Trial Outcomes & Findings for Continuous Versus Intermittent Dosing Regimens for Pomalidomide in Relapsed/Refractory Multiple Myeloma (NCT NCT01319422)
NCT ID: NCT01319422
Last Updated: 2016-08-11
Results Overview
All partial and complete responses must be confirmed with another efficacy assessment in no less than 4 weeks apart.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
40 participants
Primary outcome timeframe
Efficacy assessments will be made after the first two cycles of therapy (approximately 56 days--each cycle is 28 days)
Results posted on
2016-08-11
Participant Flow
Recruitment took place at an academic medical center.
Four screened subjects did not meet eligibility criteria. Forty subjects were enrolled, but one withdrew after randomization without receiving treatment, and is listed as a "non-completer". Thus, data are presented for 39 participants.
Participant milestones
| Measure |
Pomalidomide 2 mg/d on 28 Days/28 Day Cycle
Pomalidomide: Comparison of different dosages and schedules of drug
|
Pomalidomide 4 mg/d on 21 Days/28 Day Cycle
Pomalidomide: Comparison of different dosages and schedules of drug
|
|---|---|---|
|
Overall Study
STARTED
|
20
|
20
|
|
Overall Study
COMPLETED
|
19
|
20
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Pomalidomide 2 mg/d on 28 Days/28 Day Cycle
Pomalidomide: Comparison of different dosages and schedules of drug
|
Pomalidomide 4 mg/d on 21 Days/28 Day Cycle
Pomalidomide: Comparison of different dosages and schedules of drug
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
Baseline Characteristics
Continuous Versus Intermittent Dosing Regimens for Pomalidomide in Relapsed/Refractory Multiple Myeloma
Baseline characteristics by cohort
| Measure |
Pomalidomide 2 mg/d on 28 Days/28 Day Cycle
n=19 Participants
Pomalidomide: Comparison of different dosages and schedules of drug
|
Pomalidomide 4 mg/d on 21 Days/28 Day Cycle
n=20 Participants
Pomalidomide: Comparison of different dosages and schedules of drug
|
Total
n=39 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
63 years
n=5 Participants
|
61 years
n=7 Participants
|
61 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
22 Participants
n=5 Participants
|
|
Immunoglobulin Heavy chain (IgH) type
Immunoglobulin G (IgG)
|
8 participants
n=5 Participants
|
12 participants
n=7 Participants
|
20 participants
n=5 Participants
|
|
Immunoglobulin Heavy chain (IgH) type
Immunoglobulin A (IgA)
|
4 participants
n=5 Participants
|
1 participants
n=7 Participants
|
5 participants
n=5 Participants
|
|
Immunoglobulin Heavy chain (IgH) type
Light chain only
|
7 participants
n=5 Participants
|
7 participants
n=7 Participants
|
14 participants
n=5 Participants
|
|
Immunoglobulin Light chain (IgL) type
kappa
|
13 participants
n=5 Participants
|
15 participants
n=7 Participants
|
28 participants
n=5 Participants
|
|
Immunoglobulin Light chain (IgL) type
lambda
|
6 participants
n=5 Participants
|
5 participants
n=7 Participants
|
11 participants
n=5 Participants
|
|
serum beta 2 microglobulin
|
3.0 mg/L
n=5 Participants
|
3.4 mg/L
n=7 Participants
|
3.2 mg/L
n=5 Participants
|
|
serum albumin
|
3.9 g/dL
n=5 Participants
|
3.9 g/dL
n=7 Participants
|
3.9 g/dL
n=5 Participants
|
|
lines of prior therapy
|
4 lines
n=5 Participants
|
4 lines
n=7 Participants
|
4 lines
n=5 Participants
|
|
Deletion of chromosome 17p
yes
|
4 participants
n=5 Participants
|
3 participants
n=7 Participants
|
7 participants
n=5 Participants
|
|
Deletion of chromosome 17p
no
|
10 participants
n=5 Participants
|
9 participants
n=7 Participants
|
19 participants
n=5 Participants
|
|
Deletion of chromosome 17p
missing
|
5 participants
n=5 Participants
|
8 participants
n=7 Participants
|
13 participants
n=5 Participants
|
|
Chromosome 1q abnormalities
yes
|
10 participants
n=5 Participants
|
8 participants
n=7 Participants
|
18 participants
n=5 Participants
|
|
Chromosome 1q abnormalities
no
|
9 participants
n=5 Participants
|
12 participants
n=7 Participants
|
21 participants
n=5 Participants
|
|
Deletion chromosome 13
yes
|
5 participants
n=5 Participants
|
8 participants
n=7 Participants
|
13 participants
n=5 Participants
|
|
Deletion chromosome 13
no
|
14 participants
n=5 Participants
|
12 participants
n=7 Participants
|
26 participants
n=5 Participants
|
|
Lenalidomide (Len) as most recent therapy
yes
|
6 participants
n=5 Participants
|
9 participants
n=7 Participants
|
15 participants
n=5 Participants
|
|
Lenalidomide (Len) as most recent therapy
no
|
13 participants
n=5 Participants
|
11 participants
n=7 Participants
|
24 participants
n=5 Participants
|
|
Resistance to prior therapy-Len refractory
yes
|
19 participants
n=5 Participants
|
20 participants
n=7 Participants
|
39 participants
n=5 Participants
|
|
Resistance to prior therapy-Len refractory
no
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Resistance to prior therapy-Len+bortezomib refractory
yes
|
15 participants
n=5 Participants
|
16 participants
n=7 Participants
|
31 participants
n=5 Participants
|
|
Resistance to prior therapy-Len+bortezomib refractory
no
|
4 participants
n=5 Participants
|
4 participants
n=7 Participants
|
8 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Efficacy assessments will be made after the first two cycles of therapy (approximately 56 days--each cycle is 28 days)All partial and complete responses must be confirmed with another efficacy assessment in no less than 4 weeks apart.
Outcome measures
| Measure |
Pomalidomide 2 mg/d on 28 Days/28 Day Cycle
n=19 Participants
Pomalidomide: Comparison of different dosages and schedules of drug
|
Pomalidomide 4 mg/d on 21 Days/28 Day Cycle
n=20 Participants
Pomalidomide: Comparison of different dosages and schedules of drug
|
|---|---|---|
|
Percentage of Participants With Response, Analyzed Per International Myeloma Working Group Response Criteria
|
15.8 percentage of participants
|
20.0 percentage of participants
|
PRIMARY outcome
Timeframe: After the initial efficacy assessment at the completion of cycle 2 (at approximately 56 days), efficacy assessments will be made after every other cycle (approximately every 56 days).All partial and complete responses must be confirmed with another efficacy assessment in no less than 4 weeks apart.
Outcome measures
| Measure |
Pomalidomide 2 mg/d on 28 Days/28 Day Cycle
n=19 Participants
Pomalidomide: Comparison of different dosages and schedules of drug
|
Pomalidomide 4 mg/d on 21 Days/28 Day Cycle
n=20 Participants
Pomalidomide: Comparison of different dosages and schedules of drug
|
|---|---|---|
|
Percentage of Participants With Response, Analyzed Per International Myeloma Working Group Response Criteria
cycle 4
|
15.8 percentage of participants
|
40.0 percentage of participants
|
|
Percentage of Participants With Response, Analyzed Per International Myeloma Working Group Response Criteria
cycle 6
|
21.0 percentage of participants
|
40.0 percentage of participants
|
|
Percentage of Participants With Response, Analyzed Per International Myeloma Working Group Response Criteria
cycle 8
|
21.0 percentage of participants
|
45.0 percentage of participants
|
|
Percentage of Participants With Response, Analyzed Per International Myeloma Working Group Response Criteria
cycle 10
|
21.0 percentage of participants
|
45.0 percentage of participants
|
|
Percentage of Participants With Response, Analyzed Per International Myeloma Working Group Response Criteria
cycle 12
|
21.0 percentage of participants
|
45.0 percentage of participants
|
|
Percentage of Participants With Response, Analyzed Per International Myeloma Working Group Response Criteria
cycle 14
|
21.0 percentage of participants
|
45.0 percentage of participants
|
SECONDARY outcome
Timeframe: Research blood draw will be obtained at baseline, and at 2-4 hr (on day 1), 1 wk, and 4 wk after initiation of cycles 1 and 2.Population: N/A: data were not collected on this outcome; initial F actin polymerization assay was unreliable and not reproducible; hence, this outcome could not be pursued.
