Clinical Trials

Numerous clinical trials evaluate tauroursodeoxycholic acid across indications including neurodegenerative conditions such as amyotrophic lateral sclerosis, gastrointestinal and hepatic diseases (ulcerative colitis, intrahepatic cholestasis, liver cirrhosis), type 2 diabetes mellitus, and TTR cardiac amyloidosis. Spanning Early Phase 1 through Phase 4, these studies comprise completed, actively recruiting, terminated, and unknown recruitment statuses. They are funded by diverse entities, including pharmaceutical companies, academic medical centers, and charitable organizations.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07328009 RECRUITING
Hepato Cellular Carcinoma (HCC)
Fudan University
 2026-01-19 PHASE2
NCT06025630 RECRUITING
Hypertension
University of North Texas Health Science Center
 2023-08-15 PHASE1; PHASE2
NCT07457632 NOT_YET_RECRUITING
Diabetic Macular Edema (DME)
University of Alabama at Birmingham
 2026-10-01 PHASE2
NCT07100392 RECRUITING
Hepatocellular Carcinoma (HCC)
Fudan University
 2025-08-14 PHASE2
NCT07064668 NOT_YET_RECRUITING
Hepatocellular Carcinoma
Tongji Hospital
 2025-07-30 PHASE2
NCT07517276 NOT_YET_RECRUITING
Metabolic Dysfunction-Associated Steatotic Liver Disease Infant Development
Peking University First Hospital
 2026-04-02 PHASE2
NCT06827873 NOT_YET_RECRUITING
Influenza Vaccine Response
Tsinghua University
 2025-02
NCT05753852 UNKNOWN
Amyotrophic Lateral Sclerosis
Humanitas Mirasole SpA
 2021-10-25 PHASE3
NCT03800524 UNKNOWN
Amyotrophic Lateral Sclerosis (ALS)
Humanitas Mirasole SpA
 2019-02-22 PHASE3
NCT03481972 COMPLETED
TTR Cardiac Amyloidosis
Fondazione IRCCS Policlinico San Matteo di Pavia
 2018-04-11 PHASE3
NCT04114292 COMPLETED
Ulcerative Colitis
Washington University School of Medicine
 2019-01-17 PHASE1
NCT03423121 COMPLETED
Progressive Multiple Sclerosis
Johns Hopkins University
 2018-06-19 PHASE1; PHASE2
NCT05753852 Recruiting
Amyotrophic Lateral Sclerosis
Humanitas Mirasole SpA|University of Ulm|University of Sheffield|University Hospital Tours|KU Leuven|UMC Utrecht|University of Dublin Trinity College|Bruschettini S.r.l.|Istituto Superiore di Sanità|Motor Neurone Disease Association
 2021-10-25 Phase 3
NCT04727320 UNKNOWN
Liver Cirrhosis
The First Affiliated Hospital with Nanjing Medical University
 2021-03-05 EARLY_PHASE1
NCT03488524 COMPLETED
Amyotrophic Lateral Sclerosis; ALS
Amylyx Pharmaceuticals Inc.
 2018-03-29 PHASE2
NCT03462940 TERMINATED
Diabetes Mellitus, Type 2; Insulin Response; Endothelial Dysfunction
Boston University
 2018-04-02
NCT03533257 COMPLETED
Alzheimer Disease
Amylyx Pharmaceuticals Inc.
 2018-09-14 PHASE2
NCT02218619 COMPLETED
Type 1 Diabetes
Robin Goland, MD
 2015-08 PHASE2
NCT03127514 COMPLETED
Amyotrophic Lateral Sclerosis; Motor Neuron Disease; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Nervous System Diseases; Central Nervous System Diseases
Amylyx Pharmaceuticals Inc.
 2017-06-22 PHASE2
NCT03878654 TERMINATED
Asthma
University of Vermont
 2019-01-10 PHASE1
NCT04114292 Unknown status
Ulcerative Colitis
Washington University School of Medicine|Crohn''s and Colitis Foundation
 2019-01-17 Phase 1
NCT01998451 UNKNOWN
Gallstone
The First Affiliated Hospital of Guangzhou Medical University
 2012-12 PHASE4
NCT01877551 COMPLETED
HIV Related Insulin Resistance; Protease Inhibitor Related Insulin Resistance; Endoplasmic Reticulum Stress
Washington University School of Medicine
 2013-09
NCT03331432 COMPLETED
Type 2 Diabetes Mellitus
University of Missouri-Columbia
 2015-10-28
NCT01855360 COMPLETED
Amyloidosis; Heart (Manifestation); Senile Cardiac Amyloidosis
Brigham and Women's Hospital
 2013-06 PHASE1; PHASE2
NCT01171859 COMPLETED
Transthyretin Amyloidosis
Fondazione IRCCS Policlinico San Matteo di Pavia
 2010-07 PHASE2
NCT00771901 COMPLETED
Insulin Resistance; Diabetes; Obesity
Washington University School of Medicine
 2008-02
NCT01899703 Completed
Cholestasis Intrahepatic
GlaxoSmithKline
 2014-03-10 Phase 2
NCT01857284 COMPLETED
Primary Biliary Cirrhosis
Beijing Friendship Hospital
 2009-09 PHASE3
NCT01829698 COMPLETED
Cholestatic Liver Disease
Beijing Trendful Kangjian Medical Information Consulting Limited Company
 2009-08 PHASE3
NCT00877604 COMPLETED
Amyotrophic Lateral Sclerosis
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
 2008-06 PHASE2
NCT00004410 COMPLETED
Cholestasis
Children's Hospital Medical Center, Cincinnati
 1998-06
NCT00004441 COMPLETED
Cystic Fibrosis
Children's Hospital Medical Center, Cincinnati
 1997-09

(data from https://clinicaltrials.gov, updated on 2026-04-21)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Tauroursodeoxycholic acid acts as a hydrophilic bile acid derivative that stabilizes mitochondrial membranes and reduces endoplasmic reticulum stress, thereby blocking pro-apoptotic caspase cascades and preventing cell death. By suppressing programmed cell death and preserving organelle integrity, this mechanism provides cytoprotective benefits relevant to clinical trial evaluations in neurodegenerative conditions like amyotrophic lateral sclerosis and metabolic liver 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.