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| ID | Type | Description | Link |
|---|---|---|---|
| MT2003-18 | Other Identifier | Blood and Marrow Transplantation Program |
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Treatment with thymic shielding found safe, another study started.
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The purpose of this study is to determine whether thymic shielding during total body irradiation can be given and whether it will reduce the risk of infections in Fanconi Anemia patients undergoing alternate donor (not a matched sibling) stem cell transplants.
All subjects will be given the same treatment regimen of total body irradiation (TBI), Fludarabine, Cyclophosphamide, and anti-thymocyte globulin (ATG), followed by an alternate donor stem cell transplant. Since this treatment regimen has been given before, without thymic shielding, we will compare the outcomes of these patients with the historical data from subjects who did not receive thymic shielding.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| HSCT Patients | Experimental | Patients who received total body irradiation (450 cGy [centigray]) with thymic shielding prior to chemotherapy regimen and Hematopoietic Stem Cell Transplant (HSCT) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hematopoietic Stem Cell Transplant | Procedure | Bone marrow failure may be treated by giving patients stem cells that come from someone else. This is called a stem-cell transplant. As part of the transplant process, patients receive high doses of chemotherapy and/or radiation to treat their underlying disease. As one of its effects, this treatment also kills the healthy stem cells that are already in the marrow. The transplant provides new stem cells for the patient from a healthy donor; that replace the bone marrow and allow the blood counts to recover. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients Who Exhibited Hematopoietic Recovery and Engraftment | Calculated from Day 1 of hematopoietic cell transplant to Day 42 post-transplant. Hematopoietic recovery and engraftment is defined as the first of three consecutive days the patient's absolute neutrophil count is greater than or equal to 0.5X10^9/Liter. | Day 42 after hematopoietic cell transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients Who Exhibited Secondary Graft Failure | Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. A complication after Bone Marrow Transplant in which the transplanted stem cells do not grow in the recipient's bone marrow and thus do not produce new blood cells. | Day 100 after hematopoietic cell transplant |
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Inclusion Criteria:
Patients must be less than (<) 18 years of age with a diagnosis of Fanconi anemia.
Patients must have an HLA-A, B, DRB1 identical unrelated donor or less than or equal to (≤)1 antigen mismatched related (non-HLA-matched sibling) or <1 antigen mismatched unrelated UCB donor. Patients and donors will be typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing.
Patients with FA must have aplastic anemia (AA), myelodysplastic syndrome without excess blasts, or high risk genotype as defined below.
Adequate major organ function including
Women of child-bearing age must be using adequate birth control and have a negative pregnancy test
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Margaret MacMillan, 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 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 25824692 | Derived | MacMillan ML, DeFor TE, Young JA, Dusenbery KE, Blazar BR, Slungaard A, Zierhut H, Weisdorf DJ, Wagner JE. Alternative donor hematopoietic cell transplantation for Fanconi anemia. Blood. 2015 Jun 11;125(24):3798-804. doi: 10.1182/blood-2015-02-626002. Epub 2015 Mar 30. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Intent-To-Treat | All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy). |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Intent-To-Treat | All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy). |
| 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 |
| 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 Exhibited Hematopoietic Recovery and Engraftment | Calculated from Day 1 of hematopoietic cell transplant to Day 42 post-transplant. Hematopoietic recovery and engraftment is defined as the first of three consecutive days the patient's absolute neutrophil count is greater than or equal to 0.5X10^9/Liter. | Posted | Jun 2009 | Number | Participants | Day 42 after hematopoietic cell transplant |
|
|
Start of study through 1 year after transplant.
Adverse events were not collected for this study population as it is not routinely done. Graft versus Host Disease and treatment related toxicity is reported in the secondary outcome measure section.
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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 | Intent-To-Treat | All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy). |
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Study ended early as thymic shielding was found to be safe. Therefore, a new study was designed using thymic shielding.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Margaret MacMillan, M.D. | Masonic Cancer Center, University of Minnesota | 612-626-2778 | macmi002@umn.edu |
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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 |
|---|---|
| D033581 | Stem Cell Transplantation |
| D016026 | Bone Marrow Transplantation |
| D014916 | Whole-Body Irradiation |
| D011878 | Radiotherapy |
| C095424 | CF regimen |
| D003520 | Cyclophosphamide |
| C042382 | fludarabine phosphate |
| ID | Term |
|---|---|
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
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|
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| Thymic Shielding During Radiation | Procedure | protecting the thymus during total body radiation (450 cGy administered) |
|
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| Total Body Irradiation | Procedure | Six days before the stem cells are given (day -6), subjects will receive total body irradiation with thymic shielding. Thymic shielding is done by placing a piece of lead on the chest during the irradiation treatment so that the irradiation beams do not go to the thymus. |
|
|
| Cyclophosphamide, Fludarabine | Drug | Day -5 through Day -2, subjects will receive a chemotherapy regimen of Fludarabine, Cyclophosphamide via central line |
|
|
| Number of Patients With Acute Graft Versus-Host Disease (aGVHD) | Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack. | Day 100 after hematopoietic cell transplant |
| Number of Patients With Chronic Graft Versus-Host Disease (GVHD) | Calculated from Day 1 of hematopoietic cell transplant to 1 year after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack. | 1 year after hematopoietic cell transplant |
| Number of Patients Who Exhibited Regimen-related Toxicity (RRT) | Calculated from Day 1 of hematopoietic cell transplant to 1 year after transplant. Regimen-related toxicity involves harmful effects in an organism through exposure to the treatment given. | 1 year after hematopoietic cell transplant |
| Immune Reconstitution - Mean Value (1 Year) | Calculated mean value of patient CD4 values collected at intervals from Day 30 through 1 year post-transplant. | 1 year post-transplant. |
| Immune Reconstitution - Mean Value (2 Years) | Calculated mean value of patient CD4 values collected at intervals from Day 30 through 2 years post-transplant. | at 2 years after transplant |
| Number of Patients Alive at 1 Year | Calculated from Day 1 of hematopoietic cell transplant to 1 year post-transplant. | 1 year after transplant |
| Number of Patients Alive at 2 Years | Calculated from Day 1 of hematopoietic cell transplant to 2 years post-transplant. | 2 years after transplant |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
|
| Secondary | Number of Patients Who Exhibited Secondary Graft Failure | Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. A complication after Bone Marrow Transplant in which the transplanted stem cells do not grow in the recipient's bone marrow and thus do not produce new blood cells. | Posted | Jun 2009 | Number | Participants | Day 100 after hematopoietic cell transplant |
|
|
|
| Secondary | Number of Patients With Acute Graft Versus-Host Disease (aGVHD) | Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack. | Posted | Jun 2009 | Number | Participants | Day 100 after hematopoietic cell transplant |
|
|
|
| Secondary | Number of Patients With Chronic Graft Versus-Host Disease (GVHD) | Calculated from Day 1 of hematopoietic cell transplant to 1 year after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack. | Posted | Jun 2009 | Number | Participants | 1 year after hematopoietic cell transplant |
|
|
|
| Secondary | Number of Patients Who Exhibited Regimen-related Toxicity (RRT) | Calculated from Day 1 of hematopoietic cell transplant to 1 year after transplant. Regimen-related toxicity involves harmful effects in an organism through exposure to the treatment given. | Posted | Jun 2009 | Number | Participants | 1 year after hematopoietic cell transplant |
|
|
|
| Secondary | Immune Reconstitution - Mean Value (1 Year) | Calculated mean value of patient CD4 values collected at intervals from Day 30 through 1 year post-transplant. | Posted | Jun 2009 | Mean | Standard Deviation | Number of CD4 cells per microliter | 1 year post-transplant. |
|
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| Secondary | Immune Reconstitution - Mean Value (2 Years) | Calculated mean value of patient CD4 values collected at intervals from Day 30 through 2 years post-transplant. | Posted | Jun 2009 | Mean | Standard Deviation | Number of CD4 cells per microliter | at 2 years after transplant |
|
|
|
| Secondary | Number of Patients Alive at 1 Year | Calculated from Day 1 of hematopoietic cell transplant to 1 year post-transplant. | Posted | Jun 2009 | Number | Participants | 1 year after transplant |
|
|
|
| Secondary | Number of Patients Alive at 2 Years | Calculated from Day 1 of hematopoietic cell transplant to 2 years post-transplant. | Posted | Jun 2009 | Number | Participants | 2 years after transplant |
|
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| 0 |
| 0 |
| 0 |
| 0 |
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| 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 |
| D014180 |
| Transplantation |
| D013514 | Surgical Procedures, Operative |
| D016378 | Tissue Transplantation |
| D008919 | Investigative Techniques |
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |