Trial Outcomes & Findings for Melphalan, Bortezomib, and Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis (NCT NCT00790647)
NCT ID: NCT00790647
Last Updated: 2017-02-06
Results Overview
complete and partial hematologic response defined as: Complete response: absence of detectable monoclonal protein in serum and urine, and bone marrow biopsy \<5% plasma cells with no clonal predominance of kappa or lambda isotype. Partial response: any one of the following 1. For patients with detectable and quantifiable marrow plasmacytosis, a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells. 2. For patients with a detectable monoclonal peak on serum protein electropheresis or urine protein electropheresis, a reduction in the peak height of 50% or more. 3. For patients with quantifiable urinary kappa or lambda chain concentration, a reduction in daily light chain excretion (concentration x 24-hr urine volume).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
one year
Results posted on
2017-02-06
Participant Flow
Participant milestones
| Measure |
SCT With Bortezomib and Melphalan
Mobilization with Filgrastim Stem Cell Collection (SCC) Bortezomib Melphalan Stem Cell infusion
filgrastim: 16 mcg/kg daily beginning 3 days before SCC through day before final SCC
bortezomib: 1.0 mg/m2/dose D -6, D-3, D +1, D + 4
melphalan: 100 mg/m2/dose D -2, D -1
Stem Cell Infusion: infusion of previously collected autologous stem cells
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
SCT With Bortezomib and Melphalan
Mobilization with Filgrastim Stem Cell Collection (SCC) Bortezomib Melphalan Stem Cell infusion
filgrastim: 16 mcg/kg daily beginning 3 days before SCC through day before final SCC
bortezomib: 1.0 mg/m2/dose D -6, D-3, D +1, D + 4
melphalan: 100 mg/m2/dose D -2, D -1
Stem Cell Infusion: infusion of previously collected autologous stem cells
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
Baseline Characteristics
Melphalan, Bortezomib, and Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis
Baseline characteristics by cohort
| Measure |
SCT With Bortezomib and Melphalan
n=10 Participants
Mobilization with Filgrastim Stem Cell Collection (SCC) Bortezomib Melphalan Stem Cell infusion
filgrastim: 16 mcg/kg daily beginning 3 days before SCC through day before final SCC
bortezomib: 1.0 mg/m2/dose D -6, D-3, D +1, D + 4
melphalan: 100 mg/m2/dose D -2, D -1
Stem Cell Infusion: infusion of previously collected autologous stem cells
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
10 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Gender
Female
|
5 Participants
n=5 Participants
|
|
Gender
Male
|
5 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: one yearcomplete and partial hematologic response defined as: Complete response: absence of detectable monoclonal protein in serum and urine, and bone marrow biopsy \<5% plasma cells with no clonal predominance of kappa or lambda isotype. Partial response: any one of the following 1. For patients with detectable and quantifiable marrow plasmacytosis, a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells. 2. For patients with a detectable monoclonal peak on serum protein electropheresis or urine protein electropheresis, a reduction in the peak height of 50% or more. 3. For patients with quantifiable urinary kappa or lambda chain concentration, a reduction in daily light chain excretion (concentration x 24-hr urine volume).
Outcome measures
| Measure |
Stem Cell Transplant With Bortezomib and Melphalan
n=9 Participants
Mobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
filgrastim: 16 mcg/kg daily beginning 3 days before Stem Cell Collectionthrough day before final Stem Cell Collection
bortezomib: 1.0 mg/m2/dose D -6, D-3, D +1, D + 4
melphalan: 100 mg/m2/dose D -2, D -1
Stem Cell Infusion: infusion of previously collected autologous stem cells
|
|---|---|
|
Number of Participants With Hematologic Response
|
6 participants
|
SECONDARY outcome
Timeframe: 100 Days from transplant dateOutcome measures
| Measure |
Stem Cell Transplant With Bortezomib and Melphalan
n=9 Participants
Mobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
filgrastim: 16 mcg/kg daily beginning 3 days before Stem Cell Collectionthrough day before final Stem Cell Collection
bortezomib: 1.0 mg/m2/dose D -6, D-3, D +1, D + 4
melphalan: 100 mg/m2/dose D -2, D -1
Stem Cell Infusion: infusion of previously collected autologous stem cells
|
|---|---|
|
Number of Participants Surviving at 100 Days From Transplant
|
9 participants
|
SECONDARY outcome
Timeframe: one year from transplantOutcome measures
| Measure |
Stem Cell Transplant With Bortezomib and Melphalan
n=9 Participants
Mobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
filgrastim: 16 mcg/kg daily beginning 3 days before Stem Cell Collectionthrough day before final Stem Cell Collection
bortezomib: 1.0 mg/m2/dose D -6, D-3, D +1, D + 4
melphalan: 100 mg/m2/dose D -2, D -1
Stem Cell Infusion: infusion of previously collected autologous stem cells
|
|---|---|
|
Number of Participants Surviving at 1 Year
|
9 participants
|
SECONDARY outcome
Timeframe: 2 years from transplantOutcome measures
| Measure |
Stem Cell Transplant With Bortezomib and Melphalan
n=9 Participants
Mobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
filgrastim: 16 mcg/kg daily beginning 3 days before Stem Cell Collectionthrough day before final Stem Cell Collection
bortezomib: 1.0 mg/m2/dose D -6, D-3, D +1, D + 4
melphalan: 100 mg/m2/dose D -2, D -1
Stem Cell Infusion: infusion of previously collected autologous stem cells
|
|---|---|
|
Number of Participants Surviving at 2 Years
|
9 Participants
|
Adverse Events
SCT With Bortezomib and Melphalan
Serious events: 9 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
SCT With Bortezomib and Melphalan
n=9 participants at risk
Mobilization with Filgrastim Stem Cell Collection (SCC) Bortezomib Melphalan Stem Cell infusion
filgrastim: 16 mcg/kg daily beginning 3 days before SCC through day before final SCC
bortezomib: 1.0 mg/m2/dose D -6, D-3, D +1, D + 4
melphalan: 100 mg/m2/dose D -2, D -1
Stem Cell Infusion: infusion of previously collected autologous stem cells
|
|---|---|
|
Blood and lymphatic system disorders
pancytopenia
|
100.0%
9/9 • Number of events 9 • 1 year
|
|
Blood and lymphatic system disorders
coagulation
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
General disorders
fatigue
|
22.2%
2/9 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
nausea
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
vomit
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
diarrhea
|
33.3%
3/9 • Number of events 3 • 1 year
|
|
Gastrointestinal disorders
heartburn
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Cardiac disorders
elevated troponin I
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
General disorders
edema
|
22.2%
2/9 • Number of events 2 • 1 year
|
|
General disorders
deconditioning
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Infection
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Investigations
increased alkylase phosphate
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Investigations
creatinine increased
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Investigations
decreased phosphate
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Investigations
hypocalcemia
|
33.3%
3/9 • Number of events 3 • 1 year
|
|
Investigations
hypoalbuminemia
|
22.2%
2/9 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
GI bleed
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Blood and lymphatic system disorders
febrile neutropenia
|
22.2%
2/9 • Number of events 2 • 1 year
|
Other adverse events
| Measure |
SCT With Bortezomib and Melphalan
n=9 participants at risk
Mobilization with Filgrastim Stem Cell Collection (SCC) Bortezomib Melphalan Stem Cell infusion
filgrastim: 16 mcg/kg daily beginning 3 days before SCC through day before final SCC
bortezomib: 1.0 mg/m2/dose D -6, D-3, D +1, D + 4
melphalan: 100 mg/m2/dose D -2, D -1
Stem Cell Infusion: infusion of previously collected autologous stem cells
|
|---|---|
|
Gastrointestinal disorders
nausea
|
33.3%
3/9 • Number of events 5 • 1 year
|
|
Gastrointestinal disorders
diarrhea
|
44.4%
4/9 • Number of events 6 • 1 year
|
|
General disorders
fatigue
|
77.8%
7/9 • Number of events 7 • 1 year
|
|
Investigations
creatinine increased
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
General disorders
deconditioning
|
66.7%
6/9 • Number of events 6 • 1 year
|
|
Investigations
increased alkylase phosphate
|
88.9%
8/9 • Number of events 8 • 1 year
|
|
Investigations
hypocalcemia
|
33.3%
3/9 • Number of events 4 • 1 year
|
|
Investigations
hypoalbuminemia
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
General disorders
hiccups
|
44.4%
4/9 • Number of events 5 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
dyspnea
|
22.2%
2/9 • Number of events 2 • 1 year
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place