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

3 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	NCT04020055 (ClinicalTrials.gov)	October 31, 2022	24/6/2019	A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease	An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects With F ...	Fabry Disease	Drug: migalastat HCl 150 mg	Amicus Therapeutics	NULL	Recruiting	18 Years	N/A	All	14	Phase 3	United States;Australia;France;Japan;Spain;Belgium;Italy;United Kingdom
2	NCT03500094 (ClinicalTrials.gov)	October 11, 2018	9/4/2018	Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 1 ...	An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Trea ...	Fabry Disease	Drug: migalastat HCl 150 mg	Amicus Therapeutics	NULL	Completed	12 Years	17 Years	All	22	Phase 3	United States;Spain;United Kingdom
3	NCT02194985 (ClinicalTrials.gov)	October 2014	17/7/2014	Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease ...	An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlor ...	Fabry Disease	Drug: migalastat HCl 150 mg	Amicus Therapeutics	NULL	Completed	18 Years	N/A	All	76	Phase 3	United States;Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spa ...

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