28. 全身性アミロイドーシス Systemic amyloidosis Clinical trials / Disease details
臨床試験数 : 267 / 薬物数 : 241 - (DrugBank : 77) / 標的遺伝子数 : 68 - 標的パスウェイ数 : 180
| 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 | EUCTR2021-001427-40-SE (EUCTR) | 26/04/2022 | 28/01/2022 | Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: Eplontersen Other descriptive name: ION-682884 Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: Eplontersen Other descriptive name: ION-682884 Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: Eplontersen Other descriptive name: ION-682884 | Ionis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 165 | Phase 3 | Portugal;United States;Taiwan;Spain;Turkey;Italy;France;Canada;Argentina;Brazil;Cyprus;Australia;Germany;New Zealand;Sweden | ||
| 2 | EUCTR2021-001427-40-ES (EUCTR) | 12/04/2022 | 25/01/2022 | Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: Eplontersen Other descriptive name: ION-682884 Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: Eplontersen Other descriptive name: ION-682884 Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: Eplontersen Other descriptive name: ION-682884 | Ionis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 165 | Phase 3 | United States;Portugal;Taiwan;Spain;Turkey;Italy;France;Canada;Argentina;Brazil;Cyprus;Australia;Germany;New Zealand;Sweden | ||
| 3 | EUCTR2019-001698-10-NL (EUCTR) | 06/05/2021 | 19/03/2021 | Study to determine the effectiveness and safety of ION-682884 in patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy - NEURO – TTRANSFORM | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: ION-682884 Other descriptive name: ION-682884 Product Name: inotersen Product Code: ISIS 420915 INN or Proposed INN: INOTERSEN | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 140 | Phase 3 | Portugal;Taiwan;Estonia;Greece;Spain;Ireland;Israel;Chile;Switzerland;Italy;France;Malaysia;Cyprus;Australia;Netherlands;China;Korea, Democratic People's Republic of;Turkey;Austria;United Kingdom;Mexico;Canada;Argentina;Brazil;Belgium;Germany;New Zealand;Japan;Sweden | ||
| 4 | EUCTR2019-001698-10-CY (EUCTR) | 24/09/2020 | 11/09/2020 | Study to determine the effectiveness and safety of ION-682884 in patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy - NEURO – TTRANSFORM | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: ION-682884 Other descriptive name: ION-682884 Product Name: inotersen Product Code: ISIS 420915 INN or Proposed INN: INOTERSEN | Ionis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Portugal;Taiwan;Estonia;Greece;Spain;Ireland;Chile;Israel;Colombia;Switzerland;Italy;France;Malaysia;Cyprus;Australia;Latvia;Netherlands;China;Korea, Democratic People's Republic of;Turkey;Austria;United Kingdom;Mexico;Canada;Argentina;Brazil;Belgium;Germany;Japan;New Zealand;Sweden | ||
| 5 | EUCTR2019-001698-10-GR (EUCTR) | 17/07/2020 | 11/06/2020 | Study to determine the effectiveness and safety of ION-682884 in patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy - NEURO – TTRANSFORM | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: ION-682884 Other descriptive name: ION-682884 Product Name: inotersen Product Code: ISIS 420915 INN or Proposed INN: INOTERSEN | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 140 | Phase 3 | Portugal;Taiwan;Greece;Spain;Ireland;Turkey;United Kingdom;Italy;France;Mexico;Canada;Argentina;Brazil;Cyprus;Australia;Netherlands;Germany;Japan;New Zealand;Sweden | ||
| 6 | EUCTR2019-001698-10-SE (EUCTR) | 13/05/2020 | 24/02/2020 | Study to determine the effectiveness and safety of ION-682884 in patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy - NEURO – TTRANSFORM | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: Eplontersen Other descriptive name: ION-682884 Product Name: inotersen Product Code: ISIS 420915 INN or Proposed INN: INOTERSEN | Ionis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Portugal;Taiwan;Greece;Spain;Turkey;United Kingdom;Italy;France;Canada;Argentina;Brazil;Cyprus;Australia;Netherlands;Germany;New Zealand;Sweden | ||
| 7 | EUCTR2019-001698-10-DE (EUCTR) | 29/03/2020 | 24/10/2019 | Study to determine the effectiveness and safety of ION-682884 in patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy - NEURO – TTRANSFORM | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: Eplontersen Other descriptive name: ION-682884 Product Name: inotersen Product Code: ISIS 420915 INN or Proposed INN: INOTERSEN | Ionis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Portugal;Taiwan;Greece;Spain;Turkey;United Kingdom;Italy;France;Canada;Argentina;Brazil;Cyprus;Australia;Netherlands;Germany;New Zealand;Sweden | ||
| 8 | EUCTR2019-001698-10-PT (EUCTR) | 19/03/2020 | 18/10/2019 | Study to determine the effectiveness and safety of ION-682884 in patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy - NEURO – TTRANSFORM | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: ION-682884 Other descriptive name: ION-682884 Product Name: inotersen Product Code: ISIS 420915 INN or Proposed INN: INOTERSEN | Ionis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Portugal;Taiwan;Spain;Ireland;Chile;Switzerland;Italy;Malaysia;Cyprus;Australia;Netherlands;China;Korea, Democratic People's Republic of;Turkey;Austria;United Kingdom;Mexico;Argentina;Brazil;Belgium;Germany;Japan;New Zealand;Sweden;Israel;France;Estonia;Greece;Canada | ||
| 9 | EUCTR2019-001698-10-GB (EUCTR) | 06/03/2020 | 18/10/2019 | Study to determine the effectiveness and safety of ION-682884 in patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy - NEURO – TTRANSFORM | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: ION-682884 Other descriptive name: ION-682884 Product Name: inotersen Product Code: ISIS 420915 INN or Proposed INN: INOTERSEN | Ionis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Portugal;Taiwan;Estonia;Greece;Spain;Ireland;Chile;Israel;Switzerland;Italy;France;Malaysia;Cyprus;Australia;Netherlands;China;Korea, Democratic People's Republic of;Turkey;Austria;United Kingdom;Mexico;Canada;Argentina;Brazil;Belgium;Germany;Japan;New Zealand;Sweden | ||
| 10 | EUCTR2019-001698-10-IT (EUCTR) | 27/02/2020 | 03/12/2019 | Study to determine the effectiveness and safety of ION-682884 in patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy - NEURO – TTRANSFORM | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: ION-682884 Other descriptive name: ION-682884 Product Name: inotersen Product Code: ISIS 420915 INN or Proposed INN: INOTERSEN | Ionis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Portugal;Taiwan;Estonia;Greece;Spain;Ireland;Chile;Israel;Colombia;Switzerland;Italy;France;Malaysia;Cyprus;Australia;Latvia;Netherlands;China;Korea, Democratic People's Republic of;Turkey;Austria;United Kingdom;Mexico;Canada;Argentina;Brazil;Belgium;Germany;Japan;New Zealand;Sweden | ||
| 11 | EUCTR2019-001698-10-ES (EUCTR) | 14/02/2020 | 02/12/2019 | Study to determine the effectiveness and safety of ION-682884 in patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy - NEURO – TTRANSFORM | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10057949;Term: Familial amyloid polyneuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ION-682884 Product Code: ION-682884 INN or Proposed INN: ION-682884 Other descriptive name: ION-682884 Product Name: Inoserten Product Code: ISIS 420915 INN or Proposed INN: INOTERSEN | Ionis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Cyprus;Korea, Democratic People's Republic of;Malaysia;Portugal;Greece;Austria;Latvia;Netherlands;Sweden;China;Ireland;Brazil;Chile;France;Colombia;Argentina;Japan;United Kingdom;Switzerland;Spain;New Zealand;Canada;Turkey;Belgium;Taiwan;Italy;Mexico;Israel;Australia;Germany;Estonia |