Safety Study of Cord Blood Units for Stem Cell Transplants

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Brief Title

Safety Study of Cord Blood Units for Stem Cell Transplants

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

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.
    

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 Phase

Phase 2

Study Type

Interventional


Primary Outcome

safety of administration of the unlicensed investigational NCBP HPC-CORD BLOOD products


Condition

Aplastic Anemia

Intervention

Cord Blood Units

Study Arms / Comparison Groups

 1
Description:  At least one

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

500

Start Date

October 16, 2015

Completion Date

November 30, 2037

Primary Completion Date

November 30, 2037

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)
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Richard W Childs, M.D., (301) 451-3722, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01861093

Organization ID

130116

Secondary IDs

13-H-0116

Responsible Party

Sponsor

Study Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Collaborators

 National Cancer Institute (NCI)

Study Sponsor

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


Verification Date

April 22, 2021