Phenylbutyric acid
- Generic Name
- Phenylbutyric acid
- Brand Names
- Ammonaps, Buphenyl, Olpruva 2 Gm Pack, Pheburane, Relyvrio
- Drug Type
- Small Molecule
- Chemical Formula
- C10H12O2
- CAS Number
- 1821-12-1
- Unique Ingredient Identifier
- 7WY7YBI87E
- Background
Phenylbutyric acid is a fatty acid and a derivative of butyric acid naturally produced by colonic bacteria fermentation. It demonstrates a number of cellular and biological effects, such as relieving inflammation and acting as a chemical chaperone. It is used to treat genetic metabolic syndromes, neuropathies, and urea cycle disorders.
- Indication
Phenylbutyric acid is used for the treatment of various conditions, including urea cycle
disorders, neonatal-onset deficiency, late-onset deficiency disease in patients with a history of hyperammonemic encephalopathy. Phenylbutyric acid must be combined with dietary protein restriction and, in some cases, essential amino acid supplementation.
Phenylbutyric acid, as sodium phenylbutyrate, is used in combination with tauroursodeoxycholic acid to treat amyotrophic lateral sclerosis (ALS) in adults.
- Associated Conditions
- Amyotrophic Lateral Sclerosis (ALS), Argininosuccinate Synthetase Deficiency (Citrullinemia), Carbamyl Phosphate Synthetase Deficiency, Ornithine Transcarbamylase Deficiency, Deficiencies in enzymes of the urea cycle, Neonatal-onset deficiency
- Associated Therapies
- -
BSEP Function Rescue During Childhood Inhereditary Cholestatic Diseases
- Conditions
- Interventions
- First Posted Date
- 2020-08-31
- Last Posted Date
- 2023-02-09
- Lead Sponsor
- Children's Hospital of Fudan University
- Registration Number
- NCT04531878
PBA Use for Treatment of ATF6-/- Patients
- First Posted Date
- 2019-08-01
- Last Posted Date
- 2024-10-18
- Lead Sponsor
- Columbia University
- Target Recruit Count
- 2
- Registration Number
- NCT04041232
- Locations
- 🇺🇸
Edward S. Harkness Eye Institute, New York, New York, United States
Buphenyl Therapy for Byler's Disease
- Conditions
- First Posted Date
- 2013-02-06
- Last Posted Date
- 2013-02-06
- Lead Sponsor
- University of Pittsburgh
- Registration Number
- NCT01784718
Phenylbutyrate Therapy for Maple Syrup Urine Disease
- Conditions
- Interventions
- First Posted Date
- 2012-02-08
- Last Posted Date
- 2019-03-19
- Lead Sponsor
- Brendan Lee
- Target Recruit Count
- 20
- Registration Number
- NCT01529060
- Locations
- 🇺🇸
Baylor College of Medicine, Houston, Texas, United States
A Study of Glyceryl Tri-(4-phenylbutyrate) (GT4P)
- 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
Phase I and Pharmacokinetic Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Refractory Malignancy
- Conditions
- First Posted Date
- 2002-12-10
- Last Posted Date
- 2008-03-04
- Lead Sponsor
- National Cancer Institute (NCI)
- Target Recruit Count
- 35
- Registration Number
- NCT00001565
- Locations
- 🇺🇸
National Cancer Institute (NCI), Bethesda, Maryland, United States
Phenylbutyrate to Treat Children With Progressive or Recurrent Brain Tumors
- Conditions
- First Posted Date
- 2000-11-08
- Last Posted Date
- 2008-03-04
- Lead Sponsor
- National Cancer Institute (NCI)
- Target Recruit Count
- 120
- Registration Number
- NCT00006450
- Locations
- 🇺🇸
National Cancer Institute (NCI), Bethesda, Maryland, United States
Drug Development Updates
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