251. 尿素サイクル異常症 Urea cycle disorder Clinical trials / Disease details
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Deficiency | Ornithine Transcarbamylase Deficiency | Biological: MRT5201;Other: Placebo | Translate Bio, Inc. | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 1/Phase 2 | NULL |