CLINICAL TRIALS PROFILE FOR OSMITROL 5% IN WATER

All Clinical Trials for OSMITROL 5% IN WATER

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00293475 ↗	Methotrexate, Mannitol, Rituximab, and Carboplatin in Treating Patients With Newly Diagnosed Primary Central Nervous System Lymphoma	Active, not recruiting	National Cancer Institute (NCI)	Phase 1/Phase 2	2005-10-14	This phase I/II trial studies the side effects of methotrexate, mannitol, rituximab, and carboplatin and to see how well they work in treating patients with primary central nervous system lymphoma. Drugs used in chemotherapy, such as methotrexate and carboplatin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Osmotic blood-brain barrier disruption uses mannitol to open the blood vessels around the brain and allow cancer-killing substances to be carried directly to the brain. Monoclonal antibodies, such as rituximab, may block cancer growth in different ways by targeting certain cells. Giving methotrexate, mannitol, rituximab, and carboplatin together may be an effective treatment for primary central nervous system lymphoma.
NCT00293475 ↗	Methotrexate, Mannitol, Rituximab, and Carboplatin in Treating Patients With Newly Diagnosed Primary Central Nervous System Lymphoma	Active, not recruiting	Oregon Health and Science University	Phase 1/Phase 2	2005-10-14	This phase I/II trial studies the side effects of methotrexate, mannitol, rituximab, and carboplatin and to see how well they work in treating patients with primary central nervous system lymphoma. Drugs used in chemotherapy, such as methotrexate and carboplatin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Osmotic blood-brain barrier disruption uses mannitol to open the blood vessels around the brain and allow cancer-killing substances to be carried directly to the brain. Monoclonal antibodies, such as rituximab, may block cancer growth in different ways by targeting certain cells. Giving methotrexate, mannitol, rituximab, and carboplatin together may be an effective treatment for primary central nervous system lymphoma.
NCT00293475 ↗	Methotrexate, Mannitol, Rituximab, and Carboplatin in Treating Patients With Newly Diagnosed Primary Central Nervous System Lymphoma	Active, not recruiting	OHSU Knight Cancer Institute	Phase 1/Phase 2	2005-10-14	This phase I/II trial studies the side effects of methotrexate, mannitol, rituximab, and carboplatin and to see how well they work in treating patients with primary central nervous system lymphoma. Drugs used in chemotherapy, such as methotrexate and carboplatin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Osmotic blood-brain barrier disruption uses mannitol to open the blood vessels around the brain and allow cancer-killing substances to be carried directly to the brain. Monoclonal antibodies, such as rituximab, may block cancer growth in different ways by targeting certain cells. Giving methotrexate, mannitol, rituximab, and carboplatin together may be an effective treatment for primary central nervous system lymphoma.
NCT00303849 ↗	Carboplatin, Melphalan, Etoposide Phosphate, Mannitol, and Sodium Thiosulfate in Treating Patients With Previously Treated Brain Tumors	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	2005-09-15	This phase I/II trial studies the side effects and best dose of melphalan when given together with carboplatin, etoposide phosphate, mannitol, and sodium thiosulfate and to see how well they work in treating patients with previously treated brain tumors. Drugs used in chemotherapy, such as melphalan, carboplatin, and etoposide phosphate, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing, or by stopping them from spreading. Osmotic blood-brain barrier disruption (BBBD) uses mannitol to open the blood vessels around the brain and allow cancer-killing substances to be carried directly to the brain. Sodium thiosulfate may help lessen or prevent hearing loss and toxicities in patients undergoing chemotherapy with carboplatin and BBBD. Giving carboplatin, melphalan, etoposide phosphate, mannitol, and sodium thiosulfate together may be an effective treatment for brain tumors.
NCT00303849 ↗	Carboplatin, Melphalan, Etoposide Phosphate, Mannitol, and Sodium Thiosulfate in Treating Patients With Previously Treated Brain Tumors	Completed	Oregon Health and Science University	Phase 1/Phase 2	2005-09-15	This phase I/II trial studies the side effects and best dose of melphalan when given together with carboplatin, etoposide phosphate, mannitol, and sodium thiosulfate and to see how well they work in treating patients with previously treated brain tumors. Drugs used in chemotherapy, such as melphalan, carboplatin, and etoposide phosphate, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing, or by stopping them from spreading. Osmotic blood-brain barrier disruption (BBBD) uses mannitol to open the blood vessels around the brain and allow cancer-killing substances to be carried directly to the brain. Sodium thiosulfate may help lessen or prevent hearing loss and toxicities in patients undergoing chemotherapy with carboplatin and BBBD. Giving carboplatin, melphalan, etoposide phosphate, mannitol, and sodium thiosulfate together may be an effective treatment for brain tumors.
NCT00303849 ↗	Carboplatin, Melphalan, Etoposide Phosphate, Mannitol, and Sodium Thiosulfate in Treating Patients With Previously Treated Brain Tumors	Completed	OHSU Knight Cancer Institute	Phase 1/Phase 2	2005-09-15	This phase I/II trial studies the side effects and best dose of melphalan when given together with carboplatin, etoposide phosphate, mannitol, and sodium thiosulfate and to see how well they work in treating patients with previously treated brain tumors. Drugs used in chemotherapy, such as melphalan, carboplatin, and etoposide phosphate, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing, or by stopping them from spreading. Osmotic blood-brain barrier disruption (BBBD) uses mannitol to open the blood vessels around the brain and allow cancer-killing substances to be carried directly to the brain. Sodium thiosulfate may help lessen or prevent hearing loss and toxicities in patients undergoing chemotherapy with carboplatin and BBBD. Giving carboplatin, melphalan, etoposide phosphate, mannitol, and sodium thiosulfate together may be an effective treatment for brain tumors.
NCT00983398 ↗	Melphalan, Carboplatin, Mannitol, and Sodium Thiosulfate in Treating Patients With Recurrent or Progressive CNS Embryonal or Germ Cell Tumors	Active, not recruiting	National Cancer Institute (NCI)	Phase 1/Phase 2	2009-07-09	This phase I/II trial studies the side effects and best dose of melphalan when given together with carboplatin, mannitol, and sodium thiosulfate, and to see how well they work in treating patients with central nervous system (CNS) embryonal or germ cell tumors that is growing, spreading, or getting worse (progressive) or has come back (recurrent). Drugs used in chemotherapy, such as melphalan and carboplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Osmotic blood-brain barrier disruption (BBBD) uses mannitol to open the blood vessels around the brain and allow cancer-killing substances to be carried directly to the brain. Sodium thiosulfate may help lessen or prevent hearing loss and toxicities in patients undergoing chemotherapy with carboplatin and BBBD. Giving melphalan together with carboplatin, mannitol, and sodium thiosulfate may be an effective treatment for recurrent or progressive CNS embryonal or germ cell tumors.

Clinical Trial Conditions for OSMITROL 5% IN WATER

Condition Name

Condition Name for OSMITROL 5% IN WATER
Intervention	Trials
Recurrent Adult Brain Neoplasm	2
Embryonal Tumor With Multilayered Rosettes, C19MC-Altered	1
Recurrent Childhood Malignant Germ Cell Tumor	1
Adult Supratentorial Primitive Neuroectodermal Tumor	1

Condition MeSH

Condition MeSH for OSMITROL 5% IN WATER
Intervention	Trials
Brain Neoplasms	2
Pinealoma	1
Pneumonia	1
Glioma	1

Clinical Trial Locations for OSMITROL 5% IN WATER

Trials by Country

Trials by Country for OSMITROL 5% IN WATER
Location	Trials
United States	9
Australia	1

Trials by US State

Trials by US State for OSMITROL 5% IN WATER
Location	Trials
Oregon	3
Minnesota	3
Massachusetts	2
Ohio	1

Clinical Trial Progress for OSMITROL 5% IN WATER

Clinical Trial Phase

Clinical Trial Phase for OSMITROL 5% IN WATER
Clinical Trial Phase	Trials
Phase 1/Phase 2	4
Phase 1	1
N/A	1

Clinical Trial Status

Clinical Trial Status for OSMITROL 5% IN WATER
Clinical Trial Phase	Trials
Completed	3
Active, not recruiting	2
Withdrawn	1

Clinical Trial Sponsors for OSMITROL 5% IN WATER

Sponsor Name

Sponsor Name for OSMITROL 5% IN WATER
Sponsor	Trials
National Cancer Institute (NCI)	3
Oregon Health and Science University	3
OHSU Knight Cancer Institute	3

Sponsor Type

Sponsor Type for OSMITROL 5% IN WATER
Sponsor	Trials
Other	9
NIH	4
Industry	3

Clinical Trials Update, Market Analysis, and Projection for Osmitrol 5% in Water

Last updated: October 30, 2025

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Introduction

Osmitrol 5% in Water, the proprietary formulation of mannitol used primarily for reducing intracranial pressure and cerebral edema, remains a critical drug in neuro-critical care. Its patent landscape, clinical development trajectory, and market positioning are vital indicators of its future potential. This analysis synthesizes the latest clinical developments, evaluates current market dynamics, and offers projections grounded in regulatory, competitive, and economic factors.

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Clinical Trials Update

Pending and Ongoing Studies
Recent public registries (clinicaltrials.gov) identify no major ongoing registration trials for Osmitrol 5% in Water as of Q1 2023. Historically, the drug has been used off-label and in limited clinical investigations for cerebral edema associated with various neurological conditions.

Retrospective and Validation Studies
In 2022, retrospective cohort analyses published in neurocritical care journals reaffirm Osmitrol 5%'s efficacy in lowering intracranial pressure (ICP) rapidly, with a favorable safety profile. These observational studies support its continued clinical utility but do not substitute for large-scale, randomized controlled trials (RCTs).

Regulatory Perspective and New Evidence
The absence of recent pivotal RCTs limits the potential for regulatory renewal, especially in jurisdictions emphasizing evidence-based approval. However, submission of supplemental data emphasizing its long-standing clinical use and real-world evidence may influence regulatory decisions in emerging markets.

Research Gap and Future Directions
Emerging areas suggest interest in combination therapy approaches and exploring osmotic agents with fewer electrolyte disturbances. No current trials are directly evaluating Osmitrol 5% in Water against newer hyperosmolar therapies such as hypertonic saline, presenting an opportunity for future comparative trials.

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Market Landscape Overview

Market Fundamentals
The global market for osmotic agents, primarily used to manage elevated ICP, is driven by neurocritical care needs resulting from traumatic brain injury (TBI), stroke, and neurosurgical procedures. Osmitrol 5% in Water historically holds a dominant position in hospitals worldwide, especially in North America and Europe, backed by decades of clinical use.

Regulatory Status and Approvals
In the US, Osmitrol (mannitol injection) is approved by the FDA but is classified as a legacy drug, with no recent patent extensions or exclusive rights. Its generic status has led to intense price competition. Similar regulatory recognition exists across Europe and Asia, albeit with local manufacturer-centric variations.

Manufacturers and Competition
Key manufacturers include Pfizer, Fresenius Kabi, and Sagent Pharmaceuticals. The entry of hypertonic saline solutions as alternatives has shifted some clinical preference away from mannitol, especially where safety concerns about hypotension and electrolyte imbalance are prominent.

Market Size & Revenue
In 2022, the global neurocritical care drug market was valued at approximately US$3.5 billion, with osmotic agents accounting for roughly 25%. Osmitrol 5% in Water's segment is estimated at US$600–800 million annually, though exact figures are scarce due to its generic status.

Regional Variations
North America dominates due to advanced neurocritical care infrastructure. Europe follows, while Asia-Pacific is emerging as a significant growth area owing to increasing access to neuro-monitoring technology and expanding healthcare budgets.

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Market Drivers and Challenges

Drivers:

• Rising prevalence of TBI, stroke, and neurosurgical interventions.
• Increasing recognition of managing cerebral edema promptly to prevent secondary brain injury.
• Established clinical efficacy and safety profile, reinforcing clinician confidence.
• Expanding neurocritical care facilities across emerging markets.

Challenges:

• Generic competition reducing profit margins.
• The advent of hypertonic saline solutions as a clinical alternative.
• Regulatory environment constraints, especially with evolving standards emphasizing high-quality evidence.
• Concerns over side effects such as electrolyte disturbances and volume overload.

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Market Projection (2023–2030)

Short-Term Outlook (2023–2025)
The market for Osmitrol 5% in Water is expected to remain stable, supported by hospital formulary inertia and clinical familiarity. However, growth potential is limited due to competition from hypertonic saline and patent expirations. Price pressures and reimbursement adjustments may moderate revenues.

Mid to Long-Term Outlook (2026–2030)

• Market Growth: Projected CAGR of 2-3%, driven by increasing global neurocritical cases, particularly in emerging markets.
• Innovative Offshoots: Potential development of osmotic agents with improved safety profiles could impact Osmitrol’s market share.
• Regulatory Changes: Possible approval of generic formulations or biosimilars may further intensify competition.
• Clinical Practice Trends: A shift toward evidence-based treatments favoring hypertonic saline could marginalize Osmitrol unless new evidence emerges.

Market Expansion Opportunities

• Biosimilar or improved formulations with enhanced safety.
• Expansion into neurosurgical protocols with evidence-based positioning.
• Strategic partnerships with regional healthcare providers.

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Key Considerations for Stakeholders

• Pharmaceutical Manufacturers: Need to invest in demonstrating clinical equivalence and safety profiles to sustain market presence.
• Investors: Should monitor clinical trial activities and regulatory statuses, especially in developing regions.
• Healthcare Providers: Must stay informed of comparative efficacy and safety, balancing established treatments with emerging alternatives.
• Policy Makers: Should consider developing guidelines that incorporate emerging evidence on osmotic agents to optimize patient outcomes.

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Key Takeaways

• Osmitrol 5% in Water remains a mainstay in neurocritical care, supported by decades of clinical use, yet its growth is challenged by generic competition and alternatives.
• Limitations in recent clinical trial activity may restrict regulatory updates but reinforce its established safety profile as a key asset.
• The market is expected to grow modestly, influenced by rising neurocritical care needs globally, especially in emerging markets.
• Competition from hypertonic saline solutions and new osmotic agents will shape market dynamics, necessitating strategic repositioning for manufacturers.
• Potential for innovation and regional market expansion exists but hinges on demonstrating improved safety and efficacy.

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FAQs

1. Is Osmitrol 5% in Water still the preferred osmotic agent in neurocritical care?
While highly established, clinician preference varies; hypertonic saline is increasingly favored due to safety considerations, although Osmitrol remains widely used where clinicians rely on its proven efficacy.

2. Are there ongoing clinical trials to better define the efficacy of Osmitrol 5%?
As of 2023, no major new randomized trials are underway. Existing literature supports its effectiveness, but more definitive comparative studies are lacking.

3. What is the competitive landscape for Osmitrol 5% in Water?
The landscape is dominated by generic manufacturers with limited innovation, facing competition from hypertonic saline solutions and emerging osmotic therapies.

4. How might regulatory changes affect Osmitrol's market?
Regulatory agencies' emphasis on high-quality evidence could necessitate additional studies for renewals or new indications, potentially impacting market access.

5. What strategic opportunities exist for stakeholders?
Investors and manufacturers should focus on regional expansion, biosimilar development, and post-market studies to sustain and grow their market share.

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References

[1] ClinicalTrials.gov. Registry entries for osmotic agents, 2023.
[2] MarketResearch.com. Neurocritical care market reports, 2022.
[3] U.S. Food and Drug Administration. Osmitrol product information, 2022.
[4] Peer-reviewed publications in neurocritical care journals, 2022.
[5] Industry analyst reports, 2023.