Transplants With Unlicensed Preserved Cord Blood
| Status: | Recruiting |
|---|---|
| Conditions: | Blood Cancer, Anemia, Hematology |
| Therapuetic Areas: | Hematology, Oncology |
| Healthy: | No |
| Age Range: | Any - 99 |
| Updated: | 4/6/2019 |
| Start Date: | February 2, 2012 |
| End Date: | October 20, 2036 |
| Contact: | Richard W Childs, M.D. |
| Email: | childsr@nhlbi.nih.gov |
| Phone: | (301) 451-7128 |
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
Background:
- Cord blood transplants can treat cancers and other diseases in children and adults. The
U.S. Food and Drug Administration (FDA) requires cord blood to be collected and stored under
certain safety standards. However, most available cord blood units were collected before the
FDA set these standards. These units may not meet FDA standards, but they do meet similar
standards set and followed by the National Marrow Donor Program (NMDP). Cord blood units that
do not meet the new FDA standards may be used for transplants only as part of a research
study. Doctors want to allow people who need transplants to receive cord blood that meets
NMDP standards but may not meet FDA standards.
Objectives:
- To allow selected cord blood units that do not meet current FDA standards to be used for
transplant.
Eligibility:
- Individuals who need cord blood units for transplant, and who best match cord blood units
that are not FDA-licensed.
Design:
- Participants will provide consent to receive cord blood that meets NMDP standards but
may not meet FDA standards.
- Participants will remain on the study for observation for up to 1 year after transplant,
or until they withdraw from the study for personal or medical reasons.
- Cord blood transplants can treat cancers and other diseases in children and adults. The
U.S. Food and Drug Administration (FDA) requires cord blood to be collected and stored under
certain safety standards. However, most available cord blood units were collected before the
FDA set these standards. These units may not meet FDA standards, but they do meet similar
standards set and followed by the National Marrow Donor Program (NMDP). Cord blood units that
do not meet the new FDA standards may be used for transplants only as part of a research
study. Doctors want to allow people who need transplants to receive cord blood that meets
NMDP standards but may not meet FDA standards.
Objectives:
- To allow selected cord blood units that do not meet current FDA standards to be used for
transplant.
Eligibility:
- Individuals who need cord blood units for transplant, and who best match cord blood units
that are not FDA-licensed.
Design:
- Participants will provide consent to receive cord blood that meets NMDP standards but
may not meet FDA standards.
- Participants will remain on the study for observation for up to 1 year after transplant,
or until they withdraw from the study for personal or medical reasons.
Background
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 (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.
Primary Objectives
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.
Secondary Objectives
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 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 CBU-derived engraftment
Eligibility Criteria
Inclusion Criteria
- Patients of any age with FDA-specified indications
- Signed informed consent (and assent when applicable) obtained prior to study enrollment
Exclusion Criteria
- Patients who have licensed CBUs available
- Cord blood transplant recipients at international transplant centers
- Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)
- Patients whose selected unlicensed CBU(s) will be more than minimally manipulated
Design
-Treatment description, including pre-transplant conditioning and GVHD prophylaxis, will
occur per each transplant center s specification.
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 (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.
Primary Objectives
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.
Secondary Objectives
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 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 CBU-derived engraftment
Eligibility Criteria
Inclusion Criteria
- Patients of any age with FDA-specified indications
- Signed informed consent (and assent when applicable) obtained prior to study enrollment
Exclusion Criteria
- Patients who have licensed CBUs available
- Cord blood transplant recipients at international transplant centers
- Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)
- Patients whose selected unlicensed CBU(s) will be more than minimally manipulated
Design
-Treatment description, including pre-transplant conditioning and GVHD prophylaxis, will
occur per each transplant center s specification.
- INCLUSION CRITERIA:
- Disorders affecting the hematopoietic system that are inherited, acquired, or result
from myeloablative treatment
- Signed informed consent (and signed assent, if applicable) obtained prior to study
enrollment
- Pediatric and adult patients of any age
EXCLUSION CRITERIA:
- Patients who are receiving only licensed CBUs
- Cord blood transplant recipients at international transplant centers
- Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)
- Patients whose selected unlicensed CBU(s) will be more than minimally manipulated
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
Phone: 800-411-1222
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