Clinical Trial of Miltefosine to Treat Mucosal Leishmaniasis

Study Overview
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Brief Summary
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The purpose of this study is to determine whether miltefosine is effective in the treatment of mucosal leishmaniasis compared to meglumine antimoniate, the standard treatment.

Detailed Description
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Mucosal leishmaniasis is a rare form of the disease, that affects only 6% of the patients with cutaneous leishmaniasis in the New World. It causes deformities and may be lethal if not treated. It is part of the neglected tropical diseases because on the past sixty years there was few progress regarding other treatment options or improvement at quality of life of its patients. Also, there was little interest from the pharmaceutical industry and government authorities to develop new researches. The standard treatment, meglumine antimoniate, is toxic, invasive, requires trained personnel and has many adverse effects and restrictions. On the other hand, miltefosine is the first oral drug to demonstrate efficacy against mucocutaneous leishmaniasis. Few clinical trials have being performed in Central and South American countries, but so far, just one involved mucosal leishmaniasis patients, and compared miltefosine to amphotericin B. None studies comparing its efficacy to the standard treatment have being done.

Official Title
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Randomized Clinical Trial of Miltefosine to Treat Mucosal Leishmaniasis at Federal District, Brazil

Conditions
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Leishmaniasis, Leishmaniasis, Mucocutaneous

Intervention / Treatment
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* Drug: Miltefosine
* Drug: Standard Treatment Meglumine antimoniate


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Clinical diagnosis of mucosal leishmaniasis Not previously treated, or last treatment must have occurred more than 6 months before the enrollment on the study Use of contraceptive method, if female on child bearing age Sign the agreement and consent form Exclusion Criteria: Previous leishmanicidal treatment on the past 6 months before the enrollment on the study Electrocardiogram abnormalities on the pretreatment exams Previous kidney, liver and/or heart diseases Diabetes Mellitus Hypersensitivity to miltefosine or meglumine antimoniate Pregnant women or breastfeeding mothers Hiv patients

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 90 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Standard Treatment<br>Meglumine antimoniate as recommended by the Brazilian Ministry of Health | Drug: Standard Treatment Meglumine antimoniate<br>* 20mgKg daily intravenous Meglumine antimoniate as oriented by the Brazilian Ministry of Health<br>* Other names: pentavalent antimonial;|
| Experimental: Tested Intervention<br>Miltefosine as the tested intervention | Drug: Miltefosine<br>* 1 Capsule of 50mg, taken orally 2 times a day for 28 days<br>* Other names: Impavido;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cure | Re-epithelizations of mucosal ulcers or regression of symptoms | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse effects | laboratory tests of blood, kidney, liver and cardiac functions, partients will be asked about nausea, vomiting, diarrhea, myalgia, or other symptoms | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Leishmaniasis, Mucocutaneous Leishmaniasis, Therapeutics, meglumine antimoniate, miltefosine