19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details

臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

2 trials found

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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	EUCTR2015-003105-41-GB (EUCTR)	13/10/2015	08/09/2015	Treatment for Nonsense Mutation Mucopolysaccharidosis Type I	CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Atal ...	Nonsense mutation Mucopolysaccharidosis Type I MedDRA version: 18.1;Level: PT;Classification code 10 ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product ...	PTC Therapeutics Inc.	NULL	Not Recruiting			Female: yes Male: yes	5	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ...	United Kingdom
2	EUCTR2014-002596-28-GB (EUCTR)	28/01/2015	12/06/2015	Treatment for Nonsense Mutation Mucopolysaccharidosis Type I	A Phase 2 Study of Ataluren (PTC124) as Treatment for Nonsense Mutation Mucopolysaccharidosis Type I ...	Nonsense mutation Mucopolysaccharidosis Type I MedDRA version: 20.1;Level: PT;Classification code 10 ...	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product ...	PTC Therapeutics Inc.	NULL	Not Recruiting			Female: yes Male: yes	18	Phase 2	Germany;United Kingdom

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