Type 1 Diabetes MellitusPhase 1/2NCT00305344
Umbilical Cord Blood Infusion to Treat Type 1 Diabetes
A Phase 1/2 study testing Autologous Umbilical Cord Blood Transfusion and Cord blood in people with Type 1 Diabetes Mellitus, run by University of Florida at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Type 1 Diabetes Mellitus
- What is being tested?
- Autologous Umbilical Cord Blood Transfusion and Cord blood
- Type of treatment
- Vaccines, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2005to May 2011
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Must have a diagnosis of T1D and have stored umbilical cord blood in an AABB and/or FACT accredited cord bank.
- TID diagnosis will be defined as having a clear history of polydipsia, polyphagia, polyuria, and weight loss consistent with a clinical diagnosis, diagnosis will mot be based solely upon the presence of autoantibodies.
- Cord blood meets all selection and testing criteria (see below).
- Able to complete mixed meal tolerance / glucagon stimulation test.
- Normal screening values for CBC, Renal function and electrolytes (BMP).
- Willing to comply with intensive diabetes management
Who cannot join
- Complicating medical issues that would interfere with blood drawing or monitoring.
- Chronic use of steroids or other immunosuppressive agents for other conditions.
- Positive infectious disease markers from mothers' blood or cord at time of collection (See below for details).
- Any evidence of illness on planned infusion date (i.e. fever >38.5 C, vomiting, diarrhea, wheezing, or crackles).
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Transfusion of Autologous Umbilical Cord Blood to Reverse Hyperglycemia in Children With Type 1 Diabetes - A Pilot Study
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.
Read the 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 metabolism/immune function leading to islet regeneration.
Background/Rationale:
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 metabolism/immune 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 markers/function 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.
- Conditions
- Type 1 Diabetes Mellitus
- Being tested
- Autologous Umbilical Cord Blood Transfusion, Cord blood
- Keywords
- Type 1 Diabetes Mellitus, Umbilical Cord, Regeneration, Insulin, Islets of Langerhans
- Medical subject headings
- Diabetes Mellitus, Type 1, Insulin Resistance
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Umbilical Cord Blood TransfusionProcedure or surgery
Also called Cord Blood
Cord Blood infusion
Cord bloodBiological
Also called Vaccine
Develop Cord blood vaccine
| Study group | Role | What they receive |
|---|---|---|
| Cord Blood | Active comparator | Umbilical Cord Blood Receives: Procedure: Autologous Umbilical Cord Blood Transfusion; Biological: Cord blood |
What is being measured
The primary outcome is the main question the study is designed to answer.
Children With T1D Underwent a Single Autologous UCB Transfusion
All participants were monitored for 2 years. Baseline and post-infusion mixed meal tolerance tests were performed to determine whether autologous cord blood infusion preserved endogenous insulin production. The change in median area under the curve for C-peptide (measure of insulin production) from baseline to to 2 years during a 2 hour mixed meal tolerance test was used as the primary outcome measure and was reported in ng/ml/120 minutes
Measured: Baseline to Year 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2006 (estimated)
- Study startApr 2005
- Primary completionMay 2011
- Study completionMay 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Type 1 Diabetes MellitusStudies of the same condition, open and under way.
- More trials run by University of FloridaThe 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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Juvenile Diabetes Research Foundation, National Institutes of Health (NIH)
- Responsible party
- Sponsor
- Investigators
- Michael J Haller, MD, Principal investigator, University of FloridaDesmond A Schatz, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00305344
- Sponsor's protocol ID
- 1-2005-362
- Other IDs
- GCRC 593, UF IRB-01 125-2004
- Status last verified
- Feb 2022
- First submitted
- 17 Mar 2006
- Last updated
- 9 Feb 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Haller MJ, Wasserfall CH, Hulme MA, Cintron M, Brusko TM, McGrail KM, Sumrall TM, Wingard JR, Theriaque DW, Shuster JJ, Atkinson MA, Schatz DA. Autologous umbilical cord blood transfusion in young children with type 1 diabetes fails to preserve C-peptide. Diabetes Care. 2011 Dec;34(12):2567-9. doi: 10.2337/dc11-1406. Epub 2011 Oct 19.
PubMed 22011412Haller MJ, Wasserfall CH, McGrail KM, Cintron M, Brusko TM, Wingard JR, Kelly SS, Shuster JJ, Atkinson MA, Schatz DA. Autologous umbilical cord blood transfusion in very young children with type 1 diabetes. Diabetes Care. 2009 Nov;32(11):2041-6. doi: 10.2337/dc09-0967.
PubMed 19875605
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Transfusion of Autologous Umbilical Cord Blood to Reverse Hyperglycemia in Children With Type 1 Diabetes - A Pilot Study
- ClinicalTrials.gov ID
- NCT00305344
- Acronym / sponsor ID
- 1-2005-362
- Conditions
- Type 1 Diabetes Mellitus
- Keywords
- Type 1 Diabetes Mellitus, Umbilical Cord, Regeneration, Insulin, Islets of Langerhans
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- Juvenile Diabetes Research Foundation, National Institutes of Health (NIH)
- Study start
- Apr 2005
- Primary completion
- May 2011 (actual)
- Study completion
- May 2011 (actual)
- First posted
- 21 Mar 2006 (estimated)
- Results
- Posted 10 Jul 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 metabolism/immune function leading to islet regeneration.
Background/Rationale:
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 metabolism/immune 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 markers/function 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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cord Blood | Active comparator | Procedure: Autologous Umbilical Cord Blood Transfusion, Biological: Cord bloodUmbilical Cord Blood |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Must have a diagnosis of T1D and have stored umbilical cord blood in an AABB and/or FACT accredited cord bank.
- TID diagnosis will be defined as having a clear history of polydipsia, polyphagia, polyuria, and weight loss consistent with a clinical diagnosis, diagnosis will mot be based solely upon the presence of autoantibodies.
- Cord blood meets all selection and testing criteria (see below).
- Able to complete mixed meal tolerance / glucagon stimulation test.
- Normal screening values for CBC, Renal function and electrolytes (BMP).
Key exclusion criteria (4 listed)
- Complicating medical issues that would interfere with blood drawing or monitoring.
- Chronic use of steroids or other immunosuppressive agents for other conditions.
- Positive infectious disease markers from mothers' blood or cord at time of collection (See below for details).
- Any evidence of illness on planned infusion date (i.e. fever >38.5 C, vomiting, diarrhea, wheezing, or crackles).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous Umbilical Cord Blood TransfusionAlso: Cord BloodCord Blood infusion | Procedure or surgery | Cord Blood |
| Cord bloodAlso: VaccineDevelop Cord blood vaccine | Biological | Cord Blood |
Primary outcome
Children With T1D Underwent a Single Autologous UCB Transfusion
Time frame: Baseline to Year 2
All participants were monitored for 2 years. Baseline and post-infusion mixed meal tolerance tests were performed to determine whether autologous cord blood infusion preserved endogenous insulin production. The change in median area under the curve for C-peptide (measure of insulin production) from baseline to to 2 years during a 2 hour mixed meal tolerance test was used as the primary outcome measure and was reported in ng/ml/120 minutes
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 22011412Haller MJ, Wasserfall CH, Hulme MA, Cintron M, Brusko TM, McGrail KM, Sumrall TM, Wingard JR, Theriaque DW, Shuster JJ, Atkinson MA, Schatz DA. Autologous umbilical cord blood transfusion in young children with type 1 diabetes fails to preserve C-peptide. Diabetes Care. 2011 Dec;34(12):2567-9. doi: 10.2337/dc11-1406. Epub 2011 Oct 19.
- Results publication: PubMed 19875605Haller MJ, Wasserfall CH, McGrail KM, Cintron M, Brusko TM, Wingard JR, Kelly SS, Shuster JJ, Atkinson MA, Schatz DA. Autologous umbilical cord blood transfusion in very young children with type 1 diabetes. Diabetes Care. 2009 Nov;32(11):2041-6. doi: 10.2337/dc09-0967.
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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.
Michael J Haller, MD principal investigator
University of Florida
Desmond A Schatz, MD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Autologous Umbilical Cord Blood Transfusion and Cord blood in people with Type 1 Diabetes Mellitus, run by University of Florida 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 6 inclusion and 4 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 Feb 2022. 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Jul 2012). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Children With T1D Underwent a Single Autologous UCB Transfusion (time frame: Baseline to Year 2). 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 NCT00305344, which also lists 2 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 Feb 2022.