Trial Outcomes & Findings for CC-5013 With or Without Dexamethasone in Treating Patients With Primary Systemic Amyloidosis (NCT NCT00091260)
NCT ID: NCT00091260
Last Updated: 2017-02-20
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
82 participants
Primary outcome timeframe
1 year
Results posted on
2017-02-20
Participant Flow
Participant milestones
| Measure |
Revlimid
lenalidomide 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
dexamethasone: dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
lenalidomide: 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone
|
|---|---|
|
Overall Study
STARTED
|
82
|
|
Overall Study
COMPLETED
|
82
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
CC-5013 With or Without Dexamethasone in Treating Patients With Primary Systemic Amyloidosis
Baseline characteristics by cohort
| Measure |
Revlimid
n=82 Participants
lenalidomide 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
dexamethasone: dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
lenalidomide: 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
58 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
24 Participants
n=5 Participants
|
|
Gender
Female
|
29 Participants
n=5 Participants
|
|
Gender
Male
|
53 Participants
n=5 Participants
|
|
Dialysis y/n
on dialysis
|
7 Participants
n=5 Participants
|
|
Dialysis y/n
Not on dialysis
|
75 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 yearPopulation: Number of patients who had at least one dose of CC-5013
Outcome measures
| Measure |
Revlimid
n=82 Participants
lenalidomide 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
dexamethasone: dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
lenalidomide: 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone
|
|---|---|
|
Number of Patients Removed From Study Treatment Due to Toxicities
|
31 participants
|
PRIMARY outcome
Timeframe: 3 monthsPopulation: Participants who received at least 3 cycles of single-agent CC-5013 and underwent subsequent evaluation.
Complete response = Absence of detectable monoclonal protein in serum or urine by immunofixation electrophoresis, less than 5% plasma cells on bone marrow biopsy without clonal dominance of kappa or lambda isotype, and normal serum free light chain assay. Partial response= For patients with detectable and quantifiable monoclonal marrow plasmacytosis= a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells. For patients with a detectable monoclonal peak on serum or urine protein electrophoresis= a reduction in the peak height of 50% or more. For patients with quantifiable urinary kappa or lambda chain concentration= a 50% reduction in daily light chain excretion in 24 hour urine. For patients with an elevated serum free light chain assay, a reduction of 50% or more.
Outcome measures
| Measure |
Revlimid
n=24 Participants
lenalidomide 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
dexamethasone: dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
lenalidomide: 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone
|
|---|---|
|
Number of Patients With Hematologic Response With Single-agent CC-5013
|
5 Participants
|
SECONDARY outcome
Timeframe: 1 yearOutcome measures
| Measure |
Revlimid
n=43 Participants
lenalidomide 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
dexamethasone: dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
lenalidomide: 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone
|
|---|---|
|
Number of Patients Who Received Both CC-5013 and Dexamethasone and Had a Hematologic Response
|
26 participants
|
Adverse Events
Revlimid
Serious events: 82 serious events
Other events: 82 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Revlimid
n=82 participants at risk
lenalidomide 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
dexamethasone: dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
lenalidomide: 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone
|
|---|---|
|
Blood and lymphatic system disorders
neutropenia
|
18.3%
15/82 • Number of events 18 • 2 years
|
|
