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This study has been withdrawn prior to enrollment.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: May 3, 2013
Last verified: November 2012
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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 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: | May 3, 2013 |
| 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 May 16, 2013