Tauroursodeoxycholic Acid (TUDCA) in New-Onset Type 1 Diabetes
|ClinicalTrials.gov Identifier: NCT02218619|
Recruitment Status : Active, not recruiting
First Posted : August 18, 2014
Last Update Posted : December 4, 2018
|Condition or disease||Intervention/treatment||Phase|
|Type 1 Diabetes||Drug: Tauroursodeoxycholic Acid (TUDCA) Drug: Sugar Pill (placebo)||Phase 2|
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 an excellent 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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||20 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||Clinical Investigation of Efficacy of Tauroursodeoxycholic Acid (TUDCA) to Enhance Pancreatic Beta Cell Survival In Type 1 Diabetes by Reducing Endoplasmic Reticulum Stress|
|Study Start Date :||August 2015|
|Estimated Primary Completion Date :||October 1, 2019|
|Estimated Study Completion Date :||October 1, 2019|
Experimental: Taurourodeoxycholic Acid (TUDCA)
TUDCA 1750 mg/day x 12 months
Drug: Tauroursodeoxycholic Acid (TUDCA)
TUDCA at 1750 mg/day x 12 months
Placebo Comparator: Sugar pill (placebo)
Placebo at same dose, frequency, and duration as experimental treatment
Drug: Sugar Pill (placebo)
Other Name: Placebo
- C-peptide measurement as reflection of insulin secretion [ Time Frame: 18 months ]The primary endpoint will be the area under the stimulated C-peptide curve over the first 2 hours of a 4- hour mixed meal tolerance test conducted at screening, and during the 12 months of drug treatment at 6 and12 months and at 6 months after drug or placebo is stopped.
- 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. We will measure markers of endoplasmic reticulum stress in beta cells taken from skin biopsies taken from subjects before treatment with TUDCA or placebo.
- liver function tests [ Time Frame: 18 months ]We will measure liver function tests at 6 and 12 months and at 6 months after drug or placebo is stopped to ensure that no abnormalities of liver function occur with the drug.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02218619
|United States, New York|
|Naomi Berrie Diabetes Center, Columbia University, 1150 St. Nicholas Ave.|
|New York, New York, United States, 10032|
|Principal Investigator:||Robin Goland, MD||Columbia University|
|Principal Investigator:||Rudolph Leibel, MD||Columbia University|