Umbilical Cord Blood Infusion to Treat Type 1 Diabetes

NCT ID: NCT00305344

Last Updated: 2022-02-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 23 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2005-04-30
• Study Completion Date: 2011-05-31

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 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cord Blood

Umbilical Cord Blood

Group Type: ACTIVE_COMPARATOR

Autologous Umbilical Cord Blood Transfusion

Intervention Type: PROCEDURE

Cord Blood infusion

Cord blood

Intervention Type: BIOLOGICAL

Develop Cord blood vaccine

Interventions

Autologous Umbilical Cord Blood Transfusion

Cord Blood infusion

Intervention Type: PROCEDURE

Cord blood

Develop Cord blood vaccine

Intervention Type: BIOLOGICAL

Other Intervention Names

Cord Blood; Vaccine

Eligibility Criteria

Inclusion Criteria

1. Must have a diagnosis of T1D and have stored umbilical cord blood in an AABB and/or FACT accredited cord bank.

2. 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.

3. Cord blood meets all selection and testing criteria (see below).

4. Able to complete mixed meal tolerance / glucagon stimulation test.

5. Normal screening values for CBC, Renal function and electrolytes (BMP).

6. Willing to comply with intensive diabetes management

Exclusion Criteria

1. Complicating medical issues that would interfere with blood drawing or monitoring.

2. Chronic use of steroids or other immunosuppressive agents for other conditions.

3. Positive infectious disease markers from mothers' blood or cord at time of collection (See below for details).

4. Any evidence of illness on planned infusion date (i.e. fever >38.5 C, vomiting, diarrhea, wheezing, or crackles).

• Minimum Eligible Age: 1 Year
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Juvenile Diabetes Research Foundation

OTHER

Sponsor Role: collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role: collaborator

University of Florida

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Michael J Haller, MD

Role: PRINCIPAL_INVESTIGATOR

University of Florida

Desmond A Schatz, MD

Role: PRINCIPAL_INVESTIGATOR

University of Florida

Locations

University of Florida

Gainesville, Florida, United States

Countries

United States

References

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.

Reference Type: RESULT

PMID: 22011412 (View on PubMed)

Haller 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.

Reference Type: RESULT

PMID: 19875605 (View on PubMed)

Other Identifiers

GCRC 593

Identifier Type: OTHER

Identifier Source: secondary_id

UF IRB-01 125-2004

Identifier Type: OTHER

Identifier Source: secondary_id

1-2005-362

Identifier Type: -

Identifier Source: org_study_id