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TUDCA as a Therapy for Ulcerative Colitis (UC)

Phase 1
Conditions
Ulcerative Colitis
Interventions
Drug: Tauroursoursodeoxycholic acid, brand name Tudcabil
Registration Number
NCT04114292
Lead Sponsor
Washington University School of Medicine
Brief Summary

This is a Phase I open label study examining the efficacy and safety of TUDCA (tauroursodeoxycholic acid) in ulcerative colitis treatment.

Detailed Description

TUDCA (tauroursodeoxycholic acid) is a dietary supplement that has shown to reduce cellular stress related to inflammation. In this Phase I study, patients with active ulcerative colitis will be receive TUDCA for six weeks. Safety and tolerability will be assessed. Efficacy will be assessed using ulcerative colitis disease activity measurements as well as markers of intestinal inflammation before and after treatment. The overall goal of this project is to determine TUDCA can provide a new therapeutic option with a desirable safety profile for patients with ulcerative colitis focused on the inflamed intestinal epithelium.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
13
Inclusion Criteria
  • Ages Eligible for Study: 18 Years to 65 Years;
  • Confirmed ulcerative colitis disease through radiographic, endoscopic and/or histologic criteria;
  • Confirmed with active ulcerative colitis (defined as a complete Mayo score ≥ 5 with endoscopic subscore of ≥ 1) See Appendix for Mayo Score using recent adaptation to include any friability on endoscopy to be scored as "2".
  • On a stable dose of medications for inflammatory bowel disease (IBD) (i.e. no change in medication within 4 weeks of study enrollment) and not planning to initiate new medication other than TUDCA.
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Exclusion Criteria
  • Those that received other chemical chaperone therapies in the 3 months prior to screening;
  • Individuals accompanied by gallstones, other intestinal disorders or cancers, or any possible cholestatic pathologies that could alter the enterohepatic circulation of the bile acids, including previous cholecystectomy or short bowel syndrome;
  • Subjects with alcohol or drug abuse within the recent year;
  • Serious heart, lung, kidney, digestive, nervous, mental, or autoimmune diseases
  • Those with plans for abdominal surgery;
  • Those unable or unwilling to provide informed consent or failure to comply with the test requirements;
  • Pregnant, lactating women;
  • Those receiving or planning to receive medicines that inhibit the absorption of the bile acids in the intestine;
  • All female subjects must have birth control and not plan to become pregnant during the study. As TUDCA may interfere in the absorption of oral contraceptives, the acceptable methods of birth control should include abstinence or 2 of the following intrauterine device (IUD-with or without local hormone release), diaphragm, spermicides, cervical cap, contraceptive sponge, and /or condoms.
  • Subjects with baseline liver transamines (AST or ALT) > 1.5 X the upper limit of normal.
  • Patients with complete biliary obstruction and known hypersensitivity or intolerance to TUDCA or any of the components of Tudcabil (or to other bile acids).
  • Patients with moderate-to-severe hepatic impairment.
  • Evidence of worsening liver function based on the 2 initial laboratory values used to establish the baseline.
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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
TUDCATauroursoursodeoxycholic acid, brand name Tudcabil1.75-2 grams daily in divided dosing
Primary Outcome Measures
NameTimeMethod
Determine the effect of TUDCA treatment on ER stress in colon biopsy tissues from subjects with symptomatic ulcerative colitisBaseline and post 6 weeks of TUDCA treatment

Change in ER stress markers as measured by expression of X box binding protein-1 (XBP1), Binding immunoglobulin protein (BIP) and Eukaryotic Translation Initiation Factor 2A (eIF2a) in colon biopsy tissues before and after 6 weeks of TUDCA treatment.

Secondary Outcome Measures
NameTimeMethod
Monitoring of UC patients for TUDCA safety and tolerability: Adverse eventsEvery 2 weeks after starting TUDCA until 2 weeks after completing 6 weeks of TUDCA treatment. (i.e. study points weeks 2, 4, 6 and 8)

Monitor and define TUDCA safety/tolerability in this population through frequent monitoring and symptom assessment as detailed in the study procedures. Adverse events will be categorized according to CTCAE v5.0 criteria and grading.

Change ulcerative colitis disease activity based on Total Mayo ScoreBaseline and post 6 weeks of TUDCA treatment

The Mayo Ulcerative colitis score is an aggregate of individual parameters evaluating rectal bleeding, stool frequency, endoscopic severity and physicians global assessment. Each of these subscores has a range from 0-3 where 3 is worse. a total score can range from 0 to 12 with 0 being in remission and 12 representing the most severe disease. As this is a secondary endpoint in a small trial, patients total scores (out of 12) will be compared from baseline to week 6 using a paired students t test. Mayo score reference is Schroeder KW, Tremaine WJ, Ilstrup DM. Coated oral 5-aminosalicylic acid therapy for mildly to moderately active ulcerative colitis. A randomized study. N Engl J Med 1987;317:1625-9.

Change in inflammation on histology by Goebes indexBaseline and post 6 weeks of TUDCA treatment

The Geboes histology index is a reproducible grading scale for histological assessment of inflammation in ulcerative colitis. This is a six grade classification system for inflammation which can also be fine tuned within each grade. The grades are: 0, structural change only; 1, chronic inflammation; 2, lamina propria neutrophils; 3, neutrophils in epithelium; 4, crypt destruction; and 5, erosions or ulcers. As this is a secondary endpoint in a small trial, patients total scores (out of 20) will be compared from baseline to week 6 using a paired students t test. Higher scores represent more severe disease. The reference for the Geboes index is "Geboes K, Riddell R, Ost A, et al. A reproducible grading scale for histological assessment of inflammation in ulcerative colitis. Gut 2000;47:404-9."

Trial Locations

Locations (3)

Washington University School of Medicine

🇺🇸

Saint Louis, Missouri, United States

Barnes-Jewish West County Hospital

🇺🇸

Creve Coeur, Missouri, United States

Barnes-Jewish Hospital

🇺🇸

Saint Louis, Missouri, United States

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