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| Name | Class |
|---|---|
| Memorial Sloan Kettering Cancer Center | OTHER |
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The trial proposed is a single arm phase II treatment protocol designed to examine engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival following a novel cytoreductive regimen including busulfan, cyclophosphamide and fludarabine and anti-thymocyte globulin (ATG- a non-chemotherapy drug whose role is to kill your immune system) for the treatment of patients with Fanconi anemia who have severe aplastic anemia (SAA), or myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML), lacking HLA-genotypically identical donors using stem cell transplants derived from (1) HLA-compatible unrelated donors or (2) HLA haplotype-mismatched related donors.
We are currently recruiting patients.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Bone marrow processing | Experimental | Bone Marrow processing using the CliniMACs device |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CliniMACs device | Device | Donor Peripheral blood progenitor cells will use CD34+ selection with the use of the CliniMACs device |
|
| Measure | Description | Time Frame |
|---|---|---|
| To measure the incidence and quality of engraftment and hematopoietic reconstitution. | To measure the incidence and quality of engraftment and hematopoietic reconstitution. | 1, 3, 6 and 12 months post transplant date |
| Measure | Description | Time Frame |
|---|---|---|
| The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD | The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD | weekly for the first 30 days and then 3, 6, and 12 months post transplant date |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| David A Margolis, MD | Medical College of Wisconsin | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
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| ID | Term |
|---|---|
| D005199 | Fanconi Anemia |
| ID | Term |
|---|---|
| D029502 | Anemia, Hypoplastic, Congenital |
| D000741 | Anemia, Aplastic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D002066 | Busulfan |
| C024352 | fludarabine |
| D003520 | Cyclophosphamide |
| ID | Term |
|---|---|
| D002072 | Butylene Glycols |
| D006018 | Glycols |
| D000438 | Alcohols |
| D009930 | Organic Chemicals |
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| Busulfan | Drug | Chemotherapy administered as a part of the HSCT conditioning regimen. |
|
| Fludarabine | Drug | Chemotherapy administered as a part of the HSCT conditioning regimen. |
|
| Cyclophosphamide | Drug | Chemotherapy administered as a part of the HSCT conditioning regimen. |
|
| ATG | Drug | Chemotherapy administered as a part of the HSCT conditioning regimen. |
|
| D006425 |
| Hemic and Lymphatic Diseases |
| D000080984 | Congenital Bone Marrow Failure Syndromes |
| D000080983 | Bone Marrow Failure Disorders |
| D001855 | Bone Marrow Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D049914 | DNA Repair-Deficiency Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| 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 |