Clinical trial NCT00629798: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Acute Myeloid Leukemia
Advanced Myelodysplastic Syndromes
Keywords: Busulfan
Melphalan
Fludarabine
08-008
Lead sponsor: Memorial Sloan Kettering Cancer Center
Sponsor class: OTHER
First posted: 2008-03-06
Enrollment: 64
Sex: ALL
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: To Reduce the Early Transplant-related Mortality.
timeFrame: conclusion of study
Secondary outcomes:
measure: To Improve the Quality of Immune Reconstitution Following Transplantation.
timeFrame: conclusion of study
measure: To Reduce the Incidence Rate of Fatal Post Transplant Infectious Complications.
timeFrame: conclusion of study
Other outcomes:
Locations:
city: New York
country: United States
facility: Memorial Sloan-Kettering Cancer Center
state: New York
Study officials:
affiliation: Memorial Sloan Kettering Cancer Center
name: Roni Tamari, MD
role: PRINCIPAL_INVESTIGATOR
References: