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

This study is currently recruiting participants.
Verified April 2013 by Center for International Blood and Marrow Transplant Research
Sponsor:
Collaborator:
National Marrow Donor Program
Information provided by (Responsible Party):
Center for International Blood and Marrow Transplant Research
ClinicalTrials.gov Identifier:
NCT01351545
First received: May 9, 2011
Last updated: April 23, 2013
Last verified: April 2013
History of Changes
  Purpose

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.


Condition Intervention
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
 Drug: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)

Study Type: Observational
Study Design: Time Perspective: Prospective
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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Center for International Blood and Marrow Transplant Research:

Estimated Enrollment: 99999
Study Start Date: October 2011
Groups/Cohorts Assigned Interventions
Unlicensed CBU
The cohort includes recipients of any age receiving unlicensed cryopreserved cord blood units (CBUs) for designated indications.
 Drug: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
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

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 aim 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 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

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Recipients of unlicensed cryopreserved cord blood units who are being treated as U.S. transplant centers.

Criteria

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),and X-linked Adrenoleukodystrophy), Primary immunodeficiency diseases, Bone marrow failure, and 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01351545

  Show 137 Study Locations
Sponsors and Collaborators
Center for International Blood and Marrow Transplant Research
National Marrow Donor Program
  More Information

No publications provided

Responsible Party: Center for International Blood and Marrow Transplant Research
ClinicalTrials.gov Identifier: NCT01351545     History of Changes
Other Study ID Numbers: 10-CBA
Study First Received: May 9, 2011
Last Updated: April 23, 2013
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Adrenoleukodystrophy
Beta-Thalassemia
Neoplasms
Leukodystrophy, Globoid Cell
Immunologic Deficiency Syndromes
Pancytopenia
Thalassemia
Mucopolysaccharidosis I
Hematologic Neoplasms
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
 Hereditary Central Nervous System Demyelinating Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Leukoencephalopathies
Demyelinating Diseases
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on May 21, 2013