Protocol Details
Collection and Storage of Umbilical Cord Hematopoietic Stem Cells for Sickle Cell Disease Therapy
This study is NOT currently recruiting participants.
Summary
Number |
01-H-0122 |
Sponsoring Institute |
National Heart, Lung and Blood Institute (NHLBI) |
Recruitment Detail |
Type: Completed Study; data analyses ongoing
Gender: Male & Female
Min Age: 18 Years
Max Age: 45 Years |
Referral Letter Required |
No |
Population Exclusion(s) |
Adults who are or may become unable to consent;
Children |
Keywords |
Stem Cells;
Sickle Cell Disease;
Umbilical Cord Blood;
Natural History |
Recruitment Keyword(s) |
None |
Condition(s) |
Sickle Cell Disease;
Sickle Cell Trait |
Investigational Drug(s) |
None |
Investigational Device(s) |
None |
Intervention(s) |
None |
Supporting Site |
National Heart, Lung, and Blood Institute |
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.
Eligibility
INCLUSION CRITERIA:
Pregnant women who are at risk of having an infant or infants (in case of multiple births) with sickle cell
disease of the following types:
Hemoglobin Sbeta 0 thalassemia
Hemoglobin Sbeta + thalassemia
Hemoglobin SC
Hemoglobin SD
Hemoglobin SE
Hemoglobin SS
Or other sickle-related variant determined acceptable by PI
1. Maternal subjects must be between 18 and 45 years old.
2. Maternal subjects must be able to provide informed consent.
3. Maternal subjects must work with their obstetrician to insure appropriate cord blood collection, sample collection, and completion of related documents.
4. Maternal subjects enrolled on other studies are not excluded, unless the other study may interfere with the present one.
EXCLUSION CRITERIA:
1. 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, WNV, HTLV or ZIKV.
2. Maternal subject will not be eligible for the study if she has active syphilis, toxoplasmosis, malaria or
3. Maternal subject will not be eligible for the study if the pregnancy resulted from egg donation or sperm donation.
4. Maternal subject is unable to give informed consent.
5. 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.
6. Maternal subject has cancer or received treatment for cancer during pregnancy.
7. Infant is premature (<34 weeks).
8. 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.
9. Cord blood received by CCBB/STLC is greater than 72 hours from collection
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
Contacts:
Clinical Trials Number:
NCT00012545