A Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension (PAH)
This is a prospective, multicenter, open-label, randomized, controlled, parallel group, group-sequential, event-driven Phase 3 study to evaluate efficacy, safety and pharmacokinetics (PK) of macitentan in children.
- Pulmonary Arterial Hypertension
- Eligible Ages
- Between 2 Years and 17 Years
- Eligible Genders
- Accepts Healthy Volunteers
- Signed informed consent by the parent(s) or legally designated representative and assent from developmentally capable children prior to initiation of any study-mandated procedure.
- Males or females between ≥ 2 years and < 18 years of age.
- Subjects with body weight ≥ 10 kg at randomization.
- Pulmonary arterial hypertension (PAH) diagnosis, confirmed by historical right heart catheterization (mPAP ≥ 25 mmHg, and PAWP ≤ 15 mmHg, and PVRi > 3 WUxm2).
- PAH belonging to the Nice 2013 Updated Classification Group 1 (including subjects with Down syndrome) and of following etiologies:
- idiopathic PAH
- heritable PAH
- PAH associated with congenital heart disease (CHD)
- Drug or toxin induced PAH
- PAH associated with HIV
- PAH associated with connective tissue diseases (PAH-aCTD)
- WHO Functional class I to III.
- Females of childbearing potential must have a negative pregnancy test at Screening and at Baseline, and must agree to undertake monthly pregnancy tests, and to use a reliable method of contraception (if sexually active) up to the end of study (EOS).
- Subjects with PAH due to portal hypertension, schistosomiasis, or with pulmonary veno-occlusive disease and/or pulmonary capillary hemangiomatosis, and persistent pulmonary hypertension of the newborn.
- Subjects with PAH associated with Eisenmenger syndrome, or with moderate to large left-to-right shunts.
- Subjects receiving a combination of > 2 PAH-specific treatments at randomization.
- Treatment with i.v. or s.c. prostanoids within 4 weeks before randomization, unless given for vasoreactivity testing.
- Hemoglobin or hematocrit <75% of the lower limit of normal range
- Serum AST and/or ALT > 3 times the upper limit of normal range'
- Pregnancy (including family planning) or breastfeeding.
- Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol.
- Phase 3
- Study Type
- Intervention Model
- Parallel Assignment
- Primary Purpose
- None (Open Label)
|Macitentan is administered once daily via oral route||
|Standard-of-care as per site's clinical practice which may comprise treatment with PAH non-specific treatment and/or up to two PAH-specific medications excluding macitentan and i.v./s.c. prostanoids.||
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