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
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Open (Enrolling)
Both
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
Both
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
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
indications.
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:
Observational
Study Design:
Time Perspective: Prospective
Authority:
United States: Food and Drug Administration
Study ID:
10-CBA
NCT ID:
NCT01351545
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
Name | Location |
|---|---|
| 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 |
