Conditions: Urea Cycle Disorder
Keywords: Urea Cycle Disorder UCD Buphenyl Glycerol Phenylbutyrate Ravicti Hyperammonemia Sodium Phenylbutyrate GPB HPN-100
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 27
Healthy volunteers: no

Brief summary:
This is an open-label study consisting of a transition period to RAVICTI, followed by a safety extension period for at least 6 months and up to 24 months of treatment with RAVICTI, depending on age at enrollment. It is designed to capture information important for evaluating safety, pharmacokinetics and efficacy in young children.

Subjects who are followed by or referred to the Investigator for management of their UCD. Subjects eligible for this study will include patients ranging from newborn to \< 2 years of age with either a diagnosed or clinically suspected UCD.

Detailed description:
Study acquired from Horizon in 2024.

Arms:
arm 1: RAVICTI Oral Liquid should be administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.

Interventions: intervention 1: RAVICTI
Intervention descriptions: intervention 1: None

Protocol document: