MedPath

Donor Umbilical Cord Blood Transplantation in Treating Patients With Leukemia, Lymphoma, or Nonmalignant Hematologic Disorders

Phase 2
Completed
Conditions
Leukemia
Lymphoma
Myelodysplastic Syndromes
Myelodysplastic-Myeloproliferative Diseases
Registration Number
NCT00055653
Lead Sponsor
Roswell Park Cancer Institute
Brief Summary

RATIONALE: Umbilical cord blood transplantation may be able to replace immune cells that were destroyed by the chemotherapy or radiation therapy that was used to kill cancer cells.

PURPOSE: Phase II trial to study the effectiveness of allogeneic umbilical cord blood transplantation in treating patients who have leukemia, lymphoma, or nonmalignant hematologic disorders.

Detailed Description

OBJECTIVES:

* Determine 180-day survival in patients with malignant or nonmalignant hematologic diseases treated with allogeneic umbilical cord blood transplantation. (Severe aplastic anemia, Fanconi anemia, and marrow failure syndromes strata are closed to accrual; adult \[over 18 years of age\] patient stratum is closed to accrual.)

* Determine disease-free and long-term survival in patients treated with this regimen.

* Determine the incidence of neutrophil engraftment, primary and secondary graft failure, platelet engraftment, and red blood cell engraftment in patients treated with this regimen.

* Determine the incidence and severity of acute and chronic graft-versus-host disease in patients treated with this regimen.

* Determine the incidence of complications, including infection, veno-occlusive disease, and interstitial pneumonitis, in patients treated with this regimen.

* Determine the incidence of relapse, other malignancies, lymphoproliferative disorders, and posttransplantation myelodysplasia in patients treated with this regimen.

* Determine the immune reconstitution in patients treated with this regimen.

OUTLINE: This is a multicenter study. Patients are grouped according to the following strata:

* Stratum I: Malignant disease, 5/6 or 6/6 HLA match, age 18 and under

* Stratum II: Malignant disease, 4/6 HLA match, age 18 and under

* Stratum III: Malignant disease, 3/6 HLA match, age 18 and under

* Stratum IV: Malignant disease, 2/6 or 1/6 HLA match, age 18 and under

* Stratum V (closed to accrual): Severe aplastic anemia, Fanconi anemia, or other marrow failure syndrome

* Stratum VI: Inborn errors of metabolism/storage diseases and other nonmalignant diseases not included in stratum V

* Stratum VII: Malignant disease receiving alternative conditioning regimen comprising busulfan and melphalan

* Stratum VIII (closed to accrual): Adult patients (over age 18)

* Conditioning therapy: Patients are assigned to 1 of 5 groups according to diagnosis.

* Group I (malignant disease or severe aplastic anemia \[severe aplastic anemia closed to accrual\]): Patients undergo total body irradiation (TBI) once or twice daily on days -8 to -4. Patients then receive cyclophosphamide IV on days -3 and -2, methylprednisolone IV on days -3 to 0, and antithymocyte globulin (ATG) IV once or twice daily on days -3 to -1.

* Group II (Fanconi anemia \[closed to accrual\]): Patients undergo TBI on day -6, and then receive cyclophosphamide IV and fludarabine IV on days -5 to -2, and methylprednisolone IV and ATG IV on days -5 to -1.

* Group III (inborn errors of metabolism/storage disease): Patients receive oral busulfan 4 times daily on days -9 to -6, cyclophosphamide as in group II, and methylprednisolone and ATG as in group I.

* Group IV (other nonmalignant diseases): Patients receive conditioning therapy as in group III. Patients with familial erythrophagocytic lymphohistiocytosis or Langerhans cell histiocytosis also receive etoposide on days -5 to -3.

* Group V (non-TBI regimen for leukemia patients under 2 years of age): Patients receive oral busulfan 4 times daily on days -8 to -5, melphalan IV on days -4 to -2, and methylprednisolone and ATG as in group I.

* Allogeneic umbilical cord blood transplantation: All patients undergo umbilical cord blood transplantation on day 0. Beginning on day 0 or 1, patients receive filgrastim (G-CSF) IV or subcutaneously daily until blood counts recover.

* Graft-versus-host disease prophylaxis: Patients receive cyclosporine (IV or oral) beginning between days -3 and -1 and continuing for 1 year after transplantation and methylprednisolone twice daily beginning on day 1 and continuing until blood counts recover.

Patients are followed weekly for 14 weeks, at 100 days, and at 4, 5, 6, 9, 12, 18, 24, and 36 months.

PROJECTED ACCRUAL: A total of 360 patients will be accrued for this study.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
Not specified
Inclusion Criteria

Not provided

Read More
Exclusion Criteria

Not provided

Read More

Study & Design

Study Type
INTERVENTIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (25)

Children's Hospital Los Angeles

🇺🇸

Los Angeles, California, United States

Children's Hospital of Orange County

🇺🇸

Orange, California, United States

North Shore University Hospital

🇺🇸

Manhasset, New York, United States

Children's National Medical Center

🇺🇸

Washington, District of Columbia, United States

Jonsson Comprehensive Cancer Center, UCLA

🇺🇸

Los Angeles, California, United States

Children's Medical Center of Dallas

🇺🇸

Dallas, Texas, United States

Children's Medical Center, University of California San Francisco

🇺🇸

San Francisco, California, United States

Children's Hospital of New Orleans

🇺🇸

New Orleans, Louisiana, United States

Ireland Cancer Center

🇺🇸

Cleveland, Ohio, United States

Indiana University Cancer Center

🇺🇸

Indianapolis, Indiana, United States

Children's Hospital of Pittsburgh

🇺🇸

Pittsburgh, Pennsylvania, United States

City of Hope Comprehensive Cancer Center

🇺🇸

Duarte, California, United States

Children's Mercy Hospital

🇺🇸

Kansas City, Missouri, United States

Medical City Dallas Hospital

🇺🇸

Dallas, Texas, United States

James P. Wilmot Cancer Center at University of Rochester Medical Center

🇺🇸

Rochester, New York, United States

Spectrum Health and DeVos Children's Hospital

🇺🇸

Grand Rapids, Michigan, United States

Texas Transplant Institute

🇺🇸

San Antonio, Texas, United States

Duke Comprehensive Cancer Center

🇺🇸

Durham, North Carolina, United States

Cardinal Glennon Children's Hospital

🇺🇸

Saint Louis, Missouri, United States

Vanderbilt-Ingram Cancer Center

🇺🇸

Nashville, Tennessee, United States

University of Minnesota Cancer Center

🇺🇸

Minneapolis, Minnesota, United States

Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

Hackensack University Medical Center

🇺🇸

Hackensack, New Jersey, United States

Roswell Park Cancer Institute

🇺🇸

Buffalo, New York, United States

Fred Hutchinson Cancer Research Center

🇺🇸

Seattle, Washington, United States

© Copyright 2025. All Rights Reserved by MedPath