NCT05167825已完成3 期
A Multicenter, Open-label, Phase III Study to Assess the Efficacy, Safety, and Pharmacokinetics of Macitentan in Japanese Pediatric Patients (>=3 Months to <15 Years) With Pulmonary Arterial Hypertension
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 7
- 试验地点
- 22
- 主要终点
- Fold Change From Baseline at Week 24 in Pulmonary Vascular Resistance Index (PVRI)
研究概览
简要总结
The purpose of this study is to evaluate the effect of macitentan on hemodynamic measures at Week 24 in pediatric populations.
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
- 年龄范围
- 3 Months 至 15 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pulmonary arterial hypertension (PAH) belonging to the nice 2013 updated classification group 1
- •PAH diagnosis confirmed by historical right heart catheterization where in the absence of pulmonary vein obstruction and/or significant lung disease pulmonary artery wedge pressure (PAWP) can be replaced by left atrium pressure (LAP) or left ventricular end diastolic pressure (LVEDP) (in absence of mitral stenosis) assessed by heart catheterization
- •World Health Organization (WHO) functional class (FC) I to IV
- •PAH-specific treatment-naïve participants or participants on PAH-specific treatment
- •A female of childbearing potential must have a negative highly sensitive serum beta-human chorionic gonadotropin (beta-hCG) test at screening and a negative urine pregnancy test at the first administration of study intervention
- •A female participant must not get pregnant and must agree not to donate eggs during the study and for a period of up to 4 weeks following the end of study
排除标准
- •Participants with PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease, and/or pulmonary capillary hemangiomatosis, and persistent pulmonary hypertension of the newborn
- •Participants with the following diseases: pulmonary vein stenosis; bronchopulmonary dysplasia
- •Severe hepatic impairment, example, Child-Pugh Class C, at screening
- •Pregnant, or breast-feeding, or planning to become pregnant while enrolled in this study or within 4 weeks after the last dose of study intervention
- •Known allergies, hypersensitivity, or intolerance to macitentan or its excipients
- •Participant with PAH associated with open shunts, with congenital cardiac abnormalities such as univentricular heart, with pulmonary hypertension due to lung disease, and renal dysfunction
研究组 & 干预措施
Macitentan
Experimental
Participants will receive oral dose of macitentan based on age and weight through Week 52.
干预措施: Macitentan (Drug)
结局指标
主要结局
Fold Change From Baseline at Week 24 in Pulmonary Vascular Resistance Index (PVRI)
时间窗: Baseline (Day 1), Week 24
PVRI fold change at Week 24 was calculated as 100\*(PVRI at Week 24 divided by PVRI at baseline). PVR was determined by right heart catheterization.
次要结局
- Change From Baseline to Weeks 24 and 52 in 6-minute Walk Distance (6MWD) as Measured by the 6-minute Walk Test (6MWT)(Baseline (Day 1), Weeks 24 and 52)
- Change From Baseline to Weeks 12, 24, and 52 in Physical Activity as Measured by Accelerometry: Number of Hours of Daytime Activity(Baseline (Day 1), Weeks 12, 24, and 52)
- Change From Baseline to Weeks 12, 24, and 52 in Physical Activity as Measured by Accelerometry: Mean Count Per Minute of Daily Activity(Baseline (Day 1), Weeks 12, 24, and 52)
- Change From Baseline to Weeks 12, 24, and 52 in Physical Activity as Measured by Accelerometry: Mean Daily Time Spent in Light Physical Activity(Baseline (Day 1), Weeks 12, 24, and 52)
- Change From Baseline to Weeks 12, 24, and 52 in Physical Activity as Measured by Accelerometry: Mean Daily Time Spent in Moderate to Vigorous Physical Activity(Baseline (Day 1), Weeks 12, 24, and 52)
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)(From Baseline (Day 1) up to Week 56)
- Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)(From Baseline (Day 1) up to Week 56)
- Change From Baseline in Vital Signs: Pulse Rate(Baseline (Day 1), Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Change From Baseline in Electrocardiogram (ECG) Parameter: Heart Rate(Baseline (Day 1), Weeks 12, 24, and 52)
- Change From Baseline in Electrocardiogram (ECG) Parameter: PR, QRS, QT, Corrected QT Interval-Bazett's Formula (QTcB), and Corrected QT Interval-Fridericia's Formula (QTcF)(Baseline (Day 1), Weeks 12, 24, and 52)
- Number of Participants With AEs of Special Interest(From Baseline (Day 1) up to Week 56)
- Number of Participants With Postbaseline Markedly Abnormal Hematology Laboratory Values(Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Number of Participants With Change From Baseline at Weeks 4, 8, 12, 16, 20, 24, 28, 40, and 52 in Panama FC(Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Change From Baseline at Week 24 in Hemodynamic Variables: Pulmonary Vascular Resistance (PVR)(Baseline (Day 1), Week 24)
- Change From Baseline in Mean Right Atrial Pressure (mRAP)(Baseline (Day 1), Week 24)
- Change From Baseline at Week 24 in Hemodynamic Variables: Mean Pulmonary Arterial Pressure (mPAP)(Baseline (Day 1), Week 24)
- Change From Baseline at Week 24 in Hemodynamic Variables: Cardiac Index (CI)(Baseline (Day 1), Week 24)
- Change From Baseline at Week 24 in Hemodynamic Variables: Cardiac Output (CO)(Baseline (Day 1), Week 24)
- Change From Baseline at Week 24 in Hemodynamic Variables: Total Pulmonary Resistance (TPR)(Baseline (Day 1), Week 24)
- Change From Baseline at Week 24 in Hemodynamic Variables: Mixed Venous Oxygen Saturation (SvO2)(Baseline (Day 1), Week 24)
- Number of Participants With Change From Baseline at Weeks 4, 8, 12, 16, 20, 24, 28, 40, and 52 in World Health Organization (WHO) Functional Class (FC)(Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Change From Baseline to Week 12, 24, 28, 40, and 52 in N-terminal Pro-Brain Natriuretic Peptide (NT-proBNP)(Baseline (Day 1), Week 12, 24, 28, 40, and 52)
- Change From Baseline at Weeks 12, 24, and 52 in Tricuspid Annular Plane Systolic Excursion (TAPSE)(Baseline (Day 1), Weeks 12, 24, and 52)
- Change From Baseline at Week 12, 24, and 52 in Left Ventricular Eccentricity Index (LVEI)(Baseline (Day 1), Weeks 12, 24 and 52)
- Change From Baseline to Week 12, 24, and 52 in Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales Short Form (SF-15)(Baseline (Day 1), Weeks 12, 24, and 52)
- Change From Baseline to Week 24 and Week 52 in Borg Dyspnea Index(Baseline (Day 1), Weeks 24 and 52)
- Number of Participants With AEs Leading to Premature Discontinuation of Macitentan(From Baseline (Day 1) up to Week 52)
- Number of Participants With Postbaseline Markedly Abnormal Clinical Chemistry Laboratory Values(Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Change From Baseline in Hematology Parameters: Platelets, Leukocytes, Neutrophils Band Form, Lymphocytes, Monocytes, Eosinophils, and Basophils(Baseline (Day 1), Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Change From Baseline in Hematology Parameters: Hematocrit(Baseline (Day 1), Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Change From Baseline in Hematology Parameters: Erythrocytes(Baseline (Day 1), Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Change From Baseline in Chemistry Parameters: Creatinine, Bilirubin, and Direct Bilirubin(Baseline (Day 1), Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Change From Baseline in Chemistry Parameters: Creatinine Clearance(Baseline (Day 1), Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Change From Baseline in Chemistry Parameters: GFR From Cystatin C Adjusted for BSA(Baseline (Day 1), Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Change From Baseline in Chemistry Parameters: Aspartate Aminotransferase, Alanine Aminotransferase, and Alkaline Phosphatase(Baseline (Day 1), Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Change From Baseline in Body Weight(From baseline (Day 1) up to Week 56)
- Change From Baseline in Height(From baseline (Day 1) up to Week 56)
- Plasma Concentration of Macitentan: Participants >=2 Years Old(Baseline (Day 1) up to Week 12)
- Plasma Concentration of Aprocitentan: Participants >=2 Years Old(Baseline (Day 1) up to Week 12)
- Plasma Concentration of Macitentan: Participants <2 Years Old(Baseline (Day 1) up to Week 8)
- Plasma Concentration of Aprocitentan: Participants <2 Years Old(Baseline (Day 1) up to Week 8)
- Change From Baseline in Hematology Parameters: Hemoglobin(Baseline (Day 1), Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Change From Baseline in Chemistry Parameters: Sodium, Potassium, Urea Nitrogen, Glucose, and Calcium(Baseline (Day 1), Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
- Change From Baseline in Vital Signs: Blood Pressure(Baseline (Day 1), Weeks 4, 8, 12, 16, 20, 24, 28, 40, 52)
研究者
研究点 (22)
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