Providing Access to Cord Blood Units for Transplants

December 20, 2018 updated by: National Cancer Institute (NCI)

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

Background:

- Cord blood banks have been set up to collect and store umbilical cord blood for transplants. These transplants are used to treat different types of cancer. In October 2011, the Food and Drug Administration (FDA) began considering cord blood as a biological drug. Most of the cord blood units currently available in cord blood banks in the United States and other countries were collected before the FDA set these new standards. The units meet standards set by the National Marrow Donor Program (NMDP), but they were not collected, tested, or stored exactly according to FDA standards. As a result, the new guidelines state that they may only be used for transplant if the transplant is done as part of a study. Researchers have set up a study to provide these cord blood units to recipients and to study the effects of their use.

Objectives:

  • To provide access to cord blood units for recipients whose best choice for a unit meets NMDP but not FDA standards.
  • To study the effects of these cord blood transplants.

Eligibility:

- Individuals who need to have a cord blood transplant to treat certain types of cancer.

Design:

  • Participants will be screened with a physical exam, medical history. They will also have blood tests and imaging studies.
  • Participants will have the cord blood transplant and allow their medical data to be collected by the study researchers.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Detailed Description

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 aim of this study is to examine the incidence of neutrophil recovery of (Bullet)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 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 platelet engraftment of >20,000 mcL and >50,000 mcL
  • Determine CBU-derived engraftment

Eligibility Criteria:

Inclusion Criteria

  • Patients with FDA-specified indications (see Appendix B for further details):
  • Hematological malignancies
  • Certain lysosomal storage and peroxisomal enzyme deficiency disorders
  • Hurler syndrome (MPS I)
  • Krabbe Disease (Globoid Leukodystrophy)
  • X-linked Adrenoleukodystrophy
  • Primary immunodeficiency diseases
  • Bone marrow failure
  • Beta-thalassemia
  • Signed informed consent (and signed assent, if applicable)
  • Pediatric and adult patients of any age

Exclusion Criteria

  • Patients who are receiving only licensed CBUs
  • Cord blood transplant recipients at international transplant centers

Treatment Description:

Treatment, including pre-transplant conditioning and GVHD prophylaxis, will occur per each

transplant center s specifications.

Accrual Objective:

In this access and distribution protocol, U.S. patients undergoing transplant using unlicensed

CBUs will be enrolled and there is no accrual maximum.

Accrual Period:

The accrual period is open ended.

Study Type

Interventional

Phase

  • Phase 2

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

No older than 95 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

  • INCLUSION CRITERIA:

Patients with FDA-specified indications:

  • Hematological malignancies
  • Certain lysosomal storage and peroxisomal enzyme deficiency disorders
  • Hurler syndrome (MPS I)
  • Krabbe Disease (Globoid Leukodystrophy)
  • X-linked Adrenoleukodystrophy
  • Primary immunodeficiency diseases
  • Bone marrow failure
  • Beta-thalassemia
  • Signed informed consent (and signed assent, if applicable)
  • Pediatric and adult patients of any age

EXCLUSION CRITERIA:

  • Patients who are receiving only licensed CBUs
  • Cord blood transplant recipients at international transplant centers

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: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
The primary aim of this study is to examine the incidence of neutrophil recovery of greater than or equal to 500/mm3 after cord blood transplantation in a multi-institution setting using CBUs that are not Food and Drug Administration (FDA) licen...

Secondary Outcome Measures

Outcome Measure
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 aGVHD vs cGVHD.

Collaborators and Investigators

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

Sponsor

National Cancer Institute (NCI)

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.

General Publications

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

October 27, 2011

Primary Completion (Actual)

November 26, 2012

Study Completion (Actual)

November 26, 2012

Study Registration Dates

First Submitted

November 22, 2011

First Submitted That Met QC Criteria

November 22, 2011

First Posted (Estimate)

November 24, 2011

Study Record Updates

Last Update Posted (Actual)

December 21, 2018

Last Update Submitted That Met QC Criteria

December 20, 2018

Last Verified

November 26, 2012

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 120027
  • 12-C-0027

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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