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 | EUCTR2011-004800-40-AT (EUCTR) | 08/07/2014 | 15/05/2014 | A Study using Migalastat to see the safety and usefulness of the drug 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 is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A. MedDRA version: 17.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark | ||
| 2 | EUCTR2011-004800-40-BE (EUCTR) | 07/10/2013 | 29/08/2013 | A Study using Migalastat to see the safety and usefulness of the drug 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 is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A. MedDRA version: 17.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark | ||
| 3 | EUCTR2011-004800-40-DK (EUCTR) | 27/08/2012 | 13/07/2012 | A Study using Migalastat to see the safety and usefulness of the drug 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 is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A. MedDRA version: 17.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Denmark;Australia | ||
| 4 | EUCTR2011-004800-40-IT (EUCTR) | 15/05/2012 | 12/03/2012 | A Study using Migalastat to see the safety and usefulness of the drug 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 is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a- galactosidase A. MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Migalastat Hydrochloride Product Code: GR181413 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A | GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Egypt;United States;Belgium;Spain;Brazil;Turkey;Austria;Denmark;Australia;United Kingdom;Italy | |||
| 5 | EUCTR2011-004800-40-ES (EUCTR) | 13/02/2012 | 11/01/2012 | A Study using Migalastat to see the safety and usefulness of the drug 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 is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme alfa-galactosidase A. MedDRA version: 14.1;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Migalastat Hydrochloride Product Code: GR181413 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A | GlaxoSmithKline Research & Development Limited | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | France;Egypt;United States;Belgium;Brazil;Spain;Turkey;Austria;Denmark;Australia;Italy;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 |
| 6 | NCT01853852 (ClinicalTrials.gov) | September 2011 | 10/11/2011 | 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 | 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 | Fabry Disease | Drug: GR181413A/AT1001 solution;Drug: GR181413A/AT1001 capsule;Other: Potable water;Drug: Placebo capsule | Amicus Therapeutics | NULL | Completed | 20 Years | 55 Years | Both | 14 | Phase 1 | Australia |