Reminder capsules ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
19ライソゾーム病11

19. ライソゾーム病


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2014-002701-38-DK
(EUCTR)
15/05/201520/01/2015A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in Patients with Fabry DiseaseAn 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: 19.1;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: AT1001
INN or Proposed INN: migalastat
Other descriptive name: MIGALASTAT HYDROCHLORIDE
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: NA
Other descriptive name: Inactive Reminder Capsules
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil;Denmark;Australia;Japan
2EUCTR2014-002701-38-BE
(EUCTR)
09/02/201504/12/2014A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in Patients with Fabry DiseaseAn 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: 18.1;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: AT1001
INN or Proposed INN: migalastat
Other descriptive name: MIGALASTAT HYDROCHLORIDE
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: NA
Other descriptive name: Inactive Reminder Capsules
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Japan
3EUCTR2014-002701-38-GB
(EUCTR)
28/01/201509/12/2014A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in Patients with Fabry DiseaseAn 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: 18.1;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: AT1001
INN or Proposed INN: migalastat
Other descriptive name: MIGALASTAT HYDROCHLORIDE
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: NA
Other descriptive name: Inactive Reminder Capsules
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Japan
4EUCTR2014-002701-38-AT
(EUCTR)
17/12/201412/11/2014A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in Patients with Fabry DiseaseAn 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: 19.1;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: AT1001
INN or Proposed INN: migalastat
Other descriptive name: MIGALASTAT HYDROCHLORIDE
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: NA
Other descriptive name: Inactive Reminder Capsules
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Japan
5EUCTR2010-022636-37-DE
(EUCTR)
09/05/201210/01/2012The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry diseaseA RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT Fabry disease
MedDRA version: 15.0;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 Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
50Phase 3Hungary;Germany;Turkey;Switzerland;France;Italy;Austria;Australia;Brazil;Israel;United Kingdom;Slovakia;Russian Federation;Taiwan;Mexico;Argentina;Belgium;Denmark;Japan;United States;Greece;Poland
6EUCTR2010-022636-37-GR
(EUCTR)
02/11/201123/09/2011The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry diseaseA RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT Fabry disease
MedDRA version: 14.0;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 Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
50Phase 3United States;Taiwan;Slovakia;Greece;Turkey;Austria;Israel;Russian Federation;Italy;Switzerland;United Kingdom;France;Hungary;Mexico;Argentina;Brazil;Poland;Belgium;Denmark;Australia;Germany;Japan
7EUCTR2010-022636-37-AT
(EUCTR)
03/08/201127/07/2011The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry diseaseA RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT Fabry disease
MedDRA version: 16.0;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 Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
68Phase 3France;United States;Greece;Brazil;Belgium;Denmark;Australia;Austria;Germany;Japan;Italy;United Kingdom
8EUCTR2010-022636-37-DK
(EUCTR)
03/05/201106/04/2011The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry diseaseA RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT Fabry disease
MedDRA version: 16.1;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 Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
68Phase 3United States;France;Greece;Belgium;Brazil;Austria;Australia;Denmark;Germany;United Kingdom;Japan;Italy
9EUCTR2010-022636-37-GB
(EUCTR)
15/04/201122/12/2010The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry diseaseA RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT Fabry disease
MedDRA version: 16.1;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 Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
68Phase 3United States;France;Greece;Belgium;Brazil;Denmark;Austria;Australia;Germany;Japan;Italy;United Kingdom
10EUCTR2010-022636-37-BE
(EUCTR)
18/01/201116/12/2010The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry diseaseA RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT Fabry disease
MedDRA version: 16.1;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 Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
68Phase 3United States;France;Greece;Brazil;Belgium;Denmark;Austria;Australia;Germany;United Kingdom;Japan;Italy
11EUCTR2007-001838-13-GB
(EUCTR)
05/05/200916/05/2007Long-term safety study of AT1001 in people with Fabry diseaseOpen Label Extension Study to Evaluate the Long-term Safety Tolerability and Pharmacodynamics of AT1001 in Patients with Fabry Disease - Extension Study of AT1001 in 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Product Name: Reminder capsules
INN or Proposed INN: Not applicable
Amicus Therapeutics, Inc.NULLNot Recruiting Female: yes
Male: yes
30 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): Therapeutic use (Phase 4):United States;Brazil;Australia;United Kingdom