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Completed

NCT Number: NCT02218619

Tauroursodeoxycholic Acid (TUDCA) in New-Onset Type 1 Diabetes

Clinically, the ability to slow or prevent beta cell demise can prevent or improve the course of type 1 diabetes. The immune-mediated destruction of beta cells that is an apparent major pathological basis for the disease, has led to efforts to prevent or suppress this immune assault. Here the investigators propose to buttress the beta cell's capacity to withstand this assault by improving the function of the endoplasmic reticulum stress resolving mechanisms within these cells. The ability to do so could have a major impact on preventive and therapeutic strategies for type 1 diabetes (and possibly other types of diabetes). The type of endoplasmic reticulum stress relieving agent (TUDCA) proposed here could ultimately be applied on an anticipatory basis to individuals at high risk for type 1 diabetes.

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Key information

Conditions

Type 1 Diabetes Autoimmune Diseases Diabetes Mellitus Diabetes Mellitus, Type 1 Endocrine System Diseases Glucose Metabolism Disorders Ichthyosis prematurity syndrome Immune System Diseases Metabolic Diseases Nutritional and Metabolic Diseases

Age range

18 year–45 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Naomi Berrie Diabetes Center, Columbia University, 1150 St. Nicholas Ave.

New York, 10032, United States

About this study

Reducing endoplasmic reticulum stress will promote beta cell survival in new-onset type 1 diabetes.

The primary aim is to test the clinical efficacy of an already approved agent, TUDCA, re-purposed to reduce endoplasmic reticulum stress and improve beta cell survival in patients with new onset type 1 diabetes. The primary endpoint of this proposed double-blinded randomized placebo-controlled pilot study is c-peptide measured after mixed meal stimulation test at randomization and then at 6 and 12 months of treatment with TUDCA compared to treatment with placebo and at 6 months following treatment.

TUDCA is an oral medication with a safety profile that is approved for use in Europe for gall stones and liver disease. The drug and similar compounds has been used in children, as young as newborns, and in adults. TUDCA's ability to lower endoplasmic reticulum stress has only recently been recognized and will be applied to new-onset type 1 diabetes in this proposal. If this pilot trial is successful, future studies could include broadening the recipients to antibody-positive pre-type 1 diabetes patients and/or combining TUDCA with other agents shown to have a beneficial effect on insulin secretion in new-onset type 1 diabetes.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Type 1 diabetes according to American Diabetes Association criteria
  • Diagnosis of type 1 diabetes within 100 days of randomization
  • One positive diabetes-related autoantibody
  • Ages 18-45 years

Exclusion criteria

  • Drugs known to affect glucose other than insulin
  • Stimulated C-peptide levels < 0.2 pmol/ml measured during a mixed meal tolerance test conducted at least 21 days from diagnosis of diabetes and within one month (37 days) of randomization to either TUDCA or placebo.
  • Women during pregnancy

Treatment and study plan

tauroursodeoxycholic acid (TUDCA)

Drug

TUDCA at 1750 mg/day x 12 months

Other names: TUDCA, Taurolite

Sugar Pill (placebo)

Drug

Placebo

Other names: Placebo

Primary outcomes

  1. Change in C-peptide Measurement as Reflection of Insulin Secretion at 6 Months

    Time frame: Baseline and 6 months

    The primary endpoint will be the change from baseline in area under the stimulated C-peptide curve over the first 2 hours of a 4- hour mixed meal tolerance test conducted at 6 months.

  2. Change in C-peptide Measurement as Reflection of Insulin Secretion at 12 Months

    Time frame: Baseline and 12 months

    The primary endpoint will be the change from baseline in area under the stimulated C-peptide curve over the first 2 hours of a 4- hour mixed meal tolerance test conducted at 12 months.

  3. Change in C-peptide Measurement as Reflection of Insulin Secretion at 18 Months

    Time frame: Baseline and 18 months

    The primary endpoint will be the change from baseline in area under the stimulated C-peptide curve over the first 2 hours of a 4- hour mixed meal tolerance test conducted at 18 months

Secondary outcomes

  1. Number of Participants With Liver Function Test Abnormalities

    Time frame: 18 months

    Measure liver function tests at 6 and 12 months and at 6 months after drug or placebo is stopped to ensure that no abnormalities (liver function blood tests outside of normal reference range) of liver function occur with the drug.

  2. Change in Insulin Use at 6 Months

    Time frame: Baseline and 6 months

    Change in insulin use from baseline at 6 months

  3. Change in Insulin Use at 12 Months

    Time frame: Baseline and 12 months

    Change in insulin use from baseline at 12 months

  4. Change in Insulin Use at 18 Months

    Time frame: Baseline and 18 months

    Change in insulin use from baseline at 18 months

  5. Change in HbA1c at 6 Months

    Time frame: Baseline and 6 months

    Change in HbA1c from baseline at 6 months

  6. Change in HbA1c at 12 Months

    Time frame: Baseline and 12 months

    Change in HbA1c from baseline at 12 months

  7. Change in HbA1c at 18 Months

    Time frame: Baseline and 18 Months

    Change in HbA1c from baseline at 18 months

Other outcomes

  1. Endoplasmic Reticulum Stress

    Time frame: 1 week

    It is believed that the autoimmune assault of new onset type 1 diabetes leads to stress to the part of the beta cell that folds proteins; referred to as endoplasmic reticulum stress. When endoplasmic reticulum stress increases, changes in protein levels in beta cells occur. The investigators will measure markers of endoplasmic reticulum stress in beta cells taken from skin biopsies from subjects before treatment with TUDCA or placebo.

Sponsors and collaborators

Lead sponsor

Robin Goland, MD

Other

Collaborators

  • Juvenile Diabetes Research Foundation

Registry information

Official study title

Clinical Investigation of Efficacy of Tauroursodeoxycholic Acid (TUDCA) to Enhance Pancreatic Beta Cell Survival In Type 1 Diabetes by Reducing Endoplasmic Reticulum Stress

Important dates

Study start
2015
Primary completion
2019
Study completion
2019
First posted
Aug 18, 2014
Registry last updated
Jun 5, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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