246. メチルマロン酸血症 Methylmalonic acidemia Clinical trials / Disease details


臨床試験数 : 23 薬物数 : 31 - (DrugBank : 8) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 23

  
2 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05506254
(ClinicalTrials.gov)
July 20, 202216/8/2022Long-term Follow-up Study of Patients Who Received hLB-001 Gene TherapyA Long-term Follow-up Study of Patients in LogicBio Clinical Trials for Methylmalonic Acidemia Characterized by MMUT Mutations Who Received hLB-001Methylmalonic AcidemiaDrug: hLB-001LogicBio Therapeutics, IncAlexionActive, not recruitingN/AN/AAll8United States
2NCT04581785
(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, IncAlexionActive, not recruiting6 Months12 YearsAll8Phase 1/Phase 2United States;Saudi Arabia