Ignite Creation Date:
2024-05-05 @ 4:43 PM
Last Modification Date:
2024-10-26 @ 9:23 AM
Study NCT ID:
NCT00305344
Status:
COMPLETED
Last Update Posted:
2022-02-09
First Post:
2006-03-17
Brief Title:
Umbilical Cord Blood Infusion to Treat Type 1 Diabetes
Sponsor:
University of Florida
Organization:
University of Florida
Study Overview
Official Title:
Transfusion of Autologous Umbilical Cord Blood to Reverse Hyperglycemia in Children With Type 1 Diabetes - A Pilot Study
Status:
COMPLETED
Status Verified Date:
2022-02
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:
While this study is now completely enrolled we do hope to develop a next generation cord blood based study sometime in early 2009 Please continue to contact us if you have a child with newly diagnosed Type 1 Diabetes T1D who alo has their OWN cord blood in storage
Detailed Description:
Objective
Our goal is to transfuse autologous umbilical cord blood into 23 children with T1D in an attempt to re-establish immune tolerance and perhaps regenerate pancreatic islet insulin-producing beta cells and improve blood glucose control As secondary goals we aim to study the potential changes in metabolismimmune function leading to islet regeneration
BackgroundRationale
Stem cells provide an exciting approach towards curing T1D Autologous bone marrow transplants have been used successfully for patients undergoing high dose chemotherapy and for a variety of cancers and autoimmune disorders such as multiple sclerosis lupus and rheumatic disorders Recent studies in immunodeficient mice with chemically-induced pancreatic damage have shown that bone marrow-derived stem cells preferentially home to the pancreas and may have the capacity to initiate pancreatic regeneration thereby restoring the endothelial interactions in the pancreas and correcting the associated elevated blood sugar levels Human umbilical cord blood cells transfused into a model of amyotrophic lateral sclerosis ALS resulted in delayed disease progression of two to three weeks and increased lifespan Umbilical cord blood has shown promise as an excellent source for deriving stem cell populations and has been used successfully in transplantation for a variety of diseases including acute lymphocytic and myeloid leukemia lymphoma Fanconi anemia and sickle cell disease Furthermore umbilical cord blood-derived stem cells have the capacity to differentiate into a variety of non-blood cell types including hepatocytes neural cells and endothelial cells In addition umbilical cord blood contains a greater proportion of hematopoietic stem cells than bone marrow
In addition cord blood contains a large number of immune cells called regulatory T cells These regulatory T cells may be helpful in diminishing autoimmunity The need to re-establish tolerance in patients with established autoimmunity provides another potential mechanism for cord blood as a therapy for type 1 diabetes
Description of Project
23 children 1 year of age with T1D and stored umbilical cord blood are being be recruited The cord blood will be infused into the children in the GCRC in an attempt to regenerate pancreatic islet insulin-producing beta cells and improve blood glucose control As secondary goals we aim to track the migration of transfused cord blood stem and study the potential changes in metabolismimmune function leading to islet regeneration
Anticipated Outcome
It is hoped that there will be preservation of beta cell function assessed by mixed meal stimulated C-peptide secretion Changes in immunological markersfunction may be observed
Relevance to Type I Diabetes
Type 1 diabetes is still associated with tremendous morbidity and premature mortality Patients require multiple daily insulin injections throughout their lives as well as close monitoring of their diet and blood sugar levels to prevent complications Unfortunately there is presently no permanent cure for diabetes Whole pancreas or islet cell transplantation is available only to a very limited number of patients and necessitates potential lifelong immunosuppressive therapy The need to find a cure for T1D cannot be overstated -autologous stem cell transfusions either with their potential to differentiate into islet cells or provide immune tolerance that leads to islet regeneration appear to be a safe and potentially viable option
Our goal is to transfuse autologous umbilical cord blood into 23 children with T1D in an attempt to re-establish immune tolerance and perhaps regenerate pancreatic islet insulin-producing beta cells and improve blood glucose control As secondary goals we aim to study the potential changes in metabolismimmune function leading to islet regeneration
BackgroundRationale
Stem cells provide an exciting approach towards curing T1D Autologous bone marrow transplants have been used successfully for patients undergoing high dose chemotherapy and for a variety of cancers and autoimmune disorders such as multiple sclerosis lupus and rheumatic disorders Recent studies in immunodeficient mice with chemically-induced pancreatic damage have shown that bone marrow-derived stem cells preferentially home to the pancreas and may have the capacity to initiate pancreatic regeneration thereby restoring the endothelial interactions in the pancreas and correcting the associated elevated blood sugar levels Human umbilical cord blood cells transfused into a model of amyotrophic lateral sclerosis ALS resulted in delayed disease progression of two to three weeks and increased lifespan Umbilical cord blood has shown promise as an excellent source for deriving stem cell populations and has been used successfully in transplantation for a variety of diseases including acute lymphocytic and myeloid leukemia lymphoma Fanconi anemia and sickle cell disease Furthermore umbilical cord blood-derived stem cells have the capacity to differentiate into a variety of non-blood cell types including hepatocytes neural cells and endothelial cells In addition umbilical cord blood contains a greater proportion of hematopoietic stem cells than bone marrow
In addition cord blood contains a large number of immune cells called regulatory T cells These regulatory T cells may be helpful in diminishing autoimmunity The need to re-establish tolerance in patients with established autoimmunity provides another potential mechanism for cord blood as a therapy for type 1 diabetes
Description of Project
23 children 1 year of age with T1D and stored umbilical cord blood are being be recruited The cord blood will be infused into the children in the GCRC in an attempt to regenerate pancreatic islet insulin-producing beta cells and improve blood glucose control As secondary goals we aim to track the migration of transfused cord blood stem and study the potential changes in metabolismimmune function leading to islet regeneration
Anticipated Outcome
It is hoped that there will be preservation of beta cell function assessed by mixed meal stimulated C-peptide secretion Changes in immunological markersfunction may be observed
Relevance to Type I Diabetes
Type 1 diabetes is still associated with tremendous morbidity and premature mortality Patients require multiple daily insulin injections throughout their lives as well as close monitoring of their diet and blood sugar levels to prevent complications Unfortunately there is presently no permanent cure for diabetes Whole pancreas or islet cell transplantation is available only to a very limited number of patients and necessitates potential lifelong immunosuppressive therapy The need to find a cure for T1D cannot be overstated -autologous stem cell transfusions either with their potential to differentiate into islet cells or provide immune tolerance that leads to islet regeneration appear to be a safe and potentially viable option
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
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 |
|---|---|---|---|
| UF IRB-01 125-2004 | OTHER | UF Institutional Review Board | None |
| GCRC 593 | OTHER | None | None |