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
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(Registered Trademark) (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.
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.
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
- 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
- Patients of any age with FDA-specified indications
- Signed informed consent (and assent when applicable)
- Patients who have licensed CBUs available
- Cord blood transplant recipients at international transplant centers
-Treatment description, including pre-transplant conditioning and GVHD prophylaxis, will
occur per each transplant center's specification.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
To examine the incidence of neutrophil recovery of greater than or equal to 500/mm (3) after unlicensed CBU transplant.
Richard W Childs, M.D.
National Heart, Lung, and Blood Institute (NHLBI)
United States: Federal Government
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Bethesda, Maryland 20892|