Trial Outcomes & Findings for Busulfan, Melphalan, and Fludarabine With Peri-transplant Palifermin, Followed by a T-Cell Depleted Hematopoietic Stem Cell Transplant From HLA Matched or Mismatched Related or Unrelated Donors in Patients With Advanced Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML) (NCT NCT00629798)
NCT ID: NCT00629798
Last Updated: 2021-09-24
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
64 participants
Primary outcome timeframe
conclusion of study
Results posted on
2021-09-24
Participant Flow
Participant milestones
| Measure |
Palifermin
This is a single arm phase II trial to assess the efficacy (decrease the transplant related mortality) and safety of peri-transplant Palifermin in combination with 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 advanced MDS and AML evolved from MDS. The addition of Palifermin is to decrease the toxicity and the infection rate associated with this regimen and transplant type and to foster earlier immune reconstitution.
Busulfan, Melphalan, Fludarabine, Anti-Thymocyte Globulin, Palifermin, Stem cell transplant: Patients will receive Palifermin 60 mcg/kg/day IV on three consecutive days with the last dose administered no less than 24 and no more than 48 hr prior to start of cytoreduction. The preparative regimen to be used for transplants will consist: of busulfan administered in 12 doses over three days of 0.8 mg/kg IV for patients \> or = to 4 years of age or 1.0 mg/kg IV for patients \< 4 years of age; melphalan 70 mg/m2 IV x 2 days; and, fludarabine 25 mg/m2 IV x 5 days
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|---|---|
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Overall Study
STARTED
|
64
|
|
Overall Study
COMPLETED
|
60
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
Palifermin
This is a single arm phase II trial to assess the efficacy (decrease the transplant related mortality) and safety of peri-transplant Palifermin in combination with 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 advanced MDS and AML evolved from MDS. The addition of Palifermin is to decrease the toxicity and the infection rate associated with this regimen and transplant type and to foster earlier immune reconstitution.
Busulfan, Melphalan, Fludarabine, Anti-Thymocyte Globulin, Palifermin, Stem cell transplant: Patients will receive Palifermin 60 mcg/kg/day IV on three consecutive days with the last dose administered no less than 24 and no more than 48 hr prior to start of cytoreduction. The preparative regimen to be used for transplants will consist: of busulfan administered in 12 doses over three days of 0.8 mg/kg IV for patients \> or = to 4 years of age or 1.0 mg/kg IV for patients \< 4 years of age; melphalan 70 mg/m2 IV x 2 days; and, fludarabine 25 mg/m2 IV x 5 days
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|---|---|
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Overall Study
Not Treated
|
4
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Baseline Characteristics
Busulfan, Melphalan, and Fludarabine With Peri-transplant Palifermin, Followed by a T-Cell Depleted Hematopoietic Stem Cell Transplant From HLA Matched or Mismatched Related or Unrelated Donors in Patients With Advanced Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML)
Baseline characteristics by cohort
| Measure |
Palifermin
n=64 Participants
This is a single arm phase II trial to assess the efficacy (decrease the transplant related mortality) and safety of peri-transplant Palifermin in combination with 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 advanced MDS and AML evolved from MDS. The addition of Palifermin is to decrease the toxicity and the infection rate associated with this regimen and transplant type and to foster earlier immune reconstitution.
Busulfan, Melphalan, Fludarabine, Anti-Thymocyte Globulin, Palifermin, Stem cell transplant: Patients will receive Palifermin 60 mcg/kg/day IV on three consecutive days with the last dose administered no less than 24 and no more than 48 hr prior to start of cytoreduction. The preparative regimen to be used for transplants will consist: of busulfan administered in 12 doses over three days of 0.8 mg/kg IV for patients \> or = to 4 years of age or 1.0 mg/kg IV for patients \< 4 years of age; melphalan 70 mg/m2 IV x 2 days; and, fludarabine 25 mg/m2 IV x 5 days
|
|---|---|
|
Age, Continuous
|
60 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
34 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
30 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
61 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
5 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
58 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
64 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: conclusion of studyPopulation: Data were not collected
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: conclusion of studyPopulation: Data were not collected
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: conclusion of studyPopulation: Data were not collected
Outcome measures
Outcome data not reported
Adverse Events
Palifermin
Serious events: 28 serious events
Other events: 28 other events
Deaths: 38 deaths
Serious adverse events
| Measure |
Palifermin
n=64 participants at risk
This is a single arm phase II trial to assess the efficacy (decrease the transplant related mortality) and safety of peri-transplant Palifermin in combination with 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 advanced MDS and AML evolved from MDS. The addition of Palifermin is to decrease the toxicity and the infection rate associated with this regimen and transplant type and to foster earlier immune reconstitution.
Busulfan, Melphalan, Fludarabine, Anti-Thymocyte Globulin, Palifermin, Stem cell transplant: Patients will receive Palifermin 60 mcg/kg/day IV on three consecutive days with the last dose administered no less than 24 and no more than 48 hr prior to start of cytoreduction. The preparative regimen to be used for transplants will consist: of busulfan administered in 12 doses over three days of 0.8 mg/kg IV for patients \> or = to 4 years of age or 1.0 mg/kg IV for patients \< 4 years of age; melphalan 70 mg/m2 IV x 2 days; and, fludarabine 25 mg/m2 IV x 5 days
|
|---|---|
|
Metabolism and nutrition disorders
Acidosis (metabolic/respiratory)
|
1.6%
1/64 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Adult Respiratory Distress Synd (ARDS)
|
3.1%
2/64 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
1.6%
1/64 • 1 year
|
|
Cardiac disorders
Atrial Fibrillation
|
1.6%
1/64 • 1 year
|
|
Investigations
Bilirubin (hyperbilirubinemia)
|
1.6%
1/64 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
1.6%
1/64 • 1 year
|
|
General disorders
Death not assoc w CTCAE term - Death NOS
|
6.2%
4/64 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
1.6%
1/64 • 1 year
|
|
Nervous system disorders
Encephalopathy
|
1.6%
1/64 • 1 year
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
1.6%
1/64 • 1 year
|
|
General disorders
Fever (in the absence of neutropenia)
|
1.6%
1/64 • 1 year
|
|
Gastrointestinal disorders
Hemorrhage, Upper GI NOS
|
1.6%
1/64 • 1 year
|
|
Blood and lymphatic system disorders
Hemorrhage/Bleeding, other
|
1.6%
1/64 • 1 year
|
|
Vascular disorders
Hypotension
|
3.1%
2/64 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
17.2%
11/64 • 1 year
|
|
Infections and infestations
Inf norm ANC/gr1/2 neut-Pneumonia (lung)
|
4.7%
3/64 • 1 year
|
|
Infections and infestations
Infection, other
|
15.6%
10/64 • 1 year
|
|
Renal and urinary disorders
Liver dysfunction/failure
|
3.1%
2/64 • 1 year
|
|
Investigations
Neutrophils/granulocytes (ANC/AGC)
|
3.1%
2/64 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Pain - Extremity-limb
|
1.6%
1/64 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pain - Sinus
|
1.6%
1/64 • 1 year
|
|
Investigations
Platelets
|
1.6%
1/64 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion (non-malig)
|
1.6%
1/64 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis/pulm inflitrates
|
1.6%
1/64 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pulm/upp respiratory - Other (spec)
|
1.6%
1/64 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
3.1%
2/64 • 1 year
|
|
Renal and urinary disorders
Renal failure
|
1.6%
1/64 • 1 year
|
|
Renal and urinary disorders
Renal/Genitourinary-Other Specify
|
4.7%
3/64 • 1 year
|
|
Nervous system disorders
Seizure
|
1.6%
1/64 • 1 year
|
|
Cardiac disorders
Sinus tachycardia
|
1.6%
1/64 • 1 year
|
|
Gastrointestinal disorders
Vomiting
|
1.6%
1/64 • 1 year
|
Other adverse events
| Measure |
Palifermin
n=64 participants at risk
This is a single arm phase II trial to assess the efficacy (decrease the transplant related mortality) and safety of peri-transplant Palifermin in combination with 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 advanced MDS and AML evolved from MDS. The addition of Palifermin is to decrease the toxicity and the infection rate associated with this regimen and transplant type and to foster earlier immune reconstitution.
Busulfan, Melphalan, Fludarabine, Anti-Thymocyte Globulin, Palifermin, Stem cell transplant: Patients will receive Palifermin 60 mcg/kg/day IV on three consecutive days with the last dose administered no less than 24 and no more than 48 hr prior to start of cytoreduction. The preparative regimen to be used for transplants will consist: of busulfan administered in 12 doses over three days of 0.8 mg/kg IV for patients \> or = to 4 years of age or 1.0 mg/kg IV for patients \< 4 years of age; melphalan 70 mg/m2 IV x 2 days; and, fludarabine 25 mg/m2 IV x 5 days
|
|---|---|
|
Metabolism and nutrition disorders
Glucose, high (hyperglycemia)
|
40.6%
26/64 • 1 year
|
|
Investigations
ALT, SGPT
|
32.8%
21/64 • 1 year
|
|
Investigations
Creatinine
|
28.1%
18/64 • 1 year
|
|
Metabolism and nutrition disorders
Magnesium, high (hypermagnesemia)
|
21.9%
14/64 • 1 year
|
|
Investigations
AST/SGOT
|
18.8%
12/64 • 1 year
|
|
Investigations
Bilirubin (hyperbilirubinemia)
|
15.6%
10/64 • 1 year
|
|
Investigations
Alkaline phosphatase
|
14.1%
9/64 • 1 year
|
Additional Information
Dr. Roni Tamari, MD
Memorial Sloan Kettering Cancer Center
Phone: 646-608-3738
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place