Research

Urea Cycle Disorder Treatment with HPN-100 – Phase III

Open-Label Study of the Safety of HPN-100 for the Long-Term Treatment of Urea Cycle Disorders (HPN-007)

Protocol Description

The goal of this multi-center Phase III study is to find out if HPN-100 is safe when taken over a 12- month period for treatment of urea cycle disorders (UCD). This study will also look at whether HPN-100 can help control the ammonia levels in people with UCD. People with UCD tend to have high ammonia levels in the blood, which can lead to various health problems.

Study Level: Phase III

Eligibility Criteria

Subject to certain screening criteria, this study is open to participants of both genders, ages 6 through adulthood, who have been diagnosed with a UCD. The study will include patients who participated in a previous study, HPN-100-006.
Males: Ages 6 and up
Females: Ages 6 and up

Requirements
Participants will receive a metabolic evaluation, including blood sampling and heart monitoring at the study onset, and will be provided a supply of HPN-100 to be taken 3 times daily with meals. Participants will be seen again at 1 week and then monthly for 3 months, followed by visits every 3 months up to the first year.
Visits: 8
Duration: Up to 12 months

Status: Closed to Enrollment

Sources of Support
Hyperion Therapeutics, Inc.

Additional Resources
Study description at National Institutes of Health