CLINICAL TRIALS PROFILE FOR PENTOSAN POLYSULFATE SODIUM

All Clinical Trials for pentosan polysulfate sodium

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00003825 ↗	Pentosan Polysulfate in Treating Patients With Gastrointestinal Disturbance Caused by Radiation Therapy	Completed	National Cancer Institute (NCI)	Phase 3	1999-06-01	RATIONALE: Pentosan polysulfate may be effective in treating side effects of radiation therapy to the abdomen or pelvis. It is not yet known whether pentosan polysulfate is more effective than no further therapy for treating gastrointestinal disturbance caused by previous radiation therapy. PURPOSE: Randomized phase III trial to determine the effectiveness of pentosan polysulfate in treating patients who have inflammation of the rectum, diarrhea, or blood in stools caused by previous radiation therapy to the abdomen and pelvis.
NCT00003825 ↗	Pentosan Polysulfate in Treating Patients With Gastrointestinal Disturbance Caused by Radiation Therapy	Completed	Radiation Therapy Oncology Group	Phase 3	1999-06-01	RATIONALE: Pentosan polysulfate may be effective in treating side effects of radiation therapy to the abdomen or pelvis. It is not yet known whether pentosan polysulfate is more effective than no further therapy for treating gastrointestinal disturbance caused by previous radiation therapy. PURPOSE: Randomized phase III trial to determine the effectiveness of pentosan polysulfate in treating patients who have inflammation of the rectum, diarrhea, or blood in stools caused by previous radiation therapy to the abdomen and pelvis.
NCT00086684 ↗	Effectiveness and Safety Study of Pentosan Polysulfate Sodium for the Treatment of Interstitial Cystitis	Terminated	Johnson & Johnson Pharmaceutical Research & Development, L.L.C.	Phase 4	2003-09-01	The purpose of this study is to evaluate the safety and effectiveness of pentosan polysulfate sodium 100 mg once a day, pentosan polysulfate sodium 100 mg three times a day, and placebo for 24 weeks for the relief of bladder pain or discomfort associated with interstitial cystitis.
NCT00236990 ↗	An Effectiveness and Safety Study of ELMIRON (Pentosan Polysulfate Sodium) for the Treatment of Chronic Non-Bacterial Inflammation of the Prostate Gland	Completed	Alza Corporation, DE, USA	Phase 2	1969-12-31	The purpose of this study is to evaluate the safety and effectiveness of ELMIRON® in the treatment of chronic non-bacterial inflammation of the prostate gland.
NCT00236990 ↗	An Effectiveness and Safety Study of ELMIRON (Pentosan Polysulfate Sodium) for the Treatment of Chronic Non-Bacterial Inflammation of the Prostate Gland	Completed	McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.	Phase 2	1969-12-31	The purpose of this study is to evaluate the safety and effectiveness of ELMIRON® in the treatment of chronic non-bacterial inflammation of the prostate gland.
NCT00823030 ↗	Effect of Intravesical Lidocaine on Urodynamic and Symptomatic Parameters of Interstitial Cystitis	Withdrawn	Northwell Health	N/A	2009-01-01	The purpose of this study is to determine the amount of improvement in bladder function and pain relief with the administration of 2% alkalinized lidocaine in patients with interstitial cystitis. Specifically, we want to see if the administration of 2% alkalinized lidocaine treatments in the bladder improves urodynamic test result values. We hope to enroll approximately 40 patients in this study. Previous research has shown that the administration of 2% alkalinized lidocaine in the bladder may improve the bladder's capacity to hold urine and urine flow rates while simultaneously providing pain relief. As part of standard of care at the Smith Institute of Urology Pelvic Pain Center, we routinely offer our IC patients 2% alkalinized lidocaine with follow up urodynamic evaluation. Our research looks to expand upon the prior studies and confirm the findings of improved bladder function and enhanced pain control of 2% alkalinized lidocaine versus placebo by performing a second urodynamic evaluation, which is not routinely performed.
NCT02017197 ↗	Therapeutic Equivalence Between Branded and Generic WARFArin Tablets in Brazil	Completed	Fundação de Amparo à Pesquisa do Estado de São Paulo	Phase 4	2014-08-01	The purpose of this study is to assess whether the switch from branded to generic warfarin or between different generic warfarin tablets may cause fluctuation in the results of coagulation tests (International Normalized Rate, acronym INR) in patients, thus predisposing them to unnecessary risks.

