Trial Outcomes & Findings for Busulfan, Melphalan, Fludarabine and T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation Followed by Post Transplantation Donor Lymphocyte Infusions (NCT NCT01131169)
NCT ID: NCT01131169
Last Updated: 2021-12-02
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
66 participants
Primary outcome timeframe
2 years
Results posted on
2021-12-02
Participant Flow
Participant milestones
| Measure |
Relapsed Multiple Myeloma
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma
|
High-risk Multiple Myeloma
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma.
|
|---|---|---|
|
Overall Study
STARTED
|
47
|
19
|
|
Overall Study
COMPLETED
|
47
|
19
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Busulfan, Melphalan, Fludarabine and T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation Followed by Post Transplantation Donor Lymphocyte Infusions
Baseline characteristics by cohort
| Measure |
Relapsed Multiple Myeloma
n=47 Participants
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma
|
High-risk Multiple Myeloma
n=19 Participants
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma.
|
Total
n=66 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
55.3 years
n=5 Participants
|
49.5 years
n=7 Participants
|
54 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
35 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
49 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
42 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
59 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
4 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
4 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
|
8 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
34 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
51 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
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
46 participants
n=5 Participants
|
19 participants
n=7 Participants
|
65 participants
n=5 Participants
|
|
Region of Enrollment
Turkey
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 yearsPopulation: This objective only applies to Arm I: Participants with Relapsed Multiple Myeloma
Outcome measures
| Measure |
Relapsed Multiple Myeloma
n=47 Participants
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma
|
High-risk Multiple Myeloma
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma.
|
|---|---|---|
|
Proportion of Participants With Relapsed Multiple Myeloma With Progression-free (PFS)
|
0.31 Proportion of participants PFS
Interval 0.19 to 0.5
|
—
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: This objective is only for participants with relapsed multiple myeloma.
Outcome measures
| Measure |
Relapsed Multiple Myeloma
n=44 Participants
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma
|
High-risk Multiple Myeloma
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma.
|
|---|---|---|
|
Proportion of Participants With Relapsed Multiple Myeloma Alive at 2 Years
|
0.54 Proportion of pts alive at 2 years
Interval 0.41 to 0.72
|
—
|
SECONDARY outcome
Timeframe: 3 yearsPopulation: This group consists of participants with residual disease either because they did not reach CR from salvage and transplant conditioning or because they were given this dose at the time of progression
To compute the current multiple myeloma free survival curve in order to account for patients who relapse and are restored to remission through DLI.
Outcome measures
| Measure |
Relapsed Multiple Myeloma
n=18 Participants
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma
|
High-risk Multiple Myeloma
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma.
|
|---|---|---|
|
Number of Participants Who Relapse That Are Restored to Remission (CR)
Restored to CR, did not develop GVHD
|
4 Participants
|
—
|
|
Number of Participants Who Relapse That Are Restored to Remission (CR)
Were not restored to CR, did not develop GVHD
|
14 Participants
|
—
|
Adverse Events
Relapsed Multiple Myeloma
Serious events: 29 serious events
Other events: 47 other events
Deaths: 17 deaths
High-risk Multiple Myeloma
Serious events: 11 serious events
Other events: 19 other events
Deaths: 9 deaths
Serious adverse events
| Measure |
Relapsed Multiple Myeloma
n=47 participants at risk
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma
|
High-risk Multiple Myeloma
n=19 participants at risk
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
4.3%
2/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
Renal and urinary disorders
Acute kidney injury
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
Investigations
Alkaline phosphatase increased
|
0.00%
0/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
Blood and lymphatic system disorders
Anemia
|
4.3%
2/47 • Through study completion, an average of 3 years
|
10.5%
2/19 • Through study completion, an average of 3 years
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Investigations
Blood bilirubin increased
|
0.00%
0/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
Cardiac disorders
Cardiac disorders - Other, specify
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Infections and infestations
Catheter related infection
|
4.3%
2/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Psychiatric disorders
Confusion
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Investigations
Creatinine increased
|
0.00%
0/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
General disorders
Death NOS
|
19.1%
9/47 • Through study completion, an average of 3 years
|
10.5%
2/19 • Through study completion, an average of 3 years
|
|
General disorders
Death not assoc w CTCAE term-Disease prog NOS
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Metabolism and nutrition disorders
Dehydration
|
4.3%
2/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Gastrointestinal disorders
Diarrhea
|
6.4%
3/47 • Through study completion, an average of 3 years
|
21.1%
4/19 • Through study completion, an average of 3 years
|
|
Nervous system disorders
Dizziness
|
0.00%
0/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
6.4%
3/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
General disorders
Fatigue
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
4.3%
2/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
General disorders
Fever
|
8.5%
4/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
General disorders
Gen disorders & admin site conditions Other, spec
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Renal and urinary disorders
Hematuria
|
2.1%
1/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
Vascular disorders
Hypotension
|
4.3%
2/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
6.4%
3/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Infections and infestations
Inf norm ANC/gr1/2 neut-Pneumonia(lung)
|
2.1%
1/47 • Through study completion, an average of 3 years
|
10.5%
2/19 • Through study completion, an average of 3 years
|
|
Infections and infestations
Infection, other
|
2.1%
1/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
Infections and infestations
Infections and infestations - Other, specify
|
36.2%
17/47 • Through study completion, an average of 3 years
|
21.1%
4/19 • Through study completion, an average of 3 years
|
|
Nervous system disorders
Lethargy
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Infections and infestations
Lung infection
|
14.9%
7/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Gastrointestinal disorders
Nausea
|
12.8%
6/47 • Through study completion, an average of 3 years
|
15.8%
3/19 • Through study completion, an average of 3 years
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Nervous system disorders
Nervous system disorders - Other, specify
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Nervous system disorders
Neurology - Other (specify)
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
General disorders
Non-cardiac chest pain
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Investigations
Platelet count decreased
|
0.00%
0/47 • Through study completion, an average of 3 years
|
10.5%
2/19 • Through study completion, an average of 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
4.3%
2/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Resp, thoracic & mediastinal disorder Other, spec
|
0.00%
0/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
Infections and infestations
Sepsis
|
8.5%
4/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Infections and infestations
Sinusitis
|
2.1%
1/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Vascular disorders
Thromboembolic event
|
4.3%
2/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Infections and infestations
Upper respiratory infection
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Renal and urinary disorders
Urinary tract pain
|
2.1%
1/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
|
Renal and urinary disorders
Urinary urgency
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Eye disorders
Uveitis
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Nervous system disorders
Vasovagal reaction
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Cardiac disorders
Ventricular tachycardia
|
2.1%
1/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Gastrointestinal disorders
Vomiting
|
6.4%
3/47 • Through study completion, an average of 3 years
|
5.3%
1/19 • Through study completion, an average of 3 years
|
Other adverse events
| Measure |
Relapsed Multiple Myeloma
n=47 participants at risk
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma
|
High-risk Multiple Myeloma
n=19 participants at risk
This is a two arm phase II trial to assess the progression-free and overall survival as well as the safety and efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with relapsed or high-risk multiple myeloma.
|
|---|---|---|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
6.4%
3/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Vascular disorders
Hypotension
|
6.4%
3/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
General disorders
Pain
|
6.4%
3/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Renal and urinary disorders
Renal and urinary disorders - Other, specify
|
6.4%
3/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
General disorders
Fatigue
|
8.5%
4/47 • Through study completion, an average of 3 years
|
15.8%
3/19 • Through study completion, an average of 3 years
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
10.6%
5/47 • Through study completion, an average of 3 years
|
15.8%
3/19 • Through study completion, an average of 3 years
|
|
Investigations
Weight loss
|
10.6%
5/47 • Through study completion, an average of 3 years
|
26.3%
5/19 • Through study completion, an average of 3 years
|
|
Gastrointestinal disorders
Mucositis oral
|
10.6%
5/47 • Through study completion, an average of 3 years
|
31.6%
6/19 • Through study completion, an average of 3 years
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
10.6%
5/47 • Through study completion, an average of 3 years
|
0.00%
0/19 • Through study completion, an average of 3 years
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
12.8%
6/47 • Through study completion, an average of 3 years
|
21.1%
4/19 • Through study completion, an average of 3 years
|
|
Metabolism and nutrition disorders
Anorexia
|
12.8%
6/47 • Through study completion, an average of 3 years
|
31.6%
6/19 • Through study completion, an average of 3 years
|
|
Investigations
Activated partial thromboplastin time prolonged
|
14.9%
7/47 • Through study completion, an average of 3 years
|
15.8%
3/19 • Through study completion, an average of 3 years
|
|
Investigations
Alanine aminotransferase increased
|
70.2%
33/47 • Through study completion, an average of 3 years
|
63.2%
12/19 • Through study completion, an average of 3 years
|
|
Investigations
Alkaline phosphatase increased
|
46.8%
22/47 • Through study completion, an average of 3 years
|
36.8%
7/19 • Through study completion, an average of 3 years
|
|
Investigations
Aspartate aminotransferase increased
|
63.8%
30/47 • Through study completion, an average of 3 years
|
52.6%
10/19 • Through study completion, an average of 3 years
|
Additional Information
Sergio Giralt, MD
Memorial Sloan Kettering Cancer Center
Phone: 646-608-3731
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place