CLINICAL TRIALS PROFILE FOR ILOPROST

All Clinical Trials for iloprost

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00004786 ↗	Phase III Randomized, Double-Blind, Placebo-Controlled Study of Oral Iloprost for Raynaud's Phenomenon Secondary to Systemic Sclerosis	Completed	University of Pittsburgh	Phase 3	1995-12-01	OBJECTIVES: I. Evaluate the safety and efficacy of oral iloprost, a prostacyclin analog, in patients with Raynaud's phenomenon secondary to systemic sclerosis.
NCT00004786 ↗	Phase III Randomized, Double-Blind, Placebo-Controlled Study of Oral Iloprost for Raynaud's Phenomenon Secondary to Systemic Sclerosis	Completed	National Center for Research Resources (NCRR)	Phase 3	1995-12-01	OBJECTIVES: I. Evaluate the safety and efficacy of oral iloprost, a prostacyclin analog, in patients with Raynaud's phenomenon secondary to systemic sclerosis.
NCT00084409 ↗	Iloprost in Preventing Lung Cancer in Patients at High Risk for This Disease	Completed	National Cancer Institute (NCI)	Phase 2	2001-11-01	RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. Iloprost may be effective in preventing lung cancer. PURPOSE: This randomized phase II trial is studying how well iloprost works in preventing lung cancer in patients who are at high risk for this disease.
NCT00084409 ↗	Iloprost in Preventing Lung Cancer in Patients at High Risk for This Disease	Completed	University of Colorado, Denver	Phase 2	2001-11-01	RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. Iloprost may be effective in preventing lung cancer. PURPOSE: This randomized phase II trial is studying how well iloprost works in preventing lung cancer in patients who are at high risk for this disease.
NCT00086463 ↗	Trial of Iloprost Inhaled Solution as Add-On Therapy With Bosentan in Subjects With Pulmonary Arterial Hypertension (PAH)	Completed	Actelion	Phase 2	2004-06-01	The purpose of this study is to determine the safety and efficacy of Iloprost in subjects that have Pulmonary Arterial Hypertension who are concurrently taking bosentan (Tracleer TM).

Clinical Trial Conditions for iloprost

Condition Name

Condition Name for iloprost
Intervention	Trials
Pulmonary Hypertension	23
Pulmonary Arterial Hypertension	22
Hypertension, Pulmonary	8
Chronic Obstructive Pulmonary Disease	6

Condition MeSH

Condition MeSH for iloprost
Intervention	Trials
Hypertension	54
Hypertension, Pulmonary	37
Pulmonary Arterial Hypertension	31
Familial Primary Pulmonary Hypertension	28

Clinical Trial Locations for iloprost

Trials by Country

Trials by Country for iloprost
Location	Trials
United States	217
Germany	43
China	14
Japan	11
France	8

Trials by US State

Trials by US State for iloprost
Location	Trials
California	16
Pennsylvania	13
New York	12
Texas	12
Illinois	11

Clinical Trial Progress for iloprost

Clinical Trial Phase

Clinical Trial Phase for iloprost
Clinical Trial Phase	Trials
PHASE2	1
PHASE1	2
Phase 4	13

Clinical Trial Status

Clinical Trial Status for iloprost
Clinical Trial Phase	Trials
Completed	54
Terminated	14
Unknown status	11

Clinical Trial Sponsors for iloprost

Sponsor Name

Sponsor Name for iloprost
Sponsor	Trials
Actelion	17
Bayer	16
Yonsei University	5

Sponsor Type

Sponsor Type for iloprost
Sponsor	Trials
Other	91
Industry	47
NIH	4

Clinical Trials Update, Market Analysis, and Projection for Iloprost

Last updated: October 29, 2025

Introduction

Iloprost, a synthetic analog of prostacyclin (PGI₂), is a potent vasodilator with antiplatelet properties, primarily used in the management of pulmonary arterial hypertension (PAH) and other vascular disorders. Approved initially in several countries during the 1990s, Iloprost's evolving clinical profile and market potential merit a comprehensive review. This article synthesizes recent clinical trial developments, analyzes current market dynamics, and projects future trends for Iloprost.

Clinical Trials Update

Recent and Ongoing Clinical Research

The clinical landscape for Iloprost is characterized by a combination of established indications and exploratory research into broader therapeutic applications. Notable recent updates include:

• Paediatric and Pulmonary Hypertension Trials:
  Trials such as the phase III study (NCT number withheld for proprietary reasons) analyzed inhaled Iloprost's efficacy and safety in pediatric PAH patients. Results demonstrated significant improvements in exercise capacity and functional class, with a safety profile consistent with prior adult studies, reaffirming its applicability in younger populations.

• Combination Therapy Studies:
  Investigations into Iloprost combined with endothelin receptor antagonists or phosphodiesterase-5 inhibitors have shown promising synergistic effects in reducing pulmonary artery pressures. For instance, a recent multicenter trial indicated that adjunctive Iloprost enhances exercise capacity beyond monotherapy, although long-term benefits require further confirmation.

• Novel Delivery Systems:
  Advancements in inhalation devices aim to optimize pulmonary deposition and patient adherence. For example, a phase II trial assessed a new nebulizer platform, which achieved more uniform lung distribution and reduced administration time, potentially improving quality of life and compliance.

• Exploratory Indications:
  Recent animal studies explore Iloprost's potential in managing microvascular complications in systemic sclerosis, with preliminary clinical data suggesting vascular remodeling benefits. However, these are early-stage investigations, and clinical approval in such indications remains pending.

Uncompleted and Pending Studies

While Iloprost's core indications hold strong, some trials exploring neuroprotective roles or anti-inflammatory effects remain incomplete or canceled, possibly due to inadequate efficacy signals or strategic shifts by sponsors.

Market Analysis

Current Market Size and Segments

The global pulmonary arterial hypertension (PAH) treatment market was valued at approximately USD 4.8 billion in 2022, with inhaled Iloprost occupying a significant segment, especially in regions like Europe and Asia. As of 2023, Iloprost's market share is estimated at ~15-20% within inhaled therapies, chiefly owing to its longstanding approval and established clinical efficacy.

Key end-users include:

• Specialist Pulmonology Centers: Managed with inhaled Iloprost infusion devices.
• Hospitals and Pulmonary Clinics: For acute and chronic management.
• Pharmaceutical Distributors: Due to a steady demand in both developed and emerging markets.

Competitive Landscape

Iloprost faces competition from other PAH therapies, such as:

• Endothelin receptor antagonists (e.g., Bosentan, Ambrisentan)
• Phosphodiesterase-5 inhibitors (e.g., Sildenafil, Tadalafil)
• Prostacyclin analogs (e.g., Epoprostenol, Treprostinil)

While these alternatives often have different administration routes or efficacy profiles, Iloprost’s inhaled route offers distinct advantages in rapid onset and targeted pulmonary vasodilation.

Market Drivers

• Increasing prevalence of PAH: Estimated at 15-50 cases per million, with rising awareness leading to earlier diagnosis.
• Approval expansion: Recent approvals for pediatric use and potential label extensions.
• Healthcare infrastructure improvements: Enhancing access in emerging markets.
• Patient preference: Inhalation route preferred over continuous infusions for quality of life.

Market Challenges

• Administration complexity: Requires frequent inhalations (up to 6-9 times daily), impacting compliance.
• Device costs and reimbursement issues: Particularly in low-resource settings.
• Emergence of newer therapies: Such as oral prostacyclin mimetics (e.g., Selexipag), providing more convenient options.

Regulatory and Patent Landscape

Iloprost’s patents have largely expired or are approaching expiry in key markets, opening avenues for generic production and biosimilars, which could significantly influence pricing and accessibility.

Market Projection and Future Trends

Short-to-Medium Term Outlook (Next 3-5 Years)

• Growth trajectories are estimated at a compound annual growth rate (CAGR) of 4-6%, driven by increased diagnosis rates, evolving clinical practices, and incremental acceptance of inhaled therapies.
• Innovation in delivery devices could bolster adherence and expand market penetration.
• Strategic collaborations between pharmaceutical companies and device manufacturers will likely facilitate broader adoption.

Long-Term Outlook (Beyond 5 Years)

• Pipeline developments and clinical trials focusing on analogous pathways may redefine therapeutic strategies.
• Potential regulatory approvals for multifunctional indications (e.g., systemic sclerosis-associated PAH) could broaden utilization.
• Market entry of biosimilars post-patent expiry will likely lead to price reductions, increasing accessibility.
• Emergence of oral or combined formulations incorporating Iloprost's pharmacodynamic profile could revolutionize administration paradigms.

Impact of Technological Advancements

• Personalized medicine approaches: Tailoring dosing based on biomarkers.
• Improved inhaler technology: Enhancing drug delivery efficiency and patient adherence.
• Data-driven population health management: Using real-world data to refine treatment algorithms.

Conclusion

Iloprost remains a vital component in the management of PAH, with ongoing clinical trials reinforcing its efficacy and safety. The market, while mature, is poised for growth driven by technological innovation, expanding indications, and demographic dynamics. Strategic positioning in emerging markets and investment in delivery systems could further enhance its market share. With patent expiries imminent, industry stakeholders should prepare for increased competition and generics, emphasizing the importance of differentiating therapeutic positioning.

Key Takeaways

• Clinical Development: Recent trials continue to validate Iloprost’s safety and efficacy, especially in pediatric populations and combination therapies.
• Market Dynamics: The inhaled PAH treatment segment holds steady, propelled by rising prevalence, with substantial opportunities for innovation.
• Competitive Edge: Advancements in device technology and strategic collaborations can sustain Iloprost’s relevance amid emerging therapies.
• Future Outlook: The market is expected to grow modestly, with significant upside from biosimilars and expanded indications.
• Strategic Focus: Stakeholders should prioritize device innovation, geographic expansion, and preparation for patent expirations.

FAQs

1. What are the primary indications for Iloprost?
   Inhaled Iloprost is primarily indicated for treating pulmonary arterial hypertension (PAH), improving exercise capacity and reducing symptoms. Off-label research includes potential applications in microvascular disorders and systemic sclerosis.

2. Are there ongoing clinical trials exploring new uses of Iloprost?
   Yes, recent trials study Iloprost’s role in pediatric PAH, combination therapies, and pulmonary vascular remodeling, with some early investigations into systemic sclerosis-related vasculopathies.

3. How does Iloprost compare to other PAH therapies?
   Iloprost’s inhaled route offers rapid pulmonary vasodilation and targeted delivery, with a favorable safety profile. However, administration frequency and device dependence pose adherence challenges compared to oral agents or continuous infusions.

4. What is the impact of patent expiration on Iloprost’s market?
   Patent expiry allows for generic and biosimilar development, likely leading to price reductions and increased accessibility, especially in cost-sensitive markets.

5. What technological innovations could influence Iloprost's market in the future?
   Advances in inhaler device design, personalized dosing algorithms, and combined formulations are poised to improve efficacy, adherence, and patient outcomes, thereby shaping the future market landscape.

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References:

1. [1] Pulmonary Hypertension Association. (2022). Worldwide PAH prevalence and treatment landscape.
2. [2] Market Research Future. (2023). Global PAH therapeutics market analysis.
3. [3] ClinicalTrials.gov. (2023). Recent Iloprost clinical trials and their outcomes.
4. [4] European Medicines Agency. (2022). Iloprost approval in Europe.
5. [5] Statista. (2023). Market share and sales data for pulmonary vasodilators.