Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases

MI-összefoglaló

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The trial is looking for young children with a non-cancer blood or immune disease who need a donor stem cell transplant. It tests a donor graft with certain T cells removed to lower the risk of graft versus host disease (GVHD).

Életkor

The criteria say 6 months to 5 years (inclusive) at consent. The registry age fields say 6 Months to 50 Years.

Nem

All sexes can join.

A betegség stádiuma

A non-cancer disease treatable by donor stem cell transplant: bone marrow failure, hemoglobinopathies, primary immune deficiency, autoimmune cytopenias, immune dysregulation, HLH, or another such disease approved by the study lead. The person must be able to have a transplant after low dose (4Gy) total-body irradiation conditioning. A donor fit for the transplant is needed.

Korábbi kezelés

Cannot be on other experimental studies for preventing GVHD.

A részvétel fő kizáró okai

• Aplastic anemia, severe combined immunodeficiency (SCID), Fanconi anemia or dyskeratosis congenita
• Weight over 100 kg (220 lb), or HIV-1 or HIV-2 positive
• A current cancer or uncontrolled infection that rules out transplant
• Poor kidney, heart, lung or liver function
• Pregnant or breast-feeding, or known allergy to tacrolimus or MMF

A regiszter feltételei, szó szerint

Mit akar kideríteni a vizsgálat

This phase II trial investigates how well a naive T cell depleted graft work for the reduction of graft versus host disease in patients with non-malignant diseases requiring hematopoietic cell transplantation. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also 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. The donated stem cells may also replace the patient's immune cells and help destroy any remaining cancer cells.

A vizsgálat 2 csoportja

• Arm A (HLA-Haploidentical or mismatched unrelated donor)Egyéb csoport

  Conditioning regimen for HLA-Haploidentical or mismatched unrelated donor consisting of Cyclophosphamide (50 mg/kg x1 day), Fludarabine 35 mg/m2/day x 5 days, Thiotepa (5 mg/kg/day x 2 days), TBI (200 cGy x 2). Tacrolimus starting Day -1, MMF D0-35.

  Kezelés: ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs

• Arm B (HLA-matched related or matched unrelated donor )Egyéb csoport

  Conditioning regimen for HLA-matched related or matched unrelated donor consisting of Cyclophosphamide (50 mg/kg x1 day), Fludarabine (30 mg/m2/day x 5 days), Thiotepa (5 mg/kg/day x 2 days), TBI (200 cGy x 2). Tacrolimus starting Day -1, MMF D0-35.

  Kezelés: ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs