234. ペルオキシソーム病（副腎白質ジストロフィーを除く。）
［臨床試験数：38，薬物数：37（DrugBank：12），標的遺伝子数：12，標的パスウェイ数：42］
Searched query = "Peroxisomal disease (except Adrenoleukodystrophy)", "Peroxisomal disease", "Peroxisomal disorder", "Peroxisome biogenesis disorder", "PEX gene disord ... show all
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 | EUCTR2019-001346-17-GB (EUCTR) | 22/01/2020 | 23/07/2019 | A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1 | ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: Lumasiran | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 16 | Phase 3 | United Arab Emirates;United States;Lebanon;Turkey;Israel;United Kingdom;Switzerland;India;Egypt;France;Jordan;Belgium;Australia;Netherlands;Germany | ||
| 2 | EUCTR2019-001346-17-NL (EUCTR) | 05/12/2019 | 22/08/2019 | A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1 | ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: LUMASIRAN | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 16 | Phase 3 | United States;United Arab Emirates;Lebanon;Turkey;Israel;Switzerland;United Kingdom;India;Egypt;France;Jordan;Belgium;Australia;Netherlands;Germany | ||
| 3 | EUCTR2019-001346-17-BE (EUCTR) | 07/10/2019 | 21/08/2019 | A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1 | ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: LUMASIRAN | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 16 | Phase 3 | United Arab Emirates;United States;Lebanon;Turkey;Israel;Switzerland;United Kingdom;India;Egypt;France;Jordan;Belgium;Australia;Netherlands;Germany | ||
| 4 | EUCTR2018-004014-17-DE (EUCTR) | 23/04/2019 | 27/12/2018 | A Study of an Investigational Drug, Lumasiran (ALN-GO1) in Infants and Young Children with Primary Hyperoxaluria Type 1 | ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: LUMASIRAN | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 3 | United States;France;Israel;Germany;United Kingdom | ||
| 5 | EUCTR2018-001981-40-NL (EUCTR) | 21/03/2019 | 21/01/2019 | A Phase 3 Study of an Investigational Drug, Lumasiran (ALN-GO1) with an Extended Dosing Period in Children and Adults with Primary Hyperoxaluria Type 1 Disease | ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study with an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: lumasiran | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | France;United States;United Arab Emirates;Jordan;Israel;Germany;Netherlands;United Kingdom;Japan;Switzerland | ||
| 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 | EUCTR2018-004014-17-GB (EUCTR) | 01/03/2019 | 19/12/2018 | A Study of an Investigational Drug, Lumasiran (ALN-GO1) in Infants and Young Children with Primary Hyperoxaluria Type 1 | ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 3 | France;United States;Israel;Germany;United Kingdom | |||
| 7 | EUCTR2018-004014-17-FR (EUCTR) | 19/02/2019 | 26/12/2018 | A Study of an Investigational Drug, Lumasiran (ALN-GO1) in Infants and Young Children with Primary Hyperoxaluria Type 1 | ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 8 | Phase 3 | United States;France;Israel;Germany;United Kingdom | |||
| 8 | EUCTR2018-001981-40-DE (EUCTR) | 17/12/2018 | 11/09/2018 | A Phase 3 Study of an Investigational Drug, Lumasiran (ALN-GO1) with an Extended Dosing Period in Children and Adults with Primary Hyperoxaluria Type 1 Disease | ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study with an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: lumasiran | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | United States;United Arab Emirates;France;Jordan;Israel;Netherlands;Germany;United Kingdom;Switzerland | ||
| 9 | EUCTR2018-001981-40-GB (EUCTR) | 20/11/2018 | 07/08/2018 | A Phase 3 Study of an Investigational Drug, Lumasiran (ALN-GO1) with an Extended Dosing Period in Children and Adults with Primary Hyperoxaluria Type 1 Disease | ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study with an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Phase 3 | France;United States;United Arab Emirates;Jordan;Israel;Netherlands;Germany;Switzerland;United Kingdom | |||
| 10 | EUCTR2016-003134-24-NL (EUCTR) | 21/06/2018 | 28/09/2017 | Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.0;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: ALN-GO1 Product Code: ALN-GO1 INN or Proposed INN: ALN-65585 Other descriptive name: ALN-65585 | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | France;United States;United Arab Emirates;Jordan;Israel;Germany;Netherlands;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 | NCT03350451 (ClinicalTrials.gov) | April 4, 2018 | 17/11/2017 | An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Patients With Primary Hyperoxaluria Type 1 | A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients With Primary Hyperoxaluria Type 1 | PH1;Primary Hyperoxaluria;RNAi Therapeutic;siRNA;AGT | Drug: Lumasiran | Alnylam Pharmaceuticals | NULL | Active, not recruiting | 6 Years | N/A | All | 20 | Phase 2 | France;Germany;Israel;Netherlands;United Kingdom |
| 12 | EUCTR2016-003134-24-DE (EUCTR) | 28/12/2017 | 11/07/2017 | Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: lumasiran Other descriptive name: ALN-65585 | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | United States;United Arab Emirates;France;Jordan;Israel;Netherlands;Germany;United Kingdom | ||
| 13 | EUCTR2016-003134-24-GB (EUCTR) | 20/12/2017 | 30/08/2017 | Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: lumasiran Other descriptive name: ALN-65585 | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | France;Israel;Netherlands;Germany;United Kingdom | ||
| 14 | EUCTR2016-003134-24-FR (EUCTR) | 09/10/2017 | 20/10/2017 | Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.0;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: ALN-GO1 Product Code: ALN-GO1 INN or Proposed INN: ALN-65585 Other descriptive name: ALN-65585 | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | United States;United Arab Emirates;France;Jordan;Israel;Netherlands;Germany;United Kingdom | ||
| 15 | EUCTR2015-004407-23-NL (EUCTR) | 06/06/2017 | 15/08/2016 | The First-in-Human Study of an Investigational Drug, ALN-GO1, in Healthy Adult Subjects and Patients with Primary Hyperoxaluria Type 1 Disease | A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 19.0;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: ALN-GO1 Product Code: ALN-GO1 INN or Proposed INN: ALN-65585 | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 1;Phase 2 | United States;France;Jordan;Israel;Germany;Netherlands;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 |
| 16 | NCT02706886 (ClinicalTrials.gov) | March 8, 2016 | 3/3/2016 | Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1 | A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients With Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) | Drug: Lumasiran;Drug: Placebo | Alnylam Pharmaceuticals | NULL | Completed | 6 Years | 64 Years | All | 52 | Phase 1;Phase 2 | France;Germany;Israel;Netherlands;United Kingdom;Jordan;United States |
| 17 | EUCTR2015-004407-23-GB (EUCTR) | 10/02/2016 | 21/12/2015 | The First-in-Human Study of an Investigational Drug, ALN-GO1, in Healthy Adult Subjects and Patients with Primary Hyperoxaluria Type 1 Disease | A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: ALN-GO1 Product Code: ALN-GO1 INN or Proposed INN: ALN-65585 | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 52 | Phase 1;Phase 2 | United States;France;Jordan;Israel;Netherlands;Germany;United Kingdom | ||
| 18 | EUCTR2019-001346-17-DE (EUCTR) | 15/07/2019 | A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1 | ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: LUMASIRAN | Alnylam Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 20 | Phase 3 | United Arab Emirates;United States;Lebanon;Turkey;Israel;Switzerland;United Kingdom;India;Egypt;France;Jordan;Belgium;Australia;Netherlands;Germany | |||
| 19 | EUCTR2015-004407-23-DE (EUCTR) | 07/03/2016 | The First-in-Human Study of an Investigational Drug, ALN-GO1, in Healthy Adult Subjects and Patients with Primary Hyperoxaluria Type 1 Disease | A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 52 | Phase 1;Phase 2 | France;United States;Jordan;Israel;Netherlands;Germany;United Kingdom | ||||
| 20 | EUCTR2019-001346-17-FR (EUCTR) | 12/07/2019 | A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1 | ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: LUMASIRAN | Alnylam Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 16 | Phase 3 | United Arab Emirates;United States;Lebanon;Turkey;Israel;Switzerland;United Kingdom;India;France;Egypt;Belgium;Australia;Netherlands;Germany |