A Phase 1/2 interventional study of Hematopoietic Stem Cell Transplant and Thymic Shielding During Radiation in Fanconi Anemia, sponsored by Masonic Cancer Center, University of Minnesota. Terminated at 1 site in United States. Open to participants aged 1 Day to 18 Years. Per ClinicalTrials.gov, last updated 2019-08-26.
Sponsored by Masonic Cancer Center, University of Minnesota · Phase 1/2, Interventional, and Treatment
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.
72 studies on the registry are indexed under Fanconi Syndrome; 11 are open to participants now.
This study's enrollment of 16 is above the median of 12 across 44 interventional studies indexed under Fanconi Syndrome.
Browse Fanconi Syndrome studies →Masonic Cancer Center, University of Minnesota is the lead sponsor of 284 studies on the registry; 34 are open to participants now.
Of its 39 completed or terminated interventional studies of FDA-regulated products, 28 (72%) have results posted.
Counted across the registry records on this site, refreshed daily.
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
Exclusion Criteria:
Patients who received total body irradiation (450 cGy \[centigray\]) with thymic shielding prior to chemotherapy regimen and Hematopoietic Stem Cell Transplant (HSCT)
Procedure: Hematopoietic Stem Cell Transplant · Procedure: Thymic Shielding During Radiation · Procedure: Total Body Irradiation · Drug: Cyclophosphamide, Fludarabine
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.
Also known as: Bone Marrow Transplant
protecting the thymus during total body radiation (450 cGy administered)
Also known as: TBI
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.
Also known as: Radiation Therapy, Therapuetic Radiation
Day -5 through Day -2, subjects will receive a chemotherapy regimen of Fludarabine, Cyclophosphamide via central line
Also known as: Cytoxan, Fludara
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.
Time frame: Day 42 after hematopoietic cell transplant
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.
Time frame: Day 100 after hematopoietic cell transplant
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 1 year after transplant
Number of Patients Alive at 2 Years
Calculated from Day 1 of hematopoietic cell transplant to 2 years post-transplant.
Time frame: 2 years after transplant
| Milestone | Intent-To-Treat |
|---|---|
| Started | 16 |
| Completed | 16 |
| Not completed | 0 |
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.
| Participants | Intent-To-Treat |
|---|---|
| Number of Patients Who Exhibited Hematopoietic Recovery and Engraftment | 15 |
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.
| Participants | Intent-To-Treat |
|---|---|
| Number of Patients Who Exhibited Secondary Graft Failure | 1 |
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.
| Participants | Intent-To-Treat |
|---|---|
| Number of Patients With Acute Graft Versus-Host Disease (aGVHD) | 8 |
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.
| Participants | Intent-To-Treat |
|---|---|
| Number of Patients With Chronic Graft Versus-Host Disease (GVHD) | 2 |
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.
| Participants | Intent-To-Treat |
|---|---|
| Number of Patients Who Exhibited Regimen-related Toxicity (RRT) | 5 |
Calculated mean value of patient CD4 values collected at intervals from Day 30 through 1 year post-transplant.
| Number of CD4 cells per microliter | Intent-To-Treat |
|---|---|
| Immune Reconstitution - Mean Value (1 Year) | 860 ± 870 |
Calculated mean value of patient CD4 values collected at intervals from Day 30 through 2 years post-transplant.
| Number of CD4 cells per microliter | Intent-To-Treat |
|---|---|
| Immune Reconstitution - Mean Value (2 Years) | 1100 ± 510 |
Calculated from Day 1 of hematopoietic cell transplant to 1 year post-transplant.
| Participants | Intent-To-Treat |
|---|---|
| Number of Patients Alive at 1 Year | 11 |
Calculated from Day 1 of hematopoietic cell transplant to 2 years post-transplant.
| Participants | Intent-To-Treat |
|---|---|
| Number of Patients Alive at 2 Years | 10 |
Collected over Start of study through 1 year after transplant.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Intent-To-Treat | — | — | — |
| Age, Categorical(Participants) | Intent-To-Treat |
|---|---|
| <=18 years | 16 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Age, Continuous(years) | Intent-To-Treat |
|---|---|
| Mean | 9.8 ± 4.5 |
| Sex: Female, Male(Participants) | Intent-To-Treat |
|---|---|
| Female | 10 |
| Male | 6 |
| Region of Enrollment(participants) | Intent-To-Treat |
|---|---|
| United States | 16 |
This study is terminated, as verified in Aug 2019. You cannot join it, but the record below documents what was studied.
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Masonic Cancer Center, University of Minnesota