HLB-001 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
246メチルマロン酸血症1

246. メチルマロン酸血症


臨床試験数 : 19 薬物数 : 26 - (DrugBank : 3) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 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