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

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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