NCT04042402进行中（未招募）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
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company2 个研究点 分布在 2 个国家目标入组 75 人开始时间: 2019年7月9日最近更新:
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 75
- 试验地点
- 2
- 主要终点
- The annual rate of decline in eGFR in participants with PH1
研究概览
简要总结
The proposed study is designed to provide patients previously enrolled in Phase 1 and 2 studies of DCR-PHXC and their siblings (<18 years old) long-term access to DCR-PHXC, and to evaluate the long-term safety and efficacy of DCR-PHXC in patients with PH.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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 75 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, or the multivariate equation by Schwartz in participants aged 12 months to 17 years. In Japan, the cystatin C-based Uemura formula will be used for participants aged 12 months to <2 years, the creatinine-based Uemura formula by will be used for participants aged 2 to 17 years, and the equation by Matsuo will be used in participants aged ≥ 18 years.
排除标准
- •Renal or hepatic transplantation (prior or planned within the study period)
- •Plasma oxalate > 30 µmol/L
- •Currently on dialysis
- •Documented evidence of clinical manifestations of systemic oxalosis
研究组 & 干预措施
Open Label
Experimental
Open label, monthly subcutaneous injection
干预措施: DCR-PHXC (Drug)
结局指标
主要结局
The annual rate of decline in eGFR in participants with PH1
时间窗: Annual change from baseline
To evaluate the effect of DCR PHXC on estimated glomerular filtration rate (eGFR) in participants with PH1
次要结局
- The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal vital signs(TEAEs and SAEs are evaluated monthly for 6 years)
- The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal 12 lead electrocardiogram (ECG) readings(TEAEs and SAEs are evaluated monthly for 6 years)
- The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal physical examination findings(TEAEs and SAEs are evaluated monthly for 6 years)
- The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs related to abnormal clinical laboratory tests (hematology, chemistry, coagulation parameters, and urinalysis)(TEAEs and SAEs are evaluated monthly for 6 years)
- To identify the percentage of participants with spot urinary oxalate-to-creatinine ratio ≤ the ULN or ≤ 1.5 x ULN(Spot urine collections are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that.)
- To assess the effect of DCR-PHXC on stone events in patients with PH(Evaluated yearly for 6 years)
- To assess the effect of DCR-PHXC on stone burden grade in patients with PH(Evaluated yearly for 6 years)
- To assess the effect of DCR-PHXC in nephrocalcinosis grade in patients with PH(Evaluated yearly for 6 years)
- To identify the proportion of participants with normalized or near-normalized 24 hour urinary oxalate (Uox)(24 hour urine collections (if applicable) are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that.)
- Change from Baseline in the Short Form (36) Health Survey (SF-36®) in PH1, PH2, and PH3 participant subgroups(Surveys are administered at screening, Day 180, yearly for 3.5 years, then at Month 72 (EOS).)
- Change from Baseline in the EQ-5D-5L™ in adults in PH1, PH2, and PH3 participant subgroups(Surveys are administered at screening, Day 180, yearly for 3.5 years, then at Month 72 (EOS).)
- Change from Baseline in the Pediatric Quality of Life Inventory (PedsQL™) in children in PH1, PH2, and PH3 participant subgroups(Surveys are administered at screening, Day 180, yearly for 3.5 years, then at Month 72 (EOS).)
- To assess the efficacy of DCR PHXC in reducing Uox burden in patients with PH: TWS AUC(Monthly for 4 months (D90 through D180))
- To assess the long-term efficacy of DCR PHXC in reducing Uox burden in patients with PH(24 hour urine collections (if applicable) are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that.)
- To evaluate the incidence of chronic kidney disease (CKD) and end-stage renal disease (ESRD) in participants with PH(eGFR is evaluated monthly for 6 months (or quarterly for multidose rollovers), quarterly for 2 1/2 years, and every 6 months for 3 years after that.)
- To assess the long-term efficacy of DCR-PHXC in reducing Uox burden in patients with PH(Spot urine collections are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that.)
研究者
研究点 (2)
Loading locations...
相似试验
终止
2 期
An Extension Study of GDC-0853 in Participants With Moderate to Severe Active Systemic Lupus ErythematosusLupus Erythematosus, SystemicNCT03407482Genentech, Inc.160
已完成
2 期
Open-Label Extension Study of Patients Previously Enrolled in Study CIN-107-124HypertensionNCT05459688AstraZeneca175
已完成
2 期
A Rollover Study for Subjects That Have Participated in an Astellas Sponsored Linsitinib TrialAdvanced Solid TumorsNCT02057380Astellas Pharma Global Development, Inc.13
终止
3 期
OPen Label Study to Evaluate Long Term Treatment Effect of DiaPep277Type 1 DiabetesNCT01460251Rabin Medical Center11
终止
1 期
Study of Melphalan Flufenamide (Melflufen) + Dex With Bortezomib or Daratumumab in Patients With RRMMMultiple MyelomaNCT03481556Oncopeptides AB56