251. 尿素サイクル異常症 Urea cycle disorder Clinical trials / Disease details
臨床試験数 : 54 / 薬物数 : 61 - (DrugBank : 15) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 28
| 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 | NCT05345171 (ClinicalTrials.gov) | October 18, 2022 | 18/4/2022 | Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency | A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency | OTC Deficiency | Genetic: DTX301;Other: Placebo;Drug: Oral Corticosteroids;Drug: Placebo for oral corticosteroids;Drug: Sodium Acetate | Ultragenyx Pharmaceutical Inc | NULL | Recruiting | 12 Years | N/A | All | 50 | Phase 3 | United States;Argentina;Australia;Brazil;Canada;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom |