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

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ClinicalTrials.gov Identifier: NCT00012545
Recruitment Status : Recruiting
First Posted : March 12, 2001
Last Update Posted : March 12, 2019
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Tracking Information
First Submitted Date March 10, 2001
First Posted Date March 12, 2001
Last Update Posted Date March 12, 2019
Actual Study Start Date March 8, 2001
Primary Completion Date Not Provided
Current Primary Outcome Measures
 (submitted: October 3, 2018)
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, as controls [ Time Frame: End of study ]
To evaluate the feasibility of performing directed donor umbilical cord blood banking for families at risk for having children with congenital diseases amenable to treatment by autologous gene therapy or allogeneic hematopoietic transplantation; in this protocol, the feasibility will be studied specifically in families with risk for sickle cell anemia and related syndromes.
Original Primary Outcome Measures Not Provided
Change History Complete list of historical versions of study NCT00012545 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease
Official Title Collection and Storage of Umbilical Cord Hematopoietic Stem Cells for Sickle Cell Disease Therapy
Brief 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.

Detailed Description 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.
Study Type Observational
Study Design Observational Model: Case-Control
Time Perspective: Cross-Sectional
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population This is a pilot feasibility study for which umbilical cord blood samples will be collected and transported to the NIH Clinical Center for our developmental research. Pregnant women whose babies are at risk for sickle cell anemia will be identified and referred to the NIH Research Coordinator for evaluation and entry into the study. The NIH Research Coordinator is experienced in performing patient education and counseling concerning sickle cell disease, and obtaining informed consent.
Condition
  • Sickle Cell Disease
  • Sickle Cell Trait
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: July 23, 2009)
99999999
Original Enrollment
 (submitted: June 23, 2005)
9999999
Study Completion Date Not Provided
Primary Completion Date Not Provided
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 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.

Sex/Gender
Sexes Eligible for Study: Female
Ages 18 Years to 45 Years   (Adult)
Accepts Healthy Volunteers Yes
Contacts
Contact: Wynona Coles (301) 402-2104 wcoles@nhlbi.nih.gov
Contact: John F Tisdale, M.D. (301) 402-6497 johntis@mail.nih.gov
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT00012545
Other Study ID Numbers 010122
01-H-0122
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
Study Sponsor National Heart, Lung, and Blood Institute (NHLBI)
Collaborators Not Provided
Investigators
Principal Investigator: John F Tisdale, M.D. National Heart, Lung, and Blood Institute (NHLBI)
PRS Account National Institutes of Health Clinical Center (CC)
Verification Date December 19, 2018