Um estudo de Selexipag como tratamento complementar ao tratamento padrão em crianças com hipertensão arterial pulmonar (SALTO)
27 de agosto de 2026 atualizado por: Actelion
Um estudo randomizado, multicêntrico, duplo-cego, controlado por placebo, grupo paralelo, orientado a eventos, grupo sequencial com período de extensão aberto para avaliar a eficácia e a segurança de Selexipag como tratamento complementar ao tratamento padrão em crianças Idade >=2 a
O objetivo deste estudo é avaliar se a adição de selexipag ao tratamento padrão atrasa a progressão da doença em crianças com Hipertensão Arterial Pulmonar (HAP) em comparação com o placebo.
Visão geral do estudo
Status
Ativo, não recrutando
Condições
Descrição detalhada
A HAP pediátrica é uma doença rara e progressiva associada a considerável morbidade e mortalidade.
Dada a necessidade médica significativa de desenvolver tratamentos em crianças com HAP, são necessários mais estudos clínicos na população pediátrica para fornecer mais dados para o tratamento da HAP em crianças.
O selexipag (JNJ-67896049) é um agonista não prostanóide do receptor da prostaciclina disponível oralmente, seletivo e de longa duração, aprovado e disponível comercialmente para o tratamento de participantes adultos com HAP.
Selexipag e seu metabólito possuem propriedades antifibróticas, antiproliferativas e antitrombóticas.
Atualmente, nenhum medicamento direcionado à via da prostaciclina está aprovado para uso pediátrico na HAP.
Uma terapia eficaz e disponível por via oral atuando no receptor de prostaciclina, como selexipag, introduzida no estágio clinicamente apropriado da doença de HAP e principalmente em combinação com medicamentos específicos de primeira linha para HAP em participantes que precisam de terapia adicional devido ao controle insuficiente da doença representa um grande avanço no manejo terapêutico dos participantes pediátricos com HAP.
Este estudo consiste em um período de triagem de até 6 semanas e um período de tratamento duplo-cego, incluindo titulação e períodos de manutenção, seguido por um período de extensão aberto de 3 anos (OLEP) e um acompanhamento de segurança de 30 dias. período de alta que ocorre após a última dose da intervenção do estudo (seja duplo-cego ou aberto).
As avaliações de segurança, farmacocinética e eficácia serão realizadas durante o estudo.
Um Comitê Independente de Monitoramento de Dados (IDMC) será estabelecido para monitorar os dados de forma contínua, revisar os dados provisórios e garantir a segurança contínua dos participantes inscritos neste estudo.
A duração aproximada do estudo é de 8 anos.
Tipo de estudo
Intervencional
Inscrição (Real)
138
Estágio
- Fase 3
Contactos e Locais
Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.
Locais de estudo
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Freiburg im Breisgau, Alemanha, 70106
- Universitätsklinikum Freiburg Zentrum
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Heidelberg, Alemanha, D-69120
- Universitaetsklinikum Heidelberg
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Leipzig, Alemanha, 04289
- Herzzentrum Leipzig GmbH
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München, Alemanha, 81377
- Klinikum der Universitaet Muenchen
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South Brisbane, Austrália, 4101
- Queensland Children's Hospital
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Minsk, Bielorrússia, 220013
- State Institution Republican Scientific And Practical Center For Pediatric Surgery
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Minsk, Bielorrússia, 220118
- Health Institution 4Th City Children'S Clinical Hospital
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Blumenau, Brasil, 89030-101
- Complexo de Prevencao,Diagnostico,Terapia e Reabilitacao Respiratoria LTDA Hospital Dia do Pulmao
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Brasília, Brasil, 70310-500
- Fundacao Universitaria de Cardiologia - Instituto de Cardiologia e Transplantes do DF
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Curitiba, Brasil, 80250-060
- Hospital Pequeno Principe
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Fortaleza, Brasil, 60840-285
- Secretaria da Saude do Estado do Ceara - Hospital Doutor Carlos Alberto Studart Gomes
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Porto Alegre, Brasil, 90020-090
- Irmandade Santa Casa de Misericordia de Porto Alegre
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Porto Alegre, Brasil, 90620-001
- Fundação Universitaria de Cardiologia
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São Paulo, Brasil, 01221-020
- Irmandade Santa Casa de Misericordia de Sao Paulo
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São Paulo, Brasil, 04024 002
- SPDM - Associacao Paulista para o Desenvolvimento da Medicina - Hospital Sao Paulo
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Sofia, Bulgária, 1309
- Multiprofile Hospital For Active Treatment National Cardiology Hospital, Ead
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Brussels, Bélgica, 1070
- ULB Hôpital Erasme
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Ghent, Bélgica, 9000
- Universitair Ziekenhuis Gent
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Leuven, Bélgica, 3000
- Universitaire Ziekenhuizen Leuven
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Alberta
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Edmonton, Alberta, Canadá, T6G 2B7
- Stollery Children's Hospital
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Ontario
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Toronto, Ontario, Canadá, M5G 1X8
- Hospital for Sick Children
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Beijing, China, 100029
- Beijing Anzhen Hospital
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Guangzhou, China, 510623
- Guangzhou Women and Children's Medical Center
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Qingdao, China, 266000
- Qingdao Women and Children's Hospital
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Qingdao, China, 266000
- Qingdao Women and Children's Hospital 1
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Shanghai, China, 201102
- Children's Hospital of Fudan University
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Shanghai, China, 200127
- Shanghai Childrens Medical Center
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Shenyang, China, 110000
- The General Hospital of Northern Theater Command
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Bogotá, Colômbia
- Fundación Santa Fe de Bogotá
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Bogotá, Colômbia, 0000000
- Clinica San Rafael
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Bogotá, Colômbia, 0000000
- Fundacion Neumologica Colombiana
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Cali, Colômbia, 760042
- Clínica Imbanaco S.A.S.
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Piedecuesta, Colômbia, 681017
- Fundacion cardiovascular de Colombia
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Soledad, Colômbia, 0000000
- Hospital Universidad del Norte
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Seoul, Coréia do Sul, 03080
- Seoul National University Hospital
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Seoul, Coréia do Sul, 06351
- Samsung Medical Center
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Seoul, Coréia do Sul, 120-752
- Severance Hospital Yonsei University Health System
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Yangsan, Coréia do Sul, 50612
- Pusan National University Yangsan Hospital
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A Coruña, Espanha, 15006
- Hosp Univ A Coruna
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Barcelona, Espanha, 08035
- Hosp Univ Vall D Hebron
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Esplugues de Llobregat, Espanha, 08950
- Hosp. Sant Joan de Deu
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Madrid, Espanha, 28046
- Hosp. Univ. La Paz
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Madrid, Espanha, 28009
- Hosp. Gral. Univ. Gregorio Maranon
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Seville, Espanha, 41013
- Hosp. Virgen Del Rocio
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Arizona
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Phoenix, Arizona, Estados Unidos, 85016
- Phoenix Children's Hospital
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California
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Los Angeles, California, Estados Unidos, 90095
- UCLA Medical Center
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San Francisco, California, Estados Unidos, 94158
- UCSF
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Colorado
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Aurora, Colorado, Estados Unidos, 80045
- Childrens Hospital Colorado
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District of Columbia
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Washington D.C., District of Columbia, Estados Unidos, 20010
- Children's National Medical Center
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Florida
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Gainesville, Florida, Estados Unidos, 32610
- Congenital Heart Center of the University of Florida
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Indiana
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Indianapolis, Indiana, Estados Unidos, 46202
- Riley Hospital for Children
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Michigan
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Detroit, Michigan, Estados Unidos, 48201
- Detroit Medical Center
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Ohio
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Cincinnati, Ohio, Estados Unidos, 45229
- Cincinnati Children's Hospital Medical Center
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Pennsylvania
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Philadelphia, Pennsylvania, Estados Unidos, 19104
- Childrens Hospital of Philadelphia
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Texas
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Houston, Texas, Estados Unidos, 77030
- Texas Children's Hospital
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Utah
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Salt Lake City, Utah, Estados Unidos, 84113
- Primary Children's Hospital
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Virginia
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Charlottesville, Virginia, Estados Unidos, 22908
- University of Virginia Division of Pediatric Cardiology
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Helsinki, Finlândia, 29
- New Children's Hospital of the Helsinki University Hospital (HUS)
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Lille, França, 59037
- Hôpital Cardiologique - Chru Lille
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Marseille, França, 13385
- Hôpital de la Timone
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Montpellier, França, 34295
- CHU Arnaud de Villeneuve
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Paris, França, 75015
- Hopital Necker - Enfants Malades
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Pessac, França, 33604
- Hôpital Cardiologique Du Haut-Lévêque
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Toulouse, França, 31059
- Chu Hopital Des Enfants
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Budapest, Hungria, 1096
- Gottsegen György Országos Kardiológiai Intézet
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Dublin, Irlanda
- Our Lady's Children's Hospital
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Haifa, Israel, 3109601
- Rambam Medical Center
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Ramat Gan, Israel, 52621
- Sheba medical center
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Bologna, Itália, 40138
- Azienda Ospedaliera Policlinico S. Orsola-Malpighi
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Milan, Itália, 20162
- ASST Grande Ospedale Metropolitano Niguarda
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Padova, Itália
- Universta Degli Studi Di Padova
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Roma, Itália, 00193
- Ospedale Pediatrico Bambin Gesù
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S. Donato Milanese, Itália, 20097
- IRCCS Policlinico San Donato
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Torino, Itália, 10126
- AOU Città della Salute e della Scienza di Torino, Presidio Ospedale Infantile Regina Margherita
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Vilnius, Lituânia, LT08661
- Vilnius University Hospital Santariskiu Clinics
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Kuala Lumpur, Malásia, 50400
- National Heart Institute
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Guadalajara, México, 44160
- CICUM San Miguel
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Monterrey, México, 64718
- Unidad de Investigacion Clinica en Medicina S.C. (UDICEM)
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México, México, 52787
- Operadora de Hospitales Angeles SA de CV Hospital Angeles Lomas
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Gdansk, Polônia, 80 952
- Uniwersyteckie Centrum Kliniczne
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Krakow, Polônia, 30-663
- Uniwersytecki Szpital Dzieciecy w Krakowie
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Poznan, Polônia, 60 572
- Szpital Kliniczny im Karola Jonschera
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Warsaw, Polônia, 04-730
- Instytut Pomnik Centrum Zdrowia Dziecka
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Wroclaw, Polônia, 51 124
- Wojewodzki Szpital Specjalistyczny We Wroclawiu
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Zabrze, Polônia, 41-800
- Slaskie Centrum Chorob Serca
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Lisbon, Portugal, 1169-024
- Uls Sao Jose - Hosp. Santa Marta
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Porto, Portugal, 4200 319
- Uls Sao Joao - Hosp. Sao Joao
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Kazan', Rússia, 420012
- Kazan State Medical University
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Kazan', Rússia, 420059
- Kazan State Medical University 1
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Kemerovo, Rússia, 650002
- Scientific and Research Institution of Cardiovascular Diseases Complex Problems
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Moscow, Rússia, 125373
- Childrens City Clinical Hospital n.a. Bashlyaeva
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Moscow, Rússia, 125412
- Veltischev Research and Clinical Institute for Pediatrics of the Pirogov RNRMU
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Samara, Rússia, 443070
- Samara Regional Clinical Cardiological Dispensary
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Gothenburg, Suécia, 416 50
- Drottning Silvias barn- och ungdomssjukhus
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Lund, Suécia, 222 42
- Skånes universitetssjukhus
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Lausanne, Suíça, 1011
- Centre hospitalier universitaire vaudois CHUV
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Belgrade, Sérvia, 11000
- Univerzitetska Dečja Klinika
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Chiang Mai, Tailândia, 50200
- Chiang Mai University Hospital
