Observational Pregnancy Safety Study of Women Exposed to Nifurtimox During Pregnancy to Describe the Risk of Pregnancy and Maternal Complications and Other Events of Interest on the Developing Fetus, Neonate, and Infant

This is an observational study in which data from women with Chagas disease who will take or have already taken nifurtimox during pregnancy and the impact on their babies are studied.
Chagas disease is an inflammatory, infectious disease caused by the parasite Trypanosoma cruzi. This parasite is mainly spread by insects called triatomine bug. If Chagas disease is left untreated, it can later cause e.g. serious heart and digestive problems.
Nifurtimox has been used for more than 50 years to treat Chagas disease in children and adults.
It is not recommended to be used during pregnancy as data from animal studies indicate that it may harm the baby. Currently, there are not enough data to know if this is also the case in humans.
In this study, researchers want to collect data on the safety of nifurtimox use in pregnant women. To do this, researchers will collect the following information:
* Birth defects (abnormal and problematic structures or functions, a child is born with)
* Pregnancy outcomes (like live birth, preterm birth, still birth/death of the unborn baby, miscarriage, or abortion)
* Certain health problems of the child up to 12 months of age
* Certain health problems of the women experienced during pregnancy The data will be collected from different sources including telephone calls with the women or their doctor, CRFs (case reprt forms) or from medical records The researchers will compare the proportion of children with birth defects, pregnancy outcomes or certain health problems of the child or the women during pregnancy with available data on these outcomes in the general population.
The study will run for approximately 10 years.

Start Date30 Jun 2025

Sponsor / Collaborator

Bayer AG

CTR20220156

/ RecruitingPhase 1

硝呋莫司片在成年中枢神经系统肿瘤患者中评价耐受性、安全性、药代动力学及初步抗肿瘤活性的Ia期临床试验

[Translation] A phase Ia clinical trial evaluating the tolerability, safety, pharmacokinetics and preliminary antitumor activity of nifurtimox tablets in adult patients with central nervous system tumors

主要目的评价硝呋莫司在经标准治疗后复发或疾病进展、或无标准治疗的成年高级别脑胶质瘤、髓母细胞瘤、脑转移瘤（包括但不限于肺癌脑转移、乳腺癌脑转移、黑色素瘤脑转移等）患者的耐受性、安全性，确定最大耐受剂量（MTD）和/或II期推荐剂量（RP2D）。次要目的 1) 评价硝呋莫司在成年中枢神经系统肿瘤患者中的初步药代动力学特征； 2) 评价硝呋莫司在成年中枢神经系统肿瘤患者中的初步有效性。探索性目的探索硝呋莫司在成年中枢神经系统肿瘤患者中的给药方案。

[Translation]

Primary objective To evaluate the tolerability and safety of nifurtimox in adult patients with high-grade glioma, medulloblastoma, brain metastasis (including but not limited to brain metastasis of lung cancer, brain metastasis of breast cancer, brain metastasis of melanoma, etc.) who have relapsed or progressed after standard treatment or have no standard treatment, and to determine the maximum tolerated dose (MTD) and/or phase II recommended dose (RP2D). Secondary objectives 1) To evaluate the preliminary pharmacokinetic characteristics of nifurtimox in adult patients with central nervous system tumors; 2) To evaluate the preliminary efficacy of nifurtimox in adult patients with central nervous system tumors. Exploratory objectives To explore the dosing regimen of nifurtimox in adult patients with central nervous system tumors.

Start Date23 Feb 2023

Sponsor / Collaborator

Hengtai Jiukang (Tianjin) Pharmaceutical Technology Co., Ltd.

CTR20190136

/ RecruitingPhase 1

硝呋莫司治疗儿童复发或难治性神经母细胞瘤患者的单臂、非随机化、开放、剂量递增、I期临床试验

[Translation] A single-arm, non-randomized, open-label, dose-escalation, phase I trial of nifurtimox in the treatment of pediatric patients with relapsed or refractory neuroblastoma

主要目的评价硝呋莫司单药及联合环磷酰胺及拓扑替康治疗儿童复发或难治性神经母细胞瘤患者的安全性，确定最大耐受剂量及 II 期推荐剂量。次要目的 1) 评价硝呋莫司单药及联合用药的初步药代动力学特征； 2) 评价硝呋莫司联合用药治疗儿童复发或难治性神经母细胞瘤患者的缓解率。探索性目的探索硝呋莫司联合用药的最佳给药方案。

[Translation]

Primary objective To evaluate the safety of nifurtimox alone and in combination with cyclophosphamide and topotecan in the treatment of pediatric patients with relapsed or refractory neuroblastoma, and to determine the maximum tolerated dose and the recommended dose for phase II. Secondary objectives 1) To evaluate the preliminary pharmacokinetic characteristics of nifurtimox alone and in combination with other drugs; 2) To evaluate the remission rate of nifurtimox combined with other drugs in the treatment of pediatric patients with relapsed or refractory neuroblastoma. Exploratory objective To explore the optimal dosing regimen of nifurtimox combined with other drugs.

Start Date20 Oct 2021

Sponsor / Collaborator

Hengtai Jiukang (Tianjin) Pharmaceutical Technology Co., Ltd.

100 Clinical Results associated with Nifurtimox

100 Translational Medicine associated with Nifurtimox

100 Patents (Medical) associated with Nifurtimox

1,303

Literatures (Medical) associated with Nifurtimox

10 Apr 2025·Journal of Medicinal Chemistry

Discovery and Early Optimization of 1H-Indole-2-carboxamides with Anti-Trypanosoma cruzi Activity

Article

Author: Moraes, Carolina B. ; Dichiara, Maria ; Pollastri, Michael P. ; dos Santos, Deborah A. ; Chelucci, Rafael C. ; Dessoy, Marco A. ; Feltrin, Clarissa ; da Silva, Adriana C. ; Ferreira, Rafael A. ; Ferrins, Lori ; Mowbray, Charles E. ; Krogh, Renata ; de Oliveira, Luiz F. N. ; Kratz, Jadel M. ; Cruz, Luiza R. ; Ferreira, Leonardo L. G. ; Duarte, Simone M. ; Dias, Luiz C. ; Lee, Eun ; de Oliveira, Ramon G. ; dos Santos, Benedito M. ; Mollo, María C. ; Sjö, Peter ; Simpson, Quillon J. ; Broering, Milena F. ; Andricopulo, Adriano D. ; Koovits, Paul J.

Chagas disease (CD), caused by the flagellate protozoan Trypanosoma cruzi, is a neglected tropical disease endemic in 21 countries. The only two antiparasitic drugs approved for its treatment, benznidazole and nifurtimox, have significant drawbacks. We present herein the optimization of a series of substituted indoles that were identified through phenotypic screening against T. cruzi. Early lead compounds with balanced potency and physicochemical properties were advanced to animal studies but showed limited plasma exposure. Medicinal chemistry strategies were used to improve metabolic stability and solubility, but unfortunately, this effort failed to yield compounds with improvements in both exposure and potency. Still, the best compound was progressed for a proof-of-concept efficacy study using acute and chronic mice models of Chagas disease. Despite showing antiparasitic activity in these in vivo studies, the optimization work with this series was stopped due to unfavorable drug metabolism and pharmacokinetic (DMPK) properties and a deprioritized mechanism of action (CYP51 inhibition).

13 Mar 2025·Clinical Microbiology Reviews

Transforming the chemotherapy of human African trypanosomiasis

Review

Author: Barrett, Michael P.

SUMMARY Prior to 2019, when the orally available drug fexinidazole began its clinical use, the treatment of human African trypanosomiasis (HAT) was complex and unsatisfactory for many reasons. Two sub-species of the Trypanosoma brucei parasite are responsible for HAT, namely the rhodesiense form found in East and Southern Africa and the gambiense form found in Central and West Africa. Diseases caused by both forms manifest in two stages: stage 1 before and stage 2 after central nervous system involvement. Prior to 2019, different drugs were required for each of the two parasite sub-species at each stage. Gambiense disease required pentamidine or nifurtimox-eflornithine combination therapy, while for rhodesiense disease, suramin or melarsoprol was given for stages 1 and 2, respectively. These drugs all suffered complications including protracted administration regimens involving multiple injections with drug-induced adverse effects common. Today, a single drug, fexinidazole, can be given orally in most cases for both diseases at either stage. Another compound, acoziborole, effective in both stages 1 and 2 gambiense disease with a single dosing is anticipated to become available within a few years. Moreover, the recent engagement of multilateral organizations in seeking other compounds that could be used in HAT therapy has also been successful, and a rich vein of new trypanocides has been discovered. Here, the clinical use, modes of action, and resistance risks for drugs used against HAT are discussed.

01 Feb 2025·Acta Parasitologica

Expression Analysis of Thirteen Genes in Response to Nifurtimox and Benznidazole in Mexican Isolates of Trypanosoma cruzi by Digital PCR

Article

Author: López-Monteon, Aracely ; Ochoa-Martínez, Paulina ; Ramos-Ligonio, Angel ; Manning-Cela, Rebeca Georgina ; López-Domínguez, Jaime

Despite being the most relevant and critical option for managing Chagas disease, pharmacological therapy is currently limited by the availability of only two drugs, benznidazole and nifurtimox. Their effectiveness is further restricted in the chronic phase of the infection, as they induce severe side effects and require prolonged treatment. Additionally, the use of these drugs can lead to the emergence of substantial resistance problems, compounded by the potential natural resistance of some parasite isolates. This study analyzes the expression of 13 genes by digital PCR in four Mexican T. cruzi isolates treated with NFX and BZN. Each isolate exhibited a unique combination of enzyme expression in response to the oxidative stress induced by the antichagasic agents. Notably, we observed the overexpression of cruzipain (CZP), L-threonine dehydrogenase (TDH), and detoxification-related enzymes such as Glutathionyl spermidine synthetase (GST) and Superoxide dismutase-A (SOD). These findings highlight the need for further studies to elucidate the molecular mechanisms underlying this resistance, which pose both unexpected challenges for Chagas disease therapy and a biological barrier to the action of these drugs. These findings highlight the need for further studies to understand how these resistance mechanisms contribute to treatment failure and constitute a biological barrier to drug action.

News (Medical) associated with Nifurtimox

16 Apr 2024

·www.prnewswire.com

Health Alert: Chagas Disease, Also Known as American Trypanosomiasis, Poses Life-Threatening Risk from Trypanosoma Cruzi Parasite: BCC Research LLC

