DRKS00015024已完成2 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
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 63
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- N/a: Single Arm Study
- 盲法
- Open (masking not used)
入排标准
- 年龄范围
- 2 Years 至 18 Years（—）
- 性别
- All
入选标准
- •1. Signed and dated informed consent by the parent(s) or LAR(s) AND assent from developmentally capable children.
- •2. Males or females between = 2 and < 18 years of age with weight = 9 kg.
- •3. PAH diagnosis confirmed by documented historical RHC performed at any time before subject’s enrollment, and characterized by:
- •mPAP = 25 mmHg,
- •PAWP = 15 mmHg
- •(in the absence of pulmonary vein obstruction and/or significant lung disease PAWP, can be replaced by LAP or, in absence of mitral stenosis, by LVEDP)
- •PVRi > 3 WU × m2
- •4. PAH belonging to Nice 2013 Updated Classification Group 1 (including Down syndrome) and of one of the following etiologies:
- •aPAH-CHD:
- •– PAH with co-incidental CHD
- •– Post-operative PAH (persisting/ recurring/ developing = 6 months after repair of CHD)
- •Drug or toxin-induced
- •PAH associated with HIV
- •PAH-aCTD
- •5. WHO FC II to III.
- •6. Subjects treated with an ERA and/or a 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 plus 30 days (EOS).
排除标准
- •1.Subjects with PAH due to portal hypertension, schistosomiasis, 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 pulmonary hypertension due to lung disease (e.g., bronchopulmonary dysplasia).
- •Treatment and intervention
- •6.Previous treatment with Uptravi (selexipag) within 2 weeks prior to enrollment.
- •7.Subjects having received prostacyclin (epoprostenol) or prostacyclin analogs (i.e., treprostinil, iloprost, beraprost) within 2 months prior to enrollment or 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 as per investigator’s judgment.
- •Baseline abnormalities
- •13.Known concomitant life-threatening disease with a life expectancy < 12 months.
- •14.Uncontrolled thyroid disease as per investigator 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 initiation of a PAH-specific therapy.
- •18.Subjects with severe renal insufficiency (estimated creatinine clearance < 30 mL/min or serum creatinine > 221 µmol/L).
- •Pregnancy and breastfeeding
- •19.Pregnancy (including family planning) or breastfeeding.
- •Other categories
- •20.Known hypersensitivity to the investigational treatment or to any of the excipients of the drug formulations.
- •21.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.
- •22.Loss of 250 mL or more of blood within 3 months prior to screening.
- •23.History or clinical evidence of any disease and/or existence of any surgical or medical condition which might interfere with the absorption, distribution, metabolism or excretion of the study treatment(s) (e.g., cholecystectomy).
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