Last updated on July 2019

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

Brief description of study

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.

Detailed Study Description

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 (g). 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).

Clinical Study Identifier: NCT03492177

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