Clinical Trials

Multiple clinical trials evaluate chenodeoxycholic acid and related pathways across metabolic and hepatobiliary conditions, including bile acid malabsorption, chronic diarrhea, cholelithiasis, primary biliary cirrhosis, severe obesity, and type 2 diabetes. Spanning early phase through Phase 4 studies, these trials are supported by both academic institutions and industry sponsors. Recruitment statuses across the research portfolio vary, encompassing completed, ongoing, terminated, and withdrawn trials.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06260748 WITHDRAWN
Cerebrotendinous Xanthomatoses
Leadiant Biosciences, Inc.
 2024-05-27 PHASE3
NCT04270682 COMPLETED
CTX
Mirum Pharmaceuticals, Inc.
 2020-01-31 PHASE3
NCT06180057 COMPLETED
Cerebrotendinous Xanthomatoses
Humanis Saglık Anonim Sirketi
 2022-06-12 PHASE1
NCT05130047 Completed
Chronic Diarrhea|Irritable Bowel Syndrome With Diarrhea|Bile Acid Malabsorption|Bile Acid Diarrhea|Bile Acid Malabsorption Syndrome Type II|Functional Diarrhea
Michael Camilleri MD|NGM Biopharmaceuticals Inc|Mayo Clinic
 2021-12-01 Phase 2
NCT04640571 COMPLETED
Bile Salt Export Pump (BSEP) Transporter; Polysorbate 80
University of Maryland, Baltimore
 2021-04-01 PHASE4
NCT03168555 COMPLETED
Bile Acid Malabsorption; Cholelithiasis
Zealand University Hospital
 2017-06-22 PHASE4
NCT02952963 COMPLETED
Severe Obesity
Hvidovre University Hospital
 2016-10 PHASE4
NCT03059537 COMPLETED
Bile Acid Malabsorption
Lars Kristian Munck
 2017-03-13 PHASE4
NCT02876484 COMPLETED
Severe Obesity
Hvidovre University Hospital
 2016-06 PHASE4
NCT03168555 Completed
Bile Acid Malabsorption|Cholelithiasis
Zealand University Hospital
 2017-06-22 Phase 4
NCT02340247 COMPLETED
Severe Obesity
Hvidovre University Hospital
 2014-11 PHASE4
NCT01865812 Completed
Primary Biliary Cirrhosis
Intercept Pharmaceuticals
 2013-12-03 Phase 2
NCT01666223 COMPLETED
Type 2 Diabetes; Obesity
University Hospital, Gentofte, Copenhagen
 2012-11
NCT01570439 Unknown status
Anonymous Donors at Blood Donation Center (NUH)
National University Hospital Singapore
 2012-01 --
NCT01674946 COMPLETED
Echolocation
University Hospital, Basel, Switzerland
 2011-09 PHASE1
NCT00018694 WITHDRAWN
Cerebrotendinous Xanthomatosis
US Department of Veterans Affairs
 1999-10
NCT00912301 COMPLETED
Constipation-predominant Irritable Bowel Syndrome
Mayo Clinic
 2009-04 PHASE2
NCT00550862 TERMINATED
Liver Cirrhosis, Biliary
Intercept Pharmaceuticals
 2007-10 PHASE2
NCT00004442 TERMINATED
Infantile Refsum's Disease; Zellweger Syndrome; Bifunctional Enzyme Deficiency; Adrenoleukodystrophy
University of Cincinnati
NCT00465751 COMPLETED
Metabolic Syndrome; Familial Hypertriglyceridemia; Familial Combined Hyperlipidemia
University Hospital, Basel, Switzerland
 2004-10 EARLY_PHASE1
NCT00004346 UNKNOWN
Cerebrotendinous Xanthomatosis
National Center for Research Resources (NCRR)
 1996-01 PHASE2

(data from https://clinicaltrials.gov, updated on 2024-11-18)

Check the Chenodeoxycholic Acid product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Chenodeoxycholic acid acts as a primary bile acid that selectively activates the nuclear farnesoid X receptor (FXR), downstream of which hepatic cholesterol synthesis and intestinal absorption are suppressed. This reduction in biliary cholesterol saturation facilitates the solubilization of gallstones, providing a mechanistic basis for its therapeutic utility in cholelithiasis and bile acid malabsorption disorders.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.