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

NCT02218619 | Completed Phase 2 Results DMC

• 1 other identifier: AAAN2402
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Type 1 Diabetes

Keywords

type 1 diabetes (T1D); endoplasmic reticulum (ER) stress; induced pluripotential stem (iPS) cells; Tauroursodeoxycholic Acid (TUDCA)

Outcome Measures

Primary Outcomes (3)

• Change in C-peptide Measurement as Reflection of Insulin Secretion at 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.

  Baseline and 6 months

• Change in C-peptide Measurement as Reflection of Insulin Secretion at 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.

  Baseline and 12 months

• Change in C-peptide Measurement as Reflection of Insulin Secretion at 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

  Baseline and 18 months

Secondary Outcomes (7)

• Number of Participants With Liver Function Test Abnormalities

  18 months

• Change in Insulin Use at 6 Months

  Baseline and 6 months

• Change in Insulin Use at 12 Months

  Baseline and 12 months

• Change in Insulin Use at 18 Months

  Baseline and 18 months

• Change in HbA1c at 6 Months

  Baseline and 6 months

Other Outcomes (1)

• Endoplasmic Reticulum Stress

  1 week

Study Arms (2)

Taurourodeoxycholic Acid (TUDCA)

EXPERIMENTAL

TUDCA 1750 mg/day x 12 months

Drug: Tauroursodeoxycholic Acid (TUDCA)

Sugar pill (placebo)

PLACEBO COMPARATOR

Placebo at same dose, frequency, and duration as experimental treatment

Drug: Sugar Pill (placebo)

Interventions

Tauroursodeoxycholic Acid (TUDCA) DRUG

TUDCA at 1750 mg/day x 12 months

Also known as: TUDCA, Taurolite

Taurourodeoxycholic Acid (TUDCA)

Sugar Pill (placebo) DRUG

Placebo

Also known as: Placebo

Sugar pill (placebo)

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Robin Goland, MD: lead
• Juvenile Diabetes Research Foundation: collaborator

Study Sites (1)

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

New York, New York, 10032, United States

Location

Related Publications (1)

• Rosa LRO, Vettorazzi JF, Zangerolamo L, Carneiro EM, Barbosa HCL. TUDCA receptors and their role on pancreatic beta cells. Prog Biophys Mol Biol. 2021 Dec;167:26-31. doi: 10.1016/j.pbiomolbio.2021.09.003. Epub 2021 Sep 20.

  PMID: 34547326 DERIVED

MeSH Terms

Conditions

Diabetes Mellitus, Type 1; Ichthyosis prematurity syndrome

Interventions

ursodoxicoltaurine; Sugars

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Carbohydrates

Results Point of Contact

• Title: Robin Goland, MD
• Organization: Columbia University Irving Medical Center

rsg2@cumc.columbia.edu

212-851-5494

Study Officials

• Robin Goland, MD

  Columbia University

  PRINCIPAL INVESTIGATOR

• Rudolph Leibel, MD

  Columbia University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: J. Merrill Eastman Professor of Clinical Diabetes, Co-Director, Berrie Center

Study Record Dates

• First Submitted: August 13, 2014
• First Posted: August 18, 2014
• Study Start: August 1, 2015
• Primary Completion: December 31, 2019
• Study Completion: December 31, 2019
• Last Updated: June 5, 2025
• Results First Posted: June 5, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: 2021
• Access Criteria: email rsg2@columbia.edu

Locations

US (1)