To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)

• Status: Completed
• Trial end date: 2017-02-16
• Target enrollment: 0
• Participant gender: All

Summary

This was an open-label, long-term safety study of HPN-100 (RAVICTI; glycerol phenylbutyrate) in participants with a urea cycle disorder (UCD) who completed the safety extensions of HPN-100-005 (NCT00947544; HPN-100-005SE), HPN-100-006 (NCT00947297; HPN-100-007), or HPN-100-012 (NCT01347073; HPN-100-012SE). The initial studies were 1- to 2-week crossover studies, and their associated safety extensions were 12-month, open-label studies. All participants who completed the initial studies were eligible to enroll in the associated safety extension studies, and new participants were also permitted to enroll directly into the safety extension studies.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Horizon Therapeutics, LLC
• Treatments: 4-phenylbutyric acid; Glycerol

Criteria

Inclusion Criteria:

• Male and female subjects who completed Studies HPN-100-005SE, HPN-100-007, or HPN-100-012SE

• Signed informed consent by participant and/or participant's legally authorized representative

• Negative pregnancy test for all females of childbearing potential

Exclusion Criteria:

• Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, may have put the participant at increased risk when participating

• Known hypersensitivity to PAA (phenylacetate) or PBA (phenylbutyrate).

• Liver transplant, including hepatocellular transplant

• Pregnant, breastfeeding or lactating females