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	NCT01599286 (ClinicalTrials.gov)	September 2012	11/5/2012	Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia	Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia	Propionic Acidemia, Type I and/or Type II;Methylmalonic Acidemia;Carbamoyl-Phosphate Synthase I Deficiency Disease;Ornithine Carbamoyltransferase Deficiency	Drug: Carbaglu;Drug: Placebo;Drug: Standard of Care Treatment	Mendel Tuchman	Children's National Research Institute;Boston Children's Hospital;University Hospitals Cleveland Medical Center;University of California, Los Angeles;Children's Hospital of Philadelphia;Stanford University;Icahn School of Medicine at Mount Sinai;University of Pittsburgh;Children's Hospital Colorado	Active, not recruiting	N/A	99 Years	All	114	Phase 2	United States

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

NCT01599286
(ClinicalTrials.gov)

September 2012

11/5/2012

Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia

Propionic Acidemia, Type I and/or Type II;Methylmalonic Acidemia;Carbamoyl-Phosphate Synthase I Deficiency Disease;Ornithine Carbamoyltransferase Deficiency

Drug: Carbaglu;Drug: Placebo;Drug: Standard of Care Treatment

Mendel Tuchman

Children's National Research Institute;Boston Children's Hospital;University Hospitals Cleveland Medical Center;University of California, Los Angeles;Children's Hospital of Philadelphia;Stanford University;Icahn School of Medicine at Mount Sinai;University of Pittsburgh;Children's Hospital Colorado

Active, not recruiting

N/A

99 Years

All

114

Phase 2

United States