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

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

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	NCT02185651 (ClinicalTrials.gov)	October 2016	1/7/2014	A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction	A Pilot Study of the Effects of Oral Administration of Zavesca® on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy	Pompe Disease;Hypersensitivity Reaction	Drug: Zavesca ® Prescription	University of Florida	Amicus Therapeutics	Terminated	18 Years	65 Years	All	2	Phase 1	United States
2	EUCTR2010-024647-32-IT (EUCTR)	18/03/2011	09/05/2011	Combined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease - ND	Combined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease - ND	Patients with Pompe Disease (Type II Glycogenosis) on therapy with Enzyme Replacement Therapy (ERT) for at least 6 months MedDRA version: 13.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders	Trade Name: ZAVESCA INN or Proposed INN: Miglustat	UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO II	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes			Italy