Clinical trial NCT00514579: Adult Double Cord Blood Transplant Study
Official title: A Phase II Multicenter Trial of Myeloablative Double Unit Umbilical Cord Blood Transplantation (UCBT) in Adults With Hematologic Malignancy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cord Blood Stem Cell Transplantation
Hematologic Malignancies
Keywords: Cord Blood Stem Cell Transplantation
Hematologic Malignancies
Leukemia
Myelodysplastic Syndrome
Lead sponsor: Center for International Blood and Marrow Transplant Research
Sponsor class: NETWORK
First posted: 2007-08-10
Enrollment: 56
Sex: ALL
Minimum age (years): 22
Maximum age (years): 50
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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%.


Eligibility criteria: 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

Primary outcomes:
measure: Measure overall survival of double unit UCBT in adult patients with hematologic malignancies
timeFrame: One year
Secondary outcomes:
measure: Measure incidence of donor-derived neutrophil and platelet recovery
timeFrame: 100 Days
measure: Measure contribution of each unit to initial and sustained engraftment
timeFrame: 2 years
measure: Measure incidence and severity of acute graft-versus-host disease
timeFrame: 100 Days
measure: Measure incidence and severity of chronic GVHD
timeFrame: 1 year
measure: Measure incidence of transplant-related mortality
timeFrame: 6 months
measure: Measure incidence of malignant relapse
timeFrame: 2 years
measure: Measure incidence of serious infectious complications
timeFrame: 1 year
measure: Measure incidence of immune reconstitution
timeFrame: 2 years
measure: Measure probability of overall and disease-free survival
timeFrame: 2 years
Other outcomes:
Locations:
city: Duarte
country: United States
facility: City of Hope
state: California
city: Los Angeles
country: United States
facility: University of California at Los Angeles
state: California
city: Tampa
country: United States
facility: H. Lee Moffitt Cancer Center and Research Institute
state: Florida
city: Atlanta
country: United States
facility: Blood and Marrow Transplant Program at Northside Hospital
state: Georgia
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: Case Western Reserve University
state: Ohio
city: Columbus
country: United States
facility: Ohio State University Medical Center
state: Ohio
city: Oklahoma City
country: United States
facility: University of Oklahoma
state: Oklahoma
city: Madison
country: United States
facility: University of Wisconsin-Madison
state: Wisconsin
Study officials:
affiliation: Memorial Sloan Kettering Cancer Center
name: Juliet Barker, MBBS
role: PRINCIPAL_INVESTIGATOR
References: