Transplants With Unlicensed Preserved Cord Blood

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Brief Title

Transplants With Unlicensed Preserved Cord Blood

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

Brief Summary

      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....
    

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) obtained prior to study enrollment

      Exclusion Criteria

        -  Patients who have licensed CBUs available

        -  Cord blood transplant recipients at international transplant centers

        -  Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)

        -  Patients whose selected unlicensed CBU(s) will be more than minimally manipulated

      Design

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

Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

To examine the incidence of neutrophil recovery of greater than or equal to 500/mm (3) after unlicensed CBU transplant.

Secondary Outcome

 Assess incidence of graft rejection, transmission of infection, serious infusion reactions.

Condition

Myelodysplastic Syndrome (MDS)

Intervention

Cord Blood Transplant

Study Arms / Comparison Groups

 1
Description:  cord blood transplant with unlicensed CBU

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

February 21, 2012

Completion Date

October 20, 2036

Primary Completion Date

October 20, 2036

Eligibility 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) obtained prior to study
             enrollment

          -  Pediatric and adult patients of any age

        EXCLUSION CRITERIA:

          -  Patients who are receiving only licensed CBUs

          -  Cord blood transplant recipients at international transplant centers

          -  Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)

          -  Patients whose selected unlicensed CBU(s) will be more than minimally manipulated
      

Gender

All

Ages

N/A - 99 Years

Accepts Healthy Volunteers

No

Contacts

Richard W Childs, M.D., (301) 451-7128, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01553461

Organization ID

120064

Secondary IDs

12-H-0064

Responsible Party

Sponsor

Study Sponsor

National Heart, Lung, and Blood Institute (NHLBI)


Study Sponsor

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


Verification Date

April 16, 2021