Selexipag is available for the treatment of pulmonary arterial hypertension (PAH) in adults
in various countries. The efficacy of selexipag to delay disease progression was shown in a
previous pivotal study conducted in 1156 adult patients with PAH. Given the similarities in
the functional changes of PAH in children and adults, it is expected that children suffering
from PAH could benefit from treatment with selexipag. The aim of the present study is to
confirm the doses of selexipag to be used in pediatric patients with PAH older than 2 years.
To fulfill this aim, blood levels of selexipag (pharmacokinetic assessments) as well as the
safety, and tolerability of selexipag in children with PAH will be assessed.
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
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.
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.
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.
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
The person who is responsible for the scientific and technical direction of the entire clinical study.
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
The disease, disorder, syndrome, illness, or injury that is being studied.
The selection of the starting dose for pediatric patients is based on the pharmacokinetic
(PK) extrapolation from adults, taking into account the children body weight category, in
order to lead to an exposure similar to that in adult PAH patients at a starting dose of 200
microgram (μg). As in adults, selexipag will be up-titrated to the individual maximum
tolerated dose (iMTD) during the first 12 weeks.
Approximately 55 subjects will be enrolled in 3 different age cohorts to obtain at least 40
subjects with evaluable PK profiles: Cohort 1: ≥ 12 to < 18 years of age, Cohort 2: ≥ 6 to <
12 years of age, Cohort 3: ≥ 2 to < 6 years of age. In each age cohort the starting dose will
depend on the body weight. Enrollment will start with both Cohort 1 and Cohort 2. After
completion of PK assessments in at least 15 subjects from Cohort 1 at Week 12, a first
interim analysis will be conducted to establish the dose-exposure relationship using a
population PK model.
Results of this model-based analysis will be used to confirm or adjust the selexipag doses
initially selected. Enrollment of Cohort 3 (children ≥ 2 to < 6 years of age) will start once
the appropriate doses have been confirmed in a second interim analysis of PK data from
Cohorts 1 and 2, and if there is no safety concern based on review by an Independent Data
Monitoring Committee (IDMC).
Experimental: open label selexipag
The first dose of selexipag (Uptravi) will be administered in the evening of Day 1 and will be based on the body weight. Thereafter selexipag will be administered twice daily (morning and evening). Selexipag will be up-titrated during the first 12 weeks, with weekly increments equal to the starting dose until the subjects reach their individual maximum tolerated dose (iMTD) or until a maximum dose corresponding to their baseline weight category is achieved (which will be 8-fold of the corresponding starting dose). Up-titration is followed by a stable maintenance treatment period from Week 12 to Week 16, at the maximum tolerated dose. Thereafter, subjects will be treated with selexipag as long as the treatment is beneficial to the subject, as per investigator's decision
Drug: - selexipag (Uptravi)
Film-coated tablets for oral administration
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.
Children'S Hospital Cardiac Care Center University Of Colorado
Aurora, Colorado, 80045
University of Iowa Hospital
Iowa City, Iowa, 52242
Rochester, Minnesota, 55905
Columbia University Medical Center
New York, New York, 10032
Not yet recruiting
Vanderbilt University Medical Center
Nashville, Tennessee, 37232-2386
Seattle Children's Hospital
Seattle, Washington, 98105
State Institution Republican Scientific And Practical Center For Pediatric Surgery
Minsk, , 220013
Health Institution 4Th City Children'S Clinical Hospital
Minsk, , 220118
Gent, , 9000
Centre Hospitalier Sainte Justine
Montreal, Quebec, H3T 1C4
Stollery Children's Hospital
Edmonton, , AB T6G 2C8
Beijing Anzhen Hospital
Beijing, , 100029
Cardiovascular institute & Fuwai Hospital- Thrombus Center
Beijing, , 100037
Guangdong Provincial People's Hospital
Guangzhou, , 510080
Shanghai Children's Medical Center
Shanghai, , 200127
Wuhan Asia Heart Hospital
Wuhan, , 430022
CHU Arnaud de Villeneuve
Montpellier Cedex 5, , 34295
Hôpital Necker - Enfants Malades
Paris, , 75015
Chu Hopital Des Enfants
Toulouse Cedex 9, , 31059
Universitätsklinikum Freiburg Zentrum
Freiburg, , 70106
Justus-Liebig-Universität Gießen, Kinderherzzentrum
Giessen, , 35392
Universitäres Herzzentrum Hamburg
Hamburg, , 20246
Münster, , 48149
Gottsegen György Országos Kardiológiai Intézet, Felnőtt kardiológiai osztály
Budapest, , 1096
Schneider Children's Medical Center
Petach Tikvah, ,
Sheba Medical Center
Ramat Gan, , 52621
Sarawak General Hospital
Kota Samarahan, , 94300
Institut Jantung Negara (National Heart Institute)
Kuala Lumpur, , 50400
Uniwersyteckie Centrum Kliniczne
Gdansk, , 80-952
Uniwersytecki Szpital Dzieciecy w Krakowie
Kraków, , 30-663
Szpital Kliniczny im. Karola Jonschera
Poznan, , 60-572
Wojewódzki Szpital Specjalistyczny We Wrocławiu
Wrocław, , 51-124
Kazan State Medical University
Kazan, , 420059
Federal State Budget Scientific Institution
Kemerovo, , 650002
Moscow Scientific Research Institute For Pediatrics And Childrens Surgery Of Rosmedtechnologies
Moscow, , 125412
Samara Regional Clinical Cardiological Dispensary
Samara, , 443070
Federal State Budgetary Institution
St Petersburg, , 197341
Saint Petersburg State Pediatric Medical University
St. Petersburg, , 194100
Univerzitetska Dečja Klinika
Belgrade, , 11000
Institut Za Zdravstvenu Zaštitu Majke I Deteta Srbije ''Dr Vukan Čupić''
Belgrade, , 11070
National Cheng Kung University Hospital
Tainan, , 704
National Taiwan University Hospital
Taipei, , 100
Municipal Enterprise Of The Dnipropetrovsk Regional Council
Dnipro, , 49070
State Institution Of The Ministry Of Health Of Ukraine
Kiev, , 04050
Lviv Regional Clinical Hospital
Lviv, , 79010
Municipal Institution Of The Zaporizhzhya Regional Council
Zaporizhzhya, , 69063
Great Ormond Street Hospital
London, , WC1N 3JH