Protocol for Transplantation of Cryopreserved CBUs Compliant with National Marrow Donor Program Standards

Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units for Transplantation in Pediatric and Adult Patients with Hematologic Malignancies and Other Indications

Protocol Description

This multi-center study is an access and distribution protocol that lets doctors provide cryopreserved (frozen) cord blood units (CBUs) for patients who need them for treatment of disease, providing that the CBUs meet standards of the National Marrow Donor Program. 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.

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