MIGALASTAT ( DrugBank: Migalastat )


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63

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
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size
PhaseCountries
1NCT04020055
(ClinicalTrials.gov)
October 31, 202224/6/2019A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal DiseaseAn Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects With Fabry Disease and Amenable GLA Variants and Severe Renal Impairment or End-Stage Renal Disease Treated With HemodialysisFabry DiseaseDrug: migalastat HCl 150 mgAmicus TherapeuticsNULLRecruiting18 YearsN/AAll14Phase 3United States;Australia;France;Japan;Spain;Belgium;Italy;United Kingdom
2JPRN-jRCT2071220022
14/07/202225/06/2022A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy Fabry's diseaseDrug: Venglustat (GZ402671)
Pharmaceutical form: Tablet, Route of administration: Oral
Drug: Agalsidase alfa
Pharmaceutical form: Concentrate for solution for infusion, Route of administration: IV infusion
Drug: Agalsidase beta (GZ419828)
Pharmaceutical form: Powder for concentrate for solution for infusion, Route of administration: IV infusion
Drug: Migalastat
Pharmaceutical form: Hard capsules, Route of administration: Oral
Tanaka TomoyukiNULLRecruiting>= 18age old<= 65age oldBoth90Phase 3China;Italy;Republic of Korea;Japan
3EUCTR2021-002320-20-NL
(EUCTR)
21/06/202216/03/2022A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - CARAT Fabry’s disease
MedDRA version: 24.1;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: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
Other descriptive name: Galafold
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of
4EUCTR2021-002320-20-DK
(EUCTR)
05/05/202212/11/2021A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - CARAT Fabry’s disease
MedDRA version: 24.1;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: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Germany;Netherlands;Norway;China;Japan;Korea, Republic of
5NCT05280548
(ClinicalTrials.gov)
May 3, 20224/3/2022A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry DiseaseA Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular HypertrophyFabry's DiseaseDrug: Venglustat (GZ402671);Drug: Agalsidase alfa;Drug: Agalsidase beta (GZ419828);Drug: MigalastatGenzyme, a Sanofi CompanyNULLRecruiting18 Years65 YearsAll90Phase 3China;Czechia;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Spain;Taiwan;Turkey
6EUCTR2021-002320-20-PL
(EUCTR)
12/04/202211/01/2022A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - CARAT Fabry’s disease
MedDRA version: 24.1;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: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of
7EUCTR2021-002320-20-NO
(EUCTR)
11/04/202212/10/2021A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy Fabry’s disease
MedDRA version: 24.1;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: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Netherlands;Norway;Germany;China;Japan;Korea, Republic of
8EUCTR2021-002320-20-IT
(EUCTR)
14/01/202222/10/2021A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - . Fabry's disease
MedDRA version: 24.1;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]
Trade Name: Fabrazyme
Product Name: .
Product Code: [.]
INN or Proposed INN: AGALSIDASI BETA
Trade Name: Galafold
Product Name: .
Product Code: [.]
INN or Proposed INN: Migalastat
Trade Name: Replagal
Product Name: .
Product Code: [.]
