CLINICAL TRIALS PROFILE FOR URSODIOL

505(b)(2) Clinical Trials for URSODIOL

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
OTC	NCT00125281 ↗	SAMe to Treat Biliary Cirrhosis Symptoms	Terminated	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 2	2005-07-25	This study will examine the effect of S-adenosyl methionine (SAMe) on itching and fatigue in patients with primary biliary cirrhosis, a disease of the small bile ducts in the liver. Ursodiol, the only currently available treatment for biliary cirrhosis, does not cure the disease, and many people continue to have symptoms or liver test abnormalities despite treatment. SAMe is a naturally occurring substance found in most cells of the body. The highest levels of the substance are produced by the liver, where it helps to rid the body of toxins and breakdown products of metabolism. Studies in Europe suggest that SAMe may help to: 1) decrease the fatigue and itching that are common in persons with liver problems, and 2) decrease levels of liver enzymes in the blood, suggesting that it may decrease the amount of liver injury. Patients 21 years of age or older with primary biliary cirrhosis who are taking ursodiol and have symptoms of itching or fatigue may be eligible for this study. Candidates are screened with a medical history, physical examination, review of medical records, routine blood tests, and a symptoms rating scale. Participants stop all medications for itching 4 weeks before starting the study, but continue to take ursodiol during the 42-week trial. On entering the study, patients are assigned to take either SAMe or placebo tablets twice a day for 12 weeks. While taking the medications, they are followed in the clinic every 2 weeks for the first month and then every 4 weeks to fill out symptoms questionnaires and have a short medical evaluation and blood tests. At the end of 12 weeks, treatment is interrupted for a 2-week "wash-out" period, after which patients begin a 12-week crossover treatment; that is, patients who were taking SAMe are switched to placebo, and those who were taking placebo are switched to SAMe. After completing the second 12-week treatment course, patients come to the clinic at 4, 8, and 12 weeks to fill out symptoms questionnaires and have a medical evaluation and blood tests. At the last visit, patients are told which type of tablet they received during the two courses of treatment. SAMe is available without prescription in many forms as an over-the-counter medication.

All Clinical Trials for URSODIOL

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00004315 ↗	Phase II Pilot Study to Compare the Bioavailability of Buffered, Enteric-Coated Ursodiol With Unmodified Ursodiol for Chronic Cholestatic Liver Disease and Cystic Fibrosis-Associated Liver Disease	Unknown status	Children's Hospital Medical Center, Cincinnati	Phase 2	1995-11-01	OBJECTIVES: I. Compare the bioavailability of polymer-coated and buffered ursodiol (ursodeoxycholic acid) to unmodified ursodiol in patients with cystic fibrosis-associated liver disease or chronic cholestatic liver disease. II. Compare the differences in pruritus, weight gain, and liver function for both treatments.
NCT00004315 ↗	Phase II Pilot Study to Compare the Bioavailability of Buffered, Enteric-Coated Ursodiol With Unmodified Ursodiol for Chronic Cholestatic Liver Disease and Cystic Fibrosis-Associated Liver Disease	Unknown status	National Center for Research Resources (NCRR)	Phase 2	1995-11-01	OBJECTIVES: I. Compare the bioavailability of polymer-coated and buffered ursodiol (ursodeoxycholic acid) to unmodified ursodiol in patients with cystic fibrosis-associated liver disease or chronic cholestatic liver disease. II. Compare the differences in pruritus, weight gain, and liver function for both treatments.
NCT00004441 ↗	Study of Tauroursodeoxycholic Acid for Hepatobiliary Disease in Cystic Fibrosis	Completed	Children's Hospital Medical Center, Cincinnati	N/A	1997-09-01	OBJECTIVES: I. Determine the optimum dose of tauroursodeoxycholic acid (TUDCA) required to achieve maximal bioavailability for patients with cystic fibrosis-associated liver disease. II. Compare optimized doses of TUDCA with ursodiol (ursodeoxycholic acid; UDCA) for effects on biliary bile acid composition and metabolism, serum biochemistries, fat absorption, and fat-soluble vitamin status in these patients.
NCT00004442 ↗	Study of Bile Acids in Patients With Peroxisomal Disorders	Terminated	Children's Hospital Medical Center, Cincinnati	N/A	1969-12-31	OBJECTIVES: I. Determine the effectiveness of oral bile acid therapy with cholic acid, chenodeoxycholic acid, and ursodeoxycholic acid in patients with peroxisomal disorders involving impaired primary bile acid synthesis. II. Determine whether suppression of synthesis of atypical bile acids and enrichment of bile acid pool with this regimen is effective in treating this patient population and improving quality of life.
NCT00004442 ↗	Study of Bile Acids in Patients With Peroxisomal Disorders	Terminated	University of Cincinnati	N/A	1969-12-31	OBJECTIVES: I. Determine the effectiveness of oral bile acid therapy with cholic acid, chenodeoxycholic acid, and ursodeoxycholic acid in patients with peroxisomal disorders involving impaired primary bile acid synthesis. II. Determine whether suppression of synthesis of atypical bile acids and enrichment of bile acid pool with this regimen is effective in treating this patient population and improving quality of life.
NCT00004748 ↗	Low-Dose Oral Methotrexate Versus Colchicine for Primary Biliary Cirrhosis	Completed	Tufts Medical Center	Phase 3	1989-11-01	OBJECTIVES: I. Compare the efficacy of low-dose oral pulse methotrexate (MTX) and ursodiol versus colchicine and ursodiol in patients with primary biliary cirrhosis (PBC). II. Determine the optimum dose and duration of MTX treatment. III. Investigate the role of fibrogenic cytokines (FC) in PBC pathogenesis and the effect of treatment on FC production.

Clinical Trial Conditions for URSODIOL

Condition Name

Condition Name for URSODIOL
Intervention	Trials
Healthy	5
Liver Cirrhosis, Biliary	4
Cholestasis	2
Primary Biliary Cirrhosis	2

Condition MeSH

Condition MeSH for URSODIOL
Intervention	Trials
Fibrosis	8
Liver Cirrhosis, Biliary	6
Liver Cirrhosis	6
Syndrome	2

Clinical Trial Locations for URSODIOL

Trials by Country

Trials by Country for URSODIOL
Location	Trials
United States	51
Canada	6
Italy	2
Egypt	2
Germany	1

Trials by US State

Trials by US State for URSODIOL
Location	Trials
Texas	5
Minnesota	4
Arizona	3
Washington	3
Virginia	3

Clinical Trial Progress for URSODIOL

Clinical Trial Phase

Clinical Trial Phase for URSODIOL
Clinical Trial Phase	Trials
PHASE4	1
Phase 4	4
Phase 3	6

Clinical Trial Status

Clinical Trial Status for URSODIOL
Clinical Trial Phase	Trials
Completed	18
Terminated	8
Recruiting	4

Clinical Trial Sponsors for URSODIOL

Sponsor Name

Sponsor Name for URSODIOL
Sponsor	Trials
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	5
National Cancer Institute (NCI)	4
Children's Hospital Medical Center, Cincinnati	3

Sponsor Type

Sponsor Type for URSODIOL
Sponsor	Trials
Other	52
NIH	13
Industry	9

Clinical Trials Update, Market Analysis, and Projection for Ursodiol

Last updated: October 28, 2025

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Introduction

Ursodiol, also known as ursodeoxycholic acid (UDCA), is a naturally occurring bile acid primarily used in the treatment of various cholestatic liver diseases, gallstones, and related hepatic conditions. Approved initially for gallstone dissolution, ursodiol's therapeutic scope has expanded, supported by ongoing clinical research. This article provides a comprehensive update on ursodiol’s clinical trials, analyzes its current market landscape, and projects future growth trends based on recent developments.

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Clinical Trials Overview

Recent and Ongoing Clinical Trials

Over the past five years, numerous clinical trials have evaluated ursodiol's efficacy and safety in broader indications beyond its traditional use, including non-alcoholic fatty liver disease (NAFLD), primary biliary cholangitis (PBC), and non-responsive gallstone cases.

1. NAFLD and NASH (Non-Alcoholic Steatohepatitis):
   Recent trials like NCT04583484 examined ursodiol’s role in reducing hepatic fat accumulation and fibrosis progression. Preliminary results indicate modest biochemical improvements, but large-scale, definitive studies remain pending.

2. Primary Biliary Cholangitis (PBC):
   While ursodiol is approved for PBC, ongoing trials such as NCT03711604 are investigating optimized dosages, combination therapies with immunomodulators, and long-term safety.

3. Cholestasis in Pediatric Populations:
   Trials like NCT04452990 assess ursodiol for pediatric cholestatic conditions, with findings supporting a favorable safety profile and adequate tolerability.

4. Emerging Indications:
   Investigations into ursodiol's role in neurodegenerative diseases and certain inflammatory liver conditions are in nascent phases, with early data indicating potential immunomodulatory benefits.

Regulatory and Market Influence of Clinical Trials

The ongoing research has reinforced ursodiol’s safety profile and expanded its therapeutic landscape. Notably, data from these trials support regulatory agencies' consideration for expanded indications and dosing guidelines, likely influencing future approvals or label expansions.

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Market Analysis

Current Market Size and Key Players

Ursodiol’s global market was valued at approximately USD 300 million in 2022, driven largely by its widespread use in gallstone dissolution and primary biliary cholangitis. Major pharmaceutical companies such as Solvay Pharmaceuticals (Pr aliases: Actigall in the US) dominate the landscape, with generic manufacturers also capturing significant market share due to widespread patent expiries.

Market Drivers

• Increased Prevalence of Liver Diseases: Rising global incidence of NAFLD, obesity, and metabolic syndromes enhance demand for ursodiol as a management option.
• Expanding Indications: Growing evidence supporting off-label uses and new clinical trial data fuel market growth.
• Generics and Cost-Effectiveness: The availability of affordable generic versions broadens access, especially in emerging markets.

Market Challenges

• Indication Limitations: Currently approved primarily for gallstones and PBC, limiting broader application.
• Competitive Landscape: Presence of alternative therapies, such as obeticholic acid and new biologics, poses threat.
• Regulatory Hurdles: Pending trial results may influence future approvals, impacting market stability.

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Market Growth Projections

Based on current data and anticipated approvals, the global ursodiol market is projected to grow at a CAGR of approximately 4.5% from 2023 to 2030, potentially reaching USD 415 million by 2030.

Factors Influencing Growth

• Epidemiology Trends: Increasing burden of NAFLD and cholestatic diseases is expected to sustain demand.
• Regulatory Approvals and Expansions: Pending clinical trial outcomes and subsequent label extensions could unlock new markets.
• Innovation in Formulations: Liposomal and sustained-release formulations could improve treatment adherence and effectiveness.

Geographical Market Dynamics

• North America: Largest market due to high prevalence of liver diseases and well-established healthcare infrastructure.
• Europe: Significant growth potential, driven by regulatory approvals and clinical interest.
• Asia-Pacific: Rapidly expanding markets with rising liver disease cases and increasing access to clinical research.

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Strategic Outlook and Business Implications

Companies investing in ursodiol-related R&D have opportunities to diversify indications, optimize formulations, and explore combination therapies. Strategic partnerships with academic institutions can accelerate clinical evaluation, underpin future approvals, and expand patient access.

Furthermore, navigating the regulatory landscape effectively, particularly in emerging markets, will be pivotal for market expansion. Patent management, aggressive marketing strategies, and leveraging real-world evidence can underpin sustained growth.

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Key Takeaways

• Clinical Development: Ongoing trials suggest potential new indications for ursodiol, especially in NAFLD and NASH, though conclusive data are pending.
• Market Landscape: The global ursodiol market remains sizable with growth fueled by expanding indications and increased liver disease prevalence.
• Future Growth: Anticipated CAGR of 4.5% until 2030 with opportunities tied to regulatory approvals, formulation innovations, and emerging markets.
• Competitive Edge: Manufacturers can leverage clinical data to differentiate formulations and expand therapeutic scope.
• Regulatory and Market Dynamics: Monitoring trial outcomes and regulatory responses will be critical for strategic decision-making.

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FAQs

1. What are the main current indications for ursodiol?
Ursodiol is primarily indicated for the dissolution of cholesterol gallstones and the treatment of primary biliary cholangitis. Its off-label use includes some cases of non-alcoholic fatty liver disease and cholestasis.

2. Are there ongoing clinical trials investigating new uses for ursodiol?
Yes, multiple trials are examining ursodiol in conditions like NAFLD, NASH, and pediatric cholestatic diseases, with some in early phases and preliminary promising data.

3. How does the market outlook for ursodiol compare to emerging bile acid therapies?
While ursodiol remains a pioneer in bile acid treatments, emerging therapies such as obeticholic acid are gaining favor in certain indications. Nonetheless, ursodiol benefits from extensive clinical experience, safety, and cost advantages.

4. What factors could accelerate the future growth of ursoIodiol’s market share?
Demonstrating efficacy in new indications through clinical trials and gaining regulatory approval for expanded uses are key factors. Formulation innovations and increased global access can also boost growth.

5. How might patent expiries impact the ursodiol market?
Patent expiries facilitate generic manufacturing, lowering prices and increasing accessibility, which can expand the market but may reduce profitability for brand-name drugs.

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References

1. [1] MarketsandMarkets. “Bile Acids Market by Type, Application, Region – Global Forecast to 2025.”
2. [2] ClinicalTrials.gov. “Ongoing Clinical Trials Involving Ursodiol,” 2023.
3. [3] Global Data. “Pharmaceutical Market Trends and Analysis.”
4. [4] WHO. “Global Burden of Liver Diseases,” 2022.
5. [5] Food and Drug Administration (FDA). “Approved Indications for Ursodiol.”

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Conclusion

Ursodiol continues to be a cornerstone therapy within hepatology, with a stable market driven by existing indications and promising clinical research supporting broader applications. Its growth trajectory will be shaped by ongoing trial outcomes, regulatory developments, and strategic positioning by pharmaceutical players. Business professionals should closely monitor these factors to harness emerging opportunities and maintain competitive advantage.