Cipaglucosidase alfa (ATB200) ( DrugBank: Cipaglucosidase alfa )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
256筋型糖原病1

256. 筋型糖原病


臨床試験数 : 180 薬物数 : 153 - (DrugBank : 30) / 標的遺伝子数 : 31 - 標的パスウェイ数 : 134
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04808505
(ClinicalTrials.gov)
August 202210/3/2021Rossella: A Study to Evaluate the Safety, PK, Efficacy, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18An Open-label Study to Evaluate the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 YearsGlycogen Storage Disease Type II Infantile OnsetBiological: Cipaglucosidase alfa (ATB200);Drug: Miglustat (AT2221)Amicus TherapeuticsNULLRecruitingN/A17 YearsAll22Phase 3United States