Unrelated Umbilical Cord Blood (UBC)Transplantation

NCT01768845 | Completed Results DMC

• 1 other identifier: WVU 1909
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 1

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).

Conditions

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

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Engraftment

  Defined as neutrophil recovery associated with donor engraftment within the first 60 days of transplant

  60 days

Secondary Outcomes (1)

• Overall Survival at Day 180 Post-transplant

  180 days

Study Arms (1)

Transplant

EXPERIMENTAL

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.

Genetic: umbilical cord blood (UCB)

Interventions

umbilical cord blood (UCB) GENETIC

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.

Transplant

Eligibility Criteria

• Age: 16 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• West Virginia University: lead

Study Sites (1)

West Virginia University Hospitals Mary Babb Randolph Cancer Center

Morgantown, West Virginia, 26506, United States

Location

MeSH Terms

Conditions

Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Leukemia, Myeloid, Acute; Myelodysplastic Syndromes; Multiple Myeloma; Hodgkin Disease; Lymphoma, Non-Hodgkin; Leukemia, Lymphocytic, Chronic, B-Cell; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Anemia, Aplastic

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Neoplasms, Plasma Cell; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Lymphoma; Lymphatic Diseases; Leukemia, B-Cell; Leukemia, Lymphoid; Anemia; Bone Marrow Failure Disorders

Results Point of Contact

• Title: Michael Craig, MD
• Organization: West Virginia Universtiy

craigm@wvumedicine.org

304-293-4980

Study Officials

• Michael Craig, MD

  West Virginia University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 11, 2013
• First Posted: January 15, 2013
• Study Start: February 3, 2009
• Primary Completion: May 14, 2019
• Study Completion: April 6, 2021
• Last Updated: June 14, 2021
• Results First Posted: June 8, 2021

Record last verified: 2021-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)