DDrare: Database of Drug Development for Rare Diseases

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	NCT04581785 (ClinicalTrials.gov)	January 15, 2021	2/10/2020	Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia	A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations	Methylmalonic Acidemia	Biological: hLB-001	LogicBio Therapeutics, Inc.	NULL	Not yet recruiting	6 Months	12 Years	All	8	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

NCT04581785
(ClinicalTrials.gov)

January 15, 2021

2/10/2020

Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia

A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations

Methylmalonic Acidemia

Biological: hLB-001

LogicBio Therapeutics, Inc.

NULL

Not yet recruiting

6 Months

12 Years

All

Phase 1;Phase 2

United States