Trials / Completed
CompletedNCT01384513
A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies
A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 40 (actual)
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University · Academic / Other
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
This phase II trial studies how well reduced intensity donor stem cell transplant works in treating patients with hematologic malignancies. 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. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving tacrolimus and mycophenolate mofetil after the transplant may stop this from happening. Once the donated stem cells begin working, the patient's immune system may see the remaining cancer cells as not belonging in the patient's body and destroy them. Giving an infusion of the donor's white blood cells (donor lymphocyte infusion) may boost this effect.
Detailed description
PRIMARY OBJECTIVES: I. To compare the overall survival (OS) rate at 2 years post treatment using the Jefferson 2 step reduced intensity conditioning (RIC) approach in patients with haploidentical family donors with hematological malignancies in morphological or radiographic remission or with chemosensitive, indolent diseases to historical OS rates in similar populations after RIC matched donor HSCT as reported in the literature. SECONDARY OBJECTIVES: I. To compare the treatment-related mortality (TRM) rate at 2 years for patients treated on this study to the historical TRM rates of patients undergoing RIC matched-sibling HSCT as reported in the literature. II. To compare the 2 year relapse rates and relapse related mortality of patients with myeloid diseases to that of patients with lymphoid diseases who are treated on this Thomas Jefferson University (TJU) RIC 2 step approach. III. To determine the incidence and severity of graft-versus-host disease (GVHD) in patients undergoing treated on the TJU RIC 2 step approach. IV. To evaluate engraftment rates and lymphoid reconstitution in patients treated on the TJU RIC 2 step approach. V. To evaluate the incidence of TRM at 100 days in patients treated on the TJU RIC 2 step approach. OUTLINE: REDUCED INTENSITY CONDITIONING: Patients receive fludarabine phosphate intravenously (IV) over 60 minutes on days -11 to -8 and bulsufan IV over 3 hours on days -10 to -9. Patients undergo total body irradiation (TBI) on day -6. Patients also receive cyclophosphamide IV over 2 hours on days -3 and -2. TRANSPLANTATION: Patients undergo donor lymphocyte infusion (DLI) on day -6 and cluster of differentiation (CD)-34+ allogeneic peripheral blood stem cell transplantation (PBSCT) on day 0. GVHD PROPHYLAXIS: Beginning on day -1, patients receive tacrolimus IV or orally (PO) with taper beginning on day 42. Patients also receive mycophenolate mofetil IV twice daily (BID) on days -1 to 28. After completion of study treatment, patients are followed up periodically for 2 years.
Conditions
- Adult Acute Lymphoblastic Leukemia in Remission
- Adult Acute Myeloid Leukemia in Remission
- Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Adult Acute Myeloid Leukemia With Del(5q)
- Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
- Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)
- Adult Acute Myeloid Leukemia With t(8;21)(q22;q22)
- Adult Nasal Type Extranodal NK/T-cell Lymphoma
- Aplastic Anemia
- Childhood Acute Lymphoblastic Leukemia in Remission
- Childhood Acute Myeloid Leukemia in Remission
- Childhood Myelodysplastic Syndromes
- Chronic Eosinophilic Leukemia
- Chronic Myelomonocytic Leukemia
- Chronic Neutrophilic Leukemia
- Essential Thrombocythemia
- Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue
- Juvenile Myelomonocytic Leukemia
- Mastocytosis
- Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable
- Nodal Marginal Zone B-cell Lymphoma
- Polycythemia Vera
- Previously Treated Myelodysplastic Syndromes
- Primary Myelofibrosis
- Recurrent Adult Acute Lymphoblastic Leukemia
- Recurrent Adult Acute Myeloid Leukemia
- Recurrent Adult Diffuse Small Cleaved Cell Lymphoma
- Recurrent Adult Grade III Lymphomatoid Granulomatosis
- Recurrent Adult Hodgkin Lymphoma
- Recurrent Childhood Acute Lymphoblastic Leukemia
- Recurrent Childhood Acute Myeloid Leukemia
- Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma
- Recurrent Grade 1 Follicular Lymphoma
- Recurrent Grade 2 Follicular Lymphoma
- Recurrent Marginal Zone Lymphoma
- Recurrent Mycosis Fungoides/Sezary Syndrome
- Recurrent Small Lymphocytic Lymphoma
- Recurrent/Refractory Childhood Hodgkin Lymphoma
- Refractory Anemia
- Refractory Anemia With Ringed Sideroblasts
- Refractory Hairy Cell Leukemia
- Refractory Multiple Myeloma
- Secondary Myelodysplastic Syndromes
- Splenic Marginal Zone Lymphoma
- T-cell Large Granular Lymphocyte Leukemia
- Waldenström Macroglobulinemia
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Fludarabine | Given IV |
| DRUG | Busulfan | Given IV |
| RADIATION | Total Body Irradiation (TBI) | 2 Gy administered as part of the conditioning regimen |
| BIOLOGICAL | Donor Lymphocyte Infusion (DLI) | Undergo DLI |
| DRUG | Cyclophosphamide (CY) | Given IV |
| DRUG | Tacrolimus | Given IV or PO |
| DRUG | Mycophenolate mofetil | Given IV |
| DEVICE | Allogeneic hematopoietic stem cell transplantation | Undergo CD34+ allogeneic PBSCT |
| PROCEDURE | Peripheral blood stem cell transplantation (PBSCT) | Undergo CD34+ allogeneic PBSCT |
Timeline
- Start date
- 2011-08-04
- Primary completion
- 2020-11-13
- Completion
- 2022-11-16
- First posted
- 2011-06-29
- Last updated
- 2026-04-15
- Results posted
- 2026-04-15
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated device study
Source: ClinicalTrials.gov record NCT01384513. Inclusion in this directory is not an endorsement.