Trial Outcomes & Findings for Carfilzomib and Stem Cell Transplant for Plasma Cell Myeloma (NCT NCT01658904)
NCT ID: NCT01658904
Last Updated: 2016-06-01
Results Overview
Engraftment failure is defined as the failure to achieve neutrophil engraftment by day 21; defined from day 0, day of autologous hematopoietic cell transplantation (AHCT), as the first of three consecutive days on which the patient's absolute neutrophil count is greater than 0.5x10(9)/l following the nadir. Transplant related mortality is defined as any subject who dies in the first 100 days post-AHCT of any non-relapse related cause.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
3 participants
Primary outcome timeframe
up to day 100
Results posted on
2016-06-01
Participant Flow
The study did not progress to the phase II portion. The study was closed prematurely because the investigator left the National Institutes of Health.
Participant milestones
| Measure |
Cohort 1- CFZ 20 mg/m^2 (Day 1,2)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
Cohort 2- CFZ 20 mg/m^2 (Day 1,2,8,9)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
Cohort 3-CFZ 20 mg/m^2 (Day1,2,8,9/AHCT)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
|---|---|---|---|
|
Overall Study
STARTED
|
3
|
0
|
0
|
|
Overall Study
COMPLETED
|
1
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
0
|
0
|
Reasons for withdrawal
| Measure |
Cohort 1- CFZ 20 mg/m^2 (Day 1,2)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
Cohort 2- CFZ 20 mg/m^2 (Day 1,2,8,9)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
Cohort 3-CFZ 20 mg/m^2 (Day1,2,8,9/AHCT)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
|---|---|---|---|
|
Overall Study
Principal Investigator discretion
|
2
|
0
|
0
|
Baseline Characteristics
Carfilzomib and Stem Cell Transplant for Plasma Cell Myeloma
Baseline characteristics by cohort
| Measure |
Cohort 1- CFZ 20 mg/m^2 (Day 1,2)
n=3 Participants
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
Cohort 2- CFZ 20 mg/m^2 (Day 1,2,8,9)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
Cohort 3-CFZ 20 mg/m^2 (Day1,2,8,9/AHCT)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
Total
n=3 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 participants
n=5 Participants
|
—
|
—
|
0 participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 participants
n=5 Participants
|
—
|
—
|
3 participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 participants
n=5 Participants
|
—
|
—
|
0 participants
n=4 Participants
|
|
Age, Continuous
|
49.6 years
STANDARD_DEVIATION 10.39 • n=5 Participants
|
—
|
—
|
49.6 years
STANDARD_DEVIATION 10.39 • n=4 Participants
|
|
Gender
Female
|
1 participants
n=5 Participants
|
—
|
—
|
1 participants
n=4 Participants
|
|
Gender
Male
|
2 participants
n=5 Participants
|
—
|
—
|
2 participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 participants
n=5 Participants
|
—
|
—
|
0 participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 participants
n=5 Participants
|
—
|
—
|
3 participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 participants
n=5 Participants
|
—
|
—
|
0 participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 participants
n=5 Participants
|
—
|
—
|
0 participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 participants
n=5 Participants
|
—
|
—
|
0 participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 participants
n=5 Participants
|
—
|
—
|
0 participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 participants
n=5 Participants
|
—
|
—
|
1 participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
2 participants
n=5 Participants
|
—
|
—
|
2 participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 participants
n=5 Participants
|
—
|
—
|
0 participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 participants
n=5 Participants
|
—
|
—
|
0 participants
n=4 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
—
|
—
|
3 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: up to day 100Engraftment failure is defined as the failure to achieve neutrophil engraftment by day 21; defined from day 0, day of autologous hematopoietic cell transplantation (AHCT), as the first of three consecutive days on which the patient's absolute neutrophil count is greater than 0.5x10(9)/l following the nadir. Transplant related mortality is defined as any subject who dies in the first 100 days post-AHCT of any non-relapse related cause.
Outcome measures
| Measure |
Cohort 1- CFZ 20 mg/m^2 (Day 1,2)
n=1 Participants
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
Cohort 2- CFZ 20 mg/m^2 (Day 1,2,8,9)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
Cohort 3-CFZ 20 mg/m^2 (Day1,2,8,9/AHCT)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
|---|---|---|---|
|
Engraftment Failure Transplant Related Mortality
|
0 participants
|
—
|
—
|
PRIMARY outcome
Timeframe: 8 months and 15 daysPopulation: Analysis of dose limiting toxicities (DLTs) was planned but not performed due to study termination; this Outcome Measure captures any events that occurred.
Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module.
Outcome measures
| Measure |
Cohort 1- CFZ 20 mg/m^2 (Day 1,2)
n=3 Participants
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
Cohort 2- CFZ 20 mg/m^2 (Day 1,2,8,9)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
Cohort 3-CFZ 20 mg/m^2 (Day1,2,8,9/AHCT)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
|---|---|---|---|
|
Number of Participants With Adverse Events
|
1 participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Post-AHCT following CFZ therapyPopulation: This outcome measure was not done because the study was closed prematurely because the investigator left the National Institutes of Health.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 100 post-AHCTPopulation: This outcome measure was not done because the study was closed prematurely because the investigator left the National Institutes of Health.
Outcome measures
Outcome data not reported
Adverse Events
Cohort 1- CFZ 20 mg/m^2 (Day 1,2)
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Cohort 2- CFZ 20 mg/m^2 (Day 1,2,8,9)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Cohort 3-CFZ 20 mg/m^2 (Day1,2,8,9/AHCT)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Cohort 1- CFZ 20 mg/m^2 (Day 1,2)
n=3 participants at risk
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
Cohort 2- CFZ 20 mg/m^2 (Day 1,2,8,9)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
Cohort 3-CFZ 20 mg/m^2 (Day1,2,8,9/AHCT)
Phase I/II study on the backbone of high-dose melphalan on day -2 pre-autologous hematopoietic cell transplantation (AHCT)
•Addition of an increasing number of doses of Carfilzomib (CFZ) in the early post-AHCT period introduced in a step-wise fashion in 3 successive cohorts of 3 to 15 subjects:
Cohort 1: add CFZ 20 mg/m\^2 intravenous (IV) on days +1, +2
Cohort 2 : add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9
Cohort 3: add CFZ 20 mg/m\^2 IV on days: +1, +2, +8, +9 and add an early post-AHCT consolidation following engraftment: CFZ 20 mg/m\^2 IV given on days 42-43 then CFZ 56 mg/m\^2 IV given on days 49-50, 56-57, then on days 70.
|
|---|---|---|---|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
33.3%
1/3 • Number of events 1
|
—
0/0
|
—
0/0
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
33.3%
1/3 • Number of events 1
|
—
0/0
|
—
0/0
|
|
Metabolism and nutrition disorders
Hypokalemia
|
33.3%
1/3 • Number of events 1
|
—
0/0
|
—
0/0
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
33.3%
1/3 • Number of events 3
|
—
0/0
|
—
0/0
|
|
Investigations
Neutrophil count decreased
|
33.3%
1/3 • Number of events 2
|
—
0/0
|
—
0/0
|
|
Investigations
Platelet count decreased
|
33.3%
1/3 • Number of events 2
|
—
0/0
|
—
0/0
|
|
Investigations
White blood cell decreased
|
33.3%
1/3 • Number of events 1
|
—
0/0
|
—
0/0
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place