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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Busulfan, Melphalan, Fludarabine, Anti-Thymocyte Globulin, Palifermin, Stem cell transplant | Drug | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| To Reduce the Early Transplant-related Mortality. | conclusion of study |
| Measure | Description | Time Frame |
|---|---|---|
| To Improve the Quality of Immune Reconstitution Following Transplantation. | conclusion of study | |
| To Reduce the Incidence Rate of Fatal Post Transplant Infectious Complications. | conclusion of study |
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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.
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:
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| Name | Affiliation | Role |
|---|---|---|
| Roni Tamari, MD | Memorial Sloan Kettering Cancer Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Memorial Sloan-Kettering Cancer Center | New York | New York | 10065 | United States |
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| Label | URL |
|---|---|
| Memorial Sloan-Kettering Cancer Center | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jan 2, 2019 |
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|
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median | Full Range | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | To Reduce the Early Transplant-related Mortality. | Data were not collected | Posted | conclusion of study |
|
| ||||||||||||||||||||
| Secondary | To Improve the Quality of Immune Reconstitution Following Transplantation. | Data were not collected | Posted | conclusion of study |
|
| ||||||||||||||||||||
| Secondary | To Reduce the Incidence Rate of Fatal Post Transplant Infectious Complications. | Data were not collected | Posted | conclusion of study |
|
|
1 year
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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 | 38 | 64 | 28 | 64 | 28 | 64 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acidosis (metabolic/respiratory) | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Adult Respiratory Distress Synd (ARDS) | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Atelectasis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Atrial Fibrillation | Cardiac disorders | Systematic Assessment |
| ||
| Bilirubin (hyperbilirubinemia) | Investigations | Systematic Assessment |
| ||
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Death not assoc w CTCAE term - Death NOS | General disorders | Systematic Assessment |
| ||
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Encephalopathy | Nervous system disorders | Systematic Assessment |
| ||
| Febrile neutropenia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Fever (in the absence of neutropenia) | General disorders | Systematic Assessment |
| ||
| Hemorrhage, Upper GI NOS | Gastrointestinal disorders | Systematic Assessment |
| ||
| Hemorrhage/Bleeding, other | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Hypotension | Vascular disorders | Systematic Assessment |
| ||
| Hypoxia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Inf norm ANC/gr1/2 neut-Pneumonia (lung) | Infections and infestations | Systematic Assessment |
| ||
| Infection, other | Infections and infestations | Systematic Assessment |
| ||
| Liver dysfunction/failure | Renal and urinary disorders | Systematic Assessment |
| ||
| Neutrophils/granulocytes (ANC/AGC) | Investigations | Systematic Assessment |
| ||
| Pain - Extremity-limb | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Pain - Sinus | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Platelets | Investigations | Systematic Assessment |
| ||
| Pleural effusion (non-malig) | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pneumonitis/pulm inflitrates | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pulm/upp respiratory - Other (spec) | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Renal failure | Renal and urinary disorders | Systematic Assessment |
| ||
| Renal/Genitourinary-Other Specify | Renal and urinary disorders | Systematic Assessment |
| ||
| Seizure | Nervous system disorders | Systematic Assessment |
| ||
| Sinus tachycardia | Cardiac disorders | Systematic Assessment |
| ||
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Glucose, high (hyperglycemia) | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| ALT, SGPT | Investigations | Systematic Assessment |
| ||
| Creatinine | Investigations | Systematic Assessment |
| ||
| Magnesium, high (hypermagnesemia) | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| AST/SGOT | Investigations | Systematic Assessment |
| ||
| Bilirubin (hyperbilirubinemia) | Investigations | Systematic Assessment |
| ||
| Alkaline phosphatase | Investigations | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Roni Tamari, MD | Memorial Sloan Kettering Cancer Center | 646-608-3738 | tamarir@mskcc.org |
| Aug 27, 2021 |
| Prot_SAP_000.pdf |
| ID | Term |
|---|---|
| D015470 | Leukemia, Myeloid, Acute |
| ID | Term |
|---|---|
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| D002066 | Busulfan |
| D008558 | Melphalan |
| C024352 | fludarabine |
| D000961 | Antilymphocyte Serum |
| D051523 | Fibroblast Growth Factor 7 |
| D033581 | Stem Cell Transplantation |
| D017408 | Guidelines as Topic |
| ID | Term |
|---|---|
| D002072 | Butylene Glycols |
| D006018 | Glycols |
| D000438 | Alcohols |
| D009930 | Organic Chemicals |
| D008698 | Mesylates |
| D000476 | Alkanesulfonates |
| D017738 | Alkanesulfonic Acids |
| D000473 | Alkanes |
| D006839 | Hydrocarbons, Acyclic |
| D006838 | Hydrocarbons |
| D013451 | Sulfonic Acids |
| D013456 | Sulfur Acids |
| D013457 | Sulfur Compounds |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D010649 | Phenylalanine |
| D024322 | Amino Acids, Aromatic |
| D000598 | Amino Acids, Cyclic |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
| D007106 | Immune Sera |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| D005346 | Fibroblast Growth Factors |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D001685 | Biological Factors |
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |
| D011785 | Quality Assurance, Health Care |
| D011787 | Quality of Health Care |
| D006298 | Health Services Administration |
| D017530 | Health Care Quality, Access, and Evaluation |
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| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|