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Исследование селексипага в качестве дополнительной терапии к стандарту лечения детей с легочной артериальной гипертензией (SALTO)

2 ноября 2026 г. обновлено: Actelion

Рандомизированное, многоцентровое, двойное слепое, плацебо-контролируемое, параллельное групповое, событийно-ориентированное, групповое последовательное исследование с открытым продленным периодом для оценки эффективности и безопасности селексипага в качестве дополнительного лечения к стандарту лечения детей. Возраст от >=2 до

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.

Обзор исследования

Статус

Активный, не рекрутирующий

Условия

Вмешательство/лечение

Подробное описание

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.

Тип исследования

Интервенционный

Регистрация (Действительный)

138

Фаза

  • Фаза 3

Контакты и местонахождение

В этом разделе приведены контактные данные лиц, проводящих исследование, и информация о том, где проводится это исследование.

Места учебы

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

Критерии участия

Исследователи ищут людей, которые соответствуют определенному описанию, называемому критериям приемлемости. Некоторыми примерами этих критериев являются общее состояние здоровья человека или предшествующее лечение.

Критерии приемлемости

Возраст, подходящий для обучения

От 2 года до 17 лет (Ребенок)

Принимает здоровых добровольцев

Нет

Полы, имеющие право на обучение

Все

Описание

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

Учебный план

В этом разделе представлена ​​подробная информация о плане исследования, в том числе о том, как планируется исследование и что оно измеряет.

Как устроено исследование?

Детали дизайна

  • Основная цель: Уход
  • Распределение: Рандомизированный
  • Интервенционная модель: Параллельное назначение
  • Маскировка: Двойной

Оружие и интервенции

Группа участников / Армия
Вмешательство/лечение
Экспериментальный: 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.
Лекарство: Selexipag

Selexipag tablet will be administered orally.

Другие имена:
  • JNJ-67896049
Лекарство: Standard of Care (SOC): Endothelin receptor antagonist

ERAs will be administered as SOC therapy.

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

PDE-5 inhibitor will be administered as SOC therapy.

Лекарство: SOC: Soluble guanylate cyclase stimulator

Soluble guanylate cyclase stimulator will be administered as SOC therapy.
Плацебо Компаратор: 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.
Лекарство: Standard of Care (SOC): Endothelin receptor antagonist

ERAs will be administered as SOC therapy.

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

PDE-5 inhibitor will be administered as SOC therapy.

Лекарство: SOC: Soluble guanylate cyclase stimulator

Soluble guanylate cyclase stimulator will be administered as SOC therapy.

Лекарство: Placebo

Matching placebo tablets will be administered orally.
Экспериментальный: 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.
Лекарство: Selexipag

Selexipag tablet will be administered orally.

Другие имена:
  • JNJ-67896049
Лекарство: Standard of Care (SOC): Endothelin receptor antagonist

ERAs will be administered as SOC therapy.

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

PDE-5 inhibitor will be administered as SOC therapy.

Лекарство: SOC: Soluble guanylate cyclase stimulator

Soluble guanylate cyclase stimulator will be administered as SOC therapy.

Что измеряет исследование?

Первичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
Double-blind Period: Time to Disease Progression
Временное ограничение: 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)

Вторичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
Double-blind Period: Change in log2 N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) From Baseline to Week 24
Временное ограничение: 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)
Временное ограничение: 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
Временное ограничение: 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)
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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)
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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)
Временное ограничение: 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])
Временное ограничение: 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
Временное ограничение: 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)
Временное ограничение: 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
Временное ограничение: 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)
Временное ограничение: 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)

Соавторы и исследователи

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Спонсор

Actelion

Следователи

  • Директор по исследованиям: Actelion Clinical Trial, Actelion

Даты записи исследования

Эти даты отслеживают ход отправки отчетов об исследованиях и сводных результатов на сайт ClinicalTrials.gov. Записи исследований и сообщаемые результаты проверяются Национальной медицинской библиотекой (NLM), чтобы убедиться, что они соответствуют определенным стандартам контроля качества, прежде чем публиковать их на общедоступном веб-сайте.

Изучение основных дат

Начало исследования (Действительный)

16 января 2020 г.

Первичное завершение (Действительный)

11 октября 2024 г.

Завершение исследования (Оцененный)

1 октября 2027 г.

Даты регистрации исследования

Первый отправленный

8 ноября 2019 г.

Впервые представлено, что соответствует критериям контроля качества

21 ноября 2019 г.

Первый опубликованный (Действительный)

25 ноября 2019 г.

Обновления учебных записей

Последнее опубликованное обновление (Действительный)

3 ноября 2026 г.

Последнее отправленное обновление, отвечающее критериям контроля качества

2 ноября 2026 г.

Последняя проверка

1 ноября 2026 г.

Дополнительная информация

Термины, связанные с этим исследованием

Дополнительные соответствующие термины MeSH

Другие идентификационные номера исследования

  • CR108716
  • 2019-002817-21 (Номер EudraCT)
  • AC-065A310 (Другой идентификатор: Janssen Research & Development, LLC)
  • 2022-501012-34-00 (Идентификатор реестра: EUCT number)
  • 2019 (Грант/контракт NIH США)

Планирование данных отдельных участников (IPD)

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ДА

Описание плана IPD

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

Информация о лекарствах и устройствах, исследовательские документы

Изучает лекарственный продукт, регулируемый FDA США.

Да

Изучает продукт устройства, регулируемый Управлением по санитарному надзору за качеством пищевых продуктов и медикаментов США.

Нет

Эта информация была получена непосредственно с веб-сайта clinicaltrials.gov без каких-либо изменений. Если у вас есть запросы на изменение, удаление или обновление сведений об исследовании, обращайтесь по адресу register@clinicaltrials.gov. Как только изменение будет реализовано на clinicaltrials.gov, оно будет автоматически обновлено и на нашем веб-сайте. .

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