Providing Access to Cord Blood Units for Transplants

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 (Bullet)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.