此页面是自动翻译的，不保证翻译的准确性。请参阅 英文版 对于源文本。

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

2026年11月2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

The purpose of this study is to evaluate whether the addition of selexipag to standard of care treatment delays disease progression in children with Pulmonary Arterial Hypertension (PAH) in comparison to placebo.

研究概览

地位

主动，不招人

条件

干预/治疗

详细说明

Pediatric PAH is a rare and progressive disorder associated with considerable morbidity and mortality. Given the significant medical need to develop treatments in children with PAH, further clinical studies in the pediatric population are therefore needed to provide more data for the management of PAH in children. Selexipag (JNJ-67896049) is an orally available, selective, and long-acting non-prostanoid agonist of the prostacyclin receptor approved and commercially available for the treatment of adult participants with PAH. Selexipag and its metabolite possess anti-fibrotic, anti-proliferative, and anti-thrombotic properties. Currently, no medicines targeting prostacyclin pathway are approved for pediatric use in PAH. An effective and orally available therapy acting on the prostacyclin receptor such as selexipag introduced at medically appropriate stage of PAH disease, and primarily in combination with current first-line oral PAH-specific medicines in participants in need of additional therapy because of insufficient disease control would represents a major advance to the therapeutic management of PAH pediatric participants. This study consists of a screening period of up to 6 weeks and a double-blind treatment period, including up-titration and maintenance periods, followed by a 3-year open-label extension period (OLEP) and a 30-day safety follow-up period that occurs after the last dose of study intervention (either double-blind or open-label). Safety, pharmacokinetic and efficacy assessments will be performed during the study. An Independent Data Monitoring Committee (IDMC) will be established to monitor data on an ongoing basis, to review interim data, and to ensure the continuing safety of the participants enrolled in this study. The approximate duration of the study is 8 years.

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 Childrens 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、200127
        • Shanghai Childrens Medical Center
      • Shanghai、中国、201102
        • Children S Hospital of Fudan University
      • 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、0000000
        • Clinica San Rafael
      • Bogotá、哥伦比亚、0000000
        • Fundacion Neumologica Colombiana
      • Bogotá、哥伦比亚
        • Fundacion Santa Fe de Bogota
      • 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
        • Fundacao 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
        • Hopital de la Timone
      • Montpellier、法国、34295
        • CHU Arnaud de Villeneuve
      • Paris、法国、75015
        • Hôpital 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
        • Skanes 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
        • Children's Hospital 1
      • Ho Chi Minh City、越南、700000
        • Tam Anh Hospital
      • Ho Chi Minh City、越南、700000
        • University Medical Center Ho Chi Minh city
  • 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

有资格学习的性别

全部

描述

Inclusion Criteria:

  • Participants between greater than or equal to (>=) 2 and less than (<) 18 years of age weighing >=9 kilogram (kg) at randomization
  • Pulmonary arterial hypertension (PAH) diagnosis confirmed by documented historical right heart catheterization (RHC) performed at any time before participant's screening
  • PAH (World Health Organization [WHO] Group 1), including participants with Down syndrome, of the following etiologies: Idiopathic PAH (IPAH); Heritable PAH (HPAH); PAH associated with congenital heart disease (PAH-associated with congenital heart disease [aCHD]) (PAH with coincidental CHD [that is, a small atrial septal defect, ventricular septal defect, or patent ductus arteriosus that does not itself account for the development of elevated PVR] and if approved by the BCAC) and Post-operative PAH (persisting / recurring/ developing >=6 months after repair of CHD); Drug or toxin-induced; PAH associated with Human immunodeficiency virus (HIV)
  • WHO functional class (FC) II and III
  • Participants treated with at least 1 PAH-specific treatment, example, an Endothelin receptor antagonist (ERA) and/or a Phosphodiesterase type-5 (PDE-5) inhibitor/soluble guanylate cyclase stimulator, provided that the treatment dose(s) has been stable for at least 3 months prior to first dose of study intervention

Exclusion Criteria:

  • PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease, and/or pulmonary capillary hemangiomatosis
  • PAH associated with Eisenmenger syndrome
  • Previous exposure to Uptravi (selexipag)
  • Known concomitant life-threatening disease with a life expectancy <12 months
  • Pregnant, planning to become pregnant, or lactating
  • Known allergies, hypersensitivity, or intolerance to selexipag or its excipients

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：Selexipag

Participants will receive selexipag based on the body weight on Day 1 and will continue thereafter with twice daily dosing. Selexipag will be uptitrated during the first 12 weeks until the participants reaches the individual maximum tolerated dose (iMTD) or until a maximum dose corresponding to their baseline body-weight category is achieved. Uptitration is followed by a maintenance period after Week 12 until end of treatment (EOT), at the maximum tolerated dose. Participants will continue to receive pulmonary arterial hypertension (PAH)-specific concomitant therapies such as ERAs, PDE-5 inhibitors, and soluble guanylate cyclase stimulator as per local standard-of-care.
药品：Selexipag

Selexipag tablet will be administered orally.

其他名称：
  • JNJ-67896049
药品：Standard of Care (SOC): Endothelin receptor antagonist

ERAs will be administered as SOC therapy.

药品：SOC: Phosphodiesterase type 5 (PDE-5) inhibitor

PDE-5 inhibitor will be administered as SOC therapy.

药品：SOC: Soluble guanylate cyclase stimulator

Soluble guanylate cyclase stimulator will be administered as SOC therapy.
安慰剂比较：Placebo

Participants will receive matching placebo based on the body weight on Day 1 and will continue thereafter with twice daily dosing. Participants will continue to receive PAH-specific concomitant therapies such as ERAs, PDE-5 inhibitors, and soluble guanylate cyclase stimulator as per local standard-of-care.
药品：Standard of Care (SOC): Endothelin receptor antagonist

ERAs will be administered as SOC therapy.

药品：SOC: Phosphodiesterase type 5 (PDE-5) inhibitor

PDE-5 inhibitor will be administered as SOC therapy.

药品：SOC: Soluble guanylate cyclase stimulator

Soluble guanylate cyclase stimulator will be administered as SOC therapy.

药品：Placebo

Matching placebo tablets will be administered orally.
实验性的：Open-Label Extension Period: Selexipag

Participants with a positive benefit/risk ratio of selexipag for PAH will be offered selexipag in the open label extension period. Participants on selexipag during the double-blind treatment period will continue treatment at their iMTD during the OLEP, for those previously on placebo, the iMTD will uptitrate selexipag during first 12 weeks until participant reaches iMTD. Participants will continue to receive PAH-specific concomitant therapies such as ERAs, PDE-5 inhibitors, and soluble guanylate cyclase stimulator as per local standard-of-care.
药品：Selexipag

Selexipag tablet will be administered orally.

其他名称：
  • JNJ-67896049
药品：Standard of Care (SOC): Endothelin receptor antagonist

ERAs will be administered as SOC therapy.

药品：SOC: Phosphodiesterase type 5 (PDE-5) inhibitor

PDE-5 inhibitor will be administered as SOC therapy.

药品：SOC: Soluble guanylate cyclase stimulator

Soluble guanylate cyclase stimulator will be administered as SOC therapy.

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)

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

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11月3日

上次提交的符合 QC 标准的更新

2026年11月2日

最后验证

2026年11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
  • 2019-002817-21 (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)
  • 2019 (美国 NIH 拨款/合同)

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

The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale open Data Access (YODA) Project site at yoda.yale.edu

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

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

订阅