Sickle Cell DiseaseNCT00012545
Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease
An observational study of Sickle Cell Disease and Sickle Cell Trait, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Sickle Cell Disease, Sickle Cell Trait
- How many people
- 307 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Nov 2001to 17 Oct 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- All sexes
- No healthy volunteers
- Adult
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.
Who may be able to join
- 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
Show all 13Show fewer
- Hemoglobin SE
- Hemoglobin SS
- Or other sickle-related variant determined acceptable by PI
- Maternal subjects must be between 18 and 45 years old.
- Maternal subjects must be able to provide informed consent.
- Maternal subjects must work with their obstetrician to insure appropriate cord blood collection, sample collection, and completion of related documents.
- Maternal subjects enrolled on other studies are not excluded, unless the other study may interfere with the present one.
Who cannot join
- 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.
- Maternal subject will not be eligible for the study if she has active syphilis, toxoplasmosis, malaria or
- 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.
- Maternal subject has cancer or received treatment for cancer during pregnancy.
Show all 9Show fewer
- Infant is premature (<34 weeks).
- 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.
- Cord blood received by CCBB/STLC is greater than 72 hours from collection
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: Collection and Storage of Umbilical Cord Hematopoietic Stem Cells for Sickle Cell Disease Therapy
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.
Read more
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.
...
Read the 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 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 500 subjects until 50 CBU from newborns with homozygous sickle cell disease (HbSS) have been cryopreserved.
The NIH Sickle Cell Cord Blood Program has contracted Duke University s Carolina Cord Blood Bank/Stem Cell Transplant Laboratory (CCBB/STCL)*^, a CAP and FACTaccredited and CLIA certified laboratory, to facilitate maternal screening, cord blood kit distribution or onsite collection, processing, and storage of cord blood for maternal subjects identified as being at risk of having an infant with sickle cell disease. Mothers between the ages of 18 and 45 years who meet specified medical history criteria will deliver at her preferred hospital using CCBB/STCL s directed donation kit program. The collected CBU and needed samples will be transported to the CCBB/STCL processing facility for processing and storage. Clinical grade frozen CBUs will be transferred to the NIH for future clinical transplantation (related allogeneic transplant) or gene therapy studies (autologous transplant) under an IRB approved protocol.
*Any future mention of CCBB/STCL is understood to be a contracted service for NIH Sickle Cell Cord Blood Program.
^CCBB/STCL has been contracted for a fee-for-service to collect and store cord blood units. CCBB/STCL will not be engaged in human subject research activities (e.g. recruitment, consenting, maintenance/ processing of data, data analysis, manuscript writing, access to personal identifiable information linked to data, and/or access to a key code, etc.).
- Conditions
- Sickle Cell Disease, Sickle Cell Trait
- Keywords
- Stem Cells, Sickle Cell Disease, Umbilical Cord Blood, Natural History
- Medical subject headings
- Anemia, Sickle Cell, Sickle Cell Trait
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Healthy Pregnant Volunteers | 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 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To procure cord blood units (CBU) from newborns at risk for sickle cell disease, sickle cell trait, and related disorders as well as normal newborns, as controls
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.
Measured: end-of-study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2001 (estimated)
- Study start1 Nov 2001
- Primary completion17 Oct 2023
- Study completion17 Oct 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 307 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- John F Tisdale, M.D., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00012545
- Sponsor's protocol ID
- 010122
- Other IDs
- 01-H-0122
- Status last verified
- Jan 2026
- First submitted
- 10 Mar 2001
- Last updated
- 9 Jan 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Fraser JK, Cairo MS, Wagner EL, McCurdy PR, Baxter-Lowe LA, Carter SL, Kernan NA, Lill MC, Slone V, Wagner JE, Wallas CH, Kurtzberg J. Cord Blood Transplantation Study (COBLT): cord blood bank standard operating procedures. J Hematother. 1998 Dec;7(6):521-61. doi: 10.1089/scd.1.1998.7.521.
PubMed 9919946Klein HG, Garner RJ, Miller DM, Rosen SL, Statham NJ, Winslow RM. Automated partial exchange transfusion in sickle cell anemia. Transfusion. 1980 Sep-Oct;20(5):578-84. doi: 10.1046/j.1537-2995.1980.20581034515.x.
PubMed 7423597Sykes M, Szot GL, Swenson KA, Pearson DA. Induction of high levels of allogeneic hematopoietic reconstitution and donor-specific tolerance without myelosuppressive conditioning. Nat Med. 1997 Jul;3(7):783-7. doi: 10.1038/nm0797-783.
PubMed 9212108
Study at a glance
From the registry record, last updated by the sponsor on 9 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Collection and Storage of Umbilical Cord Hematopoietic Stem Cells for Sickle Cell Disease Therapy
- ClinicalTrials.gov ID
- NCT00012545
- Acronym / sponsor ID
- 010122
- Conditions
- Sickle Cell Disease, Sickle Cell Trait
- Keywords
- Stem Cells, Sickle Cell Disease, Umbilical Cord Blood, Natural History
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 307 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 1 Nov 2001 (actual)
- Primary completion
- 17 Oct 2023 (actual)
- Study completion
- 17 Oct 2023 (actual)
- First posted
- 12 Mar 2001 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 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 500 subjects until 50 CBU from newborns with homozygous sickle cell disease (HbSS) have been cryopreserved.
The NIH Sickle Cell Cord Blood Program has contracted Duke University s Carolina Cord Blood Bank/Stem Cell Transplant Laboratory (CCBB/STCL)*^, a CAP and FACTaccredited and CLIA certified laboratory, to facilitate maternal screening, cord blood kit distribution or onsite collection, processing, and storage of cord blood for maternal subjects identified as being at risk of having an infant with sickle cell disease. Mothers between the ages of 18 and 45 years who meet specified medical history criteria will deliver at her preferred hospital using CCBB/STCL s directed donation kit program. The collected CBU and needed samples will be transported to the CCBB/STCL processing facility for processing and storage. Clinical grade frozen CBUs will be transferred to the NIH for future clinical transplantation (related allogeneic transplant) or gene therapy studies (autologous transplant) under an IRB approved protocol.
*Any future mention of CCBB/STCL is understood to be a contracted service for NIH Sickle Cell Cord Blood Program.
^CCBB/STCL has been contracted for a fee-for-service to collect and store cord blood units. CCBB/STCL will not be engaged in human subject research activities (e.g. recruitment, consenting, maintenance/ processing of data, data analysis, manuscript writing, access to personal identifiable information linked to data, and/or access to a key code, etc.).
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Healthy Pregnant Volunteers | – | –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 |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- 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.
Key inclusion criteria (13 listed)
- Pregnant women who are at risk of having an infant or infants (in case of multiple births) with sickle cell
- Hemoglobin Sbeta 0 thalassemia
- Hemoglobin Sbeta + thalassemia
- Hemoglobin SC
- Hemoglobin SD
Key exclusion criteria (9 listed)
- 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.
- Maternal subject will not be eligible for the study if she has active syphilis, toxoplasmosis, malaria or
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To procure cord blood units (CBU) 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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jan 2026.
- Background: PubMed 9919946Fraser JK, Cairo MS, Wagner EL, McCurdy PR, Baxter-Lowe LA, Carter SL, Kernan NA, Lill MC, Slone V, Wagner JE, Wallas CH, Kurtzberg J. Cord Blood Transplantation Study (COBLT): cord blood bank standard operating procedures. J Hematother. 1998 Dec;7(6):521-61. doi: 10.1089/scd.1.1998.7.521.
- Background: PubMed 7423597Klein HG, Garner RJ, Miller DM, Rosen SL, Statham NJ, Winslow RM. Automated partial exchange transfusion in sickle cell anemia. Transfusion. 1980 Sep-Oct;20(5):578-84. doi: 10.1046/j.1537-2995.1980.20581034515.x.
- Background: PubMed 9212108Sykes M, Szot GL, Swenson KA, Pearson DA. Induction of high levels of allogeneic hematopoietic reconstitution and donor-specific tolerance without myelosuppressive conditioning. Nat Med. 1997 Jul;3(7):783-7. doi: 10.1038/nm0797-783.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John F Tisdale, M.D. principal investigator
National Heart, Lung, and Blood Institute (NHLBI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease and Sickle Cell Trait, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To procure cord blood units (CBU) 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00012545, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jan 2026.