Unrelated Umbilical Cord Blood (UBC)Transplantation

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Brief Title

Unrelated Umbilical Cord Blood (UBC)Transplantation

Official Title

Unrelated Umbilical Cord Blood (UCB) Transplantation

Brief Summary

      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).
    

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-Stem Cell
      Transplant (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 hematopoietic cell
      transplantation-specific comorbidity index (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.
    


Study Type

Interventional


Primary Outcome

Number of Participants With Engraftment

Secondary Outcome

 Overall Survival at Day 180 Post-transplant

Condition

Chronic Myelogenous Leukemia (CML)

Intervention

umbilical cord blood (UCB)

Study Arms / Comparison Groups

 Transplant
Description:  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 variable number tandem repeat (VNTR) analysis. Immune reconstitution (Digeorge Panel) will also be checked at these time points.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Genetic

Estimated Enrollment

34

Start Date

February 3, 2009

Completion Date

April 6, 2021

Primary Completion Date

May 14, 2019

Eligibility 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.

          -  Eastern Cooperative Oncology Group (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 Bone Marrow
             Transplant (BMT)
      

Gender

All

Ages

16 Years - 70 Years

Accepts Healthy Volunteers

No

Contacts

Michael Craig, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01768845

Organization ID

WVU 1909


Responsible Party

Sponsor

Study Sponsor

West Virginia University


Study Sponsor

Michael Craig, MD, Principal Investigator, West Virginia University


Verification Date

June 2021