Leishmaniasis

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:507OMIM:608207B55.0B55.1B55.2
Who is this for?
Show terms as
1FDA treatments18Active trials104Specialists8Treatment centers1Financial resources

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
Report missing data

Overview

Leishmaniasis is a group of infectious diseases caused by protozoan parasites of the genus Leishmania, transmitted to humans through the bite of infected female phlebotomine sandflies. It is not a genetic disorder but rather an infectious disease that can affect individuals of any background. The disease manifests in three main clinical forms: visceral leishmaniasis (also known as kala-azar; ICD-10 B55.0), cutaneous leishmaniasis (ICD-10 B55.1), and mucocutaneous leishmaniasis (ICD-10 B55.2). Visceral leishmaniasis is the most severe form and affects internal organs, particularly the spleen, liver, and bone marrow, causing fever, weight loss, hepatosplenomegaly (enlargement of the liver and spleen), pancytopenia (low blood cell counts), and hypergammaglobulinemia. Without treatment, visceral leishmaniasis is almost always fatal. Cutaneous leishmaniasis, the most common form, primarily affects the skin, producing ulcerated lesions at the site of the sandfly bite that can leave disfiguring scars. Mucocutaneous leishmaniasis affects the mucous membranes of the nose, mouth, and throat, potentially causing severe tissue destruction of the face. The immune response of the host plays a significant role in determining disease severity and clinical presentation, and certain host genetic factors (including polymorphisms in genes related to immune function such as those in the HLA system, SLC11A1/NRAMP1, and cytokine genes) may influence susceptibility. Treatment depends on the clinical form, the Leishmania species involved, and geographic location. For visceral leishmaniasis, treatments include liposomal amphotericin B (considered first-line in many regions), miltefosine, and pentavalent antimonial compounds. Cutaneous leishmaniasis may be treated with local therapies, cryotherapy, or systemic antileishmanial drugs depending on severity. Mucocutaneous leishmaniasis typically requires systemic treatment. Leishmaniasis is endemic in tropical and subtropical regions across approximately 90 countries, with an estimated 700,000 to 1 million new cases occurring annually worldwide. The disease disproportionately affects populations living in poverty with limited access to healthcare.

Clinical phenotype terms— hover any for plain English:

Abnormal oral cavity morphologyHP:0000163Abnormal macrophage morphologyHP:0004311Abnormal oral mucosa morphologyHP:0011830RhinitisHP:0012384
Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

10 events
Jan 2027Study Comparing Several Drugs to Understand Which Work Against Cutaneous Leishmaniasis (CL)

Institute of Tropical Medicine, Belgium — PHASE3

TrialNOT YET RECRUITING
Jan 2026Pilot Study: Miltefosine Gel (G-MTF) in Patients With Cutaneous Leishmaniasis

Universidad Industrial de Santander — PHASE1

TrialNOT YET RECRUITING
Nov 2025A Clinical Trial to Investigate Efficacy, Safety and Pharmacokinetics (PK) of Two LXE408 Oral Regimens and Oral Miltefosine as Active Control in Participants Aged ≥ 18 Years Old With Localized Cutaneous Leishmaniasis in the Region of the Americas (AMR).

Drugs for Neglected Diseases — PHASE2

TrialNOT YET RECRUITING
Oct 2025Recurrent Visceral Leishmaniasis in HIV Co-Infection

Institute of Tropical Medicine, Belgium

TrialRECRUITING
Oct 2025Arnica Tincture Fot the Treatment of Cutaneous Leishmaniasis II.

Universidad de Antioquia — PHASE3

TrialNOT YET RECRUITING
May 2025Human Bioequivalence Study of Liposomal Amphotericin B for Injection

Sichuan Huiyu Pharmaceutical Co., Ltd — NA

TrialACTIVE NOT RECRUITING
Apr 2025Antimicrobial Adjuvants to Revert the Imbalance of Skin Microbiota for Improved Outcomes of Complicated Cutaneous Leishmaniasis Treatment in Ethiopia

Institute of Tropical Medicine, Belgium — PHASE3

TrialNOT YET RECRUITING
Feb 2025Intralesional Injection of Metronidazole for the Management of Cutaneous Leishmaniasis

Hayder Adnan Fawzi — PHASE2

TrialRECRUITING
Feb 2025Intralesional Injection of Levofloxacin for the Management of Cutaneous Leishmaniasis

Hayder Adnan Fawzi — PHASE2

TrialRECRUITING
Feb 2025Ciprofloxacin Intralesional Injection for the Treatment of Cutaneous Leishmaniasis Compared to Sodium Stibogluconate

Al-Mustafa University College — PHASE2

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

1 available

IMPAVIDO

miltefosine· Knight Therapeutics (USA)■ Boxed WarningOrphan Drug
Treatment of visceral leishmaniasis due to Leishmania donovani; cutaneous leishmaniasis due to Leishmania braziliensis, Leishmania guyanensis, and Leishmania panamensis; and mucosal leishmaniasis due

Treatment of visceral leishmaniasis due to Leishmania donovani; cutaneous leishmaniasis due to Leishmania braziliensis, Leishmania guyanensis, and Leishmania panamensis; and mucosal leishmaniasis due to Leishmania braziliensis.

Clinical Trials

18 recruitingView all trials with filters →
Phase 31 trial
Randomised Clinical Trial for New Treatment Modalities for Cutaneous Leishmaniasis Caused by Leishmania Tropica, in Pakistan
Phase 3
Actively Recruiting
PI: Koert Ritmeijer (Medecins Sans Frontieres, Netherlands) · Sites: Kuchlagh, Balochistan; Quetta, Balochistan · Age: 1099 yrs
Phase 26 trials
Intralesional Injection of Levofloxacin for the Management of Cutaneous Leishmaniasis
Phase 2
Actively Recruiting
· Sites: Baghdad · Age: 1880 yrs
The Association of Miltefosine and Pentoxifylline to Treat Mucosal and Cutaneous Leishmaniasis: A Clinical Trial in Brazil
Phase 2
Active
· Sites: Brasília, Federal District · Age: 1880 yrs
A Phase II, Multicentre, Randomized, Two-arm Blinded Study to Assess the Efficacy and Safety of Two LXE408 Regimens for Treatment of Patients With Primary Visceral Leishmaniasis
Phase 2
Active
· Sites: Bihār; Patna · Age: 1899 yrs
Intralesional Injection of Metronidazole for the Management of Cutaneous Leishmaniasis
Phase 2
Actively Recruiting
· Sites: Baghdad · Age: 1880 yrs
Treatment of Bolivian L Braziliensis Mucosal Leishmaniasis with Inhaled Pentamidine Plus Oral Miltefosine
Phase 2
Actively Recruiting
· Sites: La Paz, La Paz Department · Age: 1299 yrs
Ciprofloxacin Intralesional Injection for the Treatment of Cutaneous Leishmaniasis Compared to Sodium Stibogluconate
Phase 2
Actively Recruiting
· Sites: Baghdad · Age: 1880 yrs
Phase 11 trial
Arnica Tincture Fot the Treatment of Cutaneous Leishmaniasis
Phase 1
Active
PI: Ivan D Velez, PhD (Director PECET) · Sites: Medellín, Antioquia · Age: 1865 yrs
N/A2 trials
Human Leishmaniasis: Antigen Recognition Pattern and Study of New Potential Biomarkers
N/A
Actively Recruiting
· Sites: Negrar, Verona · Age: 1899 yrs
Human Bioequivalence Study of Liposomal Amphotericin B for Injection
N/A
Active
PI: Qing Wen (Jinan Central Hospital) · Sites: Jinan, Shandong · Age: 1855 yrs
Other7 trials
Diagnosis and Treatment of Leishmania Infections
Actively Recruiting
PI: Elise M O'Connell, M.D. (National Institute of Allergy and Infectious Disea) · Sites: Bethesda, Maryland · Age: 3100 yrs
A Study to Assess Immune Response Status in Patients Before and After Treatment for Visceral Leishmaniasis
Active
PI: Paul Kaye, PhD (University of York) · Sites: Nairobi · Age: 1250 yrs
Clinical Prognostic Score to Predict Relapse in VL
Actively Recruiting
PI: Saskia van Henten, MD (Institute of Tropical Medicine Antwerp, Antwerp, B) · Sites: Gonder · Age: 1299 yrs
OPTImizing MIltefosine Treatment for Cutaneous LEISHmaniasis Patients
Actively Recruiting
PI: Saskia Van Henten (Institute of Tropical Medicine) · Sites: Addis Ababa · Age: 299 yrs
Mediterranean Visceral Leishmaniasis With Leishmania Infantum
Active
· Sites: Nice · Age: 1870 yrs
Recurrent Visceral Leishmaniasis in HIV Co-Infection
Actively Recruiting
PI: Eleni Ayele (LRTC University of Gondar) · Sites: Gonder · Age: 1899 yrs
Visceral Leishmaniasis in Emilia-Romagna (Leishmania-2019)
Actively Recruiting
PI: Pierluigi Viale, MD (IRCCS Azienda Ospedaliero-Universitaria di Bologna) · Sites: Cesena, Forlì-Cesena; Forlì, Forlì-Cesena +3 more · Age: 690 yrs

Specialists

Showing 25 of 104View all specialists →
SM
Shyam Sundar, MD
Specialist
PI on 8 active trials
JM
Jane Mbui, MD
Specialist
PI on 1 active trial
DP
Dinesh Mondal, PhD
Specialist
PI on 1 active trial
RM
Rezika Mohammed, MD
Specialist
PI on 1 active trial
JM
J Soto, MD
Specialist
PI on 1 active trial
JM
JAIME SOTO, MD
Specialist
PI on 6 active trials
SP
SERGIO C OLIVEIRA, PhD
Specialist
PI on 1 active trial
MM
Monique Wasunna, MD
Specialist
PI on 1 active trial
IP
Ivan Dario Velez, Prof
HARLINGEN, TX
Specialist
PI on 2 active trials
GM
Glenn Wortmann, MD
WASHINGTON, DC
Specialist
PI on 1 active trial1 Leishmaniasis publication
AP
Afif Ben Salah, M.D., Ph.D.
Specialist
PI on 3 active trials
JP
Joseph Olobo, MD, Prof
Specialist
PI on 1 active trial
HP
Harry S Nielsen, Ph.D.
Specialist
PI on 2 active trials
AP
Ahmed M Musa, MD, Prof
DETROIT, MI
Specialist
PI on 1 active trial
AP
Alejandro Llanos-Cuentas, MD, PhD
Specialist
PI on 2 active trials
SM
Shomik Maruf, MBBS,MPH,MSc
Specialist
PI on 1 active trial
DM
Dinesh Mondal, MBBS,MD,PhD
Specialist
PI on 1 active trial
SM
Sharan Sidhu, MD
GILROY, CA
Specialist
PI on 1 active trial
EM
Eltahir AG Khalil, MBBS
Specialist
PI on 1 active trial
CM
Charles Lacey, BMBS, MD
Specialist
PI on 2 active trials
JM
Johan van Griensven, MD,PhD
Specialist
PI on 1 active trial
JM
Julia S Ampuero-Vela, MD, MSc
Specialist
PI on 1 active trial
NP
Nancy Saravia, Ph.D.
Specialist
PI on 1 active trial
NP
Nancy Gore Saravia, Ph.D.
Specialist
PI on 1 active trial
AF
AMER EJAZ, FCPS
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Financial Resources

1 resources
IMPAVIDO(miltefosine)Knight Therapeutics (USA)

Travel Grants

No travel grants are currently matched to Leishmaniasis.

Search all travel grants →NORD Financial Assistance ↗

Community

Open LeishmaniasisForum →

No community posts yet. Be the first to share your experience with Leishmaniasis.

Start the conversation →

Latest news about Leishmaniasis

1 articles
NewsPUBMEDMar 26, 2026
[Multilocular mucocutaneous leishmaniasis with perforation of the anterior nasal septum: a rare differential diagnosis].
Leishmaniasis is a rare disease caused by tiny parasites that can affect the skin, mouth, and nose. This article describes an unusual case where a patient devel
See all news about Leishmaniasis

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Leishmaniasis

What is Leishmaniasis?

Leishmaniasis is a group of infectious diseases caused by protozoan parasites of the genus Leishmania, transmitted to humans through the bite of infected female phlebotomine sandflies. It is not a genetic disorder but rather an infectious disease that can affect individuals of any background. The disease manifests in three main clinical forms: visceral leishmaniasis (also known as kala-azar; ICD-10 B55.0), cutaneous leishmaniasis (ICD-10 B55.1), and mucocutaneous leishmaniasis (ICD-10 B55.2). Visceral leishmaniasis is the most severe form and affects internal organs, particularly the spleen, l

Are there clinical trials for Leishmaniasis?

Yes — 18 recruiting clinical trials are currently listed for Leishmaniasis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Leishmaniasis?

25 specialists and care centers treating Leishmaniasis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Leishmaniasis?

1 patient support program are currently tracked on UniteRare for Leishmaniasis. See the treatments and support programs sections for copay assistance, eligibility, and contact details.