En studie av Selexipag som tilleggsbehandling til standardbehandling hos barn med pulmonal arteriell hypertensjon (SALTO)
27. august 2026 oppdatert av: Actelion
En randomisert, multisenter, dobbeltblind, placebokontrollert, parallellgruppe, hendelsesdrevet, gruppesekvensiell studie med åpen forlengelsesperiode for å vurdere effektiviteten og sikkerheten til Selexipag som tilleggsbehandling til standarden for omsorg hos barn Alder >=2 til
Hensikten med denne studien er å evaluere om tillegg av selexipag til standardbehandling forsinker sykdomsprogresjonen hos barn med pulmonal arteriell hypertensjon (PAH) sammenlignet med placebo.
Studieoversikt
Status
Aktiv, ikke rekrutterende
Forhold
Detaljert beskrivelse
Pediatrisk PAH er en sjelden og progressiv lidelse assosiert med betydelig sykelighet og dødelighet.
Gitt det betydelige medisinske behovet for å utvikle behandlinger hos barn med PAH, er det derfor nødvendig med ytterligere kliniske studier i den pediatriske populasjonen for å gi mer data for behandling av PAH hos barn.
Selexipag (JNJ-67896049) er en oralt tilgjengelig, selektiv og langtidsvirkende ikke-prostanoid agonist av prostacyklinreseptoren godkjent og kommersielt tilgjengelig for behandling av voksne deltakere med PAH.
Selexipag og dets metabolitt har anti-fibrotiske, anti-proliferative og anti-trombotiske egenskaper.
Foreløpig er ingen medisiner rettet mot prostacyklinveien godkjent for pediatrisk bruk ved PAH.
En effektiv og oralt tilgjengelig terapi som virker på prostacyklinreseptoren slik som selexipag introdusert på medisinsk passende stadium av PAH-sykdom, og primært i kombinasjon med gjeldende førstelinje orale PAH-spesifikke medisiner hos deltakere som trenger tilleggsbehandling på grunn av utilstrekkelig sykdomskontroll. representerer et stort fremskritt for den terapeutiske behandlingen av PAH pediatriske deltakere.
Denne studien består av en screeningperiode på opptil 6 uker og en dobbeltblind behandlingsperiode, inkludert opptitrerings- og vedlikeholdsperioder, etterfulgt av en 3-års åpen forlengelsesperiode (OLEP) og en 30-dagers sikkerhetsoppfølging. opp periode som oppstår etter siste dose av studieintervensjon (enten dobbeltblind eller åpen).
Sikkerhets-, farmakokinetiske og effektvurderinger vil bli utført i løpet av studien.
En uavhengig dataovervåkingskomité (IDMC) vil bli opprettet for å overvåke data på løpende basis, for å gjennomgå midlertidige data og for å sikre den fortsatte sikkerheten til deltakerne som er registrert i denne studien.
Studiets omtrentlige varighet er 8 år.
Studietype
Intervensjonell
Registrering (Faktiske)
138
Fase
- Fase 3
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiesteder
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South Brisbane, Australia, 4101
- Queensland Children's Hospital
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Brussels, Belgia, 1070
- ULB Hôpital Erasme
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Ghent, Belgia, 9000
- Universitair Ziekenhuis Gent
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Leuven, Belgia, 3000
- Universitaire Ziekenhuizen Leuven
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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, Bulgaria, 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, Forente stater, 85016
- Phoenix Children's Hospital
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California
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Los Angeles, California, Forente stater, 90095
- UCLA Medical Center
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San Francisco, California, Forente stater, 94158
- UCSF
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Colorado
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Aurora, Colorado, Forente stater, 80045
- Childrens Hospital Colorado
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District of Columbia
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Washington D.C., District of Columbia, Forente stater, 20010
- Children's National Medical Center
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Florida
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Gainesville, Florida, Forente stater, 32610
- Congenital Heart Center of the University of Florida
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Indiana
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Indianapolis, Indiana, Forente stater, 46202
- Riley Hospital for Children
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Michigan
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Detroit, Michigan, Forente stater, 48201
- Detroit Medical Center
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Ohio
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Cincinnati, Ohio, Forente stater, 45229
- Cincinnati Children's Hospital Medical Center
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Pennsylvania
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Philadelphia, Pennsylvania, Forente stater, 19104
- Childrens Hospital of Philadelphia
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Texas
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Houston, Texas, Forente stater, 77030
- Texas Children's Hospital
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Utah
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Salt Lake City, Utah, Forente stater, 84113
- Primary Children's Hospital
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Virginia
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Charlottesville, Virginia, Forente stater, 22908
- University of Virginia Division of Pediatric Cardiology
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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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Minsk, Hviterussland, 220013
- State Institution Republican Scientific And Practical Center For Pediatric Surgery
