Clinical Trials

Numerous clinical trials evaluate the therapeutic efficacy and prophylactic utility of posaconazole across Phase 1 through Phase 4 studies and observational protocols. Supported by academic medical centers and major pharmaceutical sponsors, research targets fungal conditions—including mycoses, mucormycosis, and onychomycosis—as well as antifungal prophylaxis in patients with hematologic malignancies. The clinical landscape comprises actively recruiting, completed, terminated, and withdrawn studies.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07261150 NOT_YET_RECRUITING
Histoplasmosis
University of Minnesota
2026-09-15 PHASE3
NCT07780565 NOT_YET_RECRUITING
Acute Myeloid Leukemia (AML); Myelodysplastic Syndrome
M.D. Anderson Cancer Center
2027-01-01 PHASE1
NCT07155226 RECRUITING
Acute Lymphoblastic Leukaemia; Acute Myeloid Leukaemia; Higher-risk Myelodysplastic Syndromes
AstraZeneca
2026-01-09 PHASE1; PHASE2
NCT07723703 NOT_YET_RECRUITING
Acute Myeloid Leukaemia; High Risk Myelodysplastic Syndrome
Amphista Therapeutics Ltd
2026-09-01 PHASE1
NCT04771130 RECRUITING
Acute Myeloid Leukemia; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Neoplasm
BeOne Medicines
2021-05-24 PHASE1; PHASE2
NCT03828773 RECRUITING
Candidiasis; Fungal Infection; Acute Myeloid Leukemia; Genetic Predisposition; Aspergillosis
Bochud Pierre-Yves
2019-02-11
NCT07719348 NOT_YET_RECRUITING
Acute Myeloid Leukemia; Chronic Myelomonocytic Leukemia; Myelodysplastic Syndrome Acute Myeloid Leukemia
Servier
2026-09-15 PHASE1; PHASE2
NCT04665037 RECRUITING
Invasive Fungal Infection
Merck Sharp & Dohme LLC
2022-02-22 PHASE2
NCT06541067 RECRUITING
Hematologic Malignancy
Nantes University Hospital
2024-11-08 PHASE2
NCT06802757 RECRUITING
Breast Cancer
Shandong Cancer Hospital and Institute
2025-05-01 PHASE2
NCT05468372 COMPLETED
Mucormycosis; Pulmonary (Etiology)
Post Graduate Institute of Medical Education and Research, Chandigarh
2022-07-01 PHASE2
NCT04368559 COMPLETED
Candidemia; Mycoses; Fungal Infection; Fungemia; Invasive Candidiasis; Pneumocystis; Mold Infection; Invasive Fungal Disease; Prophylaxis of Invasive Fungal Infections; Aspergillus
Mundipharma Research Limited
2020-05-11 PHASE3
NCT03717623 RECRUITING
Posaconazole; Pharmacokinetics; Invasive Candidiases; Invasive Aspergillosis; Invasive Mycosis; Fungal Infection; Prophylaxis
Melbourne Health
2019-08-01 PHASE4
NCT03763396 NOT_YET_RECRUITING
Brain Tumor, Recurrent; Cancer, Advanced; Glioma of Brain
University Health Network, Toronto
2024-06-01 EARLY_PHASE1
NCT04966585 TERMINATED
Crohn Disease; CARD9 S12N Risk Allele
Cedars-Sinai Medical Center
2022-08-17 PHASE4
NCT04825275 TERMINATED
Glioblastoma; Glioblastoma Multiforme; Glioblastoma Multiforme of Brain; Glioblastoma Multiforme, Adult
Milton S. Hershey Medical Center
2022-05-13 EARLY_PHASE1
NCT04294641 COMPLETED
Chronic GVHD
National Cancer Institute (NCI)
2021-05-10 PHASE2
NCT06282718 Not yet recruiting
Acute Respiratory Tract Infection
European Clinical Research Alliance for Infectious Diseases (ECRAID)|UMC Utrecht|University of Oxford|Universiteit Antwerpen
2024-02 --
NCT06158360 Recruiting
Thyroid Cancer
Ilsan Cha hospital
2024-01-01 --
NCT04218851 COMPLETED
Invasive Aspergillosis
Merck Sharp & Dohme LLC
2020-07-02 PHASE2
NCT05845359 Withdrawn
Bariatric Surgery Candidate
Montefiore Medical Center
2023-09 Phase 4
NCT03318159 COMPLETED
Aplastic Anemia; Myelodysplastic Syndromes; Fungal Infection
Seoul National University Hospital
2018-04-20 PHASE2
NCT06302842 Active not recruiting
Supportive Care
Istituto Auxologico Italiano
2023-07-01 Not Applicable
NCT05617638 Recruiting
Pain
Hospital Israelita Albert Einstein|Beneficência Portuguesa de São Paulo
2023-06-27 Not Applicable
NCT03796533 UNKNOWN
Leukemia, Myeloid, Acute
Hospices Civils de Lyon
2018-12-10
NCT04966234 UNKNOWN
Cystic Fibrosis; Aspergillosis
Bambino Gesù Hospital and Research Institute
2021-04-22 PHASE2; PHASE3
NCT02203773 TERMINATED
Acute Myelogenous Leukemia; Myelogenous Leukemia; Treatment Naive AML
AbbVie
2014-10-06 PHASE1
NCT03760445 WITHDRAWN
Leukemia, Myeloid, Acute
Novartis Pharmaceuticals
2019-11-15 PHASE1; PHASE2
NCT04194086 COMPLETED
Leukemia, Acute
Zhujiang Hospital
2018-11-01 PHASE1; PHASE2
NCT03561415 UNKNOWN
Fungal Infection
Bayside Health
2018-07-02 PHASE2
NCT03378479 COMPLETED
Aspergillosis; Pulmonary, Invasive (Etiology)
Universitaire Ziekenhuizen KU Leuven
2017-12-27 PHASE4
NCT03434704 COMPLETED
Transplant-Related Hematologic Malignancy; Leukemia, Acute; Myelodysplastic Syndromes; Graft Vs Host Disease; Fungal Infection
Ciceri Fabio
2018-06-18 PHASE2
NCT03631602 UNKNOWN
IFD Post-HSCT
Fujian Medical University
2017-10-01 PHASE2; PHASE3
NCT02805946 COMPLETED
Allogeneic Stem Cell Transplant; Acute Graft Versus Host Disease; Acute Myeloid Leucaemia; Myelo Dysplastic Syndrome
Radboud University Medical Center
2017-04-28 PHASE4
NCT01782131 COMPLETED
Fungal Infections; Invasive Pulmonary Aspergillosis
Merck Sharp & Dohme LLC
2013-09-25 PHASE3
NCT02358499 COMPLETED
Fungal Infections
Children's Mercy Hospital Kansas City
2015-01 PHASE1
NCT03336502 COMPLETED
Fungal Infection
Merck Sharp & Dohme LLC
2017-12-20 PHASE1
NCT02875743 COMPLETED
Aplastic Anemia; Leukemia, Myeloid, Acute; Myelodysplastic Syndromes; Bone Marrow Transplantation
King's College Hospital NHS Trust
2016-12-07 PHASE4
NCT03614455 COMPLETED
Pharmacokinetics
Daiichi Sankyo
2018-07-13 EARLY_PHASE1
NCT03246386 COMPLETED
Morbid Obesity
Radboud University Medical Center
2017-11-05 PHASE4
NCT02452034 COMPLETED
Neutropenia
Merck Sharp & Dohme LLC
2015-09-07 PHASE1
NCT02180165 COMPLETED
Aspergillosis
Merck Sharp & Dohme LLC
2014-07-29 PHASE3
NCT03036046 WITHDRAWN
Acute Myeloid Leukemia
F2G Ltd
2017-03 PHASE1; PHASE2
NCT03095547 WITHDRAWN
Invasive Aspergillosis
F2G Ltd
2017-05 PHASE1
NCT02968134 COMPLETED
Systemic Fungal Infections
Royal Brisbane and Women's Hospital
2017-01-16 PHASE4
NCT01200355 COMPLETED
Acute Myelogenous Leukemia; Myelodysplastic Syndrome
Memorial Sloan Kettering Cancer Center
2010-09-09 PHASE4
NCT02387983 COMPLETED
Fungal Infection
Merck Sharp & Dohme LLC
2015-05-06 PHASE1
NCT01901172 COMPLETED
Neoplasms
Hoffmann-La Roche
2013-08 PHASE1
NCT01716234 TERMINATED
Fungal Infections
Merck Sharp & Dohme LLC
2008-04-17 PHASE1
NCT01419678 COMPLETED
Lung Transplant Infection; Fungal Infection
University of Pittsburgh
2011-10
NCT02020213 WITHDRAWN
Clinical Infection
Taichung Veterans General Hospital
2013-12 PHASE4
NCT02714504 COMPLETED
Fusariosis; Onychomycosis
Universidade Federal do Rio de Janeiro
2008-08
NCT02372357 COMPLETED
Hematological Malignancy
Universitaire Ziekenhuizen KU Leuven
2012-02 PHASE4
NCT01377480 COMPLETED
Chagas Disease
Merck Sharp & Dohme LLC
2011-07-06 PHASE2
NCT01984229 COMPLETED
Healthy Volunteer
Hoffmann-La Roche
2013-12 PHASE1
NCT01667107 TERMINATED
Fungal Infection
Merck Sharp & Dohme LLC
2013-01-23 PHASE4
NCT00936117 COMPLETED
Leukemia; Fungal Infection
M.D. Anderson Cancer Center
2009-09 PHASE2
NCT01162967 COMPLETED
Chagas Disease
Hospital Universitari Vall d'Hebron Research Institute
2010-09 PHASE2
NCT01075984 COMPLETED
Fungal Infection
Merck Sharp & Dohme LLC
2010-02-23 PHASE1
NCT00550732 COMPLETED
Mycoses
Merck Sharp & Dohme LLC
2007-12 PHASE2
NCT00750737 COMPLETED
Invasive Fungal Infections; Hematologic Malignancies
M.D. Anderson Cancer Center
2008-06 PHASE3
NCT01777763 COMPLETED
Fungal Infections
Merck Sharp & Dohme LLC
2009-06 PHASE1
NCT01367808 COMPLETED
Pharmacokinetics and Pharmacodynamics of Sublingual Buprenorphine
Turku University Hospital
2011-02 PHASE1
NCT00799071 COMPLETED
Chronic Granulomatous Disease
Radboud University Medical Center
2009-02 PHASE2
NCT00811928 COMPLETED
Leukopenia
Merck Sharp & Dohme LLC
2008-11 PHASE3
NCT00811642 COMPLETED
Fungal Infection
Merck Sharp & Dohme LLC
2008-11 PHASE3
NCT00817765 COMPLETED
HIV Infection; Fungal Infection
Radboud University Medical Center
2009-01 PHASE1
NCT00423267 COMPLETED
Coccidioidomycosis
Merck Sharp & Dohme LLC
2007-05 PHASE3
NCT00686543 COMPLETED
Fungal Infection; Acute Myelogenous Leukemia; Neutropenia
Merck Sharp & Dohme LLC
2007-12 PHASE4
NCT00491764 COMPLETED
Onychomycosis
Merck Sharp & Dohme LLC
2007-06 PHASE2
NCT00034632 COMPLETED
Mycoses
Merck Sharp & Dohme LLC
2001-04 PHASE3
NCT00044486 COMPLETED
Leukemia, Myelocytic, Acute; Myelodysplastic Syndromes; Neutropenia
Merck Sharp & Dohme LLC
2002-07 PHASE3
NCT00034645 COMPLETED
Mycoses
Merck Sharp & Dohme LLC
1999-01 PHASE3
NCT00034658 COMPLETED
Mycoses
Merck Sharp & Dohme LLC
1998-12 PHASE3
NCT00034671 COMPLETED
Mycoses
Merck Sharp & Dohme LLC
2001-01 PHASE2
NCT00002399 COMPLETED
Candidiasis, Oral; HIV Infections
Schering-Plough
PHASE2
NCT00177749 TERMINATED
Fungal Infection
University of Pittsburgh
2004-08 PHASE4
NCT00002446 COMPLETED
Candidiasis, Oral; HIV Infections
Schering-Plough
1998-08 PHASE3
NCT00033982 COMPLETED
Granulomatous Disease, Chronic; Job's Syndrome; Infection
National Institute of Allergy and Infectious Diseases (NIAID)
2002-04-11 PHASE3

(data from https://clinicaltrials.gov, updated on 2026-05-08)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Posaconazole potently inhibits fungal lanosterol 14α-demethylase, thereby blocking ergosterol biosynthesis and disrupting phospholipid membrane structure to impair critical membrane-bound enzyme systems and inhibit fungal cell growth. This targeted disruption of fungal cell membrane integrity provides the mechanistic foundation for using posaconazole to prevent and treat invasive mycoses and mucormycosis, particularly in immunocompromised patients with hematologic 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>).

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