CLINICAL TRIALS PROFILE FOR GLYCEROL PHENYLBUTYRATE
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All Clinical Trials for glycerol phenylbutyrate
| 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. |
| 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. |
| NCT02246218 ↗ | A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders | Completed | Horizon Therapeutics, LLC | Phase 4 | 2014-12-31 | This is an open-label study consisting of a transition period to RAVICTI, followed by a safety extension period for at least 6 months and up to 24 months of treatment with RAVICTI, depending on age at enrollment. It is designed to capture information important for evaluating safety, pharmacokinetics and efficacy in young children. Subjects who are followed by or referred to the Investigator for management of their UCD. Subjects eligible for this study will include patients ranging from newborn to < 2 years of age with either a diagnosed or clinically suspected UCD. |
| NCT02323100 ↗ | Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis) | Recruiting | Children's Hospital of Philadelphia | Phase 1/Phase 2 | 2018-12-02 | We propose to test the effectiveness of the combination of CF pancreatic enzyme replacement therapy (PERT) on absorption of Ravicti® and subsequent restoration of nasal epithelial cystic fibrosis transmembrane conductance regulator (CFTR)-mediated chloride transport during the nasal potential difference (NPD) test. Funding source FDA Office of Orphan Products Development. |
| >Trial ID | >Title | >Status | >Sponsor | >Phase | >Start Date | >Summary |
Clinical Trial Conditions for glycerol phenylbutyrate
Condition Name
| Condition Name for glycerol phenylbutyrate | |
| Intervention | Trials |
| Urea Cycle Disorder | 2 |
| STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder | 1 |
| Achromatopsia | 1 |
| Urea Cycle Disorders | 1 |
| [disabled in preview] | 0 |
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Clinical Trial Locations for glycerol phenylbutyrate
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Clinical Trial Sponsors for glycerol phenylbutyrate
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