4-PBA (4-Phenylbutyric acid) Clinical Trials

Size	Price	Qty
100mg	USD 147
500mg	USD 247

Clinical Trials

Several completed clinical trials have evaluated 4-phenylbutyric acid (4-PBA) for genetic protein misfolding disorders, focusing on Alpha 1-Antitrypsin Deficiency and Cystic Fibrosis. Sponsored by academic and research organizations including the University of Florida, the Alpha-1 Foundation, and the Children's Hospital of Philadelphia, these Phase I and Phase II studies investigated phenylbutyrate-mediated secretion rescue and combination regimen approaches.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT00016744	COMPLETED	Cystic Fibrosis	Children's Hospital of Philadelphia	2001-09	PHASE1; PHASE2
NCT00067756	COMPLETED	Alpha 1-Antitrypsin Deficiency	University of Florida	2001-11	PHASE2
NCT00067756	Completed	Alpha 1-Antitrypsin Deficiency	University of Florida\|Alpha-1 Foundation\|Brantly Mark L. M.D.	2001-11	Phase 2

(data from https://clinicaltrials.gov, updated on 2009-01-09)

Check the 4-PBA (4-Phenylbutyric acid) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

4-Phenylbutyric acid acts as a histone deacetylase inhibitor and chemical chaperone, attenuating endoplasmic reticulum stress and modulating cellular autophagy pathways. By suppressing aberrant cellular stress responses and facilitating proper protein folding and trafficking, this compound promotes secretion rescue in protein misfolding disorders such as Alpha 1-Antitrypsin Deficiency and Cystic Fibrosis.

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>).