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Clinical Trials in the UK / NCT03492177
Active, not recruiting Phase 2

A Clinical Study of to Confirm the Doses of Selexipag in Children With Pulmonary Arterial Hypertension

NCT03492177 · tracked via the Priya Life Science UK tracker
Sponsor
Actelion
Phase
Phase 2
Started
2018-07-23
Last updated
2026-08-28

Condition(s) studied

Pulmonary Arterial Hypertension

Investigational drug(s) / intervention(s)

selexipag (Uptravi)

selexipag (Uptravi): Film-coated tablets for oral administration

Study summary

The purpose of this study to confirm the selexipag starting dose(s), selected based on pharmacokinetic (PK) extrapolation from adults, that leads to similar exposure as adults doses in children from greater than or equal to (\>=) 2 to less than (˂) 18 years of age with Pulmonary Arterial Hypertension (PAH), by investigating the PK of selexipag and its active metabolite ACT-333679 in this population.

Eligibility

Sex
ALL
Min age
2 Years
Max age
17 Years
Healthy volunteers
No
Inclusion Criteria: * Signed and dated informed consent by the parent(s) or Legally authorized representative(s) AND assent from developmentally capable children * Males or females between greater than or equal to (\>=) 2 and less than (\<) 18 years of age with weight \>= 9 kilograms (kg) * Pulmonary arterial hypertension (PAH) diagnosis confirmed by documented historical right heart catheterization (RHC) performed at any time before participant's enrollment * PAH with one of the following etiologies: * idiopathic (iPAH), * heritable (hPAH), * associated with congenital heart disease (CHD): PAH with co-incidental CHD; post-operative PAH (persisting/ recurring/ developing \>= 6 months after repair of CHD) * Drug or toxin-induced * PAH associated with HIV * PAH associated with connective tissue disease * Word Health Organization functional class (WHO FC) II to III * Participants treated with an endothelin receptor antagonist (ERA) and/or a phosphodiesterase type 5 (PDE-5) inhibitor provided that the treatment dose(s) has been stable for at least 3 months prior to enrollment, or participants who are not candidates for these therapies * Females of childbearing potential must have a negative pregnancy test at Screening and at Enrollment, and must agree to undertake monthly pregnancy tests, and to use a reliable method of contraception (if sexually active) from screening up to study drug discontinuation plus 30 days (EOS) Key Exclusion Criteria: * Participants with PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease (PVOD) and/or pulmonary capillary hemangiomatosis * Participants with PAH associated with Eisenmenger syndrome * Participants with moderate to large left-to-right shunts * Participants with cyanotic congenital cardiac lesions such as transposition of the great arteries, truncus arteriosus, univentricular heart or pulmonary atresia with ventricular septal defect, as well as Participants with Fontan-palliation * Participants with pulmonary hypertension due to lung disease * Previous treatment with Uptravi (selexipag) within 2 weeks prior to enrollment * Participants having received prostacyclin (epoprostenol) or prostacyclin analogs (that is, treprostinil, iloprost, beraprost) within 2 months prior to enrollment or are scheduled to receive any of these compounds during the trial * Treatment with another investigational drug within 4 weeks prior to enrollment * History, or current suspicion of intussusception or ileus or gastrointestinal obstruction as per investigator's judgment * Uncontrolled thyroid disease as per investigator judgment * Hemoglobin or hematocrit \< 75 percentage (%) of the lower limit of normal range * Known severe or moderate hepatic impairment * Clinical signs of hypotension that in the investigator's judgment would preclude initiation of a PAH-specific therapy * Participants with severe renal insufficiency * Known hypersensitivity to the investigational treatment or to any of the excipients of the drug formulations

Primary outcome measure(s)

Trial sites (34)

FacilityCityRegionStatus
Children'S Hospital Cardiac Care Center University Of Colorado Aurora Colorado
University of Iowa Hospital Iowa City Iowa
Seattle Children's Hospital Seattle Washington
State Institution Republican Scientific And Practical Center For Pediatric Surgery Minsk Belarus
Health Institution 4Th City Children'S Clinical Hospital Minsk Belarus
UZ Gent Ghent Belgium
Centre Hospitalier Sainte Justine Montreal Quebec
Beijing Anzhen Hospital Beijing China
Shanghai Childrens Medical Center Shanghai China
CHU Arnaud de Villeneuve Montpellier France
Hôpital Necker - Enfants Malades Paris France
Chu Hopital Des Enfants Toulouse France
Universitätsklinikum Freiburg Zentrum Freiburg im Breisgau Germany
Gottsegen Gyorgy Orszagos Kardiologiai Intezet, Felnott kardiologiai osztaly Budapest Hungary
Schneider Children's Medical Center Petach Tikvah Israel
Sheba Medical Center Ramat Gan Israel
Sarawak Heart Center Kota Samarahan Malaysia
Institut Jantung Negara (National Heart Institute) Kuala Lumpur Malaysia
Wojewodzki Szpital Specjalistyczny We Wroclawiu Wroclaw Poland
Kazan State Medical University Kazan' Russia
Scientific and Research Institution of Cardiovascular Diseases Complex Problems Kemerovo Russia
Moscow Scientific Research Institute For Pediatrics And Childrens Surgery Of Rosmedtechnologies Moscow Russia
Saint Petersburg State Pediatric Medical University Saint Petersburg Russia
Almazov National Medical Research Center Of The Ministry Of Health Of The Russian Federation Saint Petersburg Russia
Samara Regional Clinical Cardiological Dispensary Samara Russia
Univerzitetska Dečja Klinika Belgrade Serbia
Institut Za Zdravstvenu Zastitu Majke I Deteta Srbije Dr Vukan Cupic Belgrade Serbia
National Cheng Kung University Hospital Tainan Taiwan
National Taiwan University Hospital Taipei Taiwan
Municipal Enterprise Of The Dnipropetrovsk Regional Council Dnipro Ukraine
State Institution Of The Ministry Of Health Of Ukraine Kiev Ukraine
Lviv Regional Clinical Hospital Lviv Ukraine
Municipal Institution Of The Zaporizhzhya Regional Council Zaporizhzhya Ukraine
Great Ormond Street Hospital London United Kingdom
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03492177 on ClinicalTrials.gov ↗ ← All trials in the UK