Clinical Trials - FOLD

NCT IDTitleStatusPhasesStart DateCompletion DatePrimary Completion Date
NCT06904261A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA VariantsRECRUITINGPHASE32025-102028-122027-12
NCT04808505A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18RECRUITINGPHASE32023-07-182027-042027-04
NCT04020055A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal DiseaseRECRUITINGPHASE32022-10-312026-12-312026-12-31
NCT03911505ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDRECRUITINGPHASE32020-02-132026-062026-06
NCT04138277A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPDCOMPLETEDPHASE32019-12-182024-12-312024-12-31
NCT04049760Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry DiseaseCOMPLETEDPHASE32019-10-112024-11-292024-11-29
NCT03729362A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe DiseaseCOMPLETEDPHASE32018-12-042021-01-152020-12-15
NCT03500094Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)COMPLETEDPHASE32018-09-272021-02-062021-02-02
NCT02675465First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221COMPLETEDPHASE1, PHASE22016-042024-08-202024-07-15
NCT02725580Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) DiseaseCOMPLETEDPHASE1, PHASE22016-03-092021-10-272021-10-27
NCT02194985Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry DiseaseCOMPLETEDPHASE32015-03-142019-10-232019-10-23
NCT02082327A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy VolunteersCOMPLETEDPHASE12014-032014-062014-06
NCT01380743Drug-drug Interaction StudyCOMPLETEDPHASE22011-10-312013-01-042013-01-04
NCT01458119Open-Label Phase 3 Long-Term Safety Study of MigalastatTERMINATEDPHASE32011-10-142016-02-172016-02-17
NCT01489995Migalastat Food Effect StudyCOMPLETEDPHASE12011-102011-122011-12
NCT01218659Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry DiseaseCOMPLETEDPHASE32011-09-082015-05-282014-05-27
NCT01853852A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese SubjectsCOMPLETEDPHASE12011-092011-122011-12
NCT01730469Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal FunctionCOMPLETEDPHASE12011-082012-042012-04
NCT01730482A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)COMPLETEDPHASE12011-082011-092011-09
NCT01196871Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry DiseaseCOMPLETEDPHASE22011-02-022012-10-092012-10-09
NCT00925301Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry DiseaseCOMPLETEDPHASE32009-10-232014-01-292012-06-12
NCT00813865A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher PatientsCOMPLETEDPHASE22009-05-112012-05-012012-05-01
NCT00875160A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101TERMINATEDPHASE12009-042009-072009-07
NCT00688597Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe DiseaseTERMINATEDPHASE22008-12-082009-12-142009-12-14
NCT00446550A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher DiseaseCOMPLETEDPHASE22008-06-112009-08-202009-08-20
NCT00526071Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 StudyTERMINATEDPHASE22007-09-172012-09-082012-09-08
NCT00433147A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement TherapyCOMPLETEDPHASE22007-03-232008-02-192008-02-19
NCT00304512A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry DiseaseCOMPLETEDPHASE22006-09-072008-05-092008-05-09
NCT00283959A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseCOMPLETEDPHASE22006-06-272008-05-082008-05-08
NCT00283933A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseCOMPLETEDPHASE22006-05-092008-03-122008-03-12
NCT00214500A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseCOMPLETEDPHASE22006-01-022008-01-292008-01-29