256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details


臨床試験数 : 180 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 105

  
4 trials found
No.TrialIDDate_
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PhaseCountries
1NCT04848779
(ClinicalTrials.gov)
June 10, 202129/3/2021A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients =6 Months of Age With Infantile-onset Pompe Disease (IOPD)A Prospective Observational Study to Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients =6 Months of Age With Infantile-onset Pompe Disease (IOPD)Glycogen Storage Disease Type IIDrug: Alglucosidase alfa GZ419829SanofiNULLRecruiting0 Days6 MonthsAll16Belgium;France;Germany;Italy;Netherlands;Taiwan;United Kingdom
2NCT03019406
(ClinicalTrials.gov)
October 12, 201720/12/2016A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase AlfaAn Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical ResponseGlycogen Storage Disease Type II-Pompe's DiseaseDrug: avalglucosidase alfa GZ402666;Drug: alglucosidase alfa GZ419829Genzyme, a Sanofi CompanyNULLActive, not recruiting6 Months17 YearsAll22Phase 2United States;France;Japan;Taiwan;United Kingdom
3NCT02782741
(ClinicalTrials.gov)
November 2, 201623/5/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients With Late-onset Pompe DiseaseGlycogen Storage Disease Type II;Pompe's DiseaseDrug: Avalglucosidase alfa (GZ402666);Drug: Alglucosidase alfa (GZ419829)Genzyme, a Sanofi CompanyNULLActive, not recruiting3 YearsN/AAll100Phase 3United States;Argentina;Australia;Austria;Belgium;Brazil;Canada;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Switzerland;Taiwan;Turkey;United Kingdom;Bulgaria;Colombia;Czech Republic;Sweden
4EUCTR2021-004047-25-Outside-EU/EEA
(EUCTR)
27/07/2021Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa TreatmentA Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa Treatment - APOLLO-IOPD Infantile-Onset Pompe Disease
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: MYOZYME®
Product Name: Alglucosidase Alfa
Product Code: GZ419829
INN or Proposed INN: ALGLUCOSIDASE ALFA
Sanofi(China) Investment Co Ltd, Shanghai BranchNULLNAFemale: yes
Male: yes
Phase 4China