We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Try the New Site
We're building a modernized ClinicalTrials.gov! Visit Beta.ClinicalTrials.gov to try the new functionality.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for Hematological Diseases

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01962636
Recruitment Status : Recruiting
First Posted : October 14, 2013
Last Update Posted : November 3, 2022
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
This is a treatment guideline for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematological diseases, including, but not limited to acute leukemias. The myeloablative preparative regimen will consist of cyclophosphamide (CY), fludarabine (FLU) and fractionated total body irradiation (TBI).

Condition or disease Intervention/treatment Phase
Acute Myeloid Leukemia (AML) Acute Lymphocytic Leukemia (ALL) Chronic Myelogenous Leukemia Plasma Cell Leukemia Myelofibrosis Myelodysplasia Chronic Lymphocytic Leukemia Small Lymphocytic Lymphoma Marginal Zone B-Cell Lymphoma Follicular Lymphoma Lymphoplasmacytic Lymphoma Mantle-Cell Lymphoma Prolymphocytic Leukemia Diffuse Large B Cell Lymphoma Lymphoblastic Lymphoma Burkitt's Lymphoma Non-Hodgkin Lymphoma Multiple Myeloma Drug: Fludarabine Drug: Cyclophosphamide Radiation: Total Body Irradiation Drug: Cyclosporine A Drug: Mycophenylate mofetil Biological: Umbilical cord blood Not Applicable

Detailed Description:

This is a study to collect routine clinical data from UCBT using unrelated single or double UCB units as an alternative, non-HLA-matched stem cell source for patients with hematological diseases.

  • data collection from transplant preparative therapy consisting of treatments with chemotherapeutic regimens and total body irradiation.
  • data collection from umbilical cord blood selection and infusion.
  • data collection from standard supportive disease and transplant related care.

Pre- and post-transplant medication, UCB selection and infusion, supportive care, and follow-up will be according to the current University of Minnesota BMT guidelines.

An average of 18 patients are expected to be treated on this protocol per year.

Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 200 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for the Treatment of Hematological Diseases
Study Start Date : December 2016
Estimated Primary Completion Date : October 2023
Estimated Study Completion Date : October 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Blood Disorders

Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
Experimental: Umbilical Cord Blood Transplant
The myeloablative preparative regimen will consist of cyclophosphamide (CY), fludarabine (FLU) and fractionated total body irradiation (TBI)followed by umbilical cord blood transplant. Immunosuppressive Cyclosporine and Mycophenylate Mofetil (MMF) will be administered pre- and post UCBT.
 Drug: Fludarabine
25 mg/m^2 IV of Fludarabine will be given over 1 hour on days -8, -7, and -6 pre-UCB transplant.
Other Name: Fludara

Drug: Cyclophosphamide
60 mg/kg IV of Cyclophosphamide will be given over 2 hours on days -7 and -6 pre-UCB transplant.
Other Name: Cytoxan

Radiation: Total Body Irradiation
165 cGy of total body irradiation will be given twice a day on days -4, -3, -2, and -1.

Drug: Cyclosporine A
Cyclosporine A (CSA) will start day -3 and will be administered PO/IV maintaining a trough level between 200 and 400 ng/mL. For adults the initial dose will be 2.5 mg/kg IV over 1 hour every 12 hours. For children < 40 kg the initial dose will be 2.5 mg/kg IV over 1 hour every 8 hours.
Other Name: CSA

Drug: Mycophenylate mofetil
Mycophenylate mofetil (MMF) 3 gram/day IV/PO for patients who are ≥ 40 kg divided in 2 or 3 doses. Pediatric patient (<40 kilograms) will receive MMF at the dose of 15 mg/kg/dose every 8 hours beginning day -3.
Other Name: MMF

Biological: Umbilical cord blood
Pre-medications and UCB infusion will be per current institutional policies/guidelines. The infusion of the first UCB unit should begin within 15 minutes, and no later than 30 minutes after arrival on the Unit. If 2 units are used, both cords will be infused within 30-60 minutes of each other as deemed clinically safe by the BMT attending or designee.
Other Name: UCB


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Survival at 1 year post-transplant [ Time Frame: 1 year ]
    The number of patients that are still living 1 year after UCBT.


Secondary Outcome Measures :
  1. Incidence of neutrophil engraftment at day 42. [ Time Frame: 42 days ]
    Number of subjects with neutrophil engraftment at day 42 post UCBT.

  2. Platelet engraftment at 1 year. [ Time Frame: 1 year ]
    Number of patients with platelet engraftment at 1 year post UCBT.

  3. Pattern of chimerism after transplant. [ Time Frame: 1 year ]
    Pattern of chimerism after transplant. Chimerism will be plotted with box-plots and described over time.

  4. Incidence of graft failure. [ Time Frame: 100 days ]
    Cumulative incidence of graft failure after UCBT.

  5. Incidence of acute graft versus host disease at 100 days. [ Time Frame: 100 days ]
    Cumulative incidence will be used to estimate acute graft versus host disease 100 days after UCBT.

  6. Incidence of chronic graft versus host disease at 1 year. [ Time Frame: 1 year ]
    Cumulative incidence will be used to estimate chronic GVHD at 1 year post UCBT.

  7. Incidence of transplant related mortality at 6 months. [ Time Frame: 6 months ]
    Cumulative incidence will be used to estimate transplant related mortality at 6 months post UCBT.

  8. Incidence of disease free survival [ Time Frame: 1, 2 years ]
    Kaplan-Meier curves will be used to estimate disease-free survival at 1 and 2 years post UCBT.

  9. Incidence of overall survival. [ Time Frame: 1, 2 years ]
    Kaplan-Meier curves will be used to estimate overall survival at 1 and 2 years post UCBT.


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   up to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Eligible Disease Status

    • Acute Myeloid Leukemia (AML): high risk CR1 (as evidenced by preceding MDS, high risk cytogenetics, ≥ 2 cycles to obtain CR, erythroblastic or megakaryocytic leukemia; CR2+. All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
    • Very high risk pediatric patients with AML: Patients <21 years, however, are eligible with (M2 marrow) with < 25% blasts in marrow after having failed one or more cycles of chemotherapy.
    • Acute Lymphocytic Leukemia (ALL): high risk CR1 as defined by cytogenetics (such as t(9;22), t (1:19), t(4;11), other MLL rearrangements, hypodiploidy, or IKZF1 abnormalities), DNA index < 0.81, > 1 cycle to obtain CR or presence minimal residual disease (MRD). Patients in CR2+ are eligible. All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
    • Very high risk pediatric patients with ALL: patients <21 years are also considered high risk CR1 if they had M2 or M3 marrow at day 42 from the initiation of induction or M3 marrow at the end of induction. They are eligible once they achieved a complete remission.
    • Chronic Myelogenous Leukemia excluding refractory blast crisis: To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to imatinib mesylate.
    • Plasma Cell Leukemia after initial therapy, who achieved at least a partial remission
    • Advanced Myelofibrosis
    • Myelodysplasia (MDS) IPSS INT-2 or High Risk (i.e. RAEB, RAEBt) or Refractory Anemia with severe pancytopenia or high risk cytogenetics: Blasts must be < 10% by a representative bone marrow aspirate morphology.
    • Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Marginal Zone B-Cell Lymphoma or Follicular Lymphoma are eligible if there was disease progression/relapse within 12 of achieving a partial or complete remission. Patients who had remissions lasting > 12 months, are eligible after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm) should be considered for de-bulking chemotherapy before transplant.
    • Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia are eligible after initial therapy in CR1+ or PR1+.
    • Large Cell NHL > CR2/> PR2: Patients in CR2/PR2 with initial short remission (<6 months) are eligible.
    • Lymphoblastic Lymphoma, Burkitt's Lymphoma, and other high-grade NHL after initial therapy if stage III/IV in CR1/PR1 or after progression if stage I/II < 1 year.
    • Multiple Myeloma beyond PR2: Patients with chromosome 13 abnormalities, first response lasting less than 6 months, or β-2 microglobulin > 3 mg/L, may be considered for this protocol after initial therapy.
    • Myeloproliferative Syndromes
  • Availability of suitable UCB unit(s)
  • 0 to 55 years
  • Voluntary written consent (adult or parental/guardian)

Exclusion Criteria:

  • previous irradiation that precludes the safe administration of TBI - Radiation Oncology will evaluate all patients who have had previous radiation therapy
  • chemotherapy refractory large cell and high grade NHL (ie progressive disease after > 2 salvage regimens)
  • if ≤ 18 years old, prior myeloablative transplant within the last 6 months. If >18 years old prior myeloablative allotransplant or autologous transplant
  • extensive prior therapy including > 12 months alkylator therapy or > 6 months alkylator therapy with extensive radiation
  • pregnant or breastfeeding
  • HIV positive
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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 ClinicalTrials.gov identifier (NCT number): NCT01962636


Contacts
Layout table for location contacts
Contact: Claudio Brunstein, MD 612-625-3918 bruns072@umn.edu

Locations
Layout table for location information
United States, Minnesota
University of Minnesota Masonic Cancer Center Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Claudio Brunstein, MD    612-625-3918    bruns072@umn.edu   
Principal Investigator: Claudio Brunstein, MD         
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
Layout table for investigator information
Principal Investigator: Claudio Brunstein, MD University of Minnesota
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT01962636    
Other Study ID Numbers: 2013OC013
First Posted: October 14, 2013    Key Record Dates
Last Update Posted: November 3, 2022
Last Verified: November 2022
Keywords provided by Masonic Cancer Center, University of Minnesota:
Umbilical Cord Transplant
Acute Myeloid Leukemia (AML)
Acute Lymphocytic Leukemia (ALL)
Chronic Myelogenous Leukemia
Plasma Cell Leukemia
Myelofibrosis
Myelodysplasia
Chronic Lymphocytic Leukemia
Small Lymphocytic Lymphoma
Marginal Zone B-Cell Lymphoma
 Follicular Lymphoma
Lymphoplasmacytic Lymphoma
Mantle-Cell Lymphoma
Prolymphocytic Leukemia
Large Cell Non-Hodgkin Lymphoma
Lymphoblastic Lymphoma
Burkitt's Lymphoma
Non-Hodgkin Lymphoma
Multiple Myeloma
Additional relevant MeSH terms:
Layout table for MeSH terms
Burkitt Lymphoma
Lymphoma
Leukemia
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Multiple Myeloma
Lymphoma, Follicular
Lymphoma, Non-Hodgkin
Leukemia, Lymphoid
Lymphoma, B-Cell
Leukemia, Lymphocytic, Chronic, B-Cell
Lymphoma, Mantle-Cell
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Lymphoma, Large B-Cell, Diffuse
Precursor Cell Lymphoblastic Leukemia-Lymphoma
 Lymphoma, B-Cell, Marginal Zone
Leukemia, Prolymphocytic
Preleukemia
Waldenstrom Macroglobulinemia
Leukemia, Plasma Cell
Primary Myelofibrosis
Myelodysplastic Syndromes
Hematologic Diseases
Neoplasms by Histologic Type
Neoplasms
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Neoplasms, Plasma Cell