INN or Proposed INN: AGALSIDASI ALFA
Product Name: Venglustat
Product Code: [SAR402671, GZ402671 o GZ/SAR402671]
INN or Proposed INN: venglustat malato
Other descriptive name: GZ/SAR402671
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENTNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Norway;Germany;Netherlands;China;Japan;Korea, Republic of
9EUCTR2019-000222-21-GB
(EUCTR)
25/08/202012/06/2020Migalastat Pediatric Long Term Extension StudyA 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 and with amenable GLA variants
MedDRA version: 20.0;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
Product Code: AT1001
INN or Proposed INN: migalastat
Other descriptive name: MIGALASTAT HYDROCHLORIDE
Amicus Therapeutics, UK LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 3United States;United Kingdom
10EUCTR2017-000146-21-IT
(EUCTR)
30/01/202023/10/2020AN 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 VARIANTSAN 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 - ASPIRE Fabry disease and with amenable GLA variants
MedDRA version: 20.0;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]
Trade Name: Galafold
Product Name: Migalastat
Product Code: [AT1001]
INN or Proposed INN: migalastat
Other descriptive name: MIGALASTAT HYDROCHLORIDE
Amicus Therapeutics UK, LtdNULLNAFemale: yes
Male: yes
20Phase 3United States;Spain;Australia;Germany;United Kingdom;Italy
11EUCTR2017-000146-21-GB
(EUCTR)
29/08/201922/03/2019ASPIRE Paediatric Fabry StudyAN 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 and with amenable GLA variants
MedDRA version: 20.0;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]
Trade Name: Galafold
Product Name: Migalastat
Product Code: AT1001
INN or Proposed INN: migalastat
Other descriptive name: MIGALASTAT HYDROCHLORIDE
Amicus Therapeutics, UK LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 3United States;Spain;Australia;Germany;Italy;United Kingdom
12NCT03949920
(ClinicalTrials.gov)
May 16, 201918/4/2019A Study of Migalastat in Fabry DiseaseA Prospective Observational Study Investigating the Impact of Migalastat on Cardiovascular Structure and Function in Fabry DiseaseFabry DiseaseDrug: MigalastatManchester University NHS Foundation TrustNorthern Care Alliance NHS Foundation TrustActive, not recruiting16 YearsN/AAll21United Kingdom
13EUCTR2017-000146-21-ES
(EUCTR)
09/05/201911/04/2019ASPIRE Pediatric Fabry StudyAN 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 and with amenable GLA variants
MedDRA version: 20.0;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]
Trade Name: Galafold
Product Name: Migalastat
Product Code: AT1001
INN or Proposed INN: migalastat
Other descriptive name: MIGALASTAT HYDROCHLORIDE
Amicus Therapeutics, UK LtdNULLNot RecruitingFemale: yes
Male: yes
20Phase 3United States;Spain;Australia;Germany;United Kingdom;Italy
14NCT03500094
(ClinicalTrials.gov)
October 11, 20189/4/2018Safety, 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 VariantsFabry DiseaseDrug: migalastat HCl 150 mgAmicus TherapeuticsNULLCompleted12 Years17 YearsAll22Phase 3United States;Spain;United Kingdom
15EUCTR2014-002701-38-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
16EUCTR2014-002701-38-ES
(EUCTR)
12/03/201516/01/2015A 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: 17.1;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
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Japan
17EUCTR2014-002701-38-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
18EUCTR2014-002701-38-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
19EUCTR2014-002701-38-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
20NCT02194985
(ClinicalTrials.gov)
October 201417/7/2014Open-Label Extension Study of the Long-Term Effects of Migalastat HCL 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 DiseaseFabry DiseaseDrug: migalastat HCl 150 mgAmicus TherapeuticsNULLCompleted18 YearsN/AAll76Phase 3United States;Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom
21EUCTR2011-004800-40-AT
(EUCTR)
08/07/201415/05/2014A 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.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark
22NCT02082327
(ClinicalTrials.gov)
March 20146/3/2014A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy VolunteersA 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 ArmFabry DiseaseDrug: IV migalastat HCl;Drug: IV placebo;Drug: oral migalastat HClAmicus TherapeuticsNULLCompleted18 Years45 YearsBoth31Phase 1Netherlands
23EUCTR2011-004800-40-BE
(EUCTR)
07/10/201329/08/2013A 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.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark
24EUCTR2011-004800-40-DK
(EUCTR)
