Clinical trial NCT01351545: A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Official title: A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients With Hematologic Malignancies and Other Indications
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hematologic Malignancies
Inherited Disorders of Metabolism
Inherited Abnormalities of Platelets
Histiocytic Disorders
Acute Myelogenous Leukemia (AML or ANLL)
Acute Lymphoblastic Leukemia (ALL)
Other Acute Leukemia
Chronic Myelogenous Leukemia (CML)
Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases
Other Leukemia
Hodgkin Lymphoma
Non-hodgkin Lymphoma
Multiple Myeloma/ Plasma Cell Disorder (PCD)
Inherited Abnormalities of Erythrocyte Differentiation or Function
Disorders of the Immune System
Autoimmune Diseases
Severe Aplastic Anemia
Lead sponsor: Center for International Blood and Marrow Transplant Research
Sponsor class: NETWORK
Collaborators: name: National Marrow Donor Program
First posted: 2011-05-11
Enrollment: 99999
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Recipients of unlicensed cryopreserved cord blood units who are being treated as U.S. transplant centers.

Brief summary: This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.

Detailed description: Principal Investigators:

The principal investigators (PIs) will be transplant physicians at all participating U.S. transplant centers.

Study Design:

This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.

Primary Objective:

The primary objective of this study is to examine the incidence of neutrophil recovery of ≥500/mm3 after cord blood transplantation in a multi-institution setting using CBUs that are not Food and Drug Administration (FDA) licensed.

Secondary Objectives:

In patients receiving a non-licensed CBU:

* Assess incidence of transmission of infection
* Assess incidence of serious infusion reaction
* Determine 1 year overall survival after cord blood transplantation
* Assess cumulative incidence of acute graft vs. host disease (GVHD) grades II to IV and grades III to IV
* Assess cumulative incidence of chronic GVHD
* Determine platelet engraftment of \>20,000 mcL and \>50,000 mcL

Eligibility criteria: Inclusion Criteria:

* Disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment
* Signed informed consent (and signed assent, if applicable) obtained prior to study enrollment
* Pediatric and adult patients of any age

Exclusion Criteria:

* Patients who are receiving only licensed CBUs
* Cord blood transplant recipients at international transplant centers
* Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)
* Patients whose selected unlicensed CBU(s) will be more than minimally manipulated

Primary outcomes:
measure: Neutrophil recovery of ≥500/mm3 after cord blood transplantation
timeFrame: Cord blood engraftment occurs in weeks following transplant is defined as defined as ANC 500/mm3 and is analyzed at 60 days post-transplant.
measure: Neutrophil recovery of ≥500/mm3 after cord blood transplantation
timeFrame: Cord blood engraftment occurs in weeks following transplant is defined as defined as ANC 500/mm3 and is analyzed at 100 days post-transplant.
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Gilbert
country: United States
facility: Banner MD Anderson Cancer Center
state: Arizona
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Phoenix
country: United States
facility: Mayo Clinic Arizona (adults)
state: Arizona
city: Tucson
country: United States
facility: University of Arizona Medical Center - Tucson
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Duarte
country: United States
facility: City of Hope
state: California
city: Glendale
country: United States
facility: Children's Hospital of Los Angeles
state: California
city: La Jolla
country: United States
facility: University of California San Diego
state: California
city: LaJolla
country: United States
facility: Scripps Green Hospital
state: California
city: Loma Linda
country: United States
facility: Loma Linda University Cancer Center
state: California
city: Los Angeles
country: United States
facility: UCLA
state: California
city: Oakland
country: United States
facility: Children's Hospital and Research Center of Oakland
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County (CHOC)
state: California
city: Sacramento
country: United States
facility: Sutter Medical Center
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital San Diego
state: California
city: San Francisco
country: United States
facility: UCSF (adults)
state: California
city: San Francisco
country: United States
facility: UCSF (peds)
state: California
city: Stanford
country: United States
facility: Stanford University Medical Center
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Aurora
country: United States
facility: University of Colorado Hospital
state: Colorado
city: Denver
country: United States
facility: Presbyterian St. Luke's/Colorado Blood Cancer Institute
state: Colorado
city: New Haven
country: United States
facility: Yale New Haven Hospital
state: Connecticut
city: Brandywine
country: United States
facility: Nemours - Alfred I. duPont Hospital for Children
state: Delaware
city: Washington DC
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Gainesville
country: United States
facility: University of Florida Shands Medical Center
state: Florida
city: Jacksonville
country: United States
facility: Nemours Children's Hospital - Mayo Jacksonville
state: Florida
city: Jacksonville
country: United States
facility: Mayo Clinic - Jacksonville
state: Florida
city: Miami
country: United States
facility: University of Miami (adult)
state: Florida
city: Miami
country: United States
facility: University of Miami (peds)
state: Florida
city: Miami
country: United States
facility: Niklaus Children's Hospital
state: Florida
city: Orlando
country: United States
facility: AdventHealth Orlando
state: Florida
city: Orlando
country: United States
facility: Florida Center for Pediatric Cellular Therapy
state: Florida
city: Orlando
country: United States
facility: Orlando Health Bone Marrow Transplant and Cellular Therapy
state: Florida
city: St. Petersburg
country: United States
facility: All Children's Hospital
state: Florida
city: Tampa
country: United States
facility: H Lee Moffitt Cancer Center
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Atlanta
country: United States
facility: Emory University Hospital
state: Georgia
city: Atlanta
country: United States
facility: Northside Hospital
state: Georgia
city: Augusta
country: United States
facility: Medical College of Georgia
state: Georgia
city: Honolulu
country: United States
facility: Kapiolani Medical Center for Women and Children
state: Hawaii
city: Chicago
country: United States
facility: Northwestern Memorial Hospital
state: Illinois
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Chicago
country: United States
facility: University of Illinois at Chicago
state: Illinois
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Maywood
country: United States
facility: Loyola University Medical Center
state: Illinois
city: Park Ridge
country: United States
facility: Advocate Lutheran General Hospital
state: Illinois
city: Zion
country: United States
facility: Midwestern Regional Medical Center
state: Illinois
city: Beech Grove
country: United States
facility: Indiana Blood & Marrow Transplantation
state: Indiana
city: Indianapolis
country: United States
facility: Indiana University/Riley Hospital for Children
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Westwood
country: United States
facility: University of Kansas
state: Kansas
city: Louisville
country: United States
facility: University of Louisville Hospital - James Brown Cancer Center
state: Kentucky
city: New Orleans
country: United States
facility: Tulane Medical Center
state: Louisiana
city: New Orleans
country: United States
facility: Children's Hospital of New Orleans/LSUHSC
state: Louisiana
city: Baltimore
country: United States
facility: Greenebaum Cancer Center - University of Maryland
state: Maryland
city: Baltimore
country: United States
facility: Johns Hopkins
state: Maryland
city: Bethesda
country: United States
facility: Nih/Niaid
state: Maryland
city: Bethesda
country: United States
facility: NIH/NCI
state: Maryland
city: Bethesda
country: United States
facility: Nih/Nhlbi
state: Maryland
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Beth Israel Deaconess
state: Massachusetts
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Worcester
country: United States
facility: University of Massachusetts
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Detroit
country: United States
facility: Children's Hospital of Michigan
state: Michigan
city: Detroit
country: United States
facility: Karmanos Cancer Institute
state: Michigan
city: Detroit
country: United States
facility: Henry Ford Health System
state: Michigan
city: Grand Rapids
country: United States
facility: Helen DeVos Children's Hospital
state: Michigan
city: Grand Rapids
country: United States
facility: Spectrum Health
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic Rochester
state: Minnesota
city: Jackson
country: United States
facility: Univeristy of Mississippi Medical Center
state: Mississippi
city: Kansas City
country: United States
facility: Children's Mercy Hospital and Clinics
state: Missouri
city: St. Louis
country: United States
facility: Cardinal Glennon Children's Medical Center
state: Missouri
city: St. Louis
country: United States
facility: St. Louis University Medical Center
state: Missouri
city: St. Louis
country: United States
facility: Washington University St. Louis
state: Missouri
city: St. Louis
country: United States
facility: Washington University/St. Louis Children's Hospital
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: New Brunswick
country: United States
facility: Robert Wood Johnson/Cancer Institute of New Jersey
state: New Jersey
city: Bronx
country: United States
facility: Children's Hospital at Montefiore
state: New York
city: Buffalo
country: United States
facility: Roswell Park Cancer Institute
state: New York
city: Lake Sucess
country: United States
facility: North Shore University Hospital
state: New York
city: New Hyde
country: United States
facility: Cohen Children's Medical Center of New York
state: New York
city: New York
country: United States
facility: New York University Langone Medical Center
state: New York
city: New York
country: United States
facility: New York Presbyterian Hospital at Cornell
state: New York
city: New York
country: United States
facility: Mount Sinai Medical Center
state: New York
city: New York
country: United States
facility: Columbia University Medical Center - Morgan Stanley Children's Hospital
state: New York
city: New York
country: United States
facility: Memorial Sloan-Kettering Cancer Center
state: New York
city: New York
country: United States
facility: Westchester Medical Center
state: New York
city: Rochester
country: United States
facility: Strong Memorial Hospital/University of Rochester
state: New York
city: Stony Brook
country: United States
facility: Stony Brook University Hospital
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina at Chapel Hill
state: North Carolina
city: Charlotte
country: United States
facility: Levine Children's Hospital
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Winston-Salem
country: United States
facility: Wake Forest University
state: North Carolina
city: Akron
country: United States
facility: Childrens Hospital Medical Center of Akron
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Cincinnati
country: United States
facility: The Jewish Hospital
state: Ohio
city: Cleveland
country: United States
facility: University Hospitals Case Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: The Arthur G. James Cancer Hospital/The Ohio State University Medical Center
state: Ohio
city: Oklahoma City
country: United States
facility: University of Oklahoma Health Science Center
state: Oklahoma
city: Portland
country: United States
facility: Oregon Health & Science University/Doernbecher Children's Hospital
state: Oregon
city: Hershey
country: United States
facility: Penn State Milton S. Hershey Cancer Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Pittsburg
country: United States
facility: Western Penn Hospital
state: Pennsylvania
city: Pittsburg
country: United States
facility: University of Pennsylvania/Thomas Jefferson
state: Pennsylvania
city: Providence
country: United States
facility: Roger Williams Medical Center
state: Rhode Island
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Sioux Falls
country: United States
facility: Avera Transplant Institute
state: South Dakota
city: Memphis
country: United States
facility: Methodist Healthcare Blood and Marrow Transplant Center
state: Tennessee
city: Memphis
country: United States
facility: St. Jude Childrens Research Hospital
state: Tennessee
city: Nashville
country: United States
facility: Sarah Cannon BMT Program
state: Tennessee
city: Nashville
country: United States
facility: VA Tennessee Valley Healthcare System
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
city: Amarillo
country: United States
facility: Amarillo Blood and Marrow Transplant Program
state: Texas
city: Dallas
country: United States
facility: Medical City Dallas
state: Texas
city: Dallas
country: United States
facility: Children's Medical Center Dallas
state: Texas
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Fort Sam Houston
country: United States
facility: Zachary & Elizabeth Fisher Bone Marrow Transplant Program (Brooke Army Medical Center)
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Hospital
state: Texas
city: Houston
country: United States
facility: M.D. Anderson Cancer Center
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital - Baylor Medical Center
state: Texas
city: Lubbock
country: United States
facility: Southwest Cancer Treatment & Research Center
state: Texas
city: San Antonio
country: United States
facility: The Children's Hospital of San Antonio
state: Texas
city: San Antonio
country: United States
facility: Methodist Healthcare System of San Antonio/Texas Transplant Institute
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Seattle
country: United States
facility: VA Puget Sound HCS
state: Washington
city: Seattle
country: United States
facility: Fred Hutchinson Cancer Research Center
state: Washington
city: Morgantown
country: United States
facility: West Virginia University
state: West Virginia
city: Madison
country: United States
facility: University of Wisconsin - Madison
state: Wisconsin
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
Study officials:
affiliation: NMDP/CIBMTR
name: John Miller, MD, PhD
role: STUDY_CHAIR
References: