Clinical Trials

Numerous clinical trials spanning Phase 1 through Phase 4 evaluate the therapeutic utility and pharmacokinetic profile of ursodiol across gastroenterological, metabolic, and oncological conditions, including neonatal cholestasis, cystic fibrosis, nonalcoholic fatty liver disease, type 2 diabetes, and high-risk pediatric solid tumors. Sponsored by academic medical centers, clinical investigators, public research organizations, and commercial entities, these studies encompass completed, terminated, unknown, and not yet recruiting cohorts.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06884748 NOT_YET_RECRUITING
Clostridioides Difficile Infection
Medical College of Wisconsin
 2026-05-01 EARLY_PHASE1
NCT03724175 TERMINATED
Ulcerative Colitis; Pouchitis
Stanford University
 2019-08-26 PHASE2; PHASE3
NCT01505569 COMPLETED
Ewing's Family Tumors; Renal Tumors; Hepatoblastoma; Rhabdomyosarcoma; Soft Tissue Sarcoma; Primary Malignant Brain Neoplasms; Retinoblastoma; Medulloblastoma; Supra-tentorial Primative Neuro-Ectodermal Tumor (PNET); Atypical Teratoid/Rhabdoid Tumor (AT/RT); CNS Tumors; Germ Cell Tumors
Masonic Cancer Center, University of Minnesota
 2011-10-20
NCT04910178 COMPLETED
Diabetes Type 2; NAFLD
Asmaa Abdelfattah Elsayed
 2020-12-01 PHASE4
NCT02748616 COMPLETED
Diarrhea
NYU Langone Health
 2017-03-01 PHASE4
NCT02319629 UNKNOWN
Morbid Obesity
HaEmek Medical Center, Israel
 2014-12
NCT03512821 COMPLETED
Healthy
Daewoong Pharmaceutical Co. LTD.
 2017-10-01 PHASE1
NCT03512808 COMPLETED
Healthy
Daewoong Pharmaceutical Co. LTD.
 2017-06-01 PHASE1
NCT02033876 COMPLETED
Type 2 Diabetes Mellitus
Mayo Clinic
 2013-10 PHASE2
NCT02244944 TERMINATED
Nonalcoholic Fatty Liver Disease (NAFLD)
Gregory Graf, PhD
 2014-09 PHASE2
NCT02385032 COMPLETED
Healthy
Daewoong Pharmaceutical Co. LTD.
 2015-02 PHASE1
NCT01097304 COMPLETED
Barrett Esophagus; Esophageal Carcinoma
National Cancer Institute (NCI)
 2010-04 PHASE2
NCT00846963 COMPLETED
Cholestasis
Ibrahim Mohamed
 2008-10 PHASE2; PHASE3
NCT00873275 COMPLETED
Colorectal Cancer
City of Hope Medical Center
 2009-03-11 PHASE1
NCT00706381 COMPLETED
Healthy Volunteers
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
 2008-06-23 PHASE3
NCT00514774 UNKNOWN
Huntington Disease
Oregon Health and Science University
 2007-08 PHASE1
NCT00846963 Completed
Cholestasis
Ibrahim Mohamed|St. Justine''s Hospital
 2008-10 Phase 2|Phase 3
NCT00909610 COMPLETED
Healthy
Teva Pharmaceuticals USA
 2006-12 PHASE1
NCT00909610 Completed
Healthy
Teva Pharmaceuticals USA
 2006-12 Phase 1
NCT00909753 COMPLETED
Healthy
Teva Pharmaceuticals USA
 2006-09 PHASE1
NCT00909753 Completed
Healthy
Teva Pharmaceuticals USA
 2006-09 Phase 1
NCT00062023 TERMINATED
Colorectal Cancer
M.D. Anderson Cancer Center
 2003-06 PHASE2
NCT00004442 TERMINATED
Infantile Refsum's Disease; Zellweger Syndrome; Bifunctional Enzyme Deficiency; Adrenoleukodystrophy
University of Cincinnati
NCT00004315 UNKNOWN
Cystic Fibrosis; Gastrointestinal Diseases; Cholestasis
National Center for Research Resources (NCRR)
 1995-11 PHASE2
NCT00004748 COMPLETED
Liver Cirrhosis, Biliary
National Center for Research Resources (NCRR)
 1989-11 PHASE3
NCT00006168 COMPLETED
Liver Cirrhosis, Biliary
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
 1994-01 PHASE3
NCT00042549 TERMINATED
Cholelithiasis
Medstone International
 2002-05 PHASE4
NCT00004441 COMPLETED
Cystic Fibrosis
Children's Hospital Medical Center, Cincinnati
 1997-09
NCT00004784 COMPLETED
Liver Cirrhosis, Biliary
National Center for Research Resources (NCRR)
 1994-01 PHASE3

(data from https://clinicaltrials.gov, updated on 2026-04-29)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Ursodiol acts by suppressing hepatic synthesis and biliary secretion of cholesterol while reducing intestinal cholesterol absorption through micellar solubilization. This reduction in biliary cholesterol saturation prevents lithogenic gallstone formation and promotes the dissolution of existing cholesterol stones, offering essential therapeutic benefit in clinical conditions such as cholestasis and nonalcoholic fatty liver disease.

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.