DDrare: Database of Drug Development for Rare Diseases

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.

Drug = CX-016492; CX-017950; Carbaglu; Carglumic Acid; Carglumic Acid (Carbaglu®); Carglumic acid; Disodium citrate; Glutamine; MRNA-3927; N-Carbamylglutamate; N-carbamylglutamate; Ornithine; Ornithine alpha ketoglutarate; Other: Standard of Care; Standard of Care Treatment

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2019-003529-36-GB (EUCTR)	18/12/2020	03/03/2020	A clinical study to evaluate the medicinal product in Patients with Propionic Acidemia	A 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, Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	36	Phase 1;Phase 2	United States;Canada;United Kingdom

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EUCTR2019-003529-36-GB
(EUCTR)

18/12/2020

03/03/2020

A clinical study to evaluate the medicinal product in Patients with Propionic Acidemia

A 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, Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 1;Phase 2

United States;Canada;United Kingdom