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 disorder", "Zellweger syndrome", "Neonatal adrenoleukodystrophy", "Rhizomelic chondrodysplasia punctata type 1", "RCDP type 1", "RCDP1", "Peroxisomal beta-oxidation enzyme deficiency", "Acyl-CoA oxidase deficiency", "AOX deficiency", "D-Bifunctional protein deficiency", "DBP deficiency", "Sterol carrier protein X deficiency", "SCPx deficiency", "2-methylacyl-CoA racemase deficiency", "Alpha-methylacyl-CoA racemase deficiency", "AMACR deficiency", "Refsum disease", "Plasmalogen biosynthesis enzyme deficiency", "Rhizomelic chondrodysplasia punctata type 2", "RCDP type 2", "RCDP2", "Rhizomelic chondrodysplasia punctata type 3", "RCDP type 3", "RCDP3", "Primary hyperoxaluria type 1", "PH1", "Acatalasemia", "Acatalasia", "Contiguous ABCD1/DXS1357E deletion syndrome", "CADDS"
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 | NCT04125472 (ClinicalTrials.gov) | January 22, 2020 | 10/10/2019 | Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1 | Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria | Drug: Lumasiran | Alnylam Pharmaceuticals | NULL | Available | N/A | N/A | All | United States;Belgium;France;French Polynesia;Germany;Italy;Switzerland;United Kingdom | ||
| 2 | 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 | ||
| 3 | NCT04152200 (ClinicalTrials.gov) | January 21, 2020 | 31/10/2019 | A Study to Evaluate Lumasiran 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;Primary Hyperoxaluria | Drug: Lumasiran | Alnylam Pharmaceuticals | NULL | Recruiting | N/A | N/A | All | 20 | Phase 3 | United States;Australia;Belgium;France;Israel;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom |
| 4 | 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 | ||
| 5 | 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 | ||
| 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-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 | ||
| 7 | NCT03905694 (ClinicalTrials.gov) | April 22, 2019 | 1/4/2019 | A Study of Lumasiran 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;Primary Hyperoxaluria Type 1 (PH1) | Drug: Lumasiran | Alnylam Pharmaceuticals | NULL | Active, not recruiting | N/A | 5 Years | All | 18 | Phase 3 | United States;France;Germany;Israel;United Kingdom |
| 8 | 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 | ||
| 9 | 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 | |||
| 10 | 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 | |||
| 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 | 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 | ||
| 12 | NCT03681184 (ClinicalTrials.gov) | November 27, 2018 | 19/9/2018 | A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 | 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) | Drug: Lumasiran;Drug: Sterile Normal Saline (0.9% NaCl) | Alnylam Pharmaceuticals | NULL | Active, not recruiting | 6 Years | N/A | All | 39 | Phase 3 | United States;France;Germany;Israel;Netherlands;Switzerland;United Arab Emirates;United Kingdom |
| 13 | 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 | |||
| 14 | 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 |
| 15 | 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 | ||
| 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 | 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 | ||
| 17 | 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 |
| 18 | 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 | |||
| 19 | 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 |