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 : 41 / 薬物数 : 37 - (DrugBank : 12) / 標的遺伝子数 : 13 - 標的パスウェイ数 : 44
| 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 | 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 | ||
| 2 | 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 | ||
| 3 | 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;Primary Hyperoxaluria Type 2;End Stage Renal Disease | Drug: DCR-PHXC | Dicerna Pharmaceuticals, Inc. | NULL | Recruiting | N/A | N/A | All | 12 | Phase 2 | United States;Spain;United Kingdom |
| 4 | 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 | ||
| 5 | NCT03847909 (ClinicalTrials.gov) | October 28, 2019 | 15/2/2019 | A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 | A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria | Primary Hyperoxaluria Type 1 (PH1);Primary Hyperoxaluria Type 2 (PH2);Kidney Diseases;Urologic Diseases;Genetic Disease | Drug: DCR-PHXC;Drug: Sterile Normal Saline (0.9% NaCl) | Dicerna Pharmaceuticals, Inc. | NULL | Completed | 6 Years | N/A | All | 35 | Phase 2 | United States;Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom |
| 6 | NCT04042402 (ClinicalTrials.gov) | July 9, 2019 | 10/7/2019 | Long Term Extension Study in Patients With Primary Hyperoxaluria | An Open-Label Roll-Over Study to Evaluate the Long-Term Safety and Efficacy of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria | Primary Hyperoxaluria Type 1 (PH1);Primary Hyperoxaluria Type 2 (PH2);Kidney Diseases;Urologic Diseases;Genetic Disease | Drug: DCR-PHXC | Dicerna Pharmaceuticals, Inc. | NULL | Enrolling by invitation | 6 Years | N/A | All | 50 | Phase 3 | United States;Australia;Canada;France;Germany;Italy;Japan;Lebanon;Netherlands;Spain;United Kingdom |