NCT03492177

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

A Prospective, Multicenter, Open Label, Single Arm, Phase 2 Study to Investigate the Safety, Tolerability and Pharmacokinetics of Selexipag in Children With Pulmonary Arterial Hypertension

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 63 (actual)
• Sponsor: Actelion · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

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.

Detailed description

The selection of the starting dose for pediatric participants is based on the 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 participants at a starting dose of 200 micrograms (mcg). As in adults, selexipag will be up-titrated to the individual maximum tolerated dose (iMTD) during the first 12 weeks. Approximately 60 participants will be enrolled in 3 different age cohorts to obtain at least 45 participants 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 participants from Cohort 1 at Week 12, a first interim analysis will be conducted to establish the dose-exposure relationship using a population PK model. The PK data from any participants in Cohort 2 who have completed their PK assessments at this time will be included in this first interim analysis. 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).

Conditions

• Pulmonary Arterial Hypertension

Interventions

Type	Name	Description
DRUG	selexipag (Uptravi)	Film-coated tablets for oral administration

Timeline

• Start date: 2018-07-23
• Primary completion: 2022-03-28
• Completion: 2026-12-31
• First posted: 2018-04-10
• Last updated: 2026-04-13
• Results posted: 2023-04-19

Locations

34 sites across 16 countries: United States, Belarus, Belgium, Canada, China, France, Germany, Hungary, Israel, Malaysia, Poland, Russia, Serbia, Taiwan, Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study