There is still no effective treatment for cryptosporidiosis even though the disease has a significant impact on HIV-infected adults and children. Following evidence of the drug's promising efficacy in vitro, a phase-1-phase-2 study of miltefosine (given at 2.5 mg/kg for 14 days, with the dose capped at 100 mg/day) was recently initiated among Zambian adults with HIV-related cryptosporidiosis. Seven patients were recruited before the trial was terminated prematurely because of lack of efficacy and the development of severe adverse events. The latter may have been entirely drug-related or the result of extreme metabolic abnormalities already present in the patients enrolled in the trial. In future trials of miltefosine, attention will have to be paid to the possibility of metabolic abnormalities in the subjects investigated.