LBCTR2020124677Other3 期
An 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 Hyperoxaluria - PHYOX3”
适应症
试验速览
- 阶段
- 3 期
- 状态
- Other
- 入组人数
- 50
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Single Arm Study
- 主要目的
- Treatment
- 盲法
- Open (masking not used)
入排标准
- 年龄范围
- 6 至 99（—）
- 性别
- All
入选标准
- •Key Inclusion Criteria:
- •Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC.
- •OR Participant is the sibling of a participant who successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC. Siblings must be younger than 18 years of age and must have genetically confirmed PH.
- •For participants rolling over from a multidose study of DCR-PHXC, enrollment should occur within a window of 25 to 60 days from the last dose of study intervention. Estimated GFR at screening = 30 mL/min normalized to 1.73 m2 body surface area (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 16 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 (Uemura et al., 2014).
排除标准
- •Key exclusion criteria include:
- •Renal or hepatic transplantation; prior or planned within the study period
- •Current dialysis
- •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).
研究者
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