Glycerol phenylbutyrate Clinical Trials

Size	Price	Qty
5mg	USD 147

Clinical Trials

Several clinical trials evaluate glycerol phenylbutyrate across metabolic and neurological conditions, including urea cycle disorder, cystic fibrosis, corticobasal syndrome, and monocarboxylate transporter 8 deficiency. Spanning Phase 1/2 through Phase 4, these studies are funded by academic institutions and pharmaceutical entities such as the Technical University of Munich, Kaplan Medical Center, and Horizon Therapeutics. Trial recruitment statuses range from completed pediatric evaluations and actively recruiting studies to terminated and unknown statuses.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT05983588	Recruiting	Corticobasal Syndrome (CBS)	Technical University of Munich	2023-12-12	Phase 2
NCT05019417	Unknown status	Monocarboxylate Transporter 8 Deficiency	Kaplan Medical Center\|Weizmann Institute of Science	2021-06-30	Phase 2\|Phase 3
NCT02323100	Terminated	Cystic Fibrosis	National Jewish Health\|University of Alabama at Birmingham\|Children''s Hospital of Philadelphia\|Johns Hopkins University\|Horizon Pharma Ireland Ltd. Dublin Ireland	2018-12-02	Phase 1\|Phase 2
NCT02246218	Completed	Urea Cycle Disorder	Horizon Therapeutics LLC\|Horizon Pharma Ireland Ltd. Dublin Ireland	2014-12-31	Phase 4

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

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Glycerol phenylbutyrate acts as a prodrug that is hydrolyzed by gastrointestinal lipases to release phenylbutyrate, a nitrogen-binding agent and sigma-2 (σ2) receptor ligand with a pKi of 8.02 that facilitates alternative waste nitrogen excretion through glutamine conjugation. This reduction in systemic nitrogen toxicity provides a mechanism for controlling hyperammonemia in urea cycle disorders, while also underlying its clinical evaluation in corticobasal syndrome, monocarboxylate transporter 8 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>).