245. プロピオン酸血症 Propionic acidemia Clinical trials / Disease details
臨床試験数 : 13 / 薬物数 : 16 - (DrugBank : 3) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 6
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 |
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1 | NCT04284917 (ClinicalTrials.gov) | November 28, 2019 | 24/2/2020 | Long-term Efficacy of Carglumic Acid in Organic Acidemia. | Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA). | Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA) | Drug: Carglumic Acid (Carbaglu®) | National Taiwan University Hospital | NULL | Enrolling by invitation | 18 Years | N/A | All | 5 | N/A | Taiwan |