Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease
| Status: | Recruiting |
|---|---|
| Conditions: | Healthy Studies, Anemia |
| Therapuetic Areas: | Hematology, Other |
| Healthy: | No |
| Age Range: | 18 - 45 |
| Updated: | 3/14/2019 |
| Start Date: | March 8, 2001 |
| Contact: | Wynona Coles |
| Email: | wcoles@nhlbi.nih.gov |
| Phone: | (301) 402-2104 |
Collection and Storage of Umbilical Cord Hematopoietic Stem Cells for Sickle Cell Disease Therapy
This study will determine the best ways to collect, process and store umbilical cord blood
from babies with sickle cell disease, sickle cell trait and unaffected babies. Sickle cell
disease is an abnormality of the hemoglobin in red blood cells that causes the cells to
change shape and clump together, preventing their normal flow in the bloodstream. This
impairs blood flow to various organs, and the resulting oxygen deprivation causes organ
damage.
Cord blood is rich in stem cells (cells produced in the bone marrow that mature to different
types of blood cells), which may prove useful in new sickle cell therapies. However, cord
blood from babies with sickle cell trait, sickle cell disease and normal babies may act
differently under laboratory conditions, so it is important to learn how best to work with
blood from all three groups of babies for future use in possible treatments.
Pregnant women between 18 and 45 years of age who are at risk of having an infant with sickle
cell disease and normal volunteers who are pregnant and not at risk for this disease may be
eligible for this study. Potential participants will be counseled about donating her infant s
blood in order to make an informed choice.
All women who participate in the study will provide a medical history and have blood
collected from the umbilical cord and placenta (afterbirth) after the baby s delivery. The
blood will be tested for various infectious diseases, processed, frozen and stored for
research purposes. In addition, blood from women with babies at risk for sickle cell disease
will be tested for the presence of the sickle cell gene, tissue typed, and used for research
as follows:
- Sickle cell disease If cord blood tests show the baby has sickle cell disease, the blood
will be frozen for an indefinite period of time for possible use in future treatment of
the child. This treatment could include stem cell transplantation or gene therapy,
treatments are not currently considered routine for sickle cell disease.
- Sickle cell trait or normal hemoglobin If cord blood tests show the baby has sickle cell
trait or is unaffected, the blood will be processed and stored for up to 3 years, during
which time it may possibly be used to treat a currently living or future sibling with
sickle cell disease. After 3 years, the participant may agree to either have the blood
discarded, given to research or moved to another facility for continued storage at the
participant s expense, if there is a storage fee. Alternatively, if there is no
anticipated future need for the collected blood, or if it does not meet standards needed
for future treatment, it will be used in NIH-approved research studies.
Participants and their family doctor or the baby s pediatrician will be contacted twice a
year for information about changes in the baby s health. Participants may also be asked
permission to perform new tests developed by researchers.
from babies with sickle cell disease, sickle cell trait and unaffected babies. Sickle cell
disease is an abnormality of the hemoglobin in red blood cells that causes the cells to
change shape and clump together, preventing their normal flow in the bloodstream. This
impairs blood flow to various organs, and the resulting oxygen deprivation causes organ
damage.
Cord blood is rich in stem cells (cells produced in the bone marrow that mature to different
types of blood cells), which may prove useful in new sickle cell therapies. However, cord
blood from babies with sickle cell trait, sickle cell disease and normal babies may act
differently under laboratory conditions, so it is important to learn how best to work with
blood from all three groups of babies for future use in possible treatments.
Pregnant women between 18 and 45 years of age who are at risk of having an infant with sickle
cell disease and normal volunteers who are pregnant and not at risk for this disease may be
eligible for this study. Potential participants will be counseled about donating her infant s
blood in order to make an informed choice.
All women who participate in the study will provide a medical history and have blood
collected from the umbilical cord and placenta (afterbirth) after the baby s delivery. The
blood will be tested for various infectious diseases, processed, frozen and stored for
research purposes. In addition, blood from women with babies at risk for sickle cell disease
will be tested for the presence of the sickle cell gene, tissue typed, and used for research
as follows:
- Sickle cell disease If cord blood tests show the baby has sickle cell disease, the blood
will be frozen for an indefinite period of time for possible use in future treatment of
the child. This treatment could include stem cell transplantation or gene therapy,
treatments are not currently considered routine for sickle cell disease.
- Sickle cell trait or normal hemoglobin If cord blood tests show the baby has sickle cell
trait or is unaffected, the blood will be processed and stored for up to 3 years, during
which time it may possibly be used to treat a currently living or future sibling with
sickle cell disease. After 3 years, the participant may agree to either have the blood
discarded, given to research or moved to another facility for continued storage at the
participant s expense, if there is a storage fee. Alternatively, if there is no
anticipated future need for the collected blood, or if it does not meet standards needed
for future treatment, it will be used in NIH-approved research studies.
Participants and their family doctor or the baby s pediatrician will be contacted twice a
year for information about changes in the baby s health. Participants may also be asked
permission to perform new tests developed by researchers.
Umbilical cord blood is a source of hematopoietic stem cells (HSCs) for transplantation or
gene therapy. Our goal is to procure umbilical cord blood (UCB) from newborns at risk for
sickle cell disease, sickle cell trait, and related disorders as well as normal newborns, for
our controls, in order to develop methods for processing and cryopreservation of umbilical
cord blood HSCs for use in future clinical transplantation or gene therapy. In order to carry
out our methods development research umbilical cord blood units will be collected from an
indefinite number of subjects until 30 cord blood units from newborns with sickle cell
disease have been cryopreserved. These units will be stored for future gene therapy. Maternal
subjects will have been identified as being at risk to have an infant with sickle cell
disease, will be between the ages of 18 and 45, and will meet specified medical history
criteria. The cord blood units will be tested for transfusion transmissible viruses,
infectious disease markers, Human Leukocyte Antigen (HLA) typing, Hemoglobin genotyping, and
enumeration of progenitor cells. The umbilical cord blood units will be used for the
developmental research on processing/cryopreservation methods but, once processed and stored,
may also be identified for future clinical use or for basic or translational research by NIH
investigators.
gene therapy. Our goal is to procure umbilical cord blood (UCB) from newborns at risk for
sickle cell disease, sickle cell trait, and related disorders as well as normal newborns, for
our controls, in order to develop methods for processing and cryopreservation of umbilical
cord blood HSCs for use in future clinical transplantation or gene therapy. In order to carry
out our methods development research umbilical cord blood units will be collected from an
indefinite number of subjects until 30 cord blood units from newborns with sickle cell
disease have been cryopreserved. These units will be stored for future gene therapy. Maternal
subjects will have been identified as being at risk to have an infant with sickle cell
disease, will be between the ages of 18 and 45, and will meet specified medical history
criteria. The cord blood units will be tested for transfusion transmissible viruses,
infectious disease markers, Human Leukocyte Antigen (HLA) typing, Hemoglobin genotyping, and
enumeration of progenitor cells. The umbilical cord blood units will be used for the
developmental research on processing/cryopreservation methods but, once processed and stored,
may also be identified for future clinical use or for basic or translational research by NIH
investigators.
- INCLUSION CRITERIA:
Pregnant women who are at risk of having an infant with sickle cell anemia (HbSS), as well
as woman who are not at risk and wish to serve as control subjects, will be identified and
referred by their health care providers or will be self-referred.
Maternal subjects must be between 18 and 45 years old, may be in their first or subsequent
pregnancy, and must be able to provide informed consent.
EXCLUSION CRITERIA:
The maternal subject will not be eligible for study if she is known to be positive for one
or more of the following diseases transmissible by blood: HIV, hepatitis B, hepatitis C, or
HTLV; is unable to give informed consent; or is known to have a fetus with a significant
congenital anomaly.
Subjects may be excluded at the time of delivery if the attending physician or collection
staff, due to unanticipated obstetrical complications, deems cord blood collection
inadvisable.
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
Phone: 800-411-1222
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