Bayer announced a Phase III clinical study evaluating the efficacy and safety of a newly developed formulation of the investigational medication Nifurtimox in children of all age groups who have been diagnosed with Chagas disease. The announcement was made at the 65th annual meeting of the American Society of Tropical Medicine & Hygiene (ASTMH) in Atlanta. Nifurtimox is not approved by the U.S. Food and Drug Administration (FDA) and there are no FDA-approved treatments for children with Chagas disease.
Chagas disease is a potentially life-threatening illness caused by the protozoan parasite Trypanosoma cruzi (T. cruzi). It’s estimated more than 300,000 people in the United States are infected with Chagas diseasei. The pathogen is often transmitted by an infected mother to her newborn during pregnancy or childbirth. Children, therefore account for the majority of new infections. In addition to systematic screening and diagnosis, treatment of infected women of child-bearing age and children is an effective tool in combating the spread of disease.
“As the manufacturer of Nifurtimox, we have a long-term commitment to the fight against this disease,” said Dieter Weinand, Member of the Board of Management of Bayer AG and President of Bayer’s Pharmaceuticals Division. “Children are the most vulnerable patient group. Every child who can be treated is a step toward getting the disease under control.”
Nifurtimox is on the World Health Organization’s (WHO) list of “Essential Medicines” for both adults and children. The WHO is distributing Nifurtimox in its efforts to control Chagas disease by 2020. Since 2004, as part of Bayer’s commitment to neglected tropical diseases, the company has given the WHO a permanent supply guarantee for Nifurtimox and provided financial assistance to support logistics and drug distribution.
“While children are particularly at risk from the disease, they also have the best chances of being completely cured,” said Dr. Pedro Albajar Vinas, Medical Officer for Neglected Tropical Diseases at the World Health Organization. “Therefore, investigating a treatment today is an utmost priority, together with broad-based health education and early diagnosis.”
“We regard Chagas as a children’s disease,” said Dr. Jaime Altcheh, Head of the Department of Parasitology and Chagas disease at the Ricardo Gutierrez Children’s Hospital in Buenos Aires. “An infected adult is usually a child that went untreated. Most infections are caused in childhood.”
About the Clinical Trial
The CHICO study (Chagas disease In Children treated with NifurtimOx) is a prospective, historically controlled study to evaluate the efficacy, safety and pharmacokinetics of Nifurtimox in children (up to the age of 17) with Chagas disease. The study involves 390 pediatric patients including newborn babies.
Researchers aim to develop an appropriate, weight-adjusted dose of Nifurtimox for newborns and children using fast-disintegrating 30mg and 120mg tablets that can be divided for weight-adjusted dosing in all age groups. The study design is a two-arm study with 30- and 60-day treatment cycles vs. historical untreated control; this is because a placebo-controlled study is not feasible. The study will be conducted at 25 locations in Argentina, Colombia and Bolivia. Patient enrollment began in January 2016.
Chagas disease is spreading beyond Latin America as a result of increased migration and travel. After successful completion of the study, Bayer plans to register Nifurtimox both in disease-endemic and in non-endemic countries with disease burden.
Filed Under: Drug Discovery