EUCTR2023-000984-30-Outside-EU/EEA进行中（未招募）1 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
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. Japanese males or females between = 3 months and < 15 years of age at the first administration of study intervention.
- •2. PAH belonging to the Nice 2013 Updated Classification Group 1 (including Down syndrome) and of the following etiologies:
- •a. Idiopathic PAH (iPAH)
- •b. Heritable PAH (hPAH)
- •c. PAH associated with CHD:
- •i. PAH with co-incidental CHD
- •ii. Post-operative PAH (persisting/ recurring/ developing = 6 months after repair of CHD)
- •d. Drug or toxin induced PAH
- •e. PAH associated with HIV
- •f. PAH associated with connective tissue disease (PAH-aCTD)
- •3. PAH diagnosis confirmed by historical right heart catheterization (RHC; characterized by mean pulmonary arterial pressure =25 mm Hg, and pulmonary arterial wedge pressure =15 mm Hg, and pulmonary vascular resistance index (PVRi) >4 Wood Units × m2), where in the absence of pulmonary vein obstruction and/or significant lung disease 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.
- •4. WHO FC I to IV.
- •5. PAH-specific treatment-naïve patients or patients on PAH-specific treatment
- •6. A woman of childbearing potential must have a negative highly sensitive serum -human chorionic gonadotropin test at Screening and a negative urine pregnancy test at the first administration of study intervention.
- •7. A woman must be
- •a. Not of childbearing potential
- •b. Of childbearing potential and
- •o Practicing a highly effective, preferably user-independent method of contraception (failure rate of <1% per year when used consistently and
- •correctly) and agrees to remain on a highly effective method while receiving study intervention and until 30 days after last dose - the end of relevant systemic exposure. The investigator should evaluate the potential for contraceptive method failure (eg, noncompliance, recently initiated) in
- •relationship to the first dose of study intervention.
- •8. A woman must agree not to donate eggs (ova, oocytes) or freeze for future use for the purposes of assisted reproduction during the study and for a period of up to 4 weeks following the EOS.
- •9. Parent(s) (preferably both if available or as per local requirements) (or their legally acceptable representative) must sign an 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. Assent is also required of children capable of understanding the nature of the study (typically 7 years of age and older) as described in Informed Consent
- •Process in Appendix 3: Regulatory, Ethical, and Study Oversight Considerations.
- •10. Willing and able to adhere to the lifestyle restrictions specified in the protocol.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 7
- •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
- •1. Patients with PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease, and/or pulmonary capillary hemangiomatosis, and persistent pulmonary hypertension of the newborn.
- •2. Patients with PAH associated with open shunts, as specified below:
- •a. Eisenmenger syndrome
- •b. Moderate to large left-to-right shunts as judged by the investigator
- •3. Patients with 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 patients with Fontan-palliation
- •4. Patients with pulmonary hypertension due to lung disease (eg, bronchopulmonary dysplasia)
- •5 Patients with the following diseases:
- •a. Patients with pulmonary vein stenosis
- •b. Patients with bronchopulmonary dysplasia
- •6. Hemoglobin or hematocrit <75% of the lower limit of normal range (LLN) at Screening.
- •7. Serum AST and/or ALT >3 × ULN at Screening.
- •8. Severe hepatic impairment, eg, Child-Pugh Class C, at Screening.
- •9. Clinical signs of hypotension which in the investigator’s judgment would preclude initiation of a PAH-specific therapy at Screening.
- •10. Severe renal dysfunction with an estimated Glomerular Filtration Rate (eGFR) <30 mL/min/1.73 m2 at Screening.
- •11. Known concomitant life-threatening disease with a life expectancy <12 months.
- •12. Patients receiving PAH-specific treatments (excluding PDE-5 inhibitor) at the first administration of study intervention.
- •13. Start or change of dose* with PDE-5 inhibitor within 90 days before RHC at Screening
- •(* Dose adjustments are permitted based on patient body-weight change).
- •14. Start or change of dose* of Calcium channel blockers and/or diuretics within 7 days before RHC (* Dose adjustments are permitted based on patient body-weight change).
- •15 Previous treatment* with macitentan at any time.
- •16. Any PAH-related surgical intervention planned, or patients listed for organ transplantation related to PAH.
- •17. Treatment with strong inducers of CYP3A4 (eg, rifabutin, rifampicin, carbamazepine, phenobarbital, phenytoin, St. John’s wort), within 4 weeks prior to the first administration of study intervention.
- •18. Systemic treatment with strong inhibitors of CYP3A4 (eg, clarithromycin, itraconazole, ketoconazole, nelfinavir, posaconazole, ritonavir, and voriconazole) within 4 weeks prior to the first administration of study intervention.
- •19. Systemic treatment with moderate dual CYP3A4/CYP2C9 inhibitor (eg, fluconazole and amiodarone), or administration of a combination of a moderate CYP3A4 inhibitor (eg, ciprofloxacin, cyclosporine, diltiazem, erythromycin, verapamil) together with a moderate CYP2C9 inhibitor (eg, miconazole) within 4 weeks prior to the first administration of study intervention.
- •20. Taken any disallowed therapies as noted in in the protocol before the planned first dose of study intervention.
- •21. Received an investigational intervention (including investigational vaccines) or used an invasive investigational medical device within 4 weeks before the planned first dose of study intervention or is currently enrolled in an investigational study.
- •22. 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.
- •23. Any condition for which, in the opinion of the investigator, participation would not be in t
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