Miltefosine to Treat Mucocutaneous Leishmaniasis

• symptomatic adverse effects: gastrointestinal [ Time Frame: days 1-28 of therapy ] [ Designated as safety issue: Yes ]
• laboratory adverse effects: creatinine or LFT elevation [ Time Frame: days 1-28 of therapy ] [ Designated as safety issue: Yes ]

The purpose of this Treatment IND is to make miltefosine available for mucocutaneous leishmaniasis patients presenting in the United States.

If entrance criteria are met, subjects with mucosal or cutaneous leishmaniasis will receive miltefosine at a targeted dose of 2.5 mg/kg/day for 28 days. During treatment at weeks 1, 2, and 4, the patient will return to the treatment facility to be assessed for adverse events. Blood for transaminase and creatinine values will be drawn at the midpoint and at the end of therapy.

Patients will return to the treatment facility to be examined clinically at 6 wks (ie, 2 wks after the end of therapy), 3 months (2 months after therapy), and 7 months (6 months after treatment) for ML and CL patients, and also at 13 months (12 months after treatment) for ML patients.

• Leishmaniasis, Mucocutaneous
• Cutaneous Leishmaniasis

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 ML or CL 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) PCR 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 109/l
   • Leukocyte count <3 x 109/l
   • Haemoglobin <10 g/100 ml
   • ASAT, 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?