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Minsk, Hviterussland, 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, Italia, 40138
- Azienda Ospedaliera Policlinico S. Orsola-Malpighi
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Milan, Italia, 20162
- ASST Grande Ospedale Metropolitano Niguarda
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Padova, Italia
- Universta Degli Studi Di Padova
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Roma, Italia, 00193
- Ospedale Pediatrico Bambin Gesù
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S. Donato Milanese, Italia, 20097
- IRCCS Policlinico San Donato
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Torino, Italia, 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', Russland, 420012
- Kazan State Medical University
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Kazan', Russland, 420059
- Kazan State Medical University 1
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Kemerovo, Russland, 650002
- Scientific and Research Institution of Cardiovascular Diseases Complex Problems
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Moscow, Russland, 125373
- Childrens City Clinical Hospital n.a. Bashlyaeva
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Moscow, Russland, 125412
- Veltischev Research and Clinical Institute for Pediatrics of the Pirogov RNRMU
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Samara, Russland, 443070
- Samara Regional Clinical Cardiological Dispensary
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Belgrade, Serbia, 11000
- Univerzitetska Dečja Klinika
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A Coruña, Spania, 15006
- Hosp Univ A Coruna
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Barcelona, Spania, 08035
- Hosp Univ Vall D Hebron
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Esplugues de Llobregat, Spania, 08950
- Hosp. Sant Joan de Deu
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Madrid, Spania, 28046
- Hosp. Univ. La Paz
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Madrid, Spania, 28009
- Hosp. Gral. Univ. Gregorio Maranon
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Seville, Spania, 41013
- Hosp. Virgen Del Rocio
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Lausanne, Sveits, 1011
- Centre hospitalier universitaire vaudois CHUV
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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, Sør -Korea, 03080
- Seoul National University Hospital
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Seoul, Sør -Korea, 06351
- Samsung Medical Center
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Seoul, Sør -Korea, 120-752
- Severance Hospital Yonsei University Health System
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Yangsan, Sør -Korea, 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, Tyrkia (Türkiye), 01790
- Cukurova Balcali Hospital Application and Research Center
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Ankara, Tyrkia (Türkiye), 06230
- Hacettepe University Medical Faculty
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Istanbul, Tyrkia (Türkiye), 34093
- CAPA Istanbul University Medical Faculty
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Istanbul, Tyrkia (Türkiye), 34303
- Mehmet Akif Ersoy Training and Research Hospital
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Izmir, Tyrkia (Türkiye), 35020
- Izmir Tepecik Training and Research Hospital
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Izmir, Tyrkia (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, 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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Deltakelseskriterier
Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
2 år til 17 år (Barn)
Tar imot friske frivillige
Nei
Beskrivelse
Inklusjonskriterier:
- Deltakere mellom større enn eller lik (>=) 2 og under (<) 18 år som veier >=9 kilogram (kg) ved randomisering
- Pulmonal arteriell hypertensjon (PAH) diagnose bekreftet av dokumentert historisk høyre hjertekateterisering (RHC) utført når som helst før deltakerens screening
- PAH (Verdens helseorganisasjon [WHO] gruppe 1), inkludert deltakere med Downs syndrom, av følgende etiologier: Idiopatisk PAH (IPAH); arvelig PAH (HPAH); PAH assosiert med medfødt hjertesykdom (PAH assosiert med medfødt hjertesykdom [aCHD]) (PAH med tilfeldig CHD [det vil si en liten atrieseptumdefekt, ventrikkelseptumdefekt eller patentert ductus arteriosus som ikke i seg selv står for utviklingen av forhøyet PVR] og hvis godkjent av BCAC) og postoperativ PAH (vedvarende/tilbakevendende/utvikler >=6 måneder etter reparasjon av CHD); Legemiddel- eller toksinindusert; PAH assosiert med humant immunsviktvirus (HIV)
- WHO funksjonsklasse (FC) II og III
- Deltakere behandlet med minst 1 PAH-spesifikk behandling, for eksempel en endotelinreseptorantagonist (ERA) og/eller en fosfodiesterase type-5 (PDE-5) hemmer/oppløselig guanylatsyklasestimulator, forutsatt at behandlingsdosen(e) er blitt behandlet. stabil i minst 3 måneder før første dose av studieintervensjon
Ekskluderingskriterier:
- PAH på grunn av portal hypertensjon, schistosomiasis, pulmonal veno-okklusiv sykdom og/eller pulmonal kapillær hemangiomatose
- PAH assosiert med Eisenmenger syndrom
- Tidligere eksponering for Uptravi (selexipag)
- Kjent samtidig livstruende sykdom med forventet levealder <12 måneder
- Gravid, planlegger å bli gravid eller ammende
- Kjente allergier, overfølsomhet eller intoleranse overfor selexipag eller dets hjelpestoffer
Studieplan
Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: Randomisert
- Intervensjonsmodell: Parallell tildeling
- Masking: Dobbelt
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
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Eksperimentell: Selexipag
Deltakerne vil motta selexipag basert på kroppsvekten på dag 1 og vil fortsette deretter med to ganger daglig dosering.
Selexipag vil bli opptitrert i løpet av de første 12 ukene inntil deltakerne når den individuelle maksimalt tolererte dosen (iMTD) eller inntil en maksimal dose som tilsvarer deres baseline kroppsvektskategori er oppnådd.
Opptitrering etterfølges av en vedlikeholdsperiode etter uke 12 inntil behandlingsslutt (EOT), ved den maksimalt tolererte dosen.
Deltakerne vil fortsette å motta pulmonal arteriell hypertensjon (PAH)-spesifikk samtidig behandling som ERAer, PDE-5-hemmere og løselig guanylatcyklase-stimulator i henhold til lokal standard behandling.
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Selexipag tablett vil bli administrert oralt.
Andre navn:
ERAs vil bli administrert som SOC-behandling.
PDE-5-hemmer vil bli administrert som standard behandling.
Løselig guanylatcyklase-stimulator vil bli administrert som SOC-terapi.
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Placebo komparator: Placebo
Deltakere vil få matchende placebo basert på kroppsvekten på dag 1 og vil fortsette deretter med dosering to ganger daglig.
Deltakere vil fortsette å motta PAH-spesifikke samtidige terapier som ERAer, PDE-5-hemmere og løyseleg guanylatcyklase-stimulator i henhold til lokal standardbehandling.
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Matchende placebotabletter vil bli administrert oralt.
ERAs vil bli administrert som SOC-behandling.
PDE-5-hemmer vil bli administrert som standard behandling.
Løselig guanylatcyklase-stimulator vil bli administrert som SOC-terapi.
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Eksperimentell: Åpen merkeutvidelsesperiode: Selexipag
Deltakere med et positivt nytte/risiko-forhold for selexipag for PAH vil bli tilbudt selexipag i den åpne utvidelsesperioden.
Deltakere på selexipag under den dobbeltblindede behandlingsperioden vil fortsette behandlingen på sin iMTD under OLEP; for de som tidligere var på placebo, vil iMTD øke selexipag i løpet av de første 12 ukene til deltakeren når iMTD.
Deltakere vil fortsette å motta PAH-spesifikke samtidige terapier som ERAer, PDE-5-hemmere og stimulator av løselig guanylatcyklase i henhold til lokal standard praksis.
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Selexipag tablett vil bli administrert oralt.
Andre navn:
ERAs vil bli administrert som SOC-behandling.
PDE-5-hemmer vil bli administrert som standard behandling.
Løselig guanylatcyklase-stimulator vil bli administrert som SOC-terapi.
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Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
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 |
Tiltaksbeskrivelse |
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)
|
Number of participants with AEs leading to premature discontinuation of study treatment during DB period was reported.
An AE was any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product.
An AE does not necessarily have a causal relationship with the intervention.
|
Placebo arm: Start of treatment (Day 1) up to last dose in the DB treatment (maximum up to 198 weeks); Selexipag arm: Start of treatment (Day 1) up to last dose in the DB treatment (maximum up to 212 weeks)
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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.
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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
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.
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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
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
|
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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)
|
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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
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)
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Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
Etterforskere
- Studieleder: Actelion Clinical Trial, Actelion
Studierekorddatoer
Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.
Studer hoveddatoer
Studiestart (Faktiske)
16. januar 2020
Primær fullføring (Faktiske)
11. oktober 2024
Studiet fullført (Antatt)
1. oktober 2027
Datoer for studieregistrering
Først innsendt
8. november 2019
Først innsendt som oppfylte QC-kriteriene
21. november 2019
Først lagt ut (Faktiske)
25. november 2019
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
28. august 2026
Siste oppdatering sendt inn som oppfylte QC-kriteriene
27. august 2026
Sist bekreftet
1. august 2026
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
- Vaskulære sykdommer
- Kardiovaskulære sykdommer
- Sykdommer i luftveiene
- Lungesykdommer
- Hypertensjon
- Hypertensjon, lunge
- Health Services Administration
- Helsevesenets kvalitet, tilgang og evaluering
- Kvalitet på helsehjelpen
- Kvalitetsindikatorer, helsehjelp
- Standard for omsorg
- selexipag
- PDE5A protein, menneske
Andre studie-ID-numre
- CR108716
- 2019 (U.S. NIH-stipend/kontrakt: Chief Medical Office (CMO) Alberta Health Services)
- 2019-002817-21 (EudraCT-nummer)
- AC-065A310 (Annen identifikator: Janssen Research & Development, LLC)
- 2022-501012-34-00 (Registeridentifikator: EUCT number)
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
JA
IPD-planbeskrivelse
Datadelingspolicyen til Janssen Pharmaceutical Companies of Johnson & Johnson er tilgjengelig på www.janssen.com/clinical-trials/transparency.
Som nevnt på dette nettstedet, kan forespørsler om tilgang til studiedata sendes gjennom Yale open Data Access (YODA) Project-nettstedet på yoda.yale.edu
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Ja
Studerer et amerikansk FDA-regulert enhetsprodukt
Nei
Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .