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| ID | Type | Description | Link |
|---|---|---|---|
| 0607M89052 | Other Identifier | IRB, University of Minnesota |
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Slow Accrual
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RATIONALE: Giving chemotherapy and colony-stimulating factors, such as G-CSF, may increase the number of stem cells in the blood. The stem cells are collected from the patient's blood and stored. Chemotherapy or radiation therapy is given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy and radiation therapy.
PURPOSE: This clinical trial is studying how well an autologous stem cell transplant works in treating patients with acute myeloid leukemia.
OBJECTIVES:
OUTLINE:
Chemotherapy and filgrastim (G-CSF) priming for PBSC collection: Patients receive cyclophosphamide IV on day 0; etoposide IV over 3 hours on days 0 and 1; and oral dexamethasone twice daily on days 0 and 1. Patients also receive G-CSF subcutaneously (SC) beginning on day 3 and continuing until apheresis is complete. After blood counts recover, apheresis is performed in 4-6 daily planned collections until the minimum CD34+ cell dose of > 2.5 x 10^6 cells/kg is achieved. If the minimum CD34+ cell dose is not achieved after 6 apheresis collections, patients undergo bone marrow examination including a bone marrow biopsy and aspiration, at the termination of the PBSC collection to confirm remission. If remission is confirmed, and if peripheral counts and marrow cellularity are sufficient, the patient remains off G-CSF for 7 days and receives sargramostim (GM-CSF) for 5 days to increase the marrow cellularity, after which a bone marrow harvest is performed.
Bone marrow harvest without prior PBSC collection: Children will undergo primed bone marrow harvest comprising GM-CSF IV or SC for 5 days prior to harvest to increase cellularity and then marrow is harvested. Marrow and blood specimens are also obtained with the initial bone marrow evaluation and at the time of harvest if a cytogenetic abnormality was previously described. Other patients who are unable to undergo PBSC collection may proceed with a bone harvest at the discretion of the protocol chairperson.
Cytoreductive regimen:
Stem cell transplantation: All patients undergo autologous PBSC and/or bone marrow infusion on day 0. Patients also receive G-CSF IV or SC beginning on day 1 and continuing until blood counts recover.
After completion of study treatment, patients are followed periodically for 5 years.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Bone Marrow Transplant (2-70 Years old) | Experimental | Patients over the age of two will receive a cytoreductive regimen of total-body irradiation and cyclophosphamide (TBI/CY) as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation). |
|
| Bone Marrow Transplant (less and 2 years old) | Experimental | Patients under the age of two, and patients who cannot receive total body irradiation (TBI), will receive a cytoreductive regimen of Busulfan and cyclophosphamide (BU/CY) as per the Johns Hopkins University Hospital regimen as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| sargramostim | Biological | Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Engraftment | Percentage of Participants with Engraftment measured by myeloid, platelet, and erythroid recovery | 30 Days Post Transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Disease Response | Disease evaluation will be completed approximately 100 days after stem cell infusion and every 6 months, 1 year, and until 2 years after infusion. | 2 years Post Transplant |
| Treatment Failure |
Not provided
Inclusion Criteria:
Children under the age of two are eligible for this protocol, but will not receive total body irradiation. Instead, children under the age of two will receive Busulfan/Cyclophosphamide (Bu/Cy) conditioning as the preparative regimen in order to obviate deleterious effects of radiation at this age. Patients who cannot receive total body irradiation (TBI) (for example those with prior radiation therapy) will also receive the Bu/CY conditioning.
Acute myeloid leukemia (AML)
Allogeneic transplant with an HLA-identical sibling will be recommended for patients <55 years. If the patient refuses allogeneic transplant, they may still be eligible for this protocol.
