A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

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Brief Title

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

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

      This study is an access and distribution protocol for unlicensed cryopreserved cord blood
      units (CBUs) in pediatric and adult patients with hematologic malignancies and other
      indications.

Detailed Description

      Principal Investigators:

      The principal investigators (PIs) will be transplant physicians at all participating U.S.
      transplant centers.

      Study Design:

      This study is an access and distribution protocol for unlicensed cryopreserved cord blood
      units (CBUs) in pediatric and adult patients with hematologic malignancies and other
      indications.

      Primary Objective:

      The primary objective of this study is to examine the incidence of neutrophil recovery of
      ≥500/mm3 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 a non-licensed CBU:

        -  Assess incidence of transmission of infection

        -  Assess incidence of serious infusion reaction

        -  Determine 1 year overall 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 mcL and >50,000 mcL

Study Type

Observational

Primary Outcome

Neutrophil recovery of ≥500/mm3 after cord blood transplantation

Condition

Hematologic Malignancies

Intervention

A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)

Study Arms / Comparison Groups

 Unlicensed CBU
Description:  The cohort includes recipients of any age receiving unlicensed cryopreserved cord blood units (CBUs) for designated indications.

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

Drug

Estimated Enrollment

99999

Start Date

October 2011

Completion Date

October 2041

Primary Completion Date

October 2041

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 - N/A

Accepts Healthy Volunteers

No

Contacts

John Miller, MD, PhD, , [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations

NCT ID

NCT01351545

Organization ID

10-CBA

Responsible Party

Sponsor

Study Sponsor

Center for International Blood and Marrow Transplant Research

Collaborators

 National Marrow Donor Program

Study Sponsor

John Miller, MD, PhD, Study Chair, NMDP/CIBMTR

Verification Date

November 2022