Umbilical Cord Blood Transplantation in Treating Patients With High-Risk Hematologic Cancer
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ClinicalTrials.gov Identifier: NCT00003335 |
Recruitment Status :
Completed
First Posted : January 27, 2003
Last Update Posted : March 15, 2012
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RATIONALE: Umbilical cord blood transplantation may allow doctors to give higher doses of chemotherapy or radiation therapy and kill more cancer cells.
PURPOSE: This phase II trial is studying allogeneic umbilical cord blood transplantation to see how well it works when given with chemotherapy or radiation therapy in treating patients with high-risk hematologic cancer.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Graft Versus Host Disease Leukemia Lymphoma Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Diseases | Biological: anti-thymocyte globulin Drug: busulfan Drug: cyclosporine Drug: melphalan Drug: methylprednisolone Procedure: umbilical cord blood transplantation Radiation: radiation therapy | Phase 2 |
OBJECTIVES:
- Determine the rates of hematologic and immune reconstitution in patients with high risk hematologic malignancies who are undergoing high dose chemoradiotherapy followed by unrelated umbilical cord blood (UCB) transplantation.
- Determine the incidence of graft-versus-host-disease in this setting.
- Describe the incidence of recurrent disease in these patients post UCB transplant.
- Describe the incidence of serious infections and secondary lymphoproliferative diseases following transplantation with UCB in these patients.
- Determine specifically whether larger recipients can be durably engrafted with unrelated UCB, and determine whether nucleated cell or progenitor cell content of the graft is predictive of hematological engraftment.
OUTLINE: Patients may undergo a back-up peripheral blood stem cell collection prior to treatment.
Patients receive 9 fractions of total body irradiation (TBI) on days -9 to -5 followed by melphalan IV for three days on days -4 to -2 and antithymocyte globulin IV or methylprednisolone IV for three days on days -3 to -1. On day 0, patients receive umbilical cord blood infusion. If TBI is not tolerated, busulfan is substituted and administered orally every 6 hours for 4 days on days -8 to -5. Cyclosporine and methylprednisolone begin on day -2 and continue for 6 months.
Patients are followed at least monthly for 1 year, then every 6 months for the second year, and then annually thereafter.
PROJECTED ACCRUAL: There will be a maximum of 48 patients accrued into this study over 4 years.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 44 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Patients With High Risk Hematologic Malignancies |
Study Start Date : | January 1998 |
Actual Primary Completion Date : | November 2007 |
Actual Study Completion Date : | January 2012 |

- Biological: anti-thymocyte globulin
antithymocyte globulin IV for three days on days -3 to -1
- Drug: busulfan
If TBI is not tolerated, busulfan is substituted and administered orally every 6 hours for 4 days on days -8 to -5.
- Drug: cyclosporine
Cyclosporine begin on day -2 and continue for 6 months.
- Drug: melphalan
melphalan IV for three days on days -4 to -2
- Drug: methylprednisolone
Methylprednisolone IV for three days on days -3 to -1. Methylprednisolone begin on day -2 and continue for 6 months.
- Procedure: umbilical cord blood transplantation
On day 0, patients receive umbilical cord blood infusion.
- Radiation: radiation therapy
9 fractions of total body irradiation (TBI) on days -9 to -5
- rates of durable engraftment in patients [ Time Frame: day 42 ]The primary study end point will be hematologic engraftment. Engraftment is defined as achieving ANC larger than or equal to 500 ul/mm3 of donor origin for three consecutive measurements on different days by day +42.
- Event-free survival by clinical and pathological disease assessment [ Time Frame: at disease progression or death ]
- incidence of recurrent disease in patients post UCB transplant [ Time Frame: post transplant ]

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Ages Eligible for Study: | up to 54 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
-
Histologically confirmed high risk malignancy including:
- Acute nonlymphocytic leukemia (ANLL) after induction failure, or in first complete remission with high risk features including stem cell or biphenotypic classification (acute myeloid leukemia (AML) M0), erythroleukemia (AML M6), acute megakaryocytic leukemia (AML M7), cytogenic markers indicative of poor prognosis, or failure to achieve complete remission after standard induction therapy
- Acute lymphocytic leukemia (ALL) or ANLL in second or subsequent remission
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Chronic myeloid leukemia (CML) in chronic phase
- CML with accelerated phase or blast crisis are eligible after reinduction chemotherapy converts disease to chronic phase
- High risk ALL in first complete remission
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Myelodysplastic syndrome with evidence of evolution to acute myeloid leukemia
- Refractory anemia with excess blasts
- Refractory anemia with excess blasts in transformation
- Non-Hodgkin's lymphoma (NHL), ANLL, or ALL with recurrent disease after autologous stem cell transplantation
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Must also meet all the following conditions:
- No HLA-ABC/DR identical related bone marrow or UCB donor
- No 5/6 antigen matched related bone marrow or UCB donor
- Condition precludes waiting to search and find a donor in the National Marrow Donor Registry
- Must have an available serologic matched umbilical cord blood unit in the New York Blood Center's Placental Blood Project
- No active CNS disease
PATIENT CHARACTERISTICS:
Age:
- Under 55 at time of umbilical cord blood transplantation
Performance status:
- Zubrod 0-1
- Karnofsky 80-100%
Life expectancy:
- At least 3 months
Hematopoietic:
-
For patients with ALL or ANLL in remission, CML in chronic phase, or NHL without marrow involvement who elect to undergo autologous peripheral blood stem cell collection and storage:
- WBC at least 3,000/mm^3
- Absolute neutrophil count at least 1,000/mm^3
- Platelet count at least 100,000/mm^3
Hepatic:
- Bilirubin no greater than 2.0 mg/dL
- ALT/AST no greater than 4 times normal
Renal:
- Creatinine no greater than 2.0 mg/dL
- Creatinine clearance at least 50 mL/min
Cardiovascular:
- Normal cardiac function by echocardiogram or radionuclide scan (shortening fraction or ejection fraction at least 80% of normal value for age)
Pulmonary:
- FVC and FEV_1 at least 60% of predicted for age
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For adults:
- DLCO at least 60% of predicted
Other:
- HIV negative
- No active infections at time of autologous stem cell harvest or pretransplant cytoreduction
- Not pregnant or nursing
- Effective contraception required of all fertile patients
PRIOR CONCURRENT THERAPY:
Biologic therapy:
- Prior autologous stem cell transplantation allowed
Chemotherapy:
- See Disease Characteristics
Endocrine therapy:
- Not specified
Radiotherapy:
- Not specified
Surgery:
- Not specified

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00003335
United States, Ohio | |
Case Medical Center, University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center | |
Cleveland, Ohio, United States, 44106-5065 |
Study Chair: | Brenda W. Cooper, MD | Case Medical Center, University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Case Comprehensive Cancer Center |
ClinicalTrials.gov Identifier: | NCT00003335 |
Other Study ID Numbers: |
CWRU4Y97 P30CA043703 ( U.S. NIH Grant/Contract ) NCI-G98-1429 CWRU4Y97 ( Other Identifier: Case Comprehensive Cancer Center ) |
First Posted: | January 27, 2003 Key Record Dates |
Last Update Posted: | March 15, 2012 |
Last Verified: | March 2012 |
recurrent childhood acute lymphoblastic leukemia recurrent childhood lymphoblastic lymphoma recurrent adult acute lymphoblastic leukemia chronic phase chronic myelogenous leukemia accelerated phase chronic myelogenous leukemia blastic phase chronic myelogenous leukemia untreated childhood acute lymphoblastic leukemia adult acute myeloid leukemia in remission adult acute lymphoblastic leukemia in remission childhood acute myeloid leukemia in remission childhood acute lymphoblastic leukemia in remission adult acute erythroid leukemia (M6) adult acute megakaryoblastic leukemia (M7) childhood acute erythroleukemia (M6) childhood acute megakaryocytic leukemia (M7) |
refractory anemia with excess blasts refractory anemia with excess blasts in transformation recurrent grade 1 follicular lymphoma recurrent grade 2 follicular lymphoma recurrent grade 3 follicular lymphoma recurrent adult diffuse small cleaved cell lymphoma recurrent adult diffuse mixed cell lymphoma recurrent adult diffuse large cell lymphoma recurrent adult immunoblastic large cell lymphoma recurrent adult lymphoblastic lymphoma recurrent adult Burkitt lymphoma de novo myelodysplastic syndromes previously treated myelodysplastic syndromes secondary myelodysplastic syndromes graft versus host disease |
Lymphoma Leukemia Preleukemia Myelodysplastic Syndromes Myeloproliferative Disorders Myelodysplastic-Myeloproliferative Diseases Graft vs Host Disease Syndrome Neoplasms by Histologic Type Neoplasms Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases Disease |
Pathologic Processes Bone Marrow Diseases Hematologic Diseases Precancerous Conditions Cyclosporine Methylprednisolone Melphalan Busulfan Cyclosporins Antilymphocyte Serum Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |