Glycerol phenylbutyrate
- Generic Name
- Glycerol phenylbutyrate
- Brand Names
- Ravicti
- Drug Type
- Small Molecule
- Chemical Formula
- C33H38O6
- 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
A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders
- Conditions
- Urea Cycle Disorders
- Interventions
- First Posted Date
- 2025-04-01
- Last Posted Date
- 2025-04-01
- Lead Sponsor
- Tongji Hospital
- Target Recruit Count
- 40
- Registration Number
- NCT06904027
- Locations
- 🇨🇳
Peking University First Hospital, Beijing, Beijing, China
🇨🇳Guangzhou Women and Children's Medical Center, Guangzhou, Guangdong, China
🇨🇳Tongji Hosipital, Wuhan, Hubei, China
PROFIL Study to Investigate the Effect of GPB on NfL Levels in Patients With Corticobasal Syndrome (CBS)
- Conditions
- Corticobasal Syndrome (CBS)
- Interventions
- Drug: Placebo
- First Posted Date
- 2023-08-09
- Last Posted Date
- 2024-01-05
- Lead Sponsor
- Technical University of Munich
- Target Recruit Count
- 32
- Registration Number
- NCT05983588
- Locations
- 🇩🇪
Klinikum rechts der Isar Technische Universität München Klinik und Poliklinik für Neurologie & German Center for Neurodegenerative Diseases (DZNE), Department of Neurology, Munich, Bavaria, Germany
🇩🇪Klinikum der Universität München (KUM), Campus Großhadern, Klinik und Poliklinik für Neurologie & German Center for Neurodegenerative Diseases (DZNE), Department of Neurology, Munich, Bavaria, Germany
Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)
- First Posted Date
- 2017-11-07
- Last Posted Date
- 2024-07-01
- Lead Sponsor
- Amgen
- Target Recruit Count
- 16
- Registration Number
- NCT03335488
- Locations
- 🇺🇸
Mount Sinai School of Medicine, New York, New York, United States
🇪🇸Hospital Materno-Infantil (HRU Carlos Haya), Málaga, Andalucia, Spain
🇮🇹Bambino Gesù Children's Research Hospital, Rome, Italy
Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)
- Conditions
- Cystic Fibrosis
- Interventions
- First Posted Date
- 2014-12-23
- Last Posted Date
- 2022-03-21
- Lead Sponsor
- National Jewish Health
- Target Recruit Count
- 13
- Registration Number
- NCT02323100
- Locations
- 🇺🇸
National Jewish Health, Denver, Colorado, United States
🇺🇸Johns Hopkins University School of Medicine, Baltimore, Maryland, United States
🇺🇸Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
- First Posted Date
- 2014-09-22
- Last Posted Date
- 2024-07-01
- Lead Sponsor
- Amgen
- Target Recruit Count
- 27
- Registration Number
- NCT02246218
- Locations
- 🇺🇸
Kaiser Permanente Regional Metabolic Center, Los Angeles, California, United States
🇺🇸Stanford Center for Clinical & Translational Research & Education, Palo Alto, California, United States
🇺🇸Shands at University of Florida, Gainesville, Florida, United States
Expanded Access Protocol for an Intermediate Size Population - RAVICTI for Byler Disease
- Conditions
- Byler Disease
- First Posted Date
- 2014-03-21
- Last Posted Date
- 2019-02-18
- Lead Sponsor
- Robert Squires, Jr.
- Registration Number
- NCT02094222
Phenylbutyrate Response as a Biomarker for Alpha-synuclein Clearance From the Brain
- First Posted Date
- 2014-01-27
- Last Posted Date
- 2022-04-25
- Lead Sponsor
- University of Colorado, Denver
- Target Recruit Count
- 38
- Registration Number
- NCT02046434
- Locations
- 🇺🇸
University of Colorado Denver Anschutz Medical Center, Aurora, Colorado, United States
Transition From Buphenyl to RAVICTI for the Therapy of Byler Disease
- Conditions
- Byler Disease
- First Posted Date
- 2013-09-24
- Last Posted Date
- 2013-09-24
- Lead Sponsor
- University of Pittsburgh
- Registration Number
- NCT01949766
Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation
- Conditions
- Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
- Interventions
- First Posted Date
- 2013-06-20
- Last Posted Date
- 2017-09-25
- Lead Sponsor
- University of Pittsburgh
- Target Recruit Count
- 4
- Registration Number
- NCT01881984
- Locations
- 🇺🇸
Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States
Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)
- First Posted Date
- 2011-05-04
- Last Posted Date
- 2024-07-11
- Lead Sponsor
- Amgen
- Target Recruit Count
- 23
- Registration Number
- NCT01347073
- Locations
- 🇺🇸
UCLA Pediatrics/Genetics, Los Angeles, California, United States
🇺🇸University Hospitals Case Medical Center, Cleveland, Ohio, United States
🇺🇸Baylor College of Medicine, Houston, Texas, United States
Drug Development Updates
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