19. Lysosomal storage disease Clinical trials / Disease details

Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191

4 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	NCT04020055 (ClinicalTrials.gov)	October 1, 2022	24/6/2019	A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variants and Severe Renal Impairment	An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Fabry Subjects With Amenable GLA Variants and Severe Renal Impairment	Fabry Disease	Drug: migalastat HCl 150 mg	Amicus Therapeutics	NULL	Not yet recruiting	16 Years	N/A	All	12	Phase 3	United States;Belgium;France;Italy;Spain;United Kingdom
2	NCT04049760 (ClinicalTrials.gov)	October 14, 2019	30/7/2019	Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease	A Long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants	Fabry Disease	Drug: migalastat HCl 150 mg	Amicus Therapeutics	NULL	Active, not recruiting	12 Years	17 Years	All	15	Phase 3	United States;United Kingdom;Spain
3	NCT03500094 (ClinicalTrials.gov)	October 11, 2018	9/4/2018	Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)	An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 12 to <18 Years) With Fabry Disease and Amenable GLA Variants	Fabry Disease	Drug: migalastat HCl 150 mg	Amicus Therapeutics	NULL	Completed	12 Years	17 Years	All	22	Phase 3	United States;Spain;United Kingdom
4	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 Hydrochloride Monotherapy in Subjects With Fabry Disease	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;Spain;Turkey;United Kingdom