Sodium phenylbutyrate ( DrugBank: Phenylbutyrate )
4 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 |
| 8 | ハンチントン病 | 1 |
| 251 | 尿素サイクル異常症 | 3 |
2. 筋萎縮性側索硬化症
臨床試験数 : 624 / 薬物数 : 611 - (DrugBank : 160) / 標的遺伝子数 : 172 - 標的パスウェイ数 : 225
| 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 | NCT05286372 (ClinicalTrials.gov) | April 2022 | 9/3/2022 | An Intermediate Size Expanded Access Protocol of AMX0035 for ALS | An Intermediate Size, Expanded Access Protocol to Provide AMX0035, a Fixed Combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO), for the Treatment of Adult Patients With Amyotrophic Lateral Sclerosis (ALS) | Amyotrophic Lateral Sclerosis | Drug: AMX0035 | Amylyx Pharmaceuticals Inc. | NULL | Available | 18 Years | N/A | All | NULL | ||
| 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 |
3. 脊髄性筋萎縮症
臨床試験数 : 217 / 薬物数 : 149 - (DrugBank : 33) / 標的遺伝子数 : 54 - 標的パスウェイ数 : 80
| 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 | 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 |
| 2 | 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 |
| 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 |
8. ハンチントン病
臨床試験数 : 229 / 薬物数 : 193 - (DrugBank : 60) / 標的遺伝子数 : 84 - 標的パスウェイ数 : 158
| 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 |
251. 尿素サイクル異常症
臨床試験数 : 48 / 薬物数 : 52 - (DrugBank : 13) / 標的遺伝子数 : 4 - 標的パスウェイ数 : 29
| 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 | 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 | ||
| 3 | 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 |