Trial Outcomes & Findings for Tauroursodeoxycholic Acid (TUDCA) in New-Onset Type 1 Diabetes (NCT NCT02218619)
NCT ID: NCT02218619
Last Updated: 2025-06-05
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
Baseline and 6 months
Results posted on
2025-06-05
Participant Flow
Participant milestones
| Measure |
Taurourodeoxycholic Acid (TUDCA)
TUDCA 1750 mg/day x 12 months
Tauroursodeoxycholic Acid (TUDCA): TUDCA at 1750 mg/day x 12 months
|
Sugar Pill (Placebo)
Placebo at same dose, frequency, and duration as experimental treatment
Sugar Pill (placebo): Placebo
|
|---|---|---|
|
Overall Study
STARTED
|
10
|
10
|
|
Overall Study
COMPLETED
|
10
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Tauroursodeoxycholic Acid (TUDCA) in New-Onset Type 1 Diabetes
Baseline characteristics by cohort
| Measure |
Sugar Pill (Placebo)
n=10 Participants
Placebo at same dose, frequency, and duration as experimental treatment
Sugar Pill (placebo): Placebo
|
Taurourodeoxycholic Acid (TUDCA)
n=10 Participants
TUDCA 1750 mg/day x 12 months
Tauroursodeoxycholic Acid (TUDCA): TUDCA at 1750 mg/day x 12 months
|
Total
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
10 Participants
n=39 Participants
|
10 Participants
n=41 Participants
|
20 Participants
n=35 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=39 Participants
|
3 Participants
n=41 Participants
|
7 Participants
n=35 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=39 Participants
|
7 Participants
n=41 Participants
|
13 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
2 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=39 Participants
|
9 Participants
n=41 Participants
|
18 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=39 Participants
|
10 Participants
n=41 Participants
|
20 Participants
n=35 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Region of Enrollment
United States
|
10 participants
n=39 Participants
|
10 participants
n=41 Participants
|
20 participants
n=35 Participants
|
|
Glutamic acid decarboxylase (GAD) Result
GAD Negative
|
0 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
|
Glutamic acid decarboxylase (GAD) Result
GAD Positive
|
10 Participants
n=39 Participants
|
9 Participants
n=41 Participants
|
19 Participants
n=35 Participants
|
|
Islet cell antigen 2 (IA2) Result
IA2 Negative
|
7 Participants
n=39 Participants
|
3 Participants
n=41 Participants
|
10 Participants
n=35 Participants
|
|
Islet cell antigen 2 (IA2) Result
IA2 Positive
|
3 Participants
n=39 Participants
|
7 Participants
n=41 Participants
|
10 Participants
n=35 Participants
|
|
Insulin Autoantibodies (IAA) Result
IAA Negative
|
5 Participants
n=39 Participants
|
5 Participants
n=41 Participants
|
10 Participants
n=35 Participants
|
|
Insulin Autoantibodies (IAA) Result
IAA Positive
|
5 Participants
n=39 Participants
|
5 Participants
n=41 Participants
|
10 Participants
n=35 Participants
|
|
Pancreatic islet-cell antibodies (ICA) Result
ICA Negative
|
4 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
5 Participants
n=35 Participants
|
|
Pancreatic islet-cell antibodies (ICA) Result
ICA Positive
|
6 Participants
n=39 Participants
|
9 Participants
n=41 Participants
|
15 Participants
n=35 Participants
|
|
Zinc Transporter 8 Autoantibody (ZnT8A) Result
ZnT8A Negative
|
4 Participants
n=39 Participants
|
4 Participants
n=41 Participants
|
8 Participants
n=35 Participants
|
|
Zinc Transporter 8 Autoantibody (ZnT8A) Result
ZnT8A Positive
|
6 Participants
n=39 Participants
|
6 Participants
n=41 Participants
|
12 Participants
n=35 Participants
|
|
Positive Autoantibodies
|
3 Positive Autoantibodies
STANDARD_DEVIATION 1.49 • n=39 Participants
|
3.6 Positive Autoantibodies
STANDARD_DEVIATION 1.17 • n=41 Participants
|
3.3 Positive Autoantibodies
STANDARD_DEVIATION 1.34 • n=35 Participants
|
|
Days from Diagnosis to Randomization
|
87.50 Days
STANDARD_DEVIATION 47.44 • n=39 Participants
|
67.70 Days
STANDARD_DEVIATION 21.41 • n=41 Participants
|
77.60 Days
STANDARD_DEVIATION 37.24 • n=35 Participants
|
|
Weight
|
72.50 Kilograms
STANDARD_DEVIATION 10.18 • n=39 Participants
|
69.05 Kilograms
STANDARD_DEVIATION 9.17 • n=41 Participants
|
70.78 Kilograms
STANDARD_DEVIATION 9.59 • n=35 Participants
|
|
Body Mass Index
|
24.91 kg/m^2
STANDARD_DEVIATION 2.85 • n=39 Participants
|
23.26 kg/m^2
STANDARD_DEVIATION 2.86 • n=41 Participants
|
24.09 kg/m^2
STANDARD_DEVIATION 2.91 • n=35 Participants
|
|
Area Under Curve (AUC) for C-Peptide
|
0.53 nmol/L*120 min
STANDARD_DEVIATION 0.20 • n=39 Participants
|
0.54 nmol/L*120 min
STANDARD_DEVIATION 0.27 • n=41 Participants
|
0.53 nmol/L*120 min
STANDARD_DEVIATION 0.23 • n=35 Participants
|
|
Hemoglobin A1C (HbA1C)
|
7.10 Percent
STANDARD_DEVIATION 0.95 • n=39 Participants
|
8.65 Percent
STANDARD_DEVIATION 2.19 • n=41 Participants
|
7.88 Percent
STANDARD_DEVIATION 1.83 • n=35 Participants
|
|
Total Daily Insulin Dose (Average 3 days before baseline visit)
|
0.22 units/kg
STANDARD_DEVIATION 0.12 • n=39 Participants
|
0.40 units/kg
STANDARD_DEVIATION 0.16 • n=41 Participants
|
0.31 units/kg
STANDARD_DEVIATION 0.17 • n=35 Participants
|
PRIMARY outcome
Timeframe: Baseline and 6 monthsThe 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.
Outcome measures
| Measure |
Sugar Pill (Placebo)
n=10 Participants
Placebo at same dose, frequency, and duration as experimental treatment
Sugar Pill (placebo): Placebo
|
Taurourodeoxycholic Acid (TUDCA)
n=10 Participants
TUDCA 1750 mg/day x 12 months
Tauroursodeoxycholic Acid (TUDCA): TUDCA at 1750 mg/day x 12 months
|
|---|---|---|
|
Change in C-peptide Measurement as Reflection of Insulin Secretion at 6 Months
|
-0.07 nmol/L*120 min
Standard Deviation 0.11
|
-0.07 nmol/L*120 min
Standard Deviation 0.10
|
PRIMARY outcome
Timeframe: Baseline and 12 monthsThe 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.
Outcome measures
| Measure |
Sugar Pill (Placebo)
n=10 Participants
Placebo at same dose, frequency, and duration as experimental treatment
Sugar Pill (placebo): Placebo
|
Taurourodeoxycholic Acid (TUDCA)
n=10 Participants
TUDCA 1750 mg/day x 12 months
Tauroursodeoxycholic Acid (TUDCA): TUDCA at 1750 mg/day x 12 months
|
|---|---|---|
|
Change in C-peptide Measurement as Reflection of Insulin Secretion at 12 Months
|
-0.10 nmol/L*120 min
Standard Deviation 0.18
|
-0.12 nmol/L*120 min
Standard Deviation 0.17
|
PRIMARY outcome
Timeframe: Baseline and 18 monthsThe 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
Outcome measures
| Measure |
Sugar Pill (Placebo)
n=10 Participants
Placebo at same dose, frequency, and duration as experimental treatment
Sugar Pill (placebo): Placebo
|
Taurourodeoxycholic Acid (TUDCA)
n=10 Participants
TUDCA 1750 mg/day x 12 months
Tauroursodeoxycholic Acid (TUDCA): TUDCA at 1750 mg/day x 12 months
|
|---|---|---|
|
Change in C-peptide Measurement as Reflection of Insulin Secretion at 18 Months
|
-0.11 nmol/L*120 min
Standard Deviation 0.21
|
-0.14 nmol/L*120 min
Standard Deviation 0.16
|
SECONDARY outcome
Timeframe: 18 monthsMeasure 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.
Outcome measures
| Measure |
Sugar Pill (Placebo)
n=10 Participants
Placebo at same dose, frequency, and duration as experimental treatment
Sugar Pill (placebo): Placebo
|
Taurourodeoxycholic Acid (TUDCA)
n=10 Participants
TUDCA 1750 mg/day x 12 months
Tauroursodeoxycholic Acid (TUDCA): TUDCA at 1750 mg/day x 12 months
|
|---|---|---|
|
Number of Participants With Liver Function Test Abnormalities
Month 6
|
0 Participants
|
0 Participants
|
|
Number of Participants With Liver Function Test Abnormalities
Month 12
|
0 Participants
|
0 Participants
|
|
Number of Participants With Liver Function Test Abnormalities
Month 18
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsChange in insulin use from baseline at 6 months
Outcome measures
| Measure |
Sugar Pill (Placebo)
n=10 Participants
Placebo at same dose, frequency, and duration as experimental treatment
Sugar Pill (placebo): Placebo
|
Taurourodeoxycholic Acid (TUDCA)
n=10 Participants
TUDCA 1750 mg/day x 12 months
Tauroursodeoxycholic Acid (TUDCA): TUDCA at 1750 mg/day x 12 months
|
|---|---|---|
|
Change in Insulin Use at 6 Months
|
-0.10 units/kg
Standard Deviation 0.18
|
0.09 units/kg
Standard Deviation 0.23
|
SECONDARY outcome
Timeframe: Baseline and 12 monthsChange in insulin use from baseline at 12 months
Outcome measures
| Measure |
Sugar Pill (Placebo)
n=10 Participants
Placebo at same dose, frequency, and duration as experimental treatment
Sugar Pill (placebo): Placebo
|
Taurourodeoxycholic Acid (TUDCA)
n=10 Participants
TUDCA 1750 mg/day x 12 months
Tauroursodeoxycholic Acid (TUDCA): TUDCA at 1750 mg/day x 12 months
|
|---|---|---|
|
Change in Insulin Use at 12 Months
|
-0.10 units/kg
Standard Deviation 0.14
|
0.09 units/kg
Standard Deviation 0.16
|
SECONDARY outcome
Timeframe: Baseline and 18 monthsChange in insulin use from baseline at 18 months
Outcome measures
| Measure |
Sugar Pill (Placebo)
n=10 Participants
Placebo at same dose, frequency, and duration as experimental treatment
Sugar Pill (placebo): Placebo
|
Taurourodeoxycholic Acid (TUDCA)
n=10 Participants
TUDCA 1750 mg/day x 12 months
Tauroursodeoxycholic Acid (TUDCA): TUDCA at 1750 mg/day x 12 months
|
|---|---|---|
|
Change in Insulin Use at 18 Months
|
-0.17 units/kg
Standard Deviation 0.18
|
0.02 units/kg
Standard Deviation 0.21
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsChange in HbA1c from baseline at 6 months
Outcome measures
| Measure |
Sugar Pill (Placebo)
n=10 Participants
Placebo at same dose, frequency, and duration as experimental treatment
Sugar Pill (placebo): Placebo
|
Taurourodeoxycholic Acid (TUDCA)
n=10 Participants
TUDCA 1750 mg/day x 12 months
Tauroursodeoxycholic Acid (TUDCA): TUDCA at 1750 mg/day x 12 months
|
|---|---|---|
|
Change in HbA1c at 6 Months
|
-0.98 Percent
Standard Deviation 1.57
|
-2.27 Percent
Standard Deviation 2.13
|
SECONDARY outcome
Timeframe: Baseline and 12 monthsChange in HbA1c from baseline at 12 months
Outcome measures
| Measure |
Sugar Pill (Placebo)
n=10 Participants
Placebo at same dose, frequency, and duration as experimental treatment
Sugar Pill (placebo): Placebo
|
Taurourodeoxycholic Acid (TUDCA)
n=10 Participants
TUDCA 1750 mg/day x 12 months
Tauroursodeoxycholic Acid (TUDCA): TUDCA at 1750 mg/day x 12 months
|
|---|---|---|
|
Change in HbA1c at 12 Months
|
-0.02 Percent
Standard Deviation 3.31
|
-2.25 Percent
Standard Deviation 2.13
|
SECONDARY outcome
Timeframe: Baseline and 18 MonthsChange in HbA1c from baseline at 18 months
Outcome measures
| Measure |
Sugar Pill (Placebo)
n=10 Participants
Placebo at same dose, frequency, and duration as experimental treatment
Sugar Pill (placebo): Placebo
|
Taurourodeoxycholic Acid (TUDCA)
n=10 Participants
TUDCA 1750 mg/day x 12 months
Tauroursodeoxycholic Acid (TUDCA): TUDCA at 1750 mg/day x 12 months
|
|---|---|---|
|
Change in HbA1c at 18 Months
|
-0.48 Percent
Standard Deviation 2.22
|
-1.89 Percent
Standard Deviation 2.14
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 1 weekPopulation: Biopsy samples were collected, but not sent for pathological analysis due to trial futility. There are no results available to report.
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.
Outcome measures
Outcome data not reported
Adverse Events
Sugar Pill (Placebo)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Taurourodeoxycholic Acid (TUDCA)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Robin Goland, MD
Columbia University Irving Medical Center
Phone: 212-851-5494
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place