19. Lysosomal storage disease Clinical trials / Disease details

Clinical trials : 899 /  Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182

  
3 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT04020055
(ClinicalTrials.gov)		October 31, 202224/6/2019A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal DiseaseAn Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects With Fabry Disease and Amenable GLA Variants and Severe Renal Impairment or End-Stage Renal Disease Treated With HemodialysisFabry DiseaseDrug: migalastat HCl 150 mgAmicus TherapeuticsNULLRecruiting18 YearsN/AAll14Phase 3United States;Australia;France;Japan;Spain;Belgium;Italy;United Kingdom
2NCT03500094
(ClinicalTrials.gov)		October 11, 20189/4/2018Safety, 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 VariantsFabry DiseaseDrug: migalastat HCl 150 mgAmicus TherapeuticsNULLCompleted12 Years17 YearsAll22Phase 3United States;Spain;United Kingdom
3NCT02194985
(ClinicalTrials.gov)		October 201417/7/2014Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry DiseaseAn Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry DiseaseFabry DiseaseDrug: migalastat HCl 150 mgAmicus TherapeuticsNULLCompleted18 YearsN/AAll76Phase 3United States;Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom