Trial Outcomes & Findings for Busulfan, Cyclophosphamide, and Autologous Stem Cell Transplant in Treating Patients With Multiple Myeloma (NCT NCT00941720)
NCT ID: NCT00941720
Last Updated: 2020-07-24
Results Overview
Number of participants without progressive disease at the end of the study period, using the definitions for complete response, partial response and progressive disease from the International Myeloma Working Group .
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
71 participants
Primary outcome timeframe
at 6 months
Results posted on
2020-07-24
Participant Flow
Participant milestones
| Measure |
Busulfan Treatment
busulfan: IV busulfan 0.8 mg/kg every 6 hours x 16 doses
cyclophosphamide: IV cyclophosphamide 60 mg/kg over 4 hours x 2 days
autologous hematopoietic stem cell transplantation: infusion of autologous hematopoietic stem cells of at least 2.0 x 106 CD34+ cells/kg on day 0
|
|---|---|
|
Overall Study
STARTED
|
71
|
|
Overall Study
COMPLETED
|
57
|
|
Overall Study
NOT COMPLETED
|
14
|
Reasons for withdrawal
| Measure |
Busulfan Treatment
busulfan: IV busulfan 0.8 mg/kg every 6 hours x 16 doses
cyclophosphamide: IV cyclophosphamide 60 mg/kg over 4 hours x 2 days
autologous hematopoietic stem cell transplantation: infusion of autologous hematopoietic stem cells of at least 2.0 x 106 CD34+ cells/kg on day 0
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Insurance denied participation
|
11
|
|
Overall Study
ineligible
|
2
|
Baseline Characteristics
Busulfan, Cyclophosphamide, and Autologous Stem Cell Transplant in Treating Patients With Multiple Myeloma
Baseline characteristics by cohort
| Measure |
Busulfan Treatment
n=57 Participants
busulfan: IV busulfan 0.8 mg/kg every 6 hours x 16 doses
cyclophosphamide: IV cyclophosphamide 60 mg/kg over 4 hours x 2 days
autologous hematopoietic stem cell transplantation: infusion of autologous hematopoietic stem cells of at least 2.0 x 106 CD34+ cells/kg on day 0
|
|---|---|
|
Age, Continuous
|
58 years
STANDARD_DEVIATION 8 • n=5 Participants
|
|
Sex: Female, Male
Female
|
24 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
33 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
47 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
57 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
57 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: at 6 monthsNumber of participants without progressive disease at the end of the study period, using the definitions for complete response, partial response and progressive disease from the International Myeloma Working Group .
Outcome measures
| Measure |
Busulfan Treatment
n=57 Participants
busulfan: IV busulfan 0.8 mg/kg every 6 hours x 16 doses
cyclophosphamide: IV cyclophosphamide 60 mg/kg over 4 hours x 2 days
autologous hematopoietic stem cell transplantation: infusion of autologous hematopoietic stem cells of at least 2.0 x 106 CD34+ cells/kg on day 0
|
|---|---|
|
Relapse-free Survival
|
48 participants
|
PRIMARY outcome
Timeframe: at 6 monthsNumber of patients alive at the end of the study period
Outcome measures
| Measure |
Busulfan Treatment
n=57 Participants
busulfan: IV busulfan 0.8 mg/kg every 6 hours x 16 doses
cyclophosphamide: IV cyclophosphamide 60 mg/kg over 4 hours x 2 days
autologous hematopoietic stem cell transplantation: infusion of autologous hematopoietic stem cells of at least 2.0 x 106 CD34+ cells/kg on day 0
|
|---|---|
|
Overall Survival
|
53 participants
|
SECONDARY outcome
Timeframe: At 6 monthsToxicity criteria will be assessed and graded according to the National Cancer Institute (NCI) Common Terminology Criteria (CTC) v3.0. Estimated using exact 95% binomial confidence intervals.
Outcome measures
| Measure |
Busulfan Treatment
n=57 Participants
busulfan: IV busulfan 0.8 mg/kg every 6 hours x 16 doses
cyclophosphamide: IV cyclophosphamide 60 mg/kg over 4 hours x 2 days
autologous hematopoietic stem cell transplantation: infusion of autologous hematopoietic stem cells of at least 2.0 x 106 CD34+ cells/kg on day 0
|
|---|---|
|
Pulmonary Toxicity
|
3.5 percentage of participants
Interval 0.4 to 12.1
|
Adverse Events
Busulfan Treatment
Serious events: 12 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Busulfan Treatment
n=57 participants at risk
busulfan: IV busulfan 0.8 mg/kg every 6 hours x 16 doses
cyclophosphamide: IV cyclophosphamide 60 mg/kg over 4 hours x 2 days
autologous hematopoietic stem cell transplantation: infusion of autologous hematopoietic stem cells of at least 2.0 x 106 CD34+ cells/kg on day 0
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Adult Respiratory Distress Syndrome
|
1.8%
1/57 • Number of events 1 • 100 days after off treatment
|
|
Immune system disorders
Allergic reaction - Angioedemia due to ACE Inhibitors
|
1.8%
1/57 • Number of events 1 • 100 days after off treatment
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders - other (Relapsed disease)
|
1.8%
1/57 • Number of events 2 • 100 days after off treatment
|
|
General disorders
Death NOS
|
8.8%
5/57 • Number of events 5 • 100 days after off treatment
|
|
Respiratory, thoracic and mediastinal disorders
Dyspenia
|
1.8%
1/57 • Number of events 1 • 100 days after off treatment
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications - Other (patient fell and hit head. Never recovered)
|
1.8%
1/57 • Number of events 2 • 100 days after off treatment
|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal edema
|
1.8%
1/57 • Number of events 2 • 100 days after off treatment
|
|
Infections and infestations
Lung Infection
|
1.8%
1/57 • Number of events 1 • 100 days after off treatment
|
Other adverse events
| Measure |
Busulfan Treatment
n=57 participants at risk
busulfan: IV busulfan 0.8 mg/kg every 6 hours x 16 doses
cyclophosphamide: IV cyclophosphamide 60 mg/kg over 4 hours x 2 days
autologous hematopoietic stem cell transplantation: infusion of autologous hematopoietic stem cells of at least 2.0 x 106 CD34+ cells/kg on day 0
|
|---|---|
|
Infections and infestations
Catheter related infection
|
1.8%
1/57 • Number of events 1 • 100 days after off treatment
|
|
Gastrointestinal disorders
Colitis - diverticulitis
|
1.8%
1/57 • Number of events 1 • 100 days after off treatment
|
|
Respiratory, thoracic and mediastinal disorders
Dyspenia
|
1.8%
1/57 • Number of events 1 • 100 days after off treatment
|
|
General disorders
Fever
|
1.8%
1/57 • Number of events 3 • 100 days after off treatment
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Clostridium difficile in urine and diarrhea
|
1.8%
1/57 • Number of events 1 • 100 days after off treatment
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place