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CLINICAL TRIALS PROFILE FOR RAVICTI

Clinical Trials for Ravicti

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Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01347073	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.
NCT01881984	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.
NCT01881984	Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation	Completed	University of Pittsburgh	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.
NCT01949766	Transition From Buphenyl to RAVICTI for the Therapy of Byler Disease	No longer available	University of Pittsburgh	N/A	1969-12-31	This is a single patient compassionate use protocol to determine whether RAVICTI will improve bile flow in a subject who previously tolerated therapy with Buphenyl.
NCT02046434	Phenylbutyrate Response as a Biomarker for Alpha-synuclein Clearance From the Brain	Active, not recruiting	University of Colorado, Denver	Phase 1	2014-01-01	This is a Phase I clinical trial of the FDA approved drug Glycerol Phenylbutyrate to see if phenylbutyrate can increase the removal of alpha-synuclein from the brain into the bloodstream. Alpha-synuclein forms abnormal protein deposits in dopamine neurons and is believed to cause the death of brain cells, leading to Parkinson's Disease.
NCT02094222	Expanded Access Protocol for an Intermediate Size Population - RAVICTI for Byler Disease	Temporarily not available	University of Pittsburgh	N/A	1969-12-31	Byler Disease is the result of a homozygous missense (G308V) mutation in the ATP8B1 gene. The disease is typically manifest in the first year of life on the basis of complications of cholestasis; common presentations include jaundice, poor growth, bleeding related to vitamin K deficiency, and/or weak bones related to vitamin D deficiency. Early management of Byler Disease is directed at nutritional issues which tend to be responsive to medical intervention, unlike the pruritus/scratching which remains a devastating problem. Progressive liver disease develops in Byler Disease and can lead to cirrhosis and end-stage liver disease. This is an open label expanded access protocol of RAVICTI in children with Byler Disease. The primary hypothesis is that the administration of RAVICTI in these children is feasible, well tolerated and safe. It is also hypothesized that RAVICTI treatment leads to an improvement in biochemical markers of liver disease and it may ameliorates or prevents the development of scratching behavior as a manifestation of pruritus attributed to the liver disease.
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