19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

  
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
1NCT05629559
(ClinicalTrials.gov)
October 16, 20224/11/20224D-310 in Adults With Fabry Disease and Cardiac InvolvementAn Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac InvolvementFabry DiseaseBiological: 4D-3104D Molecular TherapeuticsNULLActive, not recruiting18 YearsN/AAll18Phase 1/Phase 2Australia;Taiwan
2NCT04519749
(ClinicalTrials.gov)
September 1, 202014/8/2020An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry DiseaseAn Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry DiseaseFabry DiseaseBiological: 4D-3104D Molecular TherapeuticsNULLActive, not recruiting18 YearsN/AAll18Phase 1/Phase 2United States