Clinical Trials

Numerous clinical trials evaluate pentamidine across various infectious diseases—including Pneumocystis jirovecii pneumonia, cutaneous leishmaniasis, and African trypanosomiasis—as well as oncological indications like melanoma and hematologic malignancies. Spanning Phase 1 through Phase 3, these investigations encompass recruitment statuses ranging from completed and terminated to not yet recruiting. Research programs are supported by diverse sponsors, including the National Institute of Allergy and Infectious Diseases, the National Cancer Institute, academic medical centers, and international organizations.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07463040 NOT_YET_RECRUITING
Cutaneous Leihmaniasis
Institute of Tropical Medicine, Belgium
 2027-01-01 PHASE3
NCT05458752 Completed
Pneumocystis Jirovecii Infection
Central Hospital Nancy France
 2020-04-01 --
NCT03403621 TERMINATED
Hypertrophic Scar
Mayo Clinic
 2018-03-05 PHASE1; PHASE2
NCT03096457 COMPLETED
Leishmaniasis, Cutaneous; Leishmania Braziliensis Complex; Leishmaniasis, American; Leishmaniasis; American, Cutaneous
Fundacion Nacional de Dermatologia
 2017-04-15 PHASE2; PHASE3
NCT02669706 Completed
Hematologic Malignancy
University of Illinois at Chicago
 2015-03 --
NCT00729807 Terminated
Melanoma (Skin)
University of Maryland Baltimore|National Cancer Institute (NCI)
 2008-07 Phase 2
NCT00802594 Completed
Trypanosomiasis African
Immtech Pharmaceuticals Inc|Bill and Melinda Gates Foundation
 2001-08 Phase 2
NCT00000722 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
National Institute of Allergy and Infectious Diseases (NIAID)
NCT00002054 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
Fisons
NCT00002056 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
LyphoMed
NCT00000707 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
National Institute of Allergy and Infectious Diseases (NIAID)
NCT00002292 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
LyphoMed
NCT00002055 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
Fisons
NCT00001028 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
National Institute of Allergy and Infectious Diseases (NIAID)
 PHASE3
NCT00002030 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
Fisons
NCT00002053 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
Fisons
NCT00000730 TERMINATED
Pneumonia, Pneumocystis Carinii; HIV Infections
National Institute of Allergy and Infectious Diseases (NIAID)
 PHASE3
NCT00000991 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
National Institute of Allergy and Infectious Diseases (NIAID)
 PHASE3
NCT00001996 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
Glaxo Wellcome
 PHASE2
NCT00001013 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
National Institute of Allergy and Infectious Diseases (NIAID)
 PHASE3
NCT00000715 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
National Institute of Allergy and Infectious Diseases (NIAID)
 PHASE3
NCT00001003 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
National Institute of Allergy and Infectious Diseases (NIAID)
NCT00002291 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
Fisons
NCT00000727 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
National Institute of Allergy and Infectious Diseases (NIAID)
 PHASE3
NCT00000974 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
National Institute of Allergy and Infectious Diseases (NIAID)
 PHASE1
NCT00001027 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
National Institute of Allergy and Infectious Diseases (NIAID)
 PHASE1
NCT00002340 COMPLETED
Pneumonia, Pneumocystis Carinii; HIV Infections
Glaxo Wellcome
 PHASE3

(data from https://clinicaltrials.gov, updated on 2026-03-10)

Check the Pentamidine isethionate product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Pentamidine binds to and inhibits PRL phosphatases while simultaneously disrupting the biosynthesis of DNA, RNA, and proteins, thereby impairing cellular replication and survival. This inhibition of essential macromolecular pathways underlines its clinical utility in treating opportunistic infections such as Pneumocystis jirovecii pneumonia and leishmaniasis, as well as its evaluated potential in hematologic and solid malignancies.

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>).

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Note: Technical data last updated: Sep 1, 2026.