19. Lysosomal storage disease
784 clinical trials, 673 drugs (DrugBank: 101 drugs), 68 drug target genes, 184 drug target pathways
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04020055 (ClinicalTrials.gov) | March 30, 2021 | 24/6/2019 | A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variants and Severe Renal Impairment | An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Fabry Subjects With Amenable GLA Variants and Severe Renal Impairment | Fabry Disease | Drug: migalastat HCl 150 mg | Amicus Therapeutics | NULL | Not yet recruiting | 16 Years | N/A | All | 12 | Phase 3 | United States;Belgium;France;Italy;Spain;United Kingdom |
| 2 | EUCTR2019-000222-21-GB (EUCTR) | 25/08/2020 | 12/06/2020 | Migalastat Pediatric Long Term Extension Study | A 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 Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 3 | United States;United Kingdom | ||
| 3 | EUCTR2017-000146-21-IT (EUCTR) | 30/01/2020 | 23/10/2020 | 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 VARIANTS | 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 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, Ltd | NULL | NA | Female: yes Male: yes | 20 | Phase 3 | United States;Spain;Australia;Germany;United Kingdom;Italy | ||
| 4 | NCT04049760 (ClinicalTrials.gov) | October 14, 2019 | 30/7/2019 | Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease | A 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 | Drug: migalastat HCl 150 mg | Amicus Therapeutics | NULL | Recruiting | 12 Years | 17 Years | All | 20 | Phase 3 | United States;Spain;United Kingdom |
| 5 | EUCTR2017-000146-21-GB (EUCTR) | 29/08/2019 | 22/03/2019 | ASPIRE Paediatric Fabry Study | 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 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 Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 3 | United States;Spain;Australia;Germany;Italy;United Kingdom | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | EUCTR2017-000146-21-ES (EUCTR) | 09/05/2019 | 11/04/2019 | ASPIRE Pediatric Fabry Study | 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 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] | Amicus Therapeutics, UK Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 3 | United States;Spain;Australia;Germany;Italy;United Kingdom | |||
| 7 | NCT03500094 (ClinicalTrials.gov) | October 11, 2018 | 9/4/2018 | Safety, 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 Variants | Fabry Disease | Drug: migalastat HCl 150 mg | Amicus Therapeutics | NULL | Active, not recruiting | 12 Years | 17 Years | All | 22 | Phase 3 | United States;Spain;United Kingdom |
| 8 | NCT03596398 (ClinicalTrials.gov) | January 1, 2016 | 15/6/2018 | Epidemiological Study of Fabry Disease in Taiwan Young Stroke Patients | Epidemiological Study of Fabry Disease in Taiwan Young Stroke Patients | Fabry Disease | Genetic: GLA gene | Chiayi Christian Hospital | Sanofi | Enrolling by invitation | 20 Years | 55 Years | All | 1000 | NULL | |
| 9 | EUCTR2014-002701-38-DK (EUCTR) | 15/05/2015 | 20/01/2015 | A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness 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: 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. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil;Denmark;Australia;Japan | ||
| 10 | EUCTR2014-002701-38-ES (EUCTR) | 12/03/2015 | 16/01/2015 | A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness 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: 17.1;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. | 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;Japan | ||
| 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 |
| 11 | EUCTR2014-002701-38-BE (EUCTR) | 09/02/2015 | 04/12/2014 | A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness 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: 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. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Japan | ||
| 12 | EUCTR2014-002701-38-GB (EUCTR) | 28/01/2015 | 09/12/2014 | A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness 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: 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. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Japan | ||
| 13 | EUCTR2014-002701-38-AT (EUCTR) | 17/12/2014 | 12/11/2014 | A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness 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: 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. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Japan | ||
| 14 | EUCTR2011-004800-40-AT (EUCTR) | 08/07/2014 | 15/05/2014 | A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease. | An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease. | Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A. MedDRA version: 17.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark | ||
| 15 | EUCTR2011-004800-40-BE (EUCTR) | 07/10/2013 | 29/08/2013 | A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease. | An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease. | Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A. MedDRA version: 17.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark | ||
| 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 |
| 16 | EUCTR2011-004800-40-DK (EUCTR) | 27/08/2012 | 13/07/2012 | A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease. | An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease. | Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A. MedDRA version: 17.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Denmark;Australia | ||
| 17 | EUCTR2011-004800-40-IT (EUCTR) | 15/05/2012 | 12/03/2012 | A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease. | An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease. | Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a- galactosidase A. MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Migalastat Hydrochloride Product Code: GR181413 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A | GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Egypt;United States;Belgium;Spain;Brazil;Turkey;Austria;Denmark;Australia;United Kingdom;Italy | |||
| 18 | EUCTR2010-022636-37-DE (EUCTR) | 09/05/2012 | 10/01/2012 | The 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 disease | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Hungary;Germany;Turkey;Switzerland;France;Italy;Austria;Australia;Brazil;Israel;United Kingdom;Slovakia;Russian Federation;Taiwan;Mexico;Argentina;Belgium;Denmark;Japan;United States;Greece;Poland | ||
| 19 | NCT01602601 (ClinicalTrials.gov) | April 9, 2012 | 17/5/2012 | A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723 | A Study to Test the Possibility of Cross Reaction of the Antibodies Induced by the ELAPRASE (R) to GSK2788723 ELAPRASE is a Trade Mark Owned by a Third Party | Mucopolysaccharidosis II | Drug: Idursulfase;Drug: GSK2788723 | GlaxoSmithKline | NULL | Completed | N/A | N/A | All | 10 | N/A | Japan |
| 20 | EUCTR2011-004800-40-ES (EUCTR) | 13/02/2012 | 11/01/2012 | A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease. | An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease. | Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme alfa-galactosidase A. MedDRA version: 14.1;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Migalastat Hydrochloride Product Code: GR181413 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A | GlaxoSmithKline Research & Development Limited | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | France;Egypt;United States;Belgium;Brazil;Spain;Turkey;Austria;Denmark;Australia;Italy;United Kingdom | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | EUCTR2011-004800-40-GB (EUCTR) | 25/01/2012 | 25/10/2011 | A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease. | An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease. | Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A. MedDRA version: 17.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | 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 | |||
| 22 | EUCTR2010-022636-37-IT (EUCTR) | 16/12/2011 | 13/03/2012 | A 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 | A 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, INC | NULL | Not Recruiting | Female: yes Male: yes | 50 | United States;Taiwan;Greece;Turkey;Austria;Russian Federation;Italy;Switzerland;United Kingdom;Mexico;Argentina;Brazil;Belgium;Denmark;Australia;Germany;Japan | |||
| 23 | EUCTR2010-022636-37-GR (EUCTR) | 02/11/2011 | 23/09/2011 | The 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 disease | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | United States;Taiwan;Slovakia;Greece;Turkey;Austria;Israel;Russian Federation;Italy;Switzerland;United Kingdom;France;Hungary;Mexico;Argentina;Brazil;Poland;Belgium;Denmark;Australia;Germany;Japan | ||
| 24 | NCT01218659 (ClinicalTrials.gov) | September 8, 2011 | 6/10/2010 | Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease | A 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 | Fabry Disease | Drug: migalastat hydrochloride;Biological: agalsidase | Amicus Therapeutics | NULL | Completed | 16 Years | 74 Years | All | 68 | Phase 3 | United States;Australia;Austria;Belgium;Brazil;Denmark;France;Italy;Japan;United Kingdom;Argentina;Germany;Greece;Poland;Switzerland;Taiwan;Turkey |
| 25 | EUCTR2010-022636-37-AT (EUCTR) | 03/08/2011 | 27/07/2011 | The 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 disease | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 68 | Phase 3 | France;United States;Greece;Brazil;Belgium;Denmark;Australia;Austria;Germany;Japan;Italy;United Kingdom | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | EUCTR2010-022636-37-DK (EUCTR) | 03/05/2011 | 06/04/2011 | The 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 disease | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 68 | Phase 3 | United States;France;Greece;Belgium;Brazil;Austria;Australia;Denmark;Germany;United Kingdom;Japan;Italy | ||
| 27 | EUCTR2010-022636-37-GB (EUCTR) | 15/04/2011 | 22/12/2010 | The 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 disease | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 68 | Phase 3 | United States;France;Greece;Belgium;Brazil;Denmark;Austria;Australia;Germany;Japan;Italy;United Kingdom | ||
| 28 | EUCTR2010-022636-37-BE (EUCTR) | 18/01/2011 | 16/12/2010 | The 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 disease | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 68 | Phase 3 | United States;France;Greece;Brazil;Belgium;Denmark;Austria;Australia;Germany;United Kingdom;Japan;Italy | ||
| 29 | EUCTR2009-013459-31-ES (EUCTR) | 30/03/2010 | 22/12/2009 | 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. | 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Germany;United Kingdom;Netherlands;Denmark;Belgium;France;Spain;Italy | |||
| 30 | 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 | |||
| 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 |
| 31 | 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 | ||
| 32 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Germany;United Kingdom;Netherlands;Denmark;Belgium;France;Spain;Italy | |||
| 33 | EUCTR2009-013459-31-IT (EUCTR) | 02/02/2010 | 15/01/2010 | 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 - ND | 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 - ND | Fabry disease MedDRA version: 9.1;Level: LLT;Classification code 10016016 | INN or Proposed INN: migalastat hydrochloride | AMICUS THERAPEUTICS, INC | NULL | Not Recruiting | Female: yes Male: yes | 60 | France;Spain;Belgium;Denmark;Netherlands;Germany;United Kingdom;Italy | |||
| 34 | 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] | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;Spain;Turkey;Italy;United Kingdom;Egypt;France;Canada;Argentina;Brazil;Belgium;Denmark;Australia;South Africa;Germany;Netherlands | |||
| 35 | EUCTR2009-013459-31-FR (EUCTR) | 13/11/2009 | 01/10/2009 | 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 | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Germany;United Kingdom;Netherlands;Denmark;Belgium;France;Spain;Italy | |||
| 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 |
| 36 | NCT00925301 (ClinicalTrials.gov) | October 23, 2009 | 19/6/2009 | Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) 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 | Fabry Disease | Drug: migalastat hydrochloride;Drug: Placebo | Amicus Therapeutics | NULL | Completed | 16 Years | 74 Years | All | 67 | Phase 3 | United States;Argentina;Australia;Brazil;Canada;Denmark;Egypt;France;Italy;Poland;Spain;Turkey;United Kingdom;Belgium;Chile;Germany;Israel;Netherlands;South Africa |
| 37 | EUCTR2017-000146-21-DE (EUCTR) | 26/03/2019 | 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 VARIANTS | 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 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 Ltd | NULL | NA | Female: yes Male: yes | 20 | Phase 3 | United States;Spain;Australia;Germany;United Kingdom;Italy | |||
| 38 | 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 |