28. 全身性アミロイドーシス
［臨床試験数：212，薬物数：234（DrugBank：72），標的遺伝子数：54，標的パスウェイ数：170］
Searched query = "Systemic amyloidosis", "AL amyloidosis", "Immunoglobulin light chain amyloidosis", "Amyloid light-chain amyloidosis", "Familial amyloidosis", "Familial amyloid polyneuropathy", "Senile transthyretin amyloidosis", "Senile TTR amyloidosis"
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 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Portugal;Taiwan;Greece;Spain;Ireland;Turkey;United Kingdom;Italy;France;Mexico;Canada;Argentina;Brazil;Cyprus;Australia;Germany;Netherlands;New Zealand;Japan;Sweden | ||
| 2 | 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: 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 | ||
| 3 | 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: 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 | ||
| 4 | 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 | ||
| 5 | 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 | ||
| 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 | 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 | ||
| 7 | EUCTR2017-005115-13-ES (EUCTR) | 25/04/2018 | 19/02/2018 | 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 | Ionis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 164 | Phase 3 | United Kingdom;New Zealand;Italy;Portugal;France;United States;Argentina;Brazil;Spain;Germany | ||
| 8 | 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 | ||
| 9 | NCT02627820 (ClinicalTrials.gov) | January 2016 | 19/11/2015 | The Effect of an Antisense Oligonucleotide to Lower Transthyretin (TTR) Levels on the Progression of -Wild-type TTR Involving the Heart | An 18 Month Open Label Study Of The Tolerability And Efficacy Of An Antisense Oligonucleotide In Patients With Wild-Type Transthyretin Amyloid Cardiomyopathy (Senile Systemic Amyloidosis) | Amyloidosis | Drug: Isis 420915/GSK 299872 | Brigham and Women's Hospital | GlaxoSmithKline;Ionis Pharmaceuticals, Inc. | Withdrawn | 50 Years | 90 Years | Both | 0 | Phase 2 | NULL |
| 10 | EUCTR2013-004561-13-ES (EUCTR) | 28/12/2015 | 30/10/2015 | Not available | An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP) - ISIS 420915-CS3 | Familial Amyloid Polyneuropathy MedDRA version: 18.1;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 | Isis Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 195 | Portugal;France;United States;Argentina;Brazil;Spain;Bulgaria;Germany;New Zealand;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 |
| 11 | EUCTR2013-004561-13-DE (EUCTR) | 22/09/2015 | 15/06/2015 | Not available | An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP) - 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 | Ionis Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 135 | Phase 3 | Portugal;France;United States;Argentina;Brazil;Spain;Germany;New Zealand;Italy;United Kingdom | ||
| 12 | EUCTR2013-004561-13-GB (EUCTR) | 23/07/2015 | 09/02/2015 | Not available | An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP) - ISIS 420915-CS3 | Familial Amyloid Polyneuropathy MedDRA version: 19.1;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 | Ionis Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 135 | Phase 3 | Portugal;France;United States;Argentina;Brazil;Spain;Germany;Italy;United Kingdom | ||
| 13 | EUCTR2012-001831-30-BG (EUCTR) | 23/04/2015 | 23/02/2015 | A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with 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 - ISIS 420915-CS2 | Familial Amyloid Polyneuropathy MedDRA version: 18.0;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 | Isis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 195 | Phase 2;Phase 3 | Portugal;France;United States;Mexico;Argentina;Brazil;Spain;Australia;Bulgaria;Germany;Italy;United Kingdom | ||
| 14 | EUCTR2013-004561-13-PT (EUCTR) | 04/07/2014 | 07/05/2014 | Not available | An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP) - 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] | Ionis Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 135 | Phase 3 | France;United States;Portugal;Argentina;Brazil;Spain;Germany;New Zealand;Italy;United Kingdom | |||
| 15 | NCT02175004 (ClinicalTrials.gov) | June 2014 | 12/6/2014 | Open-Label Extension Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP) | An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (FAP) | FAP;Familial Amyloid Polyneuropathy;TTR;Transthyretin;Amyloidosis | Drug: IONIS-TTR Rx | Ionis Pharmaceuticals, Inc. | NULL | Active, not recruiting | 18 Years | N/A | All | 135 | Phase 3 | United States;Argentina;Brazil;France;Germany;Italy;Portugal;Spain;United Kingdom;New Zealand |
| 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 | EUCTR2012-001831-30-ES (EUCTR) | 25/04/2014 | 07/03/2014 | A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with 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 - ISIS 420915-CS2 | Familial Amyloid Polyneuropathy MedDRA version: 16.1;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 | Isis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 195 | Phase 2;Phase 3 | United States;France;Portugal;Argentina;Brazil;Spain;Bulgaria;Germany;United Kingdom;Italy | ||
| 17 | EUCTR2013-004561-13-FR (EUCTR) | 16/04/2014 | 17/06/2015 | Not available | An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP) - ISIS 420915-CS3 | Familial Amyloid Polyneuropathy MedDRA version: 18.0;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 | Isis Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 195 | Phase 3 | United States;Portugal;France;Argentina;Spain;Brazil;Germany;United Kingdom;Italy | ||
| 18 | EUCTR2012-001831-30-DE (EUCTR) | 22/08/2013 | 29/05/2013 | A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with 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 - ISIS 420915-CS2 | Familial Amyloid Polyneuropathy MedDRA version: 19.0;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] | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 135 | Phase 2;Phase 3 | Portugal;France;United States;Argentina;Brazil;Spain;Bulgaria;Germany;New Zealand;Italy;United Kingdom | |||
| 19 | EUCTR2012-001831-30-IT (EUCTR) | 04/07/2013 | 19/06/2013 | A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with 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 - ISIS 420915-CS2 | Familial Amyloid Polyneuropathy MedDRA version: 14.1;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 | Isis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 195 | Phase 2;Phase 3 | France;United States;Portugal;Spain;Brazil;Bulgaria;Germany;United Kingdom;Italy | ||
| 20 | EUCTR2012-001831-30-GB (EUCTR) | 22/04/2013 | 16/11/2012 | A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with 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 - ISIS 420915-CS2 | Familial Amyloid Polyneuropathy MedDRA version: 19.0;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 | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 135 | Phase 2;Phase 3 | Portugal;France;United States;Argentina;Brazil;Spain;Bulgaria;Germany;New Zealand;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 | 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 |
| 22 | EUCTR2012-001831-30-PT (EUCTR) | 21/02/2013 | 20/11/2012 | A controlled study to assess the effectiveness and safety of study drug, ISIS 420915, in patients with 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 - ISIS 420915-CS2 | Familial Amyloid Polyneuropathy MedDRA version: 19.1;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 | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 135 | Phase 2;Phase 3 | France;United States;Portugal;Argentina;Brazil;Spain;Bulgaria;Germany;New Zealand;Italy;United Kingdom | ||
| 23 | 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] | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 164 | Phase 3 | Portugal;France;United States;Argentina;Brazil;Spain;Germany;New Zealand;Italy;United Kingdom | ||||
| 24 | 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 |