MRT5201 ( DrugBank: - )
1 disease
| 告示番号 | 疾患名(ページ内リンク) | 臨床試験数 | 
|---|---|---|
| 251 | 尿素サイクル異常症 | 1 | 
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 | NCT03767270 (ClinicalTrials.gov)  | December 2019 | 28/11/2018 | Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency | A Phase 1/2 Single Ascending Dose Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenously Administered MRT5201 in Subjects With Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency | Biological: MRT5201;Other: Placebo | Translate Bio, Inc. | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 1/Phase 2 | NULL |