NCT01760655

Reduced-Intensity Conditioning Before Donor Stem Cell Transplant in With High-Risk Hematologic Malignancies

A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for High Risk Hematologic Malignancies

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 62 (actual)
• Sponsor: Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This phase II trial studies reduced-intensity conditioning before donor stem cell transplant in treating patients with high-risk hematologic malignancies. Giving low-doses of chemotherapy and total-body irradiation before a donor stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Giving an infusion of the donor's T cells (donor lymphocyte infusion) before the transplant may help increase this effect.

Detailed description

PRIMARY OBJECTIVES: I. To compare the rate of disease-free survival (DFS) at 1 year post hematopoietic stem cell transplant (HSCT) in patients undergoing HSCT treated on this successor Thomas Jefferson University (TJU) 2 Step reduced intensity conditioning (RIC) haploidentical regimen and compare it with that of the initial 2 Step RIC regimen. SECONDARY OBJECTIVES: I. To assess the 100 day regimen-related mortality (RRM) in patients undergoing HSCT on this treatment protocol. II. To determine the incidence and severity of graft-versus-host disease (GVHD) in patients undergoing treatment on this regimen. III. To evaluate engraftment rates and lymphoid reconstitution in patients treated on this trial. IV. To assess overall survival at 1 and 3 years past HSCT in patients treated on this trial. OUTLINE: REDUCED INTENSITY CONDITIONING: Patients receive fludarabine phosphate intravenously (IV) on days -15 to -12, busulfan IV on days -14 to -13, donor lymphocyte infusion (DLI) on day -6, and cyclophosphamide IV on days -3 and -2. Patients also undergo total-body irradiation (TBI) on day -10. TRANSPLANT: Patients undergo allogeneic peripheral blood stem cell transplant (PBSCT) on day 0. GVHD PROPHYLAXIS: Patients receive tacrolimus IV on days -1 to 42 followed by taper and mycophenolate mofetil IV twice daily (BID) on days -1 to 28. After completion of study treatment, patients are followed up periodically.

Conditions

• Acute Myeloid Leukemia With FLT3/ITD Mutation
• Acute Myeloid Leukemia With Gene Mutations
• Acute Myeloid Leukemia With Inv(3) (q21.3;q26.2); GATA2, MECOM
• Acute Myeloid Leukemia With t(6;9) (p23;q34.1); DEK-NUP214
• Aplastic Anemia
• Chronic Lymphocytic Leukemia
• Chronic Myelogenous Leukemia, BCR-ABL1 Positive
• Chronic Myelomonocytic Leukemia
• Follicular Lymphoma
• Hodgkin Lymphoma
• Myelodysplastic Syndrome
• Myelofibrosis
• Myeloid Leukemia
• Non-Hodgkin Lymphoma
• Plasma Cell Myeloma
• Polycythemia Vera
• Recurrent Adult Acute Myeloid Leukemia
• Recurrent Childhood Acute Myeloid Leukemia
• Refractory Acute Myeloid Leukemia
• Secondary Acute Myeloid Leukemia
• Therapy-Related Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	Fludarabine phosphate	Given IV
DRUG	Busulfan	Given IV
RADIATION	Total-Body Irradiation	Undergo TBI
BIOLOGICAL	Therapeutic Allogeneic Lymphocytes	Undergo DLI
DRUG	Cyclophosphamide	Given IV
PROCEDURE	Allogeneic Hematopoietic Stem Cell Transplantation	Undergo allogeneic PBSCT
PROCEDURE	Peripheral Blood Stem Cell Transplantation	Undergo allogeneic PBSCT
DRUG	Tacrolimus	Given IV
DRUG	Mycophenolate Mofetil	Given IV
OTHER	Laboratory Biomarker Analysis	Correlative studies

Timeline

• Start date: 2012-12-24
• Primary completion: 2022-04-15
• Completion: 2022-12-05
• First posted: 2013-01-04
• Last updated: 2025-10-30
• Results posted: 2025-10-30

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated device study