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Reduced Intensity Double Umbilical Cord Blood Transplantation

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Zachariah Michael DeFilipp, Massachusetts General Hospital Identifier:
First received: August 2, 2011
Last updated: March 1, 2017
Last verified: March 2017

This trial will use two cord blood units for transplantation using a reduced intensity regimen rather than using intense doses of chemotherapy and radiation therapy. Two cord blood units (double cord blood) are being used, as the numbers of blood cells in one unit are too few to allow successful growth of these cells.

Because the risk of infection, particularly virus infection, is high after double cord blood transplant, this study seeks to reduce the rise of virus infection by using a reduced intensity regimen without a medicine called antithymocyte globulin (ATG), as used in prior cord blood transplants. Subjects will receive two chemotherapy drugs, melphalan and fludarabine, and low dose of total body radiation (one treatment) instead of the ATG. The number of patients with virus infections in this study will be compared to our prior experience using the ATG.

Condition Intervention Phase
Non-Hodgkin's Lymphoma Hodgkin's Lymphoma Multiple Myeloma Chronic Lymphocytic Leukemia Acute Myelogenous Leukemia Drug: Fludarabine Drug: Melphalan Radiation: Total Body Radiation Biological: Cord Blood Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Study of Reduced Intensity Double Umbilical Cord Blood Transplantation Using Fludarabine, Melphalan, and Low Dose Total Body Radiation

Resource links provided by NLM:

Further study details as provided by Zachariah Michael DeFilipp, Massachusetts General Hospital:

Primary Outcome Measures:
  • One year significant viral infection rate [ Time Frame: 2.5 years ]
    To determine the one year significant viral infection rate (viral infections requiring medical intervention) after double umbilical cord blood transplant using a novel conditioning regimen of fludarabine/melphalan/low dose total body radiation

Secondary Outcome Measures:
  • Time to neutrophil and platelet engraftment [ Time Frame: 2.5 years ]
  • Rate of primary graft failure [ Time Frame: 2.5 years ]
  • Rates of Grade II-IV and Grade III-IV acute GVHD at 100 days [ Time Frame: 2.5 years ]
  • The rate of chronic GVHD [ Time Frame: 2.5 years ]
  • 100-day treatment related mortality [ Time Frame: 2.5 years ]
  • Immune reconstitution - CD4 count at 12 months [ Time Frame: 2.5 years ]
  • Relapse-free and overall survival at 1 and 2 years from transplantation [ Time Frame: 2.5 years ]
  • Relapse rate [ Time Frame: 2.5 years ]
  • Rate of post-transplant lymphoma [ Time Frame: 2.5 years ]
  • Thrombopoietin levels after transplant [ Time Frame: 2.5 years ]
    Optional correlate

Enrollment: 33
Study Start Date: December 2011
Estimated Study Completion Date: February 2019
Estimated Primary Completion Date: February 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Fludarabine/Melphalan/TBI
All patients receive same therapy
Drug: Fludarabine
30 mg/m2/day IV x 6 days
Other Name: Fludara
Drug: Melphalan
100 mg/m2/day IV x 1 day
Radiation: Total Body Radiation
200 cGy on Day 0
Biological: Cord Blood
2 cord blood units IV

Detailed Description:

Subjects will receive their transplants as in-patients.

  • IV-Catheter

    • one or two IV catheters will be placed on the day of hospital admission
  • Conditioning

    • Fludarabine IV six days before transplant (days -7, -6, -5. -4, -3, -2)
    • Melphalan IV (day -1)
    • Total body radiation on day 0 (same day as transplant)
  • Immunosuppressive Therapy

    • Tacrolimus and sirolimus beginning day -3, daily for 6-9 months post-transplant. Given IV as in-patient, orally as out-patient
  • Infusion of Cord Blood units

    • 2 cord blood units IV on Day 0 Routine post-transplant supportive care will be provided

Ages Eligible for Study:   18 Years to 70 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Hematologic malignancy for whom allogeneic stem cell transplantation is deemed clinically appropriate
  • Appropriate candidate for reduced intensity regimen, according to the treating physician
  • Lack of 6/6/ or 5/6 HLA-matched related, 8/8/ HLA-matched unrelated donor, or unrelated donor not available with a time frame necessary to perform a potentially curative stem cell transplant
  • Able to comply with the requirements for care after allogeneic stem cell transplantation

Exclusion Criteria:

  • Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular dysfunction
  • Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic restrictive lung disease
  • Renal disease
  • Hepatic disease
  • Neurologic disease: symptomatic leukoencephalopathy, active CNS malignancy or other neuropsychiatric abnormalities believed to preclude transplantation
  • HIV-positive
  • Uncontrolled infection
  • Pregnant or breast-feeding
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01408563

United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States, 02215
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Sponsors and Collaborators
Massachusetts General Hospital
Principal Investigator: Karen Ballen, MD Massachusetts General Hospital
  More Information

Responsible Party: Zachariah Michael DeFilipp, Hematology/Oncology, Massachusetts General Hospital Identifier: NCT01408563     History of Changes
Other Study ID Numbers: 11-085
Study First Received: August 2, 2011
Last Updated: March 1, 2017

Keywords provided by Zachariah Michael DeFilipp, Massachusetts General Hospital:
myelodysplastic disorder
aplastic anemia

Additional relevant MeSH terms:
Multiple Myeloma
Lymphoma, Non-Hodgkin
Leukemia, Lymphoid
Leukemia, Lymphocytic, Chronic, B-Cell
Leukemia, Myeloid
Hodgkin Disease
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Neoplasms, Plasma Cell
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Leukemia, B-Cell
Fludarabine phosphate
Antineoplastic Agents
Antimetabolites, Antineoplastic processed this record on September 20, 2017