Phase I Study of Sequential Cord Blood Transplants

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00299767
Recruitment Status : Completed
First Posted : March 7, 2006
Last Update Posted : May 11, 2016
Dana-Farber Cancer Institute
Information provided by (Responsible Party):
Karen Ballen, Massachusetts General Hospital

Brief Summary:
The purpose of this study is to determine the safety of sequential cord blood transplantation (2 cord blood units) for patients who have diseases that are capable of being cured by allogeneic stem cell transplant but do not have a matched family or volunteer unrelated donor.

Condition or disease Intervention/treatment Phase
Lymphoma Leukemia Multiple Myeloma Myelodysplastic Syndrome Procedure: sequential cord blood transplantation Phase 1

Detailed Description:
Eligible patients will receive conditioning therapy with fludarabine 30 mg/m2/day x 6 days, melphalan 100 mg/m2/day x 1 day, rabbit antithymocyte globulin 1.5 mg/kg/day x 4 days. GVHD prophylaxis will consist of cyclosporine starting on day -1 and mycophenolate mofetil starting on day 0. Sequential cord blood units will be infused on Day 0.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 21 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Study of Sequential Cord Blood Transplants
Study Start Date : May 2003
Actual Primary Completion Date : May 2005
Actual Study Completion Date : May 2009

Intervention Details:
    Procedure: sequential cord blood transplantation
    Infused on Day 0

Primary Outcome Measures :
  1. To evaluate the 100-day transplant-related (non-relapse) mortality, including relapse-related mortality associated with Grade 4 toxicity. [ Time Frame: 3 years ]

Secondary Outcome Measures :
  1. To evaluate the 6 month transplant related (non-relapse) mortality. [ Time Frame: 3 years ]
  2. To evaluate the days to neutrophil engraftment (ANC > 500). [ Time Frame: 3 years ]
  3. To evaluate the days of platelet engraftment (platelet count > 20K unsupported). [ Time Frame: 3 years ]
  4. To evaluate the risk of acute and chronic graft versus host disease. [ Time Frame: 3 years ]
  5. To evaluate percent donor chimerism - contribution of each cord unit. [ Time Frame: 3 years ]
  6. To evaluate relapse rate.
  7. To evaluate overall survival.
  8. To evaluate transfusion support needed to cord blood transplant recipients. [ Time Frame: 3 years ]

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Ages Eligible for Study:   up to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Disease Status: NHL, HD, or MM refractory to chemotherapy or relapsed; CLL Rai Stage III/IV, or lymphocyte doubling time of 6 months, or Stage I/II resistant to 2 chemotherapy regimens; AML or ALL in second or subsequent remission or in first remission with adverse cytogenetics or antecedent hematologic disorder
  • Estimated disease-free survival of less than one year
  • ECOG performance status of 0, 1, 2
  • Lack of 6/6 or 5/6 HLA matched related or 6/6 matched unrelated donor, or a donor is not available within the time frame necessary to perform a potentially curative stem cell transplant
  • 4/6 or greater HLA A, B, CRB1 cord blood units with a combined nucleated cell dose from of > 3.7 x 10(7) NC/kg

Exclusion Criteria:

  • Cardiac disease: symptomatic congestive hearth failure or RVG or echocardiogram determined left ventricular ejection fraction of < 45%, active angina pectoris, or uncontrolled hypertension
  • Pulmonary Disease: severe chronic obstructive lung disease, or symptomatic restrictive lung disease, or corrected DLCO of < 50%
  • Renal disease: serum creatinine > 2.0 mg/dl
  • Hepatic disease: serum bilirubin > 2.0 mg/dl (except in the case of Gilbert's syndrome), SGPT or SGOT > 3 x normal
  • Neurologic disease: symptomatic leukoencephalopathy, active DNS malignancy or other neuropsychiatric abnormalities believed to preclude transplantation
  • HIV antibody or Hepatitis B surface antigen positivity
  • Uncontrolled infection
  • Pregnancy or breast-feeding mother

Information from the National Library of Medicine

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 identifier (NCT number): NCT00299767

United States, Massachusetts
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Massachusetts General Hospital
Boston, Massachusetts, United States, 02116
Sponsors and Collaborators
Massachusetts General Hospital
Dana-Farber Cancer Institute
Principal Investigator: Karen Ballen, M.D. Massachusetts General Hospital, Harvard University
Principal Investigator: Joseph Antin, M.D. Dana Farber Cancer Institute, Harvard Univeristy
Principal Investigator: David Avigan, M.D. Beth Israel Deaconess Medical Center

Responsible Party: Karen Ballen, Director, Leukemia Program, Massachusetts General Hospital Identifier: NCT00299767     History of Changes
Other Study ID Numbers: 03-061
First Posted: March 7, 2006    Key Record Dates
Last Update Posted: May 11, 2016
Last Verified: May 2016

Additional relevant MeSH terms:
Multiple Myeloma
Myelodysplastic Syndromes
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Bone Marrow Diseases
Precancerous Conditions