234. Peroxisomal disease (except Adrenoleukodystrophy) Clinical trials / Disease details
Clinical trials : 39 / Drugs : 35 - (DrugBank : 12) / Drug target genes : 13 - Drug target pathways : 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 | 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 | ||
| 3 | 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 | ||
| 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 |