Trial record 2 of 2 for:    cord blood diabetes university of florida

Umbilical Cord Blood Infusion to Treat Type 1 Diabetes

This study has been completed.
Sponsor:
Collaborators:
Juvenile Diabetes Research Foundation
Information provided by (Responsible Party):
University of Florida
ClinicalTrials.gov Identifier:
NCT00305344
First received: March 17, 2006
Last updated: July 3, 2012
Last verified: July 2012
  Purpose

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.


Condition Intervention Phase
Type 1 Diabetes Mellitus
Procedure: Autologous Umbilical Cord Blood Transfusion
Biological: Cord blood
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Transfusion of Autologous Umbilical Cord Blood to Reverse Hyperglycemia in Children With Type 1 Diabetes - A Pilot Study

Resource links provided by NLM:


Further study details as provided by University of Florida:

Primary Outcome Measures:
  • Children With T1D Underwent a Single Autologous UCB Transfusion [ Time Frame: Baseline to Year 2 ] [ Designated as safety issue: No ]
    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 insuln 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


Enrollment: 23
Study Start Date: April 2005
Study Completion Date: May 2011
Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Cord Blood
Umbilical Cord Blood
Procedure: Autologous Umbilical Cord Blood Transfusion
Cord Blood infusion
Other Name: Cord Blood
Biological: Cord blood
Develop Cord blood vaccine
Other Name: Vaccine

  Hide Detailed Description

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.

  Eligibility

Ages Eligible for Study:   1 Year and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00305344

Locations
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
Sponsors and Collaborators
University of Florida
Juvenile Diabetes Research Foundation
Investigators
Principal Investigator: Michael J Haller, MD University of Florida
Principal Investigator: Desmond A Schatz, MD University of Florida
  More Information

Publications:
Responsible Party: University of Florida
ClinicalTrials.gov Identifier: NCT00305344     History of Changes
Other Study ID Numbers: 1-2005-362, GCRC 593, UF IRB-01 125-2004
Study First Received: March 17, 2006
Results First Received: March 2, 2012
Last Updated: July 3, 2012
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board
University of Florida: Pediatric Data Safety Monitoring Board

Keywords provided by University of Florida:
Type 1 Diabetes Mellitus
Umbilical Cord
Regeneration
Insulin
Islets of Langerhans

Additional relevant MeSH terms:
Diabetes Mellitus
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases

ClinicalTrials.gov processed this record on September 18, 2014