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 | EUCTR2020-004980-24-NL (EUCTR) | 28/07/2022 | A clinical study to evaluate mRNA-3705 in Patients with MMA | A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants with Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency | Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: mRNA-3705 INN or Proposed INN: NA Other descriptive name: Modified mRNA encoding human methylmalonyl-coenzyme A mutase | ModernaTX, Inc. | NULL | NA | Female: yes Male: yes | 33 | Phase 1;Phase 2 | France;United States;Canada;Spain;Australia;Netherlands;United Kingdom;Switzerland |