CLINICAL TRIALS PROFILE FOR BENZNIDAZOLE

505(b)(2) Clinical Trials for Benznidazole

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.

All Clinical Trials for Benznidazole

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00123916 ↗	BENEFIT: Evaluation of the Use of Antiparasital Drug (Benznidazole) in the Treatment of Chronic Chagas' Disease	Completed	Canadian Institutes of Health Research (CIHR)	Phase 3	2004-11-01	Evaluate if benznidazole, an antiparasite drug, given at a dose calculated as 5mg/kg/day for 60 days, now administered as a fixed daily dose of 300mg during 40 to 80 days of treatment - period adjusted according to the patient's body weight to a total minimum dose of 12g (corresponding to 40kg) and a total maximum dose of 24g (corresponding to 80kg) - reduces morbidity and mortality in patients with Chronic Chagas' Cardiomyopathy (CCC). The BENEFIT study is being conducted by the Population Health Research Institute (in Hamilton, Canada) and the Institute Dante Pazzanese de Cardiologia (Sao Paulo, Brazil) together with a Steering Committee, and an independent Safety Monitoring Board.
NCT00123916 ↗	BENEFIT: Evaluation of the Use of Antiparasital Drug (Benznidazole) in the Treatment of Chronic Chagas' Disease	Completed	Instituto Dante Pazzanese de Cardiologia	Phase 3	2004-11-01	Evaluate if benznidazole, an antiparasite drug, given at a dose calculated as 5mg/kg/day for 60 days, now administered as a fixed daily dose of 300mg during 40 to 80 days of treatment - period adjusted according to the patient's body weight to a total minimum dose of 12g (corresponding to 40kg) and a total maximum dose of 24g (corresponding to 80kg) - reduces morbidity and mortality in patients with Chronic Chagas' Cardiomyopathy (CCC). The BENEFIT study is being conducted by the Population Health Research Institute (in Hamilton, Canada) and the Institute Dante Pazzanese de Cardiologia (Sao Paulo, Brazil) together with a Steering Committee, and an independent Safety Monitoring Board.
NCT00123916 ↗	BENEFIT: Evaluation of the Use of Antiparasital Drug (Benznidazole) in the Treatment of Chronic Chagas' Disease	Completed	University of Sao Paulo	Phase 3	2004-11-01	Evaluate if benznidazole, an antiparasite drug, given at a dose calculated as 5mg/kg/day for 60 days, now administered as a fixed daily dose of 300mg during 40 to 80 days of treatment - period adjusted according to the patient's body weight to a total minimum dose of 12g (corresponding to 40kg) and a total maximum dose of 24g (corresponding to 80kg) - reduces morbidity and mortality in patients with Chronic Chagas' Cardiomyopathy (CCC). The BENEFIT study is being conducted by the Population Health Research Institute (in Hamilton, Canada) and the Institute Dante Pazzanese de Cardiologia (Sao Paulo, Brazil) together with a Steering Committee, and an independent Safety Monitoring Board.
NCT00123916 ↗	BENEFIT: Evaluation of the Use of Antiparasital Drug (Benznidazole) in the Treatment of Chronic Chagas' Disease	Completed	World Health Organization	Phase 3	2004-11-01	Evaluate if benznidazole, an antiparasite drug, given at a dose calculated as 5mg/kg/day for 60 days, now administered as a fixed daily dose of 300mg during 40 to 80 days of treatment - period adjusted according to the patient's body weight to a total minimum dose of 12g (corresponding to 40kg) and a total maximum dose of 24g (corresponding to 80kg) - reduces morbidity and mortality in patients with Chronic Chagas' Cardiomyopathy (CCC). The BENEFIT study is being conducted by the Population Health Research Institute (in Hamilton, Canada) and the Institute Dante Pazzanese de Cardiologia (Sao Paulo, Brazil) together with a Steering Committee, and an independent Safety Monitoring Board.

Clinical Trial Conditions for Benznidazole

Condition Name

Condition Name for Benznidazole
Intervention	Trials
Chagas Disease	18
Trypanosoma Cruzi Infection	2
Chronic Chagas Disease	2
South American Trypanosomiasis	1

Condition MeSH

Condition MeSH for Benznidazole
Intervention	Trials
Chagas Disease	22
Trypanosomiasis	3
Inflammation	1
Infections	1

Clinical Trial Locations for Benznidazole

Trials by Country

Trials by Country for Benznidazole
Location	Trials
Argentina	22
Brazil	18
Bolivia	10
Colombia	8
Spain	5

Trials by US State

Trials by US State for Benznidazole
Location	Trials
Texas	1
Massachusetts	1
Louisiana	1
California	1

Clinical Trial Progress for Benznidazole

Clinical Trial Phase

Clinical Trial Phase for Benznidazole
Clinical Trial Phase	Trials
PHASE3	1
PHASE2	1
Phase 4	3

Clinical Trial Status

Clinical Trial Status for Benznidazole
Clinical Trial Phase	Trials
Completed	11
Recruiting	5
Unknown status	5

Clinical Trial Sponsors for Benznidazole

Sponsor Name

Sponsor Name for Benznidazole
Sponsor	Trials
Drugs for Neglected Diseases	8
Hospital Universitari Vall d'Hebron Research Institute	2
Barcelona Centre for International Health Research	2

Sponsor Type

Sponsor Type for Benznidazole
Sponsor	Trials
Other	52
Industry	7
NIH	1

Benznidazole: Clinical Trial Update, Market Analysis, and Projection

Last updated: April 23, 2026

What is the current clinical trial posture for benznidazole?

Benznidazole is an established antiparasitic for Chagas disease (American trypanosomiasis). Its clinical development today is dominated by new regimens, pediatric positioning, pharmacokinetic (PK) refinements, and combination strategies rather than new single-agent regulatory starts. Trial activity is concentrated in endemic geographies and in operational studies that target treatment uptake and dosing optimization.

How trials are being structured

Across registries, benznidazole programs generally cluster into these trial archetypes:

1. Pediatric and adolescent access and dosing optimization
   • Focus: age-specific dosing confirmation, safety monitoring, and exposure targets.
2. Combination therapy and regimen shortening
   • Focus: improved cure rates and/or reduced toxicity.
3. PK, exposure-response, and adherence-focused studies
   • Focus: ensuring consistent exposure in real-world treatment settings.
4. Retreatment and late-stage disease evaluation
   • Focus: outcomes in chronic infection subsets where baseline burden varies.

What endpoints trials emphasize

Common endpoints used in current benznidazole work are:

• Parasitological conversion / serologic conversion (typically assessed over extended follow-up windows in chronic disease)
• Time-to-negative serology or sustained serologic response
• Treatment-emergent adverse events and discontinuation rates
• Exposure measures (drug levels, adherence proxies)
• ECG/neurologic safety surveillance where toxicity signals require tight monitoring

Operational note

Benznidazole’s ongoing clinical activity is constrained by the long timelines of chronic Chagas endpoints (serology conversion and sustained response), which tends to slow readouts and stretch follow-up.

Where does benznidazole sit in the competitive treatment landscape?

Benznidazole competes primarily for Chagas disease treatment in endemic markets where public-sector procurement and national treatment guidelines drive volumes. Key competitive dimensions are:

• Efficacy in chronic vs acute phases
• Tolerability and discontinuation rates
• Supply reliability and procurement pricing
• Ease of dosing in pediatric and outpatient settings

Main competitive comparators

• Nifurtimox: primary comparator historically used in some regions.
• “New” Chagas entrants: fewer direct registered late-stage options for broad uptake; most novel pipelines still face regulatory and adoption lags.

Differentiation that matters commercially

For procurement-driven markets, differentiation that moves unit volumes tends to be:

• Lower discontinuation rates at tolerable dosing
• Clear guideline alignment for age groups and disease stage
• Stable supply and bulk pricing through established manufacturers

What is the market size direction for benznidazole?

Benznidazole’s market is tied to the number of patients diagnosed and treated for Chagas disease, which depends on:

• national screening programs and case finding,
• guideline updates and clinician adoption,
• procurement budgets and tender cycles,
• and the logistics of delivering multi-week therapy.

Demand drivers

• Endemic disease burden drives baseline demand, but treated-case conversion is typically below epidemiologic prevalence due to underdiagnosis.
• Guideline incorporation (acute and early chronic treatment recommendations) drives seasonal and programmatic procurement.
• Pediatric treatment expansion creates incremental demand where pediatric dosing protocols are standardized.

Supply-side dynamics

• Benznidazole is generally generic-market dominated in many countries, which compresses pricing and ties revenue to:
  • tender terms,
  • import and local distribution capability,
  • and quality consistency for public procurement.

Pricing and channel

• The market behaves like a public procurement and donor-backed procurement ecosystem in many endemic countries.
• Private-sector uptake exists but is smaller and typically more price elastic.

How should revenue and unit demand be projected?

A robust projection for benznidazole depends on treated-case growth and sustained procurement in endemic programs, with headwinds from:

• diagnostic bottlenecks,
• treatment adherence and discontinuation,
• and budget constraints tied to disease program financing.

A practical projection framework is anchored to three levers:

1. Diagnosed-and-treated conversions (screening and referral efficiency)
2. Target population expansion (pediatric and earlier chronic stages)
3. Procurement continuity and tender timing (supply reliability)

Base case vs downside vs upside (structure)

• Base case: steady growth aligned with incremental diagnosis improvements and guideline-driven uptake, offset by pricing pressure from generics.
• Downside: slower conversion of diagnosed cases into treated cases due to system constraints or tighter procurement.
• Upside: faster pediatric and programmatic scale-up plus stable supply that supports larger tender volumes.

Expected evolution of pricing

• Because benznidazole is widely generic, commercial upside is limited by pricing competition.
• Revenue growth tends to be unit-volume-led, not price-led.

What investment-relevant signals should be monitored?

For companies exposed to benznidazole supply, licensing, or platform adjacencies (combination opportunities, pediatric formulations), monitor:

• Public procurement tender volumes and contract renewals in major endemic markets
• Guideline changes that expand eligible disease stage or age bands
• Adoption of simplified regimens if supported by clinical data
• Safety signal management (discontinuation and adverse event handling) that affects throughput and patient completion rates
• Generic entry in procurement channels that compresses pricing further

What are the key regulatory and label-relevant considerations?

Benznidazole’s core regulatory posture is anchored in its established indication for Chagas disease, with label-structure differences across jurisdictions shaped by:

• approved age groups,
• disease stage specificity,
• and dosing/safety monitoring requirements.

For commercial planning, the critical fact pattern is that benznidazole is not a “label expansion-only” story in most markets; growth is driven by program uptake and patient identification, not new marketing authorization in mature jurisdictions.

Key Takeaways

• Benznidazole’s clinical activity is concentrated on pediatric positioning, dosing/PK refinement, and combination or regimen work, with long timelines driven by chronic Chagas endpoints.
• The market is procurement-driven and structurally generic-competitive, making revenue growth most sensitive to treated-case volume rather than price.
• Projection should be modeled off diagnosed-and-treated conversion, pediatric/early chronic uptake, and procurement continuity, with pricing pressure as the dominant constraint on ASP.
• Investment and R&D planning should track tender volumes, guideline adoption, safety-driven completion rates, and supply stability rather than expecting near-term pricing upside.

FAQs

1) Is benznidazole still actively studied in clinical trials?
Yes. Current activity is largely operational and translational, emphasizing dosing, pediatric populations, and regimen optimization rather than novel first-in-class approvals.

2) What determines time to readout in benznidazole Chagas trials?
Chronic Chagas endpoints rely on serologic conversion and sustained response, which require extended follow-up.

3) What drives benznidazole demand commercially?
Demand is driven by national and program-level treated-case volumes, which depend on screening, referral pathways, and procurement execution.

4) What is the main competitive pressure in benznidazole markets?
Generic competition and tender pricing dominate, limiting price growth and shifting growth to unit volumes.

5) What safety considerations most affect completion rates?
Adverse events that lead to discontinuation can reduce effective treated volumes, making tolerability management a commercial throughput factor.

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References

[1] World Health Organization. Chagas disease (American trypanosomiasis). WHO fact sheets and technical guidance. https://www.who.int/
[2] FDA (US). Drug Approval Information for benznidazole (where applicable) and product labeling references. https://www.fda.gov/
[3] EMA (European Medicines Agency). Benznidazole-related assessment documents and orphan/antiparasitic regulatory materials (where applicable). https://www.ema.europa.eu/
[4] ClinicalTrials.gov. Search results for benznidazole and Chagas disease studies. https://clinicaltrials.gov/