This was a Phase 3, multi-center, randomized, open-label, parallel-group, active control study where 273 male and female patients with first stage Trypanosoma brucei gambiense HAT were treated at six sites: one trypanosomiasis reference center in Angola, one hospital in South Sudan, and four hospita...ls in the Democratic Republic of the Congo between August 2005 and September 2009 to support the registration of pafuramidine for treatment of first stage HAT in collaboration with the United States Food and Drug Administration. Patients were treated with either 100 mg of pafuramidine orally twice a day for 10 days or 4 mg/kg pentamidine intramuscularly once daily for 7 days to assess the efficacy and safety of pafuramidine versus pentamidine. Pregnant and lactating women as well as adolescents were included.
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The Lancet Volume 390, Issue 10110p2397-2409November 25, 2017.
Human African trypanosomiasis (HAT), also called sleeping sickness, is a parasitic infection that almost invariably progresses to death, unless treatment is provided. HAT caused devastating epidemics during the 20th century. Thanks to ...sustained and coordinated efforts during the past 15 years the number of reported cases has fallen to a historic low. Fewer than 3,000 cases were reported in 2015, and the disease is targeted for elimination by the World Health Organization. Despite recent success, HAT still poses a heavy burden on the rural communities where this highly focal disease occurs, most notably in Central Africa. Since patients are also reported from non-endemic countries outside Africa, HAT should be considered in differential diagnosis for all travellers, tourists, migrants and expatriates who have visited or lived in endemic areas. In the absence of a vaccine, disease control relies on case detection and treatment, and vector control. Available drugs are sub-optimal, but ongoing clinical trials give hope for safer and simpler treatments.
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