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Selexipag 作为儿童肺动脉高压护理标准附加治疗的研究 (SALTO)

2026年8月27日 更新者：Actelion

一项随机、多中心、双盲、安慰剂对照、平行组、事件驱动、组序贯研究，具有开放标签延长期，以评估 Selexipag 作为儿童护理标准附加治疗的疗效和安全性年龄 >=2 至

本研究的目的是评估与安慰剂相比，将 selexipag 添加到标准护理治疗中是否能延缓患有肺动脉高压 (PAH) 的儿童的疾病进展。

研究概览

地位

主动，不招人

条件

干预/治疗

详细说明

儿科 PAH 是一种罕见的进行性疾病，具有相当高的发病率和死亡率。 鉴于开发 PAH 儿童治疗的重大医疗需求，因此需要在儿科人群中进行进一步的临床研究，以便为儿童 PAH 的管理提供更多数据。 Selexipag (JNJ-67896049) 是一种口服的、选择性的、长效的前列环素受体的非前列腺素类激动剂，已获批用于治疗患有 PAH 的成年参与者，并且已上市销售。 Selexipag 及其代谢物具有抗纤维化、抗增殖和抗血栓形成的特性。 目前，没有靶向前列环素途径的药物被批准用于治疗 PAH 的儿科。 作用于前列环素受体的有效且可口服的疗法，例如在 PAH 疾病的医学适当阶段引入的 selexipag，并且主要与当前一线口服 PAH 特异性药物联合用于因疾病控制不足而需要额外治疗的参与者代表了 PAH 儿科参与者治疗管理的重大进步。 该研究包括长达 6 周的筛选期和双盲治疗期，包括滴定和维持期，随后是 3 年的开放标签延长期 (OLEP) 和 30 天的安全随访期最后一次研究干预（双盲或开放标签）后出现的上升期。 安全性、药代动力学和疗效评估将在研究期间进行。 将成立独立数据监测委员会 (IDMC) 以持续监测数据、审查中期数据并确保参加本研究的参与者的持续安全。 研究的大约持续时间为8年。

研究类型

介入性

注册 (实际的)

138

阶段

  • 第三阶段

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习地点

  • 中国
      • Beijing、中国、100029
        • Beijing Anzhen Hospital
      • Guangzhou、中国、510623
        • Guangzhou Women and Children's Medical Center
      • Qingdao、中国、266000
        • Qingdao Women and Children's Hospital
      • Qingdao、中国、266000
        • Qingdao Women and Children's Hospital 1
      • Shanghai、中国、201102
        • Children's Hospital of Fudan University
      • Shanghai、中国、200127
        • Shanghai Childrens Medical Center
      • Shenyang、中国、110000
        • The General Hospital of Northern Theater Command
  • 乌克兰
      • Dnipro、乌克兰、49006
        • Dnipropetrovsk clinical medical center of Mother and Child after prof. Rudnev
      • Dnipro、乌克兰、49070
        • MI 'Dnipropetrovsk Regional Clinical Center of Cardiology and Cardiac Surgery'
      • Kyiv、乌克兰、04050
        • Scientific Practical Medical Center for Pediatric Cardiology and Cardio Surgery of the MOH
      • Zaporizhzhya、乌克兰、69063
        • MI Zaporizhzhia Regional Clinical Childrens Hospital of Zaporizhzhia Regional Council
  • 以色列
      • Haifa、以色列、3109601
        • Rambam Medical Center
      • Ramat Gan、以色列、52621
        • Sheba medical center
  • 俄罗斯
      • Kazan'、俄罗斯、420012
        • Kazan State Medical University
      • Kazan'、俄罗斯、420059
        • Kazan State Medical University 1
      • Kemerovo、俄罗斯、650002
        • Scientific and Research Institution of Cardiovascular Diseases Complex Problems
      • Moscow、俄罗斯、125373
        • Childrens City Clinical Hospital n.a. Bashlyaeva
      • Moscow、俄罗斯、125412
        • Veltischev Research and Clinical Institute for Pediatrics of the Pirogov RNRMU
      • Samara、俄罗斯、443070
        • Samara Regional Clinical Cardiological Dispensary
  • 保加利亚
      • Sofia、保加利亚、1309
        • Multiprofile Hospital For Active Treatment National Cardiology Hospital, Ead
  • 加拿大
    • Alberta
      • Edmonton、Alberta、加拿大、T6G 2B7
        • Stollery Children's Hospital
    • Ontario
      • Toronto、Ontario、加拿大、M5G 1X8
        • Hospital for Sick Children
  • 匈牙利
      • Budapest、匈牙利、1096
        • Gottsegen György Országos Kardiológiai Intézet
  • 台湾
      • Kaohsiung City、台湾、813414
        • Kaohsiung Veterans General Hospital
      • Tainan、台湾、704
        • National Cheng Kung University Hospital
      • Taipei、台湾、100
        • National Taiwan University Hospital
  • 哥伦比亚
      • Bogotá、哥伦比亚
        • Fundación Santa Fe de Bogotá
      • Bogotá、哥伦比亚、0000000
        • Clinica San Rafael
      • Bogotá、哥伦比亚、0000000
        • Fundacion Neumologica Colombiana
      • Cali、哥伦比亚、760042
        • Clínica Imbanaco S.A.S.
      • Piedecuesta、哥伦比亚、681017
        • Fundacion cardiovascular de Colombia
      • Soledad、哥伦比亚、0000000
        • Hospital Universidad del Norte
  • 土耳其（türkiye）
      • Adana、土耳其（türkiye）、01790
        • Cukurova Balcali Hospital Application and Research Center
      • Ankara、土耳其（türkiye）、06230
        • Hacettepe University Medical Faculty
      • Istanbul、土耳其（türkiye）、34093
        • CAPA Istanbul University Medical Faculty
      • Istanbul、土耳其（türkiye）、34303
        • Mehmet Akif Ersoy Training and Research Hospital
      • Izmir、土耳其（türkiye）、35020
        • Izmir Tepecik Training and Research Hospital
      • Izmir、土耳其（türkiye）、35210
        • Behcet Uz Pediatric Diseases and Surgery Training and Research Hospital
  • 塞尔维亚
      • Belgrade、塞尔维亚、11000
        • Univerzitetska Dečja Klinika
  • 墨西哥
      • Guadalajara、墨西哥、44160
        • CICUM San Miguel
      • Monterrey、墨西哥、64718
        • Unidad de Investigacion Clinica en Medicina S.C. (UDICEM)
      • México、墨西哥、52787
        • Operadora de Hospitales Angeles SA de CV Hospital Angeles Lomas
  • 巴西
      • Blumenau、巴西、89030-101
        • Complexo de Prevencao,Diagnostico,Terapia e Reabilitacao Respiratoria LTDA Hospital Dia do Pulmao
      • Brasília、巴西、70310-500
        • Fundacao Universitaria de Cardiologia - Instituto de Cardiologia e Transplantes do DF
      • Curitiba、巴西、80250-060
        • Hospital Pequeno Principe
      • Fortaleza、巴西、60840-285
        • Secretaria da Saude do Estado do Ceara - Hospital Doutor Carlos Alberto Studart Gomes
      • Porto Alegre、巴西、90020-090
        • Irmandade Santa Casa de Misericordia de Porto Alegre
      • Porto Alegre、巴西、90620-001
        • Fundação Universitaria de Cardiologia
      • São Paulo、巴西、01221-020
        • Irmandade Santa Casa de Misericordia de Sao Paulo
      • São Paulo、巴西、04024 002
        • SPDM - Associacao Paulista para o Desenvolvimento da Medicina - Hospital Sao Paulo
  • 德国
      • Freiburg im Breisgau、德国、70106
        • Universitätsklinikum Freiburg Zentrum
      • Heidelberg、德国、D-69120
        • Universitaetsklinikum Heidelberg
      • Leipzig、德国、04289
        • Herzzentrum Leipzig GmbH
      • München、德国、81377
        • Klinikum der Universitaet Muenchen
  • 意大利
      • Bologna、意大利、40138
        • Azienda Ospedaliera Policlinico S. Orsola-Malpighi
      • Milan、意大利、20162
        • ASST Grande Ospedale Metropolitano Niguarda
      • Padova、意大利
        • Universta Degli Studi Di Padova
      • Roma、意大利、00193
        • Ospedale Pediatrico Bambin Gesù
      • S. Donato Milanese、意大利、20097
        • IRCCS Policlinico San Donato
      • Torino、意大利、10126
        • AOU Città della Salute e della Scienza di Torino, Presidio Ospedale Infantile Regina Margherita
  • 比利时
      • Brussels、比利时、1070
        • ULB Hôpital Erasme
      • Ghent、比利时、9000
        • Universitair Ziekenhuis Gent
      • Leuven、比利时、3000
        • Universitaire Ziekenhuizen Leuven
  • 法国
      • Lille、法国、59037
        • Hôpital Cardiologique - Chru Lille
      • Marseille、法国、13385
        • Hôpital de la Timone
      • Montpellier、法国、34295
        • CHU Arnaud de Villeneuve
      • Paris、法国、75015
        • Hopital Necker - Enfants Malades
      • Pessac、法国、33604
        • Hôpital Cardiologique Du Haut-Lévêque
      • Toulouse、法国、31059
        • Chu Hopital Des Enfants
  • 波兰
      • Gdansk、波兰、80 952
        • Uniwersyteckie Centrum Kliniczne
      • Krakow、波兰、30-663
        • Uniwersytecki Szpital Dzieciecy w Krakowie
      • Poznan、波兰、60 572
        • Szpital Kliniczny im Karola Jonschera
      • Warsaw、波兰、04-730
        • Instytut Pomnik Centrum Zdrowia Dziecka
      • Wroclaw、波兰、51 124
        • Wojewodzki Szpital Specjalistyczny We Wroclawiu
      • Zabrze、波兰、41-800
        • Slaskie Centrum Chorob Serca
  • 泰国
      • Chiang Mai、泰国、50200
        • Chiang Mai University Hospital
      • Songkhla、泰国、90110
        • Songklanagarind Hospital
  • 澳大利亚
      • South Brisbane、澳大利亚、4101
        • Queensland Children's Hospital
  • 爱尔兰
      • Dublin、爱尔兰
        • Our Lady's Children's Hospital
  • 瑞典
      • Gothenburg、瑞典、416 50
        • Drottning Silvias barn- och ungdomssjukhus
      • Lund、瑞典、222 42
        • Skånes universitetssjukhus
  • 瑞士
      • Lausanne、瑞士、1011
        • Centre hospitalier universitaire vaudois CHUV
  • 白俄罗斯
      • Minsk、白俄罗斯、220013
        • State Institution Republican Scientific And Practical Center For Pediatric Surgery
      • Minsk、白俄罗斯、220118
        • Health Institution 4Th City Children'S Clinical Hospital
  • 立陶宛
      • Vilnius、立陶宛、LT08661
        • Vilnius University Hospital Santariskiu Clinics
  • 美国
    • Arizona
      • Phoenix、Arizona、美国、85016
        • Phoenix Children's Hospital
    • California
      • Los Angeles、California、美国、90095
        • UCLA Medical Center
      • San Francisco、California、美国、94158
        • UCSF
    • Colorado
      • Aurora、Colorado、美国、80045
        • Childrens Hospital Colorado
    • District of Columbia
      • Washington D.C.、District of Columbia、美国、20010
        • Children's National Medical Center
    • Florida
      • Gainesville、Florida、美国、32610
        • Congenital Heart Center of the University of Florida
    • Indiana
      • Indianapolis、Indiana、美国、46202
        • Riley Hospital for Children
    • Michigan
      • Detroit、Michigan、美国、48201
        • Detroit Medical Center
    • Ohio
      • Cincinnati、Ohio、美国、45229
        • Cincinnati Children's Hospital Medical Center
    • Pennsylvania
      • Philadelphia、Pennsylvania、美国、19104
        • Childrens Hospital of Philadelphia
    • Texas
      • Houston、Texas、美国、77030
        • Texas Children's Hospital
    • Utah
      • Salt Lake City、Utah、美国、84113
        • Primary Children's Hospital
    • Virginia
      • Charlottesville、Virginia、美国、22908
        • University of Virginia Division of Pediatric Cardiology
  • 芬兰
      • Helsinki、芬兰、29
        • New Children's Hospital of the Helsinki University Hospital (HUS)
  • 葡萄牙
      • Lisbon、葡萄牙、1169-024
        • Uls Sao Jose - Hosp. Santa Marta
      • Porto、葡萄牙、4200 319
        • Uls Sao Joao - Hosp. Sao Joao
  • 西班牙
      • A Coruña、西班牙、15006
        • Hosp Univ A Coruna
      • Barcelona、西班牙、08035
        • Hosp Univ Vall D Hebron
      • Esplugues de Llobregat、西班牙、08950
        • Hosp. Sant Joan de Deu
      • Madrid、西班牙、28046
        • Hosp. Univ. La Paz
      • Madrid、西班牙、28009
        • Hosp. Gral. Univ. Gregorio Maranon
      • Seville、西班牙、41013
        • Hosp. Virgen Del Rocio
  • 越南
      • Hanoi、越南
        • Hanoi Medical University Hospital
      • Ho Chi Minh City、越南、700000
        • University Medical Center Ho Chi Minh City
      • Ho Chi Minh City、越南
        • Children's Hospital 1
      • Ho Chi Minh City、越南、700000
        • Tam Anh Hospital
  • 韩国
      • Seoul、韩国、03080
        • Seoul National University Hospital
      • Seoul、韩国、06351
        • Samsung Medical Center
      • Seoul、韩国、120-752
        • Severance Hospital Yonsei University Health System
      • Yangsan、韩国、50612
        • Pusan National University Yangsan Hospital
  • 马来西亚
      • Kuala Lumpur、马来西亚、50400
        • National Heart Institute

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

2年 至 17年 (孩子)

接受健康志愿者

不

描述

纳入标准：

  • 随机分配时体重大于或等于 (>=) 2 岁且小于 (<) 18 岁的参与者体重 >=9 千克 (kg)
  • 肺动脉高压 (PAH) 诊断通过在参与者筛选前的任何时间进行的有记录的历史性右心导管插入术 (RHC) 得到确认
  • PAH（世界卫生组织 [WHO] 第 1 组），包括具有以下病因的唐氏综合症参与者：特发性 PAH (IPAH)；遗传性多环芳烃 (HPAH)； PAH 与先天性心脏病相关（PAH-associated with congenital heart disease [aCHD]） PVR 升高]，如果得到 BCAC 的批准）和术后 PAH（CHD 修复后持续/复发/发展 >=6 个月）；药物或毒素引起的；与人类免疫缺陷病毒 (HIV) 相关的多环芳烃
  • 世界卫生组织功能等级 (FC) II 和 III
  • 接受过至少一种 PAH 特异性治疗的参与者，例如内皮素受体拮抗剂 (ERA) 和/或 5 型磷酸二酯酶 (PDE-5) 抑制剂/可溶性鸟苷酸环化酶刺激剂，前提是治疗剂量已达到在第一次研究干预之前稳定至少 3 个月

排除标准：

  • 门静脉高压症、血吸虫病、肺静脉闭塞性疾病和/或肺毛细血管瘤病引起的肺动脉高压
  • PAH 与艾森曼格综合征相关
  • 之前接触过 Uptravi (selexipag)
  • 已知伴随的危及生命的疾病，预期寿命<12个月
  • 怀孕、计划怀孕或哺乳期
  • 已知对 selexipag 或其赋形剂过敏、过敏或不耐受

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：随机化
  • 介入模型：并行分配
  • 屏蔽：双倍的

