Cord Blood Infusion for Type 1 Diabetes Mellitus (T1DM)
Primary Purpose
Type 1 Diabetes, Children
Status
Unknown status
Phase
Phase 1
Locations
Germany
Study Type
Interventional
Intervention
Umbilical Cord Blood VITA 34
Sponsored by
About this trial
This is an interventional treatment trial for Type 1 Diabetes
Eligibility Criteria
Inclusion Criteria:
- Must have a diagnosis of T1D and have stored umbilical cord blood (10 patients sought) at the cord bank Vita 34.
- 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).
- Normal screening values for CBC, Renal function and electrolytes (BMP).
- Willing to comply with intensive diabetes management
- Not younger than 1 year of age
Exclusion Criteria:
- Have complicating medical issues that would interfere with blood drawing or monitoring.
- Require chronic use of steroids or other immunosuppressive agents for other conditions.
- Cord Blood with viability < 50%.
- 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).
Sites / Locations
- Forschergruppe Diabetes der Technischen Universität
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
No Intervention
Arm Label
A
B
Arm Description
Outcomes
Primary Outcome Measures
insulin production
change in median area under the curve (AUC) for C-peptide (measure of insulin production) from baseline to 2 years during a 2h Mixed Meal Tolerance Test was used as the primary outcome measure and was reported in ng/mL/120 minutes.
Secondary Outcome Measures
Insulin Dose, Autoantibody levels, T-cell functional response assays, Cytokine levels
Insulin Dose, Autoantibody levels, T-cell functional response assays, Cytokine levels
Full Information
NCT ID
NCT00989547
First Posted
October 2, 2009
Last Updated
July 22, 2013
Sponsor
Technical University of Munich
1. Study Identification
Unique Protocol Identification Number
NCT00989547
Brief Title
Cord Blood Infusion for Type 1 Diabetes Mellitus (T1DM)
Official Title
Transfusion of Autologous Umbilical Cord Blood to Reverse Hyperglycemia in Children With Type 1 Diabetes - A Pilot Study.
Study Type
Interventional
2. Study Status
Record Verification Date
July 2013
Overall Recruitment Status
Unknown status
Study Start Date
September 2008 (undefined)
Primary Completion Date
September 2014 (Anticipated)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Technical University of Munich
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Type 1 diabetes (T1D) 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. Autologous stem cell transplants have been used successfully for ALL (acute lymphoblastic leukemia), AML (acute myeloblastic leukemia) and for the treatment of a variety of cancers including breast cancer and neuroblastomas, and more recently for the treatment of autoimmune disorders such as multiple sclerosis (MS), lupus-like disease, and rheumatic disorders. Recently it was shown that bone marrow-derived stems cells transplanted into diabetic mice led to reduced hyperglycemia within 7 days after transplantation and was sustained until they were sacrificed at 35 days post-transplantation. The investigators' goal is to transfuse autologous umbilical cord blood into 23 children (Germany 10 and 20 Controls) with T1D in an attempt to regenerate pancreatic islet insulin producing beta cells and improve blood glucose control. As secondary goals, the investigators aim to track the migration of transfused cord blood stem and study the potential changes in metabolism/immune function leading to islet regeneration.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Type 1 Diabetes, Children
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
18 (Actual)
8. Arms, Groups, and Interventions
Arm Title
A
Arm Type
Active Comparator
Arm Title
B
Arm Type
No Intervention
Intervention Type
Other
Intervention Name(s)
Umbilical Cord Blood VITA 34
Intervention Description
Intervention type: Autologous Umbilical Cord Blood Transfusion
Primary Outcome Measure Information:
Title
insulin production
Description
change in median area under the curve (AUC) for C-peptide (measure of insulin production) from baseline to 2 years during a 2h Mixed Meal Tolerance Test was used as the primary outcome measure and was reported in ng/mL/120 minutes.
Secondary Outcome Measure Information:
Title
Insulin Dose, Autoantibody levels, T-cell functional response assays, Cytokine levels
Description
Insulin Dose, Autoantibody levels, T-cell functional response assays, Cytokine levels
Other Pre-specified Outcome Measures:
Title
glycated hemoglobin (HbA1c)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Eligibility Criteria
Inclusion Criteria:
Must have a diagnosis of T1D and have stored umbilical cord blood (10 patients sought) at the cord bank Vita 34.
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).
Normal screening values for CBC, Renal function and electrolytes (BMP).
Willing to comply with intensive diabetes management
Not younger than 1 year of age
Exclusion Criteria:
Have complicating medical issues that would interfere with blood drawing or monitoring.
Require chronic use of steroids or other immunosuppressive agents for other conditions.
Cord Blood with viability < 50%.
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).
Facility Information:
Facility Name
Forschergruppe Diabetes der Technischen Universität
City
München
State/Province
Bavaria
ZIP/Postal Code
80804
Country
Germany
12. IPD Sharing Statement
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Cord Blood Infusion for Type 1 Diabetes Mellitus (T1DM)
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