Trial Outcomes & Findings for Pomalidomide in Gene Expression Profiling (GEP)-Defined High-risk Multiple Myeloma (NCT NCT01177735)
NCT ID: NCT01177735
Last Updated: 2021-04-22
Results Overview
Progression -free survival (PFS) after initiation of pomalidomide therapy. Progressive disease is defined as increase of \> 25% from lowest response value in any one or more of the following: Serum M-component and/or (the absolute increase must be \> 0.5 g/dL); Urine M-component and/or (the absolute increase must be \> 200 mg/24 h); Only in patients without measurable serum and urine M-protein levels; the difference between involved and uninvolved FLC levels. The absolute increase must be \> 10 mg/dL; Bone marrow plasma cell percentage; the absolute percentage must be \> 10%; Definite development of new bone lesions or soft tissue plasmacytomas or definite increase in the size of existing bone lesions or soft tissue plasmacytomas; Development of hypercalcaemia (corrected serum calcium \> 11.5 mg/dL or 2.65 mmol/L) that can be attributed solely to the plasma cell proliferative disorder.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
71 participants
Primary outcome timeframe
1 year following initiation of pomalidomide therapy
Results posted on
2021-04-22
Participant Flow
Participant milestones
| Measure |
Pomalidomide
Pomalidomide: Only enough CC-4047 for 1 cycle of therapy may be provided to the patient each cycle. Participants will receive CC-4047 4 mg/day for 21 days, every 28 days. Treatment will continue until disease recurrence or untoward toxicity.
|
|---|---|
|
Overall Study
STARTED
|
71
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
71
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Pomalidomide in Gene Expression Profiling (GEP)-Defined High-risk Multiple Myeloma
Baseline characteristics by cohort
| Measure |
Pomalidomide
n=71 Participants
Pomalidomide: Only enough CC-4047 for 1 cycle of therapy may be provided to the patient each cycle. Participants will receive CC-4047 4 mg/day for 21 days, every 28 days. Treatment will continue until disease recurrence or untoward toxicity.
|
|---|---|
|
Age, Continuous
|
64.25 years
STANDARD_DEVIATION 76.161 • n=5 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
30 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
41 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
28 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
43 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
71 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 year following initiation of pomalidomide therapyProgression -free survival (PFS) after initiation of pomalidomide therapy. Progressive disease is defined as increase of \> 25% from lowest response value in any one or more of the following: Serum M-component and/or (the absolute increase must be \> 0.5 g/dL); Urine M-component and/or (the absolute increase must be \> 200 mg/24 h); Only in patients without measurable serum and urine M-protein levels; the difference between involved and uninvolved FLC levels. The absolute increase must be \> 10 mg/dL; Bone marrow plasma cell percentage; the absolute percentage must be \> 10%; Definite development of new bone lesions or soft tissue plasmacytomas or definite increase in the size of existing bone lesions or soft tissue plasmacytomas; Development of hypercalcaemia (corrected serum calcium \> 11.5 mg/dL or 2.65 mmol/L) that can be attributed solely to the plasma cell proliferative disorder.
Outcome measures
| Measure |
Pomalidomide
n=71 Participants
Pomalidomide: Only enough CC-4047 for 1 cycle of therapy may be provided to the patient each cycle. Participants will receive CC-4047 4 mg/day for 21 days, every 28 days. Treatment will continue until disease recurrence or untoward toxicity.
|
|---|---|
|
Progression-free Survival (PFS) After Initiation of Pomalidomide Therapy
|
13 percentage of participants
|
Adverse Events
Pomalidomide
Serious events: 6 serious events
Other events: 71 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Pomalidomide
n=71 participants at risk
Pomalidomide: Only enough CC-4047 for 1 cycle of therapy may be provided to the patient each cycle. Participants will receive CC-4047 4 mg/day for 21 days, every 28 days. Treatment will continue until disease recurrence or untoward toxicity.
|
|---|---|
|
Renal and urinary disorders
Acute renal failure
|
2.8%
2/71 • Number of events 2
|
|
Infections and infestations
Hospitalization for sinus infection
|
1.4%
1/71 • Number of events 1
|
|
Infections and infestations
Hospialization for progressive disease, sepsis and pneumonia
|
4.2%
3/71 • Number of events 3
|
|
Injury, poisoning and procedural complications
Hospitalization for removal of port
|
1.4%
1/71 • Number of events 1
|
|
Infections and infestations
Hospitalization for fever
|
1.4%
1/71 • Number of events 1
|
|
Infections and infestations
RSV Pneumonia
|
2.8%
2/71 • Number of events 2
|
|
Infections and infestations
Fever neutropenia dehydration
|
1.4%
1/71 • Number of events 1
|
|
Infections and infestations
Hospitalization for neutropenic fever
|
5.6%
4/71 • Number of events 4
|
|
Infections and infestations
Hospitalization due to acute liver failure hepatitis B flare
|
1.4%
1/71 • Number of events 1
|
|
Infections and infestations
Hospitalization for pneumonia
|
1.4%
1/71 • Number of events 1
|
|
Cardiac disorders
Atrial Fibrilation
|
2.8%
2/71 • Number of events 2
|
|
Blood and lymphatic system disorders
Pancytopenia
|
1.4%
1/71 • Number of events 1
|
|
General disorders
Hypoxia
|
1.4%
1/71 • Number of events 1
|
|
Cardiac disorders
Hospitalization dyspnea due to congestive heart failure
|
1.4%
1/71 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Hospitalization for shortness of breath
|
1.4%
1/71 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
1.4%
1/71 • Number of events 1
|
|
Cardiac disorders
Dyspnea on exertion
|
1.4%
1/71 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Hospitalization for bronchitis
|
1.4%
1/71 • Number of events 1
|
|
Blood and lymphatic system disorders
Epistaxis
|
1.4%
1/71 • Number of events 1
|
|
Cardiac disorders
Hospitalization for hypotension
|
2.8%
2/71 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure secondary to opioid overdose
|
1.4%
1/71 • Number of events 1
|
|
Nervous system disorders
Hospitalization for intractable pain, low fever
|
1.4%
1/71 • Number of events 1
|
|
Nervous system disorders
Hospitalization for acute respiratory distress syndrome, encephalopathy, fatigue
|
1.4%
1/71 • Number of events 1
|
|
Infections and infestations
Pneumonia
|
5.6%
4/71 • Number of events 4
|
|
Surgical and medical procedures
Intrahepatic chemotherapy
|
1.4%
1/71 • Number of events 1
|
|
Nervous system disorders
Paraparesis, spinal cord compression
|
1.4%
1/71 • Number of events 1
|
|
Gastrointestinal disorders
Small bowel obstruction
|
1.4%
1/71 • Number of events 1
|
|
Injury, poisoning and procedural complications
Fracture of T7 with possible cord compression
|
1.4%
1/71 • Number of events 1
|
|
Cardiac disorders
Deep vein thrombosis and cerebrovascular accident
|
1.4%
1/71 • Number of events 1
|
|
Blood and lymphatic system disorders
Blood transfusion
|
1.4%
1/71 • Number of events 1
|
|
Surgical and medical procedures
Wrong dosage dispensed
|
1.4%
1/71 • Number of events 1
|
|
Cardiac disorders
Hospitalization with fever and low blood pressure
|
1.4%
1/71 • Number of events 1
|
|
Gastrointestinal disorders
Acute diarrhea
|
1.4%
1/71 • Number of events 1
|
|
Gastrointestinal disorders
Hospitalization for Pneumonia and gastrointestinal bleeding
|
1.4%
1/71 • Number of events 1
|
|
Gastrointestinal disorders
Hospitalization for diarrhea and dizziness
|
1.4%
1/71 • Number of events 1
|
|
Infections and infestations
Elevated liver function test
|
1.4%
1/71 • Number of events 1
|
|
Vascular disorders
Deep vein thrombosis diagnosis
|
1.4%
1/71 • Number of events 1
|
|
Infections and infestations
Hospitalization for sepsis
|
1.4%
1/71 • Number of events 1
|
|
Renal and urinary disorders
Worsening renal failure
|
1.4%
1/71 • Number of events 1
|
|
Cardiac disorders
Tachycardia
|
1.4%
1/71 • Number of events 1
|
|
Infections and infestations
Pneumonia, fever and altered mental status
|
1.4%
1/71 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Severe cough, shortness of breath and hypoxemia
|
1.4%
1/71 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of breath fever chills
|
1.4%
1/71 • Number of events 1
|
|
Infections and infestations
Hospitalization for parainfluenza type 2
|
1.4%
1/71 • Number of events 1
|
|
Psychiatric disorders
Mental status changes and bacteremia
|
1.4%
1/71 • Number of events 1
|
|
Infections and infestations
Recurrent multifocal pneumonia
|
1.4%
1/71 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory infection and fever
|
1.4%
1/71 • Number of events 1
|
|
Infections and infestations
Acute onset diarrhea and acute renal insufficiency
|
1.4%
1/71 • Number of events 1
|
|
Vascular disorders
Hospitalization due to transient ischemic attack
|
1.4%
1/71 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of breath with wheezing
|
1.4%
1/71 • Number of events 1
|
|
Infections and infestations
Recurrent bilateral pneumonia
|
1.4%
1/71 • Number of events 1
|
Other adverse events
| Measure |
Pomalidomide
n=71 participants at risk
Pomalidomide: Only enough CC-4047 for 1 cycle of therapy may be provided to the patient each cycle. Participants will receive CC-4047 4 mg/day for 21 days, every 28 days. Treatment will continue until disease recurrence or untoward toxicity.
|
|---|---|
|
Blood and lymphatic system disorders
Leukopenia
|
100.0%
71/71
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
100.0%
71/71
|
|
Blood and lymphatic system disorders
Anemia
|
100.0%
71/71
|
|
General disorders
Blood/Bone Marrow
|
100.0%
71/71
|
|
Cardiac disorders
Cardiac Arrhythmia
|
9.9%
7/71
|
|
Cardiac disorders
Cardiac General
|
16.9%
12/71
|
|
General disorders
Constitutional Symptoms
|
77.5%
55/71
|
|
Skin and subcutaneous tissue disorders
Dermatology / Skin
|
43.7%
31/71
|
|
Gastrointestinal disorders
Gastrointestinal
|
62.0%
44/71
|
|
Injury, poisoning and procedural complications
Hemorrhage / Bleeding
|
11.3%
8/71
|
|
Hepatobiliary disorders
Hepatobiliary / Pancreas
|
98.6%
70/71
|
|
Infections and infestations
Infection
|
40.8%
29/71
|
|
Blood and lymphatic system disorders
Lymphatics
|
14.1%
10/71
|
|
Metabolism and nutrition disorders
Metabolic/Laboratory
|
93.0%
66/71
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal / Soft Tissue
|
33.8%
24/71
|
|
Nervous system disorders
Neurology
|
77.5%
55/71
|
|
Eye disorders
Ocular / Vision
|
8.5%
6/71
|
|
General disorders
Pain
|
56.3%
40/71
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary / Upper Respiratory
|
56.3%
40/71
|
|
Renal and urinary disorders
Renal / Genitourinary
|
63.4%
45/71
|
Additional Information
Saad Usmani, MD
University of Arkansas for Medical Sciences
Phone: 501-526-6876
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place