Safety Study of Cord Blood Units for Stem Cell Transplants
| Status: | Recruiting |
|---|---|
| Conditions: | Blood Cancer, Lymphoma, Anemia, Hematology, Leukemia |
| Therapuetic Areas: | Hematology, Oncology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 3/1/2019 |
| Start Date: | October 16, 2015 |
| End Date: | November 30, 2037 |
| Contact: | Jennifer S Wilder, R.N. |
| Email: | jw621w@nih.gov |
| Phone: | (301) 451-3722 |
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
Background:
- Cord blood is blood that is taken from the umbilical cord and placenta of healthy newborns
after childbirth. The cord blood collected from a baby is called a cord blood unit. Cord
blood units are stored frozen in public cord blood banks. About 10,000 cord blood transplants
have been performed in children and adults for blood cancers and other diseases in the world.
These transplants have helped save lives and improve treatments. However, not all available
units of cord blood have been collected, stored, and licensed according to specific
government requirements. These unlicensed units can still be used in transplant, but they can
only be given as part of specific research studies. This study will evaluate the safety of
giving these unlicensed units by recording any problems that may occur during and after
giving the cord blood.
Objectives:
- To test the safety and effectiveness of unlicensed cord blood units in people who need stem
cell transplants.
Eligibility:
- Individuals who are scheduled to have a stem cell transplant.
Design:
- Participants will be screened with a medical history and physical exam.
- Participants will receive the cord blood unit as part of their stem cell transplant
procedure. The transplant will be performed according to the current standard of care
for the procedure.
- After the transplant, participants will be monitored for up to 1 year. Any problems or
side effects from the transplant will be treated as necessary. All outcomes will be
reported to the National Cord Blood Program and to the Center for International Blood
and Marrow Transplant.
- Cord blood is blood that is taken from the umbilical cord and placenta of healthy newborns
after childbirth. The cord blood collected from a baby is called a cord blood unit. Cord
blood units are stored frozen in public cord blood banks. About 10,000 cord blood transplants
have been performed in children and adults for blood cancers and other diseases in the world.
These transplants have helped save lives and improve treatments. However, not all available
units of cord blood have been collected, stored, and licensed according to specific
government requirements. These unlicensed units can still be used in transplant, but they can
only be given as part of specific research studies. This study will evaluate the safety of
giving these unlicensed units by recording any problems that may occur during and after
giving the cord blood.
Objectives:
- To test the safety and effectiveness of unlicensed cord blood units in people who need stem
cell transplants.
Eligibility:
- Individuals who are scheduled to have a stem cell transplant.
Design:
- Participants will be screened with a medical history and physical exam.
- Participants will receive the cord blood unit as part of their stem cell transplant
procedure. The transplant will be performed according to the current standard of care
for the procedure.
- After the transplant, participants will be monitored for up to 1 year. Any problems or
side effects from the transplant will be treated as necessary. All outcomes will be
reported to the National Cord Blood Program and to the Center for International Blood
and Marrow Transplant.
Background
- 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.
Objectives
-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.
Secondary Objectives:
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
Eligibility Criteria
Inclusion Criteria
- 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).
Exclusion Criteria
- Patients who are receiving licensed CB products (only)
- Patients who are receiving unlicensed CB products from other CB banks (i.e. NMDP)
Design
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.
- 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.
Objectives
-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.
Secondary Objectives:
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
Eligibility Criteria
Inclusion Criteria
- 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).
Exclusion Criteria
- Patients who are receiving licensed CB products (only)
- Patients who are receiving unlicensed CB products from other CB banks (i.e. NMDP)
Design
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.
- INCLUSION CRITERIA:
- 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).
EXCLUSION CRITERIA:
- Patients who are receiving licensed CB products (only)
- Patients who are receiving unlicensed CB products from other CB banks (i.e. NMDP)
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
Phone: 800-411-1222
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