Trial Outcomes & Findings for Busulfan, Melphalan, and Bortezomib Before First-Line Stem Cell Transplant in Treating Patients With Multiple Myeloma (NCT NCT01605032)
NCT ID: NCT01605032
Last Updated: 2020-08-20
Results Overview
Number of patients achieved complete response after the treatment regimen
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
19 participants
Primary outcome timeframe
Day 100
Results posted on
2020-08-20
Participant Flow
Participant milestones
| Measure |
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)
CONDITIONING: Patients receive busulfan IV over 3 hours on days -6 to -3, melphalan IV over 20 minutes on day -2, and bortezomib IV over 3-5 seconds on days -6, -3, 1, and 4.
TRANSPLANT: Patients undergo autologous PBSCT on day 0.
Busulfan: Given IV
Melphalan: Given IV
Bortezomib: Given IV
Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT
Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT
|
|---|---|
|
Overall Study
STARTED
|
19
|
|
Overall Study
COMPLETED
|
18
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)
n=19 Participants
CONDITIONING: Patients receive busulfan IV over 3 hours on days -6 to -3, melphalan IV over 20 minutes on day -2, and bortezomib IV over 3-5 seconds on days -6, -3, 1, and 4.
TRANSPLANT: Patients undergo autologous PBSCT on day 0.
Busulfan: Given IV
Melphalan: Given IV
Bortezomib: Given IV
Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT
Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT
|
|---|---|
|
Age, Continuous
|
55 years
n=19 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=19 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=19 Participants
|
|
ISS stage at diagnosis
Stage 1
|
4 participants
n=19 Participants
|
|
ISS stage at diagnosis
Stage 2
|
7 participants
n=19 Participants
|
|
ISS stage at diagnosis
Stage 3
|
5 participants
n=19 Participants
|
|
ISS stage at diagnosis
Stage unkown
|
3 participants
n=19 Participants
|
|
Cytogenetics/FISH at diagnosis
High risk
|
2 participants
n=19 Participants
|
|
Cytogenetics/FISH at diagnosis
Intermediate risk
|
2 participants
n=19 Participants
|
|
Cytogenetics/FISH at diagnosis
Standard risk
|
6 participants
n=19 Participants
|
|
Cytogenetics/FISH at diagnosis
Unknown
|
10 participants
n=19 Participants
|
|
Previous treatment
Bortezomib-containing regimen
|
17 participants
n=19 Participants
|
|
Previous treatment
Immunomodulatory agent-containing regimen
|
7 participants
n=19 Participants
|
|
Response before ASCT
Complete response
|
1 Participants
n=19 Participants
|
|
Response before ASCT
Very Good partial response
|
2 Participants
n=19 Participants
|
|
Response before ASCT
Partial response
|
10 Participants
n=19 Participants
|
|
Response before ASCT
Stable disease
|
5 Participants
n=19 Participants
|
|
Response before ASCT
Progressive disease
|
1 Participants
n=19 Participants
|
PRIMARY outcome
Timeframe: Day 100Number of patients achieved complete response after the treatment regimen
Outcome measures
| Measure |
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)
n=19 Participants
CONDITIONING: Patients receive busulfan IV over 3 hours on days -6 to -3, melphalan IV over 20 minutes on day -2, and bortezomib IV over 3-5 seconds on days -6, -3, 1, and 4.
TRANSPLANT: Patients undergo autologous PBSCT on day 0.
Busulfan: Given IV
Melphalan: Given IV
Bortezomib: Given IV
Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT
Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT
|
|---|---|
|
Rate of Complete Response as Determined by the IMWG Criteria
|
2 Participants
|
SECONDARY outcome
Timeframe: Up to day 100Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.
Outcome measures
| Measure |
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)
n=19 Participants
CONDITIONING: Patients receive busulfan IV over 3 hours on days -6 to -3, melphalan IV over 20 minutes on day -2, and bortezomib IV over 3-5 seconds on days -6, -3, 1, and 4.
TRANSPLANT: Patients undergo autologous PBSCT on day 0.
