MRNA-3927 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
245プロピオン酸血症3

245. プロピオン酸血症


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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05130437
(ClinicalTrials.gov)
November 9, 20213/11/2021A Long-Term Extension Study to Evaluate the Safety and Clinical Activity of mRNA-3927A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 StudyPropionic AcidemiaBiological: mRNA-3927ModernaTX, Inc.NULLRecruiting1 YearN/AAll36Phase 1/Phase 2United States;Canada;United Kingdom
2NCT04159103
(ClinicalTrials.gov)
April 15, 20217/11/2019Open-Label Study of mRNA-3927 in Participants 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 AcidemiaPropionic AcidemiaBiological: mRNA-3927ModernaTX, Inc.NULLRecruiting1 YearN/AAll42Phase 1/Phase 2United States;Canada;United Kingdom
3EUCTR2019-003529-36-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