Providing Access to Cord Blood Units for Transplants

• Status: Withdrawn
• Trial end date: 2012-11-26
• Target enrollment: 0
• Participant gender: All

Summary

Background:

• Cord blood banks have been set up to collect and store umbilical cord blood for transplants. These transplants are used to treat different types of cancer. In October 2011, the Food and Drug Administration (FDA) began considering cord blood as a biological drug. Most of the cord blood units currently available in cord blood banks in the United States and other countries were collected before the FDA set these new standards. The units meet standards set by the National Marrow Donor Program (NMDP), but they were not collected, tested, or stored exactly according to FDA standards. As a result, the new guidelines state that they may only be used for transplant if the transplant is done as part of a study. Researchers have set up a study to provide these cord blood units to recipients and to study the effects of their use.

Objectives:

• To provide access to cord blood units for recipients whose best choice for a unit meets NMDP but not FDA standards.

• To study the effects of these cord blood transplants.

Eligibility:

• Individuals who need to have a cord blood transplant to treat certain types of cancer.

Design:

• Participants will be screened with a physical exam, medical history. They will also have blood tests and imaging studies.

• Participants will have the cord blood transplant and allow their medical data to be collected by the study researchers.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Cancer Institute (NCI)

Criteria

• INCLUSION CRITERIA:

Patients with FDA-specified indications:

• 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