En undersøgelse af Selexipag som tilføjelsesbehandling til standardbehandling hos børn med pulmonal arteriel hypertension (SALTO)
27. august 2026 opdateret af: Actelion
En randomiseret, multicenter, dobbeltblind, placebokontrolleret, parallelgruppe, hændelsesdrevet, gruppesekventiel undersøgelse med åben forlængelsesperiode for at vurdere effektiviteten og sikkerheden af Selexipag som tilføjelsesbehandling til standarden for pleje hos børn Alder >=2 til
Formålet med denne undersøgelse er at evaluere, om tilføjelsen af selexipag til standardbehandling forsinker sygdomsprogression hos børn med pulmonal arteriel hypertension (PAH) sammenlignet med placebo.
Studieoversigt
Status
Aktiv, ikke rekrutterende
Betingelser
Detaljeret beskrivelse
Pædiatrisk PAH er en sjælden og progressiv lidelse forbundet med betydelig morbiditet og dødelighed.
I betragtning af det betydelige medicinske behov for at udvikle behandlinger til børn med PAH, er der derfor behov for yderligere kliniske undersøgelser i den pædiatriske population for at tilvejebringe flere data til behandling af PAH hos børn.
Selexipag (JNJ-67896049) er en oralt tilgængelig, selektiv og langtidsvirkende ikke-prostanoid agonist af prostacyclinreceptoren godkendt og kommercielt tilgængelig til behandling af voksne deltagere med PAH.
Selexipag og dets metabolit har anti-fibrotiske, anti-proliferative og anti-trombotiske egenskaber.
I øjeblikket er ingen medicin rettet mod prostacyclin-vejen godkendt til pædiatrisk brug ved PAH.
En effektiv og oralt tilgængelig terapi, der virker på prostacyclinreceptoren, såsom selexipag, introduceret på et medicinsk passende stadie af PAH-sygdom, og primært i kombination med nuværende første-line orale PAH-specifikke lægemidler til deltagere med behov for yderligere behandling på grund af utilstrækkelig sygdomskontrol repræsenterer et stort fremskridt til den terapeutiske behandling af PAH pædiatriske deltagere.
Denne undersøgelse består af en screeningsperiode på op til 6 uger og en dobbeltblind behandlingsperiode, inklusive optitrerings- og vedligeholdelsesperioder, efterfulgt af en 3-årig åben forlængelsesperiode (OLEP) og en 30-dages sikkerhedsopfølgning. op-periode, der opstår efter den sidste dosis af undersøgelsesintervention (enten dobbeltblind eller åben-label).
Sikkerheds-, farmakokinetiske og effektivitetsvurderinger vil blive udført under undersøgelsen.
En uafhængig dataovervågningskomité (IDMC) vil blive etableret til at overvåge data på en løbende basis, for at gennemgå foreløbige data og for at sikre den fortsatte sikkerhed for deltagerne, der er tilmeldt denne undersøgelse.
Den omtrentlige varighed af undersøgelsen er 8 år.
Undersøgelsestype
Interventionel
Tilmelding (Faktiske)
138
Fase
- Fase 3
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiesteder
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South Brisbane, Australien, 4101
- Queensland Children's 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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Alberta
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Edmonton, Alberta, Canada, T6G 2B7
- Stollery Children's Hospital
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Ontario
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Toronto, Ontario, Canada, M5G 1X8
- Hospital for Sick Children
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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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Arizona
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Phoenix, Arizona, Forenede Stater, 85016
- Phoenix Children's Hospital
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California
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Los Angeles, California, Forenede Stater, 90095
- UCLA Medical Center
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San Francisco, California, Forenede Stater, 94158
- UCSF
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Colorado
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Aurora, Colorado, Forenede Stater, 80045
- Childrens Hospital Colorado
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District of Columbia
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Washington D.C., District of Columbia, Forenede Stater, 20010
- Children's National Medical Center
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Florida
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Gainesville, Florida, Forenede Stater, 32610
- Congenital Heart Center of the University of Florida
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Indiana
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Indianapolis, Indiana, Forenede Stater, 46202
- Riley Hospital for Children
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Michigan
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Detroit, Michigan, Forenede Stater, 48201
- Detroit Medical Center
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Ohio
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Cincinnati, Ohio, Forenede Stater, 45229
- Cincinnati Children's Hospital Medical Center
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Pennsylvania
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Philadelphia, Pennsylvania, Forenede Stater, 19104
- Childrens Hospital of Philadelphia
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Texas
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Houston, Texas, Forenede Stater, 77030
- Texas Children's Hospital
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Utah
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Salt Lake City, Utah, Forenede Stater, 84113
- Primary Children's Hospital
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Virginia
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Charlottesville, Virginia, Forenede Stater, 22908
- University of Virginia Division of Pediatric Cardiology
