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CLINICAL TRIALS PROFILE FOR LAMPIT

505(b)(2) Clinical Trials for LAMPIT

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.

All Clinical Trials for LAMPIT

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00601003 ↗	Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma	Active, not recruiting	Bayer	Phase 2	2008-02-01	The purpose of this study is to determine whether nifurtimox in combination with cyclophosphamide and topotecan are effective in the treatment of relapsed or refractory neuroblastoma and medulloblastoma.
NCT00601003 ↗	Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma	Active, not recruiting	Giselle SaulnierSholler	Phase 2	2008-02-01	The purpose of this study is to determine whether nifurtimox in combination with cyclophosphamide and topotecan are effective in the treatment of relapsed or refractory neuroblastoma and medulloblastoma.
NCT00601003 ↗	Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma	Active, not recruiting	Giselle Sholler	Phase 2	2008-02-01	The purpose of this study is to determine whether nifurtimox in combination with cyclophosphamide and topotecan are effective in the treatment of relapsed or refractory neuroblastoma and medulloblastoma.
NCT01685827 ↗	Pivotal Study of Fexinidazole for Human African Trypanosomiasis in Stage 2	Completed	Drugs for Neglected Diseases	Phase 2/Phase 3	2012-10-01	This clinical trial is designed to prove the efficacy and safety of Fexinidazole as an oral treatment for human african trypanosomiasis in advanced stage. The Fexinidazole is compared to reference treatment NECT. The trial will try to demonstrate that Fexinidazole is not inferior to NECT treatment.
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.
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.

Clinical Trial Conditions for LAMPIT

Condition Name

Condition Name for LAMPIT
Intervention	Trials
Trypanosoma Cruzi Infection	1
Chagas Disease	1
Human African Trypanosomiasis (HAT)	1
Medulloblastoma	1

Condition MeSH

Condition MeSH for LAMPIT
Intervention	Trials
Chagas Disease	1
Trypanosomiasis, African	1
Trypanosomiasis	1
Neuroblastoma	1

Clinical Trial Locations for LAMPIT

Trials by Country

Trials by Country for LAMPIT
Location	Trials
United States	13
Congo	6
Bolivia	3
Congo, The Democratic Republic of the	1
Central African Republic	1

Trials by US State

Trials by US State for LAMPIT
Location	Trials
Hawaii	1
Florida	1
Connecticut	1
California	1
Utah	1

Clinical Trial Progress for LAMPIT

Clinical Trial Phase

Clinical Trial Phase for LAMPIT
Clinical Trial Phase	Trials
Phase 2/Phase 3	1
Phase 2	2

Clinical Trial Status

Clinical Trial Status for LAMPIT
Clinical Trial Phase	Trials
Active, not recruiting	2
Completed	1

Clinical Trial Sponsors for LAMPIT

Sponsor Name

Sponsor Name for LAMPIT
Sponsor	Trials
Drugs for Neglected Diseases	2
Barcelona Institute for Global Health	1
Fundación Ciencia y Estudios Aplicados para el Desarrollo en Salud y Medio Ambiente (CEADES)	1

Sponsor Type

Sponsor Type for LAMPIT
Sponsor	Trials
Other	10
NIH	1
Industry	1

LAMPIT Market Analysis and Financial Projection

LAMPIT (Nifurtimox) for Chagas Disease: Clinical Trials, Market Analysis, and Projections

Introduction to Chagas Disease and LAMPIT

Chagas disease, also known as American trypanosomiasis, is a tropical parasitic disease caused by the parasite Trypanosoma cruzi. It affects approximately 6 to 7 million people globally, with a significant presence in Latin America[2].

LAMPIT, or nifurtimox, is a crucial antiprotozoal medication that has recently gained FDA approval for the treatment of Chagas disease in pediatric patients. Here, we delve into the clinical trials, market analysis, and future projections for LAMPIT.

Clinical Trials Overview

The FDA approval of LAMPIT was based on a randomized, double-blind, prospective phase three clinical trial conducted at 25 investigating sites in Colombia, Bolivia, and Argentina. This trial involved 330 pediatric patients aged less than 18 years and weighing at least 2.5 kg[1][4][5].

