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CLINICAL TRIALS PROFILE FOR RAVICTI
Clinical Trials for Ravicti
Trial ID | Title | Status | Sponsor | Phase | Summary |
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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 | 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 | 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 | 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. |
Trial ID | Title | Status | Sponsor | Phase | Summary |
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Clinical Trial Sponsors for Ravicti
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