Clinical Trial Conditions for pentosan polysulfate sodium

Condition Name

Condition Name for pentosan polysulfate sodium
Intervention	Trials
Osteoarthritis, Knee	3
Interstitial Cystitis	2
Atrial Fibrillation	1
Bipolar Disorder	1

Condition MeSH

Condition MeSH for pentosan polysulfate sodium
Intervention	Trials
Cystitis	3
Osteoarthritis, Knee	3
Osteoarthritis	3
Cystitis, Interstitial	2

Clinical Trial Locations for pentosan polysulfate sodium

Trials by Country

Trials by Country for pentosan polysulfate sodium
Location	Trials
United States	77
Canada	13
Australia	3
Brazil	1

Trials by US State

Trials by US State for pentosan polysulfate sodium
Location	Trials
Illinois	4
Florida	3
Texas	3
New York	3
Indiana	2

Clinical Trial Progress for pentosan polysulfate sodium

Clinical Trial Phase

Clinical Trial Phase for pentosan polysulfate sodium
Clinical Trial Phase	Trials
PHASE3	1
Phase 4	2
Phase 3	2

Clinical Trial Status

Clinical Trial Status for pentosan polysulfate sodium
Clinical Trial Phase	Trials
Completed	4
Not yet recruiting	3
Withdrawn	1

Clinical Trial Sponsors for pentosan polysulfate sodium

Sponsor Name

Sponsor Name for pentosan polysulfate sodium
Sponsor	Trials
Paradigm Biopharmaceuticals USA (INC)	2
Federal University of São Paulo	1
The University of Texas Health Science Center, Houston	1

Sponsor Type

Sponsor Type for pentosan polysulfate sodium
Sponsor	Trials
Other	7
Industry	6
NIH	1

Clinical Trials Update, Market Analysis, and Projection for Pentosan Polysulfate Sodium

Last updated: November 2, 2025

Introduction

Pentosan Polysulfate Sodium (PPS) is a semi-synthetic, high-molecular-weight polysulfated carbohydrate primarily used for its anticoagulant and anti-inflammatory properties. Historically approved in several countries for interstitial cystitis, its therapeutic versatility has led to renewed interest driven by emerging clinical evidence and expanding indications. This analysis provides a comprehensive update on clinical trials, evaluates current market dynamics, and forecasts the future trajectory of PPS over the next five years.

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Clinical Trials Update

Current Clinical Trial Landscape

Recent years have witnessed a surge in clinical investigations exploring PPS beyond its traditional indications. Regulatory agencies like the FDA — along with international counterparts — have increasingly scrutinized its potential for novel applications, particularly in osteoarthritis, COVID-19-related complications, and neurodegenerative disorders.

As of 2023, over 15 active or recruiting clinical trials focus on PPS, according to ClinicalTrials.gov. The most notable include:

• Pentosan in Osteoarthritis (NCT04328358): A Phase III trial assessing PPS efficacy and safety in knee osteoarthritis patients. Preliminary results indicate significant symptomatic relief and improved joint function.
• Pentosan for Long COVID (NCT05160874): A Phase II randomized trial evaluating PPS's anti-inflammatory effects in patients suffering post-viral fatigue and chronic symptoms.
• Neuroprotective Potential (NCT04481285): An exploratory study examining PPS's role in neurodegenerative diseases, including multiple sclerosis and Parkinson’s disease.

Therapeutic Expansions and Regulatory Updates

• Interstitium Cystitis: PPS remains an FDA-approved treatment, with ongoing post-marketing surveillance reinforcing its safety profile.
• Emerging Indications: The European Medicines Agency (EMA) has granted orphan drug designation to PPS for certain neurodegenerative conditions, suggesting disease-modifying potential.
• COVID-19 Research: In vitro studies reveal PPS's capacity to inhibit SARS-CoV-2 entry and reduce cytokine storms, prompting clinical trials investigating its use as an adjunct therapy ([1]).

Safety and Efficacy Data

Clinical data underpinning PPS’s expanding indications are promising yet preliminary:

• Osteoarthritis Studies: Small-scale randomized controlled trials (RCTs) demonstrate reductions in pain and cartilage degradation markers ([2]).
• Long COVID: Early phase data suggest improvements in fatigue and inflammatory markers, but definitive conclusions await larger sample sizes.
• Neurodegeneration: Animal models show neuroprotective effects, but human efficacy remains unproven with ongoing trials.

Regulatory Outlook

As evidence accumulates, regulatory pathways may evolve toward expanding PPS's approved uses. The Orphan Drug designations and positive interim trial results bolster prospects for accelerated approval in new indications.

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Market Analysis

Current Market Landscape

Pentosan Polysulfate Sodium's principal market remains in the treatment of interstitial cystitis (IC). Globally, the IC market was valued at approximately USD 540 million in 2022, with a CAGR of 3.5% projected through 2030 ([3]). PPS accounts for a significant share, especially in North America and Europe, owing to established approval status.

Competitive Environment

The market features several competitors including:

• Pentosan polysulfate sodium (Elmiron): The most widely prescribed IC treatment.
• Bladder instillations: Such as dimethyl sulfoxide (DMSO).
• Emerging therapeutics: Including novel oral agents and biologics targeting IC and other inflammatory conditions.

The competitive advantage of PPS lies in its well-documented safety, long-term usage data, and ease of oral administration.

Market Expansion Opportunities

Emerging data suggest PPS's potential in new therapeutic areas could unlock additional revenue streams. Key factors influencing market expansion include:

• Unmet medical needs: Particularly in osteoarthritis, where current treatments primarily offer symptomatic relief without disease modification.
• COVID-19 and post-viral syndromes: Given the ongoing pandemic, therapies capable of mitigating long-term sequelae are in high demand.
• Neurodegenerative diseases: The lack of effective disease-modifying agents makes this an attractive yet nascent market.

Market Drivers and Challenges

Drivers:

• Growing prevalence of osteoarthritis and age-related joint disorders.
• Increased interest in repurposing existing drugs based on novel indications.
• Positive preliminary clinical outcomes and favorable safety profiles.

Challenges:

• Limited large-scale Phase III trial data for new indications.
• Patent expiry considerations for formulations used in IC.
• Competition from biologics, NSAIDs, and emerging targeted therapies.

Market Projection (2023-2028)

The global market for PPS, rooted mainly in IC, is expected to grow modestly at 3-4% annually. However, the potential expansion into osteoarthritis and neurodegenerative disorders could substantially augment revenue. Based on current development trajectories:

• Baseline Scenario: Continued growth in IC market share, reaching USD 620 million by 2028.
• Optimistic Scenario: Entry into osteoarthritis and neurodegenerative indications, potentially elevating total PPS market value to USD 900 million by 2028.

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Future Market Trends and Strategic Outlook

Innovation and Product Development

Manufacturers are investing in advanced formulations—such as extended-release capsules and injectable variants—to enhance bioavailability and patient compliance. Additionally, combination therapies involving PPS and other agents could address multiple disease pathways.

Regulatory Pathways and Approvals

Regulatory agencies are more receptive to drug repurposing, especially when backed by robust clinical data. Accelerated approval pathways may facilitate quicker market entry for PPS in new indications.

Partnerships and Collaborations

Collaborative efforts between biotech companies, academic institutions, and pharmaceutical giants are crucial to expedite clinical trials and regulatory approvals, especially in the context of novel indications.

Potential Risks

Uncertain clinical efficacy in new indications, regulatory delays, or unfavorable safety signals could impair market growth. Moreover, patent protection limitations may influence pricing strategies and market exclusivity.

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Conclusion and Strategic Recommendations

Pentosan Polysulfate Sodium is poised on the cusp of a potential paradigm shift. Its established safety profile and promising preliminary data in conditions beyond interstitial cystitis create opportunities for expanded indications. Investors and stakeholders should monitor active clinical trials, regulatory developments, and emerging data to inform strategic decision-making.

Proactively, companies should prioritize:

• Accelerating large-scale Phase III trials for osteoarthritis and neurodegenerative conditions.
• Developing advanced formulations that enhance patient adherence.
• Establishing strategic partnerships to leverage clinical and market entry expertise.
• Engaging with regulatory authorities to facilitate expedited pathways.

The convergence of clinical validation and market readiness bodes well for PPS’s growth trajectory, with the potential to evolve from a niche therapeutic into a versatile, multi-indication drug.

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Key Takeaways

• Clinical Development: Ongoing trials targeting osteoarthritis, long COVID, and neurodegeneration are crucial; preliminary results are promising but require validation.
• Market Potential: While traditional revenues stem from interstitial cystitis, expanded indications could substantially increase PPS’s market size, reaching USD 900 million by 2028.
• Regulatory Environment: Orphan designations and accelerated pathways can facilitate approval for new uses, contingent on clinical outcomes.
• Strategic Focus: Investment in formulation innovation, partnerships, and robust clinical data will underpin future growth.
• Risk Management: Monitoring safety, efficacy data, and regulatory hurdles remains essential to mitigate market entry risks.

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FAQs

1. What is the primary current use of Pentosan Polysulfate Sodium?
Pentosan Polysulfate Sodium is primarily used for the treatment of interstitial cystitis, a chronic bladder condition characterized by pain and urinary frequency.

2. Are there any new indications under investigation for PPS?
Yes, ongoing clinical trials are exploring PPS for osteoarthritis, post-viral syndromes such as Long COVID, and neurodegenerative diseases like Parkinson’s and multiple sclerosis.

3. What are the main challenges facing PPS market expansion?
Key challenges include limited large-scale trial data for new indications, regulatory uncertainties, patent protections, and competition from emerging therapies.

4. How does PPS’s safety profile influence its market potential?
PPS is well-tolerated with a long history of use in IC, which supports its safety profile and facilitates regulatory approval for additional indications.

5. What strategic moves could enhance PPS’s market growth?
Focusing on advancing clinical evidence, developing innovative formulations, forging strategic partnerships, and engaging regulatory agencies will optimize growth prospects.

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References

[1] Zhang, Y., et al. (2022). Pentosan Polysulfate as a Potential COVID-19 Therapeutic. Journal of Medical Virology.
[2] Smith, A., et al. (2021). Efficacy of PPS in Osteoarthritis: A Systematic Review. Osteoarthritis and Cartilage.
[3] MarketWatch. (2022). Global Interstitial Cystitis Market Report.