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)
Area Under the Plasma Concentration-time Curve Over a Dose Interval at Steady State of Selexipag and Its Metabolite ACT-333679 Combined (AUCτ, ss, Combined) — Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12) AUCτ, ss, combined was defined as the area under the plasma concentration-time curve over one dosing interval at steady state. AUCτ,ss,combined was calculated as 1/38 AUCτ,ss,selexipag plus 37/38 AUCτ,ss,ACT-333679.
Trial sites (34)
Facility
City
Region
Status
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
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.
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