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Unrelated Umbilical Cord Blood (UBC)Transplantation

Primary Purpose

Chronic Myelogenous Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome

Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
umbilical cord blood (UCB)
Sponsored by
West Virginia University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Chronic Myelogenous Leukemia (CML) focused on measuring 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

Eligibility Criteria

16 Years - 70 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

  • West Virginia University Hospitals Mary Babb Randolph Cancer Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Transplant

Arm 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.

Outcomes

Primary Outcome Measures

Number of Participants With Engraftment
Defined as neutrophil recovery associated with donor engraftment within the first 60 days of transplant

Secondary Outcome Measures

Overall Survival at Day 180 Post-transplant
Number of Participants with Overall survival at day 180 transplant.

Full Information

First Posted
January 11, 2013
Last Updated
June 7, 2021
Sponsor
West Virginia University
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1. Study Identification

Unique Protocol Identification Number
NCT01768845
Brief Title
Unrelated Umbilical Cord Blood (UBC)Transplantation
Official Title
Unrelated Umbilical Cord Blood (UCB) Transplantation
Study Type
Interventional

2. Study Status

Record Verification Date
June 2021
Overall Recruitment Status
Completed
Study Start Date
February 3, 2009 (Actual)
Primary Completion Date
May 14, 2019 (Actual)
Study Completion Date
April 6, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
West Virginia University

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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
Keywords
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
34 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Transplant
Arm Type
Experimental
Arm 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.
Intervention Type
Genetic
Intervention Name(s)
umbilical cord blood (UCB)
Intervention Description
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.
Primary Outcome Measure Information:
Title
Number of Participants With Engraftment
Description
Defined as neutrophil recovery associated with donor engraftment within the first 60 days of transplant
Time Frame
60 days
Secondary Outcome Measure Information:
Title
Overall Survival at Day 180 Post-transplant
Description
Number of Participants with Overall survival at day 180 transplant.
Time Frame
180 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Michael Craig, MD
Organizational Affiliation
West Virginia University
Official's Role
Principal Investigator
Facility Information:
Facility Name
West Virginia University Hospitals Mary Babb Randolph Cancer Center
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26506
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

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Unrelated Umbilical Cord Blood (UBC)Transplantation

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