Treatment of Leishmaniasis With Impavido® (Miltefosine): Higher-Weight Patient Registry

Study Overview
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Brief Summary
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This study is a prospective observational study in which patients undergoing treatment for leishmaniasis with miltefosine (Impavido) in the US and who weigh > 75 kg can volunteer to provide information about their clinical response to treatment up to 6 months after the start of treatment.

Detailed Description
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Objective: The purpose of this observational study is to fulfill PMR 2127-4 for the miltefosine NDA (204684): implement a higher-weight-patient registry for the time period Mar 2015-Mar 2020. Study Design: This study is a prospective observational study in which patients undergoing treatment for leishmaniasis with Impavido in the US and who weigh > 75 kg can volunteer to provide information about their clinical response to treatment up to 6 months after the start of treatment. Population: Leishmaniasis patients treated with Impavido who weigh more than 75 kg. Drug Product: Drug name: Impavido (50 mg capsules). Dosing regimen: as per the Impavido Product Label Study Procedures: Patients who weigh more than 75 kg will become aware of the Impavido Higher-Weight Patient Registry via the Impavido website. By calling 1-866-588-5405, the patient will be connected to the Impavido Higher-Weight Patient Registry Coordinating Center. A trained staff member will acquaint the patient with the goals and procedures of the study. If the patient tentatively agrees to participate in the study over the telephone, the patient will be mailed information forms, the Consent/Assent Forms, and the Consent for the patient's physician to release medical information. Receipt of the two signed Consent and/or Assent Forms by the Coordinating Center will signify patient consent. The Coordinating Center will contact the patient's physician at the end of treatment, and at 1, 3, and 6 months after completing treatment, to collect data on efficacy and adverse effects (only during treatment). Sample Size and Study Duration: Estimated 3-10 patients per year for 5 years. Outcome Parameters: Efficacy Adverse effects Analysis: Baseline data, compliance to prescribed treatment, and outcomes will be reported for individual patients and for all patients.

Official Title
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Treatment of Leishmaniasis With Impavido® (Miltefosine): Higher-Weight Patient Registry

Conditions
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Leishmaniasis

Intervention / Treatment
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* Drug: Miltefosine


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Did the subject give consent to collect data from her or her physician? Was the subject treated for leishmaniasis with miltefosine? Does the subject weigh more than 75 kg? Exclusion Criteria: [none]

Ages Eligible for Study
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Minimum Age: 12 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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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Miltefosine<br>Miltefosine : target of 2.5 mg/kg/day for 28 days. Patients 45 kg or greater were to receive one 50 mg capsule 3 times daily for 28 consecutive days. This prospective observational study in which patients undergoing treatment for leishmaniasis with miltefosine in the United States who weighed >75 kg could volunteer to provide information about their clinical response to treatment up to 6 months after the start of treatment. | Drug: Miltefosine<br>* miltefosine: target 2.5 mg/kg/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 |
| --- | --- | --- |
| Number of patients cured | Determination of possible relationship of lower mg/kg daily doses of miltefosine and efficacy | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of patients with adverse effects | Adverse events including symptoms and laboratory parameters (if available) | 6 months |