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Transplants With Unlicensed Preserved Cord Blood

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ClinicalTrials.gov Identifier: NCT01553461
Recruitment Status : Recruiting
First Posted : March 14, 2012
Last Update Posted : January 15, 2018
Information provided by (Responsible Party):

Study Description
Brief Summary:


- Cord blood transplants can treat cancers and other diseases in children and adults. The U.S. Food and Drug Administration (FDA) requires cord blood to be collected and stored under certain safety standards. However, most available cord blood units were collected before the FDA set these standards. These units may not meet FDA standards, but they do meet similar standards set and followed by the National Marrow Donor Program (NMDP). Cord blood units that do not meet the new FDA standards may be used for transplants only as part of a research study. Doctors want to allow people who need transplants to receive cord blood that meets NMDP standards but may not meet FDA standards.


- To allow selected cord blood units that do not meet current FDA standards to be used for transplant.


- Individuals who need cord blood units for transplant, and who best match cord blood units that are not FDA-licensed.


  • Participants will provide consent to receive cord blood that meets NMDP standards but may not meet FDA standards.
  • Participants will remain on the study for observation for up to 1 year after transplant, or until they withdraw from the study for personal or medical reasons.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndrome (MDS) Hematologic Neoplasms Hematologic Diseases Severe Aplastic Anemia Procedure: Cord Blood Transplant Phase 1 Phase 2

  Show Detailed Description

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 500 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 : February 2, 2012
Estimated Primary Completion Date : October 20, 2036
Estimated Study Completion Date : October 20, 2036

Arms and Interventions

Outcome Measures

Primary Outcome Measures :
  1. To examine the incidence of neutrophil recovery of greater than or equal to 500/mm (3) after unlicensed CBU transplant.

Secondary Outcome Measures :
  1. Assess incidence of graft rejection, transmission of infection, serious infusion reactions.
  2. Determine 1 year survival.
  3. Assess cumulative incidence of acute GVHD (grades II to IV), (grades III to IV), and incidence of chronic GVHD.

Eligibility Criteria

Information from the National Library of Medicine

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


  • 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
Contacts and Locations

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): NCT01553461

Contact: Richard W Childs, M.D. (301) 451-7128 childsr@nhlbi.nih.gov

United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL)    800-411-1222 ext TTY8664111010    prpl@mail.cc.nih.gov   
Sponsors and Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
Principal Investigator: Richard W Childs, M.D. National Heart, Lung, and Blood Institute (NHLBI)
More Information

Additional Information:
Responsible Party: National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier: NCT01553461     History of Changes
Other Study ID Numbers: 120064
First Posted: March 14, 2012    Key Record Dates
Last Update Posted: January 15, 2018
Last Verified: January 11, 2018

Keywords provided by National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) ):
Umbilical Cord Blood
Graft Rejection
Graft Versus Host Disease

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Anemia, Aplastic
Hematologic Diseases
Hematologic Neoplasms
Bone Marrow Diseases
Precancerous Conditions
Neoplasms by Site