Safety Study of Cord Blood Units for Stem Cell Transplants

October 21, 2023 updated by: National Heart, Lung, and Blood Institute (NHLBI)

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

500

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Jennifer S Wilder, R.N.
  • Phone Number: (301) 451-3722
  • Email: jw621w@nih.gov

Study Contact Backup

Study Locations

  • United States
    • Maryland
      • Bethesda, Maryland, United States, 20892
        • Recruiting
        • National Institutes of Health Clinical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

  • 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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 1
at least one
Biological: Cord Blood Units
cord blood units for transplants

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
safety of administration of the unlicensed investigational NCBP HPC-CORD BLOOD products
Time Frame: 1 year post transplant
safety
1 year post transplant

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Collaborators

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)

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 16, 2015

Primary Completion (Estimated)

November 30, 2037

Study Completion (Estimated)

November 30, 2037

Study Registration Dates

First Submitted

May 21, 2013

First Submitted That Met QC Criteria

May 21, 2013

First Posted (Estimated)

May 23, 2013

Study Record Updates

Last Update Posted (Actual)

October 24, 2023

Last Update Submitted That Met QC Criteria

October 21, 2023

Last Verified

October 20, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 130116
  • 13-H-0116

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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