Correlation to be determined upon completion of study treatment
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Research bone marrow aspirate is obtained at baseline and after completion of 2 cycles of therapy (approximately 56 days)Population: The actin polymerization assay that was to be used for this outcome was not reproducible and data were not analyzed.
Research bone marrow aspirate is obtained to assess response (optional, but recommended), and to document complete remission, if applicable. Correlation to be determined upon completion of study treatment
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Research blood draw will be obtained at baseline, and at 2-4 hr (on day 1), 1 wk, and 4 wk after initiation of cycles 1 and 2.Population: The actin polymerization assay that was to be used for this outcome was not reproducible; hence, the cytokine profile of actin polymerized cells could not be pursued. Data were not analyzed.
Correlation to be determined upon completion of study treatment
Outcome measures
Outcome data not reported
Adverse Events
Pomalidomide 4 mg/d on 21 Days/28 Day Cycle
Serious events: 11 serious events
Other events: 20 other events
Deaths: 0 deaths
Pomalidomide 2 mg/d on 28 Days/28 Day Cycle
Serious events: 7 serious events
Other events: 19 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Pomalidomide 4 mg/d on 21 Days/28 Day Cycle
n=20 participants at risk
Pomalidomide: Comparison of different dosages and schedules of drug
|
Pomalidomide 2 mg/d on 28 Days/28 Day Cycle
n=19 participants at risk
Pomalidomide: Comparison of different dosages and schedules of drug
|
|---|---|---|
|
Infections and infestations
Febrile neutropenia
|
5.0%
1/20
|
15.8%
3/19
|
|
Infections and infestations
Lung infection
|
15.0%
3/20
|
10.5%
2/19
|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/20
|
5.3%
1/19
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/20
|
10.5%
2/19
|
|
Blood and lymphatic system disorders
Leukopenia
|
0.00%
0/20
|
5.3%
1/19
|
|
General disorders
Fever
|
10.0%
2/20
|
10.5%
2/19
|
|
Cardiac disorders
Syncope
|
5.0%
1/20
|
5.3%
1/19
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
15.0%
3/20
|
0.00%
0/19
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
5.0%
1/20
|
0.00%
0/19
|
|
Cardiac disorders
Thromboembolic event
|
5.0%
1/20
|
0.00%
0/19
|
|
Infections and infestations
Infection (Bacteremia)
|
5.0%
1/20
|
0.00%
0/19
|
|
Cardiac disorders
Cardiac Disorders, other
|
5.0%
1/20
|
0.00%
0/19
|
|
Musculoskeletal and connective tissue disorders
Hip Fracture
|
0.00%
0/20
|
5.3%
1/19
|
|
Musculoskeletal and connective tissue disorders
Spinal fracture
|
0.00%
0/20
|
5.3%
1/19
|
|
Renal and urinary disorders
Acute Kidney Injury
|
5.0%
1/20
|
5.3%
1/19
|
|
Endocrine disorders
Hyperglycemia
|
5.0%
1/20
|
0.00%
0/19
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
5.0%
1/20
|
0.00%
0/19
|
|
Infections and infestations
Upper respiratory infection
|
5.0%
1/20
|
0.00%
0/19
|
|
Infections and infestations
Infection (Other-Influenza)
|
5.0%
1/20
|
0.00%
0/19
|
|
Infections and infestations
Skin Infection
|
5.0%
1/20
|
0.00%
0/19
|
Other adverse events
| Measure |
Pomalidomide 4 mg/d on 21 Days/28 Day Cycle
n=20 participants at risk
Pomalidomide: Comparison of different dosages and schedules of drug
|
Pomalidomide 2 mg/d on 28 Days/28 Day Cycle
n=19 participants at risk
Pomalidomide: Comparison of different dosages and schedules of drug
|
|---|---|---|
|
Blood and lymphatic system disorders
Anemia
|
50.0%
10/20
|
31.6%
6/19
|
|
Blood and lymphatic system disorders
Neutropenia
|
45.0%
9/20
|
26.3%
5/19
|
|
Blood and lymphatic system disorders
Leukopenia
|
75.0%
15/20
|
63.2%
12/19
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
45.0%
9/20
|
31.6%
6/19
|
|
General disorders
Fatigue
|
60.0%
12/20
|
78.9%
15/19
|
|
General disorders
Nausea
|
25.0%
5/20
|
21.1%
4/19
|
|
General disorders
Vomiting
|
10.0%
2/20
|
10.5%
2/19
|
|
Hepatobiliary disorders
AST increased
|
25.0%
5/20
|
15.8%
3/19
|
|
Hepatobiliary disorders
ALT increased
|
30.0%
6/20
|
10.5%
2/19
|
|
Gastrointestinal disorders
Constipation
|
20.0%
4/20
|
10.5%
2/19
|
|
Gastrointestinal disorders
Diarrhea
|
20.0%
4/20
|
26.3%
5/19
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
25.0%
5/20
|
21.1%
4/19
|
|
Nervous system disorders
Dysguesia
|
5.0%
1/20
|
15.8%
3/19
|
|
General disorders
Edema Limbs
|
15.0%
3/20
|
10.5%
2/19
|
|
Nervous system disorders
Blurred vision
|
0.00%
0/20
|
10.5%
2/19
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.0%
1/20
|
15.8%
3/19
|
|
General disorders
Fever
|
10.0%
2/20
|
10.5%
2/19
|
|
General disorders
Anorexia
|
25.0%
5/20
|
10.5%
2/19
|
|
Nervous system disorders
Tremor
|
20.0%
4/20
|
15.8%
3/19
|
|
Metabolism and nutrition disorders
Hyponatremia
|
30.0%
6/20
|
15.8%
3/19
|
|
Metabolism and nutrition disorders
Hypokalemia
|
10.0%
2/20
|
10.5%
2/19
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
25.0%
5/20
|
15.8%
3/19
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
5.0%
1/20
|
5.3%
1/19
|
|
General disorders
Pruritus
|
25.0%
5/20
|
15.8%
3/19
|
|
General disorders
Hot flashes
|
25.0%
5/20
|
10.5%
2/19
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
35.0%
7/20
|
31.6%
6/19
|
|
General disorders
Dizziness
|
20.0%
4/20
|
10.5%
2/19
|
|
General disorders
Edema
|
15.0%
3/20
|
0.00%
0/19
|
|
Renal and urinary disorders
Creatinine increased
|
15.0%
3/20
|
5.3%
1/19
|
|
Skin and subcutaneous tissue disorders
Rash
|
25.0%
5/20
|
5.3%
1/19
|
|
Nervous system disorders
Headache
|
20.0%
4/20
|
0.00%
0/19
|
|
Psychiatric disorders
Anxiety
|
10.0%
2/20
|
0.00%
0/19
|
|
Infections and infestations
Upper respiratory infection
|
15.0%
3/20
|
0.00%
0/19
|
|
Nervous system disorders
Memory impairment
|
10.0%
2/20
|
0.00%
0/19
|
|
Infections and infestations
Soft tissue infection
|
10.0%
2/20
|
0.00%
0/19
|
|
Infections and infestations
Lung infection
|
10.0%
2/20
|
5.3%
1/19
|
|
Nervous system disorders
Neuropathy
|
15.0%
3/20
|
5.3%
1/19
|
|
General disorders
Pain in extremity
|
20.0%
4/20
|
5.3%
1/19
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
10.0%
2/20
|
5.3%
1/19
|
|
General disorders
Sore throat
|
10.0%
2/20
|
0.00%
0/19
|
|
Infections and infestations
Bronchitis
|
10.0%
2/20
|
0.00%
0/19
|
|
Infections and infestations
Sinusitis
|
10.0%
2/20
|
0.00%
0/19
|
|
General disorders
Abdominal Pain
|
5.0%
1/20
|
5.3%
1/19
|
|
General disorders
Dry mouth
|
5.0%
1/20
|
5.3%
1/19
|
|
General disorders
Weight loss
|
5.0%
1/20
|
5.3%
1/19
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
0.00%
0/20
|
5.3%
1/19
|
|
Cardiac disorders
Hypertension
|
5.0%
1/20
|
5.3%
1/19
|
|
Nervous system disorders
Confusion
|
5.0%
1/20
|
5.3%
1/19
|
|
Nervous system disorders
Gait disturbance
|
5.0%
1/20
|
5.3%
1/19
|
|
Infections and infestations
Productive cough
|
5.0%
1/20
|
0.00%
0/19
|
|
Nervous system disorders
Heightened sense of smell
|
0.00%
0/20
|
5.3%
1/19
|
|
Musculoskeletal and connective tissue disorders
Jaw pain
|
0.00%
0/20
|
5.3%
1/19
|
|
Nervous system disorders
Amnesia
|
0.00%
0/20
|
5.3%
1/19
|
|
Nervous system disorders
Vertigo
|
0.00%
0/20
|
5.3%
1/19
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/20
|
5.3%
1/19
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
0.00%
0/20
|
5.3%
1/19
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/20
|
5.3%
1/19
|
|
General disorders
Insomnia
|
0.00%
0/20
|
5.3%
1/19
|
|
Renal and urinary disorders
Bladder pain
|
0.00%
0/20
|
5.3%
1/19
|
|
Endocrine disorders
TSH increased
|
0.00%
0/20
|
5.3%
1/19
|
|
Endocrine disorders
T3 increased
|
0.00%
0/20
|
5.3%
1/19
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
0.00%
0/20
|
5.3%
1/19
|
|
Infections and infestations
Periodontal disease
|
0.00%
0/20
|
5.3%
1/19
|
|
General disorders
Flu-like symptoms
|
0.00%
0/20
|
5.3%
1/19
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
5.0%
1/20
|
0.00%
0/19
|
|
General disorders
Erectile dysfunction
|
5.0%
1/20
|
0.00%
0/19
|
|
Endocrine disorders
Libido decreased
|
5.0%
1/20
|
0.00%
0/19
|
|
Nervous system disorders
Muscle weakness, lower limb
|
5.0%
1/20
|
0.00%
0/19
|
|
Gastrointestinal disorders
GERD
|
5.0%
1/20
|
0.00%
0/19
|
|
Cardiac disorders
Presyncope
|
5.0%
1/20
|
0.00%
0/19
|
|
General disorders
Flatulence
|
5.0%
1/20
|
0.00%
0/19
|
|
General disorders
Nasal congestion
|
5.0%
1/20
|
0.00%
0/19
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.0%
1/20
|
0.00%
0/19
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
5.0%
1/20
|
0.00%
0/19
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
5.0%
1/20
|
0.00%
0/19
|
|
Infections and infestations
Infection (other, oral)
|
5.0%
1/20
|
0.00%
0/19
|
|
General disorders
Weight gain
|
5.0%
1/20
|
0.00%
0/19
|
|
General disorders
Pain
|
5.0%
1/20
|
0.00%
0/19
|
|
Psychiatric disorders
Depression
|
5.0%
1/20
|
0.00%
0/19
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place