Clinical Trials

Multiple Phase I and Phase II clinical trials evaluate the safety, tolerability, pharmacokinetics, and liver-targeted pharmacodynamics of PF-05221304 in healthy participants, individuals with hepatic impairment, and patients with non-alcoholic fatty liver disease or nonalcoholic steatohepatitis with associated liver fibrosis. Sponsored primarily by Pfizer alongside academic collaborators such as Columbia University, these studies represent varying recruitment statuses, including completed and withdrawn protocols.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT04321031 COMPLETED
Nonalcoholic Fatty Liver Disease; Nonalcoholic Steatohepatitis With Liver Fibrosis
Pfizer
 2020-06-15 PHASE2
NCT04399538 COMPLETED
Nonalcoholic Steatohepatitis; Nonalcoholic Fatty Liver Disease
Pfizer
 2020-08-10 PHASE2
NCT04395950 WITHDRAWN
NASH (Nonalcoholic Steatohepatitis); NAFLD (Nonalcoholic Fatty Liver Disease)
Columbia University
 2020-12 PHASE1
NCT03776175 COMPLETED
Non-Alcoholic Fatty Liver Disease (NAFLD)
Pfizer
 2019-01-04 PHASE2
NCT03871439 COMPLETED
Healthy
Pfizer
 2019-03-13 PHASE1
NCT03248882 COMPLETED
Nonalcoholic Fatty Liver Disease; Nonalcoholic Steatohepatitis
Pfizer
 2017-08-22 PHASE2
NCT03597217 COMPLETED
Healthy
Pfizer
 2018-08-27 PHASE1
NCT03534648 COMPLETED
Healthy
Pfizer
 2018-04-19 PHASE1
NCT03309202 COMPLETED
Hepatic Impairment
Pfizer
 2017-12-19 PHASE1
NCT03448172 COMPLETED
Healthy
Pfizer
 2018-04-18 PHASE1
NCT03448172 Completed
Healthy
Pfizer
 2018-04-18 Phase 1
NCT03309202 Completed
Hepatic Impairment
Pfizer
 2017-12-19 Phase 1
NCT02871037 COMPLETED
Normal Healthy
Pfizer
 2016-08 PHASE1
NCT02871037 Completed
Normal Healthy
Pfizer
 2016-08 Phase 1

(data from https://clinicaltrials.gov, updated on 2025-03-21)

Check the PF-05221304 product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

PF-05221304 is a liver-targeted inhibitor of acetyl-CoA carboxylase (ACC) that specifically binds the enzyme to suppress the conversion of acetyl-CoA to malonyl-CoA, thereby inhibiting downstream de novo lipogenesis in hepatocytes. By restricting hepatic lipid accumulation and reducing liver fat content, this mechanism directly mitigates pathological steatosis in clinical trial conditions such as non-alcoholic fatty liver disease and nonalcoholic steatohepatitis.

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.