Phenylacetylglutamine Clinical Trials

Size	Price	Qty
25mg	USD 147
1g	USD 970

Clinical Trials

Several clinical trials have evaluated therapeutic strategies related to phenylacetylglutamine metabolism and disposition, focusing on the treatment of urea cycle disorders and pharmacokinetic assessments in healthy volunteers. Sponsored by commercial entities including Takeda and Horizon Therapeutics, these completed Phase I, Phase III, and Phase IV studies assessed the safety, tolerability, and waste-nitrogen excretion efficacy of precursor therapies across pediatric and adult cohorts.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT04155567	Completed	Healthy Volunteers	Takeda	2019-11-13	Phase 1
NCT02246218	Completed	Urea Cycle Disorder	Horizon Therapeutics LLC\|Horizon Pharma Ireland Ltd. Dublin Ireland	2014-12-31	Phase 4
NCT00992459	Completed	Urea Cycle Disorders	Horizon Pharma Ireland Ltd. Dublin Ireland	2009-10	Phase 3

(data from https://clinicaltrials.gov, updated on 2024-05-22)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Phenylacetylglutamine is formed via the enzymatic conjugation of phenylacetate with glutamine, effectively trapping excess nitrogen into a water-soluble metabolite designated for rapid renal excretion. By facilitating this alternative nitrogen elimination pathway, the generation of this compound lowers systemic ammonia levels to mitigate neurotoxicity in patients with urea cycle disorders.

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>).