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Clinical Trial VICCBMT1158


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

Principal Investigator(s)

Michael Byrne


  • Protocol No. VICCBMT1158
  • Open Date: 10/20/2011
  • Staging: N/A
  • Age Group: Both Child and Adult
  • Scope: National
  • 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.
  • Disease Sites: Hematologic
  • Therapies: Bone Marrow/Stem Cell Transplant
  • Drugs: None Specified
  • Participating Institutions: Vanderbilt University
  • National Clinical Trial ID: NCT01351545
  • Secondary Protocol No: 10-CBA


Patients are asked to take part in this study because they and their doctor have agreed that the best option for treating their disease is an umbilical cord blood transplant. In October 2011, the Food and Drug Administration (FDA) began considering cord blood as a biological drug. In the United States, drugs must meet standards set by the FDA to make sure they are safe. Cord blood units that were not collected, tested, or stored exactly according to FDA standards may be used for transplant if the transplant is done as part of a study. Umbilical cord blood is taken from the placenta immediately after birth. Human umbilical cord blood is a source of blood forming stem cells that are capable of making different types of new blood cells. Since 1989, cord blood banks have been established in six continents and over one million cord blood collections have been performed. Approximately 10,000 cord blood transplants have been performed in children and adults, for malignant and non-malignant conditions. Most of the cord blood units that are currently available in cord blood banks in the United States and other countries were collected before the FDA set these new standards. Although these cord blood units may not meet FDA standards, they do meet similar standards set and followed by the NMDP for years. Patients are going to have a cord blood transplant and one or more of the cord blood units that are considered by their doctor to be the best choice for their transplant may not meet all the new FDA standards. This study will allow the patient's doctor to use these cord blood units for the patient's transplant. Cord blood units used for transplant on this study must meet standards set by the NMDP. If a cord blood unit meets NMDP standards this means that the cord blood unit is suitable for use. This does not however guarantee that the transplant will be successful in treating the patient's disease. These standards include: Making sure the hospital where the cord blood units are collected follows safe procedures to prevent the cord blood units from coming into contact with germs. Making sure the cord blood units are stored and labeled in a certain way. Making sure the cord blood units are tested for certain viruses and bacteria that could cause illness in the recipient. The primary purposes of this study are to: Provide access to cord blood units for recipients whose best choice for a cord blood unit(s) do not meet all FDA standards, but do meet standards set by the NMDP on this study. Assess how well and how quickly blood counts return to normal after transplant in recipients on this study. This study is sponsored by the National Marrow Donor Program (NMDP). An unlimited number of patients will participate in this study from medical centers throughout the United States. About 15 people from Vanderbilt will be taking part.


Ages Eligible for Study:N/A and older
Genders Eligible for Study:All
Accepts Healthy Volunteers:No


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