HLB-001 ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
246Methylmalonic acidemia1

246. Methylmalonic acidemia


Clinical trials : 19 Drugs : 26 - (DrugBank : 3) / Drug target genes : 17 - Drug target pathways : 23
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04581785
(ClinicalTrials.gov)
May 29, 20212/10/2020Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic AcidemiaA Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT MutationsMethylmalonic AcidemiaBiological: hLB-001LogicBio Therapeutics, Inc.NULLRecruiting6 Months12 YearsAll8Phase 1/Phase 2United States;Saudi Arabia