Type 1 DiabetesPhase 2NCT02218619
Tauroursodeoxycholic Acid (TUDCA) in New-Onset Type 1 Diabetes
A Phase 2 study testing Tauroursodeoxycholic Acid (TUDCA) in people with Type 1 Diabetes, run by Robin Goland, MD at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Type 1 Diabetes
- What is being tested?
- Tauroursodeoxycholic Acid (TUDCA)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2015to 31 Dec 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Naomi Berrie Diabetes Center, Columbia University, 1150 St. Nicholas Ave.
New York, New York, 10032
About this study
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
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.
Read the detailed description
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.
- Conditions
- Type 1 Diabetes
- Being tested
- Tauroursodeoxycholic Acid (TUDCA), Sugar Pill (placebo)
- Keywords
- type 1 diabetes (T1D), endoplasmic reticulum (ER) stress, induced pluripotential stem (iPS) cells, Tauroursodeoxycholic Acid (TUDCA)
- Medical subject headings
- Diabetes Mellitus, Type 1, Ichthyosis prematurity syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tauroursodeoxycholic Acid (TUDCA)Drug
Also called TUDCA, Taurolite
TUDCA at 1750 mg/day x 12 months
Sugar Pill (placebo)Drug
Also called Placebo
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Taurourodeoxycholic Acid (TUDCA) | Experimental | TUDCA 1750 mg/day x 12 months Receives: Drug: Tauroursodeoxycholic Acid (TUDCA) |
| Sugar pill (placebo) | Placebo comparator | Placebo at same dose, frequency, and duration as experimental treatment Receives: Drug: Sugar Pill (placebo) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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
Measured: Baseline and 18 months
Secondary outcomes (7)
Number of Participants With Liver Function Test Abnormalities
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.
Measured: 18 months
Change in Insulin Use at 6 Months
Change in insulin use from baseline at 6 months
Measured: Baseline and 6 months
Change in Insulin Use at 12 Months
Change in insulin use from baseline at 12 months
Measured: Baseline and 12 months
Change in Insulin Use at 18 Months
Change in insulin use from baseline at 18 months
Measured: Baseline and 18 months
Change in HbA1c at 6 Months
Change in HbA1c from baseline at 6 months
Measured: Baseline and 6 months
Change in HbA1c at 12 Months
Change in HbA1c from baseline at 12 months
Measured: Baseline and 12 months
Change in HbA1c at 18 Months
Change in HbA1c from baseline at 18 months
Measured: Baseline and 18 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Aug 2014 (estimated)
- Study startAug 2015
- Primary completion31 Dec 2019
- Study completion31 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Type 1 DiabetesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Robin Goland, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Juvenile Diabetes Research Foundation
- Responsible party
- Sponsor-investigator, Robin Goland, MD, Columbia University
- Investigators
- Robin Goland, MD, Principal investigator, Columbia UniversityRudolph Leibel, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02218619
- Sponsor's protocol ID
- AAAN2402
- Status last verified
- May 2025
- First submitted
- 13 Aug 2014
- Last updated
- 5 Jun 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: primary data to be shared
Publications
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.
PubMed 34547326
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02218619
- Acronym / sponsor ID
- AAAN2402
- Conditions
- Type 1 Diabetes
- Keywords
- type 1 diabetes (T1D), endoplasmic reticulum (ER) stress, induced pluripotential stem (iPS) cells, Tauroursodeoxycholic Acid (TUDCA)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Robin Goland, MDUniversity, hospital or other
- Collaborators
- Juvenile Diabetes Research Foundation
- Study start
- Aug 2015 (actual)
- Primary completion
- 31 Dec 2019 (actual)
- Study completion
- 31 Dec 2019 (actual)
- First posted
- 18 Aug 2014 (estimated)
- Results
- Posted 5 Jun 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Taurourodeoxycholic Acid (TUDCA) | Experimental | Drug: Tauroursodeoxycholic Acid (TUDCA)TUDCA 1750 mg/day x 12 months |
| Sugar pill (placebo) | Placebo comparator | Drug: Sugar Pill (placebo)Placebo at same dose, frequency, and duration as experimental treatment |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Tauroursodeoxycholic Acid (TUDCA)Also: TUDCA, TauroliteTUDCA at 1750 mg/day x 12 months | Drug | Taurourodeoxycholic Acid (TUDCA) |
| Sugar Pill (placebo)Also: PlaceboPlacebo | Drug | Sugar pill (placebo) |
Primary outcome
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.
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.
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
7 secondary outcomes
Number of Participants With Liver Function Test Abnormalities
Time frame: 18 months
Change in Insulin Use at 6 Months
Time frame: Baseline and 6 months
Change in Insulin Use at 12 Months
Time frame: Baseline and 12 months
Change in Insulin Use at 18 Months
Time frame: Baseline and 18 months
Change in HbA1c at 6 Months
Time frame: Baseline and 6 months
Change in HbA1c at 12 Months
Time frame: Baseline and 12 months
Change in HbA1c at 18 Months
Time frame: Baseline and 18 Months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34547326Rosa 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.
- Individual participant dataSharing planned: Yes. primary data to be shared
Related trials for Type 1 Diabetes
Not yet recruitingPhase 3
A Study to Evaluate the Effectiveness and Safety of the Drug AZD6234 for Managing Weight in Adults Who Are Obese or Overweight. These Participants May or May Not Also Have Type 2 Diabetes
Obesity, Overweight
RecruitingPhase 1
Pharmacokinetics, Safety, and Immunogenicity Comparison of Bmab1700 and Opdivo® as Adjuvant Monotherapy in Participants With Melanoma
Melanoma
Active, not recruitingPhase 2
A Study of Botensilimab and Balstilimab for the Treatment of Colorectal Cancer
Metastatic Colorectal Cancer
Not yet recruiting
Inter-Arm Differences in Continuous Glucose Monitor Readings in Adults With Diabetes
Continuous Glucose Monitoring, Type 1 Diabetes
RecruitingObservational study
Estimated Risk of Preeclampsia in the First Trimester and Its Association With Subclinical Atherosclerosis and Cardiovascular Risk in Women With Type 1 Diabetes
Type 1 Diabetes, Preeclampsia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robin Goland, MD principal investigator
Columbia University
Rudolph Leibel, MD principal investigator
Columbia University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Tauroursodeoxycholic Acid (TUDCA) in people with Type 1 Diabetes, run by Robin Goland, MD at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Jun 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in C-peptide Measurement as Reflection of Insulin Secretion at 6 Months (time frame: Baseline and 6 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02218619, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jun 2025.