234. Peroxisomal disease (except Adrenoleukodystrophy)
38 clinical trials,   37 drugs   (DrugBank: 12 drugs),   12 drug target genes,   42 drug target pathways

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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
5 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1NCT04580420
(ClinicalTrials.gov)
October 202024/9/2020Safety & Efficacy of DCR-PHXC in Patients With PH1/2 and ESRDA 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 DialysisPrimary Hyperoxaluria;Primary Hyperoxaluria Type 2;End Stage Renal DiseaseDrug: DCR-PHXCDicerna Pharmaceuticals, Inc.NULLNot yet recruitingN/AN/AAll12Phase 2NULL
2NCT03847909
(ClinicalTrials.gov)
October 28, 201915/2/2019A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2A 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 HyperoxaluriaPrimary Hyperoxaluria Type 1 (PH1);Primary Hyperoxaluria Type 2 (PH2);Kidney Diseases;Urologic Diseases;Genetic DiseaseDrug: DCR-PHXC;Drug: Sterile Normal Saline (0.9% NaCl)Dicerna Pharmaceuticals, Inc.NULLRecruiting6 YearsN/AAll36Phase 2United States;Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom
3NCT04042402
(ClinicalTrials.gov)
July 9, 201910/7/2019Long Term Extension Study in Patients With Primary HyperoxaluriaAn 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 HyperoxaluriaPrimary Hyperoxaluria Type 1 (PH1);Primary Hyperoxaluria Type 2 (PH2);Kidney Diseases;Urologic Diseases;Genetic DiseaseDrug: DCR-PHXCDicerna Pharmaceuticals, Inc.NULLEnrolling by invitation6 YearsN/AAll50Phase 3United States;France;Germany;Netherlands;United Kingdom
4EUCTR2020-002826-97-FR
(EUCTR)
30/09/2020Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, safety and tolerability of DCR-PHXCA 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 IncNULLNAFemale: yes
Male: yes
24Phase 2United States;France;Spain;Romania;Australia;Germany;United Kingdom;Japan;Italy
5EUCTR2020-002826-97-DE
(EUCTR)
08/12/2020Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, safety and tolerability of DCR-PHXCA 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 IncNULLNAFemale: yes
Male: yes
24Phase 2United States;France;Spain;Romania;Australia;Germany;United Kingdom;Japan;Italy