| Conditions: |
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Healthy - Sickle Cell Anemia |
| Purpose: |
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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.
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| Study Summary: |
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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. This study will be a multisite collaboration with Washington
metropolitan area hospitals.
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| Criteria: |
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- 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.
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| NCT ID: |
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NCT00012545 |
| Primary Contact: |
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Principal Investigator
John F Tisdale, M.D.
National Heart, Lung, and Blood Institute (NHLBI)
Wynona Coles
Phone: (301) 402-2104
Email: wcoles@nhlbi.nih.gov
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| Backup Contact: |
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Email: johntis@mail.nih.gov
John F Tisdale, M.D.
Phone: (301) 402-6497
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| Location Contact: |
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Bethesda, Maryland 20892
United States
For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL)
Phone: 800-411-1222
Email: prpl@mail.cc.nih.gov
Site Status: Recruiting |