Glycerol phenylbutyrate

Catalog No.S6981 Synonyms: GT4P, HPN-100M, Ravicti

For research use only.

Glycerol phenylbutyrate (GT4P, HPN-100M, Ravicti) is a nitrogen-binding agent and acts as a sigma-2 (σ2) receptor ligand with a pKi of 8.02. Glycerol phenylbutyrate is a prodrug in which phenylbutyrate (PBA) is released from the glycerol backbone by lipases in the gastrointestinal tract.

Glycerol phenylbutyrate Chemical Structure

CAS No. 611168-24-2

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Biological Activity

Description Glycerol phenylbutyrate (GT4P, HPN-100M, Ravicti) is a nitrogen-binding agent and acts as a sigma-2 (σ2) receptor ligand with a pKi of 8.02. Glycerol phenylbutyrate is a prodrug in which phenylbutyrate (PBA) is released from the glycerol backbone by lipases in the gastrointestinal tract.
Targets
σ2 receptor [2]
8.02(pKi)

Protocol (from reference)

Chemical Information

Molecular Weight 530.65
Formula

C33H38O6

Density 1.13 g/mL
CAS No. 611168-24-2
Storage 3 years -20°C powder
2 years -80°C in solvent

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Clinical Trial Information

NCT Number Recruitment Interventions Conditions Sponsor/Collaborators Start Date Phases
NCT05019417 Recruiting Drug: Glycerol Phenylbutyrate 1100 MG/ML Monocarboxylate Transporter 8 Deficiency Kaplan Medical Center|Weizmann Institute of Science June 30 2021 Phase 2|Phase 3
NCT02323100 Terminated Drug: Ravicti low dose|Drug: Ravicti high dose|Drug: Placebo Cystic Fibrosis National Jewish Health|University of Alabama at Birmingham|Children''s Hospital of Philadelphia|Johns Hopkins University|Horizon Pharma Ireland Ltd. Dublin Ireland December 2 2018 Phase 1|Phase 2
NCT02246218 Completed Drug: RAVICTI Urea Cycle Disorder Horizon Therapeutics LLC|Horizon Pharma Ireland Ltd. Dublin Ireland December 31 2014 Phase 4

(data from https://clinicaltrials.gov, updated on 2022-08-01)

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Answers to questions you may have can be found in the inhibitor handling instructions. Topics include how to prepare stock solutions, how to store inhibitors, and issues that need special attention for cell-based assays and animal experiments.

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