurst R, Offner F, Dimopoulos MA. Overall survival of relapsed and refractory multiple myeloma patients after adjusting for crossover in the MM-003 trial for pomalidomide plus low-dose dexamethasone. Br J Haematol. 2015 Mar;168(6):820-3. doi: 10.1111/bjh.13227. Epub 2014 Nov 18. PubMed 25403264 ↗

Timeline

Tracking since Sep 25, 2026

ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Mar 2011 to primary completion in Jul 2014, 40 months.

Results were first posted in Jul 2015, 11 months after primary completion.

Of 104 industry-sponsored phase 3 interventional studies under Multiple Myeloma, half reached primary completion within 45 months of starting (the median), counting those still running; a quarter within 32 and three quarters within 66. This study's 40 months is below that.

Counting every sponsor, half of 172 studies reached it within 55 months; a quarter within 34 and three quarters within 95.

How these figures are worked out, and which studies are left out of them.

Registered: Mar 27, 2011
First posted: Mar 29, 2011
StartActual: Mar 1, 2011
Primary completionActual: Jul 31, 2014
CompletionActual: Jul 31, 2014
Results first posted: Jul 30, 2015
Last update: Nov 19, 2019

What these dates mean

Start.: When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.: When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.: When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.: Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.

No changes since tracking began. The registry record was last updated on Nov 19, 2019, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT01324947
Lead sponsor: Celgene
Responsible party: Sponsor
First posted: Mar 29, 2011
Start date: Mar 1, 2011
Primary completion: Jul 31, 2014
Completion: Jul 31, 2014
Results posted: Jul 30, 2015
Last update: Nov 19, 2019

Study contacts

Mohamed Zaki, MD, PhD

study director · Celgene Corporation

Oversight

Data monitoring committee: No

View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Nov 2019. You cannot join it, but the record below documents what was studied.

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Discussion

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Studies of the drug tested here: Pomalidomide

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