Protocol Number: 01-DK-0122

Title:

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

Number:

01-DK-0122

Summary:

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.

Sponsoring Institute:

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Recruitment Detail

Type: Active Accrual Of New Subjects

Gender: Female

Referral Letter Required: No

Population Exclusion(s): Male

Children

Eligibility Criteria:

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 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.

Special Instructions: Currently Not Provided

Keywords:

Processing

Cryopreservation

Gene Therapy

Transplantation

Hemoglobin

Recruitment Keywords:

Sickle Cell

Umbilical Cord Blood

Gene Therapy

Conditions:

Healthy

Sickle Cell Anemia

Investigational Drug(s):

None

Investigational Device(s):

None

Contacts:

Patient Recruitment and Public Liaison Office
Building 61
10 Cloister Court
Bethesda, Maryland 20892-4754
Toll Free: 1-800-411-1222
TTY: 301-594-9774 (local),1-866-411-1010 (toll free)
Fax: 301-480-9793

Electronic Mail:prpl@mail.cc.nih.gov

Citations:

Cord Blood Transplantation Study (COBLT): cord blood bank standard operating procedures

Automated partial exchange transfusion in sickle cell anemia

Induction of high levels of allogeneic hematopoietic reconstitution and donor-specific tolerance without myelosuppressive conditioning

• Questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.
• Technical questions regarding the Clinical Center web site, please contact the Department of Networks and Applications, CC.

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Warren Grant Magnuson Clinical Center (CC)
National Institutes of Health (NIH)
Bethesda, Maryland 20892. Last update: 10/27/2004