28. Systemic amyloidosis Clinical trials / Disease details
Clinical trials : 267 / Drugs : 241 - (DrugBank : 77) / Drug target genes : 68 - Drug target pathways : 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-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 | ||
| 4 | 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 |