Urea Cycle Disorder Treatment With Ravicti® - Phase IV

Study of Glycerol Phenylbutyrate (GPB; Ravicti®) in Children Under 2 Years of Age with Urea Cycle Disorders

Protocol Description

Through this investigation, researchers are monitoring the effectiveness of and how the body processes Ravicti® when used on patients of less than age 2 who have urea cycle disorders (UCDs). This rare genetic condition results in the accumulation of high levels of ammonia in the blood, called hyperammonemia, which can lead to brain damage and other serious problems. Ravicti works by helping to remove unwanted ammonia from the body, and it has previously been approved for use in patients older than 2.

Study Level: Phase IV

Eligibility Criteria

Subject to exclusion criteria, the study is accepting patients of both genders and under 2 years of age, who have a confirmed or suspected diagnosis of UCD.
Boys and Girls: Up to 2 years old


Enrolled patients will receive a physical examination by the study team, along with blood and urine testing. Participants will initially receive the study medication by mouth or via feeding tube 3 to 6 times a day while in the hospital. Once discharged, the patients’ parents/caregivers will provide the study medication. Checkups by the research team will be required monthly for the first six months, and quarterly thereafter.
Visits: Up to 12, depending on length of enrollment
Duration: 6 to 24 months, depending on age at enrollment

Status: Enrollment Closed

Source(s) of Support

Horizon Therapeutics

Primary Investigator

Uta Lichter-Konecki, MD, PhD

Contact Information

For more information about the study or enrollment, please contact:
Jessica Lindenberger, RN