246. Methylmalonic acidemia Clinical trials / Disease details
Clinical trials : 23 / Drugs : 31 - (DrugBank : 8) / 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 | NCT05295433 (ClinicalTrials.gov) | March 8, 2022 | 14/2/2022 | An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in the mRNA-3705-P101 Study | A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in the mRNA-3705-P101 Study | Methylmalonic Acidemia | Drug: mRNA-3705 | ModernaTX, Inc. | NULL | Recruiting | 1 Year | N/A | All | 33 | Phase 1/Phase 2 | Canada;United Kingdom |
| 2 | 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 | United States;Canada;Netherlands;United Kingdom |
| 3 | 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 |