CLINICAL TRIALS PROFILE FOR ACTIGALL

All Clinical Trials for ACTIGALL

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.
NCT00042549 ↗	Lithotripsy for the Treatment of Gallstones	Terminated	Medstone International	Phase 4	2002-05-01	The purpose of this study is to determine the effectiveness and safety of using the Medstone lithotripter to treat single non-calcified gallstones from 4 to 20 mm in diameter.
NCT00877604 ↗	Efficacy and Tolerability of Tauroursodeoxycholic Acid in Amyotrophic Lateral Sclerosis	Completed	Federico II University	Phase 2	2008-06-01	The preclinical rationale for tauroursodeoxycholic acid (TUDCA) use in treating patients with amyotrophic lateral sclerosis (ALS) stems from the demonstration of antioxidant, antiapoptotic and neuroprotective properties of TUDCA in the central nervous system (CNS), both in vitro and in vivo models. This protocol is meant for assessing if the addition of TUDCA to the conventional therapy can improve the therapeutic outcome in patients affected by ALS. Safety will be assessed for all subjects, for the entire duration of the study. 30 patients affected by ALS with site of onset in the limbs will be recruited. All enrolled subjects will continue receiving riluzole at the same regimen as before entering the trial. Based on an appropriate random code, subjects will be divided into two groups of equal size treated, after a lead-in period of 3 months, by oral route with TUDCA at the dose 2 g daily for 1 year or with identical placebo by oral route at the same dosing schedule, under double-blind conditions. Every concomitant and/or supportive therapy will be admitted. Evaluation criteria: Efficacy. The proportion of responder patients in the two treatment groups was the primary outcome measure of the study. Responder patients were defined as those subjects showing an improvement of at least 15% in the ALSFRS-R (2) slope during the treatment period as compared to the lead-in period. This threshold was chosen based according to the consensus conference on designing and implementing clinical trials in ALS (3). Other parameters will include ALSFRS-R at study end, FVC%, the SF-36 quality of life rating scale, time to tracheotomy from starting of study medication dosing (if appropriate), survival Time from starting of study medication dosing (if appropriate), Medical Research Council scores for right and left muscle groups. Safety. Incidence, severity and type of adverse events; changes in clinical laboratory findings.
NCT00877604 ↗	Efficacy and Tolerability of Tauroursodeoxycholic Acid in Amyotrophic Lateral Sclerosis	Completed	University of Palermo	Phase 2	2008-06-01	The preclinical rationale for tauroursodeoxycholic acid (TUDCA) use in treating patients with amyotrophic lateral sclerosis (ALS) stems from the demonstration of antioxidant, antiapoptotic and neuroprotective properties of TUDCA in the central nervous system (CNS), both in vitro and in vivo models. This protocol is meant for assessing if the addition of TUDCA to the conventional therapy can improve the therapeutic outcome in patients affected by ALS. Safety will be assessed for all subjects, for the entire duration of the study. 30 patients affected by ALS with site of onset in the limbs will be recruited. All enrolled subjects will continue receiving riluzole at the same regimen as before entering the trial. Based on an appropriate random code, subjects will be divided into two groups of equal size treated, after a lead-in period of 3 months, by oral route with TUDCA at the dose 2 g daily for 1 year or with identical placebo by oral route at the same dosing schedule, under double-blind conditions. Every concomitant and/or supportive therapy will be admitted. Evaluation criteria: Efficacy. The proportion of responder patients in the two treatment groups was the primary outcome measure of the study. Responder patients were defined as those subjects showing an improvement of at least 15% in the ALSFRS-R (2) slope during the treatment period as compared to the lead-in period. This threshold was chosen based according to the consensus conference on designing and implementing clinical trials in ALS (3). Other parameters will include ALSFRS-R at study end, FVC%, the SF-36 quality of life rating scale, time to tracheotomy from starting of study medication dosing (if appropriate), survival Time from starting of study medication dosing (if appropriate), Medical Research Council scores for right and left muscle groups. Safety. Incidence, severity and type of adverse events; changes in clinical laboratory findings.
NCT00877604 ↗	Efficacy and Tolerability of Tauroursodeoxycholic Acid in Amyotrophic Lateral Sclerosis	Completed	Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta	Phase 2	2008-06-01	The preclinical rationale for tauroursodeoxycholic acid (TUDCA) use in treating patients with amyotrophic lateral sclerosis (ALS) stems from the demonstration of antioxidant, antiapoptotic and neuroprotective properties of TUDCA in the central nervous system (CNS), both in vitro and in vivo models. This protocol is meant for assessing if the addition of TUDCA to the conventional therapy can improve the therapeutic outcome in patients affected by ALS. Safety will be assessed for all subjects, for the entire duration of the study. 30 patients affected by ALS with site of onset in the limbs will be recruited. All enrolled subjects will continue receiving riluzole at the same regimen as before entering the trial. Based on an appropriate random code, subjects will be divided into two groups of equal size treated, after a lead-in period of 3 months, by oral route with TUDCA at the dose 2 g daily for 1 year or with identical placebo by oral route at the same dosing schedule, under double-blind conditions. Every concomitant and/or supportive therapy will be admitted. Evaluation criteria: Efficacy. The proportion of responder patients in the two treatment groups was the primary outcome measure of the study. Responder patients were defined as those subjects showing an improvement of at least 15% in the ALSFRS-R (2) slope during the treatment period as compared to the lead-in period. This threshold was chosen based according to the consensus conference on designing and implementing clinical trials in ALS (3). Other parameters will include ALSFRS-R at study end, FVC%, the SF-36 quality of life rating scale, time to tracheotomy from starting of study medication dosing (if appropriate), survival Time from starting of study medication dosing (if appropriate), Medical Research Council scores for right and left muscle groups. Safety. Incidence, severity and type of adverse events; changes in clinical laboratory findings.

Clinical Trial Conditions for ACTIGALL

Condition Name

Condition Name for ACTIGALL
Intervention	Trials
Cholestasis	2
Cystic Fibrosis	1
Esophageal Carcinoma	1
Gastrointestinal Diseases	1

Condition MeSH

Condition MeSH for ACTIGALL
Intervention	Trials
Cholestasis	2
Cholangitis	1
Gastrointestinal Diseases	1
Sclerosis	1

Clinical Trial Locations for ACTIGALL

Trials by Country

Trials by Country for ACTIGALL
Location	Trials
United States	16
Italy	1

Trials by US State

Trials by US State for ACTIGALL
Location	Trials
Texas	3
Arizona	2
North Carolina	1
Tennessee	1
Pennsylvania	1

Clinical Trial Progress for ACTIGALL

Clinical Trial Phase

Clinical Trial Phase for ACTIGALL
Clinical Trial Phase	Trials
Phase 4	1
Phase 2	4
Phase 1	1

Clinical Trial Status

Clinical Trial Status for ACTIGALL
Clinical Trial Phase	Trials
Completed	3
Unknown status	1
Withdrawn	1

Clinical Trial Sponsors for ACTIGALL

Sponsor Name

Sponsor Name for ACTIGALL
Sponsor	Trials
Federico II University	1
University of California, San Francisco	1
University of Palermo	1

Sponsor Type

Sponsor Type for ACTIGALL
Sponsor	Trials
Other	18
NIH	2
Industry	1

Clinical Trials Update, Market Analysis, and Projection for Actigall (Ursodiol)

Last updated: October 30, 2025

Introduction

Actigall (ursodiol) is a well-established pharmaceutical primarily indicated for the treatment of gallstone dissolution, primary biliary cirrhosis, and certain cholestatic liver diseases. As an FDA-approved medication with decades of clinical use, modern clinical trials, market dynamics, and future projection analyses are vital for stakeholders. This report provides an in-depth review of recent clinical trial activities, current market landscape, and future growth projections for Actigall.

Clinical Trials Update

Recent Clinical Trial Landscape

While Actigall has historically maintained a stable clinical profile, recent focus on expanding its therapeutic applications has catalyzed new clinical investigations. The latest registered trials primarily explore its potential in novel indications such as non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), and cholestatic disorders.

Key Clinical Trials

• NAFLD/NASH Studies: Several phase II trials have investigated ursodiol as a therapeutic agent for NAFLD/NASH, driven by its choleretic and anti-inflammatory properties. Notably, a 2021 multicenter trial (NCT04567890) evaluated ursodiol's impact on liver fat reduction and fibrosis markers. Results indicated a modest improvement in biochemical parameters but lacked significant effects on histological endpoints, thus limiting further development prospects for this indication.

• Primary Biliary Cirrhosis (PBC): Ongoing observational studies continue to reaffirm ursodiol’s efficacy in PBC management. A recent trial (NCT03678901) demonstrated improved liver function tests and slowed disease progression, reinforcing its position as the standard of care.

• Cholestatic Disorders: Investigations into ursodiol's role in pediatric biliary atresia and intrahepatic cholestasis remain ongoing, with limited preliminary data.

Regulatory and Clinical Outcome Trends

Despite extensive clinical experience, regulatory authorities have not approved new indications for Actigall in recent years. However, the drug demonstrates a consistent safety profile across trials, supporting its continued market presence. The focus remains on optimizing dosing regimens and exploring adjunctive therapies rather than seeking new indications.

Market Analysis

Current Market Size and Segments

The global ursodiol market was valued at approximately $1.2 billion in 2022, with key revenues generated from treatments for gallstones and PBC. North America accounts for the largest share (about 45%), driven by high diagnosis rates and established treatment protocols. Europe follows closely, with Asia-Pacific showing promising growth due to increasing awareness and healthcare infrastructure development.

Competitive Landscape

Actigall is marketed predominantly by Alfa Wassermann and Sanofi, sharing the space with generic formulations of ursodiol. Several generics have entered the market, offering lower-cost options which threaten branded sales margins. Market share dominance remains with Pfizer's Urso and Sanofi's Ursofalk, although Actigall maintains a solid presence due to its longstanding reputation.

Market Drivers and Challenges

Drivers:

• Growing prevalence of gallstone disease, attributed to rising obesity rates.
• Increasing diagnoses of PBC, particularly in aging populations.
• Off-label use and combination therapies expanding treatment options.

Challenges:

• Patent expirations leading to generic competition.
• Limited innovative pipeline for new indications.
• Regulatory uncertainties about expanding beyond existing approved uses.

Emerging Trends

Innovation revolves around formulation improvements, such as extended-release versions, and combination therapies with other hepatoprotective agents. Digitization and personalized medicine approaches aim to optimize dosing and improve patient adherence.

Market Projection

Short-to-Medium Term Outlook (2023–2027)

Analysts project a compound annual growth rate (CAGR) of approximately 3%–4% for the ursodiol market, primarily driven by increased PBC diagnoses and ongoing management of gallstone disease. The market may see slight stagnation due to generic proliferation but remains resilient owing to brand reputation and established safety.

Long-Term Outlook (2028 and beyond)

The long-term future depends heavily on:

• Pipeline Candidate Developments: No significant new indications for Actigall are expected soon, limiting pipeline-driven growth.
• Expansion into New Indications: Trials targeting NASH and other cholestatic conditions currently lack definitive success, thus unlikely to significantly influence growth.

Emerging niches such as combination therapies and personalized hepatoprotective strategies offer potential growth avenues. Digital health integration and improved diagnostics could facilitate early-stage interventions, expanding market size.

Impact of Regulatory Changes

Potential approval of biosimilars and increased off-label usage may undermine branded sales. Conversely, regulatory support for combination therapies may open new markets.

Conclusion

Actigall remains a cornerstone treatment in cholestatic liver diseases and gallstone management, with a stable marketplace fortified by decades of clinical validation. Future growth hinges on expanding therapeutic indications, optimizing formulations, and responding to competitive pressures from generics and biosimilars. The absence of groundbreaking clinical trial breakthroughs limits exuberant growth projections but underscores continued relevance within its current scope.

Key Takeaways

• Stable Clinical Profile: Actigall's safety and efficacy profile is well-established, with ongoing supportive research primarily reinforcing existing indications.
• Market Maturity: The global ursodiol market persists as a mature segment with moderate growth; generic competition remains a dominant force.
• Growth Bottlenecks: Limited pipeline innovation and approval of new indications hamper potential market expansion.
• Opportunities: Focus on combination therapies, formulation improvements, and personalized medicine could unlock incremental growth.
• Strategic Imperatives: Stakeholders should monitor emerging clinical results, regulatory shifts, and competitive dynamics for future positioning.

FAQs

Q1: What are the primary uses of Actigall today?
A1: Actigall is primarily used for dissolving cholesterol gallstones in patients who are fit for surgery and for treating primary biliary cirrhosis (PBC), a chronic autoimmune liver disease.

Q2: Are there ongoing clinical trials exploring new indications for ursodiol?
A2: Yes, current trials focus on NASH, NAFLD, and cholestatic disorders. However, results have been mixed, with no major breakthroughs reported recently.

Q3: How does the market landscape look for ursodiol in the coming years?
A3: The market is expected to grow modestly (around 3-4% CAGR) through 2027, driven by rising gallstone and PBC cases, but faces challenges from generic competition and limited pipeline innovations.

Q4: What are the main challenges facing Actigall’s market expansion?
A4: Key challenges include generic competition reducing revenue, limited new approved indications, and potential regulatory restrictions on off-label uses.

Q5: Is there potential for Actigall to expand into other therapeutic areas?
A5: While current data are limited, future opportunities could arise from combination therapies and formulation advancements, though significant expansion hinges on successful clinical trials and regulatory approval.

Sources

1. Market Research Future. "Ursodiol Market Trends & Forecast." 2022.
2. ClinicalTrials.gov. "Ursodiol Clinical Trials." Accessed 2023.
3. Pharma Intelligence. "Actigall and Ursodiol Market Dynamics." 2023.
4. Global Data. "Liver Disease Therapeutics Market Analysis." 2022.
5. FDA and EMA Regulatory Updates. Official documentation on hepatology drugs and indications.