Bethesda, Maryland 20892

  • Sickle Cell Trait

Purpose:

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.


Study summary:

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 and store clinical grade cord blood units (CBU) for future use in clinical transplantation or gene therapy. Cord blood units will be collected from an indefinite number of subjects until 50 CBU from newborns with homozygous sickle cell disease have been cryopreserved. Maternal subjects will be identified as being at risk to have an infant with sickle cell disease, will be between the ages of 18 and 45 years, and will meet specified medical history criteria. Mothers will have to deliver at INOVA Fairfax Hospital (collection provider site) and the CB will be collected by NCBP collectors ex utero, will be transported to the NCBP CB facility and will be processed, tested and stored. Frozen CBU will be transferred to the NIH for clinical transplantation or gene therapy studies upon NIH request.


Criteria:

- INCLUSION CRITERIA: 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.) 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 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.


NCT ID:

NCT00012545


Primary Contact:

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


Backup Contact:

Email: johntis@mail.nih.gov
John F Tisdale, M.D.
Phone: (301) 402-6497


Location Contact:

Bethesda, Maryland 20892
United States

For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)
Phone: 800-411-1222
Email: prpl@cc.nih.gov

Site Status: Recruiting


Data Source: ClinicalTrials.gov

Date Processed: June 22, 2021

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