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 an oil (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? Three clinical trials have been completed, including one study in 24 healthy adults, one study in 32 healthy adults and adults with liver disease, and one study in 10 adults with UCDs. The results of these trials have been provided to the US Food and Drug Administration (FDA).
BUPHENYL^® is a registered trademark of Ucyclyd Pharma, Inc.