La Paz

Researchers are evaluating a combination treatment of inhaled pentamidine and oral miltefosine for patients with mucosal leishmaniasis caused by Leishmania braziliensis in Bolivia. This phase 2 study focuses on this severe form of leishmaniasis, which affects the nose, pharynx, palate, and larynx, and is known for its difficult treatment and poor cure rates with classical therapies. The goal is to improve cure rates for both mild and moderate-to-severe mucosal disease using a non-injectable drug regimen. Participants will receive oral miltefosine capsules—three pills daily for 28 days—alongside inhaled pentamidine administered as 10 doses of 300 mg each. This combination aims to achieve cure rates above 90% for mild disease and improve outcomes for more extensive mucosal involvement. The study will enroll up to 20 moderate-to-severe patients and may include up to 10 mild cases if they present. During the study, participants will undergo clinical evaluations and parasitological confirmation by biopsy or related tests. The primary outcome is the treatment efficacy measured over 25 months, focusing on the cure rate for moderate-to-severe cases compared to historical data for miltefosine alone. Safety monitoring, including laboratory tests and electrocardiograms, will be conducted. The total study duration includes treatment and long-term follow-up to assess sustained cure and safety.