A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

NCT ID: NCT01351545

Last Updated: 2025-12-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

99999 participants

Study Classification

OBSERVATIONAL

Study Start Date

2011-10-31

Study Completion Date

2041-10-31

Brief Summary

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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

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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

Conditions

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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

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

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Unlicensed CBU

The cohort includes recipients of any age receiving unlicensed cryopreserved cord blood units (CBUs) for designated indications.

A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)

Intervention Type DRUG

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

Interventions

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A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)

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

Intervention Type DRUG

Eligibility Criteria

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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
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Marrow Donor Program

OTHER

Sponsor Role collaborator

Center for International Blood and Marrow Transplant Research

NETWORK

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Heather Stefanski, MD, PhD

Role: STUDY_CHAIR

NMDP/CIBMTR

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status RECRUITING

Banner MD Anderson Cancer Center

Gilbert, Arizona, United States

Site Status RECRUITING

Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status RECRUITING

Mayo Clinic Arizona (adults)

Phoenix, Arizona, United States

Site Status RECRUITING

University of Arizona Medical Center - Tucson

Tucson, Arizona, United States

Site Status RECRUITING

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Site Status RECRUITING

City of Hope

Duarte, California, United States

Site Status RECRUITING

Children's Hospital of Los Angeles

Glendale, California, United States

Site Status RECRUITING

Scripps Green Hospital

La Jolla, California, United States

Site Status RECRUITING

University of California San Diego

La Jolla, California, United States

Site Status RECRUITING

Loma Linda University Cancer Center

Loma Linda, California, United States

Site Status RECRUITING

UCLA

Los Angeles, California, United States

Site Status RECRUITING

Children's Hospital and Research Center of Oakland

Oakland, California, United States

Site Status RECRUITING

Children's Hospital of Orange County (CHOC)

Orange, California, United States

Site Status RECRUITING

Sutter Medical Center

Sacramento, California, United States

Site Status RECRUITING

Rady Children's Hospital San Diego

San Diego, California, United States

Site Status RECRUITING

UCSF (adults)

San Francisco, California, United States

Site Status SUSPENDED

UCSF (Peds)

San Francisco, California, United States

Site Status RECRUITING

Stanford University Medical Center

Stanford, California, United States

Site Status RECRUITING

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status RECRUITING

University of Colorado Hospital

Aurora, Colorado, United States

Site Status RECRUITING

Presbyterian St. Luke's/Colorado Blood Cancer Institute

Denver, Colorado, United States

Site Status RECRUITING

Yale New Haven Hospital

New Haven, Connecticut, United States

Site Status RECRUITING

Nemours - Alfred I. duPont Hospital for Children

Brandywine, Delaware, United States

Site Status RECRUITING

Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status RECRUITING

University of Florida Shands Medical Center

Gainesville, Florida, United States

Site Status RECRUITING

Nemours Children's Hospital - Mayo Jacksonville

Jacksonville, Florida, United States

Site Status RECRUITING

Mayo Clinic - Jacksonville

Jacksonville, Florida, United States

Site Status RECRUITING

University of Miami (adult)

Miami, Florida, United States

Site Status COMPLETED

University of Miami (peds)

Miami, Florida, United States

Site Status RECRUITING

Niklaus Children's Hospital

Miami, Florida, United States

Site Status RECRUITING

AdventHealth Orlando

Orlando, Florida, United States

Site Status RECRUITING

Florida Center for Pediatric Cellular Therapy

Orlando, Florida, United States

Site Status RECRUITING

Orlando Health Arnold Palmer Hospital for Children

Orlando, Florida, United States

Site Status RECRUITING

Orlando Health Cancer Institute Bone Marrow Transplant and Cellular Therapy

Orlando, Florida, United States

Site Status RECRUITING

All Children's Hospital

St. Petersburg, Florida, United States

Site Status RECRUITING

H Lee Moffitt Cancer Center

Tampa, Florida, United States

Site Status TERMINATED

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status RECRUITING

Emory University Hospital

Atlanta, Georgia, United States

Site Status TERMINATED

Northside Hospital

Atlanta, Georgia, United States

Site Status RECRUITING

Georgia Cancer Center at Augusta University Health

Augusta, Georgia, United States

Site Status RECRUITING

Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, United States

Site Status RECRUITING

Northwestern Memorial Hospital

Chicago, Illinois, United States

Site Status RECRUITING

Rush University Medical Center

Chicago, Illinois, United States

Site Status RECRUITING

University of Illinois at Chicago

Chicago, Illinois, United States

Site Status RECRUITING

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status RECRUITING

University of Chicago

Chicago, Illinois, United States

Site Status RECRUITING

Loyola University Medical Center

Maywood, Illinois, United States

Site Status RECRUITING

Advocate Lutheran General Hospital

Park Ridge, Illinois, United States

Site Status TERMINATED

Midwestern Regional Medical Center

Zion, Illinois, United States

Site Status TERMINATED

Indiana Blood & Marrow Transplantation

Beech Grove, Indiana, United States

Site Status RECRUITING

Indiana University/Riley Hospital for Children

Indianapolis, Indiana, United States

Site Status RECRUITING

University of Iowa

Iowa City, Iowa, United States

Site Status RECRUITING

University of Kansas

Westwood, Kansas, United States

Site Status RECRUITING

University of Louisville Hospital - James Brown Cancer Center

Louisville, Kentucky, United States

Site Status RECRUITING

Tulane Medical Center

New Orleans, Louisiana, United States

Site Status RECRUITING

Children's Hospital of New Orleans/LSUHSC

New Orleans, Louisiana, United States

Site Status RECRUITING

Greenebaum Cancer Center - University of Maryland

Baltimore, Maryland, United States

Site Status WITHDRAWN

Johns Hopkins

Baltimore, Maryland, United States

Site Status RECRUITING

Nih/Niaid

Bethesda, Maryland, United States

Site Status RECRUITING

NIH/NCI

Bethesda, Maryland, United States

Site Status TERMINATED

Nih/Nhlbi

Bethesda, Maryland, United States

Site Status RECRUITING

Tufts Medical Center

Boston, Massachusetts, United States

Site Status RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status SUSPENDED

Beth Israel Deaconess

Boston, Massachusetts, United States

Site Status RECRUITING

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Site Status RECRUITING

University of Massachusetts

Worcester, Massachusetts, United States

Site Status RECRUITING

University of Michigan

Ann Arbor, Michigan, United States

Site Status RECRUITING

Children's Hospital of Michigan

Detroit, Michigan, United States

Site Status RECRUITING

Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status SUSPENDED

Henry Ford Health System

Detroit, Michigan, United States

Site Status RECRUITING

Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States

Site Status RECRUITING

Spectrum Health

Grand Rapids, Michigan, United States

Site Status RECRUITING

Mayo Clinic Rochester

Rochester, Minnesota, United States

Site Status TERMINATED

Univeristy of Mississippi Medical Center

Jackson, Mississippi, United States

Site Status RECRUITING

Children's Mercy Hospital and Clinics

Kansas City, Missouri, United States

Site Status SUSPENDED

Cardinal Glennon Children's Medical Center

St Louis, Missouri, United States

Site Status RECRUITING

St. Louis University Medical Center

St Louis, Missouri, United States

Site Status RECRUITING

Washington University St. Louis

St Louis, Missouri, United States

Site Status RECRUITING

Washington University/St. Louis Children's Hospital

St Louis, Missouri, United States

Site Status RECRUITING

University of Nebraska Medical Center

Omaha, Nebraska, United States

Site Status RECRUITING

Hackensack University Medical Center

Hackensack, New Jersey, United States

Site Status RECRUITING

Robert Wood Johnson/Cancer Institute of New Jersey

New Brunswick, New Jersey, United States

Site Status RECRUITING

Roswell Park Cancer Institute

Buffalo, New York, United States

Site Status RECRUITING

North Shore University Hospital

Lake Sucess, New York, United States

Site Status RECRUITING

Cohen Children's Medical Center of New York

New Hyde, New York, United States

Site Status RECRUITING

New York University Langone Medical Center

New York, New York, United States

Site Status RECRUITING

New York Presbyterian Hospital at Cornell

New York, New York, United States

Site Status RECRUITING

Mount Sinai Medical Center - New York

New York, New York, United States

Site Status RECRUITING

Columbia University Medical Center - Morgan Stanley Children's Hospital

New York, New York, United States

Site Status RECRUITING

Memorial Sloan-Kettering Cancer Center

New York, New York, United States

Site Status RECRUITING

Westchester Medical Center

New York, New York, United States

Site Status RECRUITING

Strong Memorial Hospital/University of Rochester

Rochester, New York, United States

Site Status RECRUITING

Stony Brook University Hospital

Stony Brook, New York, United States

Site Status RECRUITING

Children's Hospital at Montefiore

The Bronx, New York, United States

Site Status RECRUITING

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Site Status RECRUITING

Levine Children's Hospital

Charlotte, North Carolina, United States

Site Status RECRUITING

Duke University Medical Center

Durham, North Carolina, United States

Site Status RECRUITING

Wake Forest University

Winston-Salem, North Carolina, United States

Site Status WITHDRAWN

Childrens Hospital Medical Center of Akron

Akron, Ohio, United States

Site Status RECRUITING

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Site Status RECRUITING

The Jewish Hospital

Cincinnati, Ohio, United States

Site Status WITHDRAWN

University Hospitals Case Medical Center

Cleveland, Ohio, United States

Site Status RECRUITING

Cleveland Clinic

Cleveland, Ohio, United States

Site Status RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status RECRUITING

The Arthur G. James Cancer Hospital/The Ohio State University Medical Center

Columbus, Ohio, United States

Site Status RECRUITING

University of Oklahoma Health Science Center

Oklahoma City, Oklahoma, United States

Site Status RECRUITING

Oregon Health & Science University/Doernbecher Children's Hospital

Portland, Oregon, United States

Site Status RECRUITING

Penn State Milton S. Hershey Cancer Center

Hershey, Pennsylvania, United States

Site Status RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

Western Penn Hospital

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

University of Pennsylvania/Thomas Jefferson

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

Roger Williams Medical Center

Providence, Rhode Island, United States

Site Status RECRUITING

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status RECRUITING

Avera Transplant Institute

Sioux Falls, South Dakota, United States

Site Status RECRUITING

Methodist Healthcare Blood and Marrow Transplant Center

Memphis, Tennessee, United States

Site Status COMPLETED

St. Jude Childrens Research Hospital

Memphis, Tennessee, United States

Site Status RECRUITING

Sarah Cannon BMT Program

Nashville, Tennessee, United States

Site Status SUSPENDED

VA Tennessee Valley Healthcare System

Nashville, Tennessee, United States

Site Status WITHDRAWN

Vanderbilt University

Nashville, Tennessee, United States

Site Status RECRUITING

Amarillo Blood and Marrow Transplant Program

Amarillo, Texas, United States

Site Status TERMINATED

Medical City Dallas

Dallas, Texas, United States

Site Status RECRUITING

Children's Medical Center Dallas

Dallas, Texas, United States

Site Status RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status WITHDRAWN

Zachary & Elizabeth Fisher Bone Marrow Transplant Program (Brooke Army Medical Center)

Fort Sam Houston, Texas, United States

Site Status RECRUITING

Cook Children's Hospital

Fort Worth, Texas, United States

Site Status RECRUITING

M.D. Anderson Cancer Center

Houston, Texas, United States

Site Status RECRUITING

Texas Children's Hospital - Baylor Medical Center

Houston, Texas, United States

Site Status RECRUITING

Southwest Cancer Treatment & Research Center

Lubbock, Texas, United States

Site Status WITHDRAWN

The Children's Hospital of San Antonio

San Antonio, Texas, United States

Site Status RECRUITING

Methodist Healthcare System of San Antonio/Texas Transplant Institute

San Antonio, Texas, United States

Site Status RECRUITING

University of Utah

Salt Lake City, Utah, United States

Site Status RECRUITING

University of Virginia

Charlottesville, Virginia, United States

Site Status RECRUITING

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status RECRUITING

VA Puget Sound HCS

Seattle, Washington, United States

Site Status SUSPENDED

Fred Hutchinson Cancer Research Center

Seattle, Washington, United States

Site Status RECRUITING

West Virginia University

Morgantown, West Virginia, United States

Site Status RECRUITING

University of Wisconsin - Madison

Madison, Wisconsin, United States

Site Status RECRUITING

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Site Status RECRUITING

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Elizabeth Fosberg, B.A.

Role: CONTACT

Email: [email protected]

Facility Contacts

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Joseph Chewning, MD

Role: primary

Matthew Ulrickson, MD

Role: primary

Roberta Adams, MD

Role: primary

James Slack, MD

Role: primary

Emmanuel Katsanis, MD

Role: primary

Jason Farrar, MD

Role: primary

Monzr Al Malki, MD

Role: primary

Neena Kapoor, MD

Role: primary

James R Mason, MD

Role: primary

Dimitrios Tzachanis, MD

Role: primary

Hisham Abdel-Azim, MD

Role: primary

Gary Schiller, MD

Role: primary

Mark Walters, MD

Role: primary

Rishikesh Chavan, MD

Role: primary

Elias Kiwan, MD

Role: primary

Eric Anderson, MD

Role: primary

Sandhya Kharbanda, MD

Role: primary

Andrew Rezvani, MD

Role: primary

Michael Verneris, MD

Role: primary

Jonathan Gutman, MD

Role: primary

Alireza Eghtedar, MD

Role: primary

Aaron Flagg, MD

Role: primary

Emi Caywood, MD

Role: primary

David Jacobsohn, MD

Role: primary

Jordan Milner, MD

Role: primary

Edward Ziga, MD

Role: primary

Vivek Roy, MD

Role: primary

Edward Ziga, MD, MPH

Role: primary

Jorge Galvez-Silva, MD

Role: primary

Rushang Patel, MD

Role: primary

Susan Kelly, MD

Role: primary

Rikin Shah

Role: primary

Yasser Khaled, MD

Role: primary

Deepakbabu Chellapandian, MD

Role: primary

Ann Haight, MD

Role: primary

Scott Solomon, MD

Role: primary

Vamsi K Kota, MD

Role: primary

Randal Wada, MD

Role: primary

Kehinde Adekola, MD

Role: primary

Sunita Nathan, MD

Role: primary

Damiano Rondelli, MD

Role: primary

Sonali Chaudhury, MD

Role: primary

James LaBelle, MD, PhD

Role: primary

Patrick Hagen, MD

Role: primary

Luke Akard, MD

Role: primary

Jodi Skiles, MD

Role: primary

Margarida Silverman, MD

Role: primary

Joseph McGuirk, DO

Role: primary

Mohamed Hegazi, MD

Role: primary

Hana Safa, MD

Role: primary

Lolie Yu, MD

Role: primary

Allen Chen, MD, PhD

Role: primary

Elizabeth Kang, MD

Role: primary

Richard Childs, MD

Role: primary

Andreas Klein, MD

Role: primary

David Avigan, MD

Role: primary

Corey Cutler, MD

Role: primary

Jan Cerny, MD

Role: primary

Jessica Yu

Role: primary

Sureyya Savasan, MD

Role: primary

Nalini Janakiraman, MD

Role: primary

Troy Quigg, MD

Role: primary

Aly Abdel-Mageed, MD

Role: primary

Carolyn Bigelow, MD

Role: primary

Deepika Bhatla, MD

Role: primary

Sagun Goyal, MD

Role: primary

Geoffrey Uy, MD

Role: primary

Geoffrey Uy, MD

Role: primary

Phyllis Warkenton, MD

Role: primary

Alfred Gillio, MD

Role: primary

Matthew Matasar, MD, MS

Role: primary

Kanwaldeep Mallhi, MD

Role: primary

Ruthee L Bayer, MD

Role: primary

Jonathan Fish, MD

Role: primary

Mohammad M Abdul Hay, MD

Role: primary

Antoine Mesidor

Role: backup

Tsiporah Shore, MD

Role: primary

Alla Keyzner, MD

Role: primary

Monica Bhatia, MD

Role: primary

Ioannis Politikos, MBBS

Role: primary

Mitchell Cairo, MD

Role: primary

Omar Aljitawi, MD

Role: primary

Michael Schuster, MD

Role: primary

Mahvish Rahim, MD

Role: primary

Anson Snow, MD

Role: primary

Jeffrey Huo, MD

Role: primary

Michelle Hyland

Role: backup

Joanne Kurtzberg, MD

Role: primary

Megan Sampson, MD

Role: primary

Stella Davies, MD

Role: primary

Hillard Lazarus, MD

Role: primary

Claudio Brunstein, MD

Role: primary

Rolla Abu-Arja, MD

Role: primary

Sumithira Vasu, MD

Role: primary

Adam Asch, MD

Role: primary

Eneida Nemecek, MD

Role: primary

Shin Mineishi, MD

Role: primary

Timothy Olson, MD

Role: primary

Paul Szabolcs, MD

Role: primary

John Lister, MD

Role: primary

Alison Sehgal, MD

Role: primary

Elizabeth Hexner, MD

Role: primary

Todd Roberts, MD

Role: primary

Michelle Hudspeth, MD

Role: primary

Waqas Jehangir, MD, FACP

Role: primary

Renee Madden, MD

Role: primary

Michael Byrne, MD

Role: primary

Vikas Bhushan, MD

Role: primary

Victor Aquino, MD

Role: primary

Bradley Beeler, MD

Role: primary

Gretchen Eames, MD

Role: primary

Elizabeth Shpall, MD

Role: primary

George Carrum, MD

Role: primary

Martin Andreansky, MD, PhD

Role: primary

Mark Schambura

Role: backup

Jose Cruz, MD

Role: primary

Ahmad Rayes, MD

Role: primary

Karen Ballen, MD

Role: primary

John McCarty, MD

Role: primary

Ann Dahlberg, MD

Role: primary

Lauren Veltri, MD

Role: primary

Kenneth DeSantes, MD

Role: primary

David Margolis, MD

Role: primary

Hamadani Mehdi, MD

Role: primary

Other Identifiers

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NCT01553461

Identifier Type: -

Identifier Source: nct_alias

10-CBA

Identifier Type: -

Identifier Source: org_study_id