NCT01585025: OBADIAH1

Obeticholic Acid Treatment in Patients With Bile Acid Diarrhoea: an Open-label, Pilot Study of Mechanisms, Safety and Symptom Response.

Completed Phase 2 Results posted Last updated 8 March 2023

What this trial tests

Phase 2 trial testing Obeticholic acid in Primary Bile Acid Malabsorption in 35 participants. Completed in 1 February 2014.

Timeline

1 April 2012

Primary endpoint
1 January 2014

1 February 2014

Quick facts

Lead sponsor	Imperial College Healthcare NHS Trust
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	35
Start date	1 April 2012
Primary completion	1 January 2014
Estimated completion	1 February 2014
Sites	2 locations across United Kingdom

Drugs / interventions tested

• Obeticholic acid (OBETICHOLIC ACID) — full drug profile →

Conditions studied

• Primary Bile Acid Malabsorption — all drugs for Primary Bile Acid Malabsorption →
• Secondary Bile Acid Malabsorption — all drugs for Secondary Bile Acid Malabsorption →
• Chronic Diarrhoea — all drugs for Chronic Diarrhoea →

Sponsor

Imperial College Healthcare NHS Trust

Who can join

Adults 18 to 80, any sex, with Primary Bile Acid Malabsorption or Secondary Bile Acid Malabsorption. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Changes in Fasting FGF19
  Time frame: Day 0, Day 15
  The primary outcome measure is the change over 2 weeks in fasting serum fibroblast growth factor (FGF19) in 3 groups of patients: primary bile acid diarrhoea, secondary bile acid diarrhoea, and a control population of patients with chronic diarrhoea but with normal bile acid retention.

Sponsor's own description

The investigators propose to develop studies of obeticholic acid (OCA) in patients with bile acid diarrhoea. OCA is a semisynthetic bile acid, also known as 6αethylchenodeoxycholic acid or INT747,and is a potent farnesoid X receptor (FXR) agonist. Preliminary data suggests that patients with bile acid diarrhoea have impaired production of the ileal hormone Fibroblast Growth Factor 19 (FGF19). FGF19 is stimulated by FXR agonists, and regulates bile acid synthesis. This study is a pilot, proof-of-concept, open-label study to investigate whether OCA can stimulate FGF19 in bile acid diarrhoea patients to provide a safe and effective treatment.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. FGF/FGFR signaling in health and disease.
   Xie Y, Su N, Yang J, Tan Q, et al · · 2020 · cited 588× · PMID 32879300 · DOI 10.1038/s41392-020-00222-7
2. The response of patients with bile acid diarrhoea to the farnesoid X receptor agonist obeticholic acid.
   Walters JR, Johnston IM, Nolan JD, Vassie C, et al · · 2015 · cited 130× · PMID 25329562 · DOI 10.1111/apt.12999
3. FXR: structures, biology, and drug development for NASH and fibrosis diseases.
   Tian SY, Chen SM, Pan CX, Li Y. · · 2022 · cited 65× · PMID 35217809 · DOI 10.1038/s41401-021-00849-4
4. Review article: therapeutic aspects of bile acid signalling in the gut-liver axis.
   Simbrunner B, Trauner M, Reiberger T. · · 2021 · cited 63× · PMID 34555862 · DOI 10.1111/apt.16602
5. The Saga of Endocrine FGFs.
   Phan P, Saikia BB, Sonnaila S, Agrawal S, et al · · 2021 · cited 48× · PMID 34572066 · DOI 10.3390/cells10092418
6. Enterohepatic and non-canonical roles of farnesoid X receptor in controlling lipid and glucose metabolism.
   Zhou W, Anakk S. · · 2022 · cited 31× · PMID 35304191 · DOI 10.1016/j.mce.2022.111616
7. Nuclear receptors in health and disease: signaling pathways, biological functions and pharmaceutical interventions.
   Jin P, Duan X, Huang Z, Dong Y, et al · · 2025 · cited 21× · PMID 40717128 · DOI 10.1038/s41392-025-02270-3
8. Role of FXR in Renal Physiology and Kidney Diseases.
   Guo Y, Xie G, Zhang X. · · 2023 · cited 19× · PMID 36768731 · DOI 10.3390/ijms24032408