Diagnosis and Treatment of Leishmania Infections

Host Response to Infection and Treatment in Leishmania Infection of Humans

This study will examine the natural history of Leishmanial infections and their treatments. It will provide an opportunity for NIAID staff to learn more about leishmaniasis and perhaps to improve diagnostic tests for these infections. Patients between 2 and 80 years of age with known or suspected leishmaniasis are eligible for this study.

Participants will have routine blood tests and a biopsy to confirm leishmanial infection. The biopsy procedure will be determined by the type of infection local cutaneous leishmaniasis (LCL), mucocutaneous leishmaniasis (MCL) or visceral leishmaniasis (VL). CL will be confirmed with a punch biopsy, in which a cookie-cutter type razor is used to remove a small circular piece of skin tissue. MCL will be confirmed using a thin flexible tube inserted into the nose. This tube is used to examine the nose and upper airway and to remove a tissue sample, if an affected area is seen. VL will be confirmed with either a bone marrow or liver biopsy or a splenic aspirate. For these procedures, a small tissue sample is withdrawn through a needle placed in the hipbone, liver or spleen, respectively. Some patients may also have a skin test for leishmaniasis similar to tuberculin skin testing.

Treatment and length of hospital stay are determined by the type of infection. CL may be treated with Pentostam, amphotericin, amphotericin B, itraconazole or ketoconazole; ML with amphotericin B, or encapsulated amphotericin; and VL with Pentostam or encapsulated amphotericin. Pentostam is infused daily for 18 to 28 doses, most as an outpatient. Blood is drawn 3 times a week for safety tests and an electrocardiogram is done 2 to 3 times a week to monitor heart rhythm. Amphotericin B is infused every day or every other day for about 30 doses, all on an inpatient basis. Patients undergo hydration (infusion of a large amount of fluid) just before and immediately after each infusion to protect the kidneys. Blood is drawn every other day and urine samples are collected occasionally for routine urinalysis. Encapsulated amphotericin is infused every other day, on an outpatient basis. Blood is generally drawn every other day to every 2 days and urinalyses are done periodically. Itraconazole and ketoconazole are taken orally for at least 1 to 3 months, with blood drawn every 2 to 3 weeks.

Patients may be asked to have photographs taken before, during and after treatment to document progress. They may also be asked to provide extra blood samples for research purposes, either through a vein in the arm or through apheresis, a method for collecting large numbers of cells. For apheresis, whole blood is collected through a needle in an arm vein and circulated through a machine that separates it into its components. The desired cells are then removed, and the rest of the blood is returned to the body, either through the same needle used to draw the blood or through a second needle in the other arm.

Patients with cutaneous leishmaniasis will have a follow-up clinic visit 2 weeks to 3 months after treatment is completed. If there are no complications, their participation will end at that time. Patients with mucocutaneous leishmaniasis and visceral leishmaniasis will be followed every 3 to 6 months indefinitely for routine evaluations and re-treatment if the infection recurs.

Study Overview

• Status: Recruiting
• Conditions: Parasitic Diseases; Skin Diseases, Parasitic; Euglenozoa Infections; Leishmaniasis

Detailed Description

Patients admitted on this protocol will have, or be suspected of having, a Leishmania infection. The major objectives of the protocol are to diagnose and classify the Leishmania infection and to provide species-based therapy to study the natural history of the treated infection and further understand host and species characteristics that lead to resistant or relapsing disease. As part of the standard evaluation of these patients, biopsies to obtain parasite and host tissue will be obtained. The host response before and after therapy will be assessed to address broader questions related to diagnosis, disease pathogenesis, and response to therapy. Careful observations of the patients clinical and immunologic responses to therapy will be made, as well as long-term follow-up of these changes. It is anticipated that the patients will receive optimal clinical care for their infections and that the specimens collected from them will prove to be valuable reagents for the laboratory studies of the host responses unique to leishmanial infections.

• Study Type: Observational
• Enrollment (Estimated): 289

Contacts and Locations

• Study Contact: Name: Elise M O'Connell, M.D.; Phone Number: (301) 761-5413; Email: oconnellem@mail.nih.gov

Study Locations

• United States
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • Recruiting
      • National Institutes of Health Clinical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 100 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

This population of patients are referred from their physicians both regionally and nationally.

Description

• INCLUSION CRITERIA:
• Individuals aged 3 to 100 years.
• Diagnosis of leishmaniasis:

A: Biopsy proven

OR

B: Likely diagnosis of leishmaniasis based on :

Epidemiologic exposure, clinical manifestations, and time course

-Willingness to allow samples to be stored for future research.

EXCLUSION CRITERIA:

Any condition that, in the judgment of the investigator, may put the participant at undue risk or make them unsuitable for participation in the study.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
1; This population of patients are referred from their physicians both regionally and nationally.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Obtain phenotypic and clinical information to document refractory and relapsing clinical courses.	Obtain phenotypic and clinical information to document refractory and relapsing clinical courses.	1 year
Obtain organism genomic data at the initial time of NIH presentation and any further relapses	Obtain organism genomic data at the initial time of NIH presentation and any further relapses	1 year
Obtain a basic immune workup and host genetic information (WGS)	Obtain a basic immune workup and host genetic information (WGS)	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assess the efficacy of varying treatment regimens in different species	Assess the efficacy of varying treatment regimens in different species	1 year
Assess the safety profile of varying treatment regimens	Assess the safety profile of varying treatment regimens	1 year

Collaborators and Investigators

• Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
• Investigators: Principal Investigator: Elise M O'Connell, M.D., National Institute of Allergy and Infectious Diseases (NIAID)

Publications and helpful links

Helpful Links

• NIH Clinical Center Detailed Web Page

Study record dates

Study Major Dates

• Study Start (Actual): November 30, 2001

Study Registration Dates

• First Submitted: June 23, 2006
• First Submitted That Met QC Criteria: June 23, 2006
• First Posted (Estimated): June 26, 2006

Study Record Updates

• Last Update Posted (Actual): June 11, 2026
• Last Update Submitted That Met QC Criteria: June 10, 2026
• Last Verified: June 9, 2026

More Information

Terms related to this study

• Keywords: Natural History; Cutaneous; Visceral; Leishmanial Infection; MucocutaneousLeishmaniasis
• Additional Relevant MeSH Terms: Vector Borne Diseases; Infections; Protozoan Infections; Skin Diseases; Skin Diseases, Infectious; Skin and Connective Tissue Diseases; Parasitic Diseases; Leishmaniasis; Skin Diseases, Parasitic; Euglenozoa Infections
• Other Study ID Numbers: 010238; 01-I-0238

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: No.

Drug and device information, study documents

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