Safety Study of Cord Blood Units for Stem Cell Transplants

Sponsor
National Heart, Lung, and Blood Institute (NHLBI) (NIH)
Overall Status
Recruiting
CT.gov ID
NCT01861093
Collaborator
National Cancer Institute (NCI) (NIH), National Institute of Allergy and Infectious Diseases (NIAID) (NIH), National Cord Blood Program, New York Blood Center (Other)
500
Enrollment
1
Location
1
Arm
265.5
Anticipated Duration (Months)
1.9
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Background:
  • Cord blood is blood that is taken from the umbilical cord and placenta of healthy newborns after childbirth. The cord blood collected from a baby is called a cord blood unit. Cord blood units are stored frozen in public cord blood banks. About 10,000 cord blood transplants have been performed in children and adults for blood cancers and other diseases in the world. These transplants have helped save lives and improve treatments. However, not all available units of cord blood have been collected, stored, and licensed according to specific government requirements. These unlicensed units can still be used in transplant, but they can only be given as part of specific research studies. This study will evaluate the safety of giving these unlicensed units by recording any problems that may occur during and after giving the cord blood.
Objectives:
  • To test the safety and effectiveness of unlicensed cord blood units in people who need stem cell transplants.
Eligibility:
  • Individuals who are scheduled to have a stem cell transplant.
Design:
  • Participants will be screened with a medical history and physical exam.

  • Participants will receive the cord blood unit as part of their stem cell transplant procedure. The transplant will be performed according to the current standard of care for the procedure.

  • After the transplant, participants will be monitored for up to 1 year. Any problems or side effects from the transplant will be treated as necessary. All outcomes will be reported to the National Cord Blood Program and to the Center for International Blood and Marrow Transplant.

Condition or DiseaseIntervention/TreatmentPhase
  • Biological: Cord Blood Units
Phase 2

Detailed Description

Background

  • The requirement for licensure of human cell and blood products 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. As of October 20, 2011, those units of cord blood 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. In addition to the licensure guidance, the FDA published a guidance in August 2011 titled Investigational New Drug Applications (INDs) for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications.

  • This is a multi-center study of unlicensed, investigational cryopreserved cord blood units (CBUs) manufactured by the National Cord Blood Program (NCBP) which provides access and distribution on an Investigational New Drug (IND) application #6637 to sites with locally IRB-approved protocols for unrelated hematopoietic stem cell transplantation of pediatric and adult patients.

Objectives

-The primary objective of this study is to examine the safety of administration of the unlicensed investigational NCBP HPC-CORD Blood products, evaluating prospectively the incidence of serious adverse reactions as well as the incidence of all infusion related reactions after administration of unlicensed, investigational NCBP CBU.

Secondary Objectives:
In patients receiving a non-licensed NCBP CBU (HPC-CORD BLOOD):
  • Assess incidence of transmission of infections

  • Assess incidence of graft rejection

  • Assess incidence of neutrophil engraftment >500 /ul

  • 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/ul and >50,000/ul

  • Determine CBU-derived engraftment

Eligibility Criteria

Inclusion Criteria

  • Patients of any age or either gender with indications for receipt of investigational HPC-CORD BLOOD who are participating in an NIH-IRB approved clinical trial for unrelated hematopoietic stem cell transplantation.

  • Signed informed consent (and assent when applicable).

Exclusion Criteria

  • Patients who are receiving licensed CB products (only)

  • Patients who are receiving unlicensed CB products from other CB banks (i.e. NMDP)

Design

This study is a multi-NIH institute endeavor designed to allow NHLBI, NCI and NIAID investigators access investigational HPC-CORD BLOOD for patients participating in NIH-IRB approved clinical trials for unrelated hematopoietic stem cell transplantation. Treatment, including pre-treatment conditioning and GVHD prophylaxis will occur per the institute s NIH-IRB approved clinical trial or the transplant center s specifications.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
500 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units (CBUs) Manufactured by the National Cord Blood Program (NCBP) and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients
Actual Study Start Date :
Oct 16, 2015
Anticipated Primary Completion Date :
Nov 30, 2037
Anticipated Study Completion Date :
Nov 30, 2037

Arms and Interventions

ArmIntervention/Treatment
Experimental: 1

at least one

Biological: Cord Blood Units
cord blood units for transplants

Outcome Measures

Primary Outcome Measures

  1. safety of administration of the unlicensed investigational NCBP HPC-CORD BLOOD products [1 year post transplant]

    safety

Eligibility Criteria

Criteria

Ages Eligible for Study:
4 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
  • INCLUSION CRITERIA:

  • Patients of any age or either gender with indications for receipt of investigational HPC-CORD BLOOD who are participating in an NIH-IRB approved clinical trial for unrelated hematopoietic stem cell transplantation.

  • Signed informed consent (and assent when applicable).

EXCLUSION CRITERIA:
  • Patients who are receiving licensed CB products (only)

  • Patients who are receiving unlicensed CB products from other CB banks (i.e. NMDP)

Contacts and Locations

Locations

SiteCityStateCountryPostal Code
1National Institutes of Health Clinical Center, 9000 Rockville PikeBethesdaMarylandUnited States20892

Sponsors and Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)
  • National Cancer Institute (NCI)
  • National Institute of Allergy and Infectious Diseases (NIAID)
  • National Cord Blood Program, New York Blood Center

Investigators

  • Principal Investigator: Richard W Childs, M.D., National Heart, Lung, and Blood Institute (NHLBI)

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

Publications

None provided.
Responsible Party:
National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier:
NCT01861093
Other Study ID Numbers:
  • 130116
  • 13-H-0116
First Posted:
May 23, 2013
Last Update Posted:
Apr 8, 2022
Last Verified:
Nov 12, 2021
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by National Heart, Lung, and Blood Institute (NHLBI)
Additional relevant MeSH terms:

Study Results

No Results Posted as of Apr 8, 2022