Tauroursodeoxycholic Acid (TUDCA) in New-Onset Type 1 Diabetes
| Status: | Active, not recruiting |
|---|---|
| Conditions: | Diabetes, Diabetes |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | 18 - 45 |
| Updated: | 12/6/2018 |
| Start Date: | August 2015 |
| End Date: | October 1, 2019 |
Clinical Investigation of Efficacy of Tauroursodeoxycholic Acid (TUDCA) to Enhance Pancreatic Beta Cell Survival In Type 1 Diabetes by Reducing Endoplasmic Reticulum Stress
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 we 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.
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 we 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.
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.
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.
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
We found this trial at
1
site
New York, New York 10032
Principal Investigator: Robin Goland, MD
Phone: 212-851-5425
Click here to add this to my saved trials
