A Phase 2 interventional study of Busulfan, Melphalan, Fludarabine, Anti-Thymocyte Globulin, Palifermin, Stem cell transplant in Acute Myeloid Leukemia and Advanced Myelodysplastic Syndromes, sponsored by Memorial Sloan Kettering Cancer Center. Completed at 1 site in United States. Open to participants aged Up to 65 Years. Per ClinicalTrials.gov, last updated 2021-09-24.
Sponsored by Memorial Sloan Kettering Cancer Center · Phase 2, Interventional, and Treatment
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.
5,441 studies on the registry are indexed under Leukemia; 636 are open to participants now.
This study's enrollment of 64 is above the median of 38 across 4,247 interventional studies indexed under Leukemia.
Browse Leukemia studies →Memorial Sloan Kettering Cancer Center is the lead sponsor of 1,930 studies on the registry; 328 are open to participants now.
Of its 129 completed or terminated interventional studies of FDA-regulated products, 66 (51%) have results posted.
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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.
Exclusion Criteria:
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.
Drug: 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
Also known as: 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.
To Reduce the Early Transplant-related Mortality.
Time frame: conclusion of study
To Improve the Quality of Immune Reconstitution Following Transplantation.
Time frame: conclusion of study
To Reduce the Incidence Rate of Fatal Post Transplant Infectious Complications.
Time frame: conclusion of study
| Milestone | Palifermin |
|---|---|
| Started | 64 |
| Completed | 60 |
| Not completed | 4 |
| Withdrew: Not treated | 4 |
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
Collected over 1 year. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Palifermin | 38/64 (59.4%) | 28/64 (43.8%) | 28/64 (43.8%) |
| Event | Palifermin |
|---|---|
| HypoxiaRespiratory, thoracic and mediastinal disorders | 11/64 |
| Infection, otherInfections and infestations | 10/64 |
| Death not assoc w CTCAE term - Death NOSGeneral disorders | 4/64 |
| Inf norm ANC/gr1/2 neut-Pneumonia (lung)Infections and infestations | 3/64 |
| Renal/Genitourinary-Other SpecifyRenal and urinary disorders | 3/64 |
| Adult Respiratory Distress Synd (ARDS)Respiratory, thoracic and mediastinal disorders | 2/64 |
| HypotensionVascular disorders | 2/64 |
| Liver dysfunction/failureRenal and urinary disorders | 2/64 |
| Neutrophils/granulocytes (ANC/AGC)Investigations | 2/64 |
| Pulmonary hypertensionRespiratory, thoracic and mediastinal disorders | 2/64 |
| Event | Palifermin |
|---|---|
| Glucose, high (hyperglycemia)Metabolism and nutrition disorders | 26/64 |
| ALT, SGPTInvestigations | 21/64 |
| CreatinineInvestigations | 18/64 |
| Magnesium, high (hypermagnesemia)Metabolism and nutrition disorders | 14/64 |
| AST/SGOTInvestigations | 12/64 |
| Bilirubin (hyperbilirubinemia)Investigations | 10/64 |
| Alkaline phosphataseInvestigations | 9/64 |
| Age, Continuous(years) | Palifermin |
|---|---|
| Median | 60 (4 to 72) |
| Sex: Female, Male(Participants) | Palifermin |
|---|---|
| Female | 34 |
| Male | 30 |
| Ethnicity (NIH/OMB)(Participants) | Palifermin |
|---|---|
| Hispanic or Latino | 3 |
| Not Hispanic or Latino | 61 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Palifermin |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 5 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 58 |
| More than one race | 0 |
| Unknown or Not Reported | 1 |
| Region of Enrollment(Participants) | Palifermin |
|---|---|
| United States | 64 |
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Memorial Sloan Kettering Cancer Center