폐동맥고혈압을 앓는 소아의 표준 치료에 대한 추가 치료로서 셀렉시팍에 대한 연구 (SALTO)
2026년 8월 27일 업데이트: Actelion
소아 표준 치료에 대한 추가 치료로서 셀렉시팍의 효능 및 안전성을 평가하기 위한 공개 연장 기간을 포함하는 무작위, 다기관, 이중맹검, 위약 대조, 병렬 그룹, 사건 중심, 그룹 순차 연구 세 >=2 ~
이 연구의 목적은 표준 치료 치료에 셀렉시팍을 추가하는 것이 위약과 비교하여 폐동맥 고혈압(PAH)이 있는 소아의 질병 진행을 지연시키는지 여부를 평가하는 것입니다.
연구 개요
상태
모집하지 않고 적극적으로
정황
상세 설명
소아 PAH는 상당한 이환율 및 사망률과 관련된 희귀하고 진행성인 장애입니다.
PAH가 있는 어린이의 치료법을 개발해야 하는 상당한 의학적 필요성을 감안할 때 어린이의 PAH 관리에 대한 더 많은 데이터를 제공하기 위해 소아 인구에 대한 추가 임상 연구가 필요합니다.
Selexipag(JNJ-67896049)는 PAH가 있는 성인 참여자의 치료를 위해 승인되고 상업적으로 이용 가능한 프로스타사이클린 수용체의 경구용 선택적 장기 작용 비프로스타노이드 작용제입니다.
Selexipag 및 그 대사산물은 항섬유화, 항증식 및 항혈전 특성을 가지고 있습니다.
현재 프로스타사이클린 경로를 표적으로 하는 약물은 PAH에서 소아용으로 승인되지 않았습니다.
PAH 질병의 의학적으로 적절한 단계에 도입된 selexipag와 같은 프로스타사이클린 수용체에 작용하는 효과적이고 경구적으로 이용 가능한 치료법은 불충분한 질병 통제로 인해 추가 요법이 필요한 참여자에게 주로 현재의 1차 경구 PAH 특정 약물과 병용됩니다. PAH 소아 참가자의 치료 관리에 대한 주요 발전을 나타냅니다.
이 연구는 최대 6주의 스크리닝 기간과 상향 적정 및 유지 기간을 포함한 이중 맹검 치료 기간, 이후 3년의 오픈 라벨 연장 기간(OLEP) 및 30일의 안전성 추적으로 구성됩니다. 연구 중재(이중 맹검 또는 공개 라벨)의 마지막 투여 후 발생하는 최대 기간.
안전성, 약동학 및 효능 평가는 연구 중에 수행될 것입니다.
데이터를 지속적으로 모니터링하고 중간 데이터를 검토하며 이 연구에 등록한 참가자의 지속적인 안전을 보장하기 위해 독립 데이터 모니터링 위원회(IDMC)가 설립될 것입니다.
대략적인 연구 기간은 8년입니다.
연구 유형
중재적
등록 (실제)
138
단계
- 3단계
연락처 및 위치
이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.
연구 장소
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Kaohsiung City, 대만, 813414
- Kaohsiung Veterans General Hospital
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Tainan, 대만, 704
- National Cheng Kung University Hospital
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Taipei, 대만, 100
- National Taiwan University Hospital
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Seoul, 대한민국, 03080
- Seoul National University Hospital
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Seoul, 대한민국, 06351
- Samsung Medical Center
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Seoul, 대한민국, 120-752
- Severance Hospital Yonsei University Health System
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Yangsan, 대한민국, 50612
- Pusan National University Yangsan Hospital
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Freiburg im Breisgau, 독일, 70106
- Universitätsklinikum Freiburg Zentrum
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Heidelberg, 독일, D-69120
- Universitaetsklinikum Heidelberg
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Leipzig, 독일, 04289
- Herzzentrum Leipzig GmbH
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München, 독일, 81377
- Klinikum der Universitaet Muenchen
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Kazan', 러시아 제국, 420012
- Kazan State Medical University
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Kazan', 러시아 제국, 420059
- Kazan State Medical University 1
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Kemerovo, 러시아 제국, 650002
- Scientific and Research Institution of Cardiovascular Diseases Complex Problems
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Moscow, 러시아 제국, 125373
- Childrens City Clinical Hospital n.a. Bashlyaeva
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Moscow, 러시아 제국, 125412
- Veltischev Research and Clinical Institute for Pediatrics of the Pirogov RNRMU
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Samara, 러시아 제국, 443070
- Samara Regional Clinical Cardiological Dispensary
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Vilnius, 리투아니아, LT08661
- Vilnius University Hospital Santariskiu Clinics
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Kuala Lumpur, 말레이시아, 50400
- National Heart Institute
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Guadalajara, 멕시코, 44160
- CICUM San Miguel
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Monterrey, 멕시코, 64718
- Unidad de Investigacion Clinica en Medicina S.C. (UDICEM)
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México, 멕시코, 52787
- Operadora de Hospitales Angeles SA de CV Hospital Angeles Lomas
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Arizona
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Phoenix, Arizona, 미국, 85016
- Phoenix Children's Hospital
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California
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Los Angeles, California, 미국, 90095
- UCLA Medical Center
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San Francisco, California, 미국, 94158
- UCSF
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Colorado
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Aurora, Colorado, 미국, 80045
- Childrens Hospital Colorado
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District of Columbia
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Washington D.C., District of Columbia, 미국, 20010
- Children's National Medical Center
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Florida
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Gainesville, Florida, 미국, 32610
- Congenital Heart Center of the University of Florida
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Indiana
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Indianapolis, Indiana, 미국, 46202
- Riley Hospital for Children
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Michigan
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Detroit, Michigan, 미국, 48201
- Detroit Medical Center
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Ohio
