EUCTR2022-002754-74-PL进行中（未招募）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
ACTELION Pharmacteuticals Ltd.0 个研究点目标入组 10 人开始时间: 2022年11月15日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Pediatric participants aged 1 month to <2 years (at Screening).
- •Type of Participant and Disease Characteristic
- •2. Pulmonary arterial hypertension including participants with Down syndrome. Diagnosis must have been confirmed by (historical, any time before screening) right heart catheterization mPAP =25 mmHg, pulmonary arterial wedge pressure (PAWP) =15 mmHg, pulmonary vascular resistance index >3Wood units × m2) 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 <1.5 and saturation of peripheral oxygen =90% measured by pulse oximetry at room air), or
- •ii. Inoperable open left-to-right shunts (with a PVR >8 WU and Qp/Qs <2), or
- •iii. Co-incidental shunt (ie, 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 HIV.
- •3. WHO FC I, II, or III.
- •4. 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.
- •5. Body weight of =3.5 kg.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 10
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Medical Conditions
- •1. PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease and/or pulmonary capillary hemangiomatosis.
- •2. Persistent pulmonary hypertension of the newborn.
- •3. 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.
- •4. Pulmonary hypertension due to lung disease.
- •5. Known diagnosis of bronchopulmonary dysplasia.
- •6. Pulmonary vein stenosis.
- •7. Known concomitant life-threatening disease with life expectancy <12 months.
- •8. Alanine transaminase and/or AST >3 ×ULN.
- •9. Severe hepatic impairment, eg, Child-Pugh Class C.
- •10. Hemoglobin or hematocrit <75% of the lower limit of normal range (for age).
- •11. Clinical signs of hypotension which in the investigator’s judgment would preclude initiation of a PAH-specific therapy.
- •12. Severe renal insufficiency (estimated creatinine clearance <30 mL/min or serum creatinine >221 µmol/L).
- •13. Known allergies, hypersensitivity, or intolerance to ERAs or macitentan or its excipients.
- •14. Known hereditary fructose intolerance.
- •Prior/Concomitant Therapy
- •15. Triple combination therapy with PAH-specific treatments.
- •16. Treatment with intravenous or subcutaneous prostanoids within 4 weeks before Visit 2 (Day 1) unless given for vasoreactive testing.
- •17. Any PAH-related surgical intervention planned, or participants listed for organ transplantation related to PAH.
- •18. Treatment with strong inducers of CYP3A4 such as rifabutin, rifampicin, rifapentin, carbamazepine, phenobarbital, phenytoin, St. John’s wort (hypericum perforatum), within 4 weeks prior to Visit 2 (Day 1).
- •19. Systemic treatment with strong inhibitors of CYP3A4 such as boceprevir, clarithromycin, conivaptan, indinavir, itraconazole, ketoconazole, nefazodone, nelfinavir, posaconazole, ritonavir, saquinavir, telaprevir, telithromycin, and voriconazole within 4 weeks prior to Visit 2 (Day 1).
- •20. Systemic treatment with moderate dual CYP3A4/CYP2C9 inhibitor (eg, fluconazole and amiodarone), or administration of a combination of a moderate CYP3A4 (eg, ciprofloxacin, cyclosporine, diltiazem, erythromycin, verapamil) together with a moderate CYP2C9 inhibitor (eg, miconazole, piperine) within 4 weeks prior to Visit 2 (Day 1).
- •21. Switching PAH treatment from an ERA (eg, bosentan, ambrisentan) to macitentan when a participant’s clinical condition is unstable or undergoing continuous deterioration.
- •Prior/Concurrent Clinical Study Experience
- •22. Received an investigational intervention (including investigational vaccines) or used an invasive investigational medical device within 4 weeks prior to Visit 2 (Day 1) or is currently enrolled in an investigational study.
研究者
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