GR181413A
( DrugBank: - / KEGG DRUG: - )
1 disease
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 19 | Lysosomal storage disease | 7 |
19. Lysosomal storage disease
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
Showing 1 to 7 of 7 trials
| 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) | 08/07/201420140708 | 15/05/201420140515 | A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease. 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. An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlor ... | 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] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochlor ... | 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 United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil ... | ||
| 2 | EUCTR2011-004800-40-BE (EUCTR) | 07/10/201320131007 | 29/08/201320130829 | A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease. 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. An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlor ... | 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] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochlor ... | 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 United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil ... | ||
| 3 | EUCTR2011-004800-40-DK (EUCTR) | 27/08/201220120827 | 13/07/201220120713 | A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease. 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. An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlor ... | 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] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochlor ... | 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 United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil ... | ||
| 4 | EUCTR2011-004800-40-IT (EUCTR) | 15/05/201220120515 | 12/03/201220120312 | A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease. 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. An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlor ... | 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] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat Hydrochloride Product Code: GR181413 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A Product Name: Migalastat Hydrochloride Product Code: GR181413 INN or Proposed INN: Migalastat Hydrochl ... | 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) | 13/02/201220120213 | 11/01/201220120111 | A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease. 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. An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlor ... | 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] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat Hydrochloride Product Code: GR181413 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A Product Name: Migalastat Hydrochloride Product Code: GR181413 INN or Proposed INN: Migalastat Hydrochl ... | 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 France;Egypt;United States;Belgium;Brazil;Spain;Turkey;Austria;Denmark;Australia;Italy;United Kingdo ... | ||
| 6 | EUCTR2011-004800-40-GB (EUCTR) | 25/01/201220120125 | 25/10/201120111025 | A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease. 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. An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlor ... | 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] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochlor ... | 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 United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil ... | ||
| 7 | NCT01853852 (ClinicalTrials.gov) | September 201120110900 | 10/11/201120111110 | 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 Stu ... | 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 Stu ... | Fabry Disease | Drug: GR181413A/AT1001 solution;Drug: GR181413A/AT1001 capsule;Other: Potable water;Drug: Placebo capsule Drug: GR181413A/AT1001 solution;Drug: GR181413A/AT1001 capsule;Other: Potable water;Drug: Placebo ca ... | Amicus Therapeutics | NULL | Completed | 20 Years | 55 Years | Both | 14 | Phase 1 | Australia |