Glycerol phenylbutyrate

Generic Name: Glycerol phenylbutyrate

Brand Names: Ravicti

Drug Type: Small Molecule

Chemical Formula: C_₃₃H_₃₈O_₆

CAS Number: 611168-24-2

Unique Ingredient Identifier: ZH6F1VCV7B

Background: Glycerol phenylbutyrate is a nitrogen-binding agent. Chemically, it is a triglyceride in which three molecules of phenylbutyrate are linked to a glycerol backbone. FDA approved on February 1, 2013.

Indication: Glycerol phenylbutyrate is a nitrogen-binding agent for the chronic management of adult and pediatric patients ≥2 years of age with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.

Associated Conditions: Disorders of urea cycle metabolism

Clinical Trials

To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)

Phase 4

Completed

Conditions: Urea Cycle Disorders

Interventions: Drug: HPN-100

First Posted Date: 2010-12-10

Last Posted Date: 2024-08-22

Lead Sponsor: Amgen

Target Recruit Count: 88

Registration Number: NCT01257737

Locations: 🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
🇺🇸
University Hospitals Case Medical Center, Cleveland, Ohio, United States
🇺🇸
Seattle Children's Hospital, Seattle, Washington, United States

and more 13 locations

Double-Blind Randomized Crossover Trial to Access Electrocardiogram Effects of HPN-100

Phase 1

Completed

Conditions: Drug Toxicity

Interventions: Drug: HPN-100
Drug: Placebo
Drug: HPN-100 or Placebo
Drug: Moxifloxacin

First Posted Date: 2010-06-03

Last Posted Date: 2024-06-20

Lead Sponsor: Amgen

Target Recruit Count: 98

Registration Number: NCT01135680

Locations: 🇺🇸
Covance Clinical Pharmacology, Inc., Madison, Wisconsin, United States

A Study of Safety and Efficacy of HPN-100 in Subjects With Cirrhosis and Episodic Hepatic Encephalopathy

Phase 2

Completed

Conditions: Cirrhosis
Hepatic Encephalopathy

Interventions: Drug: HPN-100
Drug: Placebo

First Posted Date: 2009-10-21

Last Posted Date: 2024-07-10

Lead Sponsor: Amgen

Target Recruit Count: 189

Registration Number: NCT00999167

Locations: 🇺🇸
The University of Chicago Medical Center, Chicago, Illinois, United States
🇺🇸
New York Medical College / Westchester Medical Center, Valhalla, New York, United States
🇺🇸
University of Maryland, Baltimore, Maryland, United States

and more 25 locations

Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders

Phase 3

Completed

Conditions: Urea Cycle Disorders

Interventions: Drug: Buphenyl (NaPBA)
Drug: HPN-100

First Posted Date: 2009-10-09

Last Posted Date: 2024-07-09

Lead Sponsor: Amgen

Target Recruit Count: 46

Registration Number: NCT00992459

Locations: 🇺🇸
UCLA, Los Angeles, California, United States
🇺🇸
Maine Medical Center, Portland, Maine, United States
🇺🇸
SNBL-Clinical Pharmacology Center, Baltimore, Maryland, United States

and more 19 locations

A Study of Glyceryl Tri-(4-phenylbutyrate) Administered Orally as a Single Dose, and Twice Daily for Seven Consecutive Days to Subjects With Hepatic Impairment With Cirrhosis and to a Control Group

Phase 1

Completed

Conditions: Urea Cycle Disorders
Hepatic Encephalopathy

First Posted Date: 2009-09-30

Last Posted Date: 2024-06-20

Lead Sponsor: Amgen

Target Recruit Count: 32

Registration Number: NCT00986895

Locations: 🇺🇦
Department of General Surgery #2; Kharkiv State Medical University, Kharkiv, Ukraine
🇺🇦
National University of Pharmacy, Kharkiv, Ukraine

A Study of Glyceryl Tri-(4-phenylbutyrate) (GT4P)

Phase 1

Completed

Conditions: Healthy

Interventions: Drug: Ammonul
Drug: HPN-100
Drug: Buphenyl

First Posted Date: 2009-09-16

Last Posted Date: 2024-06-20

Lead Sponsor: Amgen

Target Recruit Count: 24

Registration Number: NCT00977600

Locations: 🇺🇦
Medical Sanitary Division #2, Kharkiv, Ukraine

Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders

Phase 2

Completed

Conditions: Urea Cycle Disorders

Interventions: Drug: HPN-100
Drug: NaPBA

First Posted Date: 2009-07-28

Last Posted Date: 2024-07-11

Lead Sponsor: Amgen

Target Recruit Count: 17

Registration Number: NCT00947544

Locations: 🇺🇸
Seattle Children's Hospital, Seattle, Washington, United States
🇺🇸
UCLA, Los Angeles, California, United States
🇺🇸
The George Washington DC Children's National Medical Center, Washington, District of Columbia, United States

and more 5 locations

Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

Phase 3

Completed

Conditions: Urea Cycle Disorders

Interventions: Drug: HPN-100

First Posted Date: 2009-07-28

Last Posted Date: 2024-07-10

Lead Sponsor: Amgen

Target Recruit Count: 60

Registration Number: NCT00947297

Locations: 🇺🇸
SNBL-Clinical Pharmacology Center, Baltimore, Maryland, United States
🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
🇺🇸
University of Pittsburgh, Pittsburgh, Pennsylvania, United States

and more 19 locations

Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders

Phase 2

Completed

Conditions: Urea Cycle Disorders

Interventions: Drug: HPN-100
Drug: BUPHENYL®

First Posted Date: 2007-10-30

Last Posted Date: 2024-07-10

Lead Sponsor: Amgen

Target Recruit Count: 14

Registration Number: NCT00551200

Locations: 🇺🇸
University of Minnesota, Minneapolis, Minnesota, United States
🇺🇸
Baylor College of Medicine, Houston, Texas, United States

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