Collection and Storage of Umbilical Cord Hematopoietic Stem Cells for Sickle Cell Disease Therapy

Study point of contact

John F Tisdale, M.D.
(301) 402-6497
[email protected]
Wynona Coles
(301) 402-2104
[email protected]

Locations

1 United States site

Age

< 45 Years

Genotypes

SC, SS

Study type

Observational

Gender

Female

Compensation

Unknown

About the study

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.

participation requirements

Pregnant women who are at risk of having an infant with sickle cell anemia related
diseases.

The types of sickle cell disease include the following:

– Hemoglobin S <=0 thalassemia - Hemoglobin S <=+ thalassemia - Hemoglobin SC - Hemoglobin SD - Hemoglobin SE - Hemoglobin SS - Maternal subjects must be between 18 and 45 years old. - Maternal subjects must be able to provide informed consent. - Maternal subjects deliver at INOVA Fairfax Hospital (insurance approval) - Maternal subjects enrolled on other studies are not excluded, unless the other study may interfere with the present one. - Maternal subjects in active labor will be enrolled at the discretion of her physician. (Note: in rare cases that a mother is identified as a potential candidate for the study during labor, it is at the discretion of her physician whether she could be in a condition to discuss about the study and provide informed consent (then IFH has to obtain the Informed Consent). After labor and delivery, the study will be discussed again with the mother.)

participation restrictions

– 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 or ZIKV.

– Maternal subject will not be eligible for the study if she has active syphilis,
toxoplasmosis, malaria or babesia during pregnancy.

– Maternal subject will not be eligible for the study if the pregnancy resulted from egg
donation or sperm donation.

– Maternal subject is unable to give informed consent.

– Maternal subject is known to have a fetus with a significant congenital anomaly,
either not compatible with life, or requiring immediate surgical intervention or care
in the Neonatal Intensive Care Unit or.

– Maternal subject delivers at another facility, not INOVA Fairfax Hospital.

– Maternal subject has cancer or received treatment for cancer during pregnancy

– Maternal subject may be excluded at the time of delivery if the attending physician or
collection staff deems that the cord blood collection is inadvisable, due to
unanticipated obstetrical complications.

Locations

  • Bethesda, Maryland, United States, National Institutes of Health Clinical Center, 9000 Rockville Pike, 20892 [Recruiting]