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:
Participant gender:
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.