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

1 trial 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	EUCTR2018-000192-33-GB (EUCTR)	17/08/2018	09/04/2018	A phase 1/2 study to evaluate the safety and tolerability of SB-913, a rAAV2/6-based gene therapy in ...	A Phase 1/2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Toler ...	Mucopolysaccharidosis type II (MPS II) MedDRA version: 20.1;Level: PT;Classification code 10056889;T ...	Product Name: rAAV2/6 Left ZFN Vector Product Code: SB-47171 INN or Proposed INN: Not yet assigned Othe ...	Sangamo Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	32	Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ...	United States;United Kingdom

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