Phenylbutyrate (DrugBank: Phenylbutyrate)
11 diseases| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 2 | 筋萎縮性側索硬化症 | 2 |
| 3 | 脊髄性筋萎縮症 | 3 |
| 6 | パーキンソン病 | 1 |
| 8 | ハンチントン病 | 1 |
| 15 | 封入体筋炎 | 1 |
| 93 | 原発性胆汁性胆管炎［原発性胆汁性肝硬変 (~2017.3)］ | 2 |
| 94 | 原発性硬化性胆管炎 | 2 |
| 244 | メープルシロップ尿症 | 1 |
| 251 | 尿素サイクル異常症 | 4 |
| 297 | アラジール症候群 | 2 |
| 299 | 嚢胞性線維症 | 3 |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03127514 (ClinicalTrials.gov) | June 22, 2017 | 12/4/2017 | AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) | Evaluation of the Safety, Tolerability, Efficacy and Activity of AMX0035, a Fixed Combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for the Treatment of ALS | Amyotrophic Lateral Sclerosis;Motor Neuron Disease;Neuromuscular Diseases;Neurodegenerative Diseases;Spinal Cord Diseases;TDP-43 Proteinopathies;Nervous System Diseases;Central Nervous System Diseases | Drug: AMX0035;Other: Placebo | Amylyx Pharmaceuticals Inc. | ALS Finding a Cure Foundation;ALS Association;Northeast ALS Consortium;Massachusetts General Hospital Neurology Clinical Research Institute;Leandro P. Rizzuto Foundation | Completed | 18 Years | 80 Years | All | 137 | Phase 2;Phase 3 | United States |
| 2 | NCT00107770 (ClinicalTrials.gov) | April 2005 | 7/4/2005 | Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis) | Safety and Dose Escalating Study of Oral Sodium Phenylbutyrate in Subjects With Amyotrophic Lateral Sclerosis | Amyotrophic Lateral Sclerosis | Drug: sodium phenylbutyrate | Department of Veterans Affairs | Muscular Dystrophy Association | Completed | 18 Years | N/A | Both | 40 | Phase 1;Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT00439569 (ClinicalTrials.gov) | January 2008 | 21/2/2007 | Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III | Phase I/IIa Clinical Trial of Sodium Phenylbutyrate in Pediatric Subjects With Type II/III Spinal Muscular Atrophy | Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III | Drug: sodium phenylbutyrate | Westat | National Institute of Neurological Disorders and Stroke (NINDS) | Terminated | 2 Years | 11 Years | All | 9 | Phase 1;Phase 2 | United States |
| 2 | NCT00439218 (ClinicalTrials.gov) | January 2008 | 22/2/2007 | Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I | Phase I/IIa Clinical Trial of Sodium Phenylbutyrate in Pediatric Subjects With Type I Spinal Muscular Atrophy | Spinal Muscular Atrophy Type I | Drug: sodium phenylbutyrate | Westat | National Institute of Neurological Disorders and Stroke (NINDS) | Terminated | 2 Months | 48 Months | All | 5 | Phase 1;Phase 2 | United States |
| 3 | NCT00528268 (ClinicalTrials.gov) | July 2007 | 10/9/2007 | Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy | Prospective Phase I/II Study to Evaluate Effects of Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Sodium phenylbutyrate (NaPB) | University of Utah | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Completed | N/A | 6 Months | All | 14 | Phase 1;Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02046434 (ClinicalTrials.gov) | January 2014 | 23/1/2014 | Phenylbutyrate Response as a Biomarker for Alpha-synuclein Clearance From the Brain | Phenylbutyrate Response As a Biomarker for Alpha-Synuclein Clearance From Brain | Parkinson's Disease | Drug: Glycerol Phenylbutyrate | University of Colorado, Denver | NULL | Active, not recruiting | 21 Years | 80 Years | All | 40 | Phase 1 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT00212316 (ClinicalTrials.gov) | August 2005 | 19/9/2005 | Safety and Tolerability Study of Phenylbutyrate in Huntington's Disease (PHEND-HD) | Phenylbutyrate Development for Huntington's Disease (PHEND-HD): A Multi-Center, Double-Blind, Placebo-Controlled Study With Open-Label Follow-Up to Determine the Safety and Tolerability of Phenylbutyrate in Subjects With Huntington's Disease | Huntington's Disease | Drug: sodium phenylbutyrate | University of Rochester | HP Therapeutics Foundation;Massachusetts General Hospital;Columbia University;University of Iowa;University of California, San Diego;University of Kansas;University of Alabama at Birmingham;Johns Hopkins University | Completed | 18 Years | N/A | Both | 60 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04421677 (ClinicalTrials.gov) | August 20, 2020 | 18/5/2020 | Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis | Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis | Inclusion Body Myositis;Sporadic Inclusion Body Myositis | Drug: Phenylbutyrate Oral Tablet | University of Kansas Medical Center | NULL | Active, not recruiting | 18 Years | N/A | All | 10 | Phase 1 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-UMIN000012782 | 2014/02/01 | 01/02/2014 | Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. | Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. | Group A; phenylbutyrate 6g (Child 100mg/kg)/day *7days Group B; phenylbutyrate 6g (Child 100mg/kg)/day *3days and 12g (Child 200mg/kg)/day *4days Group C; phenylbutyrate 6g (Child 100mg/kg)/day *1day, phenylbutyrate 12g (Child 200mg/kg)/day *2days and phenylbutyrate 21g (Child 300mg/kg)/day *4days | Juntendo University | NULL | Pending | Not applicable | Not applicable | Male and Female | 2 | Not selected | Japan | |
| 2 | JPRN-UMIN000003802 | 2010/04/01 | 22/06/2010 | Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. | Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. | phenylbutyrate(Child 250mg/kg/day)for 1-4months : phenylbutyrate(Child 350mg/kg/day)for 1-4months : phenylbutyrate(Child 500mg/kg/day)for 1-4months | Saiseikai Yokohama City Tobu Hospital | Laboratory of Molecular Pharmacokinetics, Graduate School of Pharmaceutical Sciences, The University of Tokyo (Tokyo) | Recruiting | Not applicable | Not applicable | Male and Female | 15 | Not selected | Japan |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-UMIN000012782 | 2014/02/01 | 01/02/2014 | Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. | Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. | Group A; phenylbutyrate 6g (Child 100mg/kg)/day *7days Group B; phenylbutyrate 6g (Child 100mg/kg)/day *3days and 12g (Child 200mg/kg)/day *4days Group C; phenylbutyrate 6g (Child 100mg/kg)/day *1day, phenylbutyrate 12g (Child 200mg/kg)/day *2days and phenylbutyrate 21g (Child 300mg/kg)/day *4days | Juntendo University | NULL | Pending | Not applicable | Not applicable | Male and Female | 2 | Not selected | Japan | |
| 2 | JPRN-UMIN000003802 | 2010/04/01 | 22/06/2010 | Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. | Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. | phenylbutyrate(Child 250mg/kg/day)for 1-4months : phenylbutyrate(Child 350mg/kg/day)for 1-4months : phenylbutyrate(Child 500mg/kg/day)for 1-4months | Saiseikai Yokohama City Tobu Hospital | Laboratory of Molecular Pharmacokinetics, Graduate School of Pharmaceutical Sciences, The University of Tokyo (Tokyo) | Recruiting | Not applicable | Not applicable | Male and Female | 15 | Not selected | Japan |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT01529060 (ClinicalTrials.gov) | February 2013 | 6/2/2012 | Phenylbutyrate Therapy for Maple Syrup Urine Disease | A Double-Blind, Randomized, Placebo-Controlled, Crossover Trial of Phenylbutyrate in the Treatment of Maple Syrup Urine Disease | Maple Syrup Urine Disease | Drug: Phenylbutyrate;Drug: Placebo powder | Brendan Lee | NULL | Completed | 3 Years | N/A | All | 20 | Phase 2;Phase 3 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03335488 (ClinicalTrials.gov) | February 20, 2018 | 18/10/2017 | Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders | A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders | Urea Cycle Disorder | Drug: RAVICTI;Drug: NaPBA | Horizon Therapeutics, LLC | NULL | Recruiting | N/A | 99 Years | All | 18 | Phase 4 | United States;Belgium;Italy;Spain;Switzerland |
| 2 | NCT02246218 (ClinicalTrials.gov) | December 31, 2014 | 17/9/2014 | A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders | An Open Label Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate (GPB; RAVICTI®) in Pediatric Subjects Under Two Years of Age With Urea Cycle Disorders (UCDs) | Urea Cycle Disorder | Drug: RAVICTI | Horizon Therapeutics, LLC | NULL | Completed | N/A | 2 Years | All | 27 | Phase 4 | United States;Canada |
| 3 | EUCTR2008-003865-23-GB (EUCTR) | 08/09/2008 | 25/07/2008 | A Phase 2, Switch-Over, Dose-Escalation Study of the Safety and Tolerability of HPN-100 (glyceryl tri-[4-phenylbutyrate]) Compared to Sodium Phenylbutyrate in Adult and Pediatric Subjects with Urea Cycle Disorders | A Phase 2, Switch-Over, Dose-Escalation Study of the Safety and Tolerability of HPN-100 (glyceryl tri-[4-phenylbutyrate]) Compared to Sodium Phenylbutyrate in Adult and Pediatric Subjects with Urea Cycle Disorders | Urea Cycle Disorder (urea cycle enzyme or transporter deficiency) MedDRA version: 9.1;Level: LLT;Classification code 10013373;Term: Disorders of urea cycle metabolism | Product Name: glyceryl tri-(4-phenylbutyrate)(GT4P) Product Code: HPN-100 INN or Proposed INN: NA Other descriptive name: glyceryl tri-(4-phenylbutyrate) | Hyperion Therapeutics Limited | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 2 | United Kingdom | ||
| 4 | NCT00345605 (ClinicalTrials.gov) | February 2008 | 26/6/2006 | Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder | A Randomized, Double-Blind, Crossover Study of Sodium Phenylbutyrate and Low-Dose Arginine Compared to High-Dose Arginine Alone on Liver Function, Ureagenesis and Subsequent Nitric Oxide Production in Patients With Argininosuccinic Aciduria | Argininosuccinic Aciduria;Amino Acid Metabolism, Inborn Errors;Urea Cycle Disorders | Drug: Sodium Phenylbutyrate;Drug: Arginine | Brendan Lee | Office of Rare Diseases (ORD);Rare Diseases Clinical Research Network;Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Completed | 5 Years | N/A | All | 12 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-UMIN000012782 | 2014/02/01 | 01/02/2014 | Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. | Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. | Group A; phenylbutyrate 6g (Child 100mg/kg)/day *7days Group B; phenylbutyrate 6g (Child 100mg/kg)/day *3days and 12g (Child 200mg/kg)/day *4days Group C; phenylbutyrate 6g (Child 100mg/kg)/day *1day, phenylbutyrate 12g (Child 200mg/kg)/day *2days and phenylbutyrate 21g (Child 300mg/kg)/day *4days | Juntendo University | NULL | Pending | Not applicable | Not applicable | Male and Female | 2 | Not selected | Japan | |
| 2 | JPRN-UMIN000003802 | 2010/04/01 | 22/06/2010 | Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. | Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. | phenylbutyrate(Child 250mg/kg/day)for 1-4months : phenylbutyrate(Child 350mg/kg/day)for 1-4months : phenylbutyrate(Child 500mg/kg/day)for 1-4months | Saiseikai Yokohama City Tobu Hospital | Laboratory of Molecular Pharmacokinetics, Graduate School of Pharmaceutical Sciences, The University of Tokyo (Tokyo) | Recruiting | Not applicable | Not applicable | Male and Female | 15 | Not selected | Japan |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02323100 (ClinicalTrials.gov) | December 2, 2018 | 18/12/2014 | Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis) | A Double Blind, Placebo Controlled, Dose Escalation Trial of Glycerol Phenylbutyrate Corrector Therapy for Cystic Fibrosis | Cystic Fibrosis | Drug: Ravicti low dose;Drug: Ravicti high dose;Drug: Placebo | National Jewish Health | University of Alabama at Birmingham;Children's Hospital of Philadelphia;Johns Hopkins University;Horizon Pharma Ireland, Ltd., Dublin Ireland | Suspended | 18 Years | N/A | All | 36 | Phase 1;Phase 2 | United States |
| 2 | NCT00590538 (ClinicalTrials.gov) | February 2003 | 27/12/2007 | Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygotes (for Cystic Fibrosis) | A Pilot Trial of Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygous Cystic Fibrosis Patients | Cystic Fibrosis | Drug: Sodium 4-Phenylbutyrate;Drug: Genistein (Unconjugated Isoflavones 100);Drug: Placebo | Children's Hospital of Philadelphia | Cystic Fibrosis Foundation Therapeutics | Terminated | 18 Years | N/A | All | 9 | Phase 1;Phase 2 | United States |
| 3 | NCT00016744 (ClinicalTrials.gov) | September 2001 | 31/5/2001 | Phenylbutyrate/Genistein Duotherapy in Delta F508-Homozygous(for Cystic Fibrosis) | A Pilot Trial of Phenylbutyrate/Genistein Duotherapy in Delta F508-Homozygous Cystic Fibrosis Patients | Cystic Fibrosis | Drug: Sodium 4-Phenylbutyrate (4PBA);Drug: Unconjugated Isoflavones 100 (PTI G-4660, 87% Genistein);Drug: Placebo | Children's Hospital of Philadelphia | Cystic Fibrosis Foundation Therapeutics;National Center for Research Resources (NCRR) | Completed | 18 Years | N/A | Both | 12 | Phase 1;Phase 2 | United States |