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 | EUCTR2019-001061-32-GB (EUCTR) | 25/10/2019 | 30/05/2019 | A clinical study to evaluate mRNA-3704 in Patients with MMA | A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients 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-3704 INN or Proposed INN: NA Other descriptive name: NA | ModernaTX, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 34 | Phase 1;Phase 2 | United States;United Kingdom | ||
| 2 | NCT03810690 (ClinicalTrials.gov) | May 28, 2019 | 14/1/2019 | Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia | A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency | Methylmalonic Acidemia (MMA);Metabolism, Inborn Errors | Biological: mRNA-3704 | ModernaTX, Inc. | NULL | Withdrawn | 1 Year | N/A | All | 0 | Phase 1/Phase 2 | United States |