CLINICAL TRIALS PROFILE FOR NIFURTIMOX

505(b)(2) Clinical Trials for NIFURTIMOX

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Dosage	NCT03981523 ↗	New Therapies and Biomarkers for Chagas Infection	Active, not recruiting	Barcelona Institute for Global Health	Phase 2	2019-12-18	Chagas disease (CD) is an endemic zoonotic disease with a significant global impact. Current approved treatments for CD (benznidazole (BZN) and nifurtimox (NFX)) were developed in the 1970s with regimens and dosing intervals derived from decades-old patient series and with very limited direct comparisons. Treatment recommendations vary significantly from country to country and the comparative evidence-base with the current treatment regimens is limited. The reported efficacy of both drugs in patients with T. cruzi infection is variable and depends on the disease stage, the drug dose, the age of patients, and the infecting T. cruzi strain or genotype. Due to a therapeutic failure of at least 20% after 12 months in chronic patients and the high rate of adverse events, together with the recent data that suggest that we may be overdosing patients, we propose to test new dosing regimens of these two old compounds. Hypotheses: - Lowering the frequency of drug dosing of BZN and NFX, the plasma drug levels of the drugs within the therapeutic range will be maintained. - The duration of treatment with BZN or NFX may be related to the effectiveness of these drugs. - Blood levels of the proposed biomarkers will significantly diminish or became negative after a relatively short interval after treatment.
New Dosage	NCT03981523 ↗	New Therapies and Biomarkers for Chagas Infection	Active, not recruiting	Drugs for Neglected Diseases	Phase 2	2019-12-18	Chagas disease (CD) is an endemic zoonotic disease with a significant global impact. Current approved treatments for CD (benznidazole (BZN) and nifurtimox (NFX)) were developed in the 1970s with regimens and dosing intervals derived from decades-old patient series and with very limited direct comparisons. Treatment recommendations vary significantly from country to country and the comparative evidence-base with the current treatment regimens is limited. The reported efficacy of both drugs in patients with T. cruzi infection is variable and depends on the disease stage, the drug dose, the age of patients, and the infecting T. cruzi strain or genotype. Due to a therapeutic failure of at least 20% after 12 months in chronic patients and the high rate of adverse events, together with the recent data that suggest that we may be overdosing patients, we propose to test new dosing regimens of these two old compounds. Hypotheses: - Lowering the frequency of drug dosing of BZN and NFX, the plasma drug levels of the drugs within the therapeutic range will be maintained. - The duration of treatment with BZN or NFX may be related to the effectiveness of these drugs. - Blood levels of the proposed biomarkers will significantly diminish or became negative after a relatively short interval after treatment.
New Dosage	NCT03981523 ↗	New Therapies and Biomarkers for Chagas Infection	Active, not recruiting	Fundación Ciencia y Estudios Aplicados para el Desarrollo en Salud y Medio Ambiente (CEADES)	Phase 2	2019-12-18	Chagas disease (CD) is an endemic zoonotic disease with a significant global impact. Current approved treatments for CD (benznidazole (BZN) and nifurtimox (NFX)) were developed in the 1970s with regimens and dosing intervals derived from decades-old patient series and with very limited direct comparisons. Treatment recommendations vary significantly from country to country and the comparative evidence-base with the current treatment regimens is limited. The reported efficacy of both drugs in patients with T. cruzi infection is variable and depends on the disease stage, the drug dose, the age of patients, and the infecting T. cruzi strain or genotype. Due to a therapeutic failure of at least 20% after 12 months in chronic patients and the high rate of adverse events, together with the recent data that suggest that we may be overdosing patients, we propose to test new dosing regimens of these two old compounds. Hypotheses: - Lowering the frequency of drug dosing of BZN and NFX, the plasma drug levels of the drugs within the therapeutic range will be maintained. - The duration of treatment with BZN or NFX may be related to the effectiveness of these drugs. - Blood levels of the proposed biomarkers will significantly diminish or became negative after a relatively short interval after treatment.
New Dosage	NCT03981523 ↗	New Therapies and Biomarkers for Chagas Infection	Active, not recruiting	Institute of Parasitology and Biomedicine Lopez Neyra	Phase 2	2019-12-18	Chagas disease (CD) is an endemic zoonotic disease with a significant global impact. Current approved treatments for CD (benznidazole (BZN) and nifurtimox (NFX)) were developed in the 1970s with regimens and dosing intervals derived from decades-old patient series and with very limited direct comparisons. Treatment recommendations vary significantly from country to country and the comparative evidence-base with the current treatment regimens is limited. The reported efficacy of both drugs in patients with T. cruzi infection is variable and depends on the disease stage, the drug dose, the age of patients, and the infecting T. cruzi strain or genotype. Due to a therapeutic failure of at least 20% after 12 months in chronic patients and the high rate of adverse events, together with the recent data that suggest that we may be overdosing patients, we propose to test new dosing regimens of these two old compounds. Hypotheses: - Lowering the frequency of drug dosing of BZN and NFX, the plasma drug levels of the drugs within the therapeutic range will be maintained. - The duration of treatment with BZN or NFX may be related to the effectiveness of these drugs. - Blood levels of the proposed biomarkers will significantly diminish or became negative after a relatively short interval after treatment.
New Dosage	NCT03981523 ↗	New Therapies and Biomarkers for Chagas Infection	Active, not recruiting	Mundo Sano Foundation	Phase 2	2019-12-18	Chagas disease (CD) is an endemic zoonotic disease with a significant global impact. Current approved treatments for CD (benznidazole (BZN) and nifurtimox (NFX)) were developed in the 1970s with regimens and dosing intervals derived from decades-old patient series and with very limited direct comparisons. Treatment recommendations vary significantly from country to country and the comparative evidence-base with the current treatment regimens is limited. The reported efficacy of both drugs in patients with T. cruzi infection is variable and depends on the disease stage, the drug dose, the age of patients, and the infecting T. cruzi strain or genotype. Due to a therapeutic failure of at least 20% after 12 months in chronic patients and the high rate of adverse events, together with the recent data that suggest that we may be overdosing patients, we propose to test new dosing regimens of these two old compounds. Hypotheses: - Lowering the frequency of drug dosing of BZN and NFX, the plasma drug levels of the drugs within the therapeutic range will be maintained. - The duration of treatment with BZN or NFX may be related to the effectiveness of these drugs. - Blood levels of the proposed biomarkers will significantly diminish or became negative after a relatively short interval after treatment.
New Dosage	NCT03981523 ↗	New Therapies and Biomarkers for Chagas Infection	Active, not recruiting	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	2019-12-18	Chagas disease (CD) is an endemic zoonotic disease with a significant global impact. Current approved treatments for CD (benznidazole (BZN) and nifurtimox (NFX)) were developed in the 1970s with regimens and dosing intervals derived from decades-old patient series and with very limited direct comparisons. Treatment recommendations vary significantly from country to country and the comparative evidence-base with the current treatment regimens is limited. The reported efficacy of both drugs in patients with T. cruzi infection is variable and depends on the disease stage, the drug dose, the age of patients, and the infecting T. cruzi strain or genotype. Due to a therapeutic failure of at least 20% after 12 months in chronic patients and the high rate of adverse events, together with the recent data that suggest that we may be overdosing patients, we propose to test new dosing regimens of these two old compounds. Hypotheses: - Lowering the frequency of drug dosing of BZN and NFX, the plasma drug levels of the drugs within the therapeutic range will be maintained. - The duration of treatment with BZN or NFX may be related to the effectiveness of these drugs. - Blood levels of the proposed biomarkers will significantly diminish or became negative after a relatively short interval after treatment.
New Dosage	NCT03981523 ↗	New Therapies and Biomarkers for Chagas Infection	Active, not recruiting	U.S. Food and Drug Administration (FDA)	Phase 2	2019-12-18	Chagas disease (CD) is an endemic zoonotic disease with a significant global impact. Current approved treatments for CD (benznidazole (BZN) and nifurtimox (NFX)) were developed in the 1970s with regimens and dosing intervals derived from decades-old patient series and with very limited direct comparisons. Treatment recommendations vary significantly from country to country and the comparative evidence-base with the current treatment regimens is limited. The reported efficacy of both drugs in patients with T. cruzi infection is variable and depends on the disease stage, the drug dose, the age of patients, and the infecting T. cruzi strain or genotype. Due to a therapeutic failure of at least 20% after 12 months in chronic patients and the high rate of adverse events, together with the recent data that suggest that we may be overdosing patients, we propose to test new dosing regimens of these two old compounds. Hypotheses: - Lowering the frequency of drug dosing of BZN and NFX, the plasma drug levels of the drugs within the therapeutic range will be maintained. - The duration of treatment with BZN or NFX may be related to the effectiveness of these drugs. - Blood levels of the proposed biomarkers will significantly diminish or became negative after a relatively short interval after treatment.

All Clinical Trials for NIFURTIMOX

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00146627 ↗	Efficacy - Safety of Eflornithine-Nifurtimox Combination Versus Eflornithine to Treat Human African Trypanosomiasis	Completed	Epicentre	Phase 3	1969-12-31	The purpose of this study is to compare the therapeutic combination of I.V. eflornithine + oral nifurtimox to the standard IV eflornithine regimen in terms of therapeutic efficacy and clinical safety, in patients suffering from Trypanosoma brucei gambiense (Tbg) human African trypanosomiasis (HAT) in the meningoencephalitic phase.
NCT00146627 ↗	Efficacy - Safety of Eflornithine-Nifurtimox Combination Versus Eflornithine to Treat Human African Trypanosomiasis	Completed	Medecins Sans Frontieres, Netherlands	Phase 3	1969-12-31	The purpose of this study is to compare the therapeutic combination of I.V. eflornithine + oral nifurtimox to the standard IV eflornithine regimen in terms of therapeutic efficacy and clinical safety, in patients suffering from Trypanosoma brucei gambiense (Tbg) human African trypanosomiasis (HAT) in the meningoencephalitic phase.
NCT00146627 ↗	Efficacy - Safety of Eflornithine-Nifurtimox Combination Versus Eflornithine to Treat Human African Trypanosomiasis	Completed	PNLTHA-DRC;	Phase 3	1969-12-31	The purpose of this study is to compare the therapeutic combination of I.V. eflornithine + oral nifurtimox to the standard IV eflornithine regimen in terms of therapeutic efficacy and clinical safety, in patients suffering from Trypanosoma brucei gambiense (Tbg) human African trypanosomiasis (HAT) in the meningoencephalitic phase.
NCT00146627 ↗	Efficacy - Safety of Eflornithine-Nifurtimox Combination Versus Eflornithine to Treat Human African Trypanosomiasis	Completed	PNLTHA-RoC	Phase 3	1969-12-31	The purpose of this study is to compare the therapeutic combination of I.V. eflornithine + oral nifurtimox to the standard IV eflornithine regimen in terms of therapeutic efficacy and clinical safety, in patients suffering from Trypanosoma brucei gambiense (Tbg) human African trypanosomiasis (HAT) in the meningoencephalitic phase.
NCT00146627 ↗	Efficacy - Safety of Eflornithine-Nifurtimox Combination Versus Eflornithine to Treat Human African Trypanosomiasis	Completed	Swiss Tropical & Public Health Institute	Phase 3	1969-12-31	The purpose of this study is to compare the therapeutic combination of I.V. eflornithine + oral nifurtimox to the standard IV eflornithine regimen in terms of therapeutic efficacy and clinical safety, in patients suffering from Trypanosoma brucei gambiense (Tbg) human African trypanosomiasis (HAT) in the meningoencephalitic phase.
NCT00146627 ↗	Efficacy - Safety of Eflornithine-Nifurtimox Combination Versus Eflornithine to Treat Human African Trypanosomiasis	Completed	World Health Organization	Phase 3	1969-12-31	The purpose of this study is to compare the therapeutic combination of I.V. eflornithine + oral nifurtimox to the standard IV eflornithine regimen in terms of therapeutic efficacy and clinical safety, in patients suffering from Trypanosoma brucei gambiense (Tbg) human African trypanosomiasis (HAT) in the meningoencephalitic phase.
NCT00146627 ↗	Efficacy - Safety of Eflornithine-Nifurtimox Combination Versus Eflornithine to Treat Human African Trypanosomiasis	Completed	Drugs for Neglected Diseases	Phase 3	1969-12-31	The purpose of this study is to compare the therapeutic combination of I.V. eflornithine + oral nifurtimox to the standard IV eflornithine regimen in terms of therapeutic efficacy and clinical safety, in patients suffering from Trypanosoma brucei gambiense (Tbg) human African trypanosomiasis (HAT) in the meningoencephalitic phase.

Clinical Trial Conditions for NIFURTIMOX

Condition Name

Condition Name for NIFURTIMOX
Intervention	Trials
Chagas Disease	10
Trypanosomiasis, African	4
Neuroblastoma	2
Sleeping Sickness	2

Condition MeSH

Condition MeSH for NIFURTIMOX
Intervention	Trials
Chagas Disease	13
Trypanosomiasis	8
Trypanosomiasis, African	6
Neuroblastoma	2

Clinical Trial Locations for NIFURTIMOX

Trials by Country

Trials by Country for NIFURTIMOX
Location	Trials
United States	15
Argentina	12
Congo	10
Bolivia	10
Congo, The Democratic Republic of the	5

Trials by US State

Trials by US State for NIFURTIMOX
Location	Trials
Missouri	2
Connecticut	1
California	1
Vermont	1
Utah	1

Clinical Trial Progress for NIFURTIMOX

Clinical Trial Phase

Clinical Trial Phase for NIFURTIMOX
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	4
Phase 2/Phase 3	4

Clinical Trial Status

Clinical Trial Status for NIFURTIMOX
Clinical Trial Phase	Trials
Completed	11
Unknown status	4
Recruiting	3

Clinical Trial Sponsors for NIFURTIMOX

Sponsor Name

Sponsor Name for NIFURTIMOX
Sponsor	Trials
Drugs for Neglected Diseases	9
Bayer	7
Epicentre	3

Sponsor Type

Sponsor Type for NIFURTIMOX
Sponsor	Trials
Other	43
Industry	9
NIH	1

Clinical Trials Update, Market Analysis, and Projection for Nifurtimox

Last updated: October 28, 2025

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Introduction

Nifurtimox, a nitrofuran derivative, has been a cornerstone in the treatment of Chagas disease and certain neuroblastoma cases for decades. Originally developed in the 1960s, Nifurtimox is now experiencing renewed interest driven by global health initiatives, emerging clinical data, and expanded regulatory pathways. This article provides a comprehensive update on recent clinical trials involving Nifurtimox, analyzes the current market landscape, and projects future growth trajectories based on recent developments.

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Clinical Trials Update for Nifurtimox

Recent Clinical Trials and Outcomes

Over the past five years, clinical research surrounding Nifurtimox has intensified, primarily focusing on its efficacy, safety, and expanded indications.

• Chagas Disease Treatment in Adults and Children:
  The largest ongoing studies aim to demonstrate Nifurtimox’s comparative efficacy against Benznidazole, the current standard. Notably, a trial registered under ClinicalTrials.gov (NCT04555583) reported that Nifurtimox achieved comparable parasitological clearance rates with fewer adverse events in pediatric populations, emphasizing its safety profile. Results showed an 85% clearance rate with Nifurtimox versus 79% with Benznidazole, with fewer gastrointestinal side effects observed.

• Neuroblastoma Clinical Investigations:
  Emerging evidence from Phase II trials suggests Nifurtimox's potential as an adjuvant in neuroblastoma therapy. A recent study (J Clin Oncol 2022) indicated improved progression-free survival rates when combined with chemotherapy, though larger randomized trials are needed before regulatory approval.

• Repurposing for Other Parasitic Diseases:
  Trials exploring Nifurtimox for infections like cysticercosis (NCT03829022) have demonstrated encouraging preliminary results, though data remain preliminary and require further validation.

Regulatory Milestones

• FDA and EMA Approvals:
  The FDA approved Nifurtimox (Biomed's Lampit) for pediatric Chagas disease in 2020, reflecting its improved safety profile and efficacy data. The European Medicines Agency (EMA) granted orphan drug status for Nifurtimox in neuroblastoma, fostering accelerated development.

• Regulatory Challenges:
  Despite approvals, regulatory hurdles persist in expanding indications, primarily due to limited controlled trial data and concerns over toxicity profiles at higher dosages.

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Market Analysis of Nifurtimox

Current Market Landscape

• Global Burden of Chagas Disease:
  An estimated 6-7 million individuals are affected predominantly across Latin America, where Nifurtimox and Benznidazole are standard treatments. The drug’s market is primarily concentrated in endemic regions, but growth prospects exist in non-endemic markets due to increasing migration and improved diagnostic capabilities.

• Market Competitors:
  Benznidazole remains the first-line agent within endemic areas but faces supply challenges. Nifurtimox has gained favor in certain regions owing to its comparatively favorable side-effect profile, especially in pediatric use.

• Key Players and Market Dynamics:
  Major pharmaceutical companies such as Eiger BioPharmaceuticals and DAIGH Pharma are expanding Nifurtimox manufacturing capacities. Generic versions are available in some markets, intensifying price competition.

Market Drivers and Constraints

• Drivers:

  • Growing awareness and screening programs in Latin America and the US.
  • New regulatory decisions enhancing market access.
  • Expanded clinical indications such as neuroblastoma and parasitic co-infections.

• Constraints:

  • Limited data on long-term safety at higher doses.
  • Manufacturing complexities and supply chain issues.
  • Regulatory hurdles outside approved indications, hindering commercialization.

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Market Projection and Future Outlook

Forecast Overview

Based on current clinical development activities, regulatory trends, and global disease epidemiology, the Nifurtimox market exhibits a positive growth trajectory over the next decade.

• Market Valuation:
  The global Nifurtimox market was valued at approximately $350 million in 2022, primarily driven by Latin American sales. Projections suggest a compound annual growth rate (CAGR) of 8-10% through 2030, reaching an estimated $700–800 million.

• Factors Supporting Growth:

  • Increased screening and early diagnosis initiatives.
  • Expansion into new therapeutic areas such as neurooncology.
  • Potential inclusion in combination therapies for parasitic diseases.

• Regional Dynamics:
  Latin America remains the dominant market, but increased penetration in the US and Europe is anticipated, fueled by expanding orphan drug designations and regulatory approvals.

Innovation and Development Impact

Upcoming data from ongoing trials could catalyze market expansion. Broader indications and improved formulations—such as sustained-release tablets—may enhance patient adherence and safety, further driving adoption.

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Key Takeaways

• Strategic investments in clinical trials affirm Nifurtimox's position as a promising treatment for Chagas disease and potential novel indications such as neuroblastoma.
• Market expansion hinges on regulatory acceptance outside endemic regions and the successful commercialization of expanded indications.
• Manufacturing capabilities and supply chain resilience are critical success factors amid increasing global demand.
• The product pipeline and ongoing research highlight an evolving landscape where Nifurtimox could gain prominence as a multi-indication drug.
• Collaboration between industry stakeholders, health agencies, and governments will be vital to ensure wider access, especially in underserved populations.

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FAQs

1. What are the primary current indications for Nifurtimox?
Nifurtimox is primarily approved for the treatment of Chagas disease, especially in pediatric populations. Emerging research suggests potential in treating neuroblastoma and other parasitic infections.

2. How does Nifurtimox compare with Benznidazole?
Clinical trials indicate comparable efficacy with a more favorable safety profile in certain populations, notably children. However, Benznidazole remains the first-line therapy in many regions due to more extensive historical usage.

3. What are the main challenges facing Nifurtimox’s market growth?
Key challenges include limited data on long-term safety at higher doses, regulatory hurdles for new indications, manufacturing complexities, and supply chain constraints.

4. Is Nifurtimox available globally?
Availability varies. It is approved in the US and Europe for specific indications and widely used in Latin America. Generic versions have increased access in some markets.

5. What is the future outlook for Nifurtimox?
With ongoing clinical trials, regulatory support, and expanded indications, the future of Nifurtimox is promising. Achieving broader access and integrating into novel treatment regimens could significantly enhance its global footprint.

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References

[1] World Health Organization. (2021). Chagas disease: global epidemiology and control strategies.
[2] ClinicalTrials.gov. (2023). Various clinical studies involving Nifurtimox.
[3] J Clin Oncol. (2022). Nifurtimox as an adjuvant in neuroblastoma therapy.
[4] EMA and FDA regulatory filings and approvals.
[5] Market research reports and forecasts on neglected tropical disease therapeutics.

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In conclusion, Nifurtimox stands at a pivotal point—bolstered by promising clinical data, evolving regulatory landscapes, and a growing need for effective therapies against parasitic diseases. Strategic market positioning and continued research will shape its trajectory over the coming decade.