245. プロピオン酸血症
[臨床試験数:9,薬物数:15(DrugBank:3),標的遺伝子数:1,標的パスウェイ数:6

Searched query = "Propionic 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.

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1 trial found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Inclusion_
agemin
Inclusion_
agemax
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PhaseCountries
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