Exclusion Criteria:
Patients can also be deemed not eligible for transplant because of specific organ toxicity. Specifically, patients with pre-existing compromise to the heart, lungs, kidney, CNS or liver may be excluded:
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| Name | Affiliation | Role |
|---|---|---|
| Daniel J. Weisdorf, MD | Masonic Cancer Center, University of Minnesota | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Masonic Cancer Center, University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Bone Marrow Transplant (2-70 Years Old) | Patients over the age of two will receive a cytoreductive regimen of total-body irradiation and cyclophosphamide (TBI/CY) as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation). sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed cyclophosphamide: 4 gm/m^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2. dexamethasone: 20 mg/m^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours etoposide: 300 mg/m^2/day x 2 days (day 0-1) over 3 hours intravenously (IV) bone marrow transplantation: Day 0 infusion of bone marrow cells peripheral blood stem cell transplantation: Day 0 infusion of peripheral blood stem cells total-body irradiation: 165 cGy/dose given twice a day on days -7 through -4. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 31, 2018 | Oct 2, 2023 |
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|
| busulfan | Drug | 4 mg/kg po in 4 divided doses (.8 mg/kg/dose orally every 6 hours) on days -7 through -4. |
|
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| cyclophosphamide | Drug | 4 gm/m^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2. |
|
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| dexamethasone | Drug | 20 mg/m^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours |
|
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| etoposide | Drug | 300 mg/m^2/day x 2 days (day 0-1) over 3 hours intravenously (IV) |
|
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| bone marrow transplantation | Procedure | Day 0 infusion of bone marrow cells |
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| hematopoietic stem cell transplantation | Procedure | Stem cell infusion (>48 hours after the last dose of cyclophosphamide) |
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| peripheral blood stem cell transplantation | Procedure | Day 0 infusion of peripheral blood stem cells |
|
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| total-body irradiation | Radiation | 165 cGy/dose given twice a day on days -7 through -4. |
|
|
Percentage of participants experiencing treatment failure.
| 2 years Post Transplant |
| Percent of Patients With Various Late Effects | Description: (e.g., thyroid function abnormalities - T4, TSH, gonadal abnormalities, cataracts, pulmonary dysfunctions, growth and development abnormalities, and second malignant neoplasms) | 2 years Post Transplant |
| Disease-free Survival | Disease-free survival 2 years Post Transplant | 2 years Post Transplant |
| Percentage of Patients With Adequate Cells Collected | The proportion of primed patients with adequate number of cells collected will be calculated. | Pre-Transplant |
| FG001 | Bone Marrow Transplant (Less and 2 Years Old) | Patients under the age of two, and patients who cannot receive total body irradiation (TBI), will receive a cytoreductive regimen of Busulfan and cyclophosphamide (BU/CY) as per the Johns Hopkins University Hospital regimen as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation). sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed busulfan: 4 mg/kg po in 4 divided doses (.8 mg/kg/dose orally every 6 hours) on days -7 through -4. cyclophosphamide: 4 gm/m^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2. dexamethasone: 20 mg/m^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours etoposide: 300 mg/m^2/day x 2 days (day 0-1) over 3 hours intravenously (IV) hematopoietic stem cell transplantation: Stem cell infusion (>48 hours after the last dose of cyclophosphamide) |
| COMPLETED |
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| NOT COMPLETED |
|
No participants enrolled under the age of 2 years old.
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| ID | Title | Description |
|---|---|---|
| BG000 | Bone Marrow Transplant (2-70 Years Old) | Patients over the age of two will receive a cytoreductive regimen of total-body irradiation and cyclophosphamide (TBI/CY) as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation). sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed cyclophosphamide: 4 gm/m^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2. dexamethasone: 20 mg/m^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours etoposide: 300 mg/m^2/day x 2 days (day 0-1) over 3 hours intravenously (IV) bone marrow transplantation: Day 0 infusion of bone marrow cells peripheral blood stem cell transplantation: Day 0 infusion of peripheral blood stem cells total-body irradiation: 165 cGy/dose given twice a day on days -7 through -4. |
| BG001 | Bone Marrow Transplant (Less and 2 Years Old) | Patients under the age of two, and patients who cannot receive total body irradiation (TBI), will receive a cytoreductive regimen of Busulfan and cyclophosphamide (BU/CY) as per the Johns Hopkins University Hospital regimen as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation). sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed busulfan: 4 mg/kg po in 4 divided doses (.8 mg/kg/dose orally every 6 hours) on days -7 through -4. cyclophosphamide: 4 gm/m^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2. dexamethasone: 20 mg/m^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours etoposide: 300 mg/m^2/day x 2 days (day 0-1) over 3 hours intravenously (IV) hematopoietic stem cell transplantation: Stem cell infusion (>48 hours after the last dose of cyclophosphamide) |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | 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 | Percentage of Participants With Engraftment | Percentage of Participants with Engraftment measured by myeloid, platelet, and erythroid recovery | Posted | Number | 95% Confidence Interval | Participants of participants | 30 Days Post Transplant |
|
|
| ||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Disease Response | Disease evaluation will be completed approximately 100 days after stem cell infusion and every 6 months, 1 year, and until 2 years after infusion. | Posted | Number | 95% Confidence Interval | Percentage of participants | 2 years Post Transplant |
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| |||||||||||||||||||||||||||
| Secondary | Treatment Failure | Percentage of participants experiencing treatment failure. | Posted | Number | Percentage of participants | 2 years Post Transplant |
|
| ||||||||||||||||||||||||||||
| Secondary | Percent of Patients With Various Late Effects | Description: (e.g., thyroid function abnormalities - T4, TSH, gonadal abnormalities, cataracts, pulmonary dysfunctions, growth and development abnormalities, and second malignant neoplasms) | Posted | Count of Participants | Participants | 2 years Post Transplant |
|
| ||||||||||||||||||||||||||||
| Secondary | Disease-free Survival | Disease-free survival 2 years Post Transplant | Posted | Number | 95% Confidence Interval | Percentage of participants with DFS | 2 years Post Transplant |
|
| |||||||||||||||||||||||||||
| Secondary | Percentage of Patients With Adequate Cells Collected | The proportion of primed patients with adequate number of cells collected will be calculated. | Posted | Number | 95% Confidence Interval | Percentage of participants | Pre-Transplant |
|
|
2 years
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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 | Bone Marrow Transplant (2-70 Years Old) | Patients over the age of two will receive a cytoreductive regimen of total-body irradiation and cyclophosphamide (TBI/CY) as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation). sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed cyclophosphamide: 4 gm/m^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2. dexamethasone: 20 mg/m^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours etoposide: 300 mg/m^2/day x 2 days (day 0-1) over 3 hours intravenously (IV) bone marrow transplantation: Day 0 infusion of bone marrow cells peripheral blood stem cell transplantation: Day 0 infusion of peripheral blood stem cells total-body irradiation: 165 cGy/dose given twice a day on days -7 through -4. | 0 | 12 | 1 | 12 | 0 | 12 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Relapse | Blood and lymphatic system disorders | Systematic Assessment | Relapse of acute promyelocytic leukaemia (APL) |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Daniel Weisdorf, MD | University of Minnesota, Masonic Cancer Center | +1 612-624-3101 | weisd001@umn.edu |
| Prot_000.pdf |
| ICF | No | No | Yes | Informed Consent Form | Nov 8, 2018 | Aug 15, 2023 | ICF_001.pdf |
| ID | Term |
|---|---|
| D007938 | Leukemia |
| D000013 | Congenital Abnormalities |
| ID | Term |
|---|---|
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C081222 | sargramostim |
| D016179 | Granulocyte Colony-Stimulating Factor |
| D002066 | Busulfan |
| D003520 | Cyclophosphamide |
| D003907 | Dexamethasone |
| D002123 | Calcium Dobesilate |
| D005047 | Etoposide |
| D016026 | Bone Marrow Transplantation |
| D018380 | Hematopoietic Stem Cell Transplantation |
| D036102 | Peripheral Blood Stem Cell Transplantation |
| D014916 | Whole-Body Irradiation |
| ID | Term |
|---|---|
| D003115 | Colony-Stimulating Factors |
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D016298 | Hematopoietic Cell Growth Factors |
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |
| 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 |
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D011246 | Pregnadienetriols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D013259 | Steroids, Fluorinated |
| D001557 | Benzenesulfonates |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D001190 | Arylsulfonates |
| D017739 | Arylsulfonic Acids |
| D011034 | Podophyllotoxin |
| D013764 | Tetrahydronaphthalenes |
| D009281 | Naphthalenes |
| D011084 | Polycyclic Aromatic Hydrocarbons |
| D005960 | Glucosides |
| D006027 | Glycosides |
| D016378 | Tissue Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |
| D033581 | Stem Cell Transplantation |
| D017690 | Cell Transplantation |
| D011878 | Radiotherapy |
| D008919 | Investigative Techniques |
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| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
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