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	EUCTR2019-003595-38-GB (EUCTR)	20/11/2020	03/07/2020	FORTIS: AAV8-Delivered Gene Therapy in Late Onset Pompe Disease	FORTIS: A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients with Late Onset Pompe Disease - FORTIS	Late 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] - Musculoskeletal Diseases [C05]	Product Name: AT845 Product Code: AT845	Audentes Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	8	Phase 1;Phase 2	Germany;Italy;United Kingdom
2	NCT04174105 (ClinicalTrials.gov)	October 27, 2020	13/11/2019	Gene Transfer Study in Patients With Late Onset Pompe Disease	A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients With Late Onset Pompe Disease	Pompe Disease (Late-onset)	Genetic: AT845	Astellas Gene Therapies	NULL	Recruiting	18 Years	80 Years	All	12	Phase 1/Phase 2	United States;United Kingdom;Germany