E.W. "Al" Thrasher
Status: Funded - Open
A study for safety and efficacy of Miltefosine for treatment of children and adolescents with Post-Kala-azar Dermal Leishmaniasis (PKDL) in Bangladesh and association of serum vitamin E and exposure to arsenic with PKDL
Dinesh Mondal, MBBS, M.D., Ph.D.
BACKGROUND: An effective and safe treatment regimen for children and adolescents with post-kala-azar dermal leishmaniasis is currently not available which demands clinical trial to identify safe and effective treatment regimen for better management of children and adolescents with PKDL. Risk factor for development of PKDL after treatment for VL is poorly investigated, but is needed for discovery of new preventive strategy against PKDL.
GAP: The study is investigating the efficacy and safety of MF at allometric dose for 12 weeks for treatment of PKDL in children and adolescents. In addition, the study will investigate the role of nutritional and environmental factors in PKDL development. The study aims to establish how safe and effective miltefosine at allometric dose for 12 weeks in children and adolescents with PKDL and looking for developing new preventive strategy against PKDL by exploring nutritional and environmental risk factor for PKDL.
HYPOTHESIS: Hypothesis of the study are: 1. Oral treatment with Miltefosine in children with PKDL at allometric daily dose for 12 weeks is safe with a cure rate of ≥95%; 2. Development of PKDL in children and adolescent is genetically predisposed and is associated with IL-10 & IFN-gamma gene polymorphism causing high serum level of IL-10 and low serum level of IFN-gamma; 3. Nutritional & environmental factors such as low serum vitamin E & arsenic exposure are associated with PKDL .
METHODS: The study design includes case-control component for investigation risk factors for PKDL and an open clinical trial with MF at allometric dose for 12 weeks for treatment of confirmed PKDL in children and adolescents. Cure assessment will be done at 12 months after treatment completion through a newly developed scoring system and analysis of skin specimens for Leishmania donovani by qPCR.
IMPACT: The findings of the study will improve management of children and adolescents with PKDL in particular safety and efficacy of miltefosine at allometric dose for 12 weeks for treatment of PKDL in these population. Study findings will help policymakers, national kala-azar elimination program and physician in selection of drug, its dose and duration of treatment for PKDL in children and adolescents.
Drug Therapy, Parasite, Prospective Cohort, Treatment, Safety