NL-OMON54596招募中3 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 - DCR-PHXC-301 (PHYOX 3)
Dicerna Pharmaceuticals Inc (a Novo Nordisk Company)0 个研究点目标入组 8 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 8
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
- 0 至 64（—）
入选标准
- •ROLL-OVER PARTICIPANTS AND THEIR PEDIATRIC SIBLINGS
- •1. Participants starting at birth are eligible for this study.
- •2.Documented diagnosis of PH, confirmed by genotyping (historically available
- •genotype information is acceptable for study eligibility)
- •3. Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of
- •DCR-PHXC or is the sibling of a participant who either successfully completed a
- •Dicerna study of DCR-PHXC successfully completed a Dicerna Pharmaceuticals,
- •Inc. study of DCR-PHXC.
- •a. For participants rolling over from a multidose study of DCR-PHXC, enrollment
- •should occur within a window of 25 to 75 days from the last dose of study
- •intervention. In order to minimize any gap in administration of DCR-PHXC, every
- •effort should be made to enroll participants as soon as all assessments from
- •the previous study have been completed. It should be noted if the participant
- •was required to repeat the end-of-study (EOS) 24-hour Uox collection for
- •violation of completeness criteria.
- •4. Estimated GFR at screening >= 30 mL/min normalized to 1.73 m2 BSA,calculated
- •using the equations found in Section8.2.4.1 (Protocol). For infants aged less
- •than 12 months, serum creatinine below The 97.5th percentile of a healthy
- •population (Boer et al. 2010)
- •In the Netherlands, children aged 0 to 5 must have PH1 to be eligible for
- •enrollment. Pediatric patients with PH2 or PH3 are not eligible for enrollment,
- •as the efficacy of DCR-PHXC has not yet been established in patients with PH2
排除标准
- •ROLL-OVER PARTICIPANTS AND THEIR PEDIATRIC SIBLING
- •1. Prior renal or hepatic transplantation; or planned transplantation within
- •the study period
- •2. Currently receiving dialysis
- •3. Documented evidence of clinical manifestations of systemic oxalosis.
研究者
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