武器和干预

参与者组/臂
干预/治疗
实验性的：司来帕格
参与者将在第1天根据体重接受司来帕格，此后继续每日两次给药。 在最初12周内，将逐步上调司来帕格的剂量，直至参与者达到个体最大耐受剂量（iMTD）或达到其基线体重类别对应的最大剂量。 剂量上调后，从第12周至治疗结束（EOT）为维持期，参与者将维持在最大耐受剂量。 参与者将继续根据当地标准治疗，接受肺动脉高压（PAH）特异性联合疗法，如内皮素受体拮抗剂、磷酸二酯酶-5抑制剂和可溶性鸟苷酸环化酶刺激剂。
药品：Selexipag
Selexipag 片剂将口服给药。
其他名称：
  • JNJ-67896049
药品：标准护理（SOC）：内皮素受体拮抗剂
ERA将作为标准治疗方案使用。
药品：SOC：磷酸二酯酶5型（PDE-5）抑制剂
PDE-5抑制剂将作为标准治疗方案给药。
药品：SOC：可溶性鸟苷酸环化酶刺激剂
可溶性鸟苷酸环化酶刺激剂将作为标准治疗进行给药。
安慰剂比较：安慰剂
参与者将在第1天根据体重接受匹配的安慰剂，并在此后继续每日两次给药。 参与者将继续接受肺动脉高压特异性伴随治疗，如内皮素受体拮抗剂、磷酸二酯酶-5抑制剂和可溶性鸟苷酸环化酶刺激剂，具体根据当地标准医疗实践。
药品：安慰剂
将口服匹配的安慰剂药片。
药品：标准护理（SOC）：内皮素受体拮抗剂
ERA将作为标准治疗方案使用。
药品：SOC：磷酸二酯酶5型（PDE-5）抑制剂
PDE-5抑制剂将作为标准治疗方案给药。
药品：SOC：可溶性鸟苷酸环化酶刺激剂
可溶性鸟苷酸环化酶刺激剂将作为标准治疗进行给药。
实验性的：开放标签扩展期：司来帕格
对于肺动脉高压（PAH）且selexipag获益/风险比为正的参与者，将在开放标签延长期提供selexipag治疗。 在双盲治疗期间使用selexipag的参与者，将在开放标签延长期继续按其个体化最大耐受剂量（iMTD）进行治疗；对于先前使用安慰剂的参与者，将在前12周内逐步上调selexipag剂量，直至达到其个体化最大耐受剂量（iMTD）。 参与者将继续根据当地标准治疗，接受肺动脉高压特异性联合治疗，如内皮素受体拮抗剂（ERAs）、磷酸二酯酶-5抑制剂（PDE-5抑制剂）和可溶性鸟苷酸环化酶刺激剂。
药品：Selexipag
Selexipag 片剂将口服给药。
其他名称：
  • JNJ-67896049
药品：标准护理（SOC）：内皮素受体拮抗剂
ERA将作为标准治疗方案使用。
药品：SOC：磷酸二酯酶5型（PDE-5）抑制剂
PDE-5抑制剂将作为标准治疗方案给药。
药品：SOC：可溶性鸟苷酸环化酶刺激剂
可溶性鸟苷酸环化酶刺激剂将作为标准治疗进行给药。

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Double-blind Period: Time to Disease Progression
大体时间：Placebo arm: Randomization (Day 1) up to 7 days after last dose in the DB treatment (maximum up to 199 weeks); Selexipag arm: Randomization (Day 1) up to 7 days after last dose in the DB treatment (maximum up to 213 weeks)
Time to disease progression: time from randomization to first Clinical Event Committee (CEC)-confirmed disease progression event occurring between date of randomization until double-blind end date (DBED) + 7 days. Disease progression event as adjudicated by CEC, defined as any of the following: death (all causes); atrial septostomy or Potts' anastomosis, or registration on lung transplant list; hospitalization due to worsening pulmonary arterial hypertension (PAH); clinical worsening of PAH. Clinical worsening of PAH was defined as initiation of new PAH-specific therapy or intravenous diuretics or continuous oxygen use and at least 1 of the following: worsening in World Health Organization (WHO) functional class (FC); new occurrence or worsening: of syncope, of at least 2 PAH symptoms (shortness of breath/dyspnea, chest pain, cyanosis, dizziness/near syncope, or fatigue), of signs of right heart failure not responding to oral diuretics. Kaplan-Meier method was used for estimation.
Placebo arm: Randomization (Day 1) up to 7 days after last dose in the DB treatment (maximum up to 199 weeks); Selexipag arm: Randomization (Day 1) up to 7 days after last dose in the DB treatment (maximum up to 213 weeks)

次要结果测量

结果测量
措施说明
大体时间
Double-blind Period: Change in log2 N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) From Baseline to Week 24
大体时间：Baseline (Day 1), Week 24
Change in log2 NT-proBNP from baseline to Week 24 was reported. Change from baseline to Week 24 in the central laboratory NT-proBNP (nanograms per liter [ng/L]) value, expressed as the log2-transformed ratio of the Week 24 NT-proBNP value over the baseline value: log2 (Week 24 NT-proBNP divided by Baseline NT-proBNP). Clinically, a reduction from baseline in NT-proBNP (ratio < 1) was considered an improvement. The baseline value was the last non-missing assessment obtained before or on the DB start date (DBSD).
Baseline (Day 1), Week 24
Double-blind Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious AEs (TESAEs)
大体时间：Placebo arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 198.42 weeks); Selexipag arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 212.42 weeks)
Number of participants with TEAEs (included serious and non-serious events) and TESAEs during DB period was reported. An AE was any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention. A serious AE was defined as any untoward medical occurrence that at any dose resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a suspected transmission of any infectious agent via a medicinal product, or was medically important. TEAEs were defined as any AE occurring on or after the initial administration of study intervention through the day of last dose in the DB treatment period plus 3 days and/or prior to start of OL study administration.
Placebo arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 198.42 weeks); Selexipag arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 212.42 weeks)
Double-blind Period: Number of Participants With AEs Leading to Premature Discontinuation of Study Treatment
大体时间：Placebo arm: Start of treatment (Day 1) up to last dose in the DB treatment (maximum up to 198 weeks); Selexipag arm: Start of treatment (Day 1) up to last dose in the DB treatment (maximum up to 212 weeks)
Number of participants with AEs leading to premature discontinuation of study treatment during DB period was reported. An AE was any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention.
Placebo arm: Start of treatment (Day 1) up to last dose in the DB treatment (maximum up to 198 weeks); Selexipag arm: Start of treatment (Day 1) up to last dose in the DB treatment (maximum up to 212 weeks)
Double-blind Period: Change From Baseline in Vital Signs: Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)
大体时间：Baseline (Day 1), Weeks 24, 48, 72, and 96
Change from baseline in vital signs parameters: systolic and diastolic blood pressure during DB period was reported. Vital signs were measured after the participant has rested at least 5 minutes in sitting position. The baseline value was the last non-missing assessment obtained before or on the DBSD.
Baseline (Day 1), Weeks 24, 48, 72, and 96
Double-blind Period: Change From Baseline in Vital Signs: Pulse Rate
大体时间：Baseline (Day 1), Weeks 24, 48, 72, and 96
Change from baseline in vital signs parameter: pulse rate during DB period was reported. Vital signs were measured after the participant has rested for at least 5 minutes in sitting position. The baseline value was the last non-missing assessment obtained before or on the DBSD.
Baseline (Day 1), Weeks 24, 48, 72, and 96
Double-blind Period: Change From Baseline in Growth Parameter: Body Weight
大体时间：Baseline (Day 1), Weeks 24, 48, 72, and 96
Change from baseline in growth parameter: body weight during DB period was reported. The baseline value was the last non-missing assessment obtained before or on the DBSD.
Baseline (Day 1), Weeks 24, 48, 72, and 96
Double-blind Period: Change From Baseline in Growth Parameter: Height
大体时间：Baseline (Day 1), Weeks 24, 48, 72, and 96
Change from baseline in growth parameter: height during DB period was reported. The baseline value was the last non-missing assessment obtained before or on the DBSD.
Baseline (Day 1), Weeks 24, 48, 72, and 96
Double-blind Period: Number of Participants With Shift From Baseline in Sexual Maturation (Tanner Stage)
大体时间：Placebo arm: Baseline (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 198.42 weeks); Selexipag arm: Baseline (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 212.42 weeks)
Tanner staging were assessed for pubic hair growth and genitalia development (GD) in males (M) and for pubic hair growth and breast development (BD) in females (F) in stages(S) 1 to 5 and missing data. If a participant had reached Tanner stage 5, no further tanner pubertal stage assessment were to be completed. Tanner stage was assessed in F >=8 years; M >=9 years. Pubic hair growth (M and F) stages: S1: No hair, S2: Downy hair, S3: More coarse and curly hair, S4: Adult-like hair quality; S5: Hair extends to medial surface of thighs. BD in females: S1: Nipple raised a little, rest of breast still flat, S2: Breast bud forms, S3: More elevated, outside areola, S4: Increased breast size, S5: Final adult-size breasts. GD in males: S1: Testes, scrotum, and penis about same size, S2: Enlargement of scrotum, testes, and penis, S3: Enlargement of penis, S4: Penis and glands became larger, S5: Genitalia size and shape same an adult male. Categories with at least 1 non-zero data are reported.
Placebo arm: Baseline (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 198.42 weeks); Selexipag arm: Baseline (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 212.42 weeks)
Double-blind Period: Number of Participants With Treatment-emergent Electrocardiogram (ECG) Abnormalities
大体时间：Placebo arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 198.42 weeks); Selexipag arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 212.42 weeks)
Number of participants with treatment-emergent ECG abnormalities were reported. Any abnormality occurring on or after the initial administration of study intervention through the day of last dose in the DB treatment period plus 3 days was considered to be treatment-emergent. Abnormalities that were not present at baseline were reported. Abnormalities were assessed as per investigator's discretion. The baseline value was the last non-missing assessment obtained before or on the DBSD.
Placebo arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 198.42 weeks); Selexipag arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 212.42 weeks)
Double-blind Period: Number of Participants With Treatment-emergent (TE) Marked Laboratory Abnormalities
大体时间：Placebo arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 198.42 weeks); Selexipag arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 212.42 weeks)
Number of participants with TE marked laboratory abnormalities during DB period was reported. TE marked laboratory abnormalities included hemoglobin, platelet count, leukocyte count, potassium, calcium, thyrotropin, thyroxine free and triiodothyronine free. Any abnormality occurring or worsening at or after the DB study treatment start date through the DB study treatment end date plus 3 days was considered to be treatment-emergent. If the abnormality was present at baseline, then any post-baseline abnormality was considered treatment-emergent only if the post-baseline abnormality was in a category worse than at baseline. HH, HHH, HHHH = marked abnormally high values; LL, LLL = marked abnormally low values. The baseline value was the last non-missing assessment obtained before or on the DBSD.
Placebo arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 198.42 weeks); Selexipag arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 212.42 weeks)
Double-blind Period: Change From Baseline in Treatment-emergent Laboratory Parameter: Thyroid Stimulating Hormone
大体时间：Baseline (Day 1), Weeks 24, 48, 72, and 96
Change from baseline in treatment-emergent laboratory parameter: thyroid stimulating hormone during DB period was reported. Any abnormality occurring or worsening at or after the DB study treatment start date through the DB study treatment end date plus 3 days was considered to be treatment-emergent. The baseline value was the last non-missing assessment obtained before or on the DBSD.
Baseline (Day 1), Weeks 24, 48, 72, and 96
Double-blind Period: Change From Baseline in Treatment-emergent Laboratory Parameter: Hemoglobin
大体时间：Baseline (Day 1), Weeks 24, 48, 72, and 96
Change from baseline in treatment-emergent laboratory parameter: hemoglobin during DB period was reported. Any abnormality occurring or worsening at or after the DB study treatment start date through the DB study treatment end date plus 3 days was considered to be treatment-emergent. The baseline value was the last non-missing assessment obtained before or on the DBSD.
Baseline (Day 1), Weeks 24, 48, 72, and 96
Double-blind Period: Change From Baseline in Treatment-emergent Laboratory Parameter: Platelets, Leukocytes
大体时间：Baseline (Day 1), Weeks 24, 48, 72, and 96
Change from baseline in treatment-emergent laboratory parameter: platelets, leukocytes during DB period was reported. Any abnormality occurring or worsening at or after the DB study treatment start date through the DB study treatment end date plus 3 days was considered to be treatment-emergent. The baseline value was the last non-missing assessment obtained before or on the DBSD.
Baseline (Day 1), Weeks 24, 48, 72, and 96
Double-blind Period: Change From Baseline in Treatment-emergent Laboratory Parameter: Electrolytes (Sodium, Potassium, Calcium, Bicarbonate and Chloride)
大体时间：Baseline (Day 1), Weeks 24, 48, 72, and 96
Change from baseline in treatment-emergent laboratory parameter: electrolytes (sodium, potassium, calcium, bicarbonate and chloride) during DB period was reported. Any abnormality occurring or worsening at or after the DB study treatment start date through the DB study treatment end date plus 3 days was considered to be treatment-emergent. The baseline value was the last non-missing assessment obtained before or on the DBSD.
Baseline (Day 1), Weeks 24, 48, 72, and 96
Double-blind Period: Change From Baseline in Treatment-emergent Laboratory Parameter: Transaminases (Alanine Aminotransferase [ALT] and Aspartate Aminotransferase [AST])
大体时间：Baseline (Day 1), Weeks 24, 48, 72, and 96
Change from baseline in treatment-emergent laboratory parameter: transaminases (ALT and AST) during DB period was reported. Any abnormality occurring or worsening at or after the DB study treatment start date through the DB study treatment end date plus 3 days was considered to be treatment-emergent. The baseline value was the last non-missing assessment obtained before or on the DBSD.
Baseline (Day 1), Weeks 24, 48, 72, and 96
Double-blind Period: Change From Baseline in Treatment-emergent Laboratory Parameter: Bilirubin
大体时间：Baseline (Day 1), Weeks 24, 48, 72, and 96
Change from baseline in treatment-emergent laboratory parameter: bilirubin during DB period was reported. Any abnormality occurring or worsening at or after the DB study treatment start date through the DB study treatment end date plus 3 days was considered to be treatment-emergent. The baseline value was the last non-missing assessment obtained before or on the DBSD.
Baseline (Day 1), Weeks 24, 48, 72, and 96
Double-blind Period: Time to First Clinical Event Committee (CEC)-Confirmed Hospitalization or Death for Pulmonary Arterial Hypertension (PAH)
大体时间：Placebo arm: Randomization (Day 1) up to 7 days after last dose in the DB treatment (maximum up to 199 weeks); Selexipag arm: Randomization (Day 1) up to 7 days after last dose in the DB treatment (maximum up to 213 weeks)
Time to first CEC-confirmed hospitalization or death for PAH was calculated as the onset date of the first CEC-confirmed death or hospitalization due to PAH minus date of randomization+1 day. Kaplan-Meier method was used for estimation.
Placebo arm: Randomization (Day 1) up to 7 days after last dose in the DB treatment (maximum up to 199 weeks); Selexipag arm: Randomization (Day 1) up to 7 days after last dose in the DB treatment (maximum up to 213 weeks)
Double-blind Period: Dose-normalized Steady-state Trough Concentrations (Ctrough,ss,dn) of Selexipag and Its Metabolite ACT-333679 by Age Cohort
大体时间：Cohort (C) 1: Predose between Week (W) 16 to end of DB treatment period (maximum up to W212); C 2: Predose between Week 16 to end of DB treatment period (maximum up to W198); C 3: Predose between Week 16 to end of DB treatment period (maximum up to W96)
Dose-normalized trough plasma concentration at steady-state (Ctrough,ss,dn) was defined as the plasma concentration just prior to the morning dose, with the last study intervention administration one day prior to the PK sampling. Steady-state was considered achieved if the participant has received a stable dose for at least 3 days prior to collection of the plasma sample. PK samples were collected at two protocol-specified visits per participant occurring between Week 16 and end of DB treatment period. Exact visit timing varied by participant.
Cohort (C) 1: Predose between Week (W) 16 to end of DB treatment period (maximum up to W212); C 2: Predose between Week 16 to end of DB treatment period (maximum up to W198); C 3: Predose between Week 16 to end of DB treatment period (maximum up to W96)
Double-blind Period: Dose-normalized Steady-state Trough Concentrations (Ctrough,ss,dn) of Selexipag and Its Metabolite ACT-333679 by Body Weight (BW)
大体时间：BW >=50kg: Predose between W16 to end of DB treatment period (maximum up to W212); BW >=25-<50kg: Predose between W16 to end of DB treatment period (maximum up to W198); BW >=9- <25kg: Predose between W16 to end of DB treatment period (maximum up to W175)
Dose-normalized steady-state trough concentration (Ctrough,ss,dn) was defined as the plasma concentration just prior to the morning dose, with the last study intervention administration one day prior to the PK sampling. Steady-state was considered achieved if the participant has received a stable dose for at least 3 days prior to collection of the plasma sample. PK samples were collected at two protocol-specified visits per participant occurring between Week 16 and end of DB treatment period. Exact visit timing varied by participant.
BW >=50kg: Predose between W16 to end of DB treatment period (maximum up to W212); BW >=25-<50kg: Predose between W16 to end of DB treatment period (maximum up to W198); BW >=9- <25kg: Predose between W16 to end of DB treatment period (maximum up to W175)

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赞助

Actelion

调查人员

  • 研究主任：Actelion Clinical Trial、Actelion

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2020年1月16日

初级完成 (实际的)

2024年10月11日

研究完成 (估计的)

2027年10月1日

研究注册日期

首次提交

2019年11月8日

首先提交符合 QC 标准的

2019年11月21日

首次发布 (实际的)

2019年11月25日

研究记录更新

最后更新发布 (实际的)

2026年8月28日

上次提交的符合 QC 标准的更新

2026年8月27日

最后验证

2026年8月1日

更多信息

与本研究相关的术语

其他相关的 MeSH 术语

其他研究编号

  • CR108716
  • 2019 (美国 NIH 拨款/合同：Chief Medical Office (CMO) Alberta Health Services)
  • 2019-002817-21 (EudraCT编号)
  • AC-065A310 (其他标识符：Janssen Research & Development, LLC)
  • 2022-501012-34-00 (注册表标识符：EUCT number)

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

是的

IPD 计划说明

强生旗下杨森制药公司的数据共享政策可在 www.janssen.com/clinical-trials/transparency 获取。 如本网站所述，可以通过耶鲁开放数据访问 (YODA) 项目网站 yoda.yale.edu 提交访问研究数据的请求

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

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