Clinical Studies Sponsored by Amicus Therapeutics | 35 results

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

Name	NCT Number	Health Conditions in Trial	Clinical Trial Start Date	Study Completion Date	Clinical Trial Study Type
‌ A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)	NCT01730482	Fabry disease	2011	2011	Interventional
‌ 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	NCT01853852	Fabry disease	2011	2011	Interventional
‌ Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa	NCT03158662	‌ Epidermolysis bullosa	April 2, 2017	June 30, 2017	Observational
‌ Natural History Study of Batten Disease	NCT04644549	‌ Batten disease	April 27, 2021	March 11, 2022	Observational
‌ A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease	NCT00515398	Glycogen storage disease type II	2007	2008	Observational
‌ STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT	NCT03347253		December 8, 2017	November 30, 2018	Observational
‌ Migalastat Food Effect Study	NCT01489995	Fabry disease	2011	2011	Interventional
‌ Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease	NCT01476163	Fabry disease
‌ Expanded Access for ATB200/AT2221 for the Treatment of IOPD	NCT04327973
‌ Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease	NCT01196871	Fabry disease	February 2, 2011	October 9, 2012	Interventional
‌ A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding	NCT04252066	Fabry disease	April 17, 2020	2030	Observational
‌ Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer	NCT04273243	‌ Batten disease CLN6	January 24, 2020	2024	Observational
‌ A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease	NCT03729362		December 4, 2018	January 15, 2021	Interventional
‌ A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease	NCT00446550		June 11, 2008	August 20, 2009	Interventional
‌ Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)	NCT03500094	Fabry disease	September 27, 2018	February 6, 2021	Interventional
‌ Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease	NCT01218659	Fabry disease	September 8, 2011	May 28, 2015	Interventional
‌ Drug-drug Interaction Study	NCT01380743		October 31, 2011	January 4, 2013	Interventional
‌ A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy	NCT04281537	Fabry disease	March 1, 2020	May 18, 2022	Observational
‌ ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD	NCT03911505		February 13, 2020	2026	Interventional
‌ A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy	NCT00433147		March 23, 2007	February 19, 2008	Interventional
‌ A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease	NCT00304512	Fabry disease	September 7, 2006	May 9, 2008	Interventional
‌ A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients	NCT00813865		May 11, 2009	May 1, 2012	Interventional
‌ A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease	NCT00283933	Fabry disease	May 9, 2006	March 12, 2008	Interventional
‌ A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease	NCT00214500	Fabry disease	January 2, 2006	January 29, 2008	Interventional
‌ Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function	NCT01730469	Fabry disease	2011	2012	Interventional