Trial Outcomes & Findings for A Randomized Trial to Compare Busulfan + Melphalan 140 mg/m2 With Melphalan 200 mg/m2 as Preparative Regimen for Autologous Hematopoietic Stem Cell Transplantation for Multiple Myeloma (NCT NCT01413178)
NCT ID: NCT01413178
Last Updated: 2020-04-21
Results Overview
Participants that are still alive and without Multiple Myeloma 3 years after Stem cell Transplantation.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
205 participants
Primary outcome timeframe
3 years after transplant
Results posted on
2020-04-21
Participant Flow
All participants were registered in MD Anderson Cancer.
Participant milestones
| Measure |
Busulfan + Melphalan
Busulfan with a target daily area under the curve (AUC) of 5000 with Melphalan 70 mg/m2 followed by a stem cell transplant
|
Melphalan
High-dose Melphalan 200 mg/m2/day followed by a stem cell transplant
|
|---|---|---|
|
Overall Study
STARTED
|
105
|
100
|
|
Overall Study
COMPLETED
|
104
|
98
|
|
Overall Study
NOT COMPLETED
|
1
|
2
|
Reasons for withdrawal
| Measure |
Busulfan + Melphalan
Busulfan with a target daily area under the curve (AUC) of 5000 with Melphalan 70 mg/m2 followed by a stem cell transplant
|
Melphalan
High-dose Melphalan 200 mg/m2/day followed by a stem cell transplant
|
|---|---|---|
|
Overall Study
Toxicity
|
1
|
0
|
|
Overall Study
Ineligible
|
0
|
2
|
Baseline Characteristics
A Randomized Trial to Compare Busulfan + Melphalan 140 mg/m2 With Melphalan 200 mg/m2 as Preparative Regimen for Autologous Hematopoietic Stem Cell Transplantation for Multiple Myeloma
Baseline characteristics by cohort
| Measure |
Busulfan + Melphalan
n=104 Participants
Busulfan with a target daily area under the curve (AUC) of 5000 with Melphalan 70 mg/m2 followed by a stem cell transplant.
|
Melphalan
n=98 Participants
High-dose Melphalan 200 mg/m2/day followed by a stem cell transplant
|
Total
n=202 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
58.9 years
n=5 Participants
|
59.5 years
n=7 Participants
|
59.2 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
43 Participants
n=5 Participants
|
43 Participants
n=7 Participants
|
86 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
61 Participants
n=5 Participants
|
55 Participants
n=7 Participants
|
116 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
5 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
15 Participants
n=5 Participants
|
24 Participants
n=7 Participants
|
39 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
75 Participants
n=5 Participants
|
67 Participants
n=7 Participants
|
142 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
9 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
104 participants
n=5 Participants
|
98 participants
n=7 Participants
|
202 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 3 years after transplantParticipants that are still alive and without Multiple Myeloma 3 years after Stem cell Transplantation.
Outcome measures
| Measure |
Busulfan + Melphalan
n=104 Participants
Busulfan with a target daily area under the curve (AUC) of 5000 with Melphalan 70 mg/m2 followed by a stem cell transplant
|
Melphalan
n=98 Participants
High-dose Melphalan 200 mg/m2/day followed by a stem cell transplant
|
|---|---|---|
|
Progression-Free Survival (PFS)
|
72 Participants
|
50 Participants
|
SECONDARY outcome
Timeframe: Evaluated 90 days from transplant.Complete response (CR), evaluated 90 days from transplant, defined as (i) negative immunofixation of the multiple myeloma (MM) protein in urine and serum, (ii) disappearance of any soft tissue plasmacytomas, and (iii) less than 5% plasma MM cells in the bone marrow. International Myeloma Working Group uniform response criteria.
Outcome measures
| Measure |
Busulfan + Melphalan
n=104 Participants
Busulfan with a target daily area under the curve (AUC) of 5000 with Melphalan 70 mg/m2 followed by a stem cell transplant
|
Melphalan
n=98 Participants
High-dose Melphalan 200 mg/m2/day followed by a stem cell transplant
|
|---|---|---|
|
Number of Participants With Complete Response (CR)
|
12 Participants
|
15 Participants
|
SECONDARY outcome
Timeframe: 100 days post treatmentOutcome measures
| Measure |
Busulfan + Melphalan
n=104 Participants
Busulfan with a target daily area under the curve (AUC) of 5000 with Melphalan 70 mg/m2 followed by a stem cell transplant
|
Melphalan
n=98 Participants
High-dose Melphalan 200 mg/m2/day followed by a stem cell transplant
|
|---|---|---|
|
Treatment-Related Mortality (TRM) Between 2 Arms.
