NCT03847909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
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 35
- 试验地点
- 1
- 主要终点
- AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of DCR-PHXC in Children and Adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2)
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Capable and willing to provide written informed consent or assent
- •Documented diagnosis of PH1 or PH2, confirmed by genotyping
- •Must meet the 24 hour urine oxalate excretion requirements
- •Less than 20% variation between the two 24-hour urinary creatinine excretion values 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
排除标准
- •Renal or hepatic transplantation (prior or planned within the study period)
- •Currently on dialysis or anticipated requirement for dialysis during the study period
- •Plasma oxalate >30 µmol/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)
- •Use of an RNA interference (RNAi) drug within the last 6 months
- •Participation in any clinical study in which you received an investigational medicinal product (IMP) within 4 months before Screening
- •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
- •Inability or unwillingness to comply with study procedures
研究组 & 干预措施
DCR-PHXC
Experimental
Intervention, drug, DCR-PHXC
干预措施: DCR-PHXC (Drug)
Placebo - Sterile Normal Saline (0.9% NaCl)
Placebo Comparator
Placebo, sterile normal saline (0.9% NaCl) for subcutaneous (SC) injection
干预措施: Sterile Normal Saline (0.9% NaCl) (Drug)
结局指标
主要结局
AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox
时间窗: From Day 90 to 180
The AUC of 24-hour urinary oxalate (Uox) from Day 90 to Day 180, based on percent change from baseline, was compared between the active treatment group and placebo group. A multiple imputation approach was used to handle missing Uox data and then calculate the AUC.
次要结局
- Change From Baseline in Clinical Hematology Laboratory Tests: Basophils/Leukocytes(Baseline, Day 180)
- Change From Baseline in Vital Signs: Height(Baseline, Day 180)
- Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea(Baseline, Day 180)
- Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes(Baseline, Day 180)
- Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes (Ery.) Mean Corpuscular Volume and Mean Platelet Volume(Baseline, Day 180)
- Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils(Baseline, Day 180)
- Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only)(Baseline, Day 180)
- Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs)(From Baseline up to Day 180)
- Change From Baseline in Electrocardiogram (ECG): Heart Rate(Baseline, Day 180)
- Change From Baseline in Vital Signs: Heart Rate(Baseline, Day 180)
- Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase(Baseline, Day 180)
- Change From Baseline in Clinical Chemistry Laboratory Tests: Protein, Albumin(Baseline, Day 180)
- Change From Baseline in Vital Signs: Weight(Baseline, Day 180)
- Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones(Baseline, Day 180)
- Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval(Baseline, Day 180)
- Change From Baseline in Vital Signs: Respiratory Rate(Baseline, Day 180)
- Change From Baseline in Vital Signs: Systolic and Diastolic Blood Pressure(Baseline, Day 180)
- Change From Baseline in Clinical Chemistry Laboratory Tests: Vitamin B6(Baseline, Day 180)
- Change From Baseline in Vital Signs: Body Mass Index (BMI)(Baseline, Day 180)
- Percent Change From Baseline to Day 180 in the Number of Kidney Stones(Baseline, Day 180)
- Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Day 180(Baseline, Day 180)
- Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination(Baseline up to Day 180)
- Change From Baseline in Clinical Chemistry Laboratory Tests: Bilirubin, Direct Bilirubin and Creatinine(Baseline, Day 180)
- Change From Baseline in Clinical Hematology Laboratory Tests: Hematocrit(Baseline, Day 180)
- Change From Baseline in Clinical Hematology Laboratory Tests: Lymphocytes/Leukocytes(Baseline, Day 180)
- Change From Baseline in Clinical Hematology Laboratory Tests: Neutrophils/Leukocytes(Baseline, Day 180)
- Change From Baseline in Clinical Urinalysis Laboratory Tests: pH(Baseline, Day 180)
- Area Under the Curve From Time of Administration to the Last Measurable Concentration (AUC0-last) of of DCR-PHXC(For adults: Day 1 and 30: predose, 5, 15, and 30 minutes and 1, 2, 4, 6, 10, and 12 hours postdose; Day 150: predose, 2, 6, and 12 hours postdose For adolescents: Days 1 and 30: predose, 30 minutes and 2 and 10 hours postdose)
- Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits(From Day 90 to 180)
- Change From Baseline in Vital Signs: Oral Body Temperature(Baseline, Day 180)
- Change From Baseline in Clinical Hematology Laboratory Tests: Monocytes/Leukocytes(Baseline, Day 180)
- Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC(For adults: Day 1 and 30: predose, 5, 15, and 30 minutes and 1, 2, 4, 6, 10, and 12 hours (hrs) postdose; Day 150: predose, 2, 6, and 12 hours postdose For adolescents: Days 1 and 30: predose, 30 minutes and 2 and 10 hours postdose)
- Change From Baseline in Clinical Hematology Laboratory Tests: Hemoglobin and Erythrocytes Mean Corpuscular Hemoglobin Concentration(Baseline, Day 180)
- Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes Mean Corpuscular Hemoglobin(Baseline, Day 180)
- Change From Baseline in Clinical Hematology Laboratory Tests: Eosinophils/Leukocyte(Baseline, Day 180)
- Change From Baseline in Clinical Urinalysis Laboratory Tests: Specific Gravity(Baseline, Day 180)
研究者
研究点 (1)
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