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

  
1 trial found
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1NCT03847909
(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.NULLCompleted6 YearsN/AAll35Phase 2United States;Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom