NL-OMON49278已完成2 期
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 - DCR-PHXC-201 (PHYOX 2)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 6
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 99（—）
入选标准
- •Key inclusion criteria:
- •- 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 GFR at screening * 30 mL/min normalized to 1.73 m2 BSA,
- •calculated using the CKD-EPI formula in participants aged * 18 years or the
- •2012 multivariate equation by Schwartz in participants aged 6 to 17 years.
排除标准
- •Key exclusion criteria:
- •- Prior renal or hepatic transplantation; or planned transplantation within
- •the study period
- •- Currently receiving dialysis or anticipating requirement for dialysis during
- •the study period
- •- Plasma oxalate > 30 *mol/L
- •- Documented evidence of clinical manifestations of systemic oxalosis
- •(including pre-existing 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.
研究者
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