Back to resultsBusulfan, 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)
Primary Purpose
Acute Myeloid Leukemia, Advanced Myelodysplastic Syndromes
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Busulfan, Melphalan, Fludarabine, Anti-Thymocyte Globulin, Palifermin, Stem cell transplant
Sponsored by
About this trial
This is an interventional treatment trial for Acute Myeloid Leukemia focused on measuring Busulfan, Melphalan, Fludarabine, 08-008
Eligibility Criteria
Inclusion Criteria:
- Patients should be < 65 years. Patients > or equal to 65 years will be accrued on a case by case basis after discussion and approval by the BMT Service.
- Patients may be of either gender or any ethnic background.
- Patients must have a Karnofsky or Lansky Performance Status > or equal to 70%.
Patients must have adequate organ function measured by:
* Cardiac: asymptomatic or if symptomatic then LVEF at rest must be > or equal to 50% and must improve with exercise.
Hepatic: < 3x ULN ALT and < 1.5 total serum bilirubin, unless there is congenital benign hyperbilirubinemia.
- Renal: serum creatinine < than or equal to 1.2 mg/dl or if serum creatinine is outside the normal range, then CrCl > 60-ml/min/1.73 m2
- Pulmonary: asymptomatic or if symptomatic, DLCO > 50% of predicted (corrected for hemoglobin)
- Each patient must be willing to participate as a research subject and must sign an informed consent form.
- Parent or legal guardians of patients who are minors will sign the informed consent form.
Exclusion Criteria:
- Active CNS or skin leukemic involvement
- Female patients who are pregnant or breast-feeding
- Active viral, bacterial or fungal infection
- Patient seropositive for HIV-I/II; HTLV -I/II
- Patients who have undergone a prior allogeneic or autologous stem cell transplant within the previous six months.
- Patients who have had a previous malignancy that is not in remission.
Sites / Locations
- Memorial Sloan-Kettering Cancer Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
1
Arm Description
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.
Outcomes
Primary Outcome Measures
To Reduce the Early Transplant-related Mortality.
Secondary Outcome Measures
To Improve the Quality of Immune Reconstitution Following Transplantation.
To Reduce the Incidence Rate of Fatal Post Transplant Infectious Complications.
Full Information
NCT ID
NCT00629798
First Posted
February 14, 2008
Last Updated
August 27, 2021
Sponsor
Memorial Sloan Kettering Cancer Center
1. Study Identification
Unique Protocol Identification Number
NCT00629798
Brief Title
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)
Official Title
A Phase II Trial of 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)
Study Type
Interventional
2. Study Status
Record Verification Date
October 2020
Overall Recruitment Status
Completed
Study Start Date
February 12, 2008 (Actual)
Primary Completion Date
October 20, 2020 (Actual)
Study Completion Date
October 20, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Memorial Sloan Kettering Cancer Center
4. Oversight
5. Study Description
Brief Summary
This study will see if the researchers can lower that risk by giving the patient Palifermin. This drug helps protect the lining of the mouth, throat, and stomach. These areas typically get sores or ulcers while the blood cell counts are very low. The patient can get infections in or from these sores. Palifermin might also help the immune system recover faster. It is currently approved for patients who receive their own stem cells. That is called an autologous transplant. This study will test the use of Palifermin for T-cell depleted allogeneic stem cell transplants.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Myeloid Leukemia, Advanced Myelodysplastic Syndromes
Keywords
Busulfan, Melphalan, Fludarabine, 08-008
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
64 (Actual)
8. Arms, Groups, and Interventions
Arm Title
1
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Busulfan, Melphalan, Fludarabine, Anti-Thymocyte Globulin, Palifermin, Stem cell transplant
Other Intervention Name(s)
Patients will receive ATG for two doses prior to transplant., G-CSF mobilized CD34+E- PBSCs (or BM if donors are unwilling or unable, to donate PBSC) obtained from the HLA compatible donor will be infused on, day 0. Patients will receive three additional daily doses of Palifermin,, the first approximately 6 hours after the stem cell infusion on day 0,, followed by two daily doses given on d+1 and d+2. Supportive care will be administered as per the BMT Service, guidelines.
Intervention Description
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
Primary Outcome Measure Information:
Title
To Reduce the Early Transplant-related Mortality.
Time Frame
conclusion of study
Secondary Outcome Measure Information:
Title
To Improve the Quality of Immune Reconstitution Following Transplantation.
Time Frame
conclusion of study
Title
To Reduce the Incidence Rate of Fatal Post Transplant Infectious Complications.
Time Frame
conclusion of study
10. Eligibility
Sex
All
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients should be < 65 years. Patients > or equal to 65 years will be accrued on a case by case basis after discussion and approval by the BMT Service.
Patients may be of either gender or any ethnic background.
Patients must have a Karnofsky or Lansky Performance Status > or equal to 70%.
Patients must have adequate organ function measured by:
* Cardiac: asymptomatic or if symptomatic then LVEF at rest must be > or equal to 50% and must improve with exercise.
Hepatic: < 3x ULN ALT and < 1.5 total serum bilirubin, unless there is congenital benign hyperbilirubinemia.
Renal: serum creatinine < than or equal to 1.2 mg/dl or if serum creatinine is outside the normal range, then CrCl > 60-ml/min/1.73 m2
Pulmonary: asymptomatic or if symptomatic, DLCO > 50% of predicted (corrected for hemoglobin)
Each patient must be willing to participate as a research subject and must sign an informed consent form.
Parent or legal guardians of patients who are minors will sign the informed consent form.
Exclusion Criteria:
Active CNS or skin leukemic involvement
Female patients who are pregnant or breast-feeding
Active viral, bacterial or fungal infection
Patient seropositive for HIV-I/II; HTLV -I/II
Patients who have undergone a prior allogeneic or autologous stem cell transplant within the previous six months.
Patients who have had a previous malignancy that is not in remission.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Roni Tamari, MD
Organizational Affiliation
Memorial Sloan Kettering Cancer Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Memorial Sloan-Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
12. IPD Sharing Statement
Links:
URL
http://www.mskcc.org
Description
Memorial Sloan-Kettering Cancer Center
Learn more about this trial
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)
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