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Lille, Frankrig, 59037
- Hôpital Cardiologique - Chru Lille
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Marseille, Frankrig, 13385
- Hôpital de la Timone
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Montpellier, Frankrig, 34295
- CHU Arnaud de Villeneuve
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Paris, Frankrig, 75015
- Hopital Necker - Enfants Malades
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Pessac, Frankrig, 33604
- Hôpital Cardiologique Du Haut-Lévêque
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Toulouse, Frankrig, 31059
- Chu Hopital Des Enfants
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Minsk, Hviderusland, 220013
- State Institution Republican Scientific And Practical Center For Pediatric Surgery
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Minsk, Hviderusland, 220118
- Health Institution 4Th City Children'S Clinical Hospital
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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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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, Mexico, 44160
- CICUM San Miguel
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Monterrey, Mexico, 64718
- Unidad de Investigacion Clinica en Medicina S.C. (UDICEM)
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México, Mexico, 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', Rusland, 420012
- Kazan State Medical University
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Kazan', Rusland, 420059
- Kazan State Medical University 1
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Kemerovo, Rusland, 650002
- Scientific and Research Institution of Cardiovascular Diseases Complex Problems
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Moscow, Rusland, 125373
- Childrens City Clinical Hospital n.a. Bashlyaeva
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Moscow, Rusland, 125412
- Veltischev Research and Clinical Institute for Pediatrics of the Pirogov RNRMU
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Samara, Rusland, 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, Tyrkiet (Türkiye), 01790
- Cukurova Balcali Hospital Application and Research Center
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Ankara, Tyrkiet (Türkiye), 06230
- Hacettepe University Medical Faculty
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Istanbul, Tyrkiet (Türkiye), 34093
- CAPA Istanbul University Medical Faculty
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Istanbul, Tyrkiet (Türkiye), 34303
- Mehmet Akif Ersoy Training and Research Hospital
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Izmir, Tyrkiet (Türkiye), 35020
- Izmir Tepecik Training and Research Hospital
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Izmir, Tyrkiet (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, Ukraine, 49006
- Dnipropetrovsk clinical medical center of Mother and Child after prof. Rudnev
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Dnipro, Ukraine, 49070
- MI 'Dnipropetrovsk Regional Clinical Center of Cardiology and Cardiac Surgery'
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Kyiv, Ukraine, 04050
- Scientific Practical Medical Center for Pediatric Cardiology and Cardio Surgery of the MOH
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Zaporizhzhya, Ukraine, 69063
- MI Zaporizhzhia Regional Clinical Childrens Hospital of Zaporizhzhia Regional Council
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Budapest, Ungarn, 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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Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
2 år til 17 år (Barn)
Tager imod sunde frivillige
Ingen
Beskrivelse
Inklusionskriterier:
- Deltagere mellem større end eller lig med (>=) 2 og under (<) 18 år, der vejer >=9 kilogram (kg) ved randomisering
- Pulmonal arteriel hypertension (PAH) diagnose bekræftet af dokumenteret historisk højre hjertekateterisering (RHC) udført på et hvilket som helst tidspunkt før deltagerens screening
- PAH (World Health Organization [WHO] Gruppe 1), inklusive deltagere med Downs syndrom, af følgende ætiologier: Idiopatisk PAH (IPAH); arvelig PAH (HPAH); PAH associeret med medfødt hjertesygdom (PAH-associeret med medfødt hjertesygdom [aCHD]) (PAH med tilfældig CHD [dvs. en lille atrial septal defekt, ventrikulær septal defekt eller patent ductus arteriosus, der ikke i sig selv forklarer udviklingen af forhøjet PVR] og hvis godkendt af BCAC) og postoperativ PAH (vedvarende/tilbagevendende/udvikling >=6 måneder efter reparation af CHD); Lægemiddel- eller toksin-induceret; PAH forbundet med humant immundefektvirus (HIV)
- WHO funktionsklasse (FC) II og III
- Deltagere behandlet med mindst 1 PAH-specifik behandling, f.eks. en endotelinreceptorantagonist (ERA) og/eller en phosphodiesterase type-5 (PDE-5) hæmmer/opløselig guanylatcyclasestimulator, forudsat at behandlingsdosis(-erne) er blevet stabil i mindst 3 måneder før første dosis af undersøgelsesintervention
Ekskluderingskriterier:
- PAH på grund af portal hypertension, schistosomiasis, pulmonal veno-okklusiv sygdom og/eller pulmonær kapillær hæmangiomatose
- PAH forbundet med Eisenmenger syndrom
- Tidligere eksponering for Uptravi (selexipag)
- Kendt samtidig livstruende sygdom med en forventet levetid <12 måneder
- Gravid, planlægger at blive gravid eller ammer
- Kendte allergier, overfølsomhed eller intolerance over for selexipag eller dets hjælpestoffer
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Randomiseret
- Interventionel model: Parallel tildeling
- Maskning: Dobbelt
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
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Eksperimentel: Selexipag
Deltagerne vil modtage selexipag baseret på kropsvægten på dag 1 og vil fortsætte herefter med to gange daglig dosering.
Selexipag vil blive optitrateret i løbet af de første 12 uger, indtil deltagerne når den individuelle maksimale tolererede dosis (iMTD) eller indtil en maksimal dosis svarende til deres baseline kropsvægtkategori er opnået.
Optitrering efterfølges af en vedligeholdelsesperiode efter uge 12 indtil behandlingens afslutning (EOT) ved den maksimalt tolererede dosis.
Deltagerne vil fortsætte med at modtage pulmonal arteriel hypertension (PAH)-specifikke samtidige behandlinger såsom ERAs, PDE-5-hæmmere og opløselig guanylatcyklase-stimulator i henhold til lokal standardbehandling.
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Selexipag tablet vil blive indgivet oralt.
Andre navne:
ERA'er vil blive administreret som SOC-terapi.
PDE-5-hæmmer vil blive administreret som SOC-terapi.
Stimulator af opløselig guanylat-cyklase vil blive administreret som SOC-terapi.
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Placebo komparator: Placebo
Deltagerne vil modtage et matchende placebo baseret på kropsvægten på dag 1 og vil fortsætte herefter med dosering to gange dagligt.
Deltagerne vil fortsat modtage PAH-specifikke samtidige behandlinger såsom ERA'er, PDE-5-hæmmere og stimulatorer af opløselig guanylatcyklase i henhold til lokal standardbehandling.
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Matchende placebotabletter vil blive indgivet oralt.
ERA'er vil blive administreret som SOC-terapi.
PDE-5-hæmmer vil blive administreret som SOC-terapi.
Stimulator af opløselig guanylat-cyklase vil blive administreret som SOC-terapi.
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Eksperimentel: Åben etiket forlængelsesperiode: Selexipag
Deltagere med et positivt fordel/risiko-forhold for selexipag til PAH vil blive tilbudt selexipag i den åbne forlængelsesperiode.
Deltagere på selexipag under den dobbeltblindede behandlingsperiode vil fortsætte behandlingen på deres iMTD under OLEP, for dem, der tidligere var på placebo, vil iMTD øges med selexipag i de første 12 uger, indtil deltageren når iMTD.
Deltagere vil fortsat modtage PAH-specifikke samtidige behandlinger såsom ERA'er, PDE-5-hæmmere og stimulator af opløselig guanylatcyklase i henhold til lokal standardbehandling.
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Selexipag tablet vil blive indgivet oralt.
Andre navne:
ERA'er vil blive administreret som SOC-terapi.
PDE-5-hæmmer vil blive administreret som SOC-terapi.
Stimulator af opløselig guanylat-cyklase vil blive administreret som SOC-terapi.
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Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
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Double-blind Period: Time to Disease Progression
Tidsramme: 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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Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
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Double-blind Period: Change in log2 N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) From Baseline to Week 24
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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])
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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)
|
Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Efterforskere
- Studieleder: Actelion Clinical Trial, Actelion
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart (Faktiske)
16. januar 2020
Primær færdiggørelse (Faktiske)
11. oktober 2024
Studieafslutning (Anslået)
1. oktober 2027
Datoer for studieregistrering
Først indsendt
8. november 2019
Først indsendt, der opfyldte QC-kriterier
21. november 2019
Først opslået (Faktiske)
25. november 2019
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
28. august 2026
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
27. august 2026
Sidst verificeret
1. august 2026
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- CR108716
- 2019 (U.S. NIH-bevilling/kontrakt: Chief Medical Office (CMO) Alberta Health Services)
- 2019-002817-21 (EudraCT nummer)
- AC-065A310 (Anden identifikator: Janssen Research & Development, LLC)
- 2022-501012-34-00 (Registry Identifier: EUCT number)
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
JA
IPD-planbeskrivelse
Datadelingspolitikken for Janssen Pharmaceutical Companies of Johnson & Johnson er tilgængelig på www.janssen.com/clinical-trials/transparency.
Som nævnt på dette websted kan anmodninger om adgang til undersøgelsesdata indsendes via Yale open Data Access (YODA) Project-websted på yoda.yale.edu
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Ja
Studerer et amerikansk FDA-reguleret enhedsprodukt
Ingen
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