DDrare: Database of Drug Development for Rare Diseases

246. メチルマロン酸血症
［臨床試験数：14，薬物数：22（DrugBank：2），標的遺伝子数：1，標的パスウェイ数：6］

Searched query = "Methylmalonic 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 = 291844; ATALUREN; Ataluren; Ataluren 1000mg; Ataluren 125mg; Ataluren 250mg; Carbaglu; Carglumic Acid; Carglumic Acid (Carbaglu®); Carglumic acid; Compound 1a; HLB-001; MRNA-3704; N-Carbamylglutamate; N-carbamylglutamate; Other: Standard of Care; PTC-0161480; PTC-124; PTC-C124; PTC124; RPS 2505; 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	NCT04284917 (ClinicalTrials.gov)	November 28, 2019	24/2/2020	Long-term Efficacy of Carglumic Acid in Organic Acidemia.	Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).	Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)	Drug: Carglumic Acid (Carbaglu®)	National Taiwan University Hospital	NULL	Enrolling by invitation	18 Years	N/A	All	5	N/A	Taiwan

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

NCT04284917
(ClinicalTrials.gov)

November 28, 2019

24/2/2020

Long-term Efficacy of Carglumic Acid in Organic Acidemia.

Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).

Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)

Drug: Carglumic Acid (Carbaglu®)

National Taiwan University Hospital

NULL

Enrolling by invitation

18 Years

N/A

All

N/A

Taiwan