Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer

• ClinicalTrials.gov Identifier: NCT00290628
• Recruitment Status: Terminated
• First Posted: February 13, 2006
• Last Update Posted: November 29, 2017
• Sponsor: Masonic Cancer Center, University of Minnesota
• Collaborator: National Cancer Institute (NCI)
• Information provided by (Responsible Party): Masonic Cancer Center, University of Minnesota

Tracking Information

• First Submitted Date: February 9, 2006
• First Posted Date: February 13, 2006
• Last Update Posted Date: November 29, 2017
• Study Start Date: October 1999
• Actual Primary Completion Date: April 2007 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures: Not Provided
• Original Primary Outcome Measures: Not Provided
• Change History: Complete list of historical versions of study NCT00290628 on ClinicalTrials.gov Archive Site
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer
• Official Title: Transplantation of Umbilical Cord Blood From Related and Unrelated Donors

Brief Summary

RATIONALE: Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. When the stem cells from a related or unrelated donor, that do not exactly match the patient's blood, are infused into the patient they may help the patient's bone marrow to make stem cells, red blood cells, white blood cells, and platelets.

PURPOSE: This clinical trial is studying how well donor umbilical cord blood transplant works in treating patients with hematologic cancer.

Detailed Description

OBJECTIVES:

Primary

• Determine the engraftment potential of umbilical cord blood (UCB) in patients with hematological cancers.
• Determine the safety of UCB transplantation in these patients.

Secondary

• Determine the rate of neutrophil and platelet recovery and the completeness of donor cell engraftment.
• Determine the incidence and severity of acute and chronic graft-versus-host disease.
• Determine the incidence of relapse in patients with malignant disease.
• Determine the probabilities of survival and event-free survival (EFS) at 1 and 2 years after UCB transplantation.

OUTLINE: Patients are stratified according to degree of HLA disparity (0-1 vs 2-3 disparities between donor and recipient), donor type (related vs unrelated), and the basis of cell dose (< 2 vs ≥ 2 x 10^7 nucleated cells/kg recipient body weight). Patients are assigned to 1 of 4 treatment groups according to disease*.

NOTE: *Patients with acute lymphocytic leukemia (ALL), secondary acute myeloid leukemia (AML), severe combined immunodeficiency, familial erythrophagocytic lymphohistiocytosis (FEL)/viral-associated hemophagocytic syndrome (VAHS), inborn errors of metabolism, aplastic anemia, Fanconi's anemia, or Diamond-Blackfan anemia who have an unrelated umbilical cord blood donor may proceed directly to transplantation.

• Preparative regimen:

  • Group 1 (patients with chronic myelogenous leukemia, AML, myelodysplastic syndromes, or ALL): Patients receive cyclophosphamide IV once daily on days -7 and -6. Patients then undergo total-body irradiation (TBI) twice daily on days -4 to -1. Patients undergoing unrelated allogeneic umbilical cord blood transplantation (UCBT) also receive methylprednisone IV and anti-thymocyte globulin (ATG) IV twice daily on days -2 and -1.
  • Group 2 (patients with infant leukemia): Patients receive busulfan orally or IV four times daily on days -9 to -6 and melphalan IV once daily on days -4 to -2. Patients undergoing unrelated allogeneic UCBT also receive methylprednisolone IV and ATG IV twice daily on days -2 and -1.
  • Group 3 (patients with inborn errors of metabolism): Patients receive busulfan orally or IV four times daily on days -9 to -6 and cyclophosphamide IV once daily on days -5 to -2. Patients undergoing unrelated allogeneic UCBT also receive methylprednisolone IV and ATG IV twice daily on days -2 and -1.
  • Group 4 (patients with aplastic anemia): Patients receive cyclophosphamide IV once daily on days -6 to -3 and ATG IV twice daily on days -5 and -3. Patients then undergo TBI once on day -2.
• Allogeneic UCBT: Patients undergo UCBT on day 0. Patients with inborn errors of metabolism receive methylprednisolone IV before and after UCBT on day 0.

• Graft-versus-host disease (GVHD) prophylaxis: Patients receive 1 of the following regimens:

  • Related donor UCBT: Patients receive cyclosporine IV over 2 hours or orally beginning on day -3 and continuing until day 60.
  • Unrelated donor UCBT and myeloablative preparative regimen: Patients receive cyclosporine orally or IV over 2 hours twice daily beginning on day -3 and continuing until day 180. Patients also receive methylprednisolone IV twice daily on days 5-19.
  • Unrelated donor UCBT and nonmyeloablative preparative regimen: Patients receive cyclosporine IV over 2 hours or orally twice daily beginning on day -3 and continuing until day 180. Patients also receive mycophenolate mofetil IV or orally beginning on day 5 and continuing until day 30 or 7 days after active GVHD is controlled.

All patients receive filgrastim (G-CSF) IV beginning on day 1 and continuing until blood counts recover.

Patients are followed periodically for 2 years.

PROJECTED ACCRUAL: A total of 60 patients will be accrued for this study.

• Study Type: Interventional
• Study Phase: Not Applicable
• Study Design: Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Chronic Myeloproliferative Disorders
• Diamond-blackfan Anemia
• Fanconi Anemia
• Graft Versus Host Disease
• Leukemia
• Lymphoma
• Multiple Myeloma and Plasma Cell Neoplasm
• Myelodysplastic Syndromes
• Myelodysplastic/Myeloproliferative Diseases

Intervention

• Drug: anti-thymocyte globulin
• Drug: busulfan
• Drug: cyclophosphamide
• Drug: cyclosporine
• Drug: filgrastim
• Drug: melphalan
• Drug: methylprednisolone
• Drug: mycophenolate mofetil
• Procedure: radiation therapy
• Procedure: umbilical cord blood transplantation

• Study Arms: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Terminated
• Actual Enrollment (submitted: October 9, 2012): 43
• Original Enrollment: Not Provided
• Actual Study Completion Date: April 2007
• Actual Primary Completion Date: April 2007 (Final data collection date for primary outcome measure)

Eligibility Criteria

DISEASE CHARACTERISTICS:

• Diagnosis of 1 of the following:

  • Leukemia including, but not limited to, the following subtypes:

    • Chronic myelogenous leukemia
    • Acute myeloid leukemia (primary or secondary)
    • Acute lymphoblastic leukemia
  • Lymphoma
  • Myelodysplastic syndrome
  • Aplastic anemia
  • Fanconi's anemia
  • Diamond-Blackfan anemia
  • Inborn errors of metabolism (e.g., Hurler syndrome, Maroteaux-Lamy syndrome, myelodysplastic syndrome VI, metachromatic leukodystrophy, adrenoleukodystrophy, and globoid cell leukodystrophy)
  • Immune deficiency disorders
• Patients must meet the eligibility requirements outlined in currently active treatment protocols of the University of Minnesota Bone Marrow Transplant Program

• HLA-identical or 1, 2, or 3 antigen mismatched umbilical cord blood (UCB) donor with at least one DRB1 match available

  • Unrelated or related donor
  • UCB specimen must contain ≥ 2.0 x 10^7 nucleated cells/kg patient body weight

PATIENT CHARACTERISTICS:

• See Disease Characteristics
• No active infection
• No history of HIV infection
• Not pregnant or nursing
• Negative pregnancy test
• Fertile patients must use effective contraception

PRIOR CONCURRENT THERAPY:

• Not specified

Sex/Gender

• Sexes Eligible for Study: All

• Ages: up to 45 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT00290628
• Other Study ID Numbers: 2000LS017; UMN-MT1999-28
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Masonic Cancer Center, University of Minnesota
• Study Sponsor: Masonic Cancer Center, University of Minnesota
• Collaborators: National Cancer Institute (NCI)

Investigators

• Study Chair: John E. Wagner, MD; Masonic Cancer Center, University of Minnesota

• PRS Account: Masonic Cancer Center, University of Minnesota
• Verification Date: November 2017