Clinical Trial of Miltefosine to Treat Mucosal Leishmaniasis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01377974
Recruitment Status : Completed
First Posted : June 22, 2011
Last Update Posted : June 9, 2015
Information provided by (Responsible Party):
Juliana Silva, Brasilia University Hospital

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Leishmaniasis Leishmaniasis, Mucocutaneous Drug: Miltefosine Drug: Standard Treatment Meglumine antimoniate Phase 2

Detailed Description:
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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Randomized Clinical Trial of Miltefosine to Treat Mucosal Leishmaniasis at Federal District, Brazil
Study Start Date : July 2009
Actual Primary Completion Date : June 2013
Actual Study Completion Date : December 2014

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Leishmaniasis

Arm Intervention/treatment
Active Comparator: Standard Treatment
Meglumine antimoniate as recommended by the Brazilian Ministry of Health
Drug: Standard Treatment Meglumine antimoniate
20mgKg daily intravenous Meglumine antimoniate as oriented by the Brazilian Ministry of Health
Other Name: pentavalent antimonial

Experimental: Tested Intervention
Miltefosine as the tested intervention
Drug: Miltefosine
1 Capsule of 50mg, taken orally 2 times a day for 28 days
Other Name: Impavido

Primary Outcome Measures :
  1. Cure [ Time Frame: 6 months ]
    Re-epithelizations of mucosal ulcers or regression of symptoms

Secondary Outcome Measures :
  1. Adverse effects [ Time Frame: 6 months ]
    laboratory tests of blood, kidney, liver and cardiac functions, partients will be asked about nausea, vomiting, diarrhea, myalgia, or other symptoms

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Ages Eligible for Study:   12 Years to 90 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01377974

Brasilia University Hospital
Brasilia, DF, Brazil, 70910-900
Sponsors and Collaborators
Brasilia University Hospital
Principal Investigator: Juliana SF Silva, MD University of Brasilia
Study Chair: Raimunda NR Sampaio, PhD University of Brasilia

Responsible Party: Juliana Silva, Dr. Juliana Silva, Brasilia University Hospital Identifier: NCT01377974     History of Changes
Other Study ID Numbers: MILTHUB
First Posted: June 22, 2011    Key Record Dates
Last Update Posted: June 9, 2015
Last Verified: June 2015

Keywords provided by Juliana Silva, Brasilia University Hospital:
Mucocutaneous Leishmaniasis
meglumine antimoniate

Additional relevant MeSH terms:
Leishmaniasis, Mucocutaneous
Euglenozoa Infections
Protozoan Infections
Parasitic Diseases
Skin Diseases, Parasitic
Skin Diseases, Infectious
Skin Diseases
Leishmaniasis, Cutaneous
Meglumine antimoniate
Antifungal Agents
Anti-Infective Agents
Antineoplastic Agents
Antiprotozoal Agents
Antiparasitic Agents