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

2 trials found

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT05164055 (ClinicalTrials.gov)	July 11, 2022	16/11/2021	Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)	A French Multicenter Phase 4 Open Label Extension Study of Long Term Safety and Efficacy in Patients With Pompe Disease Who Previously Participated in Avalglucosidase Development Studies in France	Glycogen Storage Disease Type II	Drug: Avalglucosidase alfa (GZ402666)	Genzyme, a Sanofi Company	NULL	Recruiting	6 Months	N/A	All	18	Phase 4	France
2	NCT02782741 (ClinicalTrials.gov)	November 2, 2016	23/5/2016	Study 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 Disease	A 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 Disease	Glycogen Storage Disease Type II;Pompe's Disease	Drug: Avalglucosidase alfa (GZ402666);Drug: Alglucosidase alfa (GZ419829)	Genzyme, a Sanofi Company	NULL	Active, not recruiting	3 Years	N/A	All	100	Phase 3	United 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