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
Bolivia	10
Congo	10
Colombia	5

Trials by US State

Trials by US State for nifurtimox
Location	Trials
Missouri	2
North Carolina	1
Minnesota	1
Michigan	1
Massachusetts	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

Nifurtimox Market Analysis and Financial Projection

Nifurtimox: Clinical Trials, Market Analysis, and Projections

Introduction

Nifurtimox is a crucial antiparasitic drug used in the treatment of Chagas disease, a serious and sometimes life-threatening illness caused by the parasite Trypanosoma cruzi. Here, we will delve into the latest clinical trials, market analysis, and projections for nifurtimox.

Clinical Trials Update

Efficacy and Safety in Pediatric Patients

A recent study published in 2023 confirmed the effectiveness and safety of a pediatric formulation of nifurtimox in treating Chagas disease. The CHICO clinical trial involved pediatric patients aged less than 18 years, who were treated with either a 60-day or 30-day regimen of nifurtimox. The results showed that both regimens were safe and effective, with seronegative conversion rates of 8.12% and 8.16% for the 60-day and 30-day regimens, respectively. Children under 2 years at baseline were more likely to achieve seronegative conversion during the 4-year follow-up period[1].

Comparative Trials with Benznidazole

The EQUITY trial, though still ongoing, aims to evaluate the trypanocide effect and safety of nifurtimox (NFX) and benznidazole (BZ) in asymptomatic patients with T. cruzi positive serology. Participants are divided into different treatment schemes, including conventional and shorter treatment durations. The primary outcomes include the rate of positive PCR results and safety evaluations[4].

Long-Term Follow-Up

Studies have shown that nifurtimox treatment leads to significant seroreduction and seroconversion over time. A Phase III trial indicated that approximately 50% of patients reached a serologic response to 60-day nifurtimox treatment by the 4-year follow-up. Younger patients had a higher probability of achieving seroreduction[5].

Market Analysis

Market Share and Growth

The Chagas disease market is dominated by antiparasitic treatments, with nifurtimox and benznidazole being the two key drugs. The nifurtimox segment is expected to grow during the forecast period due to its high efficacy and safety profile. Despite benznidazole holding the largest market share, nifurtimox's benefits are enhancing its adoption and contributing to substantial market growth[2].

Regional Market Growth

The European region is anticipated to grow considerably during the forecast period, driven by a well-established healthcare infrastructure and a favorable regulatory network. This environment is conducive to product launches and market revenue growth[2].

Key Market Players

Several pharmaceutical companies are key players in the Chagas disease market, including Nortec Quimica SA, Bayer AG, Laboratorio Elea Phoenix SA, Novartis Pharmaceuticals, and others. These companies are instrumental in the development, distribution, and marketing of nifurtimox and other treatments for Chagas disease[2].

Projections and Future Outlook

Increasing Demand and Awareness

The future outlook for the Chagas disease market is cautiously optimistic, with ongoing research into new treatments and the development of better diagnostic tools. Increasing global awareness of the disease is expected to drive market growth as more people are diagnosed and treated[2].

New Therapeutic Options

While current treatments like nifurtimox and benznidazole remain effective, new therapeutic options and strategies are being explored. Clinical trials such as the MULTIBENZ and EQUITY trials are evaluating different treatment schemes and dosages to improve outcomes and reduce side effects[4].

Regulatory and Healthcare Infrastructure

The favorable regulatory environment, particularly in regions like Europe, is expected to support the launch of new products and enhance market growth. The expanding healthcare industry and increasing awareness among the public will also contribute to the escalating demand for effective treatments like nifurtimox[2].

Safety and Efficacy Considerations

Toxicity Profile

Nifurtimox has a notable toxicity profile, which led to its commercialization being suspended in several countries in the early 1980s. However, recent studies have shown that when administered in age- and weight-adjusted regimens, nifurtimox is safe and effective, especially in pediatric patients[1][3].

Pharmacokinetics

Nifurtimox undergoes extensive first-pass hepatic metabolism and has little renal excretion. Its pharmacokinetic profile supports its use in various patient populations, including children and adults, when dosed appropriately[3].

Key Takeaways

• Clinical Efficacy: Nifurtimox has been shown to be effective and safe in treating Chagas disease, particularly in pediatric patients.
• Market Growth: The nifurtimox segment is expected to grow due to its high efficacy and safety profile.
• Regional Impact: The European region is anticipated to see significant growth due to its well-established healthcare infrastructure.
• Future Outlook: Ongoing research and increasing global awareness are expected to drive market growth.
• Safety and Efficacy: Nifurtimox, when administered correctly, is safe and effective, despite its historical toxicity concerns.

FAQs

What are the current clinical trials evaluating nifurtimox for Chagas disease?

Current clinical trials, such as the EQUITY and CHICO trials, are evaluating the efficacy and safety of nifurtimox in various patient populations, including pediatric and asymptomatic patients[1][4].

How does nifurtimox compare to benznidazole in treating Chagas disease?

While benznidazole is generally preferred due to its better efficacy profile, nifurtimox has shown significant effectiveness, especially in younger patients. There are no randomized clinical trials directly comparing the two drugs[3].

What is the market projection for nifurtimox in the Chagas disease market?

The nifurtimox segment is expected to grow during the forecast period due to its high efficacy and safety profile, as well as increasing global awareness and demand for effective treatments[2].

What are the safety concerns associated with nifurtimox?

Nifurtimox has a notable toxicity profile, but recent studies have shown that when administered in age- and weight-adjusted regimens, it is safe and effective. Historical concerns led to its commercialization being suspended in some countries[1][3].

How does the pharmacokinetic profile of nifurtimox support its use?

Nifurtimox undergoes extensive first-pass hepatic metabolism and has little renal excretion, supporting its use in various patient populations when dosed appropriately[3].

Sources

1. Efficacy and Safety of Nifurtimox in Pediatric Patients with Chagas Disease. PubMed, 2023.
2. Chagas Disease Market Size, Share, Growth & Analysis, 2032. Market Data Forecast.
3. Trypanocidal treatment of Chagas disease. Elsevier.
4. Clinical trials for Chagas disease: etiological and pathophysiological aspects. Frontiers in Microbiology.
5. Chagas disease treatment efficacy markers. Frontiers in Parasitology.