Trial Outcomes & Findings for Study of High Dose Carfilzomib in Multiple Myeloma Patients Who Have Progressed On Standard Dose Carfilzomib (NCT NCT01775553)
NCT ID: NCT01775553
Last Updated: 2017-06-21
Results Overview
The safety and efficacy of high dose carfilzomib who developed disease progression on the standard dosing and schedule of carfilzomib as measured by number of participants with adverse events
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
13 participants
Primary outcome timeframe
up to 4 years
Results posted on
2017-06-21
Participant Flow
Participant milestones
| Measure |
Carfilzomib
All patients will receive Carfilzomib
Carfilzomib: During Cycle 1, patients will receive either 20 mg/m2 on days 1,2 (if the subject has not received carfilzomib as part another clinical trial within the last 4 weeks) or 56 mg/m2 (if the subject is enrolling in the present study after progression of disease on carfilzomib within the last month - for example subjects enrolled in CMAP compassionate use carfilzomib). Thereafter, all subjects will receive 56 mg/m2 for the remaining doses given Cycle 1 Day 8 onwards. Each cycle is 28 days.
|
|---|---|
|
Overall Study
STARTED
|
13
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
13
|
Reasons for withdrawal
| Measure |
Carfilzomib
All patients will receive Carfilzomib
Carfilzomib: During Cycle 1, patients will receive either 20 mg/m2 on days 1,2 (if the subject has not received carfilzomib as part another clinical trial within the last 4 weeks) or 56 mg/m2 (if the subject is enrolling in the present study after progression of disease on carfilzomib within the last month - for example subjects enrolled in CMAP compassionate use carfilzomib). Thereafter, all subjects will receive 56 mg/m2 for the remaining doses given Cycle 1 Day 8 onwards. Each cycle is 28 days.
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Disease Progression
|
12
|
Baseline Characteristics
Study of High Dose Carfilzomib in Multiple Myeloma Patients Who Have Progressed On Standard Dose Carfilzomib
Baseline characteristics by cohort
| Measure |
Carfilzomib
n=13 Participants
All patients will receive Carfilzomib
Carfilzomib: During Cycle 1, patients will receive either 20 mg/m2 on days 1,2 (if the subject has not received carfilzomib as part another clinical trial within the last 4 weeks) or 56 mg/m2 (if the subject is enrolling in the present study after progression of disease on carfilzomib within the last month - for example subjects enrolled in CMAP compassionate use carfilzomib). Thereafter, all subjects will receive 56 mg/m2 for the remaining doses given Cycle 1 Day 8 onwards. Each cycle is 28 days.
|
|---|---|
|
Age, Continuous
|
62.9 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
|
Age >65 years
|
6 Participants
n=5 Participants
|
|
ECOG Performance Status
0
|
5 Participants
n=5 Participants
|
|
ECOG Performance Status
1
|
5 Participants
n=5 Participants
|
|
ECOG Performance Status
2
|
3 Participants
n=5 Participants
|
|
ISS Staging
Stage 1
|
5 Participants
n=5 Participants
|
|
ISS Staging
Stage 2
|
5 Participants
n=5 Participants
|
|
ISS Staging
Stage 3
|
3 Participants
n=5 Participants
|
|
Immunoglobulin subtype
IgG
|
10 Participants
n=5 Participants
|
|
Immunoglobulin subtype
IgA
|
3 Participants
n=5 Participants
|
|
Immunoglobulin subtype
IgM
|
0 Participants
n=5 Participants
|
|
Immunoglobulin subtype
none
|
0 Participants
n=5 Participants
|
|
Light-chain subtype
K
|
7 Participants
n=5 Participants
|
|
Light-chain subtype
L
|
6 Participants
n=5 Participants
|
|
FISH
Normal
|
7 Participants
n=5 Participants
|
|
FISH
Any abnormality
|
6 Participants
n=5 Participants
|
|
Prior therapies since diagnosis
|
7 years
n=5 Participants
|
|
Number of prior regimens
|
5 prior regimens
n=5 Participants
|
|
Prior therapy
Dexamethasone : Exposed
|
13 participants
n=5 Participants
|
|
Prior therapy
Dexamethasone : Refractory
|
13 participants
n=5 Participants
|
|
Prior therapy
Thalidomide : Exposed
|
7 participants
n=5 Participants
|
|
Prior therapy
Thalidomide : Refractory
|
1 participants
n=5 Participants
|
|
Prior therapy
Lenalidomide : Exposed
|
13 participants
n=5 Participants
|
|
Prior therapy
Lenalidomide : Refractory
|
9 participants
n=5 Participants
|
|
Prior therapy
Pomalidomide : Exposed
|
8 participants
n=5 Participants
|
|
Prior therapy
Pomalidomide : Refractory
|
8 participants
n=5 Participants
|
|
Prior therapy
Bortezomib : Exposed
|
13 participants
n=5 Participants
|
|
Prior therapy
Bortezomib : Refractory
|
9 participants
n=5 Participants
|
|
Prior Autologous Stem Cell Transplant
|
7 Participants
n=5 Participants
|
|
Carfilzomib Regimen Combination
Carfilzomib Regimen Combination
|
13 Participants
n=5 Participants
|
|
Carfilzomib Regimen Combination
Weekly 40 mg DEX
|
11 Participants
n=5 Participants
|
|
Carfilzomib Regimen Combination
Pomalidomide, DEX
|
2 Participants
n=5 Participants
|
|
Carfilzomib Regimen Combination
Cyclophosphamide, DEX
|
2 Participants
n=5 Participants
|
|
Carfilzomib Regimen Combination
Ibrutinib, DEX
|
1 Participants
n=5 Participants
|
|
Carfilzomib Regimen Combination
Bendamustine
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: up to 4 yearsThe safety and efficacy of high dose carfilzomib who developed disease progression on the standard dosing and schedule of carfilzomib as measured by number of participants with adverse events
Outcome measures
| Measure |
Carfilzomib
n=13 Participants
All patients will receive Carfilzomib
Carfilzomib: During Cycle 1, patients will receive either 20 mg/m2 on days 1,2 (if the subject has not received carfilzomib as part another clinical trial within the last 4 weeks) or 56 mg/m2 (if the subject is enrolling in the present study after progression of disease on carfilzomib within the last month - for example subjects enrolled in CMAP compassionate use carfilzomib). Thereafter, all subjects will receive 56 mg/m2 for the remaining doses given Cycle 1 Day 8 onwards. Each cycle is 28 days.
|
|---|---|
|
Safety and Efficacy of High Dose Carfilzomib
Serious Adverse Events
|
3 Participants
|
|
Safety and Efficacy of High Dose Carfilzomib
Other than Serious Adverse Events
|
13 Participants
|
SECONDARY outcome
Timeframe: up to 4 yearsOutcome measures
| Measure |
Carfilzomib
n=12 Participants
All patients will receive Carfilzomib
Carfilzomib: During Cycle 1, patients will receive either 20 mg/m2 on days 1,2 (if the subject has not received carfilzomib as part another clinical trial within the last 4 weeks) or 56 mg/m2 (if the subject is enrolling in the present study after progression of disease on carfilzomib within the last month - for example subjects enrolled in CMAP compassionate use carfilzomib). Thereafter, all subjects will receive 56 mg/m2 for the remaining doses given Cycle 1 Day 8 onwards. Each cycle is 28 days.
|
|---|---|
|
Progression Free Survival (PFS)
|
3.8 months
Interval 1.0 to 12.0
|
SECONDARY outcome
Timeframe: up to 4 yearsOverall Response Rate defined in categories
Outcome measures
| Measure |
Carfilzomib
n=12 Participants
All patients will receive Carfilzomib
Carfilzomib: During Cycle 1, patients will receive either 20 mg/m2 on days 1,2 (if the subject has not received carfilzomib as part another clinical trial within the last 4 weeks) or 56 mg/m2 (if the subject is enrolling in the present study after progression of disease on carfilzomib within the last month - for example subjects enrolled in CMAP compassionate use carfilzomib). Thereafter, all subjects will receive 56 mg/m2 for the remaining doses given Cycle 1 Day 8 onwards. Each cycle is 28 days.
|
|---|---|
|
Overall Response Rate (ORR)
Complete Response
|
0 Participants
|
|
Overall Response Rate (ORR)
Very Good Partial Response
|
1 Participants
|
|
Overall Response Rate (ORR)
Partial Response
|
4 Participants
|
|
Overall Response Rate (ORR)
Minor Response
|
2 Participants
|
|
Overall Response Rate (ORR)
Stable Disease
|
4 Participants
|
SECONDARY outcome
Timeframe: up to 4 yearsOutcome measures
| Measure |
Carfilzomib
n=12 Participants
All patients will receive Carfilzomib
Carfilzomib: During Cycle 1, patients will receive either 20 mg/m2 on days 1,2 (if the subject has not received carfilzomib as part another clinical trial within the last 4 weeks) or 56 mg/m2 (if the subject is enrolling in the present study after progression of disease on carfilzomib within the last month - for example subjects enrolled in CMAP compassionate use carfilzomib). Thereafter, all subjects will receive 56 mg/m2 for the remaining doses given Cycle 1 Day 8 onwards. Each cycle is 28 days.
|
|---|---|
|
Duration of Response to High Dose Carfilzomib
|
9 months
Interval 1.0 to 13.0
|
SECONDARY outcome
Timeframe: up to 4 yearsPopulation: data not collected
The markers of ER stress signaling (both apoptotic and prosurvival) in MM cells from patients in this study and to determine if the balance of apoptotic versus prosurvival signaling changes upon recapture of response with carfilzomib dose escalation relative to the time of study entry.
