337. ホモシスチン尿症 Homocystinuria Clinical trials / Disease details
臨床試験数 : 16 / 薬物数 : 19 - (DrugBank : 7) / 標的遺伝子数 : 2 - 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 | NCT01793090 (ClinicalTrials.gov) | January 2013 | 8/2/2013 | EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment | Phase 2, Double-Blind, Placebo Controlled Clinical Trial of EPI-743 in Subjects With Cobalamin C Defect | Methylmalonic Aciduria and Homocystinuria,Cblc Type;Genetic Disease;Retinopathy | Drug: Epi-743;Other: Placebo supplementation | Bambino Gesù Hospital and Research Institute | Catholic University of the Sacred Heart | Completed | 1 Year | 20 Years | All | 30 | Phase 2 | Italy |