Multiple Myeloma
Conditions
Keywords
Multiple Myeloma, Relapsed Multiple Myeloma, Relapsed and refractory Multiple Myeloma, Pomalidomide, Dexamethasone
Brief summary
The primary purpose of the study is to evaluate the safety and efficacy and to generate PK and biomarker data for the combination of pomalidomide and low-dose dexamethasone in patients with refractory or relapsed and refractory multiple myeloma. The study consists of a Screening phase within 28 days prior to cycle 1 day 1, a Treatment phase and a Follow-up phase which starts within 28 days of discontinuation from study treatment, every 3 months for up to 5 years. In addition, the collection of steady-state PK data from a large population will enable robust population PK and assess Pomalidomide exposure response analyses. The exploratory objectives of the study are to investigate potential markers predictive of POM response or resistance and pharmacodynamic markers.
Interventions
DRUGPomalidomide
Oral Pomalidomide at the starting dose of 4 mg on Days 1-21 of a 28-day cycle
DRUGDexamethasone
Oral Low dose Dexamethasone at the starting dose of 40mg/day (≤ 75 years old) or 20 mg/day (\> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle.
Sponsors
Celgene
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients ≥18 years old, who must understand and voluntarily sign an Informed Consent. * Patients must have documented diagnosis of Multiple Myeloma and have measurable disease. * Patients must have undergone prior treatment with ≥ 2 treatments lines, of anti-myeloma therapy. * Patients must have either refractory or relapsed and refractory disease. * Patients must have received at least 2 consecutive cycles of prior treatment that include lenalidomide and bortezomib, either alone or in combination regimens. * Patients must have received adequate alkylator therapy
Exclusion criteria
* Prior history of malignancies, other than Multiple Myeloma. * Previous therapy with Pomalidomide, hypersensitivity to thalidomide and lenalidomide or dexamethasone. * Patients who received an allogeneic bone marrow or allogeneic peripheral blood stem cell transplant. * Patients who are planning for or who are eligible for stem cell transplant. * Patients who received major surgery and any anti-myeloma drug therapy within the last 14 days of starting study treatment. * Patients with a current disease that can interfere with protocol procedures or study treatment. * Patients unable or unwilling to undergo antithrombotic prophylactic treatment. * Pregnant or breastfeeding females.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAE) | From the first dose of study treatment up to 28 days following the last dose of study treatment. The median duration of treatment with pomalidomide and LD-dex was 21.4 weeks. | An adverse event (AE) is any noxious, unintended, or untoward medical occurrence that may appear or worsen in a subject during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the subject's health, regardless of etiology. Any worsening (i.e., any significant adverse change in the frequency or intensity of a pre- existing condition) was considered an AE. The severity of AEs were graded based on the symptoms according to version 4.0 of the National Cancer Institute Common Terminology Criteria for Adverse Events. Second primary malignancies were monitored as events of interest and considered as part of the assessment of AEs. A SAE = AE occurring at any dose that: * Results in death; * Is life-threatening * Requires inpatient hospitalization or prolongation of existing hospitalization * Results in persistent or significant disability/incapacity * Is a congenital anomaly/birth defect |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to Response | Response was assessed at each treatment cycle and at treatment discontinuation; median duration of treatment with pomalidomide and LD-dex was 21.4 weeks | Time to response was defined as the time from treatment enrollment to the first documentation of response (sCR, CR, VGPR or PR) based on IMWG criteria. |
| Kaplan Meier Estimate of Duration of Response | From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months | Duration of response, calculated for responders only, was defined as time from the initial documented response (SCR, CR, VGPR or PR) to the first confirmed disease progression, or death if no disease progression was recorded. Participants without a documented progression were censored at the time of their last tumor assessment. |
| Kaplan Meier Estimate of Progression Free Survival (PFS) According to the European Medicines Agency Guidelines | From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months | Progression free survival was calculated as the time from study enrollment, defined as the IVRS enrollment date, until either PD or death (any cause). Participants without an event (either a documented PD or death) at the time of study end were censored at the time of their last documented disease assessment or at the IVRS enrollment date if no disease assessment was conducted. |
| Kaplan Meier Estimate of Time to Progression | From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months | Time to progression was calculated as the time from study enrollment until first recorded disease progression as determined by the site investigator based on the IMWG criteria, or until death due to progression. Participants not experiencing a documented progression were censored at the time of their last tumor assessment (or at the time of trial enrollment if no assessment was conducted). |
| Overall Response | Response was assessed at each treatment cycle and at treatment discontinuation; median duration of treatment with pomalidomide and LD-dex was 21.4 weeks | Overall response rate (ORR) was defined as the percentage of participants with a stringent complete response (sCR), complete response (CR), very good partial response (VGPR) or partial response (PR) according to the International Myeloma Working Group uniform response criteria (IMWG URC) assessed by the Investigator. Responses must have been confirmed at at least 2 consecutive assessments before the institution of any new therapy with no known evidence of progressive or new bone lesions |
| Pomalidomide Exposure - Apparent (Oral) Clearance (CL/F) | Cycles 1, 2, 3, 4, 5, 6 | Pharmacokinetic (PK) parameters are derived from pomalidomide concentration versus time data. |
| Pomalidomide Exposure - Apparent Volume of Distribution (V/F) | Cycles 1, 2, 3, 4, 5, 6 | Pharmacokinetic (PK) parameters are derived from Pomalidomide concentration versus time data. |
| Cytogenetic Analysis | Study entry | Cytogenetic analysis was to be performed using fluorescence in situ hybridization (FISH) methodology at a local laboratory, to evaluate the relationship between cytogenetic profiles and the combination of POM and LD-DEX in terms of response and outcome. |
| Kaplan Meier Estimate of Overall Survival (OS) | From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months | Overall survival was calculated as the time from study enrollment, defined as the IVRS enrollment date, until death due to any cause. Participants who did not have death data at the time of study end/analysis were censored at the time they were last known to be alive. |
Countries
Austria, Belgium, Denmark, Estonia, Finland, France, Germany, Greece, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Slovakia, Spain, Sweden, Switzerland, Turkey (Türkiye), United Kingdom
Participant flow
Recruitment details
The study was conducted at 112 sites: 4 in Austria, 7 in Belgium, 3 in Denmark, 1 in Estonia, 2 in Finland, 13 in France, 17 in Germany, 1 in Greece, 3 in Ireland, 15 in Italy, 5 in the Netherlands, 2 in Norway, 3 in Poland, 4 in Portugal, 1 in Slovakia, 15 in Spain, 2 in Sweden, 3 in Switzerland, 2 in Turkey, and 9 in the United Kingdom.
Pre-assignment details
Study participants had to have either refractory or relapsed and refractory disease, defined as documented disease progression during or within 60 days of completing their last myeloma therapy to be eligible to participate in the trial.
Participants by arm
| Arm | Count |
|---|---|
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (\> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator. | 682 |
| Total | 682 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 52 |
| Overall Study | Death | 57 |
| Overall Study | Lack of Efficacy | 2 |
| Overall Study | Lost to Follow-up | 1 |
| Overall Study | Miscellaneous | 31 |
| Overall Study | Participants Did Not Receive Study Drug | 6 |
| Overall Study | Progressive Disease | 504 |
| Overall Study | Transition to Commercial Treatment | 8 |
| Overall Study | Withdrawal by Subject | 21 |
Baseline characteristics
| Characteristic | Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) |
|---|---|
| Age, Continuous | 65.4 Years STANDARD_DEVIATION 9.1 |
| Beta 2 Microglobulin | 5.48 mg/L STANDARD_DEVIATION 4.713 |
| Corrected Serum Calcium | 2.43 mmol/L STANDARD_DEVIATION 0.231 |
| Eastern Cooperative Oncology Group (ECOG) Performance Status 0 = Fully active, no restrictions | 295 Participants |
| Eastern Cooperative Oncology Group (ECOG) Performance Status 1 = Restricted activity but ambulatory | 319 Participants |
| Eastern Cooperative Oncology Group (ECOG) Performance Status 2 = Ambulatory and capable of all self-care | 67 Participants |
| Eastern Cooperative Oncology Group (ECOG) Performance Status 3 = Limited self-care | 1 Participants |
| Eastern Cooperative Oncology Group (ECOG) Performance Status 4 = Completely disabled | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 52 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 626 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 4 Participants |
| Race/Ethnicity, Customized Asian | 3 Participants |
| Race/Ethnicity, Customized Black or African American | 4 Participants |
| Race/Ethnicity, Customized Missing | 4 Participants |
| Race/Ethnicity, Customized Other | 2 Participants |
| Race/Ethnicity, Customized White | 669 Participants |
| Renal Function (Cockcroft Gault Creatinine Clearance) 30 - < 45 mL/min | 57 Participants |
| Renal Function (Cockcroft Gault Creatinine Clearance) < 30 mL/min | 12 Participants |
| Renal Function (Cockcroft Gault Creatinine Clearance) 45 - < 60 mL/min | 168 Participants |
| Renal Function (Cockcroft Gault Creatinine Clearance) 60 - < 80 mL/min | 190 Participants |
| Renal Function (Cockcroft Gault Creatinine Clearance) ≥ 80 mL/min | 250 Participants |
| Renal Function (Cockcroft Gault Creatinine Clearance) Missing | 5 Participants |
| Serum Heavy Chain Type Immunoglobulin A (IgA) | 145 Participants |
| Serum Heavy Chain Type Immunoglobulin D (IgD) | 5 Participants |
| Serum Heavy Chain Type Immunoglobulin E (IgE) | 0 Participants |
| Serum Heavy Chain Type Immunoglobulin G (IgG) | 388 Participants |
| Serum Heavy Chain Type Immunoglobulin M (IgM) | 4 Participants |
| Serum Heavy Chain Type No serum heavy chain type detected | 68 Participants |
| Serum Heavy Chain Type Test not performed | 72 Participants |
| Serum Light Chain Type Kappa | 364 Participants |
| Serum Light Chain Type Lambda | 230 Participants |
| Serum Light Chain Type No Serum Light chain Type Detected | 16 Participants |
| Serum Light Chain Type Test Not Performed | 72 Participants |
| Sex: Female, Male Female | 301 Participants |
| Sex: Female, Male Male | 381 Participants |
| Time since diagnosis | 6.15 Years STANDARD_DEVIATION 3.649 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 598 / 682 |
| other Total, other adverse events | 646 / 676 |
| serious Total, serious adverse events | 448 / 676 |
Outcome results
Primary
Number of Participants With Treatment Emergent Adverse Events (TEAE)
An adverse event (AE) is any noxious, unintended, or untoward medical occurrence that may appear or worsen in a subject during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the subject's health, regardless of etiology. Any worsening (i.e., any significant adverse change in the frequency or intensity of a pre- existing condition) was considered an AE. The severity of AEs were graded based on the symptoms according to version 4.0 of the National Cancer Institute Common Terminology Criteria for Adverse Events. Second primary malignancies were monitored as events of interest and considered as part of the assessment of AEs. A SAE = AE occurring at any dose that: * Results in death; * Is life-threatening * Requires inpatient hospitalization or prolongation of existing hospitalization * Results in persistent or significant disability/incapacity * Is a congenital anomaly/birth defect
Time frame: From the first dose of study treatment up to 28 days following the last dose of study treatment. The median duration of treatment with pomalidomide and LD-dex was 21.4 weeks.
Population: The safety population includes all enrolled participants who received at least one dose of the IP.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 TEAE L/T to Stopping of Either POM or LD-DEX | 63 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥1 Study Drug Related TEAE (L/T) Stopping POM | 30 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥1 Study Drug Related TEAE L/T Stopping LD-Dex | 19 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥1 Drug Related TEAE L/T Stopping LD-Dex or POM | 38 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 TEAE L/T to Reduction (R/D) of POM | 164 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 TEAE L/T to R/D of LD-DEX | 150 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 TEAE L/T to R/D of Either POM or LD-DEX | 244 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Study Drug Related TEAE L/T to R/D of POM | 142 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Study Drug Related TEAE L/T to R/D of LD-DEX | 135 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥1 StudyDrug Related TEAE L/T to R/D POM or LD-DEX | 224 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 TEAE L/T to Interruption (I/R) of POM | 455 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 TEAE L/T to I/R of LD-DEX | 434 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 TEAE L/T to I/R of either POM or LD-DEX | 470 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Study Drug Related TEAE L/T to I/R of POM | 294 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Study Drug Related TEAE L/T to I/R of LD-DEX | 185 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥1 StudyDrug Related TEAE L/T to I/R POM or LD-DEX | 333 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ TEAE | 673 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 TEAE Related to Pomalidomide (POM) | 527 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 TEAE Related to LD-Dex | 448 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 TEAE Related to Either POM or LD-Dex | 575 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Grade (Gr) 3 or 4 TEAE | 606 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Gr 3 or 4 TEAE Related to (R/T) POM | 417 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Gr 3 or 4 TEAE R/T LD-Dex | 226 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Gr 3 or 4 TEAE R/T Either POM or LD-Dex | 448 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Grade 5 TEAE | 127 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Grade 5 TEAE R/T POM | 14 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Grade 5 TEAE R/T LD-Dex | 16 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Grade 5 TEAE R/T either POM or LD-Dex | 18 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Serious TEAE | 448 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Serious TEAE R/T POM | 187 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Serious TEAE R/T LD-Dex | 146 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Serious TEAE R/T Either POM or LD-Dex | 215 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Serious TEAE Leading to (L/T)Stopping of POM | 36 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 Serious TEAE L/T Stopping of LD-Dex | 34 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥1 Serious TEAE L/T Stopping either POM or LD-Dex | 37 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 TEAE L/T to Stopping of POM | 54 Participants |
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | ≥ 1 TEAE L/T to Stopping of LD-DEX | 61 Participants |
Secondary
Cytogenetic Analysis
Cytogenetic analysis was to be performed using fluorescence in situ hybridization (FISH) methodology at a local laboratory, to evaluate the relationship between cytogenetic profiles and the combination of POM and LD-DEX in terms of response and outcome.
Time frame: Study entry
Population: Statistical analysis could not be performed due to high level of missing cytogenetic data as well as cytogenetic probe analysis being non- compliant with study requirements which lead to an inability to accurately identify specific cytogenetic abnormalities.
