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
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
The primary aim of this study is to examine the incidence of neutrophil recovery of
after cord blood transplantation in a multi-institution setting using CBUs that are not Food
Drug Administration (FDA) licensed.
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
- 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
- Signed informed consent (and signed assent, if applicable)
- Pediatric and adult patients of any age
- Patients who are receiving only licensed CBUs
- Cord blood transplant recipients at international transplant centers
Treatment, including pre-transplant conditioning and GVHD prophylaxis, will occur per each
transplant center's specifications.
In this access and distribution protocol, U.S. patients undergoing transplant using
CBUs will be enrolled and there is no accrual maximum.
The accrual period is open ended.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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...
Karen A Kurdziel, M.D.
National Institutes of Health Clinical Center (CC)
United States: Federal Government