| ID | Type | Description | Link |
|---|---|---|---|
| 0206M26241 | Other Identifier | IRB, University of Minnesota |
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| Name | Class |
|---|---|
| National Marrow Donor Program | OTHER |
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This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies. The researchers hypothesize that these regimens will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease.
Regimen A2 has replaced Regimen A in this study. Two patients were treated on Regimen A but did not have evidence of initial engraftment thus triggering the stopping rule for that arm of this study.
Prior to transplantation, subjects will receive either:
Cyclophosphamide, Fludarabine, Campath, Total body irradiation (TBI)
Or
Busulfan, Cyclophosphamide, antithymocyte globulin (ATG), granulocyte colony-stimulating factor (GSCF)
These drugs (and the radiation) are being given to help the new stem cells take and grow. On the day of transplantation, subjects will receive stem cells transfused via intravenous (IV) catheter.
After stem cell transplantation, subjects will be given cyclosporine-A and mycophenolate (MMF)/or Methylprednisone/or Methotrexate to reduce the risk of graft-versus-host disease, the complication that occurs when the donor's stem cells react against the patient.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| RIC Bu/Flu (A) (discontinued) | Other | Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0. |
|
| MA Bu/Cy (B) | Experimental | Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days. |
|
| RIC Cy/Flu/TBI (A2) | Experimental | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Busulfan, Fludarabine, ATG, TLI | Drug | Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity | In general, grade 3 equates to moderate, grade 4 to severe and grade 5 to death. | 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| The Incidence of Chimerism at 100 Days | The number of patients whose blood and/or bone marrow contains > 10% donor cells. | 100 days |
| The Incidence of Chimerism at 6 Months | The number of patients whose blood and/or bone marrow contains > 10% donor cells. |
Not provided
Inclusion Criteria:
Patients with Sickle Cell Disease/Thalassemia (SCD/THAL) 0-50 years of age with an acceptable stem cell donor and disease characteristic defined by the following:
Patients with transfusion dependent alpha- or beta-thalassemia 0-35 years of age with an acceptable stem cell donor as defined in the criteria in section above.
Patients with other non-malignant hematologic disorders that are transfusion-dependent or involve other potentially life-threatening cytopenias (including but not limited to Severe Congenital Neutropenia, Diamond-Blackfan Anemia and Shwachman-Diamond Syndrome) who are 0-35 years of age with an acceptable stem cell donor
Second Transplants
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Angela Smith, 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 | Full Conditioning (Discontinued / A) | Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0. Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| Busulfan, Cyclophosphamide, ATG, GCSF | Drug | Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC >2500 x 2 days. |
|
|
| Campath, Fludarabine, Cyclophosphamide | Drug | Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. |
|
|
| Total Body Irradiation | Radiation | 300 cGY Day -1 |
|
|
| Stem cell infusion | Procedure | Given Day 0 |
|
|
| 6 months |
| The Incidence of Chimerism at 1 Year | The number of patients whose blood and/or bone marrow contains > 10% donor cells. | 1 year |
| The Incidence of Grade 2-4 Acute Graft Versus Host Disease (Acute GVHD) | The number of patients who experienced grades 2-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Grades 2-4 equate to mild to severe disease. Symptoms typically appear within weeks after transplant. | 100 days |
| The Incidence of Grade 3-4 Acute Graft Versus Host Disease (Acute GVHD) | The number of patients who experienced grades 3-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. IGrades 3-4 equate to moderate to severe disease. Symptoms typically appear within weeks after transplant. | 100 days |
| The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD) | The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant. | 6 months |
| The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD) | The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant. | 1 year |
| Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment | The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death. | pre-transplant |
| Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment | The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death. | 1 year |
| Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment | The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death. | 2 years |
| Determine Physical Characteristics and Biologic Effects of Mixed Populations of Donor and Host Red Blood Cells | During study |
| Determine the Concentration of Campath in the Serum | Day 0 |
| Overall Survival | Number of patients alive 100 days after transplant. | 100 days |
| Overall Survival | Number of patients alive 1 year after transplant. | 1 year |
| Disease Free Survival | Number of patients alive without disease 100 days after transplant. | 100 days |
| Disease Free Survival | Number of patients alive without disease 1 year after transplant. | 1 year |
| FG001 | Busulfan Conditioning (B) | Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days. Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC >2500 x 2 days. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
| FG002 | Campath and TBI Conditioning (A2) | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
| COMPLETED |
|
| NOT COMPLETED |
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Full Conditioning (Discontinued / A) | Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0. Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy |
| BG001 | Busulfan Conditioning (B) | Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days. Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC >2500 x 2 days. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
| BG002 | Campath and TBI Conditioning (A2) | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
| BG003 | 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 |
|
| 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 | Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity | In general, grade 3 equates to moderate, grade 4 to severe and grade 5 to death. | Posted | Count of Participants | Participants | 1 year |
|
|
| |||||||||||||||||||||||||||||||||
| Secondary | The Incidence of Chimerism at 100 Days | The number of patients whose blood and/or bone marrow contains > 10% donor cells. | One of the 3 patients treated on Arm A was retreated at Day 40 and was not evaluable. One of the 14 patients on Arm A2 died before 100 days. | Posted | Count of Participants | Participants | 100 days |
| ||||||||||||||||||||||||||||||||||
| Secondary | The Incidence of Chimerism at 6 Months | The number of patients whose blood and/or bone marrow contains > 10% donor cells. | One of the 3 patients treated on Arm A was retreated at Day 40 and was not evaluable. One of the 14 patients on Arm A2 died before 6 months. | Posted | Count of Participants | Participants | 6 months |
| ||||||||||||||||||||||||||||||||||
| Secondary | The Incidence of Chimerism at 1 Year | The number of patients whose blood and/or bone marrow contains > 10% donor cells. | One of the 3 patients treated on Arm A was retreated at Day 40 and was not evaluable. One of the 14 patients on Arm A2 died before 1 year. | Posted | Count of Participants | Participants | 1 year |
| ||||||||||||||||||||||||||||||||||
| Secondary | The Incidence of Grade 2-4 Acute Graft Versus Host Disease (Acute GVHD) | The number of patients who experienced grades 2-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Grades 2-4 equate to mild to severe disease. Symptoms typically appear within weeks after transplant. | Posted | Count of Participants | Participants | 100 days |
| |||||||||||||||||||||||||||||||||||
| Secondary | The Incidence of Grade 3-4 Acute Graft Versus Host Disease (Acute GVHD) | The number of patients who experienced grades 3-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. IGrades 3-4 equate to moderate to severe disease. Symptoms typically appear within weeks after transplant. | Posted | Count of Participants | Participants | 100 days |
| |||||||||||||||||||||||||||||||||||
| Secondary | The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD) | The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant. | Posted | Count of Participants | Participants | 6 months |
| |||||||||||||||||||||||||||||||||||
| Secondary | The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD) | The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant. | Posted | Count of Participants | Participants | 1 year |
| |||||||||||||||||||||||||||||||||||
| Secondary | Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment | The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death. | Posted | Median | Full Range | units on a scale | pre-transplant |
| ||||||||||||||||||||||||||||||||||
| Secondary | Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment | The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death. | Two of the 14 patients treated on Arm A2 died before 1 year. | Posted | Median | Full Range | units on a scale | 1 year |
| |||||||||||||||||||||||||||||||||
| Secondary | Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment | The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death. | Two of the 14 patients treated on Arm A2 died before 2 years and 2 failed their 2 year clinic appointment. | Posted | Median | Full Range | units on a scale | 2 years |
| |||||||||||||||||||||||||||||||||
| Secondary | Determine Physical Characteristics and Biologic Effects of Mixed Populations of Donor and Host Red Blood Cells | data were not collected | Posted | During study |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Determine the Concentration of Campath in the Serum | The Principal Investigator removed this as a study objective and therefore Campath concentrations were not collected. | Posted | Day 0 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Overall Survival | Number of patients alive 100 days after transplant. | Posted | Count of Participants | Participants | 100 days |
| |||||||||||||||||||||||||||||||||||
| Secondary | Overall Survival | Number of patients alive 1 year after transplant. | Posted | Count of Participants | Participants | 1 year |
| |||||||||||||||||||||||||||||||||||
| Secondary | Disease Free Survival | Number of patients alive without disease 100 days after transplant. | Posted | Count of Participants | Participants | 100 days |
| |||||||||||||||||||||||||||||||||||
| Secondary | Disease Free Survival | Number of patients alive without disease 1 year after transplant. | Posted | Count of Participants | Participants | 1 year |
|
Not provided
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Full Conditioning (Discontinued / A) | Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0. Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy | 2 | 3 | 3 | 3 | ||
| EG001 | Busulfan Conditioning (B) | Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days. Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC >2500 x 2 days. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 | 0 | 5 | 5 | 5 | ||
| EG002 | Campath and TBI Conditioning (A2) | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 | 0 | 14 | 13 | 14 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Primary Graft Failure | General disorders |
| |||
| Secondary Graft Failure | General disorders |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bacterial Infection, Blood | Infections and infestations |
| |||
| Bacterial Infection, Pulmonary | Infections and infestations |
| |||
| Conjunctival Hemorrhage | Eye disorders |
| |||
| Delayed Growth/Stature | Musculoskeletal and connective tissue disorders |
| |||
| Fungal Infection, Gastrointestinal | Infections and infestations |
| |||
| Hypertension | Vascular disorders |
| |||
| Hypothyroidism | Endocrine disorders |
| |||
| Pericardial Effusion | Cardiac disorders |
| |||
| Pneumonia | Infections and infestations |
| |||
| Renal Failure | Renal and urinary disorders |
| |||
| Respiratory Failure | Respiratory, thoracic and mediastinal disorders |
| |||
| Second Malignancy | Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
| |||
| Seizure | Nervous system disorders |
| |||
| Veno-Occlusive Disease | Hepatobiliary disorders |
| |||
| Viral Infection, Blood | Infections and infestations |
| |||
| Viral Infection, Pulmonary | Infections and infestations |
| |||
| Cystitis | Renal and urinary disorders |
| |||
| Elevated Liver Function Tests | Investigations |
| |||
| Engraftment Syndrome | Immune system disorders |
| |||
| Esophageal Varices | Gastrointestinal disorders |
| |||
| Fungal Infection, Blood | Infections and infestations |
| |||
| Fungal Infection, Genitourinary | Infections and infestations |
| |||
| Fungal Infection, Pulmonary | Infections and infestations |
| |||
| Hemochromatosis | Metabolism and nutrition disorders |
| |||
| Hepatitis | Investigations |
| |||
| Hypogonadism | Endocrine disorders |
| |||
| Intraventricular Septal Hypertrophy | Cardiac disorders |
| |||
| Joint Dysfunction | Musculoskeletal and connective tissue disorders |
| |||
| Neuro Toxicity, NOS | Nervous system disorders |
| |||
| Neuropathy | Nervous system disorders |
| |||
| Otitis Media | Infections and infestations |
| |||
| Paronychia of Bilateral Great Toes | Infections and infestations |
| |||
| Polycythemia | Blood and lymphatic system disorders |
| |||
| Polymorphic Post-Transplant Lymphoproliferative Disorder | Blood and lymphatic system disorders |
| |||
| Sepsis | Infections and infestations |
| |||
| Subarachnoid Hemorrhage | Nervous system disorders |
| |||
| Thrombus, Right Atrium | Vascular disorders |
| |||
| Upper Respiratory Stridor | Reproductive system and breast disorders |
| |||
| Viral Infection, Gastrointestinal | Infections and infestations |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Angela Smith | Masonic Cancer Center, University of Minnesota | 612-626-2778 | smith719@umn.edu |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D013789 | Thalassemia |
| C537592 | Neutropenia, Severe Congenital, Autosomal Recessive 3 |
| D029503 | Anemia, Diamond-Blackfan |
| D000081003 | Shwachman-Diamond Syndrome |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D029502 | Anemia, Hypoplastic, Congenital |
| D000741 | Anemia, Aplastic |
| D012010 | Red-Cell Aplasia, Pure |
| D000080984 | Congenital Bone Marrow Failure Syndromes |
| D000080983 | Bone Marrow Failure Disorders |
| D001855 | Bone Marrow Diseases |
| D010188 | Exocrine Pancreatic Insufficiency |
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008052 | Lipid Metabolism, Inborn Errors |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D008068 | Lipomatosis |
Not provided
Not provided
| ID | Term |
|---|---|
| D002066 | Busulfan |
| C024352 | fludarabine |
| C042382 | fludarabine phosphate |
| D003520 | Cyclophosphamide |
| D000961 | Antilymphocyte Serum |
| D016179 | Granulocyte Colony-Stimulating Factor |
| D000074323 | Alemtuzumab |
| D014916 | Whole-Body Irradiation |
| D016026 | Bone Marrow Transplantation |
| 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 |
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D007106 | Immune Sera |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| 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 |
| D001685 | Biological Factors |
| D061067 | Antibodies, Monoclonal, Humanized |
| D000911 | Antibodies, Monoclonal |
| D011878 | Radiotherapy |
| D013812 | Therapeutics |
| D008919 | Investigative Techniques |
| D016378 | Tissue Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |
Not provided
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
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| OG002 | RIC Cy/Flu/TBI (A2) | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
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| OG002 | RIC Cy/Flu/TBI (A2) | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
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| OG002 | RIC Cy/Flu/TBI (A2) | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
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| OG002 | RIC Cy/Flu/TBI (A2) | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
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| OG002 | RIC Cy/Flu/TBI (A2) | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
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| OG002 | RIC Cy/Flu/TBI (A2) | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
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| OG002 | RIC Cy/Flu/TBI (A2) | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
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| OG002 | RIC Cy/Flu/TBI (A2) | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
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| OG002 | RIC Cy/Flu/TBI (A2) | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
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| OG002 | RIC Cy/Flu/TBI (A2) | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
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Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
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Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
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Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
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Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
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Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. Total Body Irradiation: 300 cGY Day -1 Stem cell infusion: Given Day 0 |
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Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.
Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body Irradiation: 300 cGY Day -1
Stem cell infusion: Given Day 0
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