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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

Open (Enrolling)
Hematological Malignancies, Certain Lysosomal Storage and Peroxisomal Enzyme Deficiency Disorders, Hurler Syndrome (MPS I), Krabbe Disease (Globoid Leukodystrophy), X-linked Adrenoleukodystrophy, Primary Immunodeficiency Diseases, Bone Marrow Failure, Beta-thalassemia

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Trial Information

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

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

Primary Objective:

The primary aim 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 graft rejection

- Assess incidence of transmission of infection

- Assess incidence of serious infusion reaction

- Determine 1 year 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

- Determine CBU-derived engraftment

Inclusion Criteria:

- Patients with FDA-specified indications: Hematological malignancies, Certain
lysosomal storage and peroxisomal enzyme deficiency disorders (Hurler syndrome (MPS
I), Krabbe Disease (Globoid Leukodystrophy),and X-linked Adrenoleukodystrophy),
Primary immunodeficiency diseases, Bone marrow failure, and Beta-thalassemia

- Signed informed consent (and signed assent, if applicable)

- Pediatric and adult patients of any age

Exclusion Criteria:

- Patients who are receiving only licensed CBUs

- Cord blood transplant recipients at international transplant centers

Type of Study:


Study Design:

Time Perspective: Prospective


United States: Food and Drug Administration

Study ID:




Start Date:

October 2011

Completion Date:

Related Keywords:

  • Hematological Malignancies
  • Certain Lysosomal Storage and Peroxisomal Enzyme Deficiency Disorders
  • Hurler Syndrome (MPS I)
  • Krabbe Disease (Globoid Leukodystrophy)
  • X-linked Adrenoleukodystrophy
  • Primary Immunodeficiency Diseases
  • Bone Marrow Failure
  • Beta-Thalassemia
  • Beta-Thalassemia
  • Neoplasms
  • Leukodystrophy, Globoid Cell
  • Immunologic Deficiency Syndromes
  • Pancytopenia
  • Thalassemia
  • Mucopolysaccharidosis I
  • Adrenoleukodystrophy
  • Hematologic Neoplasms



