Clinical Trials for Patients with Urea Cycle Disorders
Home About HPN-100 About UCD Who should volunteer? About the Studies Participating Centers Complete safety information for BUPHENYL^®	About HPN-100 HPN-100 (generic name: glycerol phenylbutyrate) is an investigational product, which means that it is not yet FDA-approved. It is in clinical trials to determine whether it is safe and effective as a treatment for patients with UCDs. It does not contain any salt and is a light liquid (triglyceride). How does HPN-100 work? HPN-100 is believed to act like sodium phenylbutyrate (BUPHENYL^®). It is given by mouth and is converted in the body to the same active ingredient in BUPHENYL, which is phenylacetic acid. Twenty grams of BUPHENYL (40 tablets) is expected to be equivalent to about 3.5 teaspoons (~17.4 milliliters) of HPN-100. What trials have already been done on HPN-100? As of March 4, 2011, 368 subjects have received single or multiple doses of HPN-100. It has been administered to 130 healthy adults in the context of three clinical studies, 100 patients with cirrhosis and/or episodic hepatic encephalopathy (HE) in the context of two clinical studies, and 91 (65 adult and 26 pediatric) UCD subjects in the context of five clinical studies.
BUPHENYL^® is a registered trademark of Ucyclyd Pharma, Inc.