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

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.

Study Overview

• Status: Withdrawn
• Conditions: Leishmaniasis
• Intervention / Treatment: Drug: Miltefosine

Detailed Description

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.

• Study Type: Observational

Contacts and Locations

Study Locations

• United States
  • Maryland
    • Poolesville, Maryland, United States, 20837
      • Fast-Track Drugs and Biologics, LLC

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

leishmaniasis patients who are administered miltefosine

Description

Inclusion Criteria:

1. Did the subject give consent to collect data from her or her physician?
2. Was the subject treated for leishmaniasis with miltefosine?
3. Does the subject weigh more than 75 kg?

Exclusion Criteria:

[none]

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Miltefosine; 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; miltefosine: target 2.5 mg/kg/day for 28 days; Other Names: Impavido

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Knight Therapeutics (USA) Inc
• Investigators: Principal Investigator: Janet Ransom, PhD, Fast Track Drugs and Biologics LLC

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2015
• Primary Completion (Actual): March 1, 2020
• Study Completion (Actual): March 1, 2020

Study Registration Dates

• First Submitted: April 22, 2015
• First Submitted That Met QC Criteria: April 24, 2015
• First Posted (Estimate): April 29, 2015

Study Record Updates

• Last Update Posted (Actual): August 28, 2020
• Last Update Submitted That Met QC Criteria: August 26, 2020
• Last Verified: August 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Infections; Vector Borne Diseases; Parasitic Diseases; Protozoan Infections; Skin Diseases, Parasitic; Skin Diseases, Infectious; Euglenozoa Infections; Leishmaniasis; Anti-Infective Agents; Antineoplastic Agents; Antifungal Agents; Antiprotozoal Agents; Antiparasitic Agents; Miltefosine
• Other Study ID Numbers: IMP 2127-4