Miltefosine to Treat Mucocutaneous Leishmaniasis
Condition: Mucosal Leishmaniasis; Cutaneous Leishmaniasis
MeSH terms: Leishmaniasis; Leishmaniasis, Cutaneous; Leishmaniasis, Mucocutaneous
Intervention: Miltefosine
Intervention description: 2.5 mg/kg/day for 28 days
Intervention MeSH terms: Miltefosine
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 4.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: leishmaniasis; cutaneous disease; mucosal disease; miltefosine

Summary:

      The purpose of this Treatment Investigational New Drug application was to make miltefosine
      available for mucocutaneous leishmaniasis patients presenting in the United States.

      If entrance criteria were met, subjects with mucosal or cutaneous leishmaniasis received
      miltefosine at a targeted dose of 2.5 mg/kg/day for 28 days. During treatment at weeks 1, 2,
      and 4, the patient returned to the treatment facility to be assessed for adverse events.
      Blood for transaminase and creatinine values were drawn at the midpoint and at the end of
      therapy.

      Patients returned to the treatment facility to be examined clinically at 6 weeks (ie, 2 weeks
      after the end of therapy), 3 months (2 months after therapy), and 7 months (6 months after
      treatment) for mucosal leishmaniasis and cutaneous leishmaniasis patients, and also at 13
      months (12 months after treatment) for mucosal leishmaniasis patients.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Is the subject a male or female at least 18 years of age?

          2. Does the subject weigh at least 30 kg?

          3. Does the subject have a diagnosis of mucosal leishmaniasis or cutaneous leishmaniasis
             in at least one lesion by at least one of the following methods: 1) positive culture
             for promastigotes of lesion material, 2) microscopic identification of amastigotes in
             stained lesion tissue, 3) Polymerase chain reaction of lesion material?

          4. In the opinion of the investigator, is the subject capable of understanding and
             complying with the protocol?

          5. If female and of child-bearing potential, did the subject have a negative pregnancy
             test during screening and agree to use an acceptable method of birth control during
             the treatment phase and for 6 months after treatment is completed?

          6. Has the patient signed informed consent?

        Exclusion Criteria:

          1. Is the subject a female who is breast-feeding?

          2. Does the subject have a clinically significant medical disorder?

               -  Thrombocyte count <100 x 10e9/L

               -  Leukocyte count <3 x 10e9/L

               -  Haemoglobin <10 g/100 mL

               -  Aspartate transaminiase (ASAT), alanine transaminase (ALAT) >2 times upper limit
                  of normal range

               -  Bilirubin >1.5 times upper limit of normal range

               -  Serum creatinine >1.5 times upper limit of normal range

               -  Major surgery within last 2 weeks

               -  Any non-compensated or uncontrolled condition

          3. In the last 4 weeks up to the present, has the subject received other treatment for
             leishmaniasis, including any medication with pentavalent antimony; amphotericin B,
             paromomycin, or imidazoles?