240. フェニルケトン尿症 Phenylketonuria Clinical trials / Disease details
臨床試験数 : 143 / 薬物数 : 90 - (DrugBank : 10) / 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 | NCT03952156 (ClinicalTrials.gov) | June 10, 2019 | 9/5/2019 | Gene Therapy Clinical Study in Adult PKU | A Phase 1/2 Open-Label, Randomized, Concurrently-Controlled, Dose Escalation Study to Evaluate the Safety and Efficacy of HMI-102 in Adult PKU Subjects With PAH Deficiency | Phenylketonurias;PAH Deficiency | Genetic: HMI-102 | Homology Medicines, Inc | NULL | Active, not recruiting | 18 Years | 55 Years | All | 28 | Phase 1/Phase 2 | United States |