Selexipag for Pulmonary Arterial Hypertension

Recruiting in Palo Alto (17 mi)

+38 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Actelion

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial aims to find the right dose of Selexipag for children with Pulmonary Arterial Hypertension (PAH). Selexipag helps by relaxing lung blood vessels, lowering blood pressure, and improving blood flow. The goal is to ensure the treatment is safe and effective for younger patients.

Research Team

Catherine Boisson

Principal Investigator

Actelion

Eligibility Criteria

This trial is for children aged 2-18 with Pulmonary Arterial Hypertension (PAH), weighing at least 9 kg. They must have a confirmed PAH diagnosis and be in WHO functional class II to III. Those on stable PAH-specific treatments can join, except if they've used selexipag recently or certain other PAH drugs within the last two months.

Inclusion Criteria

I have been diagnosed with PAH confirmed by a heart catheterization test.

My heart condition moderately affects my daily activities.

I have been on stable doses of ERA or PDE-5 inhibitors for 3 months, or I cannot take these medications.

See 4 more

Exclusion Criteria

You have a moderate to large opening between the left and right sides of your heart.

You have certain types of serious heart conditions from birth.

I have PAH linked to Eisenmenger syndrome.

See 12 more

Treatment Details

Interventions

Selexipag (Prostacyclin Receptor Agonist)

Trial OverviewThe