Study of Cases of Cutaneous Leishmaniasis Treated With Miltefosine in French Guiana, Retrospective Study (MILT2022)

Study of Cases of Cutaneous Leishmaniasis Treated With Miltefosine in French Guiana, Retrospective Study Before Clinical Trial (MILT 2022)

Miltefosine is the only oral treatment currently available for Cutaneous Leishmaniasis.

Despite several reports of good efficiency in other countries of South America, miltefosine remains limited to a compassionate use in France. The objective of this study is to collect data regarding the efficacy, safety and acceptability of miltefosine in patients treated in French Guiana since 2017.

Study Overview

• Status: Not yet recruiting
• Conditions: Treatment Adherence; Primary Health Care; Cutaneous Leishmaniases; Drug Evaluation
• Intervention / Treatment: Other: Data collection; Other: Questionnaires

Detailed Description

In French Guiana, Cutaneous Leishmaniasis is mostly caused by Leishmania guyanensis (80% of cases), followed by L. braziliensis (10%) and rarer species. L. guyanensis is usually treated with a single injection of pentamidine. However, pentamidine-resistant infections (10%) and all L. braziliensis are treated with either amphotericine B or meglumine antimoniate. These treatments can only be used during an hospitalization, which is the source of important indirect costs and discomforts for patients from the remote areas of French Guiana. Miltefosine is the only oral treatment available for Cutaneous Leishmaniasis. While it is widely used in South America (with good reported efficacy), the data available in France are scarce. This treatment, if available on a larger scale in France, would allow a treatment without hospitalization, which would be very suitable for French Guianese patients.

Our objective is to collect data on patients already treated with miltefosine in French Guiana (current indications being failure, side effect or impossible delivery of amphotericine B and meglumine antimoniate) in order to assess its efficacy, safety and acceptability. Patients treated with miltefosine at the Cayenne Hospital Center between 2017 and 2022 will be contacted by phone so as to answer questions regarding their quality of life during the infection and their satisfaction concerning miltefosine as a treatment. Data on their symptoms and general informations will be extracted from the medical files.

Primary objective: To assess the efficacy, tolerance and acceptability of Miltefosine among patients already treated with this drug as part of its AAC in French Guiana.

Observational, retrospective and prospective, monocentric study Research Involving Human Person Category 3 (RIPH3) - Non-Interventional (RNI) - Questionnaire (Jardé Law)

• Study Type: Observational
• Enrollment (Anticipated): 20

Contacts and Locations

• Study Contact: Name: Romain BLAIZOT; Phone Number: +594 594 39 53 59; Email: romain.blaizot@ch-cayenne.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients presenting Mucocutaneous Leishmaniasis proven by culture and/or polymerase chain reaction (PCR) seen in French Guiana between 01/01/2017 and 01/04/2022, in dispensaries (CDPS) or in the Dermatology Department of the CHC and having received treatment with Miltefosine

Description

Inclusion Criteria:

• Mucocutaneous Leishmaniasis proven by culture and/or polymerase chain reaction (PCR)
• Seen in French Guiana between 01/01/2017 and 01/04/2022, in dispensaries (CDPS) or in the Dermatology Department of the CHC
• Having received treatment with Miltefosine
• Patient who consented to participate in the study
• Age equal or superior to 18 years

Exclusion Criteria:

• Clinically suspected but not parasitologically proven leishmaniasis
• Refusal to participate
• Age < 18
• Patient under legal guardianship

Study Plan

How is the study designed?

Design Details

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

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Patients; Mucocutaneous Leishmaniasis proven by culture and/or polymerase chain reaction (PCR); Seen in French Guiana between 01/01/2017 and 01/04/2022, in dispensaries (CDPS) or in the Dermatology Department of the CHC; Having received treatment with Miltefosine; Patient who consented to participate in the study; Age equal or superior to 18 years	Other: Data collection; o Retrospective data collection in database from medical records (clinical, parasitological and therapeutic data); Other: Questionnaires; o Administration of two standardized questionnaires by telephone in prospective: Dermatology Life Quality Index (DLQI); Treatment Satisfaction Questionnaire for Medication (TSQM)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Disappearance of any lesion without the appearance of a new lesion, 6 months from the start of treatment with miltefosine	The primary endpoint will be therapeutic success, defined as the disappearance of any lesion without the appearance of a new lesion, 6 months from the start of treatment with miltefosine.	baseline

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of treatment-emergent adverse events occurring during treatment	Proportion of treatment-emergent adverse events occurring during treatment, categorized as follows: non-symptomatic laboratory abnormalities (mild); symptoms not leading to discontinuation or modification of treatment (moderate intensity); symptoms leading to discontinuation or dose reduction (severe intensity)	baseline
Patients' acceptability of treatment assessed after completion of treatment	Patients' acceptability of treatment assessed after completion of treatment, by Treatment Satisfaction Questionnaire for Medication (TSQM) administered by telephone	baseline
Impact of Cutaneous Leishmaniasis on quality of life	Impact of Cutaneous Leishmaniasis on quality of life, assessed retrospectively by Dermatology Life Quality Index (DLQI) questionnaire administered by telephone	baseline

Collaborators and Investigators

• Sponsor: Centre Hospitalier de Cayenne
• Investigators: Principal Investigator: Romain BLAIZOT, Centre Hospitalier de Cayenne, Dermatology

Study record dates

Study Major Dates

• Study Start (Anticipated): August 8, 2022
• Primary Completion (Anticipated): August 8, 2022
• Study Completion (Anticipated): October 8, 2022

Study Registration Dates

• First Submitted: August 5, 2022
• First Submitted That Met QC Criteria: August 5, 2022
• First Posted (Actual): August 9, 2022

Study Record Updates

• Last Update Posted (Actual): August 9, 2022
• Last Update Submitted That Met QC Criteria: August 5, 2022
• Last Verified: August 1, 2022

More Information

Terms related to this study

• Keywords: Amphotericin B; Pentamidine; oral administration; French Guiana; Meglumine Antimoniate; intramuscular administration; intraveinous administration
• Additional Relevant MeSH Terms: Skin Diseases; Infections; Vector Borne Diseases; Parasitic Diseases; Protozoan Infections; Skin Diseases, Parasitic; Skin Diseases, Infectious; Euglenozoa Infections; Leishmaniasis; Leishmaniasis, Cutaneous
• Other Study ID Numbers: 2022_028

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No