Phase 2
N=284
Non-Myeloablative Conditioning for Unrelated Donor Umbilical Cord Blood Transplant
Myeloproliferative Disorders · Leukemia · Lymphoma · Myelodysplastic Syndromes
Bottom Line
View on ClinicalTrials.gov: NCT00305682 ↗Enrolled (actual)
284
Serious AEs
42.6%
Results posted
Nov 2020
Primary outcome: Primary: Number of Participants Who Were Alive at 1 Year Post Transplant — 59; 40; 1; 26 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- anti-thymocyte globulin (Biological); cyclophosphamide (Drug); Fludarabine (Drug); mycophenolate mofetil (Drug); umbilical cord blood transplantation (Procedure); total body irradiation (Radiation); Sirolimus (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Masonic Cancer Center, University of Minnesota
- Primary completion
- Dec 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Who Were Alive at 1 Year Post Transplant |
59; 40; 1; 26; 26; 25 | — |
| PRIMARY Number of Participants Who Were Alive at 2 Years Post Transplant |
50; 31; 1; 20; 21; 23 | — |
| SECONDARY Number of Participants Who Were Dead at 6 Months After Study Completion |
10; 20; 2; 5; 3; 6 | — |
| SECONDARY Percentage of Donor Chimerism at 21 Days |
77; 73; 57; 77; 69; 68 | — |
| SECONDARY Percentage of Donor Chimerism at 100 Days |
94; 94; 100; 93; 85; 86 | — |
| SECONDARY Percentage of Donor Chimerism at 180 Days |
96; 98; 88; 94; 91; 98 | — |
| SECONDARY Percentage of Donor Chimerism at 365 Days |
99; 98; 99; 87; 100 | — |
| SECONDARY Number of Participants With Neutrophil Engraftment |
93; 65; 6; 32; 32; 29 | — |
| SECONDARY Number of Participants With Platelet Engraftment |
75; 47; 3; 28; 34; 25 | — |
| SECONDARY Number of Participants With Acute Graft-versus-host Disease (GVHD) |
45; 24; 1; 12; 13; 13 | — |
| SECONDARY Number of Participants With Chronic Graft-Versus-Host Disease |
18; 20; 0; 3; 3; 3 | — |
| SECONDARY Number of Participants Experiencing Progression-free Survival |
43; 32; 1; 21; 16; 24 | — |
| SECONDARY Number of Participants Experiencing Progression-free Survival at 2 Years |
36; 25; 1; 17; 16; 20 | — |
| SECONDARY Number of Participants Experiencing Relapse (Incidence of Relapse) |
43; 14; 4; 7; 20; 2 | — |
| SECONDARY Number of Participants Experiencing Relapse (Incidence of Relapse) at 2 Years |
49; 19; 4; 11; 20; 4 | — |
Summary
RATIONALE: Drugs used in chemotherapy, such as fludarabine and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Radiation therapy uses high-energy x-rays to kill cancer cells. An umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy and radiation therapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving sirolimus and mycophenolate mofetil after the transplant may stop this from happening.
PURPOSE: This phase II trial is studying how well giving fludarabine and cyclophosphamide together with total-body irradiation followed by an umbilical cord blood transplant, sirolimus, and mycophenolate mofetil works in treating patients with hematologic cancer.
Eligibility Criteria
Inclusion Criteria
Age, Graft Cell Dose and Graft HLA Criteria
- Subjects must be 2 cycles to obtain CR or erythroblastic and megakaryocytic); second or greater CR.
- Acute lymphoblastic leukemia/lymphoma: high risk CR1 as evidenced by high risk cytogenetics (e.g. t(9;22), t(1;19),t(4;11), other myeloid/lymphoid or mixed lineage leukemia [MLL] rearrangements, hypodiploidy or Ikaros family zinc finger 1 [IKZF1]), > 1 cycle to obtain CR or evidence of minimal residual disease (MRD). Patients in second or greater CR are also eligible.
- Burkitt's lymphoma in CR2 or subsequent CR
- Natural Killer cell malignancies
- Chronic myelogenous leukemia: all types except refractory blast crisis. Chronic phase patients must have failed or been intolerant to Gleevec
- Myelodysplastic syndrome:
- Large-cell lymphoma, Hodgkin lymphoma and multiple myeloma with chemotherapy sensitive disease that has failed or patients who are ineligible for an autologous transplant.
- Chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), marginal zone B-cell lymphoma, follicular lymphoma, which have progressed within 12 months of achieving a partial or complete remission. Patients who had remissions lasting > 12 months, are eligible after at least two prior therapies. Patients with bulky disease should be considered for debulking chemotherapy before transplant. Patients with refractory disease are eligible, unless has bulky disease and an estimated tumor doubling time of less than one month.
- Lymphoplasmacytic lymphoma, mantle-cell lymphoma, prolymphocytic leukemia are eligible after initial therapy if chemotherapy sensitive.
- Refractory leukemia or MDS.
- Bone marrow failure syndromes, except for Fanconi Anemia
- Myeloproliferative syndromes Patients who have undergone an autologous transplant >12 months prior to allogeneic transplantation
Adequate Organ Function and Performance Status
Exclusion Criteria
- 5% blasts in normocellular bone marrow OR any % blasts if blasts have unique morphologic markers (e.g. Auer rods) or associated cytogenetic markers that allows morphologic relapse to be distinguished are not eligible.
- Chronic myelogenous leukemia (CML) in refractory blast crisis
- Large cell lymphoma, mantle cell lymphoma and Hodgkin disease that is progressive on salvage therapy. Stable disease is acceptable to move forward provided it is non-bulky.
- Active central nervous system malignancy
Data sourced from ClinicalTrials.gov (NCT00305682). 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.