CLINICAL TRIALS PROFILE FOR DESFERAL

All Clinical Trials for Desferal

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00004982 ↗	Combination Iron Chelation Therapy	Completed	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 1	1998-12-01	Patients with beta-thalassemia (Cooley's Anemia) continue to suffer from the transfusion-induced iron overload due to the inadequacies of current iron-chelation therapy. Compliance with the use of the only FDA-approved drug for removing excess iron from patients (Desferal) continues to be a major problem despite convincing evidence that it markedly reduces morbidity and prolongs life. The full potential of iron-chelation therapy will not be realized until an orally-effective drug is available. This small trial is testing the premise that a combination of drugs as a new approach to iron chelation therapy may reduce side effects and increase efficacy. If both drugs can be given orally, there may be a better chance of finding a suitable alternative to Desferal. Several combinations of experimental iron chelating drugs are being used in this trial.
NCT00110266 ↗	Study of Deferasirox for Treatment of Transfusional Iron Overload in Myelodysplastic Patients	Completed	Novartis Pharmaceuticals	Phase 2	2005-07-25	The purpose of this trial is to examine the safety and efficacy of deferasirox in patients with Myelodysplastic Syndrome (MDS) and chronic iron overload from blood transfusions.
NCT00293098 ↗	Compassionate Use of Deferiprone for Patients With Thalassemia and Iron-Induced Heart Disease	Approved for marketing	ApoPharma		2006-03-01	Patients who have iron overload due to chronic blood transfusions and have developed heart failure or who are at high risk of heart failure because of the high levels of iron in their hearts, will be treated with deferiprone, an investigational drug, in combination with deferoxamine (Desferal). Some studies suggest that deferiprone may be better than deferoxamine in removing iron from the heart and improving heart function, and that using both drugs together may remove more iron. Participants would make a clinic visit for lab studies each week, and would continue to take deferiprone for as long as their physician feels it is useful in their care.
NCT00293098 ↗	Compassionate Use of Deferiprone for Patients With Thalassemia and Iron-Induced Heart Disease	Approved for marketing	Children's Hospital of Philadelphia		2006-03-01	Patients who have iron overload due to chronic blood transfusions and have developed heart failure or who are at high risk of heart failure because of the high levels of iron in their hearts, will be treated with deferiprone, an investigational drug, in combination with deferoxamine (Desferal). Some studies suggest that deferiprone may be better than deferoxamine in removing iron from the heart and improving heart function, and that using both drugs together may remove more iron. Participants would make a clinic visit for lab studies each week, and would continue to take deferiprone for as long as their physician feels it is useful in their care.
NCT00349453 ↗	Study Using Deferiprone Alone or in Combination With Desferrioxamine in Iron Overloaded Transfusion-dependent Patients	Completed	Lipomed	Phase 2	2005-03-01	Systematical (retro- and prospective) investigation of the long-term safety (toxicity assessment according to CTCAE v3.0) and efficacy of deferiprone either given alone or in combination with desferrioxamine

Clinical Trial Conditions for Desferal

Condition Name

Condition Name for Desferal
Intervention	Trials
Iron Overload	6
Thalassemia	4
Myelodysplastic Syndrome	2
Sickle Cell Disease	2

Condition MeSH

Condition MeSH for Desferal
Intervention	Trials
Iron Overload	9
Thalassemia	8
COVID-19	3
beta-Thalassemia	3

Clinical Trial Locations for Desferal

Trials by Country

Trials by Country for Desferal
Location	Trials
United States	38
Switzerland	6
Egypt	3
Canada	3
Netherlands	2

Trials by US State

Trials by US State for Desferal
Location	Trials
New York	3
Massachusetts	3
California	2
Pennsylvania	2
Michigan	2

Clinical Trial Progress for Desferal

Clinical Trial Phase

Clinical Trial Phase for Desferal
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	3
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for Desferal
Clinical Trial Phase	Trials
Completed	8
Recruiting	3
Unknown status	3

Clinical Trial Sponsors for Desferal

Sponsor Name

Sponsor Name for Desferal
Sponsor	Trials
Lipomed	2
Ain Shams University	2
Karolinska University Hospital	2

Sponsor Type

Sponsor Type for Desferal
Sponsor	Trials
Other	22
Industry	6
NIH	1

Clinical Trials Update, Market Analysis and Projection for Desferal

Last updated: April 30, 2026

What is Desferal and what is its current commercial footprint?

Desferal is the brand name for deferoxamine mesylate (DFO), an injectable iron-chelating agent used for chronic transfusional iron overload (commonly in patients with thalassemia and other transfusion-dependent anemias) and for acute iron overload in specific settings.

Core market positioning

• Mechanism: parenteral chelation with deferoxamine binding free iron and facilitating excretion (primarily urinary).
• Clinical use pattern: chronic therapy is typically limited by the route (subcutaneous/IV delivery) and practicality versus oral alternatives.
• Primary competitive set: oral chelators, mainly deferasirox and deferiprone, which compete for chronic transfusional iron overload indications in many geographies.

Demand drivers

• Patient population tied to transfusion-dependent anemias and iron overload management.
• Uptake of oral chelators shifts share away from DFO in parts of the market, but DFO remains relevant where oral agents are not suitable, where regimens call for parenteral therapy, or where formulary practice supports DFO access.

What do the available clinical-trials signals indicate for Desferal?

A complete, current “trial-by-trial” update requires trial-level identification (NCT numbers, sponsorship, protocol stage, start/completion dates). Under the constraints of this response, no verifiable, up-to-date trial dataset is provided. As a result, this section is limited to high-level statements based on the established use profile of deferoxamine and its typical role in clinical practice.

Clinical development reality for older injectables

• For mature products like Desferal, ongoing clinical activity is commonly restricted to comparative effectiveness in specific subgroups, pharmacovigilance, formulation or device administration updates, and expanded-use studies tied to safety and dosing optimization rather than late-stage label expansions.
• Deferoxamine’s competitive environment typically shifts new enrollment toward oral chelators, which reduces the likelihood of large Phase 3 programs centered on the agent itself.

What is the market size and how should Desferal be projected?

No current, source-citable market-sizing model is included in the provided input, and this response must not invent figures. Under these rules, a projection can be produced only in a scenario framework tied to known category drivers and competitive dynamics, without assigning specific TAM/SAM values.

Projection framework (directional, evidence-aligned)

1. Base-case trajectory (modest decline or flat volume in mature markets)

   • Chronic chelation patient share tends to rotate toward oral agents as formularies and adherence dynamics favor oral dosing.
   • DFO use persists but becomes more concentrated in specific clinical circumstances (route preference, contraindications, tolerability decisions, or regimen selection).

2. Downside case (share loss accelerates)

   • Expanded oral chelator preference and tightening formulary restrictions can compress DFO volume.
   • If oral alternatives remain broadly available and clinically favored, DFO’s category share declines even if the underlying treated population grows.

3. Upside case (stabilization through access and subgroup need)

   • Where parenteral therapy remains standard, or where oral agents are less tolerated, DFO usage can stabilize.
   • Regions with lower oral chelator penetration can sustain DFO demand longer, with gradual substitution over time.

What to model for commercial planning

• Unit drivers: treated patients, chelation regimen adherence, and switching rates between DFO and oral chelators.
• Pricing drivers: reimbursement structure and loss of preferential access as newer chelators establish standard-of-care positions.
• Geography: uptake speed of oral agents and formulary penetration are key variables.

Market outlook indicators to track (actionable workplan)

• Formulary status for chronic transfusional iron overload indications (by major markets).
• Utilization mix (share of DFO versus deferasirox and deferiprone) through claims or distributor data.
• Hospital procurement patterns for parenteral chelation administration infrastructure.
• Safety/tolerability trends impacting switching behavior (GI intolerance, cytopenias, hepatic monitoring burden, and adherence).

How does the competitive landscape shape Desferal’s economics?

Desferal’s competitive position is constrained by administration practicality and clinician preference trends.

Competitive set and typical trade-offs

• Deferoxamine (DFO): parenteral delivery; often selected when oral agents are unsuitable or when parenteral management is clinically justified.
• Deferasirox: oral once-daily option; competes strongly for chronic management.
• Deferiprone: oral option with different safety monitoring profile; also competes for chronic use and specific clinical goals.

Implication for projection

• Even with stable total chelation demand, DFO’s share can decline due to oral agent substitution.
• Growth in the overall treated population does not automatically translate to DFO volume growth.

Key takeaways

• Desferal (deferoxamine mesylate) remains an established injectable chelator for iron overload tied to transfusion-dependent anemias.
• Clinical-trials activity for older injectables is typically limited in scope and often shifts toward safety, regimen refinement, and subgroup evidence rather than major new Phase 3 label expansions.
• Market projection should be modeled through chelation treated-population growth vs. switching to oral chelators, with DFO expected to face modest decline or stabilization in mature markets and longer persistence in settings where oral chelators have lower penetration.

FAQs

1. Is Desferal still used for chronic transfusional iron overload?
   Yes. It is a standard injectable chelation option in the iron overload management pathway for transfusion-dependent patients.

2. Why does Desferal typically lose share to oral competitors?
   Administration practicality and patient adherence favor oral chelators in many formularies, especially for long-term chronic regimens.

3. What determines whether clinicians keep patients on Desferal?
   Route feasibility, contraindications to oral agents, tolerability, monitoring considerations, and local reimbursement/formulary access.

4. Are there likely to be major new late-stage trials for Desferal?
   In mature products, large late-stage trials are less common; remaining studies are usually narrower in scope (safety, dosing/admin optimization, or specific subgroups).

5. What should a market projection focus on for Desferal?
   The balance between treated patient growth and the rate at which patients switch from injectable DFO to oral chelators, plus geography-specific formulary adoption.

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References (APA)

[1] FDA. (n.d.). Desferal (deferoxamine mesylate) prescribing information. U.S. Food and Drug Administration.
[2] European Medicines Agency. (n.d.). Desferal: EPAR/assessment documents and product information. European Medicines Agency.
[3] World Health Organization. (n.d.). Guidance on transfusion-dependent anemia and iron overload management. World Health Organization.