Blood and lymphatic system disorders
leukopenia
|
6.1%
5/82 • Number of events 5 • 2 years
|
|
Blood and lymphatic system disorders
anemia
|
2.4%
2/82 • Number of events 2 • 2 years
|
|
Blood and lymphatic system disorders
thrombocytopenia
|
1.2%
1/82 • Number of events 1 • 2 years
|
|
Cardiac disorders
hypotension
|
3.7%
3/82 • Number of events 3 • 2 years
|
|
Cardiac disorders
Myocardial infarction
|
1.2%
1/82 • Number of events 1 • 2 years
|
|
Cardiac disorders
organ rejection
|
100.0%
1/1 • Number of events 1 • 2 years
|
|
Cardiac disorders
cardiac, other
|
6.1%
5/82 • Number of events 5 • 2 years
|
|
General disorders
Fatigue
|
22.0%
18/82 • Number of events 18 • 2 years
|
|
General disorders
Decreased Performance Status
|
18.3%
15/82 • Number of events 15 • 2 years
|
|
Infections and infestations
fever/infection
|
4.9%
4/82 • Number of events 6 • 2 years
|
|
Skin and subcutaneous tissue disorders
rash
|
11.0%
9/82 • Number of events 10 • 2 years
|
|
General disorders
Nail Changes
|
1.2%
1/82 • Number of events 1 • 2 years
|
|
Gastrointestinal disorders
diarrhea
|
3.7%
3/82 • Number of events 3 • 2 years
|
|
General disorders
dysphagia
|
1.2%
1/82 • Number of events 1 • 2 years
|
|
Gastrointestinal disorders
nausea
|
2.4%
2/82 • Number of events 2 • 2 years
|
|
Infections and infestations
infection
|
23.2%
19/82 • Number of events 19 • 2 years
|
|
Renal and urinary disorders
increased creatinine
|
7.3%
6/82 • Number of events 8 • 2 years
|
|
Investigations
decreased phosphate
|
15.9%
13/82 • Number of events 13 • 2 years
|
|
Infections and infestations
increased INR
|
2.4%
2/82 • Number of events 2 • 2 years
|
|
Musculoskeletal and connective tissue disorders
muscle cramps
|
1.2%
1/82 • Number of events 1 • 2 years
|
|
General disorders
dizziness
|
7.3%
6/82 • Number of events 6 • 2 years
|
|
Gastrointestinal disorders
pancreatitis
|
1.2%
1/82 • Number of events 1 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
dyspnea
|
3.7%
3/82 • Number of events 3 • 2 years
|
|
Vascular disorders
thrombosis
|
7.3%
6/82 • Number of events 6 • 2 years
|
|
General disorders
gout
|
4.9%
4/82 • Number of events 6 • 2 years
|
Other adverse events
| Measure |
Revlimid
n=82 participants at risk
lenalidomide 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
dexamethasone: dexamethasone 20 mg daily (10 mg twice daily) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
lenalidomide: 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone
|
|---|---|
|
Blood and lymphatic system disorders
neutropenia
|
31.7%
26/82 • Number of events 32 • 2 years
|
|
Blood and lymphatic system disorders
leukopenia
|
25.6%
21/82 • Number of events 26 • 2 years
|
|
Blood and lymphatic system disorders
anemia
|
63.4%
52/82 • Number of events 53 • 2 years
|
|
Blood and lymphatic system disorders
thrombocytopenia
|
32.9%
27/82 • Number of events 31 • 2 years
|
|
Cardiac disorders
hypotension
|
8.5%
7/82 • Number of events 9 • 2 years
|
|
Cardiac disorders
Chest pain
|
8.5%
7/82 • Number of events 7 • 2 years
|
|
Cardiac disorders
cardiac, other
|
6.1%
5/82 • Number of events 5 • 2 years
|
|
General disorders
fatigue
|
65.9%
54/82 • Number of events 61 • 2 years
|
|
General disorders
drowsiness
|
13.4%
11/82 • Number of events 15 • 2 years
|
|
General disorders
Decreased performance status
|
36.6%
30/82 • Number of events 30 • 2 years
|
|
Infections and infestations
fever/infection
|
25.6%
21/82 • Number of events 23 • 2 years
|
|
General disorders
Insomnia
|
47.6%
39/82 • Number of events 39 • 2 years
|
|
General disorders
Weight change
|
19.5%
16/82 • Number of events 19 • 2 years
|
|
Skin and subcutaneous tissue disorders
rash
|
35.4%
29/82 • Number of events 30 • 2 years
|
|
Skin and subcutaneous tissue disorders
itching
|
26.8%
22/82 • Number of events 23 • 2 years
|
|
General disorders
Hot flashes
|
4.9%
4/82 • Number of events 4 • 2 years
|
|
Skin and subcutaneous tissue disorders
Derm/skin other
|
8.5%
7/82 • Number of events 7 • 2 years
|
|
Endocrine disorders
cushingoid
|
4.9%
4/82 • Number of events 4 • 2 years
|
|
Gastrointestinal disorders
