Human African Trypanosomiasis Development

 

  • Target disease: HAT
  • Partners (since project start): BaseCon, Denmark; Bertin Pharma, France; Venn Life Sciences (previously Cardinal Systems), France; Cardiabase, France; Médecins Sans Frontières, and other HAT Platform members; Phinc Development, France; National Control Programmes of the Democratic Republic of Congo and the Central African Republic; RCTs, France; Sanofi, France; Swiss Tropical and Public Health Institute (Swiss TPH); SGS, France; Theradis Pharma, France
  • Project start: April 2007
  • Funding (since project start): Bill & Melinda Gates Foundation, USA; Department for International Development (DFID), UK; Dutch Ministry of Foreign Affairs (DGIS), The Netherlands; French government AFD, France; GIZ on behalf of the Government of the Federal Republic of Germany, Germany; Ministry of Foreign and European Affairs (MAEE), France; Médecins Sans Frontières/Doctors without Borders, International; Norwegian Government, Norway; Republic and Canton of Geneva, International Solidarity Office, Switzerland; Spanish Agency for International Development Cooperation (AECID), Spain; Swiss Agency for Development and Cooperation (SDC), Switzerland; Other private foundations and individuals.

 

Overall Objective:

  • Develop and register fexinidazole as a new drug for the treatment of stage 2 HAT caused by T. b. gambiense, ideally also for stage 1 HAT and T. b. rhodesiense

 

The result of successful compound-mining efforts pursued by DNDi in 2005, fexinidazole entered clinical development in September 2009 and is now being co-developed with Sanofi: DNDi is undertaking clinical and pharmaceutical development whilst Sanofi is responsible for the industrial development and production. Preparations for product registration are underway.

A pivotal Phase II/III study, initiated in October 2012, aims to evaluate the safety and efficacy of fexinidazole compared to NECT, initially at eight sites in Democratic Republic of the Congo (DRC) and Central African Republic (CAR). Patient inclusion was halted in December 2013 in CAR due to conflict and security concerns in the country, and at one site in DRC due to a lack of new cases, although these sites remained open. A replacement site was opened in 2014 in DRC and, by the end of the year, the full cohort of 359 patients had been recruited. All trial safety data are regularly reviewed by the Data Safety and Management Board: serious adverse events for other indications were reviewed and no new risks identified. A strategy to accelerate the availability of fexinidazole was submitted to the regulators. Two complementary studies were initiated in May 2014 one for early stage 2 and stage 1 adult HAT patients, and another for children with HAT aged 6 to 14 years.

Last update: August 2015