Bayer's New Pediatric Formulation of Nifurtimox Demonstrates Success in Largest-Ever Phase III Trial for Chagas Disease in Children

March 18, 2019 /Bio ValleyBIOON/ -- German pharmaceutical giant Bayer recently announced the results of the Phase III clinical study CHICO, which evaluated nifurtimox for the treatment of pediatric patients with Chagas disease (also known as American trypanosomiasis). This is the largest clinical therapeutic study ever conducted in children with this disease, and the results confirmed the safety and efficacy of the new formulation of nifurtimox in treating pediatric patients with Chagas disease.

The CHICO trial is a prospective, randomized, double-blind, historically controlled Phase III study conducted between 2016 and 2018 at 25 centers in Argentina, Bolivia, and Colombia. It enrolled a total of 330 pediatric patients with acute or chronic Chagas disease to evaluate the efficacy, safety, and pharmacokinetics of scoreable and dispersible nifurtimox tablets.

The results showed that serological responses one year after the completion of treatment confirmed the superiority of 60-day nifurtimox therapy compared to historical placebo controls, thereby meeting the study’s primary endpoint. Across the entire study population, the 30-day nifurtimox regimen yielded lower serological response rates than the 60-day regimen. Weight-adjusted nifurtimox dosing demonstrated a favorable safety profile.

Dr. Jaime Altcheh, Coordinating Investigator of the CHICO Study and Head of the Department of Parasitic and Chagas Diseases at Ricardo Gutiérrez Children’s Hospital in Buenos Aires, Argentina, stated, “The dispersible formulation of nifurtimox represents a major step forward toward the goal of treating all infected children. Early treatment after infection is crucial for preventing the manifestation of disease in adulthood.”

The goal of controlling Chagas disease is to eliminate transmission and controlCarrier, treatment of girls and women of childbearing age (to prevent transplacental transmission), and pre-transfusion blood screening. Furthermore, the aim is to ensure that infected individuals have access to medical care at all stages of the disease.

Treatment of Chagas disease relies solely on two nitroheterocyclic compounds, nifurtimox and benznidazole. To date, nifurtimox has been available only as 120-mg tablets, making administration difficult, particularly in pediatric patients.BayerA 30-mg tablet has now been developed. Both tablets are now available in a rapidly dispersible formulation that can be easily split and also dissolves quickly in water to form a slurry for administration to patients who have difficulty swallowing tablets. The new formulation has been successfully used in the CHICO study, improving the accuracy, safety, and treatment adherence of medication administration in children across all age groups.

Joerg Moeller, Member of the Executive Committee and Head of Research & Development at Bayer Pharmaceuticals Division, stated that,“Children are the most vulnerable patient population affected by Chagas disease. With the successful completion of the CHICO study, we have achieved a significant milestone in our long-standing commitment to improving the treatment of Chagas disease.”

Chagas disease, caused by *Trypanosoma cruzi*, is primarily transmitted by blood-sucking triatomine bugs. It was discovered by Dr. Chagas in 1908, hence the eponym Chagas disease. The disease is predominantly endemic to Central and South America, and is therefore also known as American trypanosomiasis. *T. cruzi* parasitizes the blood and various tissue cells of humans and other mammals. Its life cycle includes two morphological forms: the trypomastigote and the amastigote. Trypomastigotes enter the human bloodstream through breaks in the skin. Infection can also occur via breastfeeding, transplacental transmission, blood transfusion, or ingestion of food contaminated with infectious feces from triatomine bugs. It is estimated that there are 20 million infected individuals in Central and South America.

Most infections are asymptomatic. Symptomatic cases present only with mild flu-like symptoms that appear immediately after infection. Approximately two months later, Chagas disease enters a chronic phase that persists for many years, leading to severe organ damage and ultimately sudden cardiac death. Education, earlyDiagnosisTreatment is key to preventing Chagas disease.

Currently, due to insufficient disease awareness and limited access to treatment, less than 1% of patients infected with Chagas disease receive therapy. Bayer is committed to expanding access to and use of nifurtimox by filing new registration applications and providing new formulations in countries with a high burden of the disease. (Bioon.com)

Original Source:Phase III clinical trial with Bayer’s Nifurtimox demonstrates safety and efficacy of new formulation to treat children with Chagas disease