AT1001
( DrugBank: - / KEGG DRUG: - )
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 | 48 |
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
Showing 1 to 10 of 48 trials
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2019-000222-21-GB (EUCTR) | 25/08/202020200825 | 12/06/202020200612 | 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 A LONG-TERM, OPEN-LABEL STUDY TO EVALUATE THE SAFETY, PHARMACODYNAMICS, AND EFFICACY OF MIGALASTAT I ... | 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] Fabry disease and with amenable GLA variants MedDRA version: 20.0;Level: PT;Classification code 1001 ... | Product Name: Migalastat Product Code: AT1001 INN or Proposed INN: migalastat Other descriptive name: MIGALASTAT HYDROCHLORIDE Product Name: Migalastat Product Code: AT1001 INN or Proposed INN: migalastat Other descriptive name: M ... | Amicus Therapeutics, UK Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 3 | United States;United Kingdom | ||
| 2 | EUCTR2017-000146-21-IT (EUCTR) | 30/01/202020200130 | 23/10/202020201023 | 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 TREA ... | 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 AN OPEN-LABEL STUDY OF THE SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF 12-MONTH TREA ... | 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] Fabry disease and with amenable GLA variants MedDRA version: 20.0;Level: PT;Classification code 1001 ... | Trade Name: Galafold Product Name: Migalastat Product Code: [AT1001] INN or Proposed INN: migalastat Other descriptive name: MIGALASTAT HYDROCHLORIDE Trade Name: Galafold Product Name: Migalastat Product Code: [AT1001] INN or Proposed INN: migalastat Oth ... | Amicus Therapeutics UK, Ltd | NULL | NA | Female: yes Male: yes | 20 | Phase 3 | United States;Spain;Australia;Germany;United Kingdom;Italy | ||
| 3 | EUCTR2017-000146-21-GB (EUCTR) | 29/08/201920190829 | 22/03/201920190322 | 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 AN OPEN-LABEL STUDY OF THE SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF 12-MONTH TREA ... | 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] Fabry disease and with amenable GLA variants MedDRA version: 20.0;Level: PT;Classification code 1001 ... | Trade Name: Galafold Product Name: Migalastat Product Code: AT1001 INN or Proposed INN: migalastat Other descriptive name: MIGALASTAT HYDROCHLORIDE Trade Name: Galafold Product Name: Migalastat Product Code: AT1001 INN or Proposed INN: migalastat Other ... | 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 | ||
| 4 | EUCTR2017-000146-21-ES (EUCTR) | 09/05/201920190509 | 11/04/201920190411 | 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 AN OPEN-LABEL STUDY OF THE SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF 12-MONTH TREA ... | 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] Fabry disease and with amenable GLA variants MedDRA version: 20.0;Level: PT;Classification code 1001 ... | Trade Name: Galafold Product Name: Migalastat Product Code: AT1001 INN or Proposed INN: migalastat Other descriptive name: MIGALASTAT HYDROCHLORIDE Trade Name: Galafold Product Name: Migalastat Product Code: AT1001 INN or Proposed INN: migalastat Other ... | Amicus Therapeutics, UK Ltd | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 3 | United States;Spain;Australia;Germany;United Kingdom;Italy | ||
| 5 | EUCTR2014-002701-38-DK (EUCTR) | 15/05/201520150515 | 20/01/201520150120 | A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in Patients with Fabry Disease A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in ... | An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in subjects with Fabry Disease An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlo ... | 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] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat 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 Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat Other descr ... | 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 United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil ... | ||
| 6 | EUCTR2014-002701-38-ES (EUCTR) | 12/03/201520150312 | 16/01/201520150116 | A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in Patients with Fabry Disease A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in ... | An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in subjects with Fabry Disease An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlo ... | 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] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat Other descriptive name: MIGALASTAT HYDROCHLORIDE Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat Other descr ... | 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 United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil ... | ||
| 7 | EUCTR2014-002701-38-BE (EUCTR) | 09/02/201520150209 | 04/12/201420141204 | A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in Patients with Fabry Disease A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in ... | An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in subjects with Fabry Disease An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlo ... | 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] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat 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 Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat Other descr ... | 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 United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil ... | ||
| 8 | EUCTR2014-002701-38-GB (EUCTR) | 28/01/201520150128 | 09/12/201420141209 | A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in Patients with Fabry Disease A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in ... | An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in subjects with Fabry Disease An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlo ... | 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] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat 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 Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat Other descr ... | 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 United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil ... | ||
| 9 | EUCTR2014-002701-38-AT (EUCTR) | 17/12/201420141217 | 12/11/201420141112 | A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in Patients with Fabry Disease A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in ... | An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in subjects with Fabry Disease An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlo ... | 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] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat 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 Product Name: Migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat Other descr ... | 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 United States;Spain;Turkey;Austria;United Kingdom;Italy;Egypt;France;Canada;Argentina;Belgium;Brazil ... | ||
| 10 | EUCTR2011-004800-40-AT (EUCTR) | 08/07/201420140708 | 15/05/201420140515 | A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease. A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease ... | An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease. An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochlor ... | Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A. MedDRA version: 17.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) th ... | Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochloride Other descriptive name: GR181413A Product Name: Migalastat Hydrochloride Product Code: AT1001 INN or Proposed INN: Migalastat Hydrochlor ... | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil;Australia;Denmark United States;Spain;Turkey;Austria;Italy;United Kingdom;Egypt;France;Canada;Argentina;Belgium;Brazil ... |