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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: 56
Selected: 0
NCT ID | Title |
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NCT06121011 | A Global Prospective Observational Registry of Patients With Pompe Disease | ||
NCT04808505 | A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 | ||
NCT04804566 | Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients | ||
NCT04644549 | Natural History Study of Batten Disease | ||
NCT04327973 | Expanded Access for ATB200/AT2221 for the Treatment of IOPD | ||
NCT04281537 | A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy. | ||
NCT04273243 | Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer | ||
NCT04252066 | A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding | ||
NCT04138277 | A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD | ||
NCT04049760 | Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease | ||
NCT04020055 | A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease | ||
NCT03911505 | ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD | ||
NCT03865836 | Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease | ||
NCT03770572 | Gene Therapy for Children With CLN3 Batten Disease | ||
NCT03729362 | A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease | ||
NCT03500094 | Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) | ||
NCT03347253 | STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT | ||
NCT03158662 | Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa | ||
NCT02725580 | Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease | ||
NCT02675465 | First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 | ||
NCT02194985 | Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease | ||
NCT02082327 | A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers | ||
NCT01853852 | A 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 | ||
NCT01730482 | A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014) | ||
NCT01730469 | Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function | ||
NCT01489995 | Migalastat Food Effect Study | ||
NCT01476163 | Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease | ||
NCT01458119 | Open-Label Phase 3 Long-Term Safety Study of Migalastat | ||
NCT01380743 | Drug-drug Interaction Study | ||
NCT01218659 | Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease | ||
NCT01196871 | Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease | ||
NCT00925301 | Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease | ||
NCT00875160 | A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101 | ||
NCT00813865 | A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients | ||
NCT00688597 | Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease | ||
NCT00526071 | Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study | ||
NCT00515398 | A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease | ||
NCT00446550 | A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease | ||
NCT00433147 | A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy | ||
NCT00351156 | Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease | ||
NCT00304512 | A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease | ||
NCT00283959 | A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease | ||
NCT00283933 | A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease | ||
NCT00214500 | A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease | ||
NCT04602364 | French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat | ||
NCT04043273 | Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences | ||
NCT03135197 | German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl. | ||
NCT02670330 | Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa | ||
NCT02384460 | ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa | ||
NCT02363153 | Diet and Exercise in Pompe Disease |