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Cincinnati, Ohio, 미국, 45229
- Cincinnati Children's Hospital Medical Center
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Pennsylvania
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Philadelphia, Pennsylvania, 미국, 19104
- Childrens Hospital Of Philadelphia
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Texas
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Houston, Texas, 미국, 77030
- Texas Children's Hospital
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Utah
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Salt Lake City, Utah, 미국, 84113
- Primary Children's Hospital
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Virginia
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Charlottesville, Virginia, 미국, 22908
- University of Virginia Division of Pediatric Cardiology
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Hanoi, 베트남
- Hanoi Medical University Hospital
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Ho Chi Minh City, 베트남, 700000
- University Medical Center Ho Chi Minh City
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Ho Chi Minh City, 베트남
- Children's Hospital 1
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Ho Chi Minh City, 베트남, 700000
- Tam Anh Hospital
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Brussels, 벨기에, 1070
- ULB Hôpital Erasme
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Ghent, 벨기에, 9000
- Universitair Ziekenhuis Gent
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Leuven, 벨기에, 3000
- Universitaire Ziekenhuizen Leuven
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Minsk, 벨라루스, 220013
- State Institution Republican Scientific And Practical Center For Pediatric Surgery
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Minsk, 벨라루스, 220118
- Health Institution 4Th City Children'S Clinical Hospital
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Sofia, 불가리아, 1309
- Multiprofile Hospital For Active Treatment National Cardiology Hospital, Ead
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Blumenau, 브라질, 89030-101
- Complexo de Prevencao,Diagnostico,Terapia e Reabilitacao Respiratoria LTDA Hospital Dia do Pulmao
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Brasília, 브라질, 70310-500
- Fundacao Universitaria de Cardiologia - Instituto de Cardiologia e Transplantes do DF
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Curitiba, 브라질, 80250-060
- Hospital Pequeno Principe
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Fortaleza, 브라질, 60840-285
- Secretaria da Saude do Estado do Ceara - Hospital Doutor Carlos Alberto Studart Gomes
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Porto Alegre, 브라질, 90020-090
- Irmandade Santa Casa de Misericordia de Porto Alegre
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Porto Alegre, 브라질, 90620-001
- Fundação Universitaria de Cardiologia
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São Paulo, 브라질, 01221-020
- Irmandade Santa Casa de Misericordia de Sao Paulo
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São Paulo, 브라질, 04024 002
- SPDM - Associacao Paulista para o Desenvolvimento da Medicina - Hospital Sao Paulo
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Belgrade, 세르비아, 11000
- Univerzitetska Dečja Klinika
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Gothenburg, 스웨덴, 416 50
- Drottning Silvias barn- och ungdomssjukhus
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Lund, 스웨덴, 222 42
- Skånes universitetssjukhus
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Lausanne, 스위스, 1011
- Centre hospitalier universitaire vaudois CHUV
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A Coruña, 스페인, 15006
- Hosp Univ A Coruna
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Barcelona, 스페인, 08035
- Hosp Univ Vall D Hebron
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Esplugues de Llobregat, 스페인, 08950
- Hosp. Sant Joan de Deu
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Madrid, 스페인, 28046
- Hosp. Univ. La Paz
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Madrid, 스페인, 28009
- Hosp. Gral. Univ. Gregorio Maranon
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Seville, 스페인, 41013
- Hosp. Virgen Del Rocio
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Dublin, 아일랜드
- Our Lady's Children's Hospital
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Dnipro, 우크라이나, 49006
- Dnipropetrovsk clinical medical center of Mother and Child after prof. Rudnev
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Dnipro, 우크라이나, 49070
- MI 'Dnipropetrovsk Regional Clinical Center of Cardiology and Cardiac Surgery'
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Kyiv, 우크라이나, 04050
- Scientific Practical Medical Center for Pediatric Cardiology and Cardio Surgery of the MOH
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Zaporizhzhya, 우크라이나, 69063
- MI Zaporizhzhia Regional Clinical Childrens Hospital of Zaporizhzhia Regional Council
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Haifa, 이스라엘, 3109601
- Rambam Medical Center
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Ramat Gan, 이스라엘, 52621
- Sheba Medical Center
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Bologna, 이탈리아, 40138
- Azienda Ospedaliera Policlinico S. Orsola-Malpighi
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Milan, 이탈리아, 20162
