Clinical Trials
Zileuton has been evaluated in numerous clinical trials spanning Early Phase 1 through Phase 4 across various pulmonary, oncologic, and systemic inflammatory conditions, including asthma, idiopathic pulmonary fibrosis, lung inflammation, chronic obstructive pulmonary disease, sickle cell disease, chronic myelogenous leukemia, and tobacco use disorder. These investigations have been sponsored by corporate entities, academic research universities, children's hospital medical centers, and the National Cancer Institute. While a significant portion of these trials have reached completion, others were terminated early or remain of unknown status.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT02348203 | COMPLETED | Tobacco Use Disorder |
National Cancer Institute (NCI) |
2016-01-13 | PHASE2 |
| NCT02047149 | TERMINATED | Chronic Myelogenous Leukemia |
University of Massachusetts, Worcester |
2014-01 | PHASE1 |
| NCT01174056 | COMPLETED | Lung Inflammation |
Washington University School of Medicine |
2011-07 | EARLY_PHASE1 |
| NCT01805687 | COMPLETED | Asthma |
Cornerstone Therapeutics Inc. |
2013-03 | PHASE4 |
| NCT01136941 | COMPLETED | Sickle Cell Disease |
Children's Hospital Medical Center, Cincinnati |
2010-09 | PHASE1 |
| NCT00467831 | TERMINATED | Hermansky-Pudlak Syndrome (HPS); Pulmonary Fibrosis; Oculocutaneous Albinism; Platelet Storage Pool Deficiency; Metabolic Disease |
National Human Genome Research Institute (NHGRI) |
2007-04 | PHASE1; PHASE2 |
| NCT01130688 | TERMINATED | Chronic Myelogenous Leukemia |
University of Massachusetts, Worcester |
2010-01 | PHASE1 |
| NCT01021215 | COMPLETED | Tobacco Use Disorder |
National Cancer Institute (NCI) |
2010-05 | PHASE1; PHASE2 |
| NCT01136941 | Completed | Sickle Cell Disease |
Children''s Hospital Medical Center Cincinnati |
2010-09 | Phase 1 |
| NCT01130688 | Terminated | Chronic Myelogenous Leukemia |
University of Massachusetts Worcester |
2010-01 | Phase 1 |
| NCT00723021 | COMPLETED | Asthma |
Pfizer |
2008-07 | PHASE2 |
| NCT00493974 | TERMINATED | Pulmonary Disease, Chronic Obstructive |
University of Minnesota |
2007-03 | PHASE3 |
| NCT00486343 | TERMINATED | Asthma |
Critical Therapeutics |
2007-07 | PHASE4 |
| NCT00534625 | COMPLETED | Asthma |
Critical Therapeutics |
2007-09 | PHASE2 |
| NCT00262405 | COMPLETED | Idiopathic Pulmonary Fibrosis |
University of Michigan |
2001-01 | PHASE2 |
| NCT00070486 | COMPLETED | Lung Cancer |
Alliance for Clinical Trials in Oncology |
2003-12 | PHASE2 |
| NCT00056004 | COMPLETED | Head and Neck Cancer; Lung Cancer |
Barbara Ann Karmanos Cancer Institute |
2003-06 | PHASE2 |
| NCT00575861 | COMPLETED | Asthma |
Gelb, Arthur F., M.D. |
2005-09 | PHASE4 |
| NCT00098358 | UNKNOWN | Acne Vulgaris |
Critical Therapeutics |
2004-11 | PHASE2 |
| NCT00299065 | COMPLETED | Asthma |
Critical Therapeutics |
2006-01 | PHASE1; PHASE2 |
(data from https://clinicaltrials.gov, updated on 2022-06-02)
Compliance for Clinical Use
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>).