246. メチルマロン酸血症 Methylmalonic acidemia Clinical trials / Disease details
臨床試験数 : 23 / 薬物数 : 31 - (DrugBank : 8) / 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 | NCT04732429 (ClinicalTrials.gov) | March 15, 2021 | 21/1/2021 | Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia | A Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia Followed by a Randomized, Double-blind, Placebo-controlled, 2-period Crossover Study and an Open-label, Long-term Extension Study | Methylmalonic Acidemia;Propionic Acidemia | Drug: HST5040;Drug: Placebo | HemoShear Therapeutics | NULL | Recruiting | 2 Years | N/A | All | 12 | Phase 2 | United States;Australia |