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Lenalidomide

Bortezomib

Pomalidomide

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Growth Inhibitors

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

pomalidomide

Mohamed Zaki, MD, PhD

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.

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

Open-label, Multi-center, Single Arm Study For The Safety And Efficacy Of Pomalidomide Monotherapy For Subjects With Refractory Or Relapsed And Refractory Multiple Myeloma. A Companion Study For Clinical Trial CC-4047-MM003

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.

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.

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

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 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.

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.

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

Celgene

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

Age, Continuous

Sex: Female, Male

American Indian or Alaska Native

Asian

Black or African American

Native Hawaiian or Other Pacific Islanders

White

Other

Not Collected

Race

Hispanic or Latino

Not Hispanic or Latino

Ethnicity

Participants with Prior Anti-Myeloma (MM) Therapies

Stage I

Stage II

Stage III

Missing

Stages are:

Stage I: Hemoglobulin \> 10 g/dL; serum calcium normal on roentgenogram; normal bone structure or solitary bone plasmacytoma only; Low M-component production rates (IgG value \< 5 g/dL, IgA value \< 3 g/dL; Bence Jones protein \< 4 g/24h)

Stage II: Overall data as minimally abnormal for stage I, and no single value as abnormal as defined for stage III

Stage III: 1 or more of below:

Hemoglobin \< 8.5 g/dL; serum calcium \> 12 mg/dL; Advanced lytic bone lesions (scale 3); High-M-component production rates (IgG value \> 7 g/dL, IgA value \> 5 g/dL; Bence Jones protein \> 12 g/24h)

Durie-Salmon Multiple Myeloma Stage before Study Entry

Time From First Pathologic Diagnosis

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

Anne McClain, Senior Manager, Clinical Trial Disclosure

Intent to Treat Population was defined as all enrolled participants.

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

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

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

Intent to treat population includes all participants enrolled.

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

Any adverse event

Grade 3-4 adverse event

AE related to pomalidomide

Grade 3-4 AE related to pomalidomide

Grade 5 AE

≥1 Serious AE (SAE)

≥1 SAE related to pomalidomide

≥1 SAE leading to stopping pomalidomide

≥AE leading to discontinuation of pomalidomide

≥1 study drug related AE leading to stopping POM

≥1 AE leading to reduction of pomalidomide

≥1 study drug related AE leading to reducing POM

≥1 AE leading to interruption of pomalidomide

≥ 1 study drug related interruption of POM

Safety population includes all participants who received at least one dose of study drug.

Number of Participants With Adverse Events and Type of Adverse Events

Intent to Treat included all participants enrolled.

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

Intent to Treat includes all participants enrolled

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

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

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

Includes those who had a partial response or better.

Spots Global Cancer Trial Database for companion

Every month we try and update this database with for companion cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for companion

Every month we try and update this database with for companion cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for companion cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.