Cord Blood Infusion for Type 1 Diabetes Mellitus (T1DM)

NCT00989547 | Unknown Phase 1 DMC

• 1 other identifier: 593
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

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.

Conditions

Type 1 Diabetes; Children

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Insulin Dose, Autoantibody levels, T-cell functional response assays, Cytokine levels

Other Outcomes (1)

• glycated hemoglobin (HbA1c)

Study Arms (2)

A

ACTIVE COMPARATOR

Other: Umbilical Cord Blood VITA 34

B

NO INTERVENTION

Interventions

Umbilical Cord Blood VITA 34 OTHER

Intervention type: Autologous Umbilical Cord Blood Transfusion

A

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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).

Sponsors & Collaborators

• Technical University of Munich: lead

Study Sites (1)

Forschergruppe Diabetes der Technischen Universität

München, Bavaria, 80804, Germany

Location

MeSH Terms

Conditions

Diabetes Mellitus, Type 1

Condition Hierarchy (Ancestors)

Diabetes Mellitus; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 2, 2009
• First Posted: October 5, 2009
• Study Start: September 1, 2008
• Primary Completion: September 1, 2014
• Last Updated: July 23, 2013

Record last verified: 2013-07

Locations

DE (1)