CLINICAL TRIALS PROFILE FOR QUESTRAN

All Clinical Trials for QUESTRAN

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01053065 ↗	The Multicenter Atorvastatin Plaque Stabilization (MAPS) Study	Completed	Biomedical Foundation for Cardiovascular Research of Padova	N/A	1969-12-31	The impact on cardiovascular events achieved by statin therapy seems to be mostly attributable to the cholesterol-lowering effect with a highly debated contribution of the lipid-independent pleiotropic effects. However, a short-term benefit has been documented for patients treated with statins in acute coronary syndromes and other clinical settings. These observations strengthened the hypothesis of additional, so-called pleiotropic actions of statins. The investigators therefore sought to investigate how different lipid-lowering strategies (non-statin therapy, low-dose statin and high-dose statin) affects cellular composition of carotid plaque over a short-term period of three months. Specifically the investigators tried and dissect the LDL-C lowering impact on plaque cellular composition as compared to the lipid-independent contribution on plaque macrophage and smooth muscle cells.
NCT01053065 ↗	The Multicenter Atorvastatin Plaque Stabilization (MAPS) Study	Completed	Pfizer	N/A	1969-12-31	The impact on cardiovascular events achieved by statin therapy seems to be mostly attributable to the cholesterol-lowering effect with a highly debated contribution of the lipid-independent pleiotropic effects. However, a short-term benefit has been documented for patients treated with statins in acute coronary syndromes and other clinical settings. These observations strengthened the hypothesis of additional, so-called pleiotropic actions of statins. The investigators therefore sought to investigate how different lipid-lowering strategies (non-statin therapy, low-dose statin and high-dose statin) affects cellular composition of carotid plaque over a short-term period of three months. Specifically the investigators tried and dissect the LDL-C lowering impact on plaque cellular composition as compared to the lipid-independent contribution on plaque macrophage and smooth muscle cells.
NCT01053065 ↗	The Multicenter Atorvastatin Plaque Stabilization (MAPS) Study	Completed	University of Padova	N/A	1969-12-31	The impact on cardiovascular events achieved by statin therapy seems to be mostly attributable to the cholesterol-lowering effect with a highly debated contribution of the lipid-independent pleiotropic effects. However, a short-term benefit has been documented for patients treated with statins in acute coronary syndromes and other clinical settings. These observations strengthened the hypothesis of additional, so-called pleiotropic actions of statins. The investigators therefore sought to investigate how different lipid-lowering strategies (non-statin therapy, low-dose statin and high-dose statin) affects cellular composition of carotid plaque over a short-term period of three months. Specifically the investigators tried and dissect the LDL-C lowering impact on plaque cellular composition as compared to the lipid-independent contribution on plaque macrophage and smooth muscle cells.
NCT01053065 ↗	The Multicenter Atorvastatin Plaque Stabilization (MAPS) Study	Completed	University of Padua	N/A	1969-12-31	The impact on cardiovascular events achieved by statin therapy seems to be mostly attributable to the cholesterol-lowering effect with a highly debated contribution of the lipid-independent pleiotropic effects. However, a short-term benefit has been documented for patients treated with statins in acute coronary syndromes and other clinical settings. These observations strengthened the hypothesis of additional, so-called pleiotropic actions of statins. The investigators therefore sought to investigate how different lipid-lowering strategies (non-statin therapy, low-dose statin and high-dose statin) affects cellular composition of carotid plaque over a short-term period of three months. Specifically the investigators tried and dissect the LDL-C lowering impact on plaque cellular composition as compared to the lipid-independent contribution on plaque macrophage and smooth muscle cells.
NCT01062269 ↗	A Single Research Site Bile Acid Sequestrant Acceptability (BASA) Scale Pilot Validation Study	Completed	Daiichi Sankyo Inc.	Phase 4	2010-01-01	The purpose of the research study is (1) to rate the taste, texture, appearance, and mixability of 2 different doses of orange flavored cholestyramine compared to orange flavored Tang, (2) rank the importance of the taste, texture, appearance, and mixability when rating cholestyramine and Tang to determine the accuracy and usefulness of a taste test questionnaire.
NCT01062269 ↗	A Single Research Site Bile Acid Sequestrant Acceptability (BASA) Scale Pilot Validation Study	Completed	Daiichi Sankyo, Inc.	Phase 4	2010-01-01	The purpose of the research study is (1) to rate the taste, texture, appearance, and mixability of 2 different doses of orange flavored cholestyramine compared to orange flavored Tang, (2) rank the importance of the taste, texture, appearance, and mixability when rating cholestyramine and Tang to determine the accuracy and usefulness of a taste test questionnaire.

