246. メチルマロン酸血症 ［臨床試験数：14，薬物数：22（DrugBank：2），標的遺伝子数：1，標的パスウェイ数：6］
Searched query = "Methylmalonic acidemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
|1||EUCTR2019-001061-32-GB||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|
|34||Phase 1;Phase 2||United States;United Kingdom|
|2||NCT03810690||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Deficiency||Methylmalonic Acidemia (MMA);Metabolism, Inborn Errors||Biological: mRNA-3704||ModernaTX, Inc.||NULL||Withdrawn||1 Year||N/A||All||0||Phase 1/Phase 2||United States|