Trials / Terminated
TerminatedNCT03333486
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
A Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem Cells
- Status
- Terminated
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 31 (actual)
- Sponsor
- Roswell Park Cancer Institute · Academic / Other
- Sex
- All
- Age
- 1 Year – 75 Years
- Healthy volunteers
- Not accepted
Summary
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer. Drugs used in chemotherapy, such as fludarabine phosphate and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays to kill cancer cells and shrink tumors. 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.
Detailed description
PRIMARY OBJECTIVES: I. To evaluate the rate of relapse, defined as recurrence of underlying disease or progression of underlying disease, at 1 year in patients who receive haploidentical peripheral blood stem cells (PBSCs) after reduced intensity conditioning and post-transplant cyclophosphamide and tocilizumab (or tocilizumab alternative). SECONDARY OBJECTIVES: I. To evaluate safety including development of acute graft versus host disease (GVHD) and death at 100 days post-transplant, as well as other treatment related toxicities including chronic GVHD, engraftment rate, non-relapse mortality, progression free survival (PFS) at one year, and overall survival (OS) at one year, as compared with historical controls. TERTIARY OBJECTIVES: I. Correlative studies will include chimerism analysis by molecular analysis and evaluation of immune reconstitution by cytomegalovirus (CMV) dextramer analysis using flow cytometry. OUTLINE: Patients receive fludarabine phosphate intravenously (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 2 hours on days -6 and -5. Patients undergo total body irradiation (TBI) on days -1 and peripheral blood stem cell transplantation (PBSCT) on day 0. After completion of study treatment, patients are followed up at 30 and 100 days.
Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- Acute Leukemia in Remission
- Acute Lymphoblastic Leukemia
- Acute Myeloid Leukemia
- Acute Myeloid Leukemia With FLT3/ITD Mutation
- Acute Myeloid Leukemia With Gene Mutations
- Aplastic Anemia
- B-Cell Non-Hodgkin Lymphoma
- CD40 Ligand Deficiency
- Chronic Granulomatous Disease
- Chronic Leukemia in Remission
- Chronic Lymphocytic Leukemia
- Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- Chronic Myelomonocytic Leukemia
- Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- Congenital Amegakaryocytic Thrombocytopenia
- Congenital Neutropenia
- Congenital Pure Red Cell Aplasia
- Glanzmann Thrombasthenia
- Immunodeficiency Syndrome
- Myelodysplastic Syndrome
- Myelofibrosis
- Myeloproliferative Neoplasm
- Paroxysmal Nocturnal Hemoglobinuria
- Plasma Cell Myeloma
- Polycythemia Vera
- Recurrent Non-Hodgkin Lymphoma
- Refractory Non-Hodgkin Lymphoma
- Secondary Acute Myeloid Leukemia
- Secondary Myelodysplastic Syndrome
- Severe Aplastic Anemia
- Shwachman-Diamond Syndrome
- Sickle Cell Disease
- T-Cell Non-Hodgkin Lymphoma
- Thalassemia
- Waldenstrom Macroglobulinemia
- Wiskott-Aldrich Syndrome
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Cyclophosphamide | Given IV |
| DRUG | Fludarabine Phosphate | Given IV |
| OTHER | Laboratory Biomarker Analysis | Correlative studies |
| PROCEDURE | Peripheral Blood Stem Cell Transplantation | Undergo PBSCT |
| RADIATION | Total-Body Irradiation | Undergo TBI |
Timeline
- Start date
- 2017-12-07
- Primary completion
- 2023-08-28
- Completion
- 2023-08-28
- First posted
- 2017-11-07
- Last updated
- 2025-07-04
- Results posted
- 2024-04-02
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03333486. Inclusion in this directory is not an endorsement.