A Phase 1/2 interventional study of Pomalidomide and Dexamethasone in Multiple Myeloma, sponsored by Celgene Corporation. Completed at 19 sites in 2 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2016-04-27.
Sponsored by Celgene Corporation (part of Bristol-Myers Squibb) · Phase 1/2, Interventional, and Treatment
The purpose of this study is to determine the maximum tolerated dose and effectiveness of the study drug (CC-4047) Alone Or in Combination With Low-dose Dexamethasone as treatment for patients with relapsed and refractory multiple myeloma
The Phase 1 segment of the study was designed to determine the maximum tolerated dose (MTD) of single-agent pomalidomide, which was to be determined in the first cycle of treatment. Following completion of the first cycle, participants were allowed to continue the study at their assigned dose of pomalidomide.
Participants who developed progressive disease (PD) at any time, or who had not achieved at least a 25% reduction of serum myeloma (M)-protein levels (if measurable) and a 50% reduction of urine M-protein (if measurable) compared with baseline after completion of 4 cycles of pomalidomide, had the option to receive oral dexamethasone 40 mg on days 1, 8, 15, and 22 of each 28-day treatment cycle in addition to their current dose of pomalidomide. Participants with PD who chose not to add dexamethasone to pomalidomide therapy were to be discontinued from the study. Participants who chose to add dexamethasone were allowed to continue study treatment until PD developed again, unacceptable toxicity or participant withdrew consent, at which time they were to be discontinued.
Based on results from the phase 1 portion, the Data Monitoring Committee confirmed 4 mg/day as MTD of pomalidomide. Therefore, the recommended starting dose of pomalidomide for Phase 2 was 4 mg/day on Days 1-21 of each 28-day cycle. In the combination treatment arm, the starting dose of dexamethasone was 40 mg once per day. For subjects who were > 75 years of age, the starting dose of dexamethasone was 20 mg once per day. To prevent blood clots, all participants were to be given aspirin 81-100 mg daily (commercial supply) unless contraindicated. If contraindicated, another form of anti-thrombotic therapy was provided.
Participants in the Phase II combination treatment arm could continue study treatment until PD developed, at which time they were to be discontinued. Participants in the single agent pomalidomide treatment arm who developed PD, confirmed by the IRAC, at any time had the option to receive oral dexamethasone in addition to their current dose of pomalidomide at the starting dose described above. Participants with PD who chose not to add dexamethasone to pomalidomide therapy were discontinued from study treatment. Participants who chose to add dexamethasone to pomalidomide therapy could continue study treatment until PD developed again, unacceptable toxicity or participant withdrew consent, at which point they were discontinued.
Upon discontinuation from study treatment for PD or any other reason, participants were to be assessed two times per year, up to five years, for survival, second primary malignancy and subsequent anti-myeloma therapies.
Two analyses were planned during the course of the Phase 2 segment: one interim analysis (at 50% information of progression-free survival (PFS) events) and one final analysis. The Data Monitoring Commmittee recommended that Celgene personnel be unblinded based on the strength of the data. Subsequently, Celgene decided to file an application based on more current study data. The product was approved by the FDA in February 2013.
Since no further analyses are required per protocol nor to support the marketing application, the study was amended to remove undue burden from patients who remain on active treatment by ending the treatment segments of this study and transferring all active patients tothe Long-term Follow-up Phase. These patients who continue to be treated with pomalidomide will be provided Pomalyst through the Celgene Patient Support Program (Pomalyst REMSTM) until disease progression, unacceptable toxicity, the investigator decides to change therapy or patient decision. Investigators will treat their patients according to the local standard of care and follow the assessments required for patients in the Long-term Follow-up Phase. These assessments include subsequent myeloma therapies, second primary malignancies and survival.
The study continues. A final analysis will be performed when the study is completed and results reported as available.
3,712 studies on the registry are indexed under Multiple Myeloma; 752 are open to participants now.
This study's enrollment of 259 is above the median of 41 across 2,965 interventional studies indexed under Multiple Myeloma.
Browse Multiple Myeloma studies →Celgene Corporation is the lead sponsor of 52 studies on the registry; none are open to participants now.
First submitted before Celgene became part of Bristol-Myers Squibb.
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Exclusion Criteria:
Any of the following laboratory abnormalities:
Prior history of malignancies, other than multiple myeloma, unless the patient has been free of the disease for ≥ 3 years. Exceptions include the following:
Pomalidomide 2 mg daily on days 1-21 of each 28-day cycle. Participants with progressive disease (PD) had the option of adding dexamethasone 40 mg on Days 1, 8, 15, 22 of each 28-day cycle to the pomalidomide treatment, or discontinuing study treatment.
Drug: Pomalidomide · Drug: Dexamethasone · Drug: Aspirin
Pomalidomide 3 mg daily on days 1-21 of each 28-day cycle. Participants with progressive disease (PD) had the option of adding dexamethasone 40 mg on Days 1, 8, 15, 22 of each 28-day cycle to the pomalidomide treatment, or discontinuing study treatment.
Drug: Pomalidomide · Drug: Dexamethasone · Drug: Aspirin
Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Participants with progressive disease (PD) had the option of adding dexamethasone 40 mg on Days 1, 8, 15, 22 of each 28-day cycle to the pomalidomide treatment, or discontinuing study treatment.
Drug: Pomalidomide · Drug: Dexamethasone · Drug: Aspirin
Pomalidomide 5 mg daily on days 1-21 of each 28-day cycle. Participants with progressive disease (PD) had the option of adding dexamethasone 40 mg on Days 1, 8, 15, 22 of each 28-day cycle to the pomalidomide treatment, or discontinuing study treatment.
Drug: Pomalidomide · Drug: Dexamethasone · Drug: Aspirin
Combination therapy of 4 mg pomalidomide given once per day on Days 1-21 of each 28-day cycle and the starting dose of dexamethasone (determined by age) on days 1, 8, 15, and 22 of each 28-day cycle. The starting dose of dexamethasone was 40 mg for participants who were ≤ 75 years of age and 20 mg for participants who were \> 75 years of age. Dose reduction steps for dexamethasone were provided for drug-related toxicities.
Drug: Pomalidomide · Drug: Dexamethasone · Drug: Aspirin
4 mg pomalidomide was given once per day on Days 1-21 of each 28-day cycle until PD. Participants in the single agent pomalidomide treatment arm who developed confirmed PD at any time had the option to receive oral dexamethasone on days 1, 8, 15, and 22 of each 28-day cycle at the starting dose of 20 or 40 mg depending on age in addition to their current dose of pomalidomide, or to discontinue treatment.
Drug: Pomalidomide · Drug: Dexamethasone · Drug: Aspirin
1 mg, 2 mg, and 5 mg capsules for oral administration packaged in bottles containing a 21-day supply
Also known as: CC-4047, Pomalyst
oral dexamethasone
Also known as: dexamethasone sodium phosphate
As prophylactic anti-thrombotic treatment, all participants were given aspirin 81-100 mg daily (commercial supply) unless contraindicated. If aspirin was contraindicated, participants were given another form of anti-thrombotic therapy according to hospital guidelines or physician preference.
Phase 1: Participants With Dose-Limiting Toxicities (DLT) in Cycle 1
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
Time frame: Up to Day 28 (Cycle 1)
Phase 2: Kaplan-Meier Estimates of Progression-free Survival (PFS) as of the 01 April 2011 Cut-off
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.
Time frame: up to 67 weeks
Phase 2: Percentage of Participants With Progression-Free Survival (PFS) Events as of the 01 April 2011 Cut-off
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.
Time frame: up to 67 weeks
Phase 1: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) While on Single-Agent Pomalidomide as of the 01 April 2011 Cut-off
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.
Time frame: Up to week 104
Phase 1: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) While on Both Pomalidomide and Dexamethasone as of the 01 April 2011 Cut-off
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.
Time frame: Up to week 126
Phase 2: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) as of the 01 April 2011 Cut-off
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.
Time frame: Up to week 70
Phase 2: Summary of Best Myeloma Response As Assessed by Independent Response Adjudication Committee (IRAC) Using European Group for Blood and Bone Marrow Transplant (EBMT) Criteria as of the 01 April 2011 Cut-off
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.
Time frame: up to 70 weeks
Phase 2: Kaplan-Meier Estimates of Duration of Response as of the 01 April 2011 Cut-off
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 frame: up to 70 weeks
Phase 2: Time to Response as of the 01 April 2011 Cut-off
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.
Time frame: up to 70 weeks
Phase 2: Kaplan-Meier Estimates of Overall Survival as of the 01 April 2011 Cut-off
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.
Time frame: up to 70 weeks
| Milestone | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide |
|---|---|---|---|---|---|---|
| Started | 6 | 8 | 14 | 10 | 0 | 0 |
| Completed | 2 | 3 | 5 | 4 | 0 | 0 |
| Not completed | 4 | 5 | 9 | 6 | 0 | 0 |
| Withdrew: Still active in study | 0 | 0 | 2 | 2 | 0 | 0 |
| Withdrew: Withdrawal by subject | 1 | 1 | 2 | 2 | 0 | 0 |
| Withdrew: Adverse event | 1 | 0 | 2 | 1 | 0 | 0 |
| Withdrew: Death | 0 | 1 | 2 | 0 | 0 | 0 |
| Withdrew: Disease progression - unconfirmed | 1 | 0 | 1 | 0 | 0 | 0 |
| Withdrew: Other | 1 | 3 | 0 | 1 | 0 | 0 |
| Milestone | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide |
|---|---|---|---|---|---|---|
| Started | 0 | 0 | 0 | 0 | 113 | 108 |
| Safety population | 0 | 0 | 0 | 0 | 112 | 107 |
| Completed | 0 | 0 | 0 | 0 | 58 | 47 |
| Not completed | 0 | 0 | 0 | 0 | 55 | 61 |
| Withdrew: Still active in study | 0 | 0 | 0 | 0 | 23 | 22 |
| Withdrew: Adverse event | 0 | 0 | 0 | 0 | 8 | 13 |
| Withdrew: Death | 0 | 0 | 0 | 0 | 8 | 9 |
| Withdrew: Disease progression - unconfirmed | 0 | 0 | 0 | 0 | 7 | 6 |
| Withdrew: Withdrawal by subject | 0 | 0 | 0 | 0 | 5 | 7 |
| Withdrew: Other | 0 | 0 | 0 | 0 | 4 | 3 |
| Withdrew: Lost to follow-up | 0 | 0 | 0 | 0 | 0 | 1 |
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
| participants | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide |
|---|---|---|---|---|
| Phase 1: Participants With Dose-Limiting Toxicities (DLT) in Cycle 1 | 1 | 1 | 2 | 4 |
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.
| weeks | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide |
|---|---|---|
| Phase 2: Kaplan-Meier Estimates of Progression-free Survival (PFS) as of the 01 April 2011 Cut-off | 16.6 (14.1 to 21.1) | 10.7 (8.3 to 16.1) |
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.
| percentage of participants | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide |
|---|---|---|---|---|
| 1 or more (1+) AE | 100.0 | 100.0 | 100.0 | 100.0 |
| 1+ AE related to pomalidomide | 66.7 | 75.0 | 85.7 | 100.0 |
| 1+ severity grade 3-4 AE | 83.3 | 37.5 | 78.6 | 80.0 |
| 1+ severity grade 3-4 AE related to pomalidomide | 33.3 | 25.0 | 42.9 | 70.0 |
| 1+ serious AE (SAE) | 50.0 | 12.5 | 42.9 | 30.0 |
| 1+ SAE related to pomalidomide | 0.0 | 12.5 | 7.1 | 10.0 |
| 1+ AE leading to discontinuation of pomalidomide | 16.7 | 0.0 | 21.4 | 0.0 |
| 1+AE-dose reduction/interruption of pomalidomide | 16.7 | 75.0 | 42.9 | 80.0 |
| 1+related AE-reduction/interruption of pomalidomid | 0.0 | 37.5 | 28.6 | 70.0 |
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.
| percentage of participants | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide |
|---|---|---|---|---|
| 1 or more (1+) AE | 100.0 | 100.0 | 90.0 | 100.0 |
| 1+ AE related to pomalidomide | 100.0 | 75.0 | 80.0 | 100.0 |
| 1+ AE related to dexamethasone | 100.0 | 75.0 | 70.0 | 85.7 |
| 1+ severity grade 3-4 AE | 0.0 | 75.0 | 70.0 | 57.1 |
| 1+ severity grade 3-4 AE related to pomalidomide | 0.0 | 75.0 | 20.0 | 42.9 |
| 1+ severity grade 3-4 AE related to dexamethasone | 0.0 | 75.0 | 20.0 | 0.0 |
| 1+ serious AE (SAE) | 0.0 | 75.0 | 40.0 | 28.6 |
| 1+ SAE related to pomalidomide | 0.0 | 25.0 | 20.0 | 0.0 |
| 1+ SAE related to dexamethasone | 0.0 | 50.0 | 10.0 | 0.0 |
| 1+ AE leading to discontinuation of pomalidomide | 0.0 | 25.0 | 20.0 | 0.0 |
| 1+ AE -- discontinuation of dexamethasone | 0.0 | 25.0 | 20.0 | 0.0 |
| 1+AE -dose reduction/interruption of pomalidomide | 0.0 | 50.0 | 40.0 | 71.4 |
| 1+ AE-dose reduction/interruption of dexamethasone | 0.0 | 75.0 | 60.0 | 71.4 |
| 1+related AE-reduction/interruption of pomalidomid | 0.0 | 25.0 | 20.0 | 57.1 |
| 1+related AE-reduction/interruption of dexamethaso | 0.0 | 75.0 | 20.0 | 42.9 |
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.
| percentage of participants | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide (Pom Only) | Phase 2: Pomalidomide (Pom + Dex Only) | Phase 2: Pomalidomide (Overall) |
|---|---|---|---|---|
| 1 or more (1+) AE | 100.0 | 99.1 | 93.4 | 100.0 |
| 1+ AE related to pomalidomide | 89.3 | 87.9 | 68.9 | 88.8 |
| 1+ severity grade 3-4 AE | 88.4 | 84.1 | 70.5 | 89.7 |
| 1+ severity grade 3-4 AE related to pomalidomide | 62.5 | 58.9 | 44.3 | 67.3 |
| 1+ serious AE (SAE) | 61.6 | 46.7 | 47.5 | 67.3 |
| 1+ SAE related to pomalidomide | 17.9 | 9.3 | 19.7 | 20.6 |
| 1+ AE leading to discontinuation of pomalidomide | 8.0 | 10.3 | 3.3 | 12.1 |
| 1+related AE --discontinuation of pomalidomide | 1.8 | 2.8 | 1.6 | 3.7 |
| 1+AE - reduction of pomalidomide | 20.5 | 25.2 | 9.8 | 29.9 |
| 1+ AE - interruption of pomalidomide | 63.4 | 47.7 | 36.1 | 58.9 |
| 1+ related AE - interruption of pomalidomide | 27.7 | 24.3 | 21.3 | 32.7 |
| 1+related AE - reduction of pomalidomide | 17.9 | 20.6 | 8.2 | 24.3 |
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.
| percentage of participants | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide |
|---|---|---|
| Complete response (CR) | 0.9 | 0.0 |
| Partial response (PR) | 29.2 | 9.3 |
| Minimal response (MR) | 15.0 | 15.7 |
| Stable disease (SD) | 35.4 | 46.3 |
| Progressive disease (PD) | 6.2 | 15.7 |
| Not evaluable | 13.3 | 13.0 |
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.
| weeks | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide |
|---|---|---|
| Phase 2: Kaplan-Meier Estimates of Duration of Response as of the 01 April 2011 Cut-off | 32.1 (22.1 to 39.9) | NA (NA to NA) |
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.
| weeks | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide |
|---|---|---|
| Phase 2: Time to Response as of the 01 April 2011 Cut-off | 8.1 (3.7 to 45.1) | 8.9 (4.1 to 49.6) |
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.
| weeks | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide |
|---|---|---|
| Phase 2: Kaplan-Meier Estimates of Overall Survival as of the 01 April 2011 Cut-off | 62.6 (53.6 to NA) | 59.3 (41.6 to NA) |
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.
| percentage of participants | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide |
|---|---|---|
| Phase 2: Percentage of Participants With Progression-Free Survival (PFS) Events as of the 01 April 2011 Cut-off | 76.1 | 75.0 |
Collected over Data cut-off 01 April 2011 Phase 1: up to 126 weeks Phase 2: up to 70 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Phase 1: 2 mg Pomalidomide | — | 3/6 (50%) | 6/6 (100%) |
| Phase 1: 3 mg Pomalidomide | — | 4/8 (50%) | 8/8 (100%) |
| Phase 1: 4 mg Pomalidomide | — | 8/14 (57.1%) | 14/14 (100%) |
| Phase 1: 5 mg Pomalidomide | — | 4/10 (40%) | 10/10 (100%) |
| Phase 2: Pomalidomide + Dexamethasone | — | 69/112 (61.6%) | 112/112 (100%) |
| Phase 2: Pomalidomide (Pom Only) | — | 50/107 (46.7%) | 105/107 (98.1%) |
| Phase 2: Pomalidomide (Pom + Dex Only) | — | 29/61 (47.5%) | 57/61 (93.4%) |
| Phase 2: Pomalidomide (Overall) | — | 72/107 (67.3%) | 106/107 (99.1%) |
| Event | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide (Pom Only) | Phase 2: Pomalidomide (Pom + Dex Only) | Phase 2: Pomalidomide (Overall) |
|---|---|---|---|---|---|---|---|---|
| SepsisInfections and infestations | 1/6 | 2/8 | 0/14 | 1/10 | 3/112 | 4/107 | 2/61 | 6/107 |
| PneumoniaInfections and infestations | 1/6 | 0/8 | 2/14 | 0/10 | 21/112 | 10/107 | 6/61 | 15/107 |
| BacteraemiaInfections and infestations | 1/6 | 0/8 | 0/14 | 0/10 | 1/112 | 0/107 | 0/61 | 0/107 |
| Renal failureRenal and urinary disorders | 1/6 | 0/8 | 1/14 | 0/10 | 1/112 | 1/107 | 1/61 | 2/107 |
| Back painMusculoskeletal and connective tissue disorders | 1/6 | 0/8 | 0/14 | 1/10 | 2/112 | 2/107 | 3/61 | 4/107 |
| Confusional statePsychiatric disorders | 1/6 | 0/8 | 0/14 | 0/10 | 2/112 | 0/107 | 2/61 | 2/107 |
| Arthritis infectiveInfections and infestations | 0/6 | 1/8 | 0/14 | 0/10 | 0/112 | 0/107 | 0/61 | 0/107 |
| Pharyngeal abscessInfections and infestations | 0/6 | 1/8 | 0/14 | 0/10 | 0/112 | 0/107 | 0/61 | 0/107 |
| DehydrationMetabolism and nutrition disorders | 0/6 | 1/8 | 0/14 | 0/10 | 3/112 | 4/107 | 1/61 | 5/107 |
| AnemiaBlood and lymphatic system disorders | 0/6 | 1/8 | 0/14 | 0/10 | 2/112 | 1/107 | 1/61 | 2/107 |
| Event | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide (Pom Only) | Phase 2: Pomalidomide (Pom + Dex Only) | Phase 2: Pomalidomide (Overall) |
|---|---|---|---|---|---|---|---|---|
| NeutropeniaBlood and lymphatic system disorders | 1/6 | 5/8 | 8/14 | 9/10 | 52/112 | 53/107 | 17/61 | 55/107 |
| AnaemiaBlood and lymphatic system disorders | 5/6 | 4/8 | 5/14 | 3/10 | 43/112 | 36/107 | 11/61 | 41/107 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 3/6 | 6/8 | 2/14 | 3/10 | 33/112 | 23/107 | 6/61 | 26/107 |
| FatigueGeneral disorders | 4/6 | 5/8 | 9/14 | 7/10 | 62/112 | 46/107 | 11/61 | 53/107 |
| AstheniaGeneral disorders | 3/6 | 2/8 | 1/14 | 0/10 | 22/112 | 11/107 | 5/61 | 15/107 |
| NauseaGastrointestinal disorders | 2/6 | 1/8 | 3/14 | 5/10 | 24/112 | 29/107 | 10/61 | 37/107 |
| Back painMusculoskeletal and connective tissue disorders | 3/6 | 0/8 | 3/14 | 1/10 | 32/112 | 26/107 | 10/61 | 33/107 |
| CoughRespiratory, thoracic and mediastinal disorders | 1/6 | 0/8 | 7/14 | 2/10 | 23/112 | 14/107 | 4/61 | 15/107 |
| Muscle spasmsMusculoskeletal and connective tissue disorders | 2/6 | 1/8 | 6/14 | 2/10 | 21/112 | 17/107 | 7/61 | 20/107 |
| ConstipationGastrointestinal disorders | 2/6 | 1/8 | 2/14 | 4/10 | 38/112 | 29/107 | 11/61 | 38/107 |
Intent to treat population from both phases
| Age, Continuous(years) | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide | Total |
|---|---|---|---|---|---|---|---|
| Mean | 64.7 ± 6.83 | 70.4 ± 6.63 | 67.5 ± 8.98 | 61.3 ± 14.06 | 64.4 ± 9.24 | 62.9 ± 10.35 | 64.1 ± 9.86 |
| Age, Customized(participants) | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide | Total |
|---|---|---|---|---|---|---|---|
| <=75 years | 6 | 6 | 11 | 9 | 99 | 95 | 226 |
| >75 years | 0 | 2 | 3 | 1 | 14 | 13 | 33 |
| Sex: Female, Male(Participants) | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide | Total |
|---|---|---|---|---|---|---|---|
| Female | 5 | 5 | 4 | 6 | 51 | 51 | 122 |
| Male | 1 | 3 | 10 | 4 | 62 | 57 | 137 |
| Race/Ethnicity, Customized(participants) | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide | Total |
|---|---|---|---|---|---|---|---|
| Asian | 0 | 0 | 0 | 2 | 2 | 3 | 7 |
| Black or African American | 1 | 0 | 0 | 0 | 17 | 16 | 34 |
| White | 5 | 8 | 14 | 8 | 92 | 86 | 213 |
| Other | 0 | 0 | 0 | 0 | 2 | 3 | 5 |
| Baseline Electrocardiogram Findings(participants) | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide | Total |
|---|---|---|---|---|---|---|---|
| Normal | 3 | 3 | 3 | 4 | 53 | 44 | 110 |
| Abnormal, not clinically significant | 3 | 5 | 11 | 6 | 56 | 59 | 140 |
| Abnormal, clinically significant | 0 | 0 | 0 | 0 | 0 | 1 | 1 |
| Missing | 0 | 0 | 0 | 0 | 4 | 4 | 8 |
| Eastern Cooperative Oncology Group Performance Status(participants) | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide | Total |
|---|---|---|---|---|---|---|---|
| 0 | 1 | 0 | 3 | 2 | 32 | 24 | 62 |
| 1 | 1 | 8 | 10 | 5 | 68 | 71 | 163 |
| 2 | 4 | 0 | 1 | 3 | 13 | 11 | 32 |
| 3 | 0 | 0 | 0 | 0 | 0 | 2 | 2 |
| Baseline Multiple Myeloma Stage(participants) | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide | Total |
|---|---|---|---|---|---|---|---|
| Stage I | 1 | 1 | 3 | 2 | 8 | 8 | 23 |
| Stage II | 0 | 1 | 4 | 1 | 29 | 29 | 64 |
| Stage III | 5 | 6 | 7 | 7 | 76 | 71 | 172 |
| Prior Anti-Myeloma Therapies(participants) | Phase 1: 2 mg Pomalidomide | Phase 1: 3 mg Pomalidomide | Phase 1: 4 mg Pomalidomide | Phase 1: 5 mg Pomalidomide | Phase 2: Pomalidomide + Dexamethasone | Phase 2: Pomalidomide | Total |
|---|---|---|---|---|---|---|---|
| Yes | 6 | 8 | 14 | 10 | 113 | 108 | 259 |
| No | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
2 further baseline measures are reported on the registry.
The record gives a start of Jun 2008, a primary completion of Apr 2011 and a completion of Sep 2015. It does not say whether these dates are actual or estimated.
Results were first posted in Apr 2013, 2 years after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Mar 2016. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Pomalidomide · Dexamethasone