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.
NCT00004748 ↗	Low-Dose Oral Methotrexate Versus Colchicine for Primary Biliary Cirrhosis	Completed	National Center for Research Resources (NCRR)	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
Gallstones	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
Washington	3
Virginia	3
Pennsylvania	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

Ursodiol: Clinical Trials Update, Market Analysis, and Projections

Last updated: February 20, 2026

What is the current status of clinical trials for ursodiol?

Ursodiol, also known as ursodeoxycholic acid, is approved primarily for the treatment of primary biliary cholangitis (PBC) and certain types of gallstones. Its pipeline development involves new formulations and indications, with a focus on improving efficacy and safety profiles.

As of 2023, there are no ongoing high-profile Phase III clinical trials for ursodiol in new indications listed on ClinicalTrials.gov. However, there are several studies in Phase II focused on using ursodiol for non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), and other liver-related conditions. These trials aim to evaluate optimal dosing, long-term safety, and efficacy enhancements.

A notable Phase II trial (NCT03904619) completed in 2022 assessed ursodiol in NASH patients, showing modest improvements in liver enzymes and fibrosis markers. No new Phase III trials have started as of mid-2023.

How is ursodiol positioned in current therapeutic markets?

Ursodiol remains the standard of care for PBC, with approximately 70% of patients responding favorably according to published data[1]. It is also prescribed for gallstones, especially when symptomatic or complicating other conditions.

The drug’s market is mature, with generic options available since patent expiration in most regions by 2000s. Its revenues are stable, estimated at approximately $400 million annually globally, mainly in North America and Europe.

Emerging markets have seen increased usage, driven by healthcare expansion and the increasing prevalence of liver diseases.

What are the current market trends influencing the future of ursodiol?

• Generic Competition: Market saturation has led to price pressure, limiting revenue growth.
• New Indications: Research into NASH and other liver fibrosis conditions could expand its use, but clinical success remains inconsistent.
• Formulation Innovation: Pills with extended-release or combination therapies aim to improve patient adherence and treatment outcomes.
• Regulatory Landscape:Approval for new uses requires substantial clinical evidence. Regulatory agencies are scrutinizing off-label applications more tightly, influencing market perception.

What are the projections for ursodiol’s market over the next five years?

Based on current trends and clinical pipeline insights, the following projections are made:

Year	Estimated Global Market Size	Major Drivers	Risks
2023	$400 million	Steady demand for PBC, gallstone treatment, generic sales	Limited growth potential, patent expiry effects
2024	$410 million	Incremental uptake in NAFLD/NASH research	Clinical trial failures for new indications
2025	$420 million	Adoption of new formulations, early Phase II successes	Regulatory hurdles for expanded indications
2026	$430 million	Emerging markets investing more in liver disease treatments	Competition from other emerging therapies
2027	$440 million	Increased awareness, potential new approved indications	Market saturation, slow regulatory approvals

The compound annual growth rate (CAGR) for the next five years is forecasted at approximately 1.5%, primarily driven by increased demand in emerging markets and incremental growth in established indications.

What are the key barriers and opportunities?

Barriers

• Low margin due to generic presence
• Limited pipeline for new indications
• Regulatory challenges for off-label expansion
• Competition from novel therapeutic approaches in liver disease

Opportunities

• Development of combination therapies
• Expanded use if future trials demonstrate strong efficacy in NASH or fibrosis
• Regulatory approvals for additional indications
• Market entry into underserved regions with increasing liver disease prevalence

Summary

Ursodiol continues to serve as a cornerstone treatment for PBC and gallstones. Its market remains stable but mature, with limited growth driven by generic competition. Clinical trial activity focuses on exploring new liver disease indications, with mixed results expected due to the complex pathophysiology of conditions like NASH.

Market projections suggest a modest growth trajectory, emphasizing incremental gains rather than disruptive expansion.

Key Takeaways

• No current Phase III trials; ongoing research centers on NASH and liver fibrosis.
• Market size remains around $400 million annually, with limited growth prospects.
• Development of formulations and targeted use expansions offer future opportunities.
• Barriers include market saturation and regulatory scrutiny.
• The future of ursodiol hinges on clinical success in emerging indications and strategic formulation innovations.

Frequently Asked Questions

1. Can ursodiol be used for conditions beyond its current approvals?
   Yes, ongoing trials are testing its efficacy in NASH and other liver diseases, but no new approved indications have emerged yet.

2. What is the primary revenue driver for ursodiol?
   Treatment of primary biliary cholangitis and gallstones remains the main source.

3. Are there significant patent protections still in place?
   Most patents expired by the early 2000s; current formulations are generally off patent, favoring generics.

4. How does ursodiol compare to alternative therapies in liver disease?
   In PBC, it is the standard of care; in other conditions like NASH, competing novel agents show promising results but lack widespread approval.

5. What is the outlook for new formulations of ursodiol?
   Innovations focusing on slow-release tablets and combination therapies aim to improve compliance and therapeutic outcomes, potentially supporting market share stability.

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References

[1] Lindor, K.D., et al. (2019). "Ursodiol Treatment and Outcomes in Patients With Primary Biliary Cholangitis." Gastroenterology, 157(3), 754-764.