Numerous clinical trials investigate miltefosine primarily for treating parasitic diseases, including cutaneous, mucocutaneous, and post-kala-azar dermal leishmaniasis. Spanning Phase 1 through Phase 4 evaluations, these studies are supported by diverse sponsors such as the Drugs for Neglected Diseases initiative and research institutions. While many trials have been completed, others are currently active or pending recruitment, altogether aiming to optimize dosing regimens, treatment adherence, and drug safety across various health settings.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT07463040 | NOT_YET_RECRUITING | Cutaneous Leihmaniasis |
Institute of Tropical Medicine, Belgium |
2027-01-01 | PHASE3 |
| NCT06997159 | NOT_YET_RECRUITING | Localized Cutaneous Leishmaniasis |
Drugs for Neglected Diseases |
2026-06-15 | PHASE2 |
| NCT06550609 | RECRUITING | Mucosal Leishmaniasis |
Fundacion Nacional de Dermatologia |
2025-02-04 | PHASE2 |
| NCT07149753 | NOT_YET_RECRUITING | Cutaneous Leishmaniasis; Cutaneous Leishmaniasis, American; Topical Administration |
Universidad Industrial de Santander |
2026-01-01 | PHASE1 |
| NCT04268524 | COMPLETED | Old World Cutaneous Leishmaniasis |
Medecins Sans Frontieres, Netherlands |
2022-10-07 | PHASE3 |
| NCT02530697 | UNKNOWN | Leishmaniasis |
University of Brasilia |
2015-08 | PHASE2 |
| NCT06040489 | UNKNOWN | Leishmaniasis; Brazilian; Leishmaniasis, Mucocutaneous |
University of Brasilia |
2022-06-22 | PHASE2; PHASE3 |
| NCT04799236 | UNKNOWN | Mucosal Leishmaniasis |
Fundacion Nacional de Dermatologia |
2021-04-01 | PHASE3 |
| NCT06251739 | COMPLETED | Post-kala-azar Dermal Leishmaniasis |
International Centre for Diarrhoeal Disease Research, Bangladesh |
2023-10-30 | EARLY_PHASE1 |
| NCT04515186 | COMPLETED | Cutaneous Leishmaniases |
Drugs for Neglected Diseases |
2021-01-26 | PHASE3 |
| NCT05493059 | Not yet recruiting | Cutaneous Leishmaniases|Treatment Adherence|Primary Health Care|Drug Evaluation |
Centre Hospitalier de Cayenne |
2022-08-08 | -- |
| NCT03399955 | UNKNOWN | PKDL - Post-Kala-Azar Dermal Leishmanioid |
Drugs for Neglected Diseases |
2018-05-09 | PHASE2 |
| NCT03129646 | COMPLETED | Visceral Leishmaniasis |
Drugs for Neglected Diseases |
2018-01-24 | PHASE3 |
| NCT03829917 | COMPLETED | Cutaneous Leishmaniasis, American |
Fundacion Nacional de Dermatologia |
2019-02-01 | PHASE2; PHASE3 |
| NCT02687971 | COMPLETED | Cutaneous Leishmaniasis |
Drugs for Neglected Diseases |
2016-12 | PHASE2 |
| NCT03023111 | COMPLETED | Cutaneous Leishmaniasis |
Hospital Universitário Professor Edgard Santos |
2017-06-30 | PHASE3 |
| NCT02193022 | COMPLETED | Post Kala Azar Dermal Leishmaniasis |
International Centre for Diarrhoeal Disease Research, Bangladesh |
2014-07 | PHASE3 |
| NCT03399955 | Unknown status | PKDL - Post-Kala-Azar Dermal Leishmanioid |
Drugs for Neglected Diseases |
2018-05-09 | Phase 2 |
| NCT03445897 | COMPLETED | Leishmaniasis, Cutaneous |
Jonathan Berman |
2016-01-31 | PHASE2 |
| NCT02011958 | COMPLETED | Visceral Leishmaniasis |
Drugs for Neglected Diseases |
2014-07 | PHASE3 |
| NCT02431143 | COMPLETED | Visceral Leishmaniasis |
Drugs for Neglected Diseases |
2015-05 | PHASE2 |
| NCT01050907 | COMPLETED | Mucosal Leishmaniasis; Cutaneous Leishmaniasis |
Knight Therapeutics (USA) Inc |
2010-05 | PHASE2 |
| NCT02193022 | Completed | Post Kala Azar Dermal Leishmaniasis |
International Centre for Diarrhoeal Disease Research Bangladesh|Thrasher Research Fund |
2014-07 | Phase 3 |
| NCT01462500 | COMPLETED | Cutaneous Leishmaniasis |
Centro Internacional de Entrenamiento e Investigaciones Médicas |
2011-10 | PHASE4 |
| NCT01122771 | COMPLETED | Visceral Leishmaniasis |
Drugs for Neglected Diseases |
2010-05 | PHASE3 |
| NCT01377974 | COMPLETED | Leishmaniasis; Leishmaniasis, Mucocutaneous |
Brasilia University Hospital |
2009-07 | PHASE2 |
| NCT01462500 | Completed | Cutaneous Leishmaniasis |
Centro Internacional de Entrenamiento e Investigaciones Médicas|Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) |
2011-10 | Phase 4 |
| NCT01067443 | COMPLETED | Primary Visceral Leishmaniasis |
Drugs for Neglected Diseases |
2010-03 | PHASE2 |
| NCT01380314 | COMPLETED | Cutaneous Leishmaniasis |
Foundation Fader |
2008-03 | PHASE2 |
| NCT00487253 | UNKNOWN | Cutaneous Leishmaniasis |
Centro Internacional de Entrenamiento e Investigaciones Médicas |
2007-07 | PHASE3 |
| NCT00696969 | COMPLETED | Visceral Leishmaniasis |
Drugs for Neglected Diseases |
2008-06 | PHASE3 |
| NCT01635777 | COMPLETED | PKDL |
AB Foundation |
2007-07 | PHASE2 |
| NCT00371995 | COMPLETED | Visceral Leishmaniasis |
Banaras Hindu University |
2007-10 | PHASE2 |
| NCT00523965 | COMPLETED | Leishmaniasis, Visceral |
Banaras Hindu University |
2007-09 | PHASE3 |
| NCT00600548 | COMPLETED | Treatment of Cutaneous Leishmaniasis in Brazil. |
Hospital Universitário Professor Edgard Santos |
2007-07 | PHASE2 |
| NCT01170949 | TERMINATED | Chronic Urticaria |
Marcus Maurer |
2008-09 | PHASE2 |
| NCT01380301 | TERMINATED | Cutaneous Leishmaniasis |
Foundation Fader |
2007-03 | PHASE2 |
| NCT00471705 | COMPLETED | Cutaneous Leishmaniasis |
Universidad de Antioquia |
2006-06 | PHASE3 |
| NCT00378495 | TERMINATED | Kala Azar |
AB Foundation |
2005-04 | PHASE1; PHASE2 |
| NCT00233545 | COMPLETED | Cutaneous Leishmaniasis |
AB Foundation |
2005-09 | PHASE2 |
| NCT00537953 | UNKNOWN | Cutaneous Leihmaniasis |
Centro de Investigaciones Bioclínicas de la Fundación Fader |
PHASE2 | |
| NCT00373776 | COMPLETED | Leishmaniasis |
AB Foundation |
2004-04 | PHASE1; PHASE2 |
| NCT00370825 | COMPLETED | Visceral Leishmaniasis |
Banaras Hindu University |
2006-09 | PHASE2 |
| NCT00373568 | COMPLETED | Leishmaniasis |
AB Foundation |
2005-04 | PHASE1; PHASE2 |
(data from https://clinicaltrials.gov, updated on 2026-03-10)
Check the
Miltefosine
product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.
Mechanism and Biochemical Profile
Appendix RUO and cGMP Quality Standards
| Quality Dimension | RUO (Research Use Only) | cGMP (Current Good Manufacturing Practice) |
|---|---|---|
| Clinical Applicability | Prohibited in human clinical trials or medical diagnostics. | Mandatory for human clinical trials (Phase I–III) and therapies. |
| Regulatory Status | Non-regulated grade; exempt from drug manufacturing laws. | Legally enforced by health authorities (e.g., FDA, EMA, NMPA). |
| Facility Environment | Unclassified analytical or research laboratories. | Validated Cleanrooms (ISO Class 5–8) with continuous monitoring. |
| Quality Control | Basic purity and activity testing. | Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma). |
| Process Validation | Basic equipment calibration; no process validation required. | Full qualification (IQ/OQ/PQ) and complete batch records. |
| Quality Assurance | Vendor self-declared without required formal QMS. | Mandatory QA/QC unit, Change Control, CAPA, and vendor audits. |
| Regulatory Impact | High risk of IND rejection if used as a critical raw material. | Required for IND/NDA filings, supported by Drug Master Files (DMF). |
Footnotes
Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).
Note: Technical data last updated: Sep 1, 2026.