En studie av Selexipag som tilläggsbehandling till standarden för vård hos barn med pulmonell arteriell hypertoni (SALTO)
27 augusti 2026 uppdaterad av: Actelion
En randomiserad, multicenter, dubbelblind, placebokontrollerad, parallellgrupps-, händelsedriven, gruppsekventiell studie med öppen förlängningsperiod för att bedöma effektiviteten och säkerheten av Selexipag som tilläggsbehandling till standarden för vård hos barn Ålder >=2 till
Syftet med denna studie är att utvärdera om tillägg av selexipag till standardbehandling fördröjer sjukdomsprogression hos barn med pulmonell arteriell hypertension (PAH) jämfört med placebo.
Studieöversikt
Status
Aktiv, inte rekryterande
Betingelser
Detaljerad beskrivning
Pediatrisk PAH är en sällsynt och progressiv sjukdom associerad med betydande sjuklighet och dödlighet.
Med tanke på det betydande medicinska behovet av att utveckla behandlingar hos barn med PAH, behövs därför ytterligare kliniska studier i den pediatriska populationen för att tillhandahålla mer data för hantering av PAH hos barn.
Selexipag (JNJ-67896049) är en oralt tillgänglig, selektiv och långverkande icke-prostanoidagonist av prostacyklinreceptorn godkänd och kommersiellt tillgänglig för behandling av vuxna deltagare med PAH.
Selexipag och dess metabolit har antifibrotiska, antiproliferativa och antitrombotiska egenskaper.
För närvarande är inga läkemedel inriktade på prostacyklinvägen godkända för pediatrisk användning vid PAH.
En effektiv och oralt tillgänglig terapi som verkar på prostacyklinreceptorn såsom selexipag som introduceras i ett medicinskt lämpligt stadium av PAH-sjukdom, och främst i kombination med nuvarande förstahands orala PAH-specifika läkemedel hos deltagare i behov av ytterligare terapi på grund av otillräcklig sjukdomskontroll skulle representerar ett stort framsteg för den terapeutiska behandlingen av PAH pediatriska deltagare.
Denna studie består av en screeningperiod på upp till 6 veckor och en dubbelblind behandlingsperiod, inklusive upptitrerings- och underhållsperioder, följt av en 3-årig öppen förlängningsperiod (OLEP) och en 30-dagars säkerhetsuppföljning. uppperiod som inträffar efter den sista dosen av studieintervention (antingen dubbelblind eller öppen).
Säkerhets-, farmakokinetiska och effektbedömningar kommer att utföras under studien.
En oberoende dataövervakningskommitté (IDMC) kommer att inrättas för att övervaka data fortlöpande, för att granska interimsdata och för att säkerställa den fortsatta säkerheten för de deltagare som är inskrivna i denna studie.
Studiens ungefärliga varaktighet är 8 år.
Studietyp
Interventionell
Inskrivning (Faktisk)
138
Fas
- Fas 3
Kontakter och platser
Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.
Studieorter
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South Brisbane, Australien, 4101
- Queensland Children's Hospital
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Minsk, Belarus, 220013
- State Institution Republican Scientific And Practical Center For Pediatric Surgery
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Minsk, Belarus, 220118
- Health Institution 4Th City Children'S Clinical Hospital
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Brussels, Belgien, 1070
- ULB Hôpital Erasme
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Ghent, Belgien, 9000
- Universitair Ziekenhuis Gent
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Leuven, Belgien, 3000
- Universitaire Ziekenhuizen Leuven
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Blumenau, Brasilien, 89030-101
- Complexo de Prevencao,Diagnostico,Terapia e Reabilitacao Respiratoria LTDA Hospital Dia do Pulmao
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Brasília, Brasilien, 70310-500
- Fundacao Universitaria de Cardiologia - Instituto de Cardiologia e Transplantes do DF
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Curitiba, Brasilien, 80250-060
- Hospital Pequeno Principe
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Fortaleza, Brasilien, 60840-285
- Secretaria da Saude do Estado do Ceara - Hospital Doutor Carlos Alberto Studart Gomes
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Porto Alegre, Brasilien, 90020-090
- Irmandade Santa Casa de Misericordia de Porto Alegre
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Porto Alegre, Brasilien, 90620-001
- Fundação Universitaria de Cardiologia
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São Paulo, Brasilien, 01221-020
- Irmandade Santa Casa de Misericordia de Sao Paulo
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São Paulo, Brasilien, 04024 002
- SPDM - Associacao Paulista para o Desenvolvimento da Medicina - Hospital Sao Paulo
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Sofia, Bulgarien, 1309
- Multiprofile Hospital For Active Treatment National Cardiology Hospital, Ead
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Bogotá, Colombia
- Fundación Santa Fe de Bogotá
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Bogotá, Colombia, 0000000
- Clinica San Rafael
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Bogotá, Colombia, 0000000
- Fundacion Neumologica Colombiana
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Cali, Colombia, 760042
- Clínica Imbanaco S.A.S.
