To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)
Completed
Horizon Therapeutics, LLC
Phase 4
2010-10-04
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.
Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)
Completed
Horizon Pharma Ireland, Ltd., Dublin Ireland
Phase 3
2011-07-01
This non-randomized, open-label study was approximately one year in duration and consisted of
a short term NaPBA to HPN-100 switchover part involving two overnight stays followed by a
12-month long term treatment period involving monthly visits.
Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation
Completed
Horizon Pharma Ireland, Ltd., Dublin Ireland
Phase 1
2013-06-01
This is a medical research study to test a medication in adult patients with a disease called
medium-chain acyl-CoA dehydrogenase (MCAD) deficiency caused by at least one copy of the
985A>G mutation. The medication is glycerol phenylbutyrate, called Ravicti, which is
currently FDA approved for the treatment of urea cycle disorders. Previous research suggests
that Ravicti may also be effective in the treatment MCAD deficiency. This study will
investigate the safety and efficacy (how well it works) of Ravicti in patients with MCAD
deficiency caused by having at least one copy of the 985A>G mutation.
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