Modified mRNA encoding human methylmalonyl-coenzyme A mutase ( DrugBank: Coenzyme A, Methylmalonyl-coenzyme A )


1 disease
IDDisease name (Link within this page)Number of trials
246Methylmalonic acidemia1

246. Methylmalonic acidemia


Clinical trials : 23 Drugs : 31 - (DrugBank : 8) / Drug target genes : 17 - Drug target pathways : 23
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2020-004980-24-NL
(EUCTR)
28/07/2022A clinical study to evaluate mRNA-3705 in Patients with MMAA 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.NULLNAFemale: yes
Male: yes
33Phase 1;Phase 2France;United States;Canada;Spain;Australia;Netherlands;United Kingdom;Switzerland