28. Systemic amyloidosis Clinical trials / Disease details
Clinical trials : 261 / Drugs : 276 - (DrugBank : 81) / Drug target genes : 68 - Drug target pathways : 178
| 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 | EUCTR2019-001698-10-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 | ||
| 2 | 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 | ||
| 3 | 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 | ||
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Portugal;Taiwan;Estonia;Greece;Spain;Ireland;Chile;Israel;Colombia;Switzerland;Italy;France;Malaysia;Cyprus;Australia;Netherlands;Latvia;China;Korea, Democratic People's Republic of;Turkey;Austria;United Kingdom;Mexico;Canada;Argentina;Brazil;Belgium;Germany;New Zealand;Japan | ||
| 10 | EUCTR2017-005115-13-PT (EUCTR) | 23/04/2018 | 06/03/2018 | A Non-Interventional Study to Monitor the Survival Status ofPatients that Discontinued from ISIS 420915-CS2 orISIS 420915-CS3 | A Non-Interventional Study to Monitor the Survival Status ofPatients that Discontinued from ISIS 420915-CS2 orISIS 420915-CS3 | Familial 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: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915 Other descriptive name: Inotersen | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 164 | Phase 3 | France;United States;Portugal;Argentina;Brazil;Spain;Germany;New Zealand;Italy;United Kingdom | ||
| 11 | NCT01737398 (ClinicalTrials.gov) | March 15, 2013 | 27/11/2012 | Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy | A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (NEURO-TTR Study) | FAP;Familial Amyloid Polyneuropathy;TTR;Transthyretin;Amyloidosis | Drug: Inotersen;Drug: Placebo | Ionis Pharmaceuticals, Inc. | NULL | Completed | 18 Years | 82 Years | All | 173 | Phase 2/Phase 3 | United States;Argentina;Brazil;France;Germany;Italy;New Zealand;Portugal;Spain;United Kingdom;Bulgaria |
| 12 | EUCTR2017-005115-13-FR (EUCTR) | 27/02/2018 | A Non-Interventional Study to Monitor the Survival Status ofPatients that Discontinued from ISIS 420915-CS2 orISIS 420915-CS3 | A Non-Interventional Study to Monitor the Survival Status ofPatients that Discontinued from ISIS 420915-CS2 orISIS 420915-CS3 | Familial 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: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915 Other descriptive name: Inotersen | Ionis Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 164 | Phase 3 | Portugal;United States;France;Argentina;Brazil;Spain;Germany;New Zealand;Italy;United Kingdom | |||
| 13 | EUCTR2017-005115-13-GB (EUCTR) | 06/03/2018 | A Non-Interventional Study to Monitor the Survival Status of Patients that Discontinued from ISIS 420915-CS2 or ISIS 420915-CS3 | A Non-Interventional Study to Monitor the Survival Status of Patients that Discontinued from ISIS 420915-CS2 or ISIS 420915-CS3 | Familial 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: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 INN or Proposed INN: ISIS 420915 Other descriptive name: Inotersen | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 164 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Portugal;France;United States;Argentina;Brazil;Spain;Germany;New Zealand;Italy;United Kingdom |