Providing Access to Cord Blood Units for Transplants

This study has been withdrawn prior to enrollment.
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )
ClinicalTrials.gov Identifier:
NCT01479582
First received: November 22, 2011
Last updated: June 25, 2014
Last verified: November 2012
  Purpose

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.

Condition Intervention Phase
Chronic Myelogous Leukemia/Other Leukemia
Acute Leukemias
MDS/MPS
Multiple Myeloma
Non-Hodgkin Lymphoma
Drug: Access to unlicensed cord blood units
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC):

Primary Outcome Measures:
  • The primary aim of this study is to examine the incidence of neutrophil recovery of greater than or equal to 500/mm3 after cord blood transplantation in a multi-institution setting using CBUs that are not Food and Drug Administration (FDA) licen...

Secondary Outcome Measures:
  • 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 aGVHD vs cGVHD.

Enrollment: 0
Study Start Date: October 2011
Study Completion Date: November 2012
Primary Completion Date: November 2012 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Access to unlicensed cord blood units
    N/A
  Show Detailed Description

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01479582

Sponsors and Collaborators
Investigators
Principal Investigator: Ronald E Gress, M.D. National Cancer Institute (NCI)
  More Information

Publications:
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )
ClinicalTrials.gov Identifier: NCT01479582     History of Changes
Other Study ID Numbers: 120027, 12-C-0027
Study First Received: November 22, 2011
Last Updated: June 25, 2014
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Adult and Pediatric
Unlicensed Cord Blood Unit IND
NMDP
Transplant
Hematologic Malignancies
Leukemia
Multiple Myeloma
Non-Hodgkin Lymphoma
CML
Chronic Myelogenous Leukemia
Myelodyplastic Syndrome
MDS
Thalassemia
Lysosomal Storage Diseases

Additional relevant MeSH terms:
Leukemia
Lymphoma
Lymphoma, Non-Hodgkin
Multiple Myeloma
Neoplasms, Plasma Cell
Hematologic Neoplasms
Neoplasms by Histologic Type
Neoplasms
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Neoplasms by Site

ClinicalTrials.gov processed this record on August 21, 2014