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Piedecuesta, Colombia, 681017
- Fundacion cardiovascular de Colombia
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Soledad, Colombia, 0000000
- Hospital Universidad del Norte
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Helsinki, Finland, 29
- New Children's Hospital of the Helsinki University Hospital (HUS)
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Lille, Frankrike, 59037
- Hôpital Cardiologique - Chru Lille
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Marseille, Frankrike, 13385
- Hôpital de la Timone
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Montpellier, Frankrike, 34295
- CHU Arnaud de Villeneuve
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Paris, Frankrike, 75015
- Hopital Necker - Enfants Malades
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Pessac, Frankrike, 33604
- Hôpital Cardiologique Du Haut-Lévêque
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Toulouse, Frankrike, 31059
- Chu Hopital Des Enfants
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Arizona
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Phoenix, Arizona, Förenta staterna, 85016
- Phoenix Children's Hospital
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California
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Los Angeles, California, Förenta staterna, 90095
- UCLA Medical Center
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San Francisco, California, Förenta staterna, 94158
- UCSF
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Colorado
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Aurora, Colorado, Förenta staterna, 80045
- Childrens Hospital Colorado
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District of Columbia
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Washington D.C., District of Columbia, Förenta staterna, 20010
- Children's National Medical Center
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Florida
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Gainesville, Florida, Förenta staterna, 32610
- Congenital Heart Center of the University of Florida
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Indiana
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Indianapolis, Indiana, Förenta staterna, 46202
- Riley Hospital for Children
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Michigan
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Detroit, Michigan, Förenta staterna, 48201
- Detroit Medical Center
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Ohio
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Cincinnati, Ohio, Förenta staterna, 45229
- Cincinnati Children's Hospital Medical Center
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Pennsylvania
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Philadelphia, Pennsylvania, Förenta staterna, 19104
- Childrens Hospital of Philadelphia
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Texas
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Houston, Texas, Förenta staterna, 77030
- Texas Children's Hospital
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Utah
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Salt Lake City, Utah, Förenta staterna, 84113
- Primary Children's Hospital
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Virginia
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Charlottesville, Virginia, Förenta staterna, 22908
- University of Virginia Division of Pediatric Cardiology
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Dublin, Irland
- 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, Italien, 40138
- Azienda Ospedaliera Policlinico S. Orsola-Malpighi
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Milan, Italien, 20162
- ASST Grande Ospedale Metropolitano Niguarda
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Padova, Italien
- Universta Degli Studi Di Padova
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Roma, Italien, 00193
- Ospedale Pediatrico Bambin Gesù
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S. Donato Milanese, Italien, 20097
- IRCCS Policlinico San Donato
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Torino, Italien, 10126
- AOU Città della Salute e della Scienza di Torino, Presidio Ospedale Infantile Regina Margherita
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Alberta
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Edmonton, Alberta, Kanada, T6G 2B7
- Stollery Children's Hospital
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Ontario
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Toronto, Ontario, Kanada, M5G 1X8
- Hospital for Sick Children
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Beijing, Kina, 100029
- Beijing Anzhen Hospital
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Guangzhou, Kina, 510623
- Guangzhou Women and Children's Medical Center
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Qingdao, Kina, 266000
- Qingdao Women and Children's Hospital
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Qingdao, Kina, 266000
- Qingdao Women and Children's Hospital 1
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Shanghai, Kina, 201102
- Children's Hospital of Fudan University
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Shanghai, Kina, 200127
- Shanghai Childrens Medical Center
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Shenyang, Kina, 110000
- The General Hospital of Northern Theater Command
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Vilnius, Litauen, LT08661
- Vilnius University Hospital Santariskiu Clinics
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Kuala Lumpur, Malaysia, 50400
- National Heart Institute
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Guadalajara, Mexiko, 44160
- CICUM San Miguel
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Monterrey, Mexiko, 64718
- Unidad de Investigacion Clinica en Medicina S.C. (UDICEM)
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México, Mexiko, 52787
- Operadora de Hospitales Angeles SA de CV Hospital Angeles Lomas
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Gdansk, Polen, 80 952
- Uniwersyteckie Centrum Kliniczne
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Krakow, Polen, 30-663
- Uniwersytecki Szpital Dzieciecy w Krakowie
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Poznan, Polen, 60 572
- Szpital Kliniczny im Karola Jonschera
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Warsaw, Polen, 04-730
- Instytut Pomnik Centrum Zdrowia Dziecka
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Wroclaw, Polen, 51 124
- Wojewodzki Szpital Specjalistyczny We Wroclawiu
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Zabrze, Polen, 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', Ryssland, 420012
- Kazan State Medical University
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Kazan', Ryssland, 420059
- Kazan State Medical University 1
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Kemerovo, Ryssland, 650002
- Scientific and Research Institution of Cardiovascular Diseases Complex Problems
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Moscow, Ryssland, 125373
- Childrens City Clinical Hospital n.a. Bashlyaeva
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Moscow, Ryssland, 125412
- Veltischev Research and Clinical Institute for Pediatrics of the Pirogov RNRMU
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Samara, Ryssland, 443070
- Samara Regional Clinical Cardiological Dispensary
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Lausanne, Schweiz, 1011
- Centre hospitalier universitaire vaudois CHUV
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Belgrade, Serbien, 11000
- Univerzitetska Dečja Klinika
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A Coruña, Spanien, 15006
- Hosp Univ A Coruna
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Barcelona, Spanien, 08035
- Hosp Univ Vall D Hebron
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Esplugues de Llobregat, Spanien, 08950
- Hosp. Sant Joan de Deu
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Madrid, Spanien, 28046
- Hosp. Univ. La Paz
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Madrid, Spanien, 28009
- Hosp. Gral. Univ. Gregorio Maranon
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Seville, Spanien, 41013
- Hosp. Virgen Del Rocio
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Gothenburg, Sverige, 416 50
- Drottning Silvias barn- och ungdomssjukhus
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Lund, Sverige, 222 42
- Skånes universitetssjukhus
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Seoul, Sydkorea, 03080
- Seoul National University Hospital
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Seoul, Sydkorea, 06351
- Samsung Medical Center
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Seoul, Sydkorea, 120-752
- Severance Hospital Yonsei University Health System
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Yangsan, Sydkorea, 50612
- Pusan National University Yangsan 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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Chiang Mai, Thailand, 50200
- Chiang Mai University Hospital
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Songkhla, Thailand, 90110
- Songklanagarind Hospital
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Adana, Turkiet (Türkiye), 01790
- Cukurova Balcali Hospital Application and Research Center
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Ankara, Turkiet (Türkiye), 06230
- Hacettepe University Medical Faculty
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Istanbul, Turkiet (Türkiye), 34093
- CAPA Istanbul University Medical Faculty
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Istanbul, Turkiet (Türkiye), 34303
- Mehmet Akif Ersoy Training and Research Hospital
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Izmir, Turkiet (Türkiye), 35020
- Izmir Tepecik Training and Research Hospital
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Izmir, Turkiet (Türkiye), 35210
- Behcet Uz Pediatric Diseases and Surgery Training and Research Hospital
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Freiburg im Breisgau, Tyskland, 70106
- Universitätsklinikum Freiburg Zentrum
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Heidelberg, Tyskland, D-69120
- Universitaetsklinikum Heidelberg
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Leipzig, Tyskland, 04289
- Herzzentrum Leipzig GmbH
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München, Tyskland, 81377
- Klinikum der Universitaet Muenchen
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Dnipro, Ukraina, 49006
- Dnipropetrovsk clinical medical center of Mother and Child after prof. Rudnev
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Dnipro, Ukraina, 49070
- MI 'Dnipropetrovsk Regional Clinical Center of Cardiology and Cardiac Surgery'
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Kyiv, Ukraina, 04050
- Scientific Practical Medical Center for Pediatric Cardiology and Cardio Surgery of the MOH
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Zaporizhzhya, Ukraina, 69063
- MI Zaporizhzhia Regional Clinical Childrens Hospital of Zaporizhzhia Regional Council
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Budapest, Ungern, 1096
- Gottsegen György Országos Kardiológiai Intézet
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Hanoi, Vietnam
- Hanoi Medical University Hospital
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Ho Chi Minh City, Vietnam, 700000
- University Medical Center Ho Chi Minh City
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Ho Chi Minh City, Vietnam
- Children's Hospital 1
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Ho Chi Minh City, Vietnam, 700000
- Tam Anh Hospital
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Deltagandekriterier
Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.
Urvalskriterier
Åldrar som är berättigade till studier
2 år till 17 år (Barn)
Tar emot friska volontärer
Nej
Beskrivning
Inklusionskriterier:
- Deltagare mellan större än eller lika med (>=) 2 och yngre än (<) 18 år som väger >=9 kilogram (kg) vid randomisering
- Diagnos av pulmonell arteriell hypertension (PAH) bekräftad av dokumenterad historisk högerhjärtkateterisering (RHC) utförd när som helst före deltagarens screening
- PAH (World Health Organization [WHO] Grupp 1), inklusive deltagare med Downs syndrom, av följande etiologier: Idiopatisk PAH (IPAH); Ärftlig PAH (HPAH); PAH associerad med medfödd hjärtsjukdom (PAH-associerad med medfödd hjärtsjukdom [aCHD]) (PAH med tillfällig hjärtsjukdom [det vill säga en liten förmaksseptumdefekt, ventrikulär septumdefekt eller patenterad ductus arteriosus som inte i sig förklarar utvecklingen av förhöjd PVR] och om det godkänns av BCAC) och postoperativ PAH (kvarstående / återkommande / utvecklas >=6 månader efter reparation av CHD); Läkemedels- eller toxin-inducerad; PAH associerad med humant immunbristvirus (HIV)
- WHO:s funktionsklass (FC) II och III
- Deltagare som behandlats med minst 1 PAH-specifik behandling, till exempel en endotelinreceptorantagonist (ERA) och/eller en fosfodiesteras typ-5 (PDE-5) hämmare/löslig guanylatcyklasstimulator, förutsatt att behandlingsdosen/-erna har varit stabil i minst 3 månader före första dos av studieintervention
Exklusions kriterier:
- PAH på grund av portal hypertoni, schistosomiasis, pulmonell veno-ocklusiv sjukdom och/eller pulmonell kapillär hemangiomatos
- PAH associerad med Eisenmengers syndrom
- Tidigare exponering för Uptravi (selexipag)
- Känd samtidig livshotande sjukdom med förväntad livslängd <12 månader
- Gravid, planerar att bli gravid eller ammar
- Kända allergier, överkänslighet eller intolerans mot selexipag eller dess hjälpämnen
Studieplan
Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Behandling
- Tilldelning: Randomiserad
- Interventionsmodell: Parallellt uppdrag
- Maskning: Dubbel
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
|---|---|
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Experimentell: Selexipag
Deltagarna kommer att få selexipag baserat på kroppsvikten på dag 1 och kommer därefter att fortsätta med två gånger daglig dosering.
Selexipag kommer att titreras upp under de första 12 veckorna tills deltagarna når den individuella maximalt tolererade dosen (iMTD) eller tills en maximal dos som motsvarar deras baslinjekroppsviktskategori uppnås.
Upptitrering följs av en underhållsperiod efter vecka 12 fram till behandlingens slut (EOT), med den maximalt tolererade dosen.
Deltagarna kommer att fortsätta att få pulmonell arteriell hypertension (PAH)-specifika samtidiga terapier såsom ERAs, PDE-5-hämmare och löslig guanylatcyklasstimulator enligt lokal standardvård.
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Selexipag tablett kommer att administreras oralt.
Andra namn:
ERAs kommer att administreras som SOC-terapi.
PDE-5-hämmare kommer att administreras som SOC-terapi.
Lösligt guanylatcyklasstimulerande medel kommer att administreras som SOC-terapi.
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Placebo-jämförare: Placebo
Deltagarna kommer att få matchande placebo baserat på kroppsvikten på dag 1 och kommer att fortsätta därefter med två gånger daglig dosering.
Deltagarna kommer att fortsätta att få PAH-specifik samtidig terapi såsom ERAs, PDE-5-hämmare och löslig guanylatcyklasstimulator enligt lokal standardvård.
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Matchande placebotabletter kommer att administreras oralt.
ERAs kommer att administreras som SOC-terapi.
PDE-5-hämmare kommer att administreras som SOC-terapi.
Lösligt guanylatcyklasstimulerande medel kommer att administreras som SOC-terapi.
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Experimentell: Öppen märkning Förlängningsperiod: Selexipag
Deltagare med ett positivt fördel-riskförhållande för selexipag för PAH kommer att erbjudas selexipag under den öppna förlängningsperioden.
Deltagare som tar selexipag under den dubbelblinda behandlingsperioden kommer att fortsätta behandlingen vid sin iMTD under OLEP. För de som tidigare fått placebo kommer iMTD att öka dosen av selexipag under de första 12 veckorna tills deltagaren når iMTD.
Deltagare kommer att fortsätta att få PAH-specifika samtidiga terapier såsom ERAs, PDE-5-hämmare och löslig guanylatcyklasstimulator enligt lokal standardvård.
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Selexipag tablett kommer att administreras oralt.
Andra namn:
ERAs kommer att administreras som SOC-terapi.
PDE-5-hämmare kommer att administreras som SOC-terapi.
Lösligt guanylatcyklasstimulerande medel kommer att administreras som SOC-terapi.
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Vad mäter studien?
Primära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
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Double-blind Period: Time to Disease Progression
Tidsram: 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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Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
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Double-blind Period: Change in log2 N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) From Baseline to Week 24
Tidsram: 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)
Tidsram: 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
Tidsram: 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)
Tidsram: 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
Tidsram: 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
Tidsram: 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.
|
Baseline (Day 1), Weeks 24, 48, 72, and 96
|
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Double-blind Period: Change From Baseline in Growth Parameter: Height
Tidsram: 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)
Tidsram: 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.
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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)
|
|
Double-blind Period: Number of Participants With Treatment-emergent Electrocardiogram (ECG) Abnormalities
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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)
Tidsram: 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])
Tidsram: 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
Tidsram: 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)
Tidsram: 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)
|
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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
Tidsram: 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)
Tidsram: 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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Samarbetspartners och utredare
Det är här du hittar personer och organisationer som är involverade i denna studie.
Sponsor
Utredare
- Studierektor: Actelion Clinical Trial, Actelion
Studieavstämningsdatum
Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.
Studera stora datum
Studiestart (Faktisk)
16 januari 2020
Primärt slutförande (Faktisk)
11 oktober 2024
Avslutad studie (Beräknad)
1 oktober 2027
Studieregistreringsdatum
Först inskickad
8 november 2019
Först inskickad som uppfyllde QC-kriterierna
21 november 2019
Första postat (Faktisk)
25 november 2019
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
28 augusti 2026
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
27 augusti 2026
Senast verifierad
1 augusti 2026
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- CR108716
- 2019 (U.S.S. NIH-anslag/kontrakt: Chief Medical Office (CMO) Alberta Health Services)
- 2019-002817-21 (EudraCT-nummer)
- AC-065A310 (Annan identifierare: Janssen Research & Development, LLC)
- 2022-501012-34-00 (Registeridentifierare: EUCT number)
Plan för individuella deltagardata (IPD)
Planerar du att dela individuella deltagardata (IPD)?
JA
IPD-planbeskrivning
Datadelningspolicyn för Janssen Pharmaceutical Companies of Johnson & Johnson finns på www.janssen.com/clinical-trials/transparency.
Som nämnts på den här webbplatsen kan förfrågningar om åtkomst till studiedata skickas in via Yale Open Data Access (YODA) projektwebbplats på yoda.yale.edu
Läkemedels- och apparatinformation, studiedokument
Studerar en amerikansk FDA-reglerad läkemedelsprodukt
Ja
Studerar en amerikansk FDA-reglerad produktprodukt
Nej
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