NCT05731492撤回1 期
A Multicenter, Open-label, Single-arm Study to Assess the Pharmacokinetics and Safety of Macitentan in Children Aged 1 Month to <2 Years With Pulmonary Arterial Hypertension
Actelion4 个研究点 分布在 2 个国家开始时间: 2024年3月14日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 发起方
- 试验地点
- 4
- 主要终点
- Trough Concentration of Macitentan and its Active Metabolite Aprocitentan at Week 4 in Steady-State
研究概览
简要总结
The purpose of this study is to learn what happens to macitentan and its active metabolite (aprocitentan) in the body of children aged between 1 month and 2 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- 1 Month 至 2 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pulmonary arterial hypertension (PAH): 1) including participants with Down syndrome. Diagnosis must have been confirmed by (historical, any time before screening) right heart catheterization mean pulmonary arterial pressure (mPAP) greater than or equal to (>=) 25 millimeter of mercury (mmHg), pulmonary arterial wedge pressure (PAWP) less than or equal to (=<)15 mmHg, pulmonary vascular resistance index greater than (>) 3 Wood units * meter square (m^2) where in the absence of pulmonary vein obstruction and/or significant lung disease PAWP can be replaced left atrium pressure or left ventricular end diastolic pressure (in the absence of mitral stenosis) assessed by heart catheterization. a) Idiopathic PAH, or b) Heritable PAH, or c) PAH associated with congenital heart disease: i) Eisenmenger syndrome (Qp/Qs less than (<) 1.5 and saturation of peripheral oxygen ≤ 90 percent (%) measured by pulse oximetry at room air), or ii) Inoperable open left-to-right shunts (with a Pulmonary vascular resistance [PVR] > 8 WU and Qp/Qs <2), or iii) Co-incidental shunt (that is, not explaining hemodynamically the presence of PAH), or iv) Post-operative PAH (persisting/recurring/developing ≥ 6 months after repair of shunt), or d) Drug or toxin induced PAH, or e) PAH associated with Human immunodeficiency viruses (HIV)
- •World Health Organization Functional Class (WHO FC) I, II, or III
- •PAH-specific treatment-naive participants or participants on PAH specific monotherapy or combination of 2 therapies. Use of macitentan before or during screening is allowed
- •Body weight of greater than or equal to (>=) 3.5 kilogram (kg)
- •Parent(s) (preferably both if available or as per local requirements) or participant's legally designated representative must sign an informed consent form (ICF) indicating that they understand the purpose of, and procedures required for, the study and is/are willing to allow the child to participate in the study
排除标准
- •PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease and/or pulmonary capillary hemangiomatosis
- •Persistent pulmonary hypertension of the newborn
- •The following congenital cardiac abnormalities: a) Cyanotic congenital cardiac lesions such as transposition of the great arteries, truncus arteriosus, pulmonary atresia with ventricular septal defect, unless operatively repaired and with no residual shunt. b) Univentricular heart and/or participants with Fontan-palliation
- •Pulmonary hypertension due to lung disease
- •Known diagnosis of bronchopulmonary dysplasia
研究组 & 干预措施
Open-label Core Treatment Period: Macitentan
Experimental
Participants will receive macitentan as a monotherapy or add-on to an existing therapy daily for 24 weeks during core treatment period. Optional treatment extension period of up to 1 year for those participants who completed the core treatment period.
干预措施: Macitentan (Drug)
结局指标
主要结局
Trough Concentration of Macitentan and its Active Metabolite Aprocitentan at Week 4 in Steady-State
时间窗: Predose (at Week 4)
Trough concentration of macitentan and its active metabolite aprocitentan at week 4 in steady-state will be reported.
次要结局
- Number of Participants with Serious Adverse Events (SAEs)(Up to 1.5 years)
- Number of Participants with Clinical Laboratory Abnormalities(Up to 1.5 years)
- Number of Participants with Change from Baseline in Clinical laboratory Values.(Up to 1.5 years)
- Change From Baseline in Heart Rate(Up to 1.5 years)
- Number of Participants with Adverse Events (AEs)(Up to 1.5 years)
- Change from Baseline in Blood Pressure(Up to 1.5 years)
- Change from Baseline in Height(Up to 1.5 years)
- Trough Concentrations of Macitentan and its Active Metabolite Aprocitentan at Week 8 in Steady-State Conditions(Predose (at Week 8))
- Change From Baseline in Length(Up to 1.5 years)
- Number of Participants with AEs Leading to Premature Discontinuation of Macitentan(Up to 1.5 years)
- Change From Baseline in Body Weight(Up to 1.5 years)
- Number of Participants with Adverse Event of Special Interests (AESIs)(Up to 1.5 years)
- Plasma Concentration of Macitentan and its Active Metabolite (Aprocitentan) for Macitentan Naive Participants(At 2, 5, and 24 hours after the first dose of macitentan on Day 1)
研究者
研究点 (4)
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