27/08/201213/07/2012A 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.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Denmark;Australia
25EUCTR2010-022709-16-GB
(EUCTR)
23/08/201221/05/2012A study to find out if an investigational new drug called AT1001 can help people with Fabry disease and if it is safe for use in combination with enzyme replacement therapy (ERT).AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS 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
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
18Phase 2Belgium;United Kingdom
26EUCTR2011-004800-40-IT
(EUCTR)
15/05/201212/03/2012A 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.NULLNot RecruitingFemale: yes
Male: yes
100Egypt;United States;Belgium;Spain;Brazil;Turkey;Austria;Denmark;Australia;United Kingdom;Italy
27EUCTR2010-022636-37-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
28EUCTR2011-004800-40-ES
(EUCTR)
13/02/201211/01/2012A 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
100Phase 3France;Egypt;United States;Belgium;Brazil;Spain;Turkey;Austria;Denmark;Australia;Italy;United Kingdom
29EUCTR2011-004800-40-GB
(EUCTR)
25/01/201225/10/2011A 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.NULLNot Recruiting Female: yes
Male: yes
100 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark
30EUCTR2010-022636-37-IT
(EUCTR)
16/12/201113/03/2012A 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 - AT1001-012A 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 - AT1001-012 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 hydrocloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrocloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Trade Name: REPLAGAL
INN or Proposed INN: Agalsidase alfa
Other descriptive name: NA
Trade Name: FABRAZYME
INN or Proposed INN: Agalsidase beta
Other descriptive name: NA
AMICUS THERAPEUTICS, INCNULLNot RecruitingFemale: yes
Male: yes
50United States;Taiwan;Greece;Turkey;Austria;Russian Federation;Italy;Switzerland;United Kingdom;Mexico;Argentina;Brazil;Belgium;Denmark;Australia;Germany;Japan
31NCT01476163
(ClinicalTrials.gov)
December 201117/11/2011Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry DiseasePhysician Initiated Expanded Access Request for Treatment Use of Migalastat Hydrochloride (AT1001), an Investigational Treatment for Individual Patients With Fabry Disease (AT1001-188)Fabry DiseaseDrug: migalastat HClAmicus TherapeuticsNULLApproved for marketing16 Years74 YearsAllUnited States;Australia
32EUCTR2010-022636-37-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
33NCT01458119
(ClinicalTrials.gov)
October 14, 201120/10/2011Open-Label Phase 3 Long-Term Safety Study of MigalastatAn Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry DiseaseFabry DiseaseDrug: migalastat hydrochlorideAmicus TherapeuticsNULLTerminated16 YearsN/AAll85Phase 3United States;Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;Austria;Japan
34NCT01489995
(ClinicalTrials.gov)
October 201120/10/2011Migalastat Food Effect StudyA Randomized, Open-Label, 5-Period Crossover Study to Evaluate the Effect of Meal Type and Timing on the Pharmacokinetics of Migalastat Hydrochloride in Healthy Volunteers.Fabry DiseaseDrug: A (migalastat);Drug: B (migalastat);Drug: C (migalastat);Drug: D (migalastat);Drug: E (migalastat)Amicus TherapeuticsNULLCompleted18 Years65 YearsBoth20Phase 1United States
35EUCTR2010-022709-16-BE
(EUCTR)
12/09/201110/08/2011A study to find out if an investigational new drug called AT1001 can help people with Fabry disease and if it is safe for use in combination with enzyme replacement therapy (ERT).AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS 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
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
24Phase 2France;Canada;Belgium;Australia;Netherlands;United Kingdom;Switzerland
36NCT01218659
(ClinicalTrials.gov)
September 8, 20116/10/2010Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in 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 ERTFabry DiseaseDrug: migalastat hydrochloride;Biological: agalsidaseAmicus TherapeuticsNULLCompleted16 Years74 YearsAll68Phase 3United States;Australia;Austria;Belgium;Brazil;Denmark;France;Italy;Japan;United Kingdom;Argentina;Germany;Greece;Poland;Switzerland;Taiwan;Turkey
37EUCTR2010-022636-37-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
38NCT01730482
(ClinicalTrials.gov)
August 20118/11/2012A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)A Phase 1 Study to Investigate the Absorption, Metabolism and Excretion of [14C] AT1001 (Migalastat Hydrochloride) Following a Single Oral Administration in Healthy Volunteers (AT1001-014)Fabry DiseaseDrug: [14C] AT1001Amicus TherapeuticsNULLCompleted30 Years55 YearsMale6Phase 1NULL
39NCT01730469
(ClinicalTrials.gov)
August 20118/11/2012Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal FunctionAn 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 DiseaseDrug: AT1001 150 mgAmicus TherapeuticsNULLCompleted18 Years75 YearsAll32Phase 1United States
40EUCTR2010-022636-37-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
41EUCTR2010-022636-37-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
42NCT01196871
(ClinicalTrials.gov)
February 2, 20117/9/2010Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry DiseaseAn Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Subjects With Fabry DiseaseFabry DiseaseDrug: Migalastat HCl;Biological: Agalsidase Beta;Biological: Agalsidase AlfaAmicus TherapeuticsNULLCompleted18 Years65 YearsMale20Phase 2United States;Australia;Belgium;Canada;Netherlands;France;United Kingdom
43EUCTR2010-022636-37-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
44EUCTR2009-013459-31-ES
(EUCTR)
30/03/201022/12/2009ESTUDIO DOBLE CIEGO, ALEATORIZADO Y CONTROLADO CON PLACEBO PARA EVALUAR LA EFICACIA, LA SEGURIDAD Y LA FARMACODINAMIA DE AT1001 EN PACIENTES CON ENFERMEDAD DE FABRY Y MUTACIONES EN GLA SENSIBLES A AT1001.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.ESTUDIO DOBLE CIEGO, ALEATORIZADO Y CONTROLADO CON PLACEBO PARA EVALUAR LA EFICACIA, LA SEGURIDAD Y LA FARMACODINAMIA DE AT1001 EN PACIENTES CON ENFERMEDAD DE FABRY Y MUTACIONES EN GLA SENSIBLES A AT1001.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. Enfermedad de Fabry.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
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
60Phase 3France;Belgium;Spain;Denmark;Netherlands;Germany;Italy;United Kingdom
45EUCTR2009-013459-31-DK
(EUCTR)
11/03/201027/01/2010A CLINICAL STUDY THAT WILL MEASURE THE EFFECTIVENESS AND SAFETY OF A NEW INVESTIGATIONAL MEDICATION FOR THE TREATMENT OF PATIENTS WITH FABRY DISEASEA 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.NULLNot RecruitingFemale: yes
Male: yes
60United States;Spain;Turkey;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Denmark;Australia;South Africa;Germany;Netherlands
46EUCTR2009-013459-31-BE
(EUCTR)
08/03/201016/12/2009A 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 DiseaseA 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.NULLNot RecruitingFemale: yes
Male: yes
60Phase 3France;Spain;Belgium;Denmark;Netherlands;Germany;Italy;United Kingdom
47EUCTR2009-013459-31-NL
(EUCTR)
03/03/201020/11/2009A 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 DiseaseA 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.NULLNot RecruitingFemale: yes
Male: yes
60Phase 3France;Spain;Belgium;Denmark;Germany;Netherlands;Italy;United Kingdom
48EUCTR2009-013459-31-IT
(EUCTR)
02/02/201015/01/2010A 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 - NDA 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 - ND Fabry disease
MedDRA version: 9.1;Level: LLT;Classification code 10016016
INN or Proposed INN: migalastat hydrochlorideAMICUS THERAPEUTICS, INCNULLNot RecruitingFemale: yes
Male: yes
60France;Spain;Belgium;Denmark;Netherlands;Germany;United Kingdom;Italy
49EUCTR2009-013459-31-GB
(EUCTR)
10/12/200911/09/2009A CLINICAL STUDY THAT WILL MEASURE THE EFFECTIVENESS AND SAFETY OF A NEW INVESTIGATIONAL MEDICATION FOR THE TREAMENT OF PATIENTS WITH FABRY DISEASEA 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.NULLNot 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): noUnited States;Spain;Turkey;Italy;United Kingdom;Egypt;France;Canada;Argentina;Brazil;Belgium;Denmark;Australia;South Africa;Germany;Netherlands
50EUCTR2009-013459-31-FR
(EUCTR)
13/11/200901/10/2009A 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 DiseaseA 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
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
60Phase 3France;Spain;Belgium;Denmark;Netherlands;Germany;Italy;United Kingdom
51NCT00925301
(ClinicalTrials.gov)
October 23, 200919/6/2009Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry DiseaseA Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Pharmacodynamics of AT1001 in Patients With Fabry Disease and AT1001-Responsive GLA MutationsFabry DiseaseDrug: migalastat hydrochloride;Drug: PlaceboAmicus TherapeuticsNULLCompleted16 Years74 YearsAll67Phase 3United States;Argentina;Australia;Brazil;Canada;Denmark;Egypt;France;Italy;Poland;Spain;Turkey;United Kingdom;Belgium;Chile;Germany;Israel;Netherlands;South Africa
52EUCTR2007-001838-13-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
53NCT00526071
(ClinicalTrials.gov)
September 17, 20075/9/2007Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 StudyOpen-label Extension Study to Evaluate the Long-term Safety, Tolerability and Pharmacodynamics of AT1001 in Patients With Fabry DiseaseFabry DiseaseDrug: migalastat HClAmicus TherapeuticsNULLTerminated18 YearsN/AAll23Phase 2United States;Australia;Brazil;France;United Kingdom
54NCT00304512
(ClinicalTrials.gov)
September 7, 200617/3/2006A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry DiseaseA Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Female Patients With Fabry DiseaseFabry DiseaseDrug: migalastat HClAmicus TherapeuticsNULLCompleted18 Years65 YearsFemale9Phase 2United States;Australia;Brazil;Canada;France;United Kingdom
55NCT00283959
(ClinicalTrials.gov)
June 27, 200627/1/2006A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseA Phase 2, Open-Label, Single Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients With Fabry DiseaseFabry DiseaseDrug: migalastat HClAmicus TherapeuticsNULLCompleted18 Years65 YearsMale4Phase 2Australia;Brazil
56NCT00283933
(ClinicalTrials.gov)
May 9, 200627/1/2006A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseA Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients With Fabry DiseaseFabry DiseaseDrug: migalastat HClAmicus TherapeuticsNULLCompleted18 Years65 YearsMale5Phase 2France;United Kingdom;Canada
57EUCTR2006-000181-36-GB
(EUCTR)
17/03/200613/02/2006A 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 - Phase 2 Study of AT1001 in Female Patients with Fabry DiseaseA 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 - Phase 2 Study of AT1001 in Female Patients with Fabry Disease Fabry disease
MedDRA version: 8.1;Level: LLT;Classification code 10016016
Product Name: 1-deoxygalactonojirimycin hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Amicus Therapeutics, Inc.NULLNot Recruiting Female: yes
Male: no
12 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom
58NCT00214500
(ClinicalTrials.gov)
January 2, 200613/9/2005A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry DiseaseA Phase 2, Open-Label, Multicenter, 12-Week Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AT1001 in Patients With Fabry DiseaseFabry DiseaseDrug: migalastat HClAmicus TherapeuticsNULLCompleted18 Years55 YearsMale9Phase 2United States
59EUCTR2005-004384-33-GB
(EUCTR)
19/12/200517/10/2005A Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients with Fabry Disease - Phase 2 study of AT1001 in Fabry DiseaseA Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients with Fabry Disease - Phase 2 study of AT1001 in Fabry Disease Fabry Disease
MedDRA version: 8.1;Level: LLT;Classification code 10016016
Product Name: 1- Deoxygalactonojirimycin
Product Code: AT1001
INN or Proposed INN: N/A
Other descriptive name: Migalastat hydrochloride
Amicus TherapeuticsNULLNot Recruiting Female: no
Male: yes
8 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom
60EUCTR2009-013459-31-DE
(EUCTR)
03/11/2009A 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 DiseaseA 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.NULLNot RecruitingFemale: yes
Male: yes
60France;Spain;Belgium;Denmark;Netherlands;Germany;Italy;United Kingdom
61EUCTR2017-000146-21-DE
(EUCTR)
26/03/2019AN 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 VARIANTSAN 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 and with amenable GLA variants
MedDRA version: 20.0;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]
Trade Name: Galafold
Product Name: Migalastat
Product Code: AT1001
INN or Proposed INN: migalastat
Other descriptive name: MIGALASTAT HYDROCHLORIDE
Amicus Therapeutics, UK LtdNULLNAFemale: yes
Male: yes
20Phase 3United States;Spain;Australia;Germany;United Kingdom;Italy
62EUCTR2019-000065-20-NO
(EUCTR)
31/10/2019To assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme®) versus continuing on migalastat (Galafold®) in male patients with classic Fabry diseaseA randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme®) versus continuing on migalastat (Galafold®) in male patients with classic Fabry disease - BCLEAR2 Fabry disease
MedDRA version: 20.0;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]
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Trade Name: Galafold
INN or Proposed INN: MIGALASTAT
Other descriptive name: Galafold
Sanofi Aventis Groupe (SAG)NULLNot RecruitingFemale: no
Male: yes
35Phase 4United States;France;Canada;Norway
63EUCTR2021-002320-20-ES
(EUCTR)
19/11/2021A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy Fabry’s disease
MedDRA version: 24.1;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: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
Other descriptive name: Migalastat
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of