Reminder Product ( DrugBank: - )
1 disease
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 19 | Lysosomal storage disease | 5 |
19. Lysosomal storage disease
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 | EUCTR2009-013459-31-DK (EUCTR) | 11/03/2010 | 27/01/2010 | A CLINICAL STUDY THAT WILL MEASURE THE EFFECTIVENESS AND SAFETY OF A NEW INVESTIGATIONAL MEDICATION FOR THE TREATMENT OF PATIENTS WITH FABRY DISEASE | A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY AND PHARMACODYNAMICS OF AT1001 IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS - Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease | Fabry Disease MedDRA version: 15.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Product INN or Proposed INN: Not applicable | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | United States;Spain;Turkey;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Denmark;Australia;South Africa;Germany;Netherlands | |||
| 2 | EUCTR2009-013459-31-BE (EUCTR) | 08/03/2010 | 16/12/2009 | A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY AND PHARMACODYNAMICS OF AT1001 IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS - Study of the Effects of Oral AT1001 in Patients with Fabry Disease | A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY AND PHARMACODYNAMICS OF AT1001 IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS - Study of the Effects of Oral AT1001 in Patients with Fabry Disease | Fabry Disease MedDRA version: 12.0;Level: LLT;Classification code 10016016;Term: Fabry's disease | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Reminder Product INN or Proposed INN: Not applicable | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;Spain;Belgium;Denmark;Netherlands;Germany;Italy;United Kingdom | ||
| 3 | EUCTR2009-013459-31-NL (EUCTR) | 03/03/2010 | 20/11/2009 | A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY AND PHARMACODYNAMICS OF AT1001 IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS - Study of the Effects of Oral AT1001 in Patients with Fabry Disease | A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY AND PHARMACODYNAMICS OF AT1001 IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS - Study of the Effects of Oral AT1001 in Patients with Fabry Disease | Fabry Disease MedDRA version: 12.0;Level: LLT;Classification code 10016016;Term: Fabry's disease | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Reminder Product INN or Proposed INN: Not applicable | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;Spain;Belgium;Denmark;Germany;Netherlands;Italy;United Kingdom | ||
| 4 | EUCTR2009-013459-31-GB (EUCTR) | 10/12/2009 | 11/09/2009 | A CLINICAL STUDY THAT WILL MEASURE THE EFFECTIVENESS AND SAFETY OF A NEW INVESTIGATIONAL MEDICATION FOR THE TREAMENT OF PATIENTS WITH FABRY DISEASE | A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY AND PHARMACODYNAMICS OF AT1001 IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS - Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease | Fabry Disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Product Product Code: Inactive Reminder Product INN or Proposed INN: Not applicable | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Italy;United Kingdom;Egypt;France;Canada;Argentina;Brazil;Belgium;Denmark;Australia;South Africa;Germany;Netherlands | ||
| 5 | EUCTR2009-013459-31-DE (EUCTR) | 03/11/2009 | A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY AND PHARMACODYNAMICS OF AT1001 IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS - Study of the Effects of Oral AT1001 in Patients with Fabry Disease | A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY AND PHARMACODYNAMICS OF AT1001 IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS - Study of the Effects of Oral AT1001 in Patients with Fabry Disease | Fabry Disease MedDRA version: 12.0;Level: LLT;Classification code 10016016;Term: Fabry's disease | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Reminder Product INN or Proposed INN: Not applicable | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | France;Spain;Belgium;Denmark;Netherlands;Germany;Italy;United Kingdom |