Phase 2
N=64
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)
Acute Myeloid Leukemia · Advanced Myelodysplastic Syndromes
Bottom Line
View on ClinicalTrials.gov: NCT00629798 ↗Enrolled (actual)
64
Serious AEs
43.8%
Results posted
Sep 2021
Primary outcome: Primary: To Reduce the Early Transplant-related Mortality.
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Busulfan, Melphalan, Fludarabine, Anti-Thymocyte Globulin, Palifermin, Stem cell transplant (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Memorial Sloan Kettering Cancer Center
- Primary completion
- Oct 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY To Reduce the Early Transplant-related Mortality. |
— | — |
| SECONDARY To Improve the Quality of Immune Reconstitution Following Transplantation. |
— | — |
| SECONDARY To Reduce the Incidence Rate of Fatal Post Transplant Infectious Complications. |
— | — |
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 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: 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.
Data sourced from ClinicalTrials.gov (NCT00629798). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.