246. Methylmalonic acidemia
14 clinical trials,   22 drugs   (DrugBank: 2 drugs),   1 drug target gene,   6 drug target pathways

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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
2 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2019-001061-32-GB
(EUCTR)
25/10/201930/05/2019A clinical study to evaluate mRNA-3704 in Patients with MMAA 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.NULLNot RecruitingFemale: yes
Male: yes
34Phase 1;Phase 2United States;United Kingdom
2NCT03810690
(ClinicalTrials.gov)
May 28, 201914/1/2019Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic AcidemiaA 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 DeficiencyMethylmalonic Acidemia (MMA);Metabolism, Inborn ErrorsBiological: mRNA-3704ModernaTX, Inc.NULLWithdrawn1 YearN/AAll0Phase 1;Phase 2United States