Outcome measures
Outcome data not reported
Adverse Events
Carfilzomib
Serious events: 3 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Carfilzomib
n=13 participants at risk
All patients will receive Carfilzomib
Carfilzomib: During Cycle 1, patients will receive either 20 mg/m2 on days 1,2 (if the subject has not received carfilzomib as part another clinical trial within the last 4 weeks) or 56 mg/m2 (if the subject is enrolling in the present study after progression of disease on carfilzomib within the last month - for example subjects enrolled in CMAP compassionate use carfilzomib). Thereafter, all subjects will receive 56 mg/m2 for the remaining doses given Cycle 1 Day 8 onwards. Each cycle is 28 days.
|
|---|---|
|
Blood and lymphatic system disorders
Acute Pulmonary Embolism
|
7.7%
1/13
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
7.7%
1/13
|
|
Musculoskeletal and connective tissue disorders
Leg pain
|
7.7%
1/13
|
Other adverse events
| Measure |
Carfilzomib
n=13 participants at risk
All patients will receive Carfilzomib
Carfilzomib: During Cycle 1, patients will receive either 20 mg/m2 on days 1,2 (if the subject has not received carfilzomib as part another clinical trial within the last 4 weeks) or 56 mg/m2 (if the subject is enrolling in the present study after progression of disease on carfilzomib within the last month - for example subjects enrolled in CMAP compassionate use carfilzomib). Thereafter, all subjects will receive 56 mg/m2 for the remaining doses given Cycle 1 Day 8 onwards. Each cycle is 28 days.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Acute dyspnea
|
7.7%
1/13 • Number of events 1
|
|
Renal and urinary disorders
Acute kidney injury
|
7.7%
1/13 • Number of events 1
|
|
Renal and urinary disorders
Acute renal failure
|
7.7%
1/13 • Number of events 1
|
|
Metabolism and nutrition disorders
alanine aminotransferase increased
|
7.7%
1/13 • Number of events 1
|
|
Metabolism and nutrition disorders
alkaline phosphatase increased
|
7.7%
1/13 • Number of events 2
|
|
Blood and lymphatic system disorders
anemia
|
61.5%
8/13 • Number of events 33
|
|
Metabolism and nutrition disorders
ankle swelling
|
7.7%
1/13 • Number of events 1
|
|
General disorders
anorexia
|
7.7%
1/13 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
anterolisthesis of L5 over S1
|
7.7%
1/13 • Number of events 1
|
|
Metabolism and nutrition disorders
asparate aminotransferase increased
|
23.1%
3/13 • Number of events 3
|
|
Musculoskeletal and connective tissue disorders
bilateral lower extremity leg pain
|
7.7%
1/13 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
back pain
|
7.7%
1/13 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
bilateral hip pain
|
7.7%
1/13 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
bone pain
|
7.7%
1/13 • Number of events 1
|
|
Infections and infestations
cellulitis
|
7.7%
1/13 • Number of events 1
|
|
General disorders
chest heaviness
|
7.7%
1/13 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
chest pain
|
15.4%
2/13 • Number of events 4
|
|
Gastrointestinal disorders
constipation
|
30.8%
4/13 • Number of events 4
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
23.1%
3/13 • Number of events 3
|
|
Renal and urinary disorders
creatinine elevated
|
30.8%
4/13 • Number of events 4
|
|
Renal and urinary disorders
decreased creatinine clearance
|
7.7%
1/13 • Number of events 1
|
|
General disorders
diaphoresis
|
7.7%
1/13 • Number of events 1
|
|
General disorders
dizziness
|
7.7%
1/13 • Number of events 1
|
|
Nervous system disorders
drift when walking
|
7.7%
1/13 • Number of events 1
|
|
Gastrointestinal disorders
dyspepsia
|
7.7%
1/13 • Number of events 1
|
|
Cardiac disorders
ejection fraction drop
|
15.4%
2/13 • Number of events 2
|
|
Metabolism and nutrition disorders
Elevated bilirubin
|
7.7%
1/13 • Number of events 1
|
|
Metabolism and nutrition disorders
elevated GGT
|
7.7%
1/13 • Number of events 1
|
|
General disorders
epigastric pain
|
7.7%
1/13 • Number of events 1
|
|
General disorders
Fatigue
|
23.1%
3/13 • Number of events 3
|
|
General disorders
Fever
|
23.1%
3/13 • Number of events 4
|
|
Gastrointestinal disorders
gastroesophageal reflux disease
|
7.7%
1/13 • Number of events 1
|
|
Nervous system disorders
hallucinations
|
7.7%
1/13 • Number of events 1
|
|
Nervous system disorders
Headache
|
46.2%
6/13 • Number of events 19
|
|
General disorders
high blood pressure
|
7.7%
1/13 • Number of events 1
|
|
Metabolism and nutrition disorders
hypercalcemia
|
46.2%
6/13 • Number of events 6
|
|
Metabolism and nutrition disorders
hyperglycemia
|
7.7%
1/13 • Number of events 1
|
|
Metabolism and nutrition disorders
hyperkalemia
|
15.4%
2/13 • Number of events 3
|
|
Metabolism and nutrition disorders
hypernatremia
|
7.7%
1/13 • Number of events 1
|
|
Metabolism and nutrition disorders
hyperphostphatemia
|
23.1%
3/13 • Number of events 4
|
|
Cardiac disorders
hypertension
|
53.8%
7/13 • Number of events 22
|
|
Cardiac disorders
hypertension chronic and intermittent fluctuation
|
7.7%
1/13 • Number of events 1
|
|
Metabolism and nutrition disorders
hypokalemia
|
7.7%
1/13 • Number of events 1
|
|
Cardiac disorders
hypotension
|
7.7%
1/13 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
intermittent sternal pain
|
7.7%
1/13 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
joint pain
|
7.7%
1/13 • Number of events 1
|
|
Blood and lymphatic system disorders
lower extremity edema
|
7.7%
1/13 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
left rib pain
|
7.7%
1/13 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
left thigh pain
|
7.7%
1/13 • Number of events 1
|
|
Blood and lymphatic system disorders
leukocyte count decreased
|
23.1%
3/13 • Number of events 3
|
|
Blood and lymphatic system disorders
lymphocyte count decreased
|
23.1%
3/13 • Number of events 18
|
|
Blood and lymphatic system disorders
lymphopenia
|
7.7%
1/13 • Number of events 1
|
|
General disorders
malaise
|
7.7%
1/13 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
minor cramps
|
7.7%
1/13 • Number of events 1
|
|
General disorders
nausea
|
15.4%
2/13 • Number of events 5
|
|
Nervous system disorders
neuropathy
|
7.7%
1/13 • Number of events 1
|
|
Blood and lymphatic system disorders
neutrophil count decreased
|
30.8%
4/13 • Number of events 38
|
|
Respiratory, thoracic and mediastinal disorders
non productive cough
|
7.7%
1/13 • Number of events 1
|
|
General disorders
pain
|
23.1%
3/13 • Number of events 3
|
|
General disorders
left mandible pain
|
7.7%
1/13 • Number of events 1
|
|
Blood and lymphatic system disorders
platelet count decreased
|
53.8%
7/13 • Number of events 29
|
|
Respiratory, thoracic and mediastinal disorders
pneumonitis
|
7.7%
1/13 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
pruritic reddish hands with pain on hands wrist and arms
|
7.7%
1/13 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
right iliac bone pain
|
7.7%
1/13 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
right rib pain
|
15.4%
2/13 • Number of events 3
|
|
Nervous system disorders
seizure
|
7.7%
1/13 • Number of events 1
|
|
General disorders
shakiness/weakness
|
7.7%
1/13 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
shortness of breath
|
7.7%
1/13 • Number of events 2
|
|
General disorders
sinusitis
|
7.7%
1/13 • Number of events 1
|
|
Blood and lymphatic system disorders
swelling in lower ankles
|
7.7%
1/13 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
upper respiratory infection
|
23.1%
3/13 • Number of events 3
|
|
Reproductive system and breast disorders
vaginal pain
|
7.7%
1/13 • Number of events 1
|
|
General disorders
vomiting
|
7.7%
1/13 • Number of events 3
|
|
General disorders
weight loss
|
7.7%
1/13 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
wheezing
|
7.7%
1/13 • Number of events 1
|
|
Cardiac disorders
white blood cell decrease
|
7.7%
1/13 • Number of events 1
|
|
Infections and infestations
yeast infection
|
7.7%
1/13 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place