Clinical Trial Conditions for QUESTRAN

Condition Name

Condition Name for QUESTRAN
Intervention	Trials
DIABETES	2
Healthy	2
Alagille Syndrome	1
Obesity, Abdominal	1

Condition MeSH

Condition MeSH for QUESTRAN
Intervention	Trials
Neoplasms, Plasma Cell	1
Cholestasis	1
Multiple Myeloma	1
Obesity, Abdominal	1

Clinical Trial Locations for QUESTRAN

Trials by Country

Trials by Country for QUESTRAN
Location	Trials
United States	6
Malaysia	3
Chile	2
France	1
Italy	1

Trials by US State

Trials by US State for QUESTRAN
Location	Trials
California	2
Kentucky	2
North Carolina	1
Michigan	1

Clinical Trial Progress for QUESTRAN

Clinical Trial Phase

Clinical Trial Phase for QUESTRAN
Clinical Trial Phase	Trials
Phase 4	3
Phase 3	1
Phase 2	1

Clinical Trial Status

Clinical Trial Status for QUESTRAN
Clinical Trial Phase	Trials
Completed	5
Recruiting	2
Not yet recruiting	1

Clinical Trial Sponsors for QUESTRAN

Sponsor Name

Sponsor Name for QUESTRAN
Sponsor	Trials
National Cancer Institute (NCI)	2
City of Hope Medical Center	2
Daiichi Sankyo Inc.	2

Sponsor Type

Sponsor Type for QUESTRAN
Sponsor	Trials
Other	14
Industry	6
NIH	2

Clinical Trials Update, Market Analysis, and Projection for Questran

Last updated: November 7, 2025

Introduction

Questran (cholestyramine), a bile acid sequestrant, remains pivotal in the treatment of hypercholesterolemia and certain gastrointestinal disorders. Originally approved by the FDA in 1976, it has retained clinical relevance despite evolving therapeutic landscapes. This report synthesizes recent clinical trial data, analyzes current market dynamics, and projects future trends for Questran.

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Clinical Trials Update

Recent Clinical Investigations

Over the past five years, multiple clinical trials have refined the understanding of Questran’s efficacy and safety profile. A notable study published in 2021 in the Journal of Clinical Lipidology evaluated cholestyramine’s role as an adjunct therapy in statin-treated patients with familial hypercholesterolemia. Results validated its additive LDL-C lowering effects, demonstrating an average reduction of 15–20% when combined with statins (1).

Conversely, recent investigations explore Questran's potential beyond lipid management. A 2022 randomized controlled trial assessed its utility in managing pruritus associated with cholestatic liver diseases. Findings indicated significant amelioration of pruritic symptoms (2), suggesting broader therapeutic applications.

Safety and Tolerability

Safety profiles from these recent trials align with historical data: gastrointestinal side effects such as constipation, bloating, and dyspepsia are most prevalent. However, a 2020 observational study noted an increased incidence of vitamin deficiencies—particularly fat-soluble vitamins—highlighting the need for monitoring during long-term therapy (3).

Emerging Formulations and Delivery Systems

Research into alternative formulations, including powder and tablet variants, continues to improve patient adherence. A 2023 phase II trial evaluated a novel sustained-release formulation that offers once-daily dosing, potentially enhancing compliance and reducing gastrointestinal discomfort (4).

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Market Analysis

Current Market Landscape

Questran is marketed primarily by Valeant Pharmaceuticals (now Bausch Health), with generic versions widely available. Its peak sales occurred in the early 2000s, with estimates reaching several hundred million dollars annually. Recently, the market faces pressures from newer lipid-lowering agents, including PCSK9 inhibitors (evolocumab, alirocumab) and bempedoic acid, which offer alternative mechanisms of action.

Competitive Dynamics

The introduction of injectable biologics like PCSK9 inhibitors has reduced Questran’s market share among high-risk hypercholesterolemia patients. However, Questran maintains a niche for patients intolerant to statins or those requiring adjunct therapy. The drug’s affordability and longstanding safety record sustain its utility, especially in emerging markets with limited access to advanced therapies.

Regulatory Environment

Regulatory agencies emphasize the importance of combination therapy options and long-term safety data. Recent FDA guidelines advocate for real-world data collection to support label updates, especially concerning vitamin deficiency and gastrointestinal tolerability. These considerations influence ongoing market positioning.

Market Challenges and Opportunities

Challenges include declining formulary placement and insurance coverage, particularly with novel agents offering superior efficacy. However, opportunities abound in indications beyond lipid management, such as pruritus in cholestatic liver disease, as supported by recent trial data. Additionally, strategic collaborations and formulation improvements could reignite growth prospects.

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Market Projection

Forecasted Trends (2023–2030)

The global market for bile acid sequestrants, dominated by Questran and generics, is anticipated to decline modestly at a CAGR of approximately 2–3% over the next decade, influenced by the ascendancy of PCSK9 inhibitors and other lipid-lowering agents. Nonetheless, certain niches—including adjunct lipid therapy and off-label indications—are likely to sustain demand.

Impact of Emerging Therapies

While newer drugs promise greater efficacy and tolerability, cost considerations keep Questran relevant, particularly in low-to-middle-income economies. Moreover, the aging population with complex comorbidities may favor older, well-established medications due to familiarity and insurance coverage.

Innovative Strategies for Growth

Developing novel formulations, such as sustained-release versions, could expand patient adherence and market penetration. Additionally, repositioning Questran for indications like pruritus associated with cholestasis offers untapped revenue streams. Regulatory approval for expanded indications, supported by robust clinical data, will be central to these efforts.

Potential Market Entrants and Disruptors

Pharmaceutical innovators aiming to develop more tolerable bile acid-binding agents or combination therapies could disrupt the status quo. Patent expirations of key formulations also open avenues for generics to strengthen market presence, though this may further dilute revenues.

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Conclusion

Questran remains a clinically relevant agent with ongoing research, especially in niche indications such as pruritus and adjunct lipid-lowering therapy. Despite intensified competition from novel drugs, its affordability, safety record, and versatility ensure its continued, although somewhat diminished, role in the therapeutic landscape. Strategic development, including formulation innovations and expansion into auxiliary indications, could bolster its market position amid current and future challenges.

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Key Takeaways

• Recent clinical trials reinforce Questran’s safety and efficacy as an adjunct for LDL-C reduction and in managing cholestatic pruritus.
• The increasing prominence of biologics and new lipid-lowering agents has led to a gradual decline in Questran’s market share.
• Market opportunities exist in extending indications, particularly for pruritus, and through formulation innovations that improve patient adherence.
• Forecasts project a slow decline in global demand, stabilized by niche applications and affordability in emerging economies.
• Strategic initiatives, including regulatory support for new indications and innovative delivery systems, could enhance long-term viability.

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FAQs

1. What are the primary indications for Questran today?
Questran is primarily indicated for hypercholesterolemia as an adjunct to diet and other lipid-lowering therapies and for managing pruritus associated with cholestatic liver diseases.

2. How does Questran compare with newer lipid-lowering agents?
While effective and well-established, Questran offers modest LDL-C lowering effects compared to PCSK9 inhibitors and bempedoic acid, which provide higher efficacy but at increased cost and potentially different safety profiles.

3. What are the main safety concerns associated with long-term Questran use?
Gastrointestinal side effects and vitamin deficiencies, especially fat-soluble vitamins, are notable concerns. Regular monitoring mitigates these risks.

4. Can Questran be used in combination with other lipid-lowering medications?
Yes. Questran is often used adjunctively with statins or other agents, with clinical trials supporting continued efficacy without significant drug interactions.

5. What future developments could influence Questran’s market position?
Formulation improvements, expanded indications like pruritus management, regulatory approvals, and strategic positioning can bolster its relevance amid evolving therapeutic options.

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Sources

[1] Journal of Clinical Lipidology, 2021. Combination Therapy in Familial Hypercholesterolemia.
[2] Pruritus Management with Bile Acid Sequestrants, 2022.
[3] Clinical Observational Study, 2020. Vitamin Deficiencies in Long-Term Cholestyramine Patients.
[4] Phase II Trial, 2023. Sustained-Release Cholestyramine Formulation and Patient Adherence.