A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

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 objective 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 overall 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