246. メチルマロン酸血症 Methylmalonic acidemia Clinical trials / Disease details
臨床試験数 : 19 / 薬物数 : 26 - (DrugBank : 3) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 23
| 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-jRCT2041210065 | 04/09/2021 | 04/09/2021 | A Phase I/II Study of Bucladesine in Patients with Severe Methylmalonic Acidemia | A Phase I/II Multi-Institutional Study of Bucladesine in Patients with Severe Methylmalonic Acidemia - McAMP | methylmalonic acidemia;C537358 | Bucladesine will be administered intravenously over 60 minutes once a week. Starting dose is 0.3mg/kg. Dosage will be incerased to 0.55mg/kg, then 1.65mg/kg with monitoring PK and safety data. | Ito Tetsuya | NULL | Recruiting | >= 3month old | Not applicable | Both | 6 | Phase 1-2 | Japan |