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	EUCTR2019-001061-32-GB (EUCTR)	25/10/2019	30/05/2019	A clinical study to evaluate mRNA-3704 in Patients with MMA	A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients with Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency	Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]	Product Name: mRNA-3704 INN or Proposed INN: NA Other descriptive name: NA	ModernaTX, Inc.	NULL	Not Recruiting			Female: yes Male: yes	34	Phase 1;Phase 2	United States;United Kingdom
2	NCT03810690 (ClinicalTrials.gov)	May 28, 2019	14/1/2019	Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia	A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency	Methylmalonic Acidemia (MMA);Metabolism, Inborn Errors	Biological: mRNA-3704	ModernaTX, Inc.	NULL	Withdrawn	1 Year	N/A	All	0	Phase 1;Phase 2	United States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

EUCTR2019-001061-32-GB
(EUCTR)

25/10/2019

30/05/2019

A clinical study to evaluate mRNA-3704 in Patients with MMA

A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients with Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency

Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Product Name: mRNA-3704
INN or Proposed INN: NA
Other descriptive name: NA

ModernaTX, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 1;Phase 2

United States;United Kingdom

NCT03810690
(ClinicalTrials.gov)

May 28, 2019

14/1/2019

Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia

A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency

Methylmalonic Acidemia (MMA);Metabolism, Inborn Errors

Biological: mRNA-3704

ModernaTX, Inc.

NULL

Withdrawn

1 Year

N/A

All

Phase 1;Phase 2

United States