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: At day 90 post SCT (Stem Cell Transplantation)Outcome measures
| Measure |
Busulfan + Melphalan
n=104 Participants
Busulfan with a target daily area under the curve (AUC) of 5000 with Melphalan 70 mg/m2 followed by a stem cell transplant
|
Melphalan
n=98 Participants
High-dose Melphalan 200 mg/m2/day followed by a stem cell transplant
|
|---|---|---|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Diarrhea
|
5 participants
|
4 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Elevated ALT
|
3 participants
|
0 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Elevated Tbili
|
2 participants
|
0 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Neutropenic fever
|
73 participants
|
32 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Pneumonia
|
1 participants
|
1 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Tachycardia
|
1 participants
|
0 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Transient blindness
|
1 participants
|
0 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Anorexia
|
1 participants
|
0 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Myocardial ischemia
|
1 participants
|
1 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Pleural effusion
|
0 participants
|
1 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Rash
|
0 participants
|
1 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Infection
|
16 participants
|
5 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Mucositis
|
15 participants
|
0 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Nausea
|
5 participants
|
2 participants
|
|
Number of Participants That Had Grade 3-4 Toxicities.
Grade 3 - Neuropathy
|
1 participants
|
0 participants
|
SECONDARY outcome
Timeframe: From time of ASCT to 3 yearsOutcome measures
| Measure |
Busulfan + Melphalan
n=104 Participants
Busulfan with a target daily area under the curve (AUC) of 5000 with Melphalan 70 mg/m2 followed by a stem cell transplant
|
Melphalan
n=98 Participants
High-dose Melphalan 200 mg/m2/day followed by a stem cell transplant
|
|---|---|---|
|
Overall Survival (OS)
|
91 Participants
|
79 Participants
|
Adverse Events
Busulfan + Melphalan
Serious events: 73 serious events
Other events: 101 other events
Deaths: 23 deaths
Melphalan
Serious events: 32 serious events
Other events: 98 other events
Deaths: 21 deaths
Serious adverse events
| Measure |
Busulfan + Melphalan
n=104 participants at risk
Busulfan with a target daily area under the curve (AUC) of 5000 with Melphalan 70 mg/m2 followed by a stem cell transplant
|
Melphalan
n=98 participants at risk
High-dose Melphalan 200 mg/m2/day followed by a stem cell transplant
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
4.8%
5/104 • From the time of ASCT until 12 months.
|
4.1%
4/98 • From the time of ASCT until 12 months.
|
|
Hepatobiliary disorders
Elevated ALT
|
2.9%
3/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Hepatobiliary disorders
Elevated Tbili
|
1.9%
2/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Infections and infestations
Infection
|
15.4%
16/104 • From the time of ASCT until 12 months.
|
6.1%
6/98 • From the time of ASCT until 12 months.
|
|
Gastrointestinal disorders
Mucositis
|
14.4%
15/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Cardiac disorders
Myocardial ischemia
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
1.0%
1/98 • From the time of ASCT until 12 months.
|
|
Nervous system disorders
Neuropathy
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Infections and infestations
Neutropenic fever
|
70.2%
73/104 • From the time of ASCT until 12 months.
|
32.7%
32/98 • From the time of ASCT until 12 months.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Cardiac disorders
Tachycardia
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Eye disorders
Transient blindness
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Gastrointestinal disorders
Nausea
|
4.8%
5/104 • From the time of ASCT until 12 months.
|
2.0%
2/98 • From the time of ASCT until 12 months.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/104 • From the time of ASCT until 12 months.
|
1.0%
1/98 • From the time of ASCT until 12 months.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/104 • From the time of ASCT until 12 months.
|
1.0%
1/98 • From the time of ASCT until 12 months.
|
Other adverse events
| Measure |
Busulfan + Melphalan
n=104 participants at risk
Busulfan with a target daily area under the curve (AUC) of 5000 with Melphalan 70 mg/m2 followed by a stem cell transplant
|
Melphalan
n=98 participants at risk
High-dose Melphalan 200 mg/m2/day followed by a stem cell transplant
|
|---|---|---|
|
Cardiac disorders
Tachycardia
|
3.8%
4/104 • From the time of ASCT until 12 months.
|
1.0%
1/98 • From the time of ASCT until 12 months.
|
|
Ear and labyrinth disorders
Tinnitus
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Musculoskeletal and connective tissue disorders
Weakness of extremities
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Psychiatric disorders
Anorexia
|
0.00%
0/104 • From the time of ASCT until 12 months.
|
3.1%
3/98 • From the time of ASCT until 12 months.
|
|
Musculoskeletal and connective tissue disorders
involuntary leg movements
|
0.00%
0/104 • From the time of ASCT until 12 months.
|
1.0%
1/98 • From the time of ASCT until 12 months.
|
|
Skin and subcutaneous tissue disorders
Lip ulcer
|
0.00%
0/104 • From the time of ASCT until 12 months.
|
1.0%
1/98 • From the time of ASCT until 12 months.
|
|
Cardiac disorders
Arrhythmia
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Cardiac disorders
Atrial fibrillation
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
1.0%
1/98 • From the time of ASCT until 12 months.
|
|
Skin and subcutaneous tissue disorders
Chemo burns
|
2.9%
3/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Gastrointestinal disorders
Diarrhea
|
54.8%
57/104 • From the time of ASCT until 12 months.
|
83.7%
82/98 • From the time of ASCT until 12 months.
|
|
Nervous system disorders
Dizziness
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Reproductive system and breast disorders
Dysuria
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Hepatobiliary disorders
Elevated ALT
|
29.8%
31/104 • From the time of ASCT until 12 months.
|
1.0%
1/98 • From the time of ASCT until 12 months.
|
|
Hepatobiliary disorders
Elevated AST
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
1.0%
1/98 • From the time of ASCT until 12 months.
|
|
Blood and lymphatic system disorders
Elevated creatinine
|
2.9%
3/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Hepatobiliary disorders
Elevated Tbili
|
7.7%
8/104 • From the time of ASCT until 12 months.
|
9.2%
9/98 • From the time of ASCT until 12 months.
|
|
Infections and infestations
Fever
|
16.3%
17/104 • From the time of ASCT until 12 months.
|
8.2%
8/98 • From the time of ASCT until 12 months.
|
|
Cardiac disorders
Fluid overload
|
60.6%
63/104 • From the time of ASCT until 12 months.
|
22.4%
22/98 • From the time of ASCT until 12 months.
|
|
Nervous system disorders
Headache
|
1.9%
2/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Renal and urinary disorders
Hematuria
|
2.9%
3/104 • From the time of ASCT until 12 months.
|
2.0%
2/98 • From the time of ASCT until 12 months.
|
|
Cardiac disorders
Hypertension
|
15.4%
16/104 • From the time of ASCT until 12 months.
|
5.1%
5/98 • From the time of ASCT until 12 months.
|
|
Cardiac disorders
Hypotension
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Infections and infestations
Infection
|
17.3%
18/104 • From the time of ASCT until 12 months.
|
17.3%
17/98 • From the time of ASCT until 12 months.
|
|
Gastrointestinal disorders
Mucositis
|
82.7%
86/104 • From the time of ASCT until 12 months.
|
50.0%
49/98 • From the time of ASCT until 12 months.
|
|
Gastrointestinal disorders
Nausea
|
95.2%
99/104 • From the time of ASCT until 12 months.
|
100.0%
98/98 • From the time of ASCT until 12 months.
|
|
Infections and infestations
Neutropenic fever
|
0.96%
1/104 • From the time of ASCT until 12 months.
|
33.7%
33/98 • From the time of ASCT until 12 months.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
4.8%
5/104 • From the time of ASCT until 12 months.
|
4.1%
4/98 • From the time of ASCT until 12 months.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
1.9%
2/104 • From the time of ASCT until 12 months.
|
0.00%
0/98 • From the time of ASCT until 12 months.
|
|
Skin and subcutaneous tissue disorders
Rash
|
4.8%
5/104 • From the time of ASCT until 12 months.
|
4.1%
4/98 • From the time of ASCT until 12 months.
|
|
Cardiac disorders
Myocardial ischemia
|
0.00%
0/104 • From the time of ASCT until 12 months.
|
1.0%
1/98 • From the time of ASCT until 12 months.
|
|
Nervous system disorders
Neuropathy
|
0.00%
0/104 • From the time of ASCT until 12 months.
|
1.0%
1/98 • From the time of ASCT until 12 months.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/104 • From the time of ASCT until 12 months.
|
1.0%
1/98 • From the time of ASCT until 12 months.
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of breath
|
0.00%
0/104 • From the time of ASCT until 12 months.
|
1.0%
1/98 • From the time of ASCT until 12 months.
|
Additional Information
Dr. Muzaffar H. Qazilbash / Stem Cell Transplantation and Cellular Therapy
U.T. MD Anderson Cancer Center
Phone: 713-745-3219
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place