Wake Forest University Winston-Salem, North Carolina  27103
University of Iowa Iowa City, Iowa  52242
University of Michigan Ann Arbor, Michigan  48109-0624
Roswell Park Cancer Institute Buffalo, New York  14263
Memorial Sloan-Kettering Cancer Center New York, New York  10021
Children's Hospital of Philadelphia Philadelphia, Pennsylvania  19104
Stanford University Medical Center Stanford, California  94305-5408
Medical University of South Carolina Charleston, South Carolina  29425-0721
Children's Hospital of Michigan Detroit, Michigan  48201
Loyola University Medical Center Maywood, Illinois  60153
Medical College of Wisconsin Milwaukee, Wisconsin  53226
Northwestern Memorial Hospital Chicago, Illinois  60611
New York Medical College Valhalla, New York  10595
Rush University Medical Center Chicago, Illinois  60612-3824
Cedars-Sinai Medical Center Los Angeles, California  90048
Midwestern Regional Medical Center Zion, Illinois  60099
North Shore University Hospital Manhasset, New York  11030
University of Nebraska Medical Center Omaha, Nebraska  68198-3330
Hackensack University Medical Center Hackensack, New Jersey  07601
Children's National Medical Center Washington, District of Columbia  20010-2970
Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania  15213
Miami Children's Hospital Miami, Florida  33155-4069
Florida Hospital Cancer Institute Orlando, Florida  32804
All Children's Hospital St. Petersburg, Florida  33701
Phoenix Children's Hospital Phoenix, Arizona  85016-7710
University of Illinois at Chicago Chicago, Illinois  60612
Roger Williams Medical Center Providence, Rhode Island  02908-4735
Mount Sinai Medical Center New York, New York  10029
Nationwide Children's Hospital Columbus, Ohio  43205-2696
Baylor University Medical Center Dallas, Texas  75246
University of California San Diego La Jolla, California  92093
Massachusetts General Hospital Boston, Massachusetts  02114-2617
University of Pittsburgh Medical Center Pittsburgh, Pennsylvania  15213
Seattle Cancer Care Alliance Seattle, Washington  98109
City of Hope Duarte, California  91010
University of Virginia Charlottesville, Virginia  22908
University of Alabama at Birmingham Birmingham, Alabama  35294-3300
University of Utah Salt Lake City, Utah  
Vanderbilt University Nashville, Tennessee  37232-6305
Kapiolani Medical Center for Women and Children Honolulu, Hawaii  96826
Children's Hospital of Wisconsin Milwaukee, Wisconsin  53201
Henry Ford Health System Detroit, Michigan  48202
Cleveland Clinic Cleveland, Ohio  44195
Dana Farber Cancer Institute Boston, Massachusetts  02115
University of Colorado Hospital Denver, Colorado  80262
M.D. Anderson Cancer Center Houston, Texas  77030
H Lee Moffitt Cancer Center Tampa, Florida  33612
University of Chicago Chicago, Illinois  60637
University of Texas Southwestern Medical Center Dallas, Texas  
UCLA Los Angeles, California  90095
University of Kentucky Lexington, Kentucky  40536-0098
Stony Brook University Hospital Stony Brook, New York  11794-8174
Texas Transplant Institute San Antonio, Texas  78229
Children's Memorial Hospital Chicago, Illinois  60614
UC Davis Cancer Center Sacramento, California  95817
Tufts Medical Center Boston, Massachusetts  02111
Virginia Commonwealth University Richmond, Virginia  
St. Louis University St. Louis, Missouri  63110
Karmanos Cancer Institute Detroit, Michigan  48201
Scripps Green Hospital La Jolla, California  92037
Children's Healthcare of Atlanta Atlanta, Georgia  30342
University of Massachusetts Worcester, Massachusetts  01655
Northside Hospital Atlanta, Georgia  30342
West Virginia University Morgantown, West Virginia  26506
Johns Hopkins Baltimore, Maryland  21231
Mayo Clinic Rochester Rochester, Minnesota  55905
University of North Carolina at Chapel Hill Chapel Hill, North Carolina  27599
Emory University Hospital Atlanta, Georgia  30322
University of Oklahoma Health Science Center Oklahoma City, Oklahoma  73104
Cincinnati Children's Hospital Cincinnati, Ohio  45229
Duke University Medical Center (Peds) Durham, North Carolina  27705
University Hospitals Case Medical Center Cleveland, Ohio  44106
Children's Hospital of Los Angeles Los Angeles, California  90027
Rady Children's Hospital San Diego San Diego, California  92123
Yale New Haven Hospital New Haven, Connecticut  06520
The Jewish Hospital Cincinnati, Ohio  45236
Children's Mercy Hospital and Clinics Kansas City, Missouri  64108
Beth Israel Deaconess Boston, Massachusetts  02215
Sutter Medical Center Sacramento, California  95816
Helen DeVos Children's Hospital Grand Rapids, Michigan  49503
Cardinal Glennon Children's Medical Center St. Louis, Missouri  63104
Cook Children's Hospital Fort Worth, Texas  76104
Children's Medical Center Dallas Dallas, Texas  75235
University Of Kansas Kansas City, Kansas  66160
Greenebaum Cancer Center - University of Maryland Baltimore, Maryland  21201
University of Wisconsin - Madison Madison, Wisconsin  53792
Florida Center for Cellular Therapy Orlando, Florida  32804
VA Puget Sound HCS Seattle, Washington  98108
Children's Hospital of Orange County (CHOC) Orange, California  92868
Children's Hospital Colorado Aurora, Colorado  80045
Washington University St. Louis St. Louis, Missouri  63110
St. Jude Childrens Research Hospital Memphis, Tennessee  38105
Mayo Clinic Arizona (adults) Phoenix, Arizona  85054
University of Arizona Medical Center - Tucson Tucson, Arizona  85224
Loma Linda Universoty Cancer Center Loma Linda, California  92350
Children's Hospital and Research Center of Oakland Oakland, California  94609
UCSF (adults) San Francisco, California  94143
UCSF (peds) San Francisco, California  94143
Presbyterian St. Luke's/Colorado Blood Cancer Institute Denver, Colorado  80218
Nemours - Alfred I. duPont Hospital for Children Brandywine, Delaware  19803
University of Florida Shands Medical Center Gainesville, Florida  32610
Nemours Children's Hospital - Mayo Jacksonville Jacksonville, Florida  32207
Mayo Clinic - Jacksonville (adults) Jacksonville, Florida  32224
University of Miami (adult) Miami, Florida  33136
University of Miami (peds) Miami, Florida  
Indiana Blood & Marrow Transplantation Beech Grove, Illinois  46107
Indiana University Bone Marrow and SCT Program Indianapolis, Indiana  46202
University of Louisville Hospital - James Brown Cancer Center Louisville, Kentucky  40202
Tulane Medical Center New Orleans, Louisiana  70112
Children's Hospital of New Orleans/LSUHSC New Orleans, Louisiana  70118
Nih/Niaid Bethesda, Maryland  20892-1456
NIH/NCI Bethesda, Maryland  20892
Nih/Nhlbi Bethesda, Maryland  20892
Univeristy of Mississippi Medical Center Jackson, Michigan  39216
St. Louis Children's St. Louis, Missouri  63110
Robert Wood Johnson/Cancer Institute of New Jersey New Brunswick, New Jersey  08901
Cohen Children's Medical Center of New York New Hyde, New York  11040
New York Presbyterian Hospital at Cornell New York, New York  10021
Columbia University Medical Center - Morgan Stanley Children's Hospital New York, New York  10032
Strong Memorial Hospital/University of Rochester Rochester, New York  14642-8704
Duke Universoty Medical Center (adults) Durham, North Carolina  27705
Childrens Hospital Medical Center of Akron Akron, Ohio  44308
The Arthur G. James Cancer Hospital/The Ohio State University Medical Center Columbus, Ohio  43210
Oregon Health & Science University/Doernbecher Children's Hospital Portland, Oregon  97239
Penn State Milton S. Hershey Cancer Center Hershey, Pennsylvania  17033
University of Pennsylvania/Thomas Jefferson Pittsburg, Pennsylvania  19104
West Penn Hospital Pittsburg, Pennsylvania  15224
Avera Transplant Institute Sioux Falls, South Dakota  57105
Vanderbilt Veterans Hospital Nashville, Tennessee  37212
Amarillo Blood and Marrow Transplant Program Amarillo, Texas  79106
Medical City Dallas Dallas, Texas  75230
Zachary and Elizabeth M Fisher Bome Marrow Transplant Unit Fork Sam Houston, Texas  78234-6200
Texas Children's Hospital - Baylor Medical Center Houston, Texas  77030
Southwest Cancer Treatment & Research Center Lubbock, Texas  79415
Intermountain Blood and Marrow Transplant Program Salt Lake City, Utah  84143
Scottsdale Healthcare/Cancer Transplant Institute at Virginia G. Piper Cancer Center Scottsdale, Arizona  85260
Levine Children's Hospital/Carolinas Medical Center Charlotte, North Carolina