240. フェニルケトン尿症 Phenylketonuria Clinical trials / Disease details
臨床試験数 : 138 / 薬物数 : 106 - (DrugBank : 11) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5
| 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 | NCT02677870 (ClinicalTrials.gov) | January 2018 | 26/1/2016 | The Effectiveness of Kuvan in Amish PKU Patients | The Effectiveness of High-Dose Synthetic BH4 (Saproterin Dihydrochloride or Kuvan) in Amish PKU Patients | Phenylketonuria | Drug: saproterin dihydrochloride;Other: Diet treatment | University Hospitals Cleveland Medical Center | BioMarin Pharmaceutical | Recruiting | 2 Years | 60 Years | All | 25 | Phase 4 | United States |
| 2 | EUCTR2005-003778-13-DE (EUCTR) | 13/07/2006 | 14/06/2006 | A Phase 2, Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Hyperphenylalaninemia Due to Primary BH4 Deficiency | A Phase 2, Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Hyperphenylalaninemia Due to Primary BH4 Deficiency | Phenylketonuria (PKU) MedDRA version: 8.1;Level: LLT;Classification code 10034872;Term: Phenylketonuria | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin Other descriptive name: saproterin dihydrochloride | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | Germany |