Ignite Creation Date:
2024-05-05 @ 11:22 AM
Last Modification Date:
2024-10-26 @ 9:06 AM
Study NCT ID:
NCT00012545
Status:
RECRUITING
Last Update Posted:
2024-07-15
First Post:
2001-03-10
Brief Title:
Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Collection and Storage of Umbilical Cord Hematopoietic Stem Cells for Sickle Cell Disease Therapy
Status:
RECRUITING
Status Verified Date:
2024-05-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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 BankStem Cell Transplant Laboratory CCBBSTCL 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 CCBBSTCL s directed donation kit program The collected CBU and needed samples will be transported to the CCBBSTCL 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 CCBBSTCL is understood to be a contracted service for NIH Sickle Cell Cord Blood Program
CCBBSTCL has been contracted for a fee-for-service to collect and store cord blood units CCBBSTCL will not be engaged in human subject research activities eg recruitment consenting maintenance processing of data data analysis manuscript writing access to personal identifiable information linked to data andor access to a key code etc
The NIH Sickle Cell Cord Blood Program has contracted Duke University s Carolina Cord Blood BankStem Cell Transplant Laboratory CCBBSTCL 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 CCBBSTCL s directed donation kit program The collected CBU and needed samples will be transported to the CCBBSTCL 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 CCBBSTCL is understood to be a contracted service for NIH Sickle Cell Cord Blood Program
CCBBSTCL has been contracted for a fee-for-service to collect and store cord blood units CCBBSTCL will not be engaged in human subject research activities eg recruitment consenting maintenance processing of data data analysis manuscript writing access to personal identifiable information linked to data andor access to a key code etc
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 01-H-0122 | None | None | None |