19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04020055 (ClinicalTrials.gov) | October 31, 2022 | 24/6/2019 | A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease | An 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 Hemodialysis | Fabry Disease | Drug: migalastat HCl 150 mg | Amicus Therapeutics | NULL | Recruiting | 18 Years | N/A | All | 14 | Phase 3 | United States;Australia;France;Japan;Spain;Belgium;Italy;United Kingdom |
| 2 | NCT03500094 (ClinicalTrials.gov) | October 11, 2018 | 9/4/2018 | Safety, 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 Variants | Fabry Disease | Drug: migalastat HCl 150 mg | Amicus Therapeutics | NULL | Completed | 12 Years | 17 Years | All | 22 | Phase 3 | United States;Spain;United Kingdom |
| 3 | NCT02194985 (ClinicalTrials.gov) | October 2014 | 17/7/2014 | Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease | An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease | Fabry Disease | Drug: migalastat HCl 150 mg | Amicus Therapeutics | NULL | Completed | 18 Years | N/A | All | 76 | Phase 3 | United States;Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom |
| 4 | NCT02082327 (ClinicalTrials.gov) | March 2014 | 6/3/2014 | A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers | A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Dose Escalation Trial to Evaluate the Safety, Tolerability and Pharmacokinetics of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers With an Open-Label, Randomized, Two-Way Crossover Arm | Fabry Disease | Drug: IV migalastat HCl;Drug: IV placebo;Drug: oral migalastat HCl | Amicus Therapeutics | NULL | Completed | 18 Years | 45 Years | Both | 31 | Phase 1 | Netherlands |
| 5 | NCT01476163 (ClinicalTrials.gov) | December 2011 | 17/11/2011 | Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease | Physician Initiated Expanded Access Request for Treatment Use of Migalastat Hydrochloride (AT1001), an Investigational Treatment for Individual Patients With Fabry Disease (AT1001-188) | Fabry Disease | Drug: migalastat HCl | Amicus Therapeutics | NULL | Approved for marketing | 16 Years | 74 Years | All | United States;Australia | ||
| 6 | NCT01730469 (ClinicalTrials.gov) | August 2011 | 8/11/2012 | Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function | An Open-Label Study to Determine the Safety and Pharmacokinetics of AT1001 in Subjects With Impaired Renal Function and Healthy Subjects With Normal Renal Function (AT1001-015) | Fabry Disease | Drug: AT1001 150 mg | Amicus Therapeutics | NULL | Completed | 18 Years | 75 Years | All | 32 | Phase 1 | United States |
| 7 | NCT01196871 (ClinicalTrials.gov) | February 2, 2011 | 7/9/2010 | Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease | An Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Subjects With Fabry Disease | Fabry Disease | Drug: Migalastat HCl;Biological: Agalsidase Beta;Biological: Agalsidase Alfa | Amicus Therapeutics | NULL | Completed | 18 Years | 65 Years | Male | 20 | Phase 2 | United States;Australia;Belgium;Canada;Netherlands;France;United Kingdom |
| 8 | NCT00526071 (ClinicalTrials.gov) | September 17, 2007 | 5/9/2007 | Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study | Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Pharmacodynamics of AT1001 in Patients With Fabry Disease | Fabry Disease | Drug: migalastat HCl | Amicus Therapeutics | NULL | Terminated | 18 Years | N/A | All | 23 | Phase 2 | United States;Australia;Brazil;France;United Kingdom |
| 9 | NCT00304512 (ClinicalTrials.gov) | September 7, 2006 | 17/3/2006 | A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease | A Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Female Patients With Fabry Disease | Fabry Disease | Drug: migalastat HCl | Amicus Therapeutics | NULL | Completed | 18 Years | 65 Years | Female | 9 | Phase 2 | United States;Australia;Brazil;Canada;France;United Kingdom |
| 10 | NCT00283959 (ClinicalTrials.gov) | June 27, 2006 | 27/1/2006 | A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease | A Phase 2, Open-Label, Single Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients With Fabry Disease | Fabry Disease | Drug: migalastat HCl | Amicus Therapeutics | NULL | Completed | 18 Years | 65 Years | Male | 4 | Phase 2 | Australia;Brazil |
| 11 | NCT00283933 (ClinicalTrials.gov) | May 9, 2006 | 27/1/2006 | A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease | A Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients With Fabry Disease | Fabry Disease | Drug: migalastat HCl | Amicus Therapeutics | NULL | Completed | 18 Years | 65 Years | Male | 5 | Phase 2 | France;United Kingdom;Canada |
| 12 | NCT00214500 (ClinicalTrials.gov) | January 2, 2006 | 13/9/2005 | A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease | A Phase 2, Open-Label, Multicenter, 12-Week Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AT1001 in Patients With Fabry Disease | Fabry Disease | Drug: migalastat HCl | Amicus Therapeutics | NULL | Completed | 18 Years | 55 Years | Male | 9 | Phase 2 | United States |