Cord Blood Transplant

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2013 by West Virginia University
Sponsor:
Information provided by (Responsible Party):
Michael Craig, MD, West Virginia University
ClinicalTrials.gov Identifier:
NCT01768845
First received: January 11, 2013
Last updated: NA
Last verified: January 2013
History: No changes posted
  Purpose

Hematopoietic progenitor cell (HPC- primitive cells in the blood, bone marrow and umbilical cord that can restore the bone marrow) transplant can be a curative therapy for the treatment of hematologic malignancies (a disease of the bone marrow and lymph nodes). The source of cells used for the transplant comes from related (sibling) and in cases where there is no sibling match, from unrelated donors through the National Marrow Donor Program. The availability of a suitable donor can be a significant obstacle for patients who need a transplant but do not have a matched donor. Cord blood that has been harvested from an umbilical cord shortly after birth has a rich supply of cells needed for transplant. These stored cord bloods are now being used to transplant adults without a matched donor

Advantages to using cord blood includes a readily available source of cells with no risk to the donor during the collection process, immediate source of cells in urgent situations (no lengthy donor work-up)and a reduction in infectious disease transmission to the recipient.

One of the main disadvantages is the cord blood has a small number of cells needed for transplant. In an adult, usually two cords are needed and large recipients do not qualify because they need too many cells.

This study will use two different preparative regimens (chemotherapy and radiation) followed by one or two umbilical cord units (UBC). The preparative regimen used will be chosen by the physician and is based on patient's age, disease and medical condition at the time of transplant.

Multiple objectives for this study include disease-free and overall survival, treatment related mortality, rate of cells taking hold, and the incidence and severity of the transplant complication called graft versus host disease (GVHD).


Condition Intervention Phase
Chronic Myelogenous Leukemia (CML)
Acute Myelogenous Leukemia (AML)
Myelodysplastic Syndrome
Multiple Myeloma
Hodgkin Lymphoma
Non-Hodgkin Lymphoma
Chronic Lymphocytic Leukemia (CLL)
Acute Lymphocytic Leukemia (ALL)
Severe Aplastic Anemia
Genetic: umbilical cord transplant (UCB)
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Unrelated Umbilical Cord Blood (UCB) Transplantation

Resource links provided by NLM:


Further study details as provided by West Virginia University:

Primary Outcome Measures:
  • Engraftment [ Time Frame: 60 days ] [ Designated as safety issue: No ]
    Defined as neutrophil recovery associated with donor engraftment within the first 60 days of transplant


Secondary Outcome Measures:
  • Overall survival [ Time Frame: 180 days ] [ Designated as safety issue: Yes ]
    Overall survival at day 180 transplant.


Estimated Enrollment: 40
Study Start Date: February 2009
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Transplant
After a preparative regimen the patient will receive an infusion of one or two umbilical cord blood unit(s) (UBC). The UBC unit(s) will be thawed according to methods of Rubinstein et al. If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis. On day +30, +60, +100, +180, and +365 the chimeric status of patients will be interpreted by VNTR analysis. Immune reconstitution (Digeorge Panel) will also be checked at these time points.
Genetic: umbilical cord transplant (UCB)
Infusion will occur after preparative regimen in one or two UCB unit(s). If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis.

Detailed Description:

Allogeneic hematopoietic cell transplantation (allo- HCT) is a curative therapy for the treatment of hematological and non-hematological malignancies and certain non-malignant conditions. Bone marrow or peripheral blood from a Human Leukocyte Antigen (HLA) matched sibling donor is the most commonly used source of allogeneic stem cells. However, HLA matched siblings are available for less than one third of the patients who require allo- SCT. In the absence of an HLA matched sibling, volunteer unrelated donors or partially mismatched related donors (PMRD), stored cord blood may be used as a source of allogeneic stem cells. Stored cord blood has been used as a source of allogeneic stem cells in infants and children, but had early skepticism in adults because of concerns about the engraftment potential of the relatively limited number of stem cells. The number of stem cells in a unit of cord blood is generally one log less than the number of stem cells on an average collection of bone marrow from an adult for transplantation.

After the success of the first allogeneic umbilical cord blood transplantation in 1988, programs for banking screened unrelated donor CBSC have been initiated both in the United States and Europe. Dr Pablo Rubenstein started the first such bank at the New York Blood Center (NYBC) in 1993. Since its inception, the NYBC has provided unrelated donor cord blood stem cells for over 1000 transplants. Analysis of outcomes for the initial 562 transplant recipients from the NYBC revealed a cumulative rate of engraftment of 81% by day 42 for PMNs. and 85% by day 180 for platelets. Currently, approximately more than 100,000 cord blood units are available in cord blood banks worldwide and more than 2000 patients have received cord blood transplants from these banks. NetCord, an international cooperative group of cord blood banks, has developed a detailed set of standards for cord blood banking to facilitate international exchanges and to guarantee the quality of these products.

Cord Blood Unit Selection:

UCB units will be required to be a 4 to 6 of 6 HLA-A, -B antigen and -DRB1 allele match with the patient. Typing at HLA-C and -DQ will be obtained but not required in the match strategy. A minimum total nucleated cell (TNC) dose of >2.0 x 107/kg at the time of freezing will be utilized when possible. When using double units, each unit should contain a minimum pre-cryopreserved TNC dose of 1.5 x 107/kg.

UCB Transplant Procedure:

There will be a myeloablative and reduced-intensity preparative regimen that can be given prior to infusion of cord product. The myeloablative approach will be selected in younger patients (<50yo) with a HCT-CI score <3. The reduced-intensity regimen will be selected for all older patients (>50) or younger patients with a HCT-CI score >3. The reduced-intensity regimen will also be chosen for any patients being transplanted for indolent/follicular lymphomas, CLL, myeloma, or Hodgkin lymphoma; irrelevant of age or HCT-CI score. On a case by case basis, patients may receive a preparative regimen outside of their designated category as noted above with the approval of the PI, if deemed in the patient's best interest.

  Eligibility

Ages Eligible for Study:   16 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age: 16-70 years
  • Available 4/6, 5/6, or 6/6 HLA antigen match (using A, B, and DRB1) cord blood unit.
  • ECOG performance status of 0-2 (Karnofsky greater than or equal to 70%)
  • Serum bilirubin less than 2 x upper limit of normal
  • Serum creatinine less than 2 mg/dl
  • DLCO or FEV1 greater than or equal to 50% predicted
  • Left ventricular ejection fraction greater than or equal to 35%
  • no uncontrolled infection
  • If female, not pregnant
  • Informed consent given
  • No major organ dysfunction precluding transplantation.
  • One of the following malignancies or bone marrow failure syndromes:

    • Chronic myelogenous leukemia (CML)
    • Acute myelogenous leukemia (AML)
    • Myelodysplastic syndrome
    • Multiple myeloma
    • Hodgkin lymphoma
    • Non-Hodgkin lymphoma
    • Chronic lymphocytic leukemia (CLL)
    • Acute lymphocytic leukemia (ALL)
    • Severe Aplastic Anemia

Exclusion Criteria:

  • Patient pregnant
  • Age less than 16, greater than 70
  • ECOG performance status of greater than 2 (Karnofsky less than 70%)
  • Psychiatric disorder or mental deficiency of the patient sufficiently severe as to make compliance with the BMT treatment unlikely, or making informed consent impossible
  • Serum bilirubin greater than or equal to 2 x upper limit of normal, transaminases greater than 3 x upper limit of normal
  • Serum creatinine greater than or equal to 2 mg/dl
  • DLCO less than 50% predicted
  • Left ventricular ejection fraction less than 35%
  • Major anticipated illness or organ failure incompatible with survival from BMT
  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 ClinicalTrials.gov identifier: NCT01768845

Contacts
Contact: Pam Bunner, MT, CCRC 304-598-4511 bunnerp@wvuhealthcare.com
Contact: Crystal Street 304-598-4500 streetc@wvuhealthcare.com

Locations
United States, West Virginia
West Virginia University Hospitals Mary Babb Randolph Cancer Center Recruiting
Morgantown, West Virginia, United States, 26506
Principal Investigator: Michael Craig, MD         
Sub-Investigator: Aaron Cumpston, PharmD         
Sub-Investigator: Jame Abraham, MD         
Sub-Investigator: Scot Remick, MD         
Sub-Investigator: Mehdi Hamdani, MD         
Sub-Investigator: William Tse, MD         
Sub-Investigator: Soumit Basu, MD         
Sponsors and Collaborators
Michael Craig, MD
Investigators
Principal Investigator: Michael Craig, MD West Virginia University
  More Information

No publications provided

Responsible Party: Michael Craig, MD, Associate Professor, West Virginia University
ClinicalTrials.gov Identifier: NCT01768845     History of Changes
Other Study ID Numbers: WVU 1909
Study First Received: January 11, 2013
Last Updated: January 11, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by West Virginia University:
Chronic myelogenous leukemia (CML)
Acute myelogenous leukemia (AML)
Myelodysplastic syndrome
Multiple myeloma
Hodgkin lymphoma
Non Hodgkin lymphoma
Chronic lymphocytic leukemia (CLL)
Acute lymphocytic leukemia (ALL)
Severe Aplastic Anemia
Cord Blood
Umbilical cord blood transplantation
UBC transplantation

Additional relevant MeSH terms:
Anemia, Aplastic
Hodgkin Disease
Leukemia
Leukemia, Lymphocytic, Chronic, B-Cell
Leukemia, Lymphoid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Lymphoma
Lymphoma, Non-Hodgkin
Multiple Myeloma
Myelodysplastic Syndromes
Neoplasms, Plasma Cell
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Preleukemia
Syndrome
Anemia
Blood Protein Disorders
Bone Marrow Diseases
Cardiovascular Diseases
Disease
Hematologic Diseases
Hemorrhagic Disorders
Hemostatic Disorders
Immune System Diseases
Immunoproliferative Disorders
Leukemia, B-Cell
Lymphatic Diseases
Lymphoproliferative Disorders
Myeloproliferative Disorders

ClinicalTrials.gov processed this record on October 29, 2014