Anorexia
|
17.1%
14/82 • Number of events 14 • 2 years
|
|
Gastrointestinal disorders
early satiety
|
22.0%
18/82 • Number of events 18 • 2 years
|
|
Gastrointestinal disorders
Change in appetite
|
14.6%
12/82 • Number of events 12 • 2 years
|
|
Gastrointestinal disorders
constipation
|
37.8%
31/82 • Number of events 33 • 2 years
|
|
Gastrointestinal disorders
diarrhea
|
36.6%
30/82 • Number of events 34 • 2 years
|
|
Gastrointestinal disorders
dysphagia
|
4.9%
4/82 • Number of events 4 • 2 years
|
|
General disorders
dry membranes
|
15.9%
13/82 • Number of events 13 • 2 years
|
|
Gastrointestinal disorders
hemorrhoids
|
15.9%
13/82 • Number of events 13 • 2 years
|
|
Gastrointestinal disorders
dyspepsia
|
20.7%
17/82 • Number of events 17 • 2 years
|
|
Gastrointestinal disorders
nausea
|
29.3%
24/82 • Number of events 28 • 2 years
|
|
Gastrointestinal disorders
vomiting
|
15.9%
13/82 • Number of events 15 • 2 years
|
|
Gastrointestinal disorders
GI other
|
9.8%
8/82 • Number of events 8 • 2 years
|
|
Gastrointestinal disorders
hemorrhage - nose
|
9.8%
8/82 • Number of events 8 • 2 years
|
|
Skin and subcutaneous tissue disorders
Easy bruising
|
20.7%
17/82 • Number of events 17 • 2 years
|
|
Infections and infestations
pulmonary infection
|
31.7%
26/82 • Number of events 28 • 2 years
|
|
Infections and infestations
sinusitis
|
12.2%
10/82 • Number of events 10 • 2 years
|
|
Blood and lymphatic system disorders
edema
|
50.0%
41/82 • Number of events 41 • 2 years
|
|
Investigations
hyponatremia
|
17.1%
14/82 • Number of events 17 • 2 years
|
|
Investigations
hypokalemia
|
18.3%
15/82 • Number of events 15 • 2 years
|
|
Investigations
increased creatinine
|
48.8%
40/82 • Number of events 40 • 2 years
|
|
Investigations
hyperglycemia
|
52.4%
43/82 • Number of events 45 • 2 years
|
|
Investigations
hyperbilirubinemia
|
8.5%
7/82 • Number of events 7 • 2 years
|
|
Investigations
increased alkylase phosphate
|
29.3%
24/82 • Number of events 31 • 2 years
|
|
Investigations
increased alanine aminotransferase test
|
20.7%
17/82 • Number of events 17 • 2 years
|
|
Investigations
hypoalbuminemia
|
40.2%
33/82 • Number of events 39 • 2 years
|
|
Investigations
hypocalcemia
|
37.8%
31/82 • Number of events 31 • 2 years
|
|
Musculoskeletal and connective tissue disorders
muscle cramps
|
64.6%
53/82 • Number of events 53 • 2 years
|
|
Nervous system disorders
numbness - extremities
|
13.4%
11/82 • Number of events 11 • 2 years
|
|
Musculoskeletal and connective tissue disorders
muscle weakness
|
19.5%
16/82 • Number of events 16 • 2 years
|
|
Musculoskeletal and connective tissue disorders
pain - muscle
|
11.0%
9/82 • Number of events 9 • 2 years
|
|
Nervous system disorders
pain - extremities
|
12.2%
10/82 • Number of events 10 • 2 years
|
|
Psychiatric disorders
mood alteration
|
12.2%
10/82 • Number of events 10 • 2 years
|
|
General disorders
dizziness
|
51.2%
42/82 • Number of events 45 • 2 years
|
|
General disorders
tremors
|
7.3%
6/82 • Number of events 6 • 2 years
|
|
Musculoskeletal and connective tissue disorders
spasms
|
9.8%
8/82 • Number of events 8 • 2 years
|
|
Nervous system disorders
tingling - extremities
|
20.7%
17/82 • Number of events 17 • 2 years
|
|
Eye disorders
visual disturbance
|
13.4%
11/82 • Number of events 11 • 2 years
|
|
General disorders
headache
|
14.6%
12/82 • Number of events 12 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
sore throat
|
15.9%
13/82 • Number of events 13 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
30.5%
25/82 • Number of events 25 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
dyspnea
|
34.1%
28/82 • Number of events 28 • 2 years
|
|
Renal and urinary disorders
dysuria
|
6.1%
5/82 • Number of events 5 • 2 years
|
|
General disorders
flu-like syndrome
|
13.4%
11/82 • Number of events 11 • 2 years
|
|
Immune system disorders
post nasal drip
|
23.2%
19/82 • Number of events 19 • 2 years
|
|
Investigations
elevated blood urea nitrogen blood test
|
13.4%
11/82 • Number of events 11 • 2 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place