Busulfan: Given IV
Melphalan: Given IV
Bortezomib: Given IV
Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT
Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT
|
|---|---|
|
Overall Response Rate
Complete response
|
2 Participants
|
|
Overall Response Rate
Partial response
|
17 Participants
|
SECONDARY outcome
Timeframe: Up to day 100Outcome measures
| Measure |
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)
n=19 Participants
CONDITIONING: Patients receive busulfan IV over 3 hours on days -6 to -3, melphalan IV over 20 minutes on day -2, and bortezomib IV over 3-5 seconds on days -6, -3, 1, and 4.
TRANSPLANT: Patients undergo autologous PBSCT on day 0.
Busulfan: Given IV
Melphalan: Given IV
Bortezomib: Given IV
Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT
Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT
|
|---|---|
|
Mortality
|
1 Participants
|
SECONDARY outcome
Timeframe: From start of treatment to disease progression with deaths, up to 2 yearsPopulation: The outcome measure 'Time to Progression' was not collected
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 yearsThe progression free survival was assessed over a period of 2 years
Outcome measures
| Measure |
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)
n=19 Participants
CONDITIONING: Patients receive busulfan IV over 3 hours on days -6 to -3, melphalan IV over 20 minutes on day -2, and bortezomib IV over 3-5 seconds on days -6, -3, 1, and 4.
TRANSPLANT: Patients undergo autologous PBSCT on day 0.
Busulfan: Given IV
Melphalan: Given IV
Bortezomib: Given IV
Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT
Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT
|
|---|---|
|
Progression-free Survival
|
11 Participants
|
SECONDARY outcome
Timeframe: 2 yearsThe overall survival of patients was measured of a period of 2 years.
Outcome measures
| Measure |
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)
n=19 Participants
CONDITIONING: Patients receive busulfan IV over 3 hours on days -6 to -3, melphalan IV over 20 minutes on day -2, and bortezomib IV over 3-5 seconds on days -6, -3, 1, and 4.
TRANSPLANT: Patients undergo autologous PBSCT on day 0.
Busulfan: Given IV
Melphalan: Given IV
Bortezomib: Given IV
Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT
Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT
|
|---|---|
|
Overall Survival
|
18 Participants
|
Adverse Events
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)
Serious events: 1 serious events
Other events: 19 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)
n=19 participants at risk
CONDITIONING: Patients receive busulfan IV over 3 hours on days -6 to -3, melphalan IV over 20 minutes on day -2, and bortezomib IV over 3-5 seconds on days -6, -3, 1, and 4.
TRANSPLANT: Patients undergo autologous PBSCT on day 0.
Busulfan: Given IV
Melphalan: Given IV
Bortezomib: Given IV
Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT
Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Death - 70 days after the autologous stem cell transplant
|
5.3%
1/19 • Up to 2 years
|
Other adverse events
| Measure |
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)
n=19 participants at risk
CONDITIONING: Patients receive busulfan IV over 3 hours on days -6 to -3, melphalan IV over 20 minutes on day -2, and bortezomib IV over 3-5 seconds on days -6, -3, 1, and 4.
TRANSPLANT: Patients undergo autologous PBSCT on day 0.
Busulfan: Given IV
Melphalan: Given IV
Bortezomib: Given IV
Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT
Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT
|
|---|---|
|
General disorders
Electrolyte abnormalities
|
89.5%
17/19 • Up to 2 years
|
|
Gastrointestinal disorders
Oral mucositis
|
94.7%
18/19 • Up to 2 years
|
|
Gastrointestinal disorders
Dysphagia/Odynophagia
|
89.5%
17/19 • Up to 2 years
|
|
Endocrine disorders
Transaminitis
|
68.4%
13/19 • Up to 2 years
|
|
Gastrointestinal disorders
Nausea and/or vomitting
|
94.7%
18/19 • Up to 2 years
|
|
Gastrointestinal disorders
Diarrhea
|
78.9%
15/19 • Up to 2 years
|
|
Gastrointestinal disorders
Abdominal pain
|
15.8%
3/19 • Up to 2 years
|
|
General disorders
Fever without neutropenia
|
21.1%
4/19 • Up to 2 years
|
|
Immune system disorders
Febrile neutropenia
|
73.7%
14/19 • Up to 2 years
|
|
Infections and infestations
Infections
|
42.1%
8/19 • Up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Noninfectious pulmonary toxicity
|
10.5%
2/19 • Up to 2 years
|
Additional Information
Ira Braunschweig, MD
Albert Einstein College of Medicine/Montefiore Medical Center
Phone: 718-920-4057
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place