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Protocol No. UW13090

Principal Investigator Otto, Mario

Phase I

Age Group Children

Scope Local

Sponsor Type Institutional

Title TCR-a/b+ and CD19+ depleted KIR/KIR ligand-mismatched Haploidentical Hematopoietic Stem Cell Transplant and Zoledronate for Pediatric Relapsed/Refractory Hematologic Malignancies and High Risk Solid Tumors

Objective In this study, we are attempting to reduce the problems caused by the conditioning (the combination of drugs given to transplant recipients before an hematopoietic stem cell transplantation) regimen by using a combination of drugs that is less intense compared to what is typically used for this type of transplant. The "reduced intensity" conditioning regimen may cause fewer side effects compared with a standard regimen, but may also be associated with an increased risk of graft rejection (the new stem cells don't grow). To prevent life-threatening immune reations some immune cells (alpha/beta T Cells and B cells) will be eliminated from the donated stem cell graft. To eliminate them from the donated stem cells, we will run them through a lab instrument that sorts the cells to remove the ones that may cause graft versus host disease. This is an experimental process that has not yet been approved by the FDA. In summary, the overall approach to treat your child with haploidentical hematopoietic stem cell transplantation using donor cells that have been run through a lab instrument to eliminate alpha/beta T cells and B cells is experimental.

Treatment Twelve days before the transplant, your child will be hospitalized and begin a series of treatments in preparation for the transplant. This is called the "conditioning regimen". The chemotherapy that your child will get uses FDA approved drugs that have been used by doctors in the past for a similar treatment. This includes the following: ATG, Fludarabine, Thiotepa, and Melphalan. Depending on when your child enrolls in this study, he/she may also receive two doses of a drug called Zoledronate. This drug is not chemotherapy. Zoledronate is an FDA approved drug but its use to stimulate immune cells after stem cell transplant is experimental. It has been used shortly after stem cell transplant to strengthen bone and reduce bone pain.

Description Stem cell transplant involves getting chemotherapy strong enough to destroy your child's normal blood cell production and immune system. The conditioning chemotherapy is followed by an infusion (transplant) of stem cells that have been collected from your child's relative through a central venous catheter. This process takes about 30 minutes. Your child may be hospitalized for about 5 - 6 weeks. Click here for more info

Key Eligibility
  • Patients must be > 6 months and ≤ 21 years of age
  • Patients with a hematologic malignancy or solid tumor
  • Patients with more than one malignancy (hematologic or solid tumor) are eligible
  • No HLA identical sibling or suitable unrelated donor, or time needed to find an acceptable unrelated donor match would likely result in disease progression such that the patient may become ineligible for any type of potentially curative transplant
  • Relapsed or primary therapy-refractory AML with bone marrow blast < 20%
  • High-risk refractory or relapsed ALL in patients for whom transplantation is deemed indicated
  • Patients with relapsed Hodgkin lymphoma unable to achieve 2nd remission or very good partial remission (VGPR) and therefore ineligible to receive autologous stem cell transplantation
  • Patients with Hodgkin lymphoma relapsing after autologous stem cell transplant
  • Patients with primary refractory or relapsed non-Hodgkin lymphoma (NHL) unable to achieve 2nd remission or very good partial remission (VGPR) and therefore ineligible to receive autologous stem cell transplantation
  • Patients with NHL relapsing after autologous stem cell transplant
  • Patients with myelodysplastic syndrome (MDS)/Myeloproliferative Syndrome (MPS)
  • Failed or ineligible to receive autologous transplant or if autologous transplant would not offer > 20% chance of cure
  • Neuroblastoma - high risk with relapsed or refractory disease
  • Soft tissue sarcoma - relapsed or primary refractory metastatic; 1st complete remission, but very high-risk features
  • Osteosarcoma - failure to achieve CR following initial therapy; relapsed with pulmonary or bone metastases and did not achieve a CR with surgery and/or chemotherapy
  • Study enrollment no earlier than 3 months after preceding hematopoietic cell transplantation (HSCT)
  • Female patients who are pregnant or breast-feeding or of reproductive potential not willing to use an effective method of birth control during study treatment and for at least 12 months thereafter are excluded
  • Patients with HIV infection are excluded

Applicable Disease Sites Brain/Central Nervous System; Hematologic cancers, other; Leukemia; Lymphoma; Neuroblastoma; Sarcoma

Therapies Involved Bone Marrow Transplant; Cytotoxic Chemotherapy; Other

Drugs Involved Zometa (zoledronate); alkeran (melphalan); f-ara-amp (fludarabine); flamp (fludarabine); fludara (fludarabine); fludarabine; lpam (melphalan); melphalan; rabbit antithymocyte globulin; thiotepa; thymoglobulin (rabbit antithymocyte globulin)

Status Open

Participating Institutions UW Hospital and Clinics