- Asst Grande Ospedale Metropolitano Niguarda
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Padova, 이탈리아
- Universta Degli Studi Di Padova
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Roma, 이탈리아, 00193
- Ospedale Pediatrico Bambin Gesù
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S. Donato Milanese, 이탈리아, 20097
- IRCCS Policlinico San Donato
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Torino, 이탈리아, 10126
- AOU Città della Salute e della Scienza di Torino, Presidio Ospedale Infantile Regina Margherita
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Beijing, 중국, 100029
- Beijing Anzhen Hospital
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Guangzhou, 중국, 510623
- Guangzhou Women And Children's Medical Center
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Qingdao, 중국, 266000
- Qingdao Women and Children's Hospital
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Qingdao, 중국, 266000
- Qingdao Women and Children's Hospital 1
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Shanghai, 중국, 201102
- Children's Hospital of Fudan University
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Shanghai, 중국, 200127
- Shanghai Childrens Medical Center
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Shenyang, 중국, 110000
- The General Hospital of Northern Theater Command
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Alberta
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Edmonton, Alberta, 캐나다, T6G 2B7
- Stollery Children'S Hospital
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Ontario
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Toronto, Ontario, 캐나다, M5G 1X8
- Hospital for Sick Children
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Bogotá, 콜롬비아
- Fundacion Santa Fe de Bogota
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Bogotá, 콜롬비아, 0000000
- Clinica San Rafael
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Bogotá, 콜롬비아, 0000000
- Fundacion Neumologica Colombiana
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Cali, 콜롬비아, 760042
- Clínica Imbanaco S.A.S.
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Piedecuesta, 콜롬비아, 681017
- Fundación Cardiovascular de Colombia
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Soledad, 콜롬비아, 0000000
- Hospital Universidad del Norte
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Chiang Mai, 태국, 50200
- Chiang Mai University Hospital
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Songkhla, 태국, 90110
- Songklanagarind hospital
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Adana, 터키 (Türkiye), 01790
- Cukurova Balcali Hospital Application and Research Center
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Ankara, 터키 (Türkiye), 06230
- Hacettepe University Medical Faculty
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Istanbul, 터키 (Türkiye), 34093
- CAPA Istanbul University Medical Faculty
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Istanbul, 터키 (Türkiye), 34303
- Mehmet Akif Ersoy Training and Research Hospital
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Izmir, 터키 (Türkiye), 35020
- Izmir Tepecik Training and Research Hospital
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Izmir, 터키 (Türkiye), 35210
- Behcet Uz Pediatric Diseases and Surgery Training and Research Hospital
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Lisbon, 포르투갈, 1169-024
- Uls Sao Jose - Hosp. Santa Marta
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Porto, 포르투갈, 4200 319
- Uls Sao Joao - Hosp. Sao Joao
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Gdansk, 폴란드, 80 952
- Uniwersyteckie Centrum Kliniczne
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Krakow, 폴란드, 30-663
- Uniwersytecki Szpital Dzieciecy w Krakowie
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Poznan, 폴란드, 60 572
- Szpital Kliniczny im Karola Jonschera
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Warsaw, 폴란드, 04-730
- Instytut Pomnik Centrum Zdrowia Dziecka
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Wroclaw, 폴란드, 51 124
- Wojewodzki Szpital Specjalistyczny We Wroclawiu
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Zabrze, 폴란드, 41-800
- Slaskie Centrum Chorob Serca
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Lille, 프랑스, 59037
- Hôpital Cardiologique - Chru Lille
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Marseille, 프랑스, 13385
- Hopital de la Timone
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Montpellier, 프랑스, 34295
- CHU Arnaud de Villeneuve
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Paris, 프랑스, 75015
- Hopital Necker - Enfants Malades
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Pessac, 프랑스, 33604
- Hôpital Cardiologique Du Haut-Lévêque
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Toulouse, 프랑스, 31059
- Chu Hopital Des Enfants
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Helsinki, 핀란드, 29
- New Children's Hospital of the Helsinki University Hospital (HUS)
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Budapest, 헝가리, 1096
- Gottsegen György Országos Kardiológiai Intézet
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South Brisbane, 호주, 4101
- Queensland Children's Hospital
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참여기준
연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.
자격 기준
공부할 수 있는 나이
2년 (어린이)
건강한 자원 봉사자를 받아들입니다
아니
설명
포함 기준:
- 2 이상(>=) 2에서 18세 미만(<) 사이의 참가자(무작위화 시 체중 >=9 kg)
- 참가자의 스크리닝 전 언제든지 수행된 문서화된 과거 우심장 도관법(RHC)에 의해 확인된 폐동맥 고혈압(PAH) 진단
- PAH(세계보건기구[WHO] 그룹 1), 다음 병인의 다운 증후군 참가자 포함: 특발성 PAH(IPAH); 유전성 PAH(HPAH); 선천성 심장 질환과 관련된 PAH(선천성 심장 질환[aCHD]와 관련된 PAH)(일치하는 CHD[즉, 작은 심방 중격 결손, 심실 중격 결손 또는 상승된 PVR] 및 BCAC에서 승인한 경우) 및 수술 후 PAH(CHD 수리 후 >=6개월 지속/반복/진행); 약물 또는 독소 유도; 인간 면역결핍 바이러스(HIV)와 관련된 PAH
- WHO 기능 등급(FC) II 및 III
- 최소 1개의 PAH 특정 치료, 예를 들어 엔도텔린 수용체 길항제(ERA) 및/또는 포스포디에스테라제 5형(PDE-5) 억제제/가용성 구아닐레이트 시클라제 자극제로 치료받은 참가자. 단, 치료 용량은 연구 개입의 첫 투여 전 최소 3개월 동안 안정적
제외 기준:
- 문맥압항진증, 주혈흡충증, 폐정맥 폐쇄성 질환 및/또는 폐모세혈관종증으로 인한 PAH
- 아이젠멩거 증후군과 관련된 PAH
- Uptravi(selexipag)에 대한 이전 노출
- 기대 수명이 12개월 미만인 것으로 알려진 수반되는 생명을 위협하는 질병
- 임신, 임신 계획 또는 수유
- 셀렉시팍 또는 그 부형제에 대한 알려진 알레르기, 과민성 또는 편협
공부 계획
이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 병렬 할당
- 마스킹: 더블
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
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실험적: 셀렉시파그
참가자는 1일차에 체중에 기반하여 셀렉시파그를 투여받으며, 이후 하루 두 번 투여를 계속합니다.
셀렉시파그는 첫 12주 동안 참가자가 개별 최대 내약 용량(iMTD)에 도달하거나 기준 체중 범주에 해당하는 최대 용량이 달성될 때까지 용량을 증량합니다.
용량 증량 후 12주부터 치료 종료(EOT)까지 최대 내약 용량으로 유지 기간이 이어집니다.
참가자는 지역 표준 치료에 따라 폐동맥 고혈압(PAH)-특이적 동반 치료제(예: ERA, PDE-5 억제제, 가용성 구아닐산 고리화효소 자극제)를 계속 투여받습니다.
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Selexipag 정제는 구두로 투여됩니다.
다른 이름들:
ERAs는 표준 치료법으로 투여될 예정입니다.
PDE-5 억제제는 SOC 요법으로 투여될 것입니다.
용해성 구아닐산 사이클라제 자극제가 SOC 요법으로 투여될 것입니다.
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위약 비교기: 플라시보
참가자는 체중에 따라 Day 1에 위약을 투여받고, 이후 하루 두 번 투여를 계속하게 됩니다.
참가자는 지역 표준 치료에 따라 ERA, PDE-5 억제제, 가용성 구아닐산 시클라제 자극제와 같은 PAH 특이적 병용 치료를 계속해서 받게 됩니다.
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일치하는 위약 정제가 구두로 투여됩니다.
ERAs는 표준 치료법으로 투여될 예정입니다.
PDE-5 억제제는 SOC 요법으로 투여될 것입니다.
용해성 구아닐산 사이클라제 자극제가 SOC 요법으로 투여될 것입니다.
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실험적: 오픈라벨 연장 기간: 셀렉시팍
PAH에 대한 셀렉시팝의 긍정적 위험/이익 비율을 보인 참가자들은 오픈 라벨 확장 기간에 셀렉시팝을 제공받게 됩니다.
이중 맹검 치료 기간 동안 셀렉시팝을 복용 중이던 참가자들은 OLEP 기간 동안 개별 최대 내약 용량(iMTD)으로 치료를 계속하며, 이전에 위약을 복용했던 참가자들의 경우 첫 12주 동안 셀렉시팝 용량을 증량하여 참가자가 iMTD에 도달할 때까지 진행됩니다.
참가자들은 지역 표준 치료에 따라 ERA, PDE-5 억제제, 가용성 구아닐산 사이클라제 자극제와 같은 PAH 특이적 병용 치료를 계속 받게 됩니다.
|
Selexipag 정제는 구두로 투여됩니다.
다른 이름들:
ERAs는 표준 치료법으로 투여될 예정입니다.
PDE-5 억제제는 SOC 요법으로 투여될 것입니다.
용해성 구아닐산 사이클라제 자극제가 SOC 요법으로 투여될 것입니다.
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Double-blind Period: Time to Disease Progression
기간: 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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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Double-blind Period: Change in log2 N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) From Baseline to Week 24
기간: 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)
기간: 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
기간: 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)
기간: 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
기간: 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
기간: Baseline (Day 1), Weeks 24, 48, 72, and 96
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Change from baseline in growth parameter: body weight during DB period was reported.
The baseline value was the last non-missing assessment obtained before or on the DBSD.
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Baseline (Day 1), Weeks 24, 48, 72, and 96
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Double-blind Period: Change From Baseline in Growth Parameter: Height
기간: Baseline (Day 1), Weeks 24, 48, 72, and 96
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Change from baseline in growth parameter: height during DB period was reported.
The baseline value was the last non-missing assessment obtained before or on the DBSD.
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Baseline (Day 1), Weeks 24, 48, 72, and 96
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Double-blind Period: Number of Participants With Shift From Baseline in Sexual Maturation (Tanner Stage)
기간: Placebo arm: Baseline (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 198.42 weeks); Selexipag arm: Baseline (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 212.42 weeks)
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Tanner staging were assessed for pubic hair growth and genitalia development (GD) in males (M) and for pubic hair growth and breast development (BD) in females (F) in stages(S) 1 to 5 and missing data.
If a participant had reached Tanner stage 5, no further tanner pubertal stage assessment were to be completed.
Tanner stage was assessed in F >=8 years; M >=9 years.
Pubic hair growth (M and F) stages: S1: No hair, S2: Downy hair, S3: More coarse and curly hair, S4: Adult-like hair quality; S5: Hair extends to medial surface of thighs.
BD in females: S1: Nipple raised a little, rest of breast still flat, S2: Breast bud forms, S3: More elevated, outside areola, S4: Increased breast size, S5: Final adult-size breasts.
GD in males: S1: Testes, scrotum, and penis about same size, S2: Enlargement of scrotum, testes, and penis, S3: Enlargement of penis, S4: Penis and glands became larger, S5: Genitalia size and shape same an adult male.
Categories with at least 1 non-zero data are reported.
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Placebo arm: Baseline (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 198.42 weeks); Selexipag arm: Baseline (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 212.42 weeks)
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Double-blind Period: Number of Participants With Treatment-emergent Electrocardiogram (ECG) Abnormalities
기간: 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 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.
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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 Treatment-emergent (TE) Marked Laboratory Abnormalities
기간: 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 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.
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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: Change From Baseline in Treatment-emergent Laboratory Parameter: Thyroid Stimulating Hormone
기간: Baseline (Day 1), Weeks 24, 48, 72, and 96
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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.
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Baseline (Day 1), Weeks 24, 48, 72, and 96
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Double-blind Period: Change From Baseline in Treatment-emergent Laboratory Parameter: Hemoglobin
기간: Baseline (Day 1), Weeks 24, 48, 72, and 96
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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.
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Baseline (Day 1), Weeks 24, 48, 72, and 96
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Double-blind Period: Change From Baseline in Treatment-emergent Laboratory Parameter: Platelets, Leukocytes
기간: Baseline (Day 1), Weeks 24, 48, 72, and 96
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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.
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Baseline (Day 1), Weeks 24, 48, 72, and 96
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Double-blind Period: Change From Baseline in Treatment-emergent Laboratory Parameter: Electrolytes (Sodium, Potassium, Calcium, Bicarbonate and Chloride)
기간: Baseline (Day 1), Weeks 24, 48, 72, and 96
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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.
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Baseline (Day 1), Weeks 24, 48, 72, and 96
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Double-blind Period: Change From Baseline in Treatment-emergent Laboratory Parameter: Transaminases (Alanine Aminotransferase [ALT] and Aspartate Aminotransferase [AST])
기간: Baseline (Day 1), Weeks 24, 48, 72, and 96
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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.
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Baseline (Day 1), Weeks 24, 48, 72, and 96
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Double-blind Period: Change From Baseline in Treatment-emergent Laboratory Parameter: Bilirubin
기간: Baseline (Day 1), Weeks 24, 48, 72, and 96
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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.
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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)
기간: Placebo arm: Randomization (Day 1) up to 7 days after last dose in the DB treatment (maximum up to 199 weeks); Selexipag arm: Randomization (Day 1) up to 7 days after last dose in the DB treatment (maximum up to 213 weeks)
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Time to first CEC-confirmed hospitalization or death for PAH was calculated as the onset date of the first CEC-confirmed death or hospitalization due to PAH minus date of randomization+1 day.
Kaplan-Meier method was used for estimation.
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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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Double-blind Period: Dose-normalized Steady-state Trough Concentrations (Ctrough,ss,dn) of Selexipag and Its Metabolite ACT-333679 by Age Cohort
기간: Cohort (C) 1: Predose between Week (W) 16 to end of DB treatment period (maximum up to W212); C 2: Predose between Week 16 to end of DB treatment period (maximum up to W198); C 3: Predose between Week 16 to end of DB treatment period (maximum up to W96)
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Dose-normalized trough plasma concentration at steady-state (Ctrough,ss,dn) was defined as the plasma concentration just prior to the morning dose, with the last study intervention administration one day prior to the PK sampling.
Steady-state was considered achieved if the participant has received a stable dose for at least 3 days prior to collection of the plasma sample.
PK samples were collected at two protocol-specified visits per participant occurring between Week 16 and end of DB treatment period.
Exact visit timing varied by participant.
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Cohort (C) 1: Predose between Week (W) 16 to end of DB treatment period (maximum up to W212); C 2: Predose between Week 16 to end of DB treatment period (maximum up to W198); C 3: Predose between Week 16 to end of DB treatment period (maximum up to W96)
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Double-blind Period: Dose-normalized Steady-state Trough Concentrations (Ctrough,ss,dn) of Selexipag and Its Metabolite ACT-333679 by Body Weight (BW)
기간: 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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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.
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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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공동 작업자 및 조사자
여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.
스폰서
수사관
- 연구 책임자: Actelion Clinical Trial, Actelion
연구 기록 날짜
이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.
연구 주요 날짜
연구 시작 (실제)
2020년 1월 16일
기본 완료 (실제)
2024년 10월 11일
연구 완료 (추정된)
2027년 10월 1일
연구 등록 날짜
최초 제출
2019년 11월 8일
QC 기준을 충족하는 최초 제출
2019년 11월 21일
처음 게시됨 (실제)
2019년 11월 25일
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
2026년 8월 28일
QC 기준을 충족하는 마지막 업데이트 제출
2026년 8월 27일
마지막으로 확인됨
2026년 8월 1일
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- CR108716
- 2019 (미국 NIH 보조금/계약: Chief Medical Office (CMO) Alberta Health Services)
- 2019-002817-21 (EudraCT 번호)
- AC-065A310 (기타 식별자: Janssen Research & Development, LLC)
- 2022-501012-34-00 (레지스트리 식별자: EUCT number)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
예
IPD 계획 설명
Johnson & Johnson의 Janssen 제약 회사의 데이터 공유 정책은 www.janssen.com/clinical-trials/transparency에서 확인할 수 있습니다.
이 사이트에 명시된 바와 같이 연구 데이터에 대한 액세스 요청은 Yale open Data Access(YODA) 프로젝트 사이트(yoda.yale.edu)를 통해 제출할 수 있습니다.
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
예
미국 FDA 규제 기기 제품 연구
아니
이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .