Sebelipase alfa (SBC-102) ( DrugBank: Sebelipase alfa, SBC-102 )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
19ライソゾーム病1

19. ライソゾーム病


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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT01371825
(ClinicalTrials.gov)
May 4, 20119/6/2011Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase DeficiencyAn Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase DeficiencyLysosomal Acid Lipase Deficiency;Wolman DiseaseDrug: Sebelipase alfa (SBC-102)Alexion PharmaceuticalsNULLCompletedN/A24 MonthsAll9Phase 2/Phase 3United States;Egypt;France;Ireland;United Kingdom;Germany;India;Italy;Saudi Arabia;Taiwan;Turkey