Secondary
Kaplan Meier Estimate of Duration of Response
Duration of response, calculated for responders only, was defined as time from the initial documented response (SCR, CR, VGPR or PR) to the first confirmed disease progression, or death if no disease progression was recorded. Participants without a documented progression were censored at the time of their last tumor assessment.
Time frame: From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months
Population: Includes participants with a response (sCR, VGPR or PR).
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Kaplan Meier Estimate of Duration of Response | 7.9 Months |
Secondary
Kaplan Meier Estimate of Overall Survival (OS)
Overall survival was calculated as the time from study enrollment, defined as the IVRS enrollment date, until death due to any cause. Participants who did not have death data at the time of study end/analysis were censored at the time they were last known to be alive.
Time frame: From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months
Population: The intent to treat (ITT) population was defined as all enrolled participants (participants who received an interactive voice response system (IVRS) enrollment date) regardless of whether they received any investigational product (IP) or not.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Kaplan Meier Estimate of Overall Survival (OS) | 11.9 Months |
Secondary
Kaplan Meier Estimate of Progression Free Survival (PFS) According to the European Medicines Agency Guidelines
Progression free survival was calculated as the time from study enrollment, defined as the IVRS enrollment date, until either PD or death (any cause). Participants without an event (either a documented PD or death) at the time of study end were censored at the time of their last documented disease assessment or at the IVRS enrollment date if no disease assessment was conducted.
Time frame: From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months
Population: The intent to treat (ITT) population was defined as all enrolled participants (participants who received an interactive voice response system (IVRS) enrollment date) regardless of whether they received any investigational product (IP) or not.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Kaplan Meier Estimate of Progression Free Survival (PFS) According to the European Medicines Agency Guidelines | 4.6 Months |
Secondary
Kaplan Meier Estimate of Time to Progression
Time to progression was calculated as the time from study enrollment until first recorded disease progression as determined by the site investigator based on the IMWG criteria, or until death due to progression. Participants not experiencing a documented progression were censored at the time of their last tumor assessment (or at the time of trial enrollment if no assessment was conducted).
Time frame: From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months
Population: The intent to treat (ITT) population was defined as all enrolled participants (participants who received an interactive voice response system (IVRS) enrollment date) regardless of whether they received any investigational product (IP) or not.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Kaplan Meier Estimate of Time to Progression | 4.8 Months |
Secondary
Overall Response
Overall response rate (ORR) was defined as the percentage of participants with a stringent complete response (sCR), complete response (CR), very good partial response (VGPR) or partial response (PR) according to the International Myeloma Working Group uniform response criteria (IMWG URC) assessed by the Investigator. Responses must have been confirmed at at least 2 consecutive assessments before the institution of any new therapy with no known evidence of progressive or new bone lesions
Time frame: Response was assessed at each treatment cycle and at treatment discontinuation; median duration of treatment with pomalidomide and LD-dex was 21.4 weeks
Population: The intent to treat (ITT) population was defined as all enrolled participants (participants who received an interactive voice response system (IVRS) enrollment date) regardless of whether they received any investigational product (IP) or not.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Overall Response | 33.4 Percentage of Participants |
Secondary
Pomalidomide Exposure - Apparent (Oral) Clearance (CL/F)
Pharmacokinetic (PK) parameters are derived from pomalidomide concentration versus time data.
Time frame: Cycles 1, 2, 3, 4, 5, 6
Population: Pharmacokinetic population~Pharmacokinetic samples from 476 participants included in studies CC-4047-MM-005 and CC 4047 MM 010 were used for the population PK analysis of pomalidomide
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Pomalidomide Exposure - Apparent (Oral) Clearance (CL/F) | 6.02 Liters/hour |
Secondary
Pomalidomide Exposure - Apparent Volume of Distribution (V/F)
Pharmacokinetic (PK) parameters are derived from Pomalidomide concentration versus time data.
Time frame: Cycles 1, 2, 3, 4, 5, 6
Population: Pharmacokinetic population~Pharmacokinetic samples from 476 participants included in studies CC-4047-MM-005 and CC 4047 MM 010 were used for the population PK analysis of pomalidomide
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Pomalidomide Exposure - Apparent Volume of Distribution (V/F) | 75.10 Liters |
Secondary
Time to Response
Time to response was defined as the time from treatment enrollment to the first documentation of response (sCR, CR, VGPR or PR) based on IMWG criteria.
Time frame: Response was assessed at each treatment cycle and at treatment discontinuation; median duration of treatment with pomalidomide and LD-dex was 21.4 weeks
Population: Includes participants with a response (sCR, VGPR or PR).
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pomalidomide Plus Low Dose Dexamethasone (LD-Dex) | Time to Response | 8.1 Weeks |