The Children's Hospital University of Illinois
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Northwestern University - Ann and Robert H. Lurie Children's Hospital of Chicago
Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant (ASCENT)
The ASCENT Trial is a single arm, multi-center, phase II study. The primary objective is to determine the rejection-free, severe graft-versus-host disease (GVHD)-free survival in pediatric patients with serious non-malignant hematologic diseases (NMHD) undergoing mismatched unrelated donor (URD) hematopoietic stem cell transplantation (HSCT) with abatacept added to conventional GVHD prophylaxis. The secondary objective is to characterize the impact of abatacept on infection and the reconstitution of protective immunity to infection. Transplanted patients will be followed for 3 years. Weight-based peripheral blood samples will be drawn longitudinally through two years to evaluate immune reconstitution. The study will enroll 28 pediatric patients with serious NMHD undergoing mismatched URD HSCT. The trial will include two strata, based on donor matching. Stratum 1 (n=14) will be for patients with 7/8 donors and stratum 2 (n=14) will be for those with 8/8 (matched) donors. All participants will receive 8 doses of abatacept (10 mg/kg intravenously on days -1, +5, +14, +28, +56, +84, +112, and +150). Recruitment is expected to last for about 2 years and participants will be followed for up to 3 years. This trial will test the hypothesis that extended abatacept administration (combined with a standard regimen of tacrolimus and mycophenolate mofetil) will effectively prevent acute and chronic GVHD in children and adolescents receiving mismatched URD HSCT, without compromising their engraftment or reconstitution of protective immunity to infection.
September 1, 2020- More Fanconi Anemia – Northwestern University - Ann and Robert H. Lurie Children's Hospital of Chicago posts
Medical College of Wisconsin - Children's Hospital of Wisconsin
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Rush University Children's Hospital
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University of Chicago - Comer Children's Hospital
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University of Iowa - Stead Family Children's Hospital
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University of Minnesota - Masonic Children's Hospital
T Cell Receptor α/β TCD HCT in Patients With Fanconi Anemia
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) hematopoietic cell transplantation (HCT) transplantation in patients with Fanconi anemia (FA) to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.
September 1, 2020Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
RATIONALE: A bone marrow or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Giving combination chemotherapy before a donor stem cell transplant may make the transplant more likely to work. This may be an effective treatment for patients with high risk Fanconi's anemia. PURPOSE: This clinical trial is studying how well combination chemotherapy works in treating high risk patients who are undergoing a donor stem cell transplant for Fanconi's anemia.
September 1, 2020Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia
RATIONALE: Giving chemotherapy, such as cyclophosphamide and fludarabine, before a donor stem cell transplant helps to remove the patient's cells to allow for the transplant cells to take and grow. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells can make an immune response against the body's normal cells. Giving antithymocyte globulin and removing the T cells from the donor cells before transplant and giving cyclosporine before and after transplant may stop this from happening. PURPOSE: This phase I/II trial is studying the side effects of cyclophosphamide, fludarabine, and antithymocyte globulin followed by donor stem cell transplant and to see how well it works in treating patients with Fanconi anemia.
September 1, 2020TBI Dose De-escalation for Fanconi Anemia
This is a single arm, total body irradiation (TBI) trial. All patients will be prescribed TBI 300 cGy with the goal of evaluating secondary endpoints.
September 1, 2020- More Fanconi Anemia - University of Minnesota - Masonic Children's Hospital posts
University of Wisconsin - American Family Children's Hospital
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