Adult Double Cord Blood Transplant Study

A Phase II Multicenter Trial of Myeloablative Double Unit Umbilical Cord Blood Transplantation (UCBT) in Adults With Hematologic Malignancy

The hypothesis of the study is double unit umbilical cord blood transplantation in adults will be associated with a one year survival rate of at least 40%.

Study Overview

• Status: Completed
• Conditions: Hematologic Malignancies; Cord Blood Stem Cell Transplantation
• Intervention / Treatment: Procedure: Cord blood transplantation

• Study Type: Interventional
• Enrollment (Actual): 56
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Duarte, California, United States, 91010
      • City of Hope
    • Los Angeles, California, United States, 90095
      • University of California at Los Angeles
  • Florida
    • Tampa, Florida, United States, 33612
      • H. Lee Moffitt Cancer Center and Research Institute
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • Blood and Marrow Transplant Program at Northside Hospital
  • New York
    • New York, New York, United States, 10065
      • Memorial Sloan Kettering Cancer Center
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • Case Western Reserve University
    • Columbus, Ohio, United States, 43210
      • Ohio State University Medical Center
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • University of Oklahoma
  • Wisconsin
    • Madison, Wisconsin, United States, 53792
      • University of Wisconsin-Madison

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 22 years to 50 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Age 22 - 50 years

• Patients will have one of the following hematological malignancies:

  • Acute myelogenous leukemia (AML): Complete first remission (CR1) at high risk for relapse or Complete second remission (CR2)
  • Acute lymphoblastic leukemia (ALL): Complete first remission (CR1) at high risk for relapse or Complete second remission (CR2)
  • Acute undifferentiated leukemia (AUL) or biphenotypic leukemia: CR1 or CR2
  • Myelodysplastic Syndrome (MDS) with one of the following: Low and Intermediate-1 International Prognostic Scoring System (IPSS) score with Life-threatening neutropenia or thrombocytopenia; or Platelet transfusion dependence Intermediate-2 or High IPSS score Therapy-related disease: patient with history of chemotherapy and current evidence of MDS
• Patients with adequate organ function and performance status criteria
• Two Suitable Umbilical Cord Blood Units: a cryopreserved dose of at least 1.5 x 107 TNC/kg. If the unit contains red cells at time of cryopreservation, the cryopreserved dose must be at least 2.0 x 107 TNC/kg. Each unit must be at least 4/6 HLA-A and B antigen, and DRB1 allele matched with the recipient. Each unit must be at least 3/6 HLA-A, B DRB1 antigen matched to each other.

Exclusion Criteria:

• Patient with suitable related donor
• AML, ALL, AUL, biphenotypic leukemia beyond CR2
• AML evolved from myelofibrosis

• Any acute leukemia with:

  • Morphologic relapse or persistent disease in the BM
  • Active extra-medullary leukemia including active CNS leukemia
  • Requiring greater than two cycles of chemotherapy to obtain present remission status
• Bone marrow aplasia (defined as BM cellularity < 5% at transplant work-up)
• MDS with 10% or greater bone marrow blasts at pre-transplant workup
• Prior autologous or allogeneic HSC transplant at any time
• Prior radiation therapy rendering patient ineligible for TBI
• Any uncontrolled infection at time of study enrollment
• Seropositive or NAT positive for HIV or HTLV1
• Females who are pregnant or breast feeding
• Patient unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, follow-up, and research tests

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Myeloablative double unit UCBT; Myeloablative preparative regimen of chemotherapy and radiation followed by double unit umbilical cord blood transplantation	Procedure: Cord blood transplantation; Myeloablative preparative regimen of chemotherapy and radiation followed by double unit umbilical cord blood transplantation

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Measure overall survival of double unit UCBT in adult patients with hematologic malignancies	One year

Secondary Outcome Measures

Outcome Measure	Time Frame
Measure incidence of donor-derived neutrophil and platelet recovery	100 Days
Measure contribution of each unit to initial and sustained engraftment	2 years
Measure incidence and severity of acute graft-versus-host disease	100 Days
Measure incidence and severity of chronic GVHD	1 year
Measure incidence of transplant-related mortality	6 months
Measure incidence of malignant relapse	2 years
Measure incidence of serious infectious complications	1 year
Measure incidence of immune reconstitution	2 years
Measure probability of overall and disease-free survival	2 years

Collaborators and Investigators

• Sponsor: Center for International Blood and Marrow Transplant Research
• Investigators: Principal Investigator: Juliet Barker, MBBS, Memorial Sloan Kettering Cancer Center

Study record dates

Study Major Dates

• Study Start: August 1, 2007
• Primary Completion (Actual): August 1, 2013
• Study Completion (Actual): August 1, 2013

Study Registration Dates

• First Submitted: August 8, 2007
• First Submitted That Met QC Criteria: August 9, 2007
• First Posted (Estimate): August 10, 2007

Study Record Updates

• Last Update Posted (Estimate): March 10, 2014
• Last Update Submitted That Met QC Criteria: March 7, 2014
• Last Verified: March 1, 2014

More Information

Terms related to this study

• Keywords: Leukemia; Myelodysplastic Syndrome; Hematologic Malignancies; Cord Blood Stem Cell Transplantation
• Additional Relevant MeSH Terms: Neoplasms by Site; Hematologic Diseases; Neoplasms; Hematologic Neoplasms
• Other Study ID Numbers: 05-DCB

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No