A Phase 3, Randomized, Double-Blind, Cross-Over, Active-Controlled Study of the Efficacy and Safety of HPN-100, Glyceryl Tri-(4-phenylbutyrate), for the Treatment of Adults With Urea Cycle Disorders (Help UCD)
Latest Information Update: 18 Jul 2024
At a glance
- Drugs Glycerol phenylbutyrate (Primary)
- Indications Hyperammonaemia
- Focus Pharmacokinetics; Registrational
- Acronyms Help UCD
- Sponsors Hyperion Therapeutics
Most Recent Events
- 30 Nov 2015 According to Horizon Pharma media release, the European Commission has approved glycerol phenylbutyrate oral liquid (RAVICTI) for treatment of urea cycle disorders in patients two months of age and older.
- 27 Jun 2014 New trial record