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The U.S. National Library of Medicine provides a database of clinical trials worldwide. Use the fields below to search the database. Expand your results and select terms or phrases and right-click to search using Google, MalaCards, MedlinePlus, PubMed, or Wikipedia.

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Clinical Trials Search Results

Query: "AREA[SponsorSearch](Amicus Therapeutics)"
Count: 55
Selected: 0
NCT IDTitle
NCT04808505Rossella: A Study to Evaluate the Safety, PK, Efficacy, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
NCT04804566Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
NCT04644549Natural History Study of Batten Disease
NCT04327973Expanded Access for ATB200/AT2221 for the Treatment of IOPD
NCT04281537A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
NCT04273243Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
NCT04252066A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
NCT04138277A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD
NCT04049760Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
NCT04020055A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variants and Severe Renal Impairment
NCT03911505ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
NCT03865836Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
NCT03770572Gene Therapy for Children With CLN3 Batten Disease
NCT03729362PROPEL Study - A Study Comparing ATB200/AT2221 With Alglucosidase/Placebo in Adult Subjects With LOPD
NCT03500094Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
NCT03347253STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
NCT03158662Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa
NCT02725580Gene Therapy for Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease
NCT02675465First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
NCT02194985Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
NCT02082327A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers
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 Subjects
NCT01730482A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)
NCT01730469Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function
NCT01489995Migalastat Food Effect Study
NCT01476163Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
NCT01458119Open-Label Phase 3 Long-Term Safety Study of Migalastat
NCT01380743Drug-drug Interaction Study
NCT01218659Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
NCT01196871Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease
NCT00925301Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
NCT00875160A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101
NCT00813865A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
NCT00688597Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
NCT00526071Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study
NCT00515398A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
NCT00446550A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
NCT00433147A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
NCT00351156Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
NCT00304512A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
NCT00283959A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
NCT00283933A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
NCT00214500A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
NCT04602364French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat
NCT04043273Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences
NCT03135197German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl.
NCT02670330Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa
NCT02384460ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
NCT02363153Diet and Exercise in Pompe Disease
NCT02185651A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction