Clinical Trials

Numerous clinical trials spanning Early Phase 1 to Phase 4 evaluate Nystatin across diverse indications, including oral candidosis, denture stomatitis, periodontal disease, HIV-1 infection, and systemic fungal prophylaxis in very low birth weight infants. Spearheaded by academic hospitals, research institutes, and government agencies, these studies evaluate therapeutic efficacy and safety with recruitment statuses encompassing completed, recruiting, active not recruiting, terminated, and withdrawn.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT03178149 RECRUITING
Age-Related Macular Degeneration
Astellas Institute for Regenerative Medicine
 2018-07-13 PHASE1
NCT06616168 ACTIVE_NOT_RECRUITING
Bacterial Vaginosis
Danylo Halytsky Lviv National Medical University
 2023-09-01 PHASE1
NCT04550546 COMPLETED
Dental Caries
University of Rochester
 2021-05-15 PHASE1
NCT03862079 WITHDRAWN
Graft-versus-host Disease Prevention
M.D. Anderson Cancer Center
 2020-06-01 PHASE2
NCT04837664 UNKNOWN
Candida Albicans Infection; Cigarette Smoking
King Saud University
 2018-07-03 PHASE3
NCT03237533 UNKNOWN
Oral Lichen Planus
University of Faisalabad
 2017-06-01 EARLY_PHASE1
NCT02186145 UNKNOWN
Bacterial Vaginosis; Fungal Vaginal Infections
Marjan Industria e Comercio ltda
 2016-01 PHASE3
NCT07793331 COMPLETED
Denture Stomatitis; Oral Candidosis
Kerman University of Medical Sciences
 2016-01-01 PHASE2
NCT07694024 COMPLETED
Denture Stomatitis; Chronic Atrophic Candidiasis
Kerman University of Medical Sciences
 2016-01-17 PHASE2
NCT02662374 UNKNOWN
Oral Mucositis
Riyadh Colleges of Dentistry and Pharmacy
 2015-09 PHASE4
NCT01743690 COMPLETED
Oral Lichen Planus; Candida Infection
University of Copenhagen
 2012-03
NCT01798537 UNKNOWN
Bacteremia Associated With Intravascular Line; Ventilator Associated Pneumonia; Bacteremia
Rambam Health Care Campus
 2013-06 PHASE2; PHASE3
NCT04532060 COMPLETED
Health Care Associated Infection
São Paulo State University
 2015-02-01 PHASE2
NCT00845819 COMPLETED
Oral Mucositis
Seoul National University Hospital
 2009-02 PHASE2
NCT01718912 COMPLETED
Periodontal Disease
Oravital Inc
 2013-11
NCT01531192 COMPLETED
Very Low Birth Weight Infants
Zekai Tahir Burak Women's Health Research and Education Hospital
 2012-06 PHASE4
NCT03390374 COMPLETED
Fungal Infections Systemic
Dr Cipto Mangunkusumo General Hospital
 2010-10 PHASE4
NCT01427738 COMPLETED
HIV-1 Infection
Advancing Clinical Therapeutics Globally for HIV/AIDS and Other Infections
 2011-06 PHASE3
NCT01531192 Completed
Very Low Birth Weight Infants
Zekai Tahir Burak Women''s Health Research and Education Hospital
 2012-06 Phase 4
NCT01411748 UNKNOWN
Anticandidal Property of Saccharomyces Boulardii on Very Low Birth Weight Infants
Zekai Tahir Burak Women's Health Research and Education Hospital
 2011-07 PHASE4
NCT01411748 Unknown status
Anticandidal Property of Saccharomyces Boulardii on Very Low Birth Weight Infants
Zekai Tahir Burak Women''s Health Research and Education Hospital
 2011-07 Phase 4
NCT01427738 Completed
HIV-1 Infection
Advancing Clinical Therapeutics Globally for HIV/AIDS and Other Infections|National Institute of Allergy and Infectious Diseases (NIAID)
 2011-06 Phase 3
NCT02642900 COMPLETED
Photochemotherapy Reaction
University of Sao Paulo
 2009-07 PHASE4
NCT03390374 Completed
Fungal Infections Systemic
Dr Cipto Mangunkusumo General Hospital
 2010-10 Phase 4
NCT01198223 COMPLETED
Denture Stomatitis
Qazvin University Of Medical Sciences
 2010-07 PHASE2
NCT00078559 COMPLETED
Kidney Transplantation; Kidney Disease
University of Wisconsin, Madison
 2003-11 PHASE1; PHASE2
NCT00629122 COMPLETED
Kidney Failure, Chronic
Weill Medical College of Cornell University
 2008-02 PHASE4
NCT02444481 COMPLETED
Vaginitis
Laboratoire Innotech International
 2009-04 PHASE4
NCT01495039 COMPLETED
Fungal Prophylaxis; Candida Infection; Critical Illness; Surgery
Policlinico Hospital
 2008-11 PHASE4
NCT02642900 Completed
Photochemotherapy Reaction
University of Sao Paulo
 2009-07 Phase 4
NCT01867164 COMPLETED
Vaginitis; Infectious Vaginosis
Janssen-Cilag, S.A.
 2007-12 PHASE3
NCT00002097 COMPLETED
HIV Infections
Argus Pharmaceuticals
 PHASE1
NCT00002293 COMPLETED
Candidiasis, Oral; HIV Infections
Bristol-Myers Squibb
NCT00001998 COMPLETED
HIV Infections
Argus Pharmaceuticals
NCT00002057 COMPLETED
Candidiasis, Oral; HIV Infections
Bristol-Myers Squibb
NCT00002742 COMPLETED
Chronic Myeloproliferative Disorders; Infection; Leukemia; Lymphoma; Multiple Myeloma and Plasma Cell Neoplasm; Myelodysplastic Syndromes
Aronex Pharmaceuticals
 1996-01 PHASE3
NCT00002112 COMPLETED
Candidiasis, Oral; HIV Infections
Pfizer
NCT00001812 COMPLETED
Candidiasis, Oral; Gastrointestinal Diseases; Stomatitis
National Cancer Institute (NCI)
 1999-04 PHASE3
NCT00128323 COMPLETED
Candidiasis, Oral
Kamuzu University of Health Sciences
 2002-11 PHASE3
NCT00002313 COMPLETED
HIV Infections
Argus Pharmaceuticals
 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-09-03)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Nystatin interacts with cytochrome P450 14-demethylase to inhibit ergosterol biosynthesis, leading to severe disruption of fungal membrane integrity and increased cellular permeability. This membrane compromise ultimately induces fungal cell death, underlying its therapeutic efficacy against fungal infections such as oral candidosis, denture stomatitis, and pediatric systemic candidiasis evaluated in clinical trials.

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.