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


Phoenix/Scottsdale, Ariz., Jacksonville, Fla., Rochester, Minn.

Trial status:

Open for Enrollment

Why is this study being done?

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 aim 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 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

Who is eligible to participate?

Inclusion Criteria: - Disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment - Signed informed consent (and signed assent, if applicable) - Pediatric and adult patients of any age Exclusion Criteria: - Patients who are receiving only licensed CBUs - Cord blood transplant recipients at international transplant centers

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