A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension
Study Purpose
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.
Recruitment Criteria
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Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms |
No |
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Study Type
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies. |
Interventional |
| Eligible Ages | 2 Years - 17 Years |
| Gender | All |
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.
Trial Details
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Trial ID:
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries. |
NCT04175600 |
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Phase
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use. |
Phase 3 |
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Lead Sponsor
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data. |
Actelion |
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Principal Investigator
The person who is responsible for the scientific and technical direction of the entire clinical study. |
Actelion Clinical Trial |
| Principal Investigator Affiliation | Actelion |
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Agency Class
Category of organization(s) involved as sponsor (and collaborator) supporting the trial. |
Industry |
| Overall Status | Active, not recruiting |
| Countries | Australia, Belarus, Belgium, Brazil, Bulgaria, Canada, China, Colombia, Finland, France, Germany, Hungary, Ireland, Israel, Italy, Korea, Republic of, Lithuania, Malaysia, Mexico, Poland, Portugal, Russian Federation, Serbia, Spain, Sweden, Switzerland, Taiwan, Thailand, Turkey, Ukraine, United States, Vietnam |
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Conditions
The disease, disorder, syndrome, illness, or injury that is being studied. |
Hypertension, Pulmonary |
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.
Arms
Experimental: 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.
Placebo Comparator: Placebo
Participants will receive matching placebo based on the body weight on Day 1 and will continue thereafter with twice daily dosing.
Interventions
Drug: - Selexipag
Selexipag tablet will be administered orally.
Drug: - Placebo
Matching placebo tablets will be administered orally.
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.
Status
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Phoenix Children's Hospital
Phoenix, Arizona, 85016
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UCLA Medical Center
Los Angeles, California, 90095
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UCSF
San Francisco, California, 94158
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Childrens Hospital Colorado
Aurora, Colorado, 80045
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Children's National Medical Center
Washington, District of Columbia, 20010
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Congenital Heart Center of the University of Florida
Gainesville, Florida, 32610
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Riley Hospital for Children
Indianapolis, Indiana, 46202
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Detroit Medical Center
Detroit, Michigan, 48201
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
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Childrens Hospital Of Philadelphia
Philadelphia, Pennsylvania, 19104
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Texas Children's Hospital
Houston, Texas, 77030
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Primary Children's Hospital
Salt Lake City, Utah, 84113
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University of Virginia Division of Pediatric Cardiology
Charlottesville, Virginia, 22908
International Sites
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Queensland CHILDREN'S HOSPITAL
South Brisbane, , 4101
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State Institution Republican Scientific And Practical Center For Pediatric Surgery
Minsk, , 220013
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Health Institution 4Th City Children'S Clinical Hospital
Minsk, , 220118
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ULB Hôpital Erasme
Brussels, , 1070
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Universitair Ziekenhuis Gent
Gent, , 9000
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Universitaire Ziekenhuizen Leuven
Leuven, , 3000
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Complexo de Prevencao,Diagnostico,Terapia e Reabilitacao Respiratoria LTDA Hospital Dia do Pulmao
Blumenau, , 89030-101
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Fundacao Universitaria de Cardiologia - Instituto de Cardiologia e Transplantes do DF
Brasilia, , 70310-500
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Hospital Pequeno Principe
Curitiba, , 80250-060
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Secretaria da Saude do Estado do Ceara - Hospital Doutor Carlos Alberto Studart Gomes
Fortaleza, , 60840-285
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Address
Irmandade Santa Casa de Misericordia de Porto Alegre
Porto Alegre, , 90020-090
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Fundacao Universitaria de Cardiologia
Porto Alegre, , 90620-001
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Address
Irmandade Santa Casa de Misericordia de Sao Paulo
Sao Paulo, , 01221-020
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Address
SPDM - Associacao Paulista para o Desenvolvimento da Medicina - Hospital Sao Paulo
São Paulo, , 04024-002
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Address
Multiprofile Hospital For Active Treatment National Cardiology Hospital, Ead
Sofia, , 1309
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Stollery Children's Hospital
Edmonton, Alberta, T6G 2B7
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Hospital For Sick Children
Toronto, Ontario, M5G 1X8
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Beijing Anzhen Hospital
Beijing, , 100029
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Guangzhou Women And Children's Medical Center
Guangzhou, , 510623
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Qingdao Women and Children's Hospital
Qingdao, , 266000
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Shanghai Childrens Medical Center
Shanghai, , 200127
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Children's Hospital of Fudan University
Shanghai, , 201102
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The General Hospital of Northern Theater Command
Shenyang, , 110000
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Clinica San Rafael
Bogota, , 0000000
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Fundacion Neumologica Colombiana
Bogota, , 0000000
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Fundacion Santa Fe de Bogota
Bogota, ,
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Clínica Imbanaco S.A.S.
Cali, , 760042
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Fundacion Cardiovascular de Colombia
Piedecuesta, , 681017
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Hospital Universidad del Norte
Soledad, , 0000000
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New Children's Hospital of the Helsinki University Hospital (HUS)
Helsinki, , 29
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Hôpital Cardiologique - Chru Lille
Lille Cedex, , 59037
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Hopital de la Timone
Marseille Cedex 5, , 13385
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CHU Arnaud de Villeneuve
Montpellier Cedex 5, , 34295
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Hôpital Necker - Enfants Malades
Paris, , 75015
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Hôpital Cardiologique Du Haut-Lévêque
Pessac, , 33604
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Chu Hopital Des Enfants
Toulouse Cedex 9, , 31059
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Universitätsklinikum Freiburg Zentrum
Freiburg, , 70106
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Universitaetsklinikum Heidelberg
Heidelberg, , D-69120
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Herzzentrum Leipzig GmbH
Leipzig, , 04289
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Klinikum der Universitaet Muenchen
München, , 81377
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Gottsegen György Országos Kardiológiai Intézet
Budapest, , 1096
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Our Lady's Children's Hospital
Dublin, ,
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Rambam Medical Center
Haifa, , 3109601
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Sheba Medical Center
Ramat Gan, , 52621
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Address
Azienda Ospedaliera Policlinico S. Orsola-Malpighi
Bologna, , 40138
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ASST Grande Ospedale Metropolitano Niguarda
Milano, , 20162
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Universta Degli Studi Di Padova
Padova, ,
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Ospedale Pediatrico Bambin Gesù
Roma, , 00193
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IRCCS Policlinico San Donato
S. Donato Milanese, , 20097
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AOU Città della Salute e della Scienza di Torino, Presidio Ospedale Infantile Regina Margherita
Torino, , 10126
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Seoul National University Hospital
Seoul, , 03080
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Address
Samsung Medical Center
Seoul, , 06351
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Severance Hospital Yonsei University Health System
Seoul, , 120-752
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Pusan National University Yangsan Hospital
Yangsan-si, , 50612
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Vilnius University Hospital Santariskiu Clinics
Vilnius, , LT08661
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National Heart Institute
Kuala Lumpur, , 50400
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CICUM San Miguel
Guadalajara, , 44160
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Operadora de Hospitales Angeles SA de CV Hospital Angeles Lomas
Mexico, , 52787
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Unidad de Investigacion Clinica en Medicina S.C. (UDICEM)
Monterrey, , 64718
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Uniwersyteckie Centrum Kliniczne
Gdansk, , 80-952
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Uniwersytecki Szpital Dzieciecy w Krakowie
Kraków, , 30-663
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Szpital Kliniczny im. Karola Jonschera
Poznan, , 60-572
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Instytut Pomnik Centrum Zdrowia Dziecka
Warszawa, , 04-730
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Wojewodzki Szpital Specjalistyczny we Wroclawiu
Wroclaw, , 51-124
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Slaskie Centrum Chorob Serca
Zabrze, , 41-800
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Hospital De Santa Marta
Lisboa, , 1169-024
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Centro Hospitalar de Sao Joao EPE
Porto, , 4200-319
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Kazan State Medical University
Kazan, , 420012
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Kazan State Medical University
Kazan, , 420059
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Cardiovascular Pathology Research Institute of Siberian Branch of RAMS
Kemerovo, , 650002
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Childrens City Clinical Hospital n.a. Bashlyaeva
Moscow, , 125373
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Address
Veltischev Research and Clinical Institute for Pediatrics of the Pirogov RNRMU
Moscow, , 125412
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Samara Regional Clinical Cardiological Dispensary
Samara, , 443070
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Univerzitetska Dečja Klinika
Belgrade, , 11000
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Hosp. Univ. A Coruna
A Coruña, , 15006
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Hosp. Univ. Vall D Hebron
Barcelona, , 08035
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Hosp. Sant Joan de Deu
Esplugues de Llobregat, , 08950
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Hosp. Gral. Univ. Gregorio Maranon
Madrid, , 28009
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Hosp. Univ. La Paz
Madrid, , 28046
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Hosp. Virgen Del Rocio
Sevilla, , 41013
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Drottning Silvias barn- och ungdomssjukhus
Gothenburg, , 416 50
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Skanes universitetssjukhus
Lund, , 222 42
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Centre Hospitalier Universitaire Vaudois (CHUV)
Lausanne, , 1011
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Kaohsiung Veterans General Hospital
Kaohsiung, , 813414
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National Cheng Kung University Hospital
Tainan, , 704
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National Taiwan University Hospital
Taipei, , 100
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Chiang Mai University Hospital
Chiang Mai, , 50200
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Songklanagarind hospital
Songkhla, , 90110
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Cukurova Balcali Hospital Application and Research Center
Adana, , 01380
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Hacettepe University Medical Faculty
Ankara, , 06230
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CAPA Istanbul University Medical Faculty
Istanbul, , 34093
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Mehmet Akif Ersoy Training and Research Hospital
Istanbul, , 34303
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Behcet Uz Pediatric Diseases and Surgery Training and Research Hospital
Izmir, , 35110
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Izmir Tepecik Training and Research Hospital
Izmir, , 35110
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Dnipropetrovsk clinical medical center of Mother and Child after prof. Rudnev
Dnipro, , 49006
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Address
MI 'Dnipropetrovsk Regional Clinical Center of Cardiology and Cardiac Surgery'
Dnipro, , 49070
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Address
Scientific Practical Medical Center for Pediatric Cardiology and Cardio Surgery of the MOH
Kyiv, , 04050
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MI Zaporizhzhia Regional Clinical Childrens Hospital of Zaporizhzhia Regional Council
Zaporizhzhya, , 69063
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Hanoi Medical University Hospital
Hanoi, ,
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Tam Anh Hospital
Ho Chi Minh, , 700000
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University Medical Center Ho Chi Minh city
Ho Chi Minh, , 700000
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Children's Hospital 1
Ho Chi Minh, ,
