Providing Access to Cord Blood Units for Transplants

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.

ClinicalTrials.gov Identifier: NCT01479582

Recruitment Status : Withdrawn

First Posted : November 24, 2011

Last Update Posted : December 21, 2018

View this study on Beta.ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )

Study Description

Brief 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.

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

Show detailed description

Detailed Description:

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.

Study Design

Layout table for study information

Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	0 participants
Allocation:	Non-Randomized
Intervention Model:	Single Group Assignment
Masking:	None (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
Study Start Date :	October 27, 2011
Actual Primary Completion Date :	November 26, 2012
Actual Study Completion Date :	November 26, 2012

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Multiple myeloma

Genetic and Rare Diseases Information Center resources: Multiple Myeloma Lymphosarcoma Myeloid Leukemia Chronic Graft Versus Host Disease Chronic Myeloid Leukemia Thalassemia Acute Graft Versus Host Disease

U.S. FDA Resources

Arms and Interventions

Outcome Measures

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.

Eligibility Criteria

Information from the National Library of Medicine

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, Learn About Clinical Studies.

Layout table for eligibility information

Ages Eligible for Study:	up to 99 Years (Child, Adult, Older Adult)
Sexes Eligible for Study:	All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01479582

Sponsors and Collaborators

National Cancer Institute (NCI)

Investigators

Layout table for investigator information

Principal Investigator:	Ronald E Gress, M.D.	National Cancer Institute (NCI)

More Information

Publications:

Laughlin MJ, Barker J, Bambach B, Koc ON, Rizzieri DA, Wagner JE, Gerson SL, Lazarus HM, Cairo M, Stevens CE, Rubinstein P, Kurtzberg J. Hematopoietic engraftment and survival in adult recipients of umbilical-cord blood from unrelated donors. N Engl J Med. 2001 Jun 14;344(24):1815-22. doi: 10.1056/NEJM200106143442402.

Kurtzberg J, Laughlin M, Graham ML, Smith C, Olson JF, Halperin EC, Ciocci G, Carrier C, Stevens CE, Rubinstein P. Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients. N Engl J Med. 1996 Jul 18;335(3):157-66. doi: 10.1056/NEJM199607183350303.

Rocha V, Labopin M, Sanz G, Arcese W, Schwerdtfeger R, Bosi A, Jacobsen N, Ruutu T, de Lima M, Finke J, Frassoni F, Gluckman E; Acute Leukemia Working Party of European Blood and Marrow Transplant Group; Eurocord-Netcord Registry. Transplants of umbilical-cord blood or bone marrow from unrelated donors in adults with acute leukemia. N Engl J Med. 2004 Nov 25;351(22):2276-85. doi: 10.1056/NEJMoa041469.

Layout table for additonal information

Responsible Party:	National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:	NCT01479582
Other Study ID Numbers:	120027 12-C-0027
First Posted:	November 24, 2011 Key Record Dates
Last Update Posted:	December 21, 2018
Last Verified:	November 26, 2012

Keywords provided by National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) ):


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:

Layout table for MeSH terms

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

To Top