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.

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
Leukemia, Lymphoid	1
Subarachnoid Hemorrhage	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
Recruiting	2
Terminated	2
Completed	2

Clinical Trial Sponsors for DEFEROXAMINE MESYLATE

Sponsor Name

Sponsor Name for DEFEROXAMINE MESYLATE
Sponsor	Trials
National Institute of Neurological Disorders and Stroke (NINDS)	3
Beth Israel Deaconess Medical Center	3
Hartford Hospital	3

Sponsor Type

Sponsor Type for DEFEROXAMINE MESYLATE
Sponsor	Trials
Other	95
NIH	3

Last updated: July 30, 2026

Deferoxamine Mesylate Clinical Trials Update, Market Analysis, and Forecast

Deferoxamine mesylate (deferoxamine) is an iron-chelation drug used for conditions involving excess iron. Public clinical-trial and commercialization data are sparse and fragmented, and market sizing varies materially by data vendor and by whether sales of closely related injectable chelators are grouped together. No current, material, late-stage development program or near-term FDA exclusivity-driven launch window can be evidenced from the available public record for this specific API.

What is the current clinical trial status for deferoxamine mesylate (late-stage studies, active trials, results)?

Answer: No clear, consistently reported Phase 3 (or registrational) study program for deferoxamine mesylate can be tied to a single, up-to-date sponsor-sponsored pipeline entry in the major public registries from the information available in this context.

How to read the latest trial signal for deferoxamine

For an older, established chelator like deferoxamine, the “clinical trials update” typically reflects:

• Small investigator-led studies (dose optimization, infusion protocols, pediatric endpoints, iron-overload subtypes)
• Comparative studies versus oral chelators where deferasirox or deferiprone are the active comparators
• Observational or pharmacokinetic studies rather than pivotal efficacy trials

Which trial categories show up most often for iron chelators

• Pharmacokinetics (subcutaneous vs IV schedules, pediatric dosing)
• Safety in special populations (pediatrics, renal impairment, cardiac iron)
• Iron biomarker outcomes (serum ferritin, transferrin saturation, cardiac MRI metrics in transfusion-dependent populations)

Why late-stage registrational updates are hard to evidence

Deferoxamine is older than most current trial registries and has historically been supported by:

• Established labeling for specific indications
• Incremental protocol studies rather than full Phase 3 re-registration cycles

Which indications are still relevant for deferoxamine mesylate in 2026 (transfusional iron overload, acute iron intoxication, other use cases)?

Answer: Deferoxamine is used to bind free iron and is prescribed for iron overload and iron intoxication. The exact label scope depends on jurisdiction and the approved formulation and route (IV/SC).

Indication buckets used in market and trials

1. Transfusional iron overload in certain patient groups
2. Acute iron poisoning (clinical setting emergency use)
3. Iron overload states where parenteral chelation is selected

How indication framing affects commercial projections

Market forecasts for chelators are driven by:

• Uptake in transfusion-dependent patient pools
• Physician shift toward oral chelators
• Hospital procurement patterns for IV/SC options
• Country-specific reimbursement and formulary inclusion

How does deferoxamine mesylate compete against deferasirox and deferiprone (market share drivers, switching risks, payer behavior)?

Answer: Deferoxamine competes primarily on:

• Route and logistics (parenteral infusion or pump use)
• Clinical scenarios where IV/SC chelation is preferred
• Relative tolerability profiles and adherence burden versus oral alternatives

Key competitive dynamics

• Oral chelators (deferasirox, deferiprone) often win long-term adherence and convenience.
• Deferoxamine maintains relevance where parenteral chelation is chosen, including specific clinical scenarios and where payer or guideline constraints exist.

What most impacts switching

• Patient adherence and caregiver burden
• Reimbursement changes favoring oral agents
• Safety monitoring feasibility (audits for renal/hearing/vision in some settings)
• Clinical preference for route in special populations

What is the Orange Book status of deferoxamine mesylate (patents, exclusivity, listed products)?

Answer: Specific Orange Book listing details cannot be provided in a complete and accurate way from the information available in this context.

What matters for exclusivity and generic entry

For small-molecule injectables like deferoxamine:

• Composition-of-matter, salt-form, and formulation patents historically drive the patent landscape
• Generic access tends to be constrained less by exclusivity now and more by manufacturing and labeling lineage

When does deferoxamine mesylate lose exclusivity (patent expiration timeline and generic launch risk)?

Answer: A complete, accurate exclusivity and patent-expiration timeline for deferoxamine mesylate cannot be produced from the available information in this context.

What investors typically model for older chelators

• Patent cliff risk for the last brand/formulation (not the API)
• New entrants through abbreviated approval pathways for injectables
• Market share shifts driven by procurement cycles rather than breakthrough launches

What patents protect deferoxamine mesylate (composition, formulation, method-of-use) and how strong is the estate?

Answer: A complete patent estate with numbers, jurisdictions, assignees, and expiration dates cannot be compiled accurately from the available information in this context.

Typical patent buckets in chelators

• Deferoxamine free base vs mesylate salt claims
• Injectable composition claims (buffering, stability, solubilizers)
• Administration device or regimen claims (pump/SC schedules) if filed

Is there any Paragraph IV (Hatch-Waxman) litigation or settlement risk for deferoxamine mesylate?

Answer: No accurate, docket-level Paragraph IV litigation status can be stated for deferoxamine mesylate from the information available in this context.

Why Paragraph IV is still relevant even for older drugs

• Injectables can have ongoing patent protection on specific strengths, containers, or formulation stability improvements
• Carve-outs by NDA/ANDA holder can create incremental litigation, even when the API is long off-patent

What is the FDA regulatory status for deferoxamine mesylate (approved pathway, labeling, safety communications)?

Answer: Deferoxamine mesylate is an FDA-approved chelator in the US, but the specific, current labeling and any up-to-date safety communications cannot be enumerated accurately in this context.

Regulatory signals that drive market access

• Label changes affecting indicated populations and administration routes
• REMS where applicable (typically rare for established chelators)
• Safety communications impacting prescribing patterns

What is the market size for deferoxamine mesylate (global and US) and how do vendors define it?

Answer: A precise market size and revenue estimate for deferoxamine mesylate cannot be delivered accurately from the available information in this context. Forecasts in iron chelators are highly sensitive to how analysts classify products:

• API-only vs finished dosage form (strength-specific)
• Inclusive vs exclusive grouping with other chelators
• Inclusion of emergency iron poisoning use vs chronic transfusional overload

How analysts typically segment iron chelators

• By chelator class (deferoxamine, deferasirox, deferiprone)
• By formulation type (IV/SC vs oral)
• By indication (transfusion-dependent thalassemia, MDS-related overload, iron poisoning)

How should investors and commercial teams project deferoxamine mesylate revenue (2026-2036 scenarios)?

Answer: Scenario modeling can be framed, but a numeric forecast cannot be produced accurately from the information available in this context.

High-confidence forecast drivers (qualitative)

• Continued price pressure from generics for parenteral injectables
• Clinical preference shift to oral chelators in transfusional overload
• Persisting demand in clinical settings requiring parenteral chelation
• Geographic formulary and tender dynamics for hospital products

Most common modeling approach for older chelators

1. Start with treated patient pool estimates by indication and route preference
2. Apply penetration assumptions (share of parenteral chelation)
3. Model price erosion (generic competition and procurement cycles)
4. Incorporate competitive substitution by oral chelators
5. Track stock-outs and tender outcomes (hospital injector volatility)

What generic entry risks exist for deferoxamine mesylate (manufacturing barriers, supply constraints, ANDA landscape)?

Answer: Specific generic entry and ANDA landscape details cannot be stated accurately from the information available in this context.

Manufacturing and supply constraints to watch

• Sterile injectable capacity and cold-chain constraints (if applicable)
• Container closure stability and particle control
• Batch release and regulatory inspection outcomes

Which companies market deferoxamine mesylate and how concentrated is the competitive landscape?

Answer: Company-by-company market share and sales concentration cannot be stated accurately from the available information in this context.

What concentration typically looks like in branded injectables

• Brand originators retain distribution in certain channels
• Generics dominate price-sensitive tenders
• Distribution fragmentation increases with multiple labelers and NDC-level differences

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Key Takeaways

• A current, registrational-scale clinical-trials signal for deferoxamine mesylate cannot be evidenced from the information available in this context.
• Competitive pressure from oral chelators remains the dominant commercial overhang for parenteral chelation.
• Orange Book status, exclusivity timelines, and detailed patent estate cannot be enumerated accurately in this context.
• Numeric market sizing and a 2026–2036 forecast cannot be produced accurately from the available information in this context.

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FAQs

1. What routes (IV vs SC) drive prescribing patterns for deferoxamine mesylate?
2. How do iron chelation guidelines influence demand for deferoxamine versus oral alternatives?
3. What safety monitoring requirements most affect real-world use of parenteral iron chelators?
4. How do hospital tenders and procurement cycles impact pricing stability for deferoxamine injectables?
5. What regulatory labeling changes have historically shifted use of deferoxamine mesylate?

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References (APA)

1. FDA. Orange Book: Approved Drug Products with Therapeutic Equivalence Evaluations. US FDA.
2. ClinicalTrials.gov. Deferoxamine mesylate studies and results. US National Library of Medicine.