CLINICAL TRIALS PROFILE FOR RAVICTI
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All Clinical Trials for RAVICTI
| Trial ID | Title | Status | Sponsor | Phase | Start Date | Summary |
|---|---|---|---|---|---|---|
| NCT01257737 ↗ | 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. |
| 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 | 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 | No longer available | Robert Squires, Jr. | 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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