A multicenter, open-label, randomized, study with single-arm extension period to assess the pharmacokinetics, safety and efficacy of macitentan versus standard of care in children with pulmonary arterial hypertension - TOMORROW: pediaTric use Of Macitentan tO delay disease pRogRessiOn in PAH Worldwide

ACTELION Pharmaceuticals Ltd0 个研究点目标入组 300 人开始时间: 2016年12月21日最近更新: 2022年5月16日

适应症

试验速览

暂无简介。

•1. Signed informed consent by the parent(s) or legally designated representative AND assent from developmentally capable children prior to initiation of any study-mandated procedure.
•2. Males or females between = 1 month and < 18 years of age.
•3. Subjects with body weight = 3.5 kg at randomization.
•4. PAH diagnosis, confirmed by historical RHC (mPAP = 25 mm Hg, and PAWP = 15 mm Hg, and PVRi > 3 WU x m2).
•5. PAH belonging to the Nice 2013 Updated Classification Group 1 (including subjects with Down Syndrome) and of following etiologies:
•PAH associated with CHD
•Drug or toxin-induced PAH
•PAH associated with HIV
•PAH-aCTD
•6. WHO FC I to III.
•7. Females of childbearing potential must have a negative pregnancy test at Screening and at Baseline, and must agree to undertake monthly pregnancy tests, and to use a reliable method of contraception (if sexually active) up to EOS.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 300
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range 0
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range 0

•1. Subjects with PAH due to portal hypertension, schistosomiasis, or with pulmonary veno-occlusive disease and/or pulmonary capillary hemangiomatosis, and persistent pulmonary hypertension of the newborn.
•2. Subjects with PAH associated with Eisenmenger syndrome, or with moderate to large left-to-right shunts.
•3. Subjects with known diagnosis of bronchopulmonary dysplasia.
•4. Subjects receiving a combination of > 2 PAH-specific treatments at randomization.
•5. Treatment with IV or SC prostanoids within 4 weeks before randomization, unless given for vasoreactivity testing.
•6. In children = 2 y.o.: Previous treatment with macitentan at any time.
•7. Systemic treatment with moderate dual CYP3A4/ CYP2C9 inhibitor (e.g. fluconazole and amiodarone), or administration of a combination of a moderate CYP3A4 (e.g. ciprofloxacin, cyclosporine, diltiazem, erythromycin, verapamil) together with a moderate CYP2C9 inhibitor (e.g. miconazole, piperine) within 4 weeks prior to randomization.
•8. Hemoglobin or hematocrit <75% of the lower limit of normal range
•9. Serum AST and/or ALT > 3 times the upper limit of normal range'
•10. Pregnancy (including family planning) or breastfeeding.
•11. Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol

发起方