Protocol for Transplantation of Cryopreserved CBUs from the National Cord Blood Program

Study of Unlicensed, Investigational Cryopreserved Cord Blood Units Manufactured by the National Cord Blood Program and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients

Protocol Description

This multi-center study enables access to, and documents the use of, unlicensed cryopreserved (frozen) cord blood units (CBUs) in people who need stem cell transplants. Since 2011, the Food and Drug Administration (FDA) has considered CBUs a biological drug that must meet certain FDA standards to be used. Most CBUs available in the United States were collected before the FDA established these standards; however, the FDA allows use of these “unlicensed” CBUs, if the transplant is being done as part of a study. CBUs in this study have been collected, processed, tested, and stored by the National Cord Blood Program and meet the criteria for transplantation, as approved by NetCord-FACT (Foundation for the Accreditation of Cellular Therapy).

Eligibility Criteria

Subject to certain exclusion criteria, this study is enrolling participants of any age who need a CBU transplant to treat a disorder affecting the hematopoietic system.
Males and Females: All ages


CBUs will be infused through a vein in accordance with standard protocols for the patient’s condition and treatment. Medical information about each participant’s disease, transplant, and outcome will be sent to the Center for International Blood and Marrow Transplant Research (CIBMT) by the participant’s doctor. Participation in the study will end a year after the patient’s cord blood transplant.
Visits: None beyond normal clinical care for the condition
Duration: 1 year post transplant

Status: Open to Enrollment

Primary Investigator

Paul Szabolcs, MD

Contact Information

For more information about the study or enrollment, please contact:
Shawna McIntyre, Clinical Research Supervisor