Hyperion Therapeutics has begun enrolling patients in its THRIVE study, a long-term registry of patients with urea cycle disorders (UCDs) which will capture clinical outcomes and comparative effectiveness data. Hyperion received FDA approval for Ravicti, an oral medication for the long-term management of high blood levels of ammonia, in February 2013.

The THRIVE study is a multi-center, prospective, non-interventional study designed to collect data in as many as 500 UCD patients as part of their routine management. The study will track long-term outcomes, including hyperammonemic crises, ammonia levels, growth and development, and neurocognitive outcomes. Eligible patients will be enrolled in the study and followed for as long as 10 years with annual interim data reports planned. At the time of enrollment, retrospective and baseline data will be collected. Data will also be collected during routine office visits. In addition, the study will capture the duration, dose, frequency, and route of administration of all current standard of care treatments, including ammonia-scavenging agents sodium phenylbutyrate, glycerol phenylbutyrate, or sodium benzoate.