Cord Blood Infusion for Type 1 Diabetes Mellitus (T1DM)

Type 1 Diabetes Clinical Trial

Official title:

Transfusion of Autologous Umbilical Cord Blood to Reverse Hyperglycemia in Children With Type 1 Diabetes - A Pilot Study.

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT00989547
• Other study ID #: 593
• Status: Active, not recruiting
• Phase: Phase 1
• First received: October 2, 2009
• Last updated: July 22, 2013
• Start date: September 2008

Study information

• Verified date: July 2013
• Source: Technische Universität München
• Contact: n/a
• Is FDA regulated: No
• Health authority: Germany: Paul-Ehrlich-Institut
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 18
• Est. primary completion date: September 2014
• Gender: Both
• Age group: 1 Year and older

Eligibility

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).

Study Design

Allocation: Non-Randomized, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Children
• Diabetes Mellitus, Type 1
• Type 1 Diabetes

Intervention

Other:
• Umbilical Cord Blood VITA 34
Intervention type: Autologous Umbilical Cord Blood Transfusion

Locations

Country	Name	City	State
Germany	Forschergruppe Diabetes der Technischen Universität	München	Bavaria

Sponsors (1)

Lead Sponsor	Collaborator
Technische Universität München

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	glycated hemoglobin (HbA1c)			No
Primary	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.		No
Secondary	Insulin Dose, Autoantibody levels, T-cell functional response assays, Cytokine levels	Insulin Dose, Autoantibody levels, T-cell functional response assays, Cytokine levels		No