폐동맥고혈압을 앓는 소아의 표준 치료에 대한 추가 치료로서 셀렉시팍에 대한 연구 (SALTO)
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 ~
The purpose of this study is to evaluate whether the addition of selexipag to standard of care treatment delays disease progression in children with Pulmonary Arterial Hypertension (PAH) in comparison to placebo.
연구 개요
상태
상태
정황
정황
개입 / 치료
개입 / 치료
상세 설명
Pediatric PAH is a rare and progressive disorder associated with considerable morbidity and mortality. Given the significant medical need to develop treatments in children with PAH, further clinical studies in the pediatric population are therefore needed to provide more data for the management of PAH in children. Selexipag (JNJ-67896049) is an orally available, selective, and long-acting non-prostanoid agonist of the prostacyclin receptor approved and commercially available for the treatment of adult participants with PAH. Selexipag and its metabolite possess anti-fibrotic, anti-proliferative, and anti-thrombotic properties. Currently, no medicines targeting prostacyclin pathway are approved for pediatric use in PAH. An effective and orally available therapy acting on the prostacyclin receptor such as selexipag introduced at medically appropriate stage of PAH disease, and primarily in combination with current first-line oral PAH-specific medicines in participants in need of additional therapy because of insufficient disease control would represents a major advance to the therapeutic management of PAH pediatric participants. This study consists of a screening period of up to 6 weeks and a double-blind treatment period, including up-titration and maintenance periods, followed by a 3-year open-label extension period (OLEP) and a 30-day safety follow-up period that occurs after the last dose of study intervention (either double-blind or open-label). Safety, pharmacokinetic and efficacy assessments will be performed during the study. An Independent Data Monitoring Committee (IDMC) will be established to monitor data on an ongoing basis, to review interim data, and to ensure the continuing safety of the participants enrolled in this study. The approximate duration of the study is 8 years.
연구 유형
연구 유형
등록 (실제)
등록
단계
단계
- 3단계
연락처 및 위치
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
- Children's Hospital 1
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Ho Chi Minh City, 베트남, 700000
- Tam Anh Hospital
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Ho Chi Minh City, 베트남, 700000
- University Medical Center Ho Chi Minh city
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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
- Fundacao 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
- Skanes 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 Childrens 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, 200127
- Shanghai Childrens Medical Center
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Shanghai, 중국, 201102
- Children S Hospital of Fudan University
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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, 0000000
- Clinica San Rafael
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Bogotá, 콜롬비아, 0000000
- Fundacion Neumologica Colombiana
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Bogotá, 콜롬비아
- Fundacion Santa Fe de Bogota
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Cali, 콜롬비아, 760042
- Clínica Imbanaco S.A.S.
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Piedecuesta, 콜롬비아, 681017
- Fundacion 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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-
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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
- Hôpital 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
자격 기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
- Participants between greater than or equal to (>=) 2 and less than (<) 18 years of age weighing >=9 kilogram (kg) at randomization
- Pulmonary arterial hypertension (PAH) diagnosis confirmed by documented historical right heart catheterization (RHC) performed at any time before participant's screening
- PAH (World Health Organization [WHO] Group 1), including participants with Down syndrome, of the following etiologies: Idiopathic PAH (IPAH); Heritable PAH (HPAH); PAH associated with congenital heart disease (PAH-associated with congenital heart disease [aCHD]) (PAH with coincidental CHD [that is, a small atrial septal defect, ventricular septal defect, or patent ductus arteriosus that does not itself account for the development of elevated PVR] and if approved by the BCAC) and Post-operative PAH (persisting / recurring/ developing >=6 months after repair of CHD); Drug or toxin-induced; PAH associated with Human immunodeficiency virus (HIV)
- WHO functional class (FC) II and III
- Participants treated with at least 1 PAH-specific treatment, example, an Endothelin receptor antagonist (ERA) and/or a Phosphodiesterase type-5 (PDE-5) inhibitor/soluble guanylate cyclase stimulator, provided that the treatment dose(s) has been stable for at least 3 months prior to first dose of study intervention
Exclusion Criteria:
- PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease, and/or pulmonary capillary hemangiomatosis
- PAH associated with Eisenmenger syndrome
- Previous exposure to Uptravi (selexipag)
- Known concomitant life-threatening disease with a life expectancy <12 months
- Pregnant, planning to become pregnant, or lactating
- Known allergies, hypersensitivity, or intolerance to selexipag or its excipients
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 병렬 할당
- 마스킹: 더블
팔의 수
무기와 개입
참가자 그룹 / 팔참가자 그룹 / 팔 |
개입 / 치료개입 / 치료 |
|---|---|
|
실험적: Selexipag
Participants will receive selexipag based on the body weight on Day 1 and will continue thereafter with twice daily dosing. Selexipag will be uptitrated during the first 12 weeks until the participants reaches the individual maximum tolerated dose (iMTD) or until a maximum dose corresponding to their baseline body-weight category is achieved. Uptitration is followed by a maintenance period after Week 12 until end of treatment (EOT), at the maximum tolerated dose. Participants will continue to receive pulmonary arterial hypertension (PAH)-specific concomitant therapies such as ERAs, PDE-5 inhibitors, and soluble guanylate cyclase stimulator as per local standard-of-care. |
Selexipag tablet will be administered orally.
다른 이름들:
ERAs will be administered as SOC therapy. PDE-5 inhibitor will be administered as SOC therapy. Soluble guanylate cyclase stimulator will be administered as SOC therapy. |
|
위약 비교기: Placebo
Participants will receive matching placebo based on the body weight on Day 1 and will continue thereafter with twice daily dosing. Participants will continue to receive PAH-specific concomitant therapies such as ERAs, PDE-5 inhibitors, and soluble guanylate cyclase stimulator as per local standard-of-care. |
ERAs will be administered as SOC therapy. PDE-5 inhibitor will be administered as SOC therapy. Soluble guanylate cyclase stimulator will be administered as SOC therapy. Matching placebo tablets will be administered orally. |
|
실험적: Open-Label Extension Period: Selexipag
Participants with a positive benefit/risk ratio of selexipag for PAH will be offered selexipag in the open label extension period. Participants on selexipag during the double-blind treatment period will continue treatment at their iMTD during the OLEP, for those previously on placebo, the iMTD will uptitrate selexipag during first 12 weeks until participant reaches iMTD. Participants will continue to receive PAH-specific concomitant therapies such as ERAs, PDE-5 inhibitors, and soluble guanylate cyclase stimulator as per local standard-of-care. |
Selexipag tablet will be administered orally.
다른 이름들:
ERAs will be administered as SOC therapy. PDE-5 inhibitor will be administered as SOC therapy. Soluble guanylate cyclase stimulator will be administered as SOC therapy. |
연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
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)
|
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. |
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)
|
2차 결과 측정
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
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
|
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). |
Baseline (Day 1), Week 24
|
|
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)
|
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. |
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 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)
|
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)
|
|
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
|
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. |
Baseline (Day 1), Weeks 24, 48, 72, and 96
|
|
Double-blind Period: Change From Baseline in Vital Signs: Pulse Rate
기간: 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
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. |
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. |
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. |
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)
|
Number of participants with treatment-emergent ECG abnormalities were reported. Any abnormality occurring on or after the initial administration of study intervention through the day of last dose in the DB treatment period plus 3 days was considered to be treatment-emergent. Abnormalities that were not present at baseline were reported. Abnormalities were assessed as per investigator's discretion. The baseline value was the last non-missing assessment obtained before or on the DBSD. |
Placebo arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 198.42 weeks); Selexipag arm: Start of treatment (Day 1) up to 3 days after last dose in the DB treatment (maximum up to 212.42 weeks)
|
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Double-blind Period: Number of Participants With Treatment-emergent (TE) Marked Laboratory Abnormalities
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)
|
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)
|
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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. |
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. |
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. |
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. |
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. |
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. |
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)
|
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
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. |
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)
|
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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공동 작업자 및 조사자
수사관
수사관
- 연구 책임자: Actelion Clinical Trial, Actelion
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
연구 시작
기본 완료 (실제)
기본 완료
연구 완료 (추정된)
연구 완료
연구 등록 날짜
최초 제출
최초 제출
QC 기준을 충족하는 최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
처음 게시됨
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
마지막 업데이트 게시됨
QC 기준을 충족하는 마지막 업데이트 제출
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
기타 연구 ID 번호
- CR108716
- 2019-002817-21 (EudraCT 번호)
- AC-065A310 (기타 식별자: Janssen Research & Development, LLC)
- 2022-501012-34-00 (레지스트리 식별자: EUCT number)
- 2019 (미국 NIH 보조금/계약)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale open Data Access (YODA) Project site at yoda.yale.edu
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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