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 - PHYOX2

Dicerna Pharmaceuticals, Inc0 个研究点目标入组 36 人开始时间: 2022年5月23日最近更新: 2024年8月19日

适应症

试验速览

暂无简介。

•24-hour Uox excretion = 0.7 mmol (adjusted per 1.73 m2 body surface area [BSA] in participants < 18 years of age) in both collections performed in the screening period. Of the first 24 participants enrolled, at least 12 (50%) must have at least one 24-hour Uox excretion = 1.6 mmol (adjusted per 1.73 m2 BSA in participants aged < 18 years).
•Less than 20% variation between the two 24-hour urinary creatinine excretion values [mmol/24 hr/kg] derived from the two 24-hour urine collections in the screening period.
•Estimated glomerular filtration rate (eGFR) at screening = 30 mL/min normalized to 1.73 m2 BSA calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula in participants aged = 18 years (Levey & Stevens, 2010), or the formula by Schwartz in participants aged 6 to 17 years, (Schwartz et al., 2009; National Kidney Foundation, 2002). In Japan, the formula by Uemura et al. will be used for
•participants aged 6 to 17 years.

•Renal or hepatic transplantation (prior or planned within the study period)
•Current dialysis or anticipated requirement for dialysis during the study period
•Plasma oxalate > 30 µmol/L
•Documented evidence of clinical manifestations of systemic oxalosis (including preexisting retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)
•Liver function test (LFT) abnormalities: Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) >1.5 times upper limit of normal (ULN) for age and gender.

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