245. プロピオン酸血症 Propionic acidemia Clinical trials / Disease details


臨床試験数 : 13 薬物数 : 16 - (DrugBank : 3) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 6

  
1 trial found
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1EUCTR2019-003529-36-GB
(EUCTR)
18/12/202003/03/2020A clinical study to evaluate the medicinal product in Patients with Propionic AcidemiaA Global, Phase 1/2, Open-label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia Propionic Acidemia- Propionic acidemia is an autosomal recessive disorder caused by loss-of-function mutations in PCCA or PCCB (and in rare instances, mutations in both PCCA and PCCB), resulting in the lossof enzymatic activity of the mitochondrial complex PCC
MedDRA version: 21.0;Level: LLT;Classification code 10080615;Term: Propionic acidemia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: mRNA-3927
INN or Proposed INN: N/A
Other descriptive name: CX-016492
INN or Proposed INN: NA
Other descriptive name: CX-017950
ModernaTX, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
36Phase 1;Phase 2United States;Canada;United Kingdom