19. Lysosomal storage disease
784 clinical trials, 673 drugs (DrugBank: 101 drugs), 68 drug target genes, 184 drug target pathways
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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) | March 30, 2021 | 24/6/2019 | A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variants and Severe Renal Impairment | An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Fabry Subjects With Amenable GLA Variants and Severe Renal Impairment | Fabry Disease | Drug: migalastat HCl 150 mg | Amicus Therapeutics | NULL | Not yet recruiting | 16 Years | N/A | All | 12 | Phase 3 | United States;Belgium;France;Italy;Spain;United Kingdom |
| 2 | NCT04049760 (ClinicalTrials.gov) | October 14, 2019 | 30/7/2019 | Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease | A Long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants | Fabry Disease | Drug: migalastat HCl 150 mg | Amicus Therapeutics | NULL | Recruiting | 12 Years | 17 Years | All | 20 | Phase 3 | United States;Spain;United Kingdom |
| 3 | 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 | Active, not recruiting | 12 Years | 17 Years | All | 22 | Phase 3 | United States;Spain;United Kingdom |
| 4 | 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 |
| 5 | 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 |
| 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 |
| 6 | 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 | ||
| 7 | 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 |
| 8 | 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 |
| 9 | 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 |
| 10 | 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 |
| 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 |
| 11 | 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 |
| 12 | 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 |
| 13 | 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 |