Trial Information

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

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 graft rejection

- 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

- Determine CBU-derived engraftment

Eligibility Criteria:

Inclusion Criteria

- Patients with FDA-specified indications (see Appendix B for further details):

- Hematological malignancies

- Certain lysosomal storage and peroxisomal enzyme deficiency disorders

- Hurler syndrome (MPS I)

- Krabbe Disease (Globoid Leukodystrophy)

- X-linked Adrenoleukodystrophy

- Primary immunodeficiency diseases

- Bone marrow failure

- Beta-thalassemia

- 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

Treatment Description:

Treatment, including pre-transplant conditioning and GVHD prophylaxis, will occur per each

transplant center's specifications.

Accrual Objective:

In this access and distribution protocol, U.S. patients undergoing transplant using
unlicensed

CBUs will be enrolled and there is no accrual maximum.

Accrual Period:

The accrual period is open ended.