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Songkhla, Tailândia, 90110
- Songklanagarind Hospital
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Kaohsiung City, Taiwan, 813414
- Kaohsiung Veterans General Hospital
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Tainan, Taiwan, 704
- National Cheng Kung University Hospital
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Taipei, Taiwan, 100
- National Taiwan University Hospital
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Adana, Turquia (Türkiye), 01790
- Cukurova Balcali Hospital Application and Research Center
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Ankara, Turquia (Türkiye), 06230
- Hacettepe University Medical Faculty
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Istanbul, Turquia (Türkiye), 34093
- CAPA Istanbul University Medical Faculty
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Istanbul, Turquia (Türkiye), 34303
- Mehmet Akif Ersoy Training and Research Hospital
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Izmir, Turquia (Türkiye), 35020
- Izmir Tepecik Training and Research Hospital
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Izmir, Turquia (Türkiye), 35210
- Behcet Uz Pediatric Diseases and Surgery Training and Research Hospital
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Dnipro, Ucrânia, 49006
- Dnipropetrovsk clinical medical center of Mother and Child after prof. Rudnev
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Dnipro, Ucrânia, 49070
- MI 'Dnipropetrovsk Regional Clinical Center of Cardiology and Cardiac Surgery'
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Kyiv, Ucrânia, 04050
- Scientific Practical Medical Center for Pediatric Cardiology and Cardio Surgery of the MOH
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Zaporizhzhya, Ucrânia, 69063
- MI Zaporizhzhia Regional Clinical Childrens Hospital of Zaporizhzhia Regional Council
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Hanoi, Vietnã
- Hanoi Medical University Hospital
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Ho Chi Minh City, Vietnã, 700000
- University Medical Center Ho Chi Minh City
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Ho Chi Minh City, Vietnã
- Children's Hospital 1
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Ho Chi Minh City, Vietnã, 700000
- Tam Anh Hospital
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Critérios de participação
Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.
Critérios de elegibilidade
Idades elegíveis para estudo
2 anos a 17 anos (Filho)
Aceita Voluntários Saudáveis
Não
Descrição
Critério de inclusão:
- Participantes entre maior ou igual a (>=) 2 e menor que (<) 18 anos de idade com peso >=9 quilogramas (kg) na randomização
- Diagnóstico de hipertensão arterial pulmonar (HAP) confirmado por histórico documentado de cateterismo cardíaco direito (RHC) realizado a qualquer momento antes da triagem do participante
- HAP (Organização Mundial da Saúde [OMS] Grupo 1), incluindo participantes com síndrome de Down, das seguintes etiologias: HAP idiopática (HAPI); HAP hereditária (HPAH); HAP associada a doença cardíaca congênita (HAP associada a doença cardíaca congênita [aCHD]) (HAP com CHD coincidente [isto é, um pequeno defeito do septo atrial, defeito do septo ventricular ou persistência do canal arterial que não é responsável pelo desenvolvimento de PVR elevado] e se aprovado pelo BCAC) e HAP pós-operatória (persistente/recorrente/desenvolvendo >=6 meses após correção de CHD); Induzido por drogas ou toxinas; HAP associada ao vírus da imunodeficiência humana (HIV)
- Classe funcional da OMS (CF) II e III
- Participantes tratados com pelo menos 1 tratamento específico para HAP, por exemplo, um antagonista do receptor de endotelina (ERA) e/ou um inibidor de fosfodiesterase tipo 5 (PDE-5)/estimulador de guanilato ciclase solúvel, desde que a(s) dose(s) de tratamento tenha(m) sido estável por pelo menos 3 meses antes da primeira dose da intervenção do estudo
Critério de exclusão:
- HAP devido a hipertensão portal, esquistossomose, doença veno-oclusiva pulmonar e/ou hemangiomatose capilar pulmonar
- HAP associada à síndrome de Eisenmenger
- Exposição anterior ao Uptravi (selexipag)
- Doença concomitante com risco de vida conhecida com expectativa de vida <12 meses
- Grávida, planejando engravidar ou amamentando
- Alergias, hipersensibilidade ou intolerância conhecidas ao selexipag ou seus excipientes
Plano de estudo
Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: Randomizado
- Modelo Intervencional: Atribuição Paralela
- Mascaramento: Dobro
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
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Experimental: Selexipag
Os participantes receberão selexipag com base no peso corporal no Dia 1 e continuarão posteriormente com dosagem duas vezes por dia.
O selexipag será aumentado durante as primeiras 12 semanas até que os participantes atinjam a dose máxima tolerada individual (iMTD) ou até que seja alcançada uma dose máxima correspondente à sua categoria de peso corporal basal.
O aumento é seguido por um período de manutenção após a Semana 12 até ao final do tratamento (EOT), na dose máxima tolerada.
Os participantes continuarão a receber terapias concomitantes específicas para hipertensão arterial pulmonar (HAP), como ERA, inibidores da PDE-5 e estimulador de guanilato ciclase solúvel, de acordo com o padrão local de cuidados.
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O comprimido de Selexipag será administrado por via oral.
Outros nomes:
Os ERA serão administrados como terapia SOC.
O inibidor da PDE-5 será administrado como terapia SOC.
O estimulador de guanilato ciclase solúvel será administrado como terapia SOC.
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Comparador de Placebo: Placebo
Os participantes receberão placebo correspondente com base no peso corporal no Dia 1 e continuarão posteriormente com administração duas vezes ao dia.
Os participantes continuarão a receber terapias concomitantes específicas para HAP, como ERA, inibidores da PDE-5 e estimulador da guanilato ciclase solúvel, de acordo com o padrão de cuidados local.
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Os comprimidos de placebo correspondentes serão administrados por via oral.
Os ERA serão administrados como terapia SOC.
O inibidor da PDE-5 será administrado como terapia SOC.
O estimulador de guanilato ciclase solúvel será administrado como terapia SOC.
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Experimental: Período de Extensão em Acesso Aberto: Selexipag
Os participantes com uma relação benefício/risco positiva do selexipag para a HAP receberão selexipag no período de extensão de etiqueta aberta.
Os participantes em selexipag durante o período de tratamento duplo-cego continuarão o tratamento na sua iMTD durante o OLEP; para os que estavam anteriormente em placebo, a iMTD aumentará o selexipag durante as primeiras 12 semanas até o participante atingir a iMTD.
Os participantes continuarão a receber terapias concomitantes específicas para a HAP, tais como ERAs, inibidores da PDE-5 e estimulador da guanilato ciclase solúvel, de acordo com o padrão de cuidados local.
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O comprimido de Selexipag será administrado por via oral.
Outros nomes:
Os ERA serão administrados como terapia SOC.
O inibidor da PDE-5 será administrado como terapia SOC.
O estimulador de guanilato ciclase solúvel será administrado como terapia SOC.
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O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Double-blind Period: Time to Disease Progression
Prazo: 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)
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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.
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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)
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Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Double-blind Period: Change in log2 N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) From Baseline to Week 24
Prazo: Baseline (Day 1), Week 24
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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).
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Baseline (Day 1), Week 24
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Double-blind Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious AEs (TESAEs)
Prazo: 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)
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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.
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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)
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Double-blind Period: Number of Participants With AEs Leading to Premature Discontinuation of Study Treatment
Prazo: 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)
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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.
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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)
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Double-blind Period: Change From Baseline in Vital Signs: Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)
Prazo: Baseline (Day 1), Weeks 24, 48, 72, and 96
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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.
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Baseline (Day 1), Weeks 24, 48, 72, and 96
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Double-blind Period: Change From Baseline in Vital Signs: Pulse Rate
Prazo: Baseline (Day 1), Weeks 24, 48, 72, and 96
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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.
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Baseline (Day 1), Weeks 24, 48, 72, and 96
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Double-blind Period: Change From Baseline in Growth Parameter: Body Weight
Prazo: Baseline (Day 1), Weeks 24, 48, 72, and 96
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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.
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Baseline (Day 1), Weeks 24, 48, 72, and 96
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Double-blind Period: Change From Baseline in Growth Parameter: Height
Prazo: 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.
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Baseline (Day 1), Weeks 24, 48, 72, and 96
|
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Double-blind Period: Number of Participants With Shift From Baseline in Sexual Maturation (Tanner Stage)
Prazo: 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)
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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.
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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)
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Double-blind Period: Number of Participants With Treatment-emergent Electrocardiogram (ECG) Abnormalities
Prazo: 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)
|
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Double-blind Period: Number of Participants With Treatment-emergent (TE) Marked Laboratory Abnormalities
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
|
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Double-blind Period: Change From Baseline in Treatment-emergent Laboratory Parameter: Electrolytes (Sodium, Potassium, Calcium, Bicarbonate and Chloride)
Prazo: 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
|
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Double-blind Period: Change From Baseline in Treatment-emergent Laboratory Parameter: Transaminases (Alanine Aminotransferase [ALT] and Aspartate Aminotransferase [AST])
Prazo: 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
Prazo: 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
|
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Double-blind Period: Time to First Clinical Event Committee (CEC)-Confirmed Hospitalization or Death for Pulmonary Arterial Hypertension (PAH)
Prazo: 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)
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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)
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Double-blind Period: Dose-normalized Steady-state Trough Concentrations (Ctrough,ss,dn) of Selexipag and Its Metabolite ACT-333679 by Age Cohort
Prazo: 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)
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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.
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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)
|
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Double-blind Period: Dose-normalized Steady-state Trough Concentrations (Ctrough,ss,dn) of Selexipag and Its Metabolite ACT-333679 by Body Weight (BW)
Prazo: BW >=50kg: Predose between W16 to end of DB treatment period (maximum up to W212); BW >=25-<50kg: Predose between W16 to end of DB treatment period (maximum up to W198); BW >=9- <25kg: Predose between W16 to end of DB treatment period (maximum up to W175)
|
Dose-normalized steady-state trough concentration (Ctrough,ss,dn) was defined as the plasma concentration just prior to the morning dose, with the last study intervention administration one day prior to the PK sampling.
Steady-state was considered achieved if the participant has received a stable dose for at least 3 days prior to collection of the plasma sample.
PK samples were collected at two protocol-specified visits per participant occurring between Week 16 and end of DB treatment period.
Exact visit timing varied by participant.
|
BW >=50kg: Predose between W16 to end of DB treatment period (maximum up to W212); BW >=25-<50kg: Predose between W16 to end of DB treatment period (maximum up to W198); BW >=9- <25kg: Predose between W16 to end of DB treatment period (maximum up to W175)
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Colaboradores e Investigadores
É aqui que você encontrará pessoas e organizações envolvidas com este estudo.
Patrocinador
Investigadores
- Diretor de estudo: Actelion Clinical Trial, Actelion
Datas de registro do estudo
Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.
Datas Principais do Estudo
Início do estudo (Real)
16 de janeiro de 2020
Conclusão Primária (Real)
11 de outubro de 2024
Conclusão do estudo (Estimado)
1 de outubro de 2027
Datas de inscrição no estudo
Enviado pela primeira vez
8 de novembro de 2019
Enviado pela primeira vez que atendeu aos critérios de CQ
21 de novembro de 2019
Primeira postagem (Real)
25 de novembro de 2019
Atualizações de registro de estudo
Última Atualização Postada (Real)
28 de agosto de 2026
Última atualização enviada que atendeu aos critérios de controle de qualidade
27 de agosto de 2026
Última verificação
1 de agosto de 2026
Mais Informações
Termos relacionados a este estudo
Termos MeSH relevantes adicionais
- Doenças Vasculares
- Doenças cardiovasculares
- Doenças Respiratórias
- Doenças pulmonares
- Hipertensão
- Hipertensão Pulmonar
- Administração de Serviços de Saúde
- Qualidade, acesso e avaliação da assistência médica
- Qualidade de assistência médica
- Indicadores de qualidade, assistência médica
- Padrão de atendimento
- SELEXIPAG
- Proteína PDE5A, humana
Outros números de identificação do estudo
- CR108716
- 2019 (Concessão/Contrato do NIH dos EUA: Chief Medical Office (CMO) Alberta Health Services)
- 2019-002817-21 (Número EudraCT)
- AC-065A310 (Outro identificador: Janssen Research & Development, LLC)
- 2022-501012-34-00 (Identificador de registro: EUCT number)
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
SIM
Descrição do plano IPD
A política de compartilhamento de dados da Janssen Pharmaceutical Companies of Johnson & Johnson está disponível em www.janssen.com/clinical-trials/transparency.
Conforme observado neste site, as solicitações de acesso aos dados do estudo podem ser enviadas por meio do site do Projeto Yale Open Data Access (YODA) em yoda.yale.edu
Informações sobre medicamentos e dispositivos, documentos de estudo
Estuda um medicamento regulamentado pela FDA dos EUA
Sim
Estuda um produto de dispositivo regulamentado pela FDA dos EUA
Não
Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .