246. メチルマロン酸血症 Methylmalonic acidemia Clinical trials / Disease details


臨床試験数 : 23 薬物数 : 31 - (DrugBank : 8) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 23

  
3 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1NCT05295433
(ClinicalTrials.gov)
March 8, 202214/2/2022An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in the mRNA-3705-P101 StudyA 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 StudyMethylmalonic AcidemiaDrug: mRNA-3705ModernaTX, Inc.NULLRecruiting1 YearN/AAll33Phase 1/Phase 2Canada;United Kingdom
2NCT04899310
(ClinicalTrials.gov)
August 6, 202119/5/2021A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic AcidemiaA 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 DeficiencyMethylmalonic AcidemiaBiological: mRNA-3705ModernaTX, Inc.NULLRecruiting1 YearN/AAll33Phase 1/Phase 2United States;Canada;Netherlands;United Kingdom
3EUCTR2020-004980-24-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