Chagas disease, which is also known as American trypanosomiasis, occurs when you get infected with a small parasite called Trypanosoma cruzi. A doctor named Carlos RJ Chagas from Brazil discovered this parasite and how it causes illness in people way back in 1909. BOSTON, April 16, 2024 /PRNewswire/ -- The new research gives a complete picture of the Chagas Disease: American trypanosomiasis market. It looks at trends in the CD market, including how common it is in different regions, and talks about what's happening in the market right now. The report also looks closely at the competition between different treatments. It focuses on treatments available across Latin America. Because many people with Chagas Disease don't have enough money to buy medicine, there isn't much demand for these products. That's why pharmaceutical companies aren't putting a lot of effort into creating new treatments for Chagas disease now. Key Facts to Know About Chagas Disease: A Quick Overview About 6-7 million people, mostly in Latin America, have a bug bite disease called Chagas disease. It can be passed on by bugs, eating unsafe food, getting blood or organs from infected people, passing it from mother to baby, or accidents in labs. If treated early, Chagas disease caused by Trypanosoma cruzi can be cured. Treatment can also help stop the disease from getting worse and prevent passing it from mother to baby. Some people with long-term Chagas disease might have heart problems, and others might have stomach or brain issues. They might need special treatment for these. Ways to stop Chagas disease include controlling bugs in Latin America, checking blood before giv ing it to someone, and testing and treating girls, women who can have babies, newborns, and brothers and sisters of infected moms who haven't had treatment before. The Transmission of Chagas Disease in the American Market: Key Insights In Latin America, Chagas disease mostly spreads from poop or pee of blood-sucking bugs called kissing bugs. These bugs hide in cracks in houses and come out at night to feed on blood. When they bite, they often do it on the face. If someone scratches the bite and gets the poop or pee in their body, they can get infected with the parasite. T. cruzi can also be transmitted by: If you eat or drink something with T. cruzi in it, like food or drinks with poop or pee from infected bugs or opossums, you can get sick. This often causes outbreaks where people get very sick, and some even die. When a mom who has Chagas disease gives birth, she can pass it on to her baby. Getting blood or something made from blood from someone who has Chagas disease can also give you the disease. Signs and symptoms Chagas disease has two phases. In the first phase, which lasts about 2 months, a lot of parasites are in the blood, but most people don't have any symptoms or just feel a little unwell. Sometimes, people might see a spot on their skin or swelling around one eye. They could also have fever, headache, swollen glands, pale skin, muscle pain, trouble breathing, or pain in the belly or chest. In the second phase, the parasites hide in the heart and stomach muscles. After one to three decades, about a third of patients have heart problems, and about one in ten have stomach issues. Over time, the infection can damage the nerves and heart muscles, causing heart problems, irregular heartbeats, heart failure, or sudden death. Chagas Disease Treatment in the American Market Chagas disease can be treated with medicines like benznidazole or nifurtimox to kill the parasite. These drugs work best if given early, especially during the first phase of the disease. Treatment can also help prevent the disease from getting worse. However, treatment can have side effects, and it's not recommended for pregnant women or people with certain health conditions. In some cases, additional treatment may be needed for heart, digestive, or nerve problems caused by the disease. Control and Prevention In different places, the World Health Organization WHO (World Health Organisation) suggests various ways to prevent and control Chagas disease: Using bug sprays inside homes and nearby areas to kill insects and keep them away. Fixing up homes and keeping them clean to stop bugs from getting in. Using bed nets and practicing good hygiene when handling food, during transport, and storage to prevent infection. Creating easy-to-understand information and education programs for different situations and people about how to prevent the disease and use surveillance tools. Checking blood donors to make sure they don't have the disease. Checking organ, tissue, or cell donors and recipients to make sure they don't have the disease Here are the top 5 key companies in the Chagas Disease (American trypanosomiasis) market along with brief descriptions: Bayer AG: A multinational pharmaceutical company known for its commitment to global health initiatives, including research and development of treatments for neglected tropical diseases like Chagas Disease. Sanofi S.A.: A leading pharmaceutical company with a focus on innovative treatments for various diseases, including neglected tropical diseases such as Chagas Disease. GlaxoSmithKline plc: A global healthcare company dedicated to developing vaccines and treatments for infectious diseases, including Chagas Disease. Novartis AG: A pharmaceutical company known for its research and development efforts in addressing unmet medical needs, including the development of therapies for neglected tropical diseases like Chagas Disease. Merck & Co., Inc.: A leading global healthcare company committed to developing innovative treatments and vaccines for a wide range of diseases, including Chagas Disease. Directly Purchase a copy of the report with BCC Research. For further information or to make a purchase, please get in touch with [email protected]. About BCC Research BCC Research provides objective, unbiased measurement and assessment of market opportunities with detailed market research reports. Our experienced industry analysts' goal is to help you make informed business decisions, free of noise and hype. Contact Us Corporate HQ: 50 Milk St. Ste 16, Boston, MA 02109, USA Email: [email protected], Phone: +1 781-489-7301 For media inquiries, email [email protected] or visit our media page for access to our market research library. Data and analysis extracted from this press release must be accompanied by a statement identifying BCC Research LLC as the source and publisher. Logo: SOURCE BCC Research LLC

Vaccine

08 Feb 2024

·www.prnewswire.com

Chagas Disease (T. cruzi Infection) Epidemiological Review and Competitive Landscape Analysis 2024-2028

DUBLIN , Feb. 8, 2024 /PRNewswire/ -- The "Chagas Disease: American trypanosomiasis Market" report has been added to ResearchAndMarkets.com's offering. The protozoan Trypanosoma cruzi is the cause of Chagas disease (CD), also known as American trypanosomiasis, a chronic, systemic, parasitic illness with substantial risk factors associated with low socioeconomic status. It is believed that Chagas sickness is a neglected tropical illness. It is widespread throughout 21 American countries, but it can spread to non-endemic nations worldwide through the movement of infected individuals. The effects of CD extend beyond Latin American rural communities where vector-borne transmission - diseases spread by insects - occurs. The epidemiology and geographic distribution of CD have changed due to significant population shifts from rural to urban areas in Latin America and other parts of the world. Control measures should concentrate on preventing transmission from organ transplants, blood transfusions and mother-to-child (congenital) transmission in the U.S. and other areas where CD is currently present but is not endemic. Treatment for T. cruzi infection can begin as soon as the infection occurs. Antiparasitic therapy may stop or slow the progression of chronic illnesses in infected people, as well as stop infections from spreading from mother to child. Two therapeutic modalities have the potential to save lives: Antiparasitic medication, which eliminates the parasite. Symptomatic therapy, which is intended to control infection-related symptoms and indicators. Treatment with antiparasitic drugs is most beneficial early in the course of infection, albeit not exclusively for acute or critical cases. There are two kinds of treatments offered in the U.S. Benznidazole is available commercially and has been licensed by the FDA for use in children ages 2 to 12. Pharmacies can buy LAMPIT (nifurtimox) commercially from several medication wholesalers. It is FDA-approved for treating children under the age of 18. Healthcare providers can consult with Centers for Disease Control and Prevention (CDC) personnel regarding patient care, and for the most part, therapy does not require hospitalization. The research report provides a thorough overview of the Chagas Disease market. This report analyzes CD market trends with epidemiological and regional markets, highlights the current market and offers a detailed competitive environment analysis. The scope of this report covers treatment throughout Latin America. The Report Includes An overview of the global chagas disease (T. cruzi infection) market Analyses of the global market trends, with historical market revenue from 2021 to 2022, estimates for 2023, forecasts for 2024, and projections of compound annual growth rates (CAGRs) through 2028 Estimate of the market size and revenue forecast for global chagas disease market, and corresponding market share analysis based on the product type, treatment, and region Overview of the significant driving trends and challenges that affect the global market and its vendor landscape Review of the epidemiology of chagas disease and potential of key marketed drugs and recent approvals Discussion of sustainability trends and factors in the chagas disease market, with emphasis on consumer attitudes, ESG score analysis, future of ESG, case study, and the ESG practices followed Analysis of the competitive intelligence based on recent developments, key financials and segmental revenues, and product launch strategies Detailed profiles of leading market participants Bayer and Insud Pharma, providing a descriptive overview of their respective businesses Key Topics Covered: Chapter 1 Introduction Chapter 2 Summary and Highlights Chapter 3 Market Overview History Trypanosoma Cruzi Vector and T. cruzi Transmission Routes Clinical Forms Future Perspectives Novel Drugs Chapter 4 Chagas Disease by Drugs Benznidazole Toxicity of Benznidazole Nifurtimox Toxicity of Nifurtimox Chapter 5 Chagas Disease by Diagnosis Serology (Antibody Detection) Molecular Testing Chapter 6 Competitive Landscape Chapter 7 Company Profiles Bayer Insud Pharma For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets

Drug Approval

07 Jul 2009

·www.pharmatimes.com

Research into developing world diseases on rise – IFPMA

The pharmaceutical industry has been stepping up its efforts to find treatments for tropical diseases, according to a new analysis. The International Federation of Pharmaceutical Manufacturers & has just published the 2009 edition of its Status Report on Pharmaceutical Industry R&D for Diseases of the Developing World . It documents projects that are being conducted by IFPMA member companies, alone or with partners, to develop drugs and vaccines for the ten diseases of the developing world prioritised by the United Nations. The ten are human African trypanosomiasis (sleeping sickness), Chagas disease, dengue fever, leishmaniasis, leprosy, lymphatic filariasis, malaria, onchocerciasis, schistosomiasis and tuberculosis and the number of projects covering these areas have increased from 67 last year to 84 this year. IFPMA noted that while the number of tuberculosis and malaria studies has grown slightly, projects for the other eight have risen “markedly” from 11 in 2008 to 25 this year. Noting that “it is obvious that the world does not have all the medicines it needs” to address these diseases, Michael Boyd, acting director-general of IFPMA, said, claiming that the report “underlines the commitment of the research-based pharmaceutical industry and its many partners to fill these gaps”. He added that “the public-private partnership model is delivering increased R&D efforts on diseases primarily affecting the developing world”. The report notes that the last year has seen the approval of two new treatments for these types of diseases, namely Novartis and the Medicines for Malaria Venture’s paediatric Coartem (artemether and lumefantrine) and the combination of oral nifurtimox and intravenous eflornithine for sleeping sickness. The latter was developed through a collaboration between Bayer, Sanofi, the World Health Organisation’s TDR, the Drugs for Neglected Diseases initiative, Epicentre, Medecins sans Frontieres and the Swiss Tropical Institute.

Vaccine

100 Deals associated with Nifurtimox

External Link

KEGG	Wiki	ATC	Drug Bank
D00833	Nifurtimox	P01CC01	DB11820

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Chagas Disease	United States	Bayer HealthCare Pharmaceuticals, Inc.	06 Aug 2020

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Medulloblastoma	Phase 2	United States	Bayer AG	14 Jan 2008
Neuroblastoma recurrent	Phase 2	United States	Bayer AG	14 Jan 2008
Neuroblastoma	Phase 1	China	Hengtai Jiukang (Tianjin) Pharmaceutical Technology Co., Ltd.	20 Oct 2021
Chagas Disease	Discovery	Colombia	Bayer AG	27 Jan 2016
Chagas Disease	Discovery	Argentina	Bayer AG	27 Jan 2016
Chagas Disease	Discovery	Bolivia	Bayer AG	27 Jan 2016
Chagas Cardiomyopathy	Discovery	Argentina	Bayer AG	01 Nov 2013

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT00601003 (CTgov)	Phase 2	Neuroblastoma recurrent \| Medulloblastoma	112	Cyclophosphamide+Topotecan+Nifurtimox	butcidybva(kuvsffzlvd) = csmgiarcnn anavzqsyvj (ebailbsgzi, pgaalewlcx - fvnlpaidfo) View more	-	29 Dec 2022
NCT03784391 (Pubmed \| PLoS Negl Trop Dis)	Not Applicable	Chagas Disease	3,000	Nifurtimox	yzfhwypmyq(cgmgqugedx) = rahbgaxwoo nrppjopzeg (yikhfxseyi, 1.0 - 4.5) View more	-	01 Oct 2021
				nifurtimox (No antitrypanosomal treatment)	yzfhwypmyq(cgmgqugedx) = zftcldbnbc nrppjopzeg (yikhfxseyi, 1.0 - 4.2)
NCT02625974 (CHICO, Pubmed \| PLoS Negl Trop Dis) Manual	Phase 3	Chagas Disease	330	Nifurtimox	bwehrutsrr(nkwjyrvfag): difference = 14.0 (95% CI, 3.7 - 24.2)	Positive	07 Jan 2021
				placebo

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