Trial Design and Outcomes

• Treatment Regimens: Patients were randomized at a 2:1 ratio to receive either a 60-day or 30-day nifurtimox treatment regimen. They were followed up for one year to assess the efficacy and safety of the drug.
• Efficacy Results: The 60-day treatment arm showed superior results compared to the 30-day arm. The serological response, measured using lysate ELISA and recombinant ELISA, was 32% and 35% respectively in the 60-day arm, compared to 19% and 22% in the 30-day arm[1][4].
• Side Effects: Common side effects observed included abdominal pain, vomiting, diarrhea, decreased appetite, nausea, anemia, eosinophilia, decreased weight, urticaria, dizziness, pyrexia, rash, and headache[1][4].

FDA Approval and Regulatory Status

LAMPIT received accelerated approval from the FDA in August 2020 for the treatment of Chagas disease in children from birth to less than 18 years of age and weighing at least 2.5 kg. This approval was granted under the FDA’s accelerated approval program, which allows for earlier patient access to promising new drugs while the company continues to conduct clinical trials to confirm efficacy and safety[2][4][5].

Orphan Drug Designation

LAMPIT also holds orphan drug designation from the FDA, which provides incentives for the development of drugs for rare diseases[1].

Market Analysis

Current Market Presence

LAMPIT is currently registered in several Latin American countries, including Argentina, Uruguay, Chile, El Salvador, Guatemala, and Honduras, for the treatment of acute and chronic Chagas disease in adults and children[1][5].

Global Need and Market Potential

Given the significant global burden of Chagas disease, LAMPIT fills a critical gap in the treatment options available, especially for pediatric patients. The drug's approval and availability are expected to improve access to treatment, particularly in regions where the disease is endemic.

Competitive Landscape

LAMPIT is one of only two drugs available globally for the treatment of Chagas disease. This limited competition, combined with the drug's efficacy and the growing need for effective treatments, positions LAMPIT favorably in the market[5].

Future Projections

Ongoing and Future Clinical Trials

Bayer will continue with the second part of the LAMPIT program to confirm the drug's efficacy and safety over an additional three years. This ongoing research will further solidify LAMPIT's place in the treatment landscape for Chagas disease[2].

Expansion of Access

Bayer is working to expand the number of countries with registrations for LAMPIT, aiming to improve global access to this critical medication. This expansion is expected to increase the drug's market reach and impact[5].

Economic Impact

The approval and increasing availability of LAMPIT are likely to have a positive economic impact, particularly in regions heavily affected by Chagas disease. By reducing the morbidity and mortality associated with the disease, LAMPIT can contribute to improved public health and economic productivity.

Key Takeaways

• Clinical Trials: LAMPIT's FDA approval was based on a phase three clinical trial showing superior efficacy with a 60-day treatment regimen.
• Regulatory Status: LAMPIT has received accelerated approval and orphan drug designation from the FDA.
• Market Presence: Currently registered in several Latin American countries, with plans for further global expansion.
• Market Potential: Significant due to the global burden of Chagas disease and limited treatment options.
• Future Projections: Ongoing clinical trials and expansion of access are expected to enhance LAMPIT's market presence and impact.

FAQs

What is LAMPIT used for?

LAMPIT (nifurtimox) is used for the treatment of Chagas disease, a tropical parasitic disease caused by Trypanosoma cruzi, in pediatric patients from birth to less than 18 years of age and weighing at least 2.5 kg[2][5].

What were the key findings of the clinical trials for LAMPIT?

The clinical trials showed that a 60-day treatment regimen of LAMPIT resulted in a higher serological response compared to a 30-day regimen, with 32% and 35% response rates using lysate ELISA and recombinant ELISA, respectively[1][4].

What are the common side effects of LAMPIT?

Common side effects include abdominal pain, vomiting, diarrhea, decreased appetite, nausea, anemia, eosinophilia, decreased weight, urticaria, dizziness, pyrexia, rash, and headache[1][4].

Is LAMPIT available globally?

Currently, LAMPIT is registered in several Latin American countries. Bayer is working to expand its registration to more countries to improve global access[1][5].

What is the significance of LAMPIT's FDA approval?

LAMPIT's FDA approval marks the first and only treatment approved in the U.S. for Chagas disease in children, highlighting its importance in addressing a significant public health need[2][5].

Sources

1. Clinical Trials Arena: Lampit (nifurtimox) for the Treatment of Chagas Disease.
2. S&P Global: Bayer's Chagas disease drug Lampit wins US FDA approval for pediatric use.
3. Business Wire: Claudin 18.2 Targeted Therapy Market Forecast & Clinical Trials Insights Report 2024-2029.
4. FDA: Drug Trials Snapshots: LAMPIT.
5. BioSpace: U.S. Food and Drug Administration Approves Lampit® (nifurtimox) for the Treatment of Chagas Disease in Children.