Clinical Trials

Multiple clinical trials evaluate pemafibrate across Phase 1 through Phase 4, focusing primarily on metabolic and cardiovascular conditions, including dyslipidemia, hypertriglyceridemia, type 2 diabetes, and non-alcoholic fatty liver disease. Sponsored by both industry entities and academic institutions, these studies encompass diverse recruitment statuses ranging from completed pharmacokinetic evaluations in healthy volunteers to active, recruiting, and terminated trials.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07562178 RECRUITING
Intracranial Atherosclerotic Disease (ICAD); Hypertriglyceridaemia; Stroke (CVA) or TIA
Tokyo Women's Medical University
 2026-05-26
NCT07634484 RECRUITING
Hypercholesterolemia
Kowa Company, Ltd.
 2026-07-01 PHASE4
NCT06247735 COMPLETED
Primary Biliary Cholangitis
Kowa Research Institute, Inc.
 2024-02-07 PHASE2
NCT06623539 ACTIVE_NOT_RECRUITING
Non Alcoholic Fatty Liver Disease
Yokohama City University
 2020-12-23 PHASE2
NCT06525311 COMPLETED
Primary Biliary Cholangitis; Compensated Cirrhosis
Kowa Research Institute, Inc.
 2024-10-01 PHASE1
NCT05923281 COMPLETED
Hypercholesterolemia
Kowa Company, Ltd.
 2023-05-01 PHASE3
NCT05722262 Completed
Healthy
Kowa Research Institute Inc.
 2023-02-24 Phase 1
NCT03071692 TERMINATED
Type2 Diabetes; Dyslipidemia
Kowa Research Institute, Inc.
 2017-03-23 PHASE3
NCT04742218 Completed
Healthy
Kowa Research Institute Inc.
 2021-01-13 Phase 1
NCT04079530 COMPLETED
Dyslipidemias
Kowa Company, Ltd.
 2019-09-13 PHASE2
NCT03350165 COMPLETED
Non-Alcoholic Fatty Liver Disease
Kowa Company, Ltd.
 2017-12-27 PHASE2
NCT03345901 TERMINATED
Diabetic Retinopathy; Diabetic Macular Edema
Jaeb Center for Health Research
 2018-01-15 PHASE3
NCT02922465 Completed
Healthy
Kowa Research Institute Inc.
 2016-09 Phase 1

(data from https://clinicaltrials.gov, updated on 2026-08-03)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Pemafibrate acts as a selective agonist of peroxisome proliferator-activated receptor alpha (PPARα) with an IC50 of 10 µM, binding to the receptor to upregulate target genes involved in fatty acid beta-oxidation and lipid transport while suppressing pro-inflammatory signaling pathways. This transcriptional modulation promotes systemic lipid clearance and reduces hepatic lipid accumulation, providing a mechanistic basis for its clinical investigation in dyslipidemia, hypertriglyceridaemia, and non-alcoholic fatty liver disease.

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.