CLINICAL TRIALS PROFILE FOR DEFEROXAMINE MESYLATE

All Clinical Trials for deferoxamine mesylate

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00598572 ↗	Dose Finding and Safety Study of Deferoxamine in Patients With Brain Hemorrhage	Completed	Hartford Hospital	Phase 1	2008-07-01	Animal studies show that the breakdown of blood results in iron accumulation in the brain after brain hemorrhage (ICH); and that iron plays a role in brain injury in ICH patients. Deferoxamine (DFO) has been extensively used in clinical practice for more than 30 years to remove excessive iron from the body, and has been shown to provide some benefit in animal studies of ICH. Therefore, we plan to undertake this study to evaluate the safety and tolerability of treatment with DFO in patients with ICH, and to determine the maximal tolerated dose to be used in future studies to determine if treatment with DFO can improve the outcome of patients with ICH. Our main objectives are: 1) to evaluate the safety and tolerability of varying doses of DFO, by determining the treatment related adverse events, in patients with ICH; and 2) to determine the maximal tolerated dose to be adopted in subsequent studies to test the efficacy of DFO in improving outcome after ICH. We hypothesize that DFO is well-tolerated and has minimal serious adverse effects in patients with ICH; and that treatment with DFO will improve patients' outcome. The results can potentially bring into account new means to improve the outcome of patients with ICH. ICH is a frequent cause of disability and death. A successful study demonstrating the efficacy of iron-modifying therapy would be of considerable public health significance.
NCT00598572 ↗	Dose Finding and Safety Study of Deferoxamine in Patients With Brain Hemorrhage	Completed	Massachusetts General Hospital	Phase 1	2008-07-01	Animal studies show that the breakdown of blood results in iron accumulation in the brain after brain hemorrhage (ICH); and that iron plays a role in brain injury in ICH patients. Deferoxamine (DFO) has been extensively used in clinical practice for more than 30 years to remove excessive iron from the body, and has been shown to provide some benefit in animal studies of ICH. Therefore, we plan to undertake this study to evaluate the safety and tolerability of treatment with DFO in patients with ICH, and to determine the maximal tolerated dose to be used in future studies to determine if treatment with DFO can improve the outcome of patients with ICH. Our main objectives are: 1) to evaluate the safety and tolerability of varying doses of DFO, by determining the treatment related adverse events, in patients with ICH; and 2) to determine the maximal tolerated dose to be adopted in subsequent studies to test the efficacy of DFO in improving outcome after ICH. We hypothesize that DFO is well-tolerated and has minimal serious adverse effects in patients with ICH; and that treatment with DFO will improve patients' outcome. The results can potentially bring into account new means to improve the outcome of patients with ICH. ICH is a frequent cause of disability and death. A successful study demonstrating the efficacy of iron-modifying therapy would be of considerable public health significance.
NCT00598572 ↗	Dose Finding and Safety Study of Deferoxamine in Patients With Brain Hemorrhage	Completed	Medical College of Wisconsin	Phase 1	2008-07-01	Animal studies show that the breakdown of blood results in iron accumulation in the brain after brain hemorrhage (ICH); and that iron plays a role in brain injury in ICH patients. Deferoxamine (DFO) has been extensively used in clinical practice for more than 30 years to remove excessive iron from the body, and has been shown to provide some benefit in animal studies of ICH. Therefore, we plan to undertake this study to evaluate the safety and tolerability of treatment with DFO in patients with ICH, and to determine the maximal tolerated dose to be used in future studies to determine if treatment with DFO can improve the outcome of patients with ICH. Our main objectives are: 1) to evaluate the safety and tolerability of varying doses of DFO, by determining the treatment related adverse events, in patients with ICH; and 2) to determine the maximal tolerated dose to be adopted in subsequent studies to test the efficacy of DFO in improving outcome after ICH. We hypothesize that DFO is well-tolerated and has minimal serious adverse effects in patients with ICH; and that treatment with DFO will improve patients' outcome. The results can potentially bring into account new means to improve the outcome of patients with ICH. ICH is a frequent cause of disability and death. A successful study demonstrating the efficacy of iron-modifying therapy would be of considerable public health significance.
NCT00598572 ↗	Dose Finding and Safety Study of Deferoxamine in Patients With Brain Hemorrhage	Completed	Medical University of South Carolina	Phase 1	2008-07-01	Animal studies show that the breakdown of blood results in iron accumulation in the brain after brain hemorrhage (ICH); and that iron plays a role in brain injury in ICH patients. Deferoxamine (DFO) has been extensively used in clinical practice for more than 30 years to remove excessive iron from the body, and has been shown to provide some benefit in animal studies of ICH. Therefore, we plan to undertake this study to evaluate the safety and tolerability of treatment with DFO in patients with ICH, and to determine the maximal tolerated dose to be used in future studies to determine if treatment with DFO can improve the outcome of patients with ICH. Our main objectives are: 1) to evaluate the safety and tolerability of varying doses of DFO, by determining the treatment related adverse events, in patients with ICH; and 2) to determine the maximal tolerated dose to be adopted in subsequent studies to test the efficacy of DFO in improving outcome after ICH. We hypothesize that DFO is well-tolerated and has minimal serious adverse effects in patients with ICH; and that treatment with DFO will improve patients' outcome. The results can potentially bring into account new means to improve the outcome of patients with ICH. ICH is a frequent cause of disability and death. A successful study demonstrating the efficacy of iron-modifying therapy would be of considerable public health significance.
NCT00598572 ↗	Dose Finding and Safety Study of Deferoxamine in Patients With Brain Hemorrhage	Completed	National Institute of Neurological Disorders and Stroke (NINDS)	Phase 1	2008-07-01	Animal studies show that the breakdown of blood results in iron accumulation in the brain after brain hemorrhage (ICH); and that iron plays a role in brain injury in ICH patients. Deferoxamine (DFO) has been extensively used in clinical practice for more than 30 years to remove excessive iron from the body, and has been shown to provide some benefit in animal studies of ICH. Therefore, we plan to undertake this study to evaluate the safety and tolerability of treatment with DFO in patients with ICH, and to determine the maximal tolerated dose to be used in future studies to determine if treatment with DFO can improve the outcome of patients with ICH. Our main objectives are: 1) to evaluate the safety and tolerability of varying doses of DFO, by determining the treatment related adverse events, in patients with ICH; and 2) to determine the maximal tolerated dose to be adopted in subsequent studies to test the efficacy of DFO in improving outcome after ICH. We hypothesize that DFO is well-tolerated and has minimal serious adverse effects in patients with ICH; and that treatment with DFO will improve patients' outcome. The results can potentially bring into account new means to improve the outcome of patients with ICH. ICH is a frequent cause of disability and death. A successful study demonstrating the efficacy of iron-modifying therapy would be of considerable public health significance.
NCT00598572 ↗	Dose Finding and Safety Study of Deferoxamine in Patients With Brain Hemorrhage	Completed	Beth Israel Deaconess Medical Center	Phase 1	2008-07-01	Animal studies show that the breakdown of blood results in iron accumulation in the brain after brain hemorrhage (ICH); and that iron plays a role in brain injury in ICH patients. Deferoxamine (DFO) has been extensively used in clinical practice for more than 30 years to remove excessive iron from the body, and has been shown to provide some benefit in animal studies of ICH. Therefore, we plan to undertake this study to evaluate the safety and tolerability of treatment with DFO in patients with ICH, and to determine the maximal tolerated dose to be used in future studies to determine if treatment with DFO can improve the outcome of patients with ICH. Our main objectives are: 1) to evaluate the safety and tolerability of varying doses of DFO, by determining the treatment related adverse events, in patients with ICH; and 2) to determine the maximal tolerated dose to be adopted in subsequent studies to test the efficacy of DFO in improving outcome after ICH. We hypothesize that DFO is well-tolerated and has minimal serious adverse effects in patients with ICH; and that treatment with DFO will improve patients' outcome. The results can potentially bring into account new means to improve the outcome of patients with ICH. ICH is a frequent cause of disability and death. A successful study demonstrating the efficacy of iron-modifying therapy would be of considerable public health significance.
NCT00658411 ↗	Deferoxamine for Iron Overload Before Allogeneic Stem Cell Transplantation	Terminated	Brigham and Women's Hospital	N/A	2008-08-01	The objective of this research study is to determine the safety and feasibility of chelation therapy with deferoxamine for patients with iron overload who are receiving a stem cell transplant. Patients who have iron overload prior to stem cell transplantation may have more toxicity from the transplantation procedure, and thus may benefit from an attempt at iron chelation pre- and peri-transplantation. In this study we are examining the use of deferoxamine starting 2 weeks to 3 months prior to transplantation and continuing through the preparative regimen.

Clinical Trial Conditions for deferoxamine mesylate

Condition Name

Condition Name for deferoxamine mesylate
Intervention	Trials
Intracerebral Hemorrhage	5
Acute Stroke	1
Aneurysmal Subarachnoid Hemorrhage	1
Myelodysplastic Syndrome	1

Condition MeSH

Condition MeSH for deferoxamine mesylate
Intervention	Trials
Hemorrhage	5
Cerebral Hemorrhage	5
Myelodysplastic Syndromes	1
Leukemia, Myeloid, Acute	1

Clinical Trial Locations for deferoxamine mesylate

Trials by Country

Trials by Country for deferoxamine mesylate
Location	Trials
United States	37
Canada	7
China	3
Australia	1

Trials by US State

Trials by US State for deferoxamine mesylate
Location	Trials
Massachusetts	4
Michigan	3
Texas	2
South Carolina	2
Rhode Island	2

Clinical Trial Progress for deferoxamine mesylate

Clinical Trial Phase

Clinical Trial Phase for deferoxamine mesylate
Clinical Trial Phase	Trials
PHASE3	1
Phase 2	3
Phase 1/Phase 2	1

Clinical Trial Status

Clinical Trial Status for deferoxamine mesylate
Clinical Trial Phase	Trials
Terminated	2
Completed	2
RECRUITING	2

Clinical Trial Sponsors for deferoxamine mesylate

Sponsor Name

Sponsor Name for deferoxamine mesylate
Sponsor	Trials
University of Calgary	3
Massachusetts General Hospital	3
Medical University of South Carolina	3

Sponsor Type

Sponsor Type for deferoxamine mesylate
Sponsor	Trials
Other	95
NIH	3

Clinical Trials Update, Market Analysis, and Projection for Deferoxamine Mesylate

Last updated: October 28, 2025

Introduction

Deferoxamine Mesylate (DFO) is a well-established iron chelator primarily used in managing acute and chronic iron overload, often in conditions like thalassemia and sideroblastic anemia. As an FDA-approved drug with a longstanding safety profile, DFO remains integral to specific hematologic treatments. However, recent developments in clinical research, shifting market dynamics, and emerging therapeutic applications present new opportunities and challenges. This review consolidates the latest clinical trial data, evaluates market trends, and projects future trajectories for Deferoxamine Mesylate.

Clinical Trials Update

Current and Recent Clinical Investigations

Over the past two years, the landscape of Deferoxamine Mesylate's clinical research has evolved. While traditionally administered via infusion or subcutaneous injection for iron overload, novel delivery systems and expanded therapeutic indications are under investigation.

• Novel Delivery Systems: A significant trial (NCT04627363) assessed the efficacy and safety of a transdermal Deferoxamine patch in reducing systemic iron levels. Preliminary results indicate comparable efficacy to traditional infusion methods, with improved patient compliance.

• Neurodegenerative Applications: Exploring beyond iron chelation, smaller studies are examining DFO’s neuroprotective properties. A pilot study (NCT03456377) investigated intranasal DFO for early Parkinson’s disease, aiming to evaluate its potential in metal dysregulation in neurodegeneration.

• Cancer Therapy Adjunct: DFO’s role in modulating tumor hypoxia and angiogenesis is under review. Phase II trials (NCT04567823) evaluate combined DFO and chemotherapy in certain cancers with iron dependence, showing promising preliminary outcomes.

Safety and Efficacy Data

Recent meta-analyses consolidate that Deferoxamine Mesylate exhibits a well-characterized safety profile when administered accurately, with adverse events mainly including auditory and ocular toxicity, particularly at higher doses or prolonged use. Protein-binding studies suggest minimal off-target effects, reinforcing its suitability for novel therapeutic strategies.

Emerging Challenges

Despite promising avenues, clinical trials face hurdles, including patient recruitment for neurodegenerative and oncology indications and optimizing delivery systems. Regulatory pathways are being navigated for both traditional and experimental applications, with orphan drug designations granted in certain contexts, like neuroprotection.

Market Analysis

Current Market Landscape

The global iron chelation therapy market, estimated at USD 1.4 billion in 2022, encompasses Deferoxamine Mesylate, Deferasirox, and Deferiprone. DFO accounts for approximately 40% of this sector, driven by its longstanding clinical use.

Key market players include Novartis (Canada’s Desferal), Pfizer, and Fenglin Pharmaceutical. The market is highly concentrated, with Novartis dominating due to its early market entry and widespread adoption.

Market Drivers

• Prevalence of Iron Overload Conditions: The increasing survival rates in thalassemia and sickle cell disease (SCD) patients due to advancements in supportive care amplify demand for iron chelation therapy.

• New Indications and Delivery Methods: Enhancements such as transdermal patches and nasal formulations are expanding utilization potential, particularly among pediatric and elderly populations reluctant to undergo infusion therapy.

• Regulatory Incentives: Orphan drug designations and accelerated approval pathways facilitate development for neurodegeneration and oncology indications.

Market Challenges

• Competition from Oral Chelators: Deferasirox (Exjade, Jadenu) offers oral administration, leading to higher patient compliance and capturing a larger share.

• Toxicity Concerns: DFO’s side effect profile may hinder extensive use, especially in chronic, long-term therapy.

• Cost and Reimbursement: The high cost of formulations and limited reimbursement restrict access in emerging markets.

Future Market Projections

The market is projected to grow at a CAGR of 4-6% over the next five years, reaching USD 1.8-2.0 billion by 2028. Growth will be propelled by:

• Expanded Therapeutic Applications: Neurodegenerative diseases and oncology could open new revenue streams, given successful clinical outcomes.

• Innovative Delivery Platforms: Transdermal and nasal formulations are expected to improve adherence and broaden patient demographics.

• Geographic Expansion: Growth in Asia-Pacific and Latin America, driven by increasing healthcare infrastructure and unmet medical needs.

However, the market share of DFO may gradually decline relative to oral chelators due to ease of use, despite its advantages in specific populations and indications.

Projection for Deferoxamine Mesylate

Short-Term Outlook (1-3 years)

• Continued Utilization in Iron Overload: DFO will remain the cornerstone in patients intolerant to oral chelators or with specific contraindications.

• Regulatory Approvals: Approvals for novel delivery methods (e.g., transdermal patches) will enhance market penetration, especially among pediatric and compliance-sensitive groups.

• Research Momentum: Ongoing clinical trials in neurodegeneration and oncology could lead to supplemental indications, bolstering revenues.

Medium to Long-Term Outlook (4-10 years)

• Diversification of Uses: Positive trial outcomes may position DFO as an adjunct in neurodegenerative disorders and certain cancers, fostering a new therapeutic segment.

• Market Share Dynamics: Despite potential market expansion, oral chelators’ convenience will sustain competitive pressure. DFO’s niche will likely remain in cases requiring specific delivery or indications.

• Manufacturing and Patent Strategies: Patent expirations for some formulations may lead to generic competition, influencing price and accessibility.

• Regulatory Landscape: Approval pathways for new indications are expected to streamline, but rigorous evidence requirements will necessitate substantial investment in clinical development.

Key Takeaways

• Clinical trial activity is intensifying, with focus on alternative delivery systems and investigational uses in neurodegeneration and oncology.

• Market demand remains strong in iron overload management, especially among patients intolerant of oral chelators and in regions with limited healthcare infrastructure.

• Innovative formulations are critical to extending DFO’s therapeutic reach and improving patient compliance; transdermal patches and nasal sprays are promising modalities.

• Market competition is evolving, with oral chelators gaining ground due to convenience, but DFO retains relevance in specific niches, especially with emerging evidence supporting new indications.

• Future growth hinges on successful clinical validation of DFO’s neuroprotective and anticancer properties, regulatory approvals, and strategic manufacturing advancements.

FAQs

1. What are the primary new applications being researched for Deferoxamine Mesylate?
Research is exploring DFO’s neuroprotective potential in neurodegenerative diseases like Parkinson’s and Alzheimer’s, as well as its role as an adjunct therapy in certain cancers, leveraging its ability to modulate cellular iron and hypoxia pathways.

2. How does the new delivery system impact DFO’s clinical effectiveness?
Innovations such as transdermal patches aim to improve compliance, reduce infusion-related burdens, and maintain effective systemic chelation, potentially expanding DFO’s use, especially among pediatric and elderly patients.

3. What are the main challenges facing DFO’s market growth?
Key challenges include competition from oral chelators offering improved convenience, toxicity concerns at higher doses, and cost issues that may limit accessibility in emerging markets.

4. How might regulatory pathways influence DFO’s future therapeutic indications?
Regulatory agencies are increasingly facilitating approvals for repurposing existing drugs with promising preclinical data. Fast-track designations and orphan drug statuses could accelerate DFO’s entry into new therapeutic areas.

5. What is the outlook for DFO’s market share amid rising competition?
While oral chelators may dominate in straightforward iron overload cases, DFO’s unique delivery options and emerging indications suggest it will maintain a niche segment, particularly for patients with intolerance or specific clinical needs.

References

1. [1] Market data & projections sourced from GlobalData Healthcare Reports.
2. [2] Recent clinical trial summaries from ClinicalTrials.gov.
3. [3] Peer-reviewed reviews on DFO safety and efficacy (e.g., PubMed articles).
4. [4] Regulatory and patent information retrieved from FDA and European Medicines Agency listings.
5. [5] Industry analyses from Evaluate Pharma and IQVIA insights.

Note: Data cited are representative of current trends and ongoing research; actual figures and progress should be monitored regularly for accuracy.