234. ペルオキシソーム病（副腎白質ジストロフィーを除く。） Peroxisomal disease (except Adrenoleukodystrophy) Clinical trials / Disease details
臨床試験数 : 39 / 薬物数 : 35 - (DrugBank : 12) / 標的遺伝子数 : 13 - 標的パスウェイ数 : 45
| 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 | NCT05001269 (ClinicalTrials.gov) | February 22, 2022 | 30/6/2021 | Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function | A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients From Birth to 11Years of Age With Primary Hyperoxaluria and Relatively Intact Renal Function | Primary Hyperoxaluria;Primary Hyperoxaluria Type 1;Primary Hyperoxaluria Type 2;Primary Hyperoxaluria Type 3 | Drug: nedosiran | Dicerna Pharmaceuticals, Inc. | NULL | Recruiting | N/A | 11 Years | All | 20 | Phase 2 | United States;Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Poland;Spain;Turkey;United Arab Emirates;United Kingdom |
| 2 | NCT04125472 (ClinicalTrials.gov) | June 2, 2021 | 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 | Approved for marketing | N/A | N/A | All | Belgium;Canada;France;French Polynesia;Germany;Italy;Netherlands;Spain;Switzerland;United Kingdom;United States | ||
| 3 | EUCTR2019-001346-17-IT (EUCTR) | 14/05/2021 | 07/06/2021 | 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) - ILLUMINATE-C | 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 Arab Emirates;United States;Lebanon;Turkey;Israel;United Kingdom;Switzerland;Italy;India;Egypt;France;Jordan;Belgium;Australia;Netherlands;Germany | ||
| 4 | EUCTR2020-002826-97-IT (EUCTR) | 12/05/2021 | 07/06/2021 | Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, safety and tolerability of DCR-PHXC | A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCRPHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe RenalImpairment, With or Without Dialysis - PHYOX 7: Safety & Efficacy of DCR-PHXC in patients with PH1/2 and ESRD | Primary Hyperoxaluria 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: Nedosiran Product Code: [DCR-PHXC] INN or Proposed INN: DCR-L1360 | Dicerna Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 2 | France;United States;Spain;Romania;Australia;Germany;United Kingdom;Italy | ||
| 5 | EUCTR2020-002826-97-DE (EUCTR) | 20/04/2021 | 08/12/2020 | Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, safety and tolerability of DCR-PHXC | A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis - PHYOX 7: Safety & Efficacy of DCR-PHXC in patients with PH1/2 and ESRD | Primary Hyperoxaluria 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: Nedosiran Product Code: DCR-PHXC INN or Proposed INN: DCR-L1360 Other descriptive name: Nedosiran | Dicerna Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 2 | United States;France;Spain;Romania;Germany;United Kingdom;Italy | ||
| 6 | NCT04580420 (ClinicalTrials.gov) | April 15, 2021 | 24/9/2020 | Safety & Efficacy of DCR-PHXC in Patients With PH1/2 and ESRD | A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis | Primary Hyperoxaluria Type 1;Primary Hyperoxaluria Type 2;End Stage Renal Disease | Drug: DCR-PHXC | Dicerna Pharmaceuticals, Inc. | NULL | Recruiting | N/A | N/A | All | 17 | Phase 2 | United States;Spain;United Kingdom |
| 7 | EUCTR2020-002826-97-FR (EUCTR) | 29/01/2021 | 30/09/2020 | Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, safety and tolerability of DCR-PHXC | A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis - PHYOX 7: Safety & Efficacy of DCR-PHXC in patients with PH1/2 and ESRD | Primary Hyperoxaluria 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: Nedosiran Product Code: DCR-PHXC INN or Proposed INN: DCR-L1360 Other descriptive name: Nedosiran | Dicerna Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 2 | United States;France;Spain;Romania;Australia;Germany;United Kingdom;Japan;Italy | ||
| 8 | 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 | ||
| 9 | 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 | Active, not recruiting | N/A | N/A | All | 21 | Phase 3 | United States;Australia;Belgium;France;Israel;Italy;Jordan;Lebanon;Netherlands;Turkey;United Arab Emirates;Switzerland;United Kingdom |
| 10 | 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 | ||
| 11 | 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 States;Egypt;United Kingdom;United Arab Emirates;Switzerland;India;Lebanon;Netherlands;Turkey;Belgium;Italy;Israel;Australia;France;Jordan;Germany | ||
| 12 | 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 | France;United States;Israel;Germany;United Kingdom | ||
| 13 | 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 | 0 Years | 5 Years | All | 18 | Phase 3 | United States;France;Germany;Israel;United Kingdom |
| 14 | 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 | ||
| 15 | 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] | 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Israel;Germany;United Kingdom | ||
| 16 | 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] | 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 | 8 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;France;Israel;Germany;United Kingdom | ||
| 17 | NCT03856866 (ClinicalTrials.gov) | January 11, 2019 | 5/11/2018 | Hydroxychloroquine Administration for Reduction of Pexophagy | Hydroxychloroquine Administration for Reduction of Pexophagy | Zellweger Syndrome;Peroxisome Biogenesis Disorders | Drug: Hydroxychloroquine;Drug: Placebo | The Hospital for Sick Children | NULL | Recruiting | 6 Months | 40 Years | All | 5 | Phase 2 | Canada |
| 18 | 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 | France;United States;United Arab Emirates;Jordan;Israel;Netherlands;Germany;Switzerland;United Kingdom | ||
| 19 | 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: Placebo;Drug: Lumasiran | 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 |
| 20 | 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] | 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 | 39 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;United Arab Emirates;Jordan;Israel;Netherlands;Germany;Switzerland;United Kingdom | ||
| 21 | 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 | ||
| 22 | 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 |
| 23 | 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 | France;United States;United Arab Emirates;Jordan;Israel;Netherlands;Germany;United Kingdom | ||
| 24 | 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 | ||
| 25 | 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 | ||
| 26 | 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 | ||
| 27 | EUCTR2015-003142-51-NL (EUCTR) | 21/07/2016 | 17/11/2015 | An Early Phase Study of DCR-PH1 in Patients with an Inherited Disorder Resulting in Overproduction of Oxalate | A Phase 1 Study of DCR-PH1 in Patients with Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 1 MedDRA version: 18.1;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: DCR-PH1 INN or Proposed INN: Not Yet Assigned Other descriptive name: DCR-1171X | Dicerna Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 42 | Phase 1 | United States;France;Israel;Germany;Netherlands;United Kingdom | ||
| 28 | NCT02795325 (ClinicalTrials.gov) | May 13, 2016 | 18/5/2016 | A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1) | A Phase 1 Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 1 | Drug: DCR-PH1;Other: Placebo | Dicerna Pharmaceuticals, Inc. | NULL | Terminated | 12 Years | N/A | All | 41 | Phase 1 | Germany;Netherlands |
| 29 | EUCTR2015-003142-51-GB (EUCTR) | 11/04/2016 | 06/10/2015 | An Early Phase Study of DCR-PH1 in Patients with an Inherited Disorder Resulting in Overproduction of Oxalate | A Phase 1 Study of DCR-PH1 in Patients with Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 1 MedDRA version: 18.0;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: DCR-PH1 INN or Proposed INN: Not Yet Assigned Other descriptive name: DCR-1171X | Dicerna Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 42 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Israel;Netherlands;Germany;United Kingdom | ||
| 30 | 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 |
| 31 | 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 | ||
| 32 | NCT02171104 (ClinicalTrials.gov) | July 10, 2014 | 20/6/2014 | MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG | Mucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome;Alpha-Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Glycoprotein Metabolic Disorders;Sphingolipidoses;Recessive Leukodystrophies;Globoid Cell Leukodystrophy;Metachromatic Leukodystrophy;Niemann-Pick B;Niemann-Pick C Subtype 2;Sphingomyelin Deficiency;Peroxisomal Disorders;Adrenoleukodystrophy With Cerebral Involvement;Zellweger Syndrome;Neonatal Adrenoleukodystrophy;Infantile Refsum Disease;Acyl-CoA Oxidase Deficiency;D-Bifunctional Enzyme Deficiency;Multifunctional Enzyme Deficiency;Alpha-methylacyl-CoA Racmase Deficiency;Mitochondrial Neurogastrointestingal Encephalopathy;Severe Osteopetrosis;Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation);Inherited Metabolic Disorders | Biological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Preparative Regimen;Drug: Osteopetrosis Haploidentical Only Preparative Regimen;Drug: cALD SR-A (Standard-Risk, Regimen A);Drug: cALD SR-B (Standard-Risk, Regimen B);Drug: cALD HR-D (High-Risk, Regimen C);Drug: cALD HR-D (High-Risk, Regimen D) | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 55 Years | All | 100 | Phase 2 | United States |
| 33 | NCT01626092 (ClinicalTrials.gov) | July 11, 2012 | 20/6/2012 | Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders | Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT) | Lysosomal Storage Disease;Peroxisomal Disorder | Drug: Campath-1H;Drug: Clofarabine;Drug: Melphalan;Radiation: Total Body Irradiation with Marrow Boosting;Biological: Hematopoietic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate mofetil | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 55 Years | All | 3 | N/A | United States |
| 34 | NCT00004442 (ClinicalTrials.gov) | September 1997 | 18/10/1999 | Study of Bile Acids in Patients With Peroxisomal Disorders | Infantile Refsum's Disease;Zellweger Syndrome;Bifunctional Enzyme Deficiency;Adrenoleukodystrophy | Drug: chenodeoxycholic acid;Drug: cholic acid;Drug: ursodiol | Children's Hospital Research Foundation University of Cincinnati | Children's Hospital Medical Center, Cincinnati | Terminated | N/A | 5 Years | Both | 25 | N/A | NULL | |
| 35 | NCT00007020 (ClinicalTrials.gov) | January 1992 | 6/12/2000 | Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid | Investigation in the Pathogenesis of Liver Disease in Patients With Inborn Errors of Bile Acid Metabolism | Infantile Refsum's Disease;Zellweger Syndrome;Adrenoleukodystrophy;Peroxisomal Disorders;Cholestasis | Drug: Cholic Acids | Travere Therapeutics, Inc. | Children's Hospital Medical Center, Cincinnati | Completed | N/A | N/A | All | 85 | Phase 3 | United States |
| 36 | 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 | |||
| 37 | 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 | |||
| 38 | 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] | 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 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Jordan;Israel;Netherlands;Germany;United Kingdom | |||
| 39 | EUCTR2010-022046-25-NL (EUCTR) | 27/10/2010 | Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in Zellweger | Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in Zellweger | Peroxisomal biogenesis disorder Synonym: Zellweger spectrum disorderThis syndrome encompasses a spectrum of severity in symptomsPreviously the disorder was subdivided into three disorders based on the severity of symptoms; Infantile Refsum disease (IRD) is the mildest form, neonatal adrenoleucodystrofia (NALD) has more severe symptoms and Zellweger syndrome has the most severe clinical symptoms. MedDRA version: 12.1;Level: LLT;Classification code 10053706;Term: Zellweger syndrome MedDRA version: 12.1;Classification code 10038275;Term: Refsum's disease | Product Name: cholic acid Product Code: CA INN or Proposed INN: Cholic Acid Other descriptive name: CHOLIC ACID | Department of Pediatric Gastroenterology, Academic Medical Centre | NULL | NA | Female: yes Male: yes | Phase 2 | Netherlands |