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肺動脈性肺高血圧症の小児における標準治療への追加治療としてのセレキシパグの研究 (SALTO)

2026年11月2日 更新者：Actelion

小児の標準治療に対する追加治療としてのセレキシパグの有効性と安全性を評価するための無作為化、多施設共同、二重盲検、プラセボ対照、並行群間、イベント駆動型、群逐次試験による非盲検延長期間年齢 >=2 ～

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.

調査の概要

状態

積極的、募集していない

条件

介入・治療

詳細な説明

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.

研究の種類

介入

入学 (実際)

138

段階

  • フェーズ 3

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究場所

  • アイルランド
      • Dublin、アイルランド
        • Our Lady's Children's Hospital
  • アメリカ
    • Arizona
      • Phoenix、Arizona、アメリカ、85016
        • Phoenix Children's Hospital
    • California
      • Los Angeles、California、アメリカ、90095
        • UCLA Medical Center
      • San Francisco、California、アメリカ、94158
        • UCSF
    • Colorado
      • Aurora、Colorado、アメリカ、80045
        • Childrens Hospital Colorado
    • District of Columbia
      • Washington D.C.、District of Columbia、アメリカ、20010
        • Children's National Medical Center
    • Florida
      • Gainesville、Florida、アメリカ、32610
        • Congenital Heart Center of the University of Florida
    • Indiana
      • Indianapolis、Indiana、アメリカ、46202
        • Riley Hospital for Children
    • Michigan
      • Detroit、Michigan、アメリカ、48201
        • Detroit Medical Center
    • Ohio
      • Cincinnati、Ohio、アメリカ、45229
        • Cincinnati Children's Hospital Medical Center
    • Pennsylvania
      • Philadelphia、Pennsylvania、アメリカ、19104
        • Childrens Hospital Of Philadelphia
    • Texas
      • Houston、Texas、アメリカ、77030
        • Texas Children's Hospital
    • Utah
      • Salt Lake City、Utah、アメリカ、84113
        • Primary Children's Hospital
    • Virginia
      • Charlottesville、Virginia、アメリカ、22908
        • University of Virginia Division of Pediatric Cardiology
  • イスラエル
      • Haifa、イスラエル、3109601
        • Rambam Medical Center
      • Ramat Gan、イスラエル、52621
        • Sheba Medical Center
  • イタリア
      • Bologna、イタリア、40138
        • Azienda Ospedaliera Policlinico S. Orsola-Malpighi
      • Milan、イタリア、20162
        • ASST Grande Ospedale Metropolitano Niguarda
      • Padova、イタリア
        • Universta Degli Studi Di Padova
      • Roma、イタリア、00193
        • Ospedale Pediatrico Bambin Gesù
      • S. Donato Milanese、イタリア、20097
        • IRCCS Policlinico San Donato
      • Torino、イタリア、10126
        • AOU Città della Salute e della Scienza di Torino, Presidio Ospedale Infantile Regina Margherita
  • ウクライナ
      • Dnipro、ウクライナ、49006
        • Dnipropetrovsk clinical medical center of Mother and Child after prof. Rudnev
      • Dnipro、ウクライナ、49070
        • MI 'Dnipropetrovsk Regional Clinical Center of Cardiology and Cardiac Surgery'
      • Kyiv、ウクライナ、04050
        • Scientific Practical Medical Center for Pediatric Cardiology and Cardio Surgery of the MOH
      • Zaporizhzhya、ウクライナ、69063
        • MI Zaporizhzhia Regional Clinical Childrens Hospital of Zaporizhzhia Regional Council
  • オーストラリア
      • South Brisbane、オーストラリア、4101
        • Queensland CHILDREN'S HOSPITAL
  • カナダ
    • Alberta
      • Edmonton、Alberta、カナダ、T6G 2B7
        • Stollery Children's Hospital
    • Ontario
      • Toronto、Ontario、カナダ、M5G 1X8
        • Hospital For Sick Children
  • コロンビア
      • Bogotá、コロンビア、0000000
        • Clinica San Rafael
      • Bogotá、コロンビア、0000000
        • Fundacion Neumologica Colombiana
      • Bogotá、コロンビア
        • Fundacion Santa Fe de Bogota
      • Cali、コロンビア、760042
        • Clínica Imbanaco S.A.S.
      • Piedecuesta、コロンビア、681017
        • Fundacion Cardiovascular de Colombia
      • Soledad、コロンビア、0000000
        • Hospital Universidad del Norte
  • スイス
      • Lausanne、スイス、1011
        • Centre Hospitalier Universitaire Vaudois CHUV
  • スウェーデン
      • Gothenburg、スウェーデン、416 50
        • Drottning Silvias barn- och ungdomssjukhus
      • Lund、スウェーデン、222 42
        • Skanes universitetssjukhus
  • スペイン
      • A Coruña、スペイン、15006
        • Hosp Univ A Coruna
      • Barcelona、スペイン、08035
        • Hosp Univ Vall D Hebron
      • Esplugues de Llobregat、スペイン、08950
        • Hosp. Sant Joan de Deu
      • Madrid、スペイン、28046
        • Hosp. Univ. La Paz
      • Madrid、スペイン、28009
        • Hosp. Gral. Univ. Gregorio Maranon
      • Seville、スペイン、41013
        • Hosp. Virgen Del Rocio
  • セルビア
      • Belgrade、セルビア、11000
        • Univerzitetska Dečja Klinika
  • タイ
      • Chiang Mai、タイ、50200
        • Chiang Mai University Hospital
      • Songkhla、タイ、90110
        • Songklanagarind hospital
  • トルコ（Türkiye）
      • Adana、トルコ（Türkiye）、01790
        • Cukurova Balcali Hospital Application and Research Center
      • Ankara、トルコ（Türkiye）、06230
        • Hacettepe University Medical Faculty
      • Istanbul、トルコ（Türkiye）、34093
        • CAPA Istanbul University Medical Faculty
      • Istanbul、トルコ（Türkiye）、34303
        • Mehmet Akif Ersoy Training and Research Hospital
      • Izmir、トルコ（Türkiye）、35020
        • Izmir Tepecik Training and Research Hospital
      • Izmir、トルコ（Türkiye）、35210
        • Behcet Uz Pediatric Diseases and Surgery Training and Research Hospital
  • ドイツ
      • Freiburg im Breisgau、ドイツ、70106
        • Universitätsklinikum Freiburg Zentrum
      • Heidelberg、ドイツ、D-69120
        • Universitaetsklinikum Heidelberg
      • Leipzig、ドイツ、04289
        • Herzzentrum Leipzig GmbH
      • München、ドイツ、81377
        • Klinikum der Universitaet Muenchen
  • ハンガリー
      • Budapest、ハンガリー、1096
        • Gottsegen György Országos Kardiológiai Intézet
  • フィンランド
      • Helsinki、フィンランド、29
        • New Children's Hospital of the Helsinki University Hospital (HUS)
  • フランス
      • Lille、フランス、59037
        • Hôpital Cardiologique - Chru Lille
      • Marseille、フランス、13385
        • Hopital de la Timone
      • Montpellier、フランス、34295
        • CHU Arnaud de Villeneuve
      • Paris、フランス、75015
        • Hôpital Necker - Enfants Malades
      • Pessac、フランス、33604
        • Hôpital Cardiologique Du Haut-Lévêque
      • Toulouse、フランス、31059
        • Chu Hopital Des Enfants
  • ブラジル
      • Blumenau、ブラジル、89030-101
        • Complexo de Prevencao,Diagnostico,Terapia e Reabilitacao Respiratoria LTDA Hospital Dia do Pulmao
      • Brasília、ブラジル、70310-500
        • Fundacao Universitaria de Cardiologia - Instituto de Cardiologia e Transplantes do DF
      • Curitiba、ブラジル、80250-060
        • Hospital Pequeno Principe
      • Fortaleza、ブラジル、60840-285
        • Secretaria da Saude do Estado do Ceara - Hospital Doutor Carlos Alberto Studart Gomes
      • Porto Alegre、ブラジル、90020-090
        • Irmandade Santa Casa de Misericordia de Porto Alegre
      • Porto Alegre、ブラジル、90620-001
        • Fundacao Universitaria de Cardiologia
      • São Paulo、ブラジル、01221-020
        • Irmandade Santa Casa de Misericordia de Sao Paulo
      • São Paulo、ブラジル、04024 002
        • SPDM - Associacao Paulista para o Desenvolvimento da Medicina - Hospital Sao Paulo
  • ブルガリア
      • Sofia、ブルガリア、1309
        • Multiprofile Hospital For Active Treatment National Cardiology Hospital, Ead
  • ベトナム
      • Hanoi、ベトナム
        • Hanoi Medical University Hospital
      • Ho Chi Minh City、ベトナム
        • Children's Hospital 1
      • Ho Chi Minh City、ベトナム、700000
        • Tam Anh Hospital
      • Ho Chi Minh City、ベトナム、700000
        • University Medical Center Ho Chi Minh city
  • ベラルーシ
      • Minsk、ベラルーシ、220013
        • State Institution Republican Scientific And Practical Center For Pediatric Surgery
      • Minsk、ベラルーシ、220118
        • Health Institution 4Th City Children'S Clinical Hospital
  • ベルギー
      • Brussels、ベルギー、1070
        • ULB Hôpital Erasme
      • Ghent、ベルギー、9000
        • Universitair Ziekenhuis Gent
      • Leuven、ベルギー、3000
        • Universitaire Ziekenhuizen Leuven
  • ポルトガル
      • Lisbon、ポルトガル、1169-024
        • Uls Sao Jose - Hosp. Santa Marta
      • Porto、ポルトガル、4200 319
        • Uls Sao Joao - Hosp. Sao Joao
  • ポーランド
      • Gdansk、ポーランド、80 952
        • Uniwersyteckie Centrum Kliniczne
      • Krakow、ポーランド、30-663
        • Uniwersytecki Szpital Dzieciecy w Krakowie
      • Poznan、ポーランド、60 572
        • Szpital Kliniczny im Karola Jonschera
      • Warsaw、ポーランド、04-730
        • Instytut Pomnik Centrum Zdrowia Dziecka
      • Wroclaw、ポーランド、51 124
        • Wojewodzki Szpital Specjalistyczny we Wroclawiu
      • Zabrze、ポーランド、41-800
        • Slaskie Centrum Chorob Serca
  • マレーシア
      • Kuala Lumpur、マレーシア、50400
        • National Heart Institute
  • メキシコ
      • Guadalajara、メキシコ、44160
        • CICUM San Miguel
      • Monterrey、メキシコ、64718
        • Unidad de Investigacion Clinica en Medicina S.C. (UDICEM)
      • México、メキシコ、52787
        • Operadora de Hospitales Angeles SA de CV Hospital Angeles Lomas
  • リトアニア
      • Vilnius、リトアニア、LT08661
        • Vilnius University Hospital Santariskiu Clinics
  • ロシア
      • Kazan'、ロシア、420012
        • Kazan State Medical University
      • Kazan'、ロシア、420059
        • Kazan State Medical University 1
      • Kemerovo、ロシア、650002
        • Scientific and Research Institution of Cardiovascular Diseases Complex Problems
      • Moscow、ロシア、125373
        • Childrens City Clinical Hospital n.a. Bashlyaeva
      • Moscow、ロシア、125412
        • Veltischev Research and Clinical Institute for Pediatrics of the Pirogov RNRMU
      • Samara、ロシア、443070
        • Samara Regional Clinical Cardiological Dispensary
  • 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
  • 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
  • 韓国
      • Seoul、韓国、03080
        • Seoul National University Hospital
      • Seoul、韓国、06351
        • Samsung Medical Center
      • Seoul、韓国、120-752
        • Severance Hospital Yonsei University Health System
      • Yangsan、韓国、50612
        • Pusan National University Yangsan Hospital

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

2年～17年 (子)

健康ボランティアの受け入れ

いいえ

受講資格のある性別

全て

説明

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

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか？

デザインの詳細

  • 主な目的：処理
  • 割り当て：ランダム化
  • 介入モデル：並列代入
  • マスキング：ダブル

武器と介入

参加者グループ / アーム
介入・治療
実験的：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.
薬：Selexipag

Selexipag tablet will be administered orally.

他の名前：
  • JNJ-67896049
薬：Standard of Care (SOC): Endothelin receptor antagonist

ERAs will be administered as SOC therapy.

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

PDE-5 inhibitor will be administered as SOC therapy.

薬：SOC: Soluble guanylate cyclase stimulator

Soluble guanylate cyclase stimulator will be administered as SOC therapy.
プラセボコンパレーター：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.
薬：Standard of Care (SOC): Endothelin receptor antagonist

ERAs will be administered as SOC therapy.

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

PDE-5 inhibitor will be administered as SOC therapy.

薬：SOC: Soluble guanylate cyclase stimulator

Soluble guanylate cyclase stimulator will be administered as SOC therapy.

薬：Placebo

Matching placebo tablets will be administered orally.
実験的：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.
薬：Selexipag

Selexipag tablet will be administered orally.

他の名前：
  • JNJ-67896049
薬：Standard of Care (SOC): Endothelin receptor antagonist

ERAs will be administered as SOC therapy.

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

PDE-5 inhibitor will be administered as SOC therapy.

薬：SOC: Soluble guanylate cyclase stimulator

Soluble guanylate cyclase stimulator will be administered as SOC therapy.

この研究は何を測定していますか？

主要な結果の測定

結果測定
メジャーの説明
時間枠
Double-blind Period: Time to Disease Progression
時間枠：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)

二次結果の測定

結果測定
メジャーの説明
時間枠
Double-blind Period: Change in log2 N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) From Baseline to Week 24
時間枠：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)
時間枠：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
時間枠：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)
時間枠：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
時間枠：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
時間枠：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
時間枠：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)
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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)
時間枠：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])
時間枠：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
時間枠：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)
時間枠：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
時間枠：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)
時間枠：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)

協力者と研究者

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スポンサー

Actelion

捜査官

  • スタディディレクター：Actelion Clinical Trial、Actelion

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (実際)

2020年1月16日

一次修了 (実際)

2024年10月11日

研究の完了 (推定)

2027年10月1日

試験登録日

最初に提出

2019年11月8日

QC基準を満たした最初の提出物

2019年11月21日

最初の投稿 (実際)

2019年11月25日

学習記録の更新

投稿された最後の更新 (実際)

2026年11月3日

QC基準を満たした最後の更新が送信されました

2026年11月2日

最終確認日

2026年11月1日

詳しくは

本研究に関する用語

追加の関連 MeSH 用語

その他の研究ID番号

  • CR108716
  • 2019-002817-21 (EudraCT番号)
  • AC-065A310 (その他の識別子：Janssen Research & Development, LLC)
  • 2022-501012-34-00 (レジストリ識別子：EUCT number)
  • 2019 (米国 NIH グラント/契約)

個々の参加者データ (IPD) の計画

個々の参加者データ (IPD) を共有する予定はありますか?

はい

IPD プランの説明

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

医薬品およびデバイス情報、研究文書

米国FDA規制医薬品の研究

はい

米国FDA規制機器製品の研究

いいえ

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