Trial Outcomes & Findings for MTD, Safety, and Efficacy of Pomalidomide (CC-4047) Alone or With Low-dose Dexamethasone in Patients With Relapsed and Refractory Multiple Myeloma (NCT NCT00833833)

Phase 2: Participants were stratified by age (≤ 75 vs. \> 75), prior number of treatments (2 vs. \> 2), and prior thalidomide exposure (yes vs. no).

MTD, Safety, and Efficacy of Pomalidomide (CC-4047) Alone or With Low-dose Dexamethasone in Patients With Relapsed and Refractory Multiple Myeloma

The maximum tolerated dose was defined as the highest dose level at which no more than 1 of 6 participants experiences a DLT within the first 28-day cycle. DLTs were defined as: * Grade 4 neutropenia or thrombocytopenia * Febrile neutropenia * Grade 3 or 4 nausea, vomiting or diarrhea despite optimal symptomatic treatment * Serum transaminase \> 20 \* upper limit of normal (ULN) * Serum transaminase \> 5 \* ULN for \>= 7 days * Delay of the start of cycle 2 by \>7 days due to pomalidomide-related adverse event

Progression free survival (PFS) is the time from randomization to the first documentation of disease progression or death from any cause during study, whichever occurs earlier. Disease progression was assessed by the Independent Response Adjudication Committee (IRAC). For the primary PFS analysis, participants who withdrew for any reason or received another antimyeloma therapy (except adding dexamethasone to the Phase 2: Pomalidomide arm) without documented PD (as determined by the IRAC review) were censored on the date of their last adequate response assessment, prior to receiving any other anti-myeloma therapy. Subjects who were still active at the time of the data cut-off date without PD (as determined by the IRAC) were censored on the date of their last adequate response assessment. Data collection is ongoing and future data results will be included as available.

Percentage of participants with the progression-free survival events: disease progression and death. Disease progression was assessed by the Independent Response Adjudication Committee (IRAC). Data collection is ongoing and future data results will be included as available.

Relation to study drug was assessed by the Investigator as either suspected or not suspected. Counts represent the suspected relationship. Severity was assessed using National Cancer Institute Common Toxicity Terminology Criteria for Adverse Events version 3.0 (NCI CTCAE v3.0): 1= Mild 2= Moderate 3= Severe 4= Life-threatening and 5= Death related to AE. Serious AEs (SAEs) are those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention to prevent one of the outcomes listed above. Data collection is ongoing and future data results will be included as available.

TEAEs that occurred during Phase 1 after dexamethasone was added to pomalidomide treatment. Relation to study drug was assessed by the Investigator as either suspected or not suspected. Counts represent the suspected relationship. Severity was assessed using National Cancer Institute Common Toxicity Terminology Criteria for Adverse Events version 3.0 (NCI CTCAE v3.0): 1= Mild 2= Moderate 3= Severe 4= Life-threatening and 5= Death related to AE. Serious AEs (SAEs) are those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention to prevent one of the outcomes listed above. Data collection is ongoing and future data results will be included as available.

Relation to study drug was assessed by the Investigator as either suspected or not suspected. Counts represent the suspected relationship. Severity was assessed using National Cancer Institute Common Toxicity Terminology Criteria for Adverse Events version 3.0 (NCI CTCAE v3.0): 1= Mild 2= Moderate 3= Severe 4= Life-threatening and 5= Death related to AE. Serious AEs (SAEs) are those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention to prevent one of the outcomes listed above. Data collection is ongoing and future data results will be included as available.

IRAC used EBMT criteria to assess myeloma response: * Complete Response (CR)-absence of serum and urine monoclonal paraprotein for 6 weeks, plus no increase in size or number of lytic bone lesions, plus other factors) * Partial Response (PR)-not all CR criteria, plus \>=50% reduction in serum monoclonal paraprotein plus others * Minimal Response (MR)- 25-49% reduction in serum monoclonal paraprotein plus others * Stable Disease (SD)- not MR or progressive disease (PD) * Progressive Disease (PD)- reappearance of monoclonal paraprotein, lytic bone lesions, other * Not Evaluable (NE). Data collection is ongoing and future data results will be included as available.

Duration of myeloma response is defined as the time from when the response criteria are first met for partial response (PR) or better, until the first date the response criteria are met for progressive disease (PD) or until the participant dies from any cause, whichever occurs first. Duration of response for participants last known to be alive with no progression after a complete response (CR) or PR was censored at the date of last adequate response assessment. Participants with confirmed responses that occur after receiving any other anti-myeloma therapy (except for adding dexamethasone to the pomalidomide treatment arm), including radiation therapy initiated after baseline, was censored at the last adequate assessment prior to the initiation of such treatment. Response was assessed by the Independent Response Adjudication Committee (IRAC) using European Group for Blood and Bone Marrow Transplant (EBMT) criteria as described in the previous outcome.

Time to myeloma response is defined as the time from randomization to the time the response criteria for complete response (CR) or partial response (PR) are first met. Response was assessed by the Independent Response Adjudication Committee (IRAC) using European Group for Blood and Bone Marrow Transplant (EBMT) criteria as described previously. Data collection is ongoing and future data results will be included as available.

Overall survival was defined as the time between randomization and death. Participants who die, regardless of the cause of the death, were considered to have had an event. All participants who were lost to follow-up prior to the end of the trial or who were withdrawn from the trial were censored at the time of last contact. Participants who were still being treated were censored at the last available date the subject was known to be alive, or clinical cut-off date if it was earlier. Data collection is ongoing and future data results will be included as available.