Transplants With Unlicensed Preserved Cord Blood

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by National Institutes of Health Clinical Center (CC)
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier:
NCT01553461
First received: March 13, 2012
Last updated: September 12, 2014
Last verified: August 2014
  Purpose

Background:

- Cord blood transplants can treat cancers and other diseases in children and adults. The U.S. Food and Drug Administration (FDA) requires cord blood to be collected and stored under certain safety standards. However, most available cord blood units were collected before the FDA set these standards. These units may not meet FDA standards, but they do meet similar standards set and followed by the National Marrow Donor Program (NMDP). Cord blood units that do not meet the new FDA standards may be used for transplants only as part of a research study. Doctors want to allow people who need transplants to receive cord blood that meets NMDP standards but may not meet FDA standards.

Objectives:

- To allow selected cord blood units that do not meet current FDA standards to be used for transplant.

Eligibility:

- Individuals who need cord blood units for transplant, and who best match cord blood units that are not FDA-licensed.

Design:

  • Participants will provide consent to receive cord blood that meets NMDP standards but may not meet FDA standards.
  • Participants will remain on the study for observation for up to 1 year after transplant, or until they withdraw from the study for personal or medical reasons.

Condition Intervention Phase
Myelodysplastic Syndrome (MDS)
Hematologic Neoplasms
Hematologic Diseases
Severe Aplastic Anemia
Procedure: Cord Blood Transplant
Phase 1
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:
  • To examine the incidence of neutrophil recovery of greater than or equal to 500/mm (3) after unlicensed CBU transplant.

Secondary Outcome Measures:
  • Assess incidence of graft rejection, transmission of infection, serious infusion reactions.
  • Determine 1 year survival.
  • Assess cumulative incidence of acute GVHD (grades II to IV), (grades III to IV), and incidence of chronic GVHD.

Estimated Enrollment: 500
Study Start Date: February 2012
Estimated Study Completion Date: October 2036
Estimated Primary Completion Date: October 2036 (Final data collection date for primary outcome measure)
Intervention Details:
    Procedure: Cord Blood Transplant
    N/A
Detailed Description:

Background

The requirement for licensure became part of the FDA final rules for Human Cells, Tissues, and Tissue-Based Products (HCT/Ps) (March 29, 2004 and May 25, 2005) in an effort to prevent transmission of communicable disease, minimize contamination and preserved integrity and function during processing, outline safety and effectiveness requirements for cells from unrelated donors or when HCT/Ps are more than minimally manipulated, assure labeling is clear accurate and not misleading and monitor and communicate with industry via establishment registration. However, as of October 20, 2011, those units that do not meet the manufacturing requirements for licensure can only be distributed for transplantation if the transplant will occur under an IND research protocol. These units are in current and future inventory at domestic and international cord blood banks that cannot be demonstrated to meet licensing requirements. In addition to the licensure guidance, the FDA published a guidance in August 2011 entitled Investigational New Drug Applications (INDs) for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications.

This study is an access and distribution protocol conducted by the National Marrow Donor Program (NMDP) for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients, in which sites wishing to receive NMDP unrelated cord blood units for treatment or research must participate with an locally IRB- approved protocol.

Primary Objectives

The primary objective of this study is to examine the incidence of neutrophil recovery of greater than or equal to 500/mm(3) 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 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 CBU-derived engraftment

Eligibility Criteria

Inclusion Criteria

  • Patients of any age with FDA-specified indications
  • Signed informed consent (and assent when applicable)

Exclusion Criteria

  • Patients who have licensed CBUs available
  • Cord blood transplant recipients at international transplant centers

Design

-Treatment description, including pre-transplant conditioning and GVHD prophylaxis, will occur per each transplant center s specification.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria
  • INCLUSION CRITERIA:

Disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment Signed informed consent (and signed assent, if applicable) Pediatric and adult patients of any age

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: NCT01553461

Contacts
Contact: Richard W Childs, M.D. (301) 451-7128 childsr@nhlbi.nih.gov

Locations
United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL)    800-411-1222 ext TTY8664111010    prpl@mail.cc.nih.gov   
Sponsors and Collaborators
Investigators
Principal Investigator: Richard W Childs, M.D. National Heart, Lung, and Blood Institute (NHLBI)
  More Information

Additional Information:
Publications:
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier: NCT01553461     History of Changes
Other Study ID Numbers: 120064, 12-H-0064
Study First Received: March 13, 2012
Last Updated: September 12, 2014
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Transplant
Umbilical Cord Blood
Graft Rejection
Graft Versus Host Disease

Additional relevant MeSH terms:
Anemia, Aplastic
Myelodysplastic Syndromes
Preleukemia
Hematologic Diseases
Hematologic Neoplasms
Anemia
Bone Marrow Diseases
Precancerous Conditions
Neoplasms
Neoplasms by Site

ClinicalTrials.gov processed this record on September 16, 2014