Phase 2
N=213
Myeloablative Umbilical Cord Blood Transplantation in Hematological Diseases
Acute Myeloid Leukemia · Acute Lymphocytic Leukemia · Chronic Myelogenous Leukemia · Myelofibrosis · MDS
Bottom Line
View on ClinicalTrials.gov: NCT00309842 ↗Enrolled (actual)
213
Serious AEs
43.4%
Results posted
Sep 2020
Primary outcome: Primary: Number of Participants Who Were Alive at 1 Year Transplant Overall Survival — 130 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- filgrastim (Biological); cyclophosphamide (Drug); cyclosporine (Drug); fludarabine phosphate (Drug); mycophenolate mofetil (Drug); umbilical cord blood transplantation (Procedure); total-body irradiation (Radiation)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Masonic Cancer Center, University of Minnesota
- Primary completion
- Jul 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Who Were Alive at 1 Year Transplant Overall Survival |
130 | — |
| SECONDARY Number of Participants Who Died Due to Transplant |
58 | — |
| SECONDARY Number of Participants With Platelet Engraftment |
159 | — |
| SECONDARY Number of Participants With Neutrophil Engraftment |
21 | — |
| SECONDARY Number of Participants With Acute Graft-Versus-Host Disease |
106; 49 | — |
| SECONDARY Number of Participants With Chronic Graft-Versus-Host Disease |
39 | — |
| SECONDARY Percentage Chimerism on Day 21 |
92.6 | — |
| SECONDARY Percentage Chimerism on Day 100 |
98.1 | — |
| SECONDARY Percentage Chimerism at 6 Months |
98.1 | — |
| SECONDARY Percentage Chimerism at 1 Year |
99.1 | — |
| SECONDARY Percentage Chimerism at 2 Years |
100 | — |
Summary
RATIONALE: Giving chemotherapy drugs, such as fludarabine and cyclophosphamide, and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells and prepares the patient's bone marrow for the 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. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil may stop this from happening.
PURPOSE: This phase II trial is studying how well giving fludarabine and cyclophosphamide together with total-body irradiation works in treating patients who are undergoing an umbilical cord blood transplant for hematologic cancer.
Eligibility Criteria
Inclusion Criteria
- Acute myeloid leukemia (AML): high risk CR1 (as evidenced by preceding myelodysplastic syndrome [MDS], high risk cytogenetics, ≥ 2 cycles to obtain complete remission [CR], erythroblastic or megakaryocytic leukemia; CR2+. All patients must be in CR as defined by hematological recovery, AND 1 cycle to obtain CR or presence minimal residual disease (MRD). Patients in CR2+ are eligible. All patients must be in CR as defined by hematological recovery, AND 12 months, are eligible after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm) should be considered for debulking chemotherapy before transplant.
- Lymphoplasmacytic lymphoma, mantle-cell lymphoma, prolymphocytic leukemia are eligible after initial therapy in CR1+ or PR1+.
- Large cell NHL > CR2/> PR2. Patients in CR2/PR2 with initial short remission ( 3 mg/L, may be considered for this protocol after initial therapy.
- Recipients must have a Karnofsky score (adults) ≥ 80 % or Lansky score ≥ 50% (pediatrics), and proper organ function.
Exclusion Criteria
- Active infection at time of transplantation
- History of human immunodeficiency virus (HIV) infection
- Pregnant or breast feeding.
- Chemotherapy refractory large cell and high grade NHL
- If 18 years old prior myeloablative allotransplant or autologous transplant
- Extensive prior therapy including > 12 months alkylator therapy or > 6 months alkylator therapy with extensive radiation.
- Patients who have received Y-90 ibritumomab (Zevalin) or I-131 tostumomab (Bexxar), as part of their salvage therapy.
Data sourced from ClinicalTrials.gov (NCT00309842). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.