246. Methylmalonic acidemia Clinical trials / Disease details
Clinical trials : 19 / Drugs : 26 - (DrugBank : 3) / Drug target genes : 17 - Drug target pathways : 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 | NCT04899310 (ClinicalTrials.gov) | August 6, 2021 | 19/5/2021 | A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia | 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 | Methylmalonic Acidemia | Biological: mRNA-3705 | ModernaTX, Inc. | NULL | Recruiting | 1 Year | N/A | All | 33 | Phase 1/Phase 2 | Canada;United Kingdom |