NCT02433483

Microtransplantation to Treat Refractory or Relapsed Hematologic Malignancies in Younger Patients

A Phase II Study of Microtransplantation in Patients With Refractory or Relapsed Hematologic Malignancies

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: St. Jude Children's Research Hospital · Academic / Other
• Sex: All
• Age: 21 Years
• Healthy volunteers: Not accepted

Summary

Allogeneic transplant can sometimes be an effective treatment for leukemia. In a traditional allogeneic transplant, patients receive very high doses of chemotherapy and/or radiation therapy, followed by an infusion of their donor's bone marrow or blood stem cells. The high-dose chemotherapy drugs and radiation are given to remove the leukemia cells in the body. The infusion of the donor's bone marrow or blood stem cells is given to replace the diseased bone marrow destroyed by the chemotherapy and/or radiation therapy. However, there are risks associated with allogeneic transplant. Many people have life-threatening or even fatal complications, like severe infections and a condition called graft-versus-host disease, which is caused when cells from the donor attack the normal tissue of the transplant patient. Recently, several hospitals around the world have been using a different type of allogeneic transplant called a microtransplant. In this type of transplant, the donor is usually a family member who is not an exact match. In a microtransplant, leukemia patients get lower doses of chemotherapy than are used in traditional allogeneic transplants. The chemotherapy is followed by an infusion of their donor's peripheral blood stem cells. The objective of the microtransplant is to suppress the bone marrow by giving just enough chemotherapy to allow the donor cells to temporarily engraft (implant), but only at very low levels. The hope is that the donor cells will cause the body to mount an immunologic attack against the leukemia, generating a response called the "graft-versus-leukemia" effect or "graft-versus-cancer" effect, without causing the potentially serious complication of graft-versus-host disease. With this research study, the investigators hope to find out whether or not microtransplantation will be a safe and effective treatment for children, adolescents and young adults with relapsed or refractory hematologic malignancies

Detailed description

PRIMARY OBJECTIVES: * To assess the safety and feasibility of standard chemotherapy plus GCSF-mobilized Hematopoietic Progenitor Cell, Apheresis (HPC-A) in pediatric patients with relapsed or refractory hematologic malignancies. * To estimate the response rates to standard chemotherapy plus GCSF-mobilized HPC-A in pediatric patients with relapsed or refractory hematologic malignancies. SECONDARY OBJECTIVES: * To describe the event-free and overall survival of patients treated with standard chemotherapy plus GCSF-mobilized HPC-A. * To estimate the time to neutrophil and platelet recovery after treatment with standard chemotherapy plus GCSF-mobilized HPC-A. * To determine the cumulative incidence of acute and chronic graft-versus-host disease (GVHD). OTHER PRESPECIFIED OBJECTIVES: * To characterize donor chimerism and microchimerism. Patients will receive standard chemotherapy followed by infusion of donor peripheral blood mononuclear cells 2 days after the completion of chemotherapy. Patients who have at least a partial response are eligible to receive a second cycle. Diagnostic lumbar puncture and intrathecal (IT) chemotherapy will be given prior to cycle 1. Patients without evidence of central nervous system (CNS) leukemia will receive no further IT therapy during cycle 1. Patients with CNS disease will receive weekly IT therapy (age-adjusted methotrexate, hydrocortisone, and cytarabine) until the cerebrospinal fluid (CSF) becomes free of leukemia (minimum of 4 doses). Bone marrow aspiration (BMA) and biopsy to assess response will be performed on approximately day 29 of therapy. For hematopoietic stem cell mobilization, donors will receive G-CSF (Filgrastim) (Neupogen®) each day for 5 days given subcutaneously (SQ) prior to HPC-A collected by leukapheresis on day 6.

Conditions

• Acute Myeloid Leukemia (AML)
• Myelodysplastic Syndrome (MDS)

Interventions

Type	Name	Description
DRUG	Cytarabine	Given by either intrathecal (IT) or intravenous (IV) route.
DRUG	Intrathecal Triples	given IT.
BIOLOGICAL	HPC-A	Given IV.

Timeline

• Start date: 2015-05-22
• Primary completion: 2017-05-08
• Completion: 2017-05-08
• First posted: 2015-05-05
• Last updated: 2017-11-01
• Results posted: 2017-11-01

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study