EUCTR2018-000145-39-PL进行中（未招募）1 期
A prospective, multicenter, open-label, single-arm, Phase 2 study to investigate the safety, tolerability and pharmacokinetics of selexipag in children with pulmonary arterial hypertension
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 55
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. Signed and dated informed consent by the parent(s) or Legally authorized representative(s) AND assent from developmentally capable children.
- •2. Males or females between = 2 and < 18 years of age at Baseline / Enrollment / Visit 2 weighing = 9 kg.
- •3. PAH diagnosis confirmed by documented historical right heart catheterization (RHC) performed at any time before the subject’s enrollment
- •4. PAH with one of the following etiologies:
- •idiopathic (iPAH),
- •heritable (hPAH),
- •associated with congenital heart disease (CHD):
- •– PAH with co-incidental CHD
- •– Post-operative PAH (persisting/ recurring/ developing = 6 months after repair of CHD)
- •Drug or toxin-induced PAH
- •PAH associated with HIV
- •PAH associated with connective tissue disease
- •5. Word Health Organizaition functional class (WHO FC) II to III.
- •6. Subjects treated with an endothelin receptor antagonist (ERA) and/or a phosphodiesterase type 5 (PDE-5) inhibitor, provided that the treatment dose(s) has been stable for at least 3 months prior to enrollment, or patients who are not candidates for these therapies.
- •7. Females of childbearing potential must have a negative pregnancy test at Screening and at Enrollment, and must agree to undertake monthly pregnancy tests, and to use a reliable method of contraception (if sexually active) from screening up to study drug discontinuation + 30 days (EOS).
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 55
- •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. Subjects with PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease (PVOD) and/or pulmonary capillary hemangiomatosis.
- •2. Subjects with PAH associated with Eisenmenger syndrome.
- •3. Subjects with moderate to large left-to-right shunts.
- •4. Subjects with cyanotic congenital cardiac lesions such as transposition of the great arteries, truncus arteriosus, univentricular heart, or pulmonary atresia with ventricular septal defect, as well as subjects with Fontan palliation.
- •5. Subjects with PH due to lung disease and/or hypoxia. For subjects with Down syndrome, exclusion of lung disease and hypoxia causing PH should be documented (e.g., computed tomography scan,
- •polysomnography, lung function tests).
- •Treatment and intervention
- •6. Previous treatment with Uptravi® (selexipag) within 2 weeks prior to enrollment.
- •7. Subjects having received prostacyclin (epoprostenol) or prostacyclin analogs2 (i.e., treprostinil, iloprost, beraprost) within 2 months prior to enrollment or who are scheduled to receive any of these compounds during the trial.
- •8. Treatment with another investigational drug within 4 weeks prior to enrollment.
- •9. Treatment with strong and moderate inhibitors of CYP2C8 (e.g., gemfibrozil, clopidogrel, deferasirox, teriflunomide) within 2 weeks prior to enrollment until the last dose of selexipag + 3 days.
- •10. Treatment with inhibitors of UGT1A3 and UGT2B7 (valproic acid, probenecid, and fluconazole) are prohibited from 2 weeks prior to enrollment and until the last dose of selexipag + 3 days.
- •11. Any PAH-related surgical intervention planned, or subjects listed for organ transplantation related to PAH.
- •12. History or current suspicion of intussusception or ileus or gastrointestinal obstruction, per investigator’s judgment.
- •Baseline abnormalities
- •13. Known concomitant life-threatening disease with a life expectancy < 12 months.
- •14. Uncontrolled thyroid disease, per investigator’s judgment.
- •15. Hemoglobin or hematocrit < 75% of the lower limit of normal range.
- •16. Known severe or moderate hepatic impairment, i.e., Child-Pugh Class B or C [see Appendix 4].
- •17. Clinical signs of hypotension that, in the investigator’s judgment, would preclude the initiation of a PAH-specific therapy.
- •18. Subjects with severe renal insufficiency (estimated creatinine clearance < 30 mL/min or serum creatinine > 221 µmol/L).
- •19. Severe coronary heart disease or unstable angina as assessed by the investigator.
- •20. Myocardial infarction within the last 6 months prior to enrollment.
- •21. Decompensated cardiac failure if not under close supervision.
- •22. Severe arrhythmias as assessed by the investigator.
- •23. Cerebrovascular events (e.g., transient ischemic attack, stroke) within the last 3 months prior to enrollment.
- •24. Congenital or acquired valvular defects with clinically relevant myocardial function disorders not related to PH.
- •Pregnancy and breastfeeding
- •25. Pregnancy (including family planning) or breastfeeding.
- •Other categories
- •26. Known hypersensitivity to the investigational treatment or to any of the excipients of the drug formulations.
- •27. Drug or substance abuse, or any condition that, in the opinion of the investigator, may prevent compliance with the protocol or adherence to study treatment.
- •28. Loss of 250 mL or more of blood within 3 months prior to screening.
- •29. History or clinical evidence of any disease and/or existence of any surgical or medical condition that m
研究者
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