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Providing Access to Cord Blood Units for Transplants

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01479582
Recruitment Status : Withdrawn
First Posted : November 24, 2011
Last Update Posted : December 21, 2018
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )

Brief Summary:


- Cord blood banks have been set up to collect and store umbilical cord blood for transplants. These transplants are used to treat different types of cancer. In October 2011, the Food and Drug Administration (FDA) began considering cord blood as a biological drug. Most of the cord blood units currently available in cord blood banks in the United States and other countries were collected before the FDA set these new standards. The units meet standards set by the National Marrow Donor Program (NMDP), but they were not collected, tested, or stored exactly according to FDA standards. As a result, the new guidelines state that they may only be used for transplant if the transplant is done as part of a study. Researchers have set up a study to provide these cord blood units to recipients and to study the effects of their use.


  • To provide access to cord blood units for recipients whose best choice for a unit meets NMDP but not FDA standards.
  • To study the effects of these cord blood transplants.


- Individuals who need to have a cord blood transplant to treat certain types of cancer.


  • Participants will be screened with a physical exam, medical history. They will also have blood tests and imaging studies.
  • Participants will have the cord blood transplant and allow their medical data to be collected by the study researchers.

Condition or disease Intervention/treatment Phase
Chronic Myelogous Leukemia/Other Leukemia Acute Leukemias MDS/MPS Multiple Myeloma Non-Hodgkin Lymphoma Drug: Access to unlicensed cord blood units Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : October 27, 2011
Actual Primary Completion Date : November 26, 2012
Actual Study Completion Date : November 26, 2012

Primary Outcome Measures :
  1. The primary aim of this study is to examine the incidence of neutrophil recovery of greater than or equal to 500/mm3 after cord blood transplantation in a multi-institution setting using CBUs that are not Food and Drug Administration (FDA) licen...

Secondary Outcome Measures :
  1. Assess incidence of graft rejection.
  2. Assess incidence of transmission of infection.
  3. Assess incidence of serious infusion reaction.
  4. Determine 1 year survival after cord blood transplantation.
  5. Assess cumulative incidence of aGVHD vs cGVHD.

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 99 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Patients with FDA-specified indications:

  • 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
  • Signed informed consent (and signed assent, if applicable)
  • Pediatric and adult patients of any age


  • Patients who are receiving only licensed CBUs
  • Cord blood transplant recipients at international transplant centers

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01479582

Sponsors and Collaborators
National Cancer Institute (NCI)
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Principal Investigator: Ronald E Gress, M.D. National Cancer Institute (NCI)
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Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT01479582    
Other Study ID Numbers: 120027
First Posted: November 24, 2011    Key Record Dates
Last Update Posted: December 21, 2018
Last Verified: November 26, 2012
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) ):
Adult and Pediatric
Unlicensed Cord Blood Unit IND
Hematologic Malignancies
Multiple Myeloma
Non-Hodgkin Lymphoma
Chronic Myelogenous Leukemia
Myelodyplastic Syndrome
Lysosomal Storage Diseases
Additional relevant MeSH terms:
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Multiple Myeloma
Lymphoma, Non-Hodgkin
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Neoplasms, Plasma Cell
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders