A Clinical Study of to Confirm the Doses of Selexipag in Children With Pulmonary Arterial Hypertension
Condition: Pulmonary Arterial Hypertension
MeSH terms: Pulmonary Arterial Hypertension; Familial Primary Pulmonary Hypertension; Hypertension
Intervention: selexipag (Uptravi)
Intervention description: Film-coated tablets for oral administration
Intervention MeSH terms: Selexipag
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 63.0
Sex: All
Minimum age: 2 Years
Maximum age: 17 Years
Healthy volunteers: No
Keywords: Pediatric

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 criteria:

        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