A Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units (CBUs) Manufactured by the National Cord Blood Program (NCBP) and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients
- The requirement for licensure of human cell and blood products 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.
As of October 20, 2011, those units of cord blood 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. In addition to the licensure
guidance, the FDA published a guidance in August 2011 titled Investigational New Drug
Applications (INDs) for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical
Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications.
- This is a multi-center study of unlicensed, investigational cryopreserved cord blood
units (CBUs) manufactured by the National Cord Blood Program (NCBP) which provides
access and distribution on an Investigational New Drug (IND) application #6637 to sites
with locally IRB-approved protocols for unrelated hematopoietic stem cell
transplantation of pediatric and adult patients.
-The primary objective of this study is to examine the safety of administration of the
unlicensed investigational NCBP HPC-CORD Blood products, evaluating prospectively the
incidence of serious adverse reactions as well as the incidence of all infusion related
reactions after administration of unlicensed, investigational NCBP CBU.
In patients receiving a non-licensed NCBP CBU (HPC-CORD BLOOD):
- Assess incidence of transmission of infections
- Assess incidence of graft rejection
- Assess incidence of neutrophil engraftment > 500 /ul
- 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/ul and > 50,000/ul
- Determine CBU-derived engraftment
- Patients of any age or either gender with indications for receipt of investigational
HPC-CORD BLOOD who are participating in an NIH-IRB approved clinical trial for
unrelated hematopoietic stem cell transplantation.
- Signed informed consent (and assent when applicable).
- Patients who are receiving licensed CB products (only)
- Patients who are receiving unlicensed CB products from other CB banks (i.e. NMDP)
This study is a multi-NIH institute endeavor designed to allow NHLBI, NCI and NIAID
investigators access investigational HPC-CORD BLOOD for patients participating in NIH-IRB
approved clinical trials for unrelated hematopoietic stem cell transplantation. Treatment,
including pre-treatment conditioning and GVHD prophylaxis will occur per the institute's
NIH-IRB approved clinical trial or the transplant center's specifications.
Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Examine the safety of administration of the unlicensed investigational NCBP HPC-CORD Blood products
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|