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

12 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 HClin Subjects With Fa ...	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 ...
4	NCT02082327 (ClinicalTrials.gov)	March 2014	6/3/2014	A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Vo ...	A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Dose Escalation Trial to Evaluate th ...	Fabry Disease	Drug: IV migalastat HCl;Drug: IV placebo;Drug: oral migalastat HCl	Amicus Therapeutics	NULL	Completed	18 Years	45 Years	Both	31	Phase 1	Netherlands
5	NCT01476163 (ClinicalTrials.gov)	December 2011	17/11/2011	Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease ...	Physician Initiated Expanded Access Request for Treatment Use of Migalastat Hydrochloride (AT1001), ...	Fabry Disease	Drug: migalastat HCl	Amicus Therapeutics	NULL	Approved for marketing	16 Years	74 Years	All			United States;Australia
6	NCT01730469 (ClinicalTrials.gov)	August 2011	8/11/2012	Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaire ...	An Open-Label Study to Determine the Safety and Pharmacokinetics of AT1001 in Subjects With Impaired ...	Fabry Disease	Drug: AT1001 150 mg	Amicus Therapeutics	NULL	Completed	18 Years	75 Years	All	32	Phase 1	United States
7	NCT01196871 (ClinicalTrials.gov)	February 2, 2011	7/9/2010	Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants ...	An Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 (Migalastat Hydroc ...	Fabry Disease	Drug: Migalastat HCl;Biological: Agalsidase Beta;Biological: Agalsidase Alfa	Amicus Therapeutics	NULL	Completed	18 Years	65 Years	Male	20	Phase 2	United States;Australia;Belgium;Canada;Netherlands;France;United Kingdom
8	NCT00526071 (ClinicalTrials.gov)	September 17, 2007	5/9/2007	Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Di ...	Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Pharmacodynamics of AT ...	Fabry Disease	Drug: migalastat HCl	Amicus Therapeutics	NULL	Terminated	18 Years	N/A	All	23	Phase 2	United States;Australia;Brazil;France;United Kingdom
9	NCT00304512 (ClinicalTrials.gov)	September 7, 2006	17/3/2006	A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participan ...	A Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and ...	Fabry Disease	Drug: migalastat HCl	Amicus Therapeutics	NULL	Completed	18 Years	65 Years	Female	9	Phase 2	United States;Australia;Brazil;Canada;France;United Kingdom
10	NCT00283959 (ClinicalTrials.gov)	June 27, 2006	27/1/2006	A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With ...	A Phase 2, Open-Label, Single Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Ph ...	Fabry Disease	Drug: migalastat HCl	Amicus Therapeutics	NULL	Completed	18 Years	65 Years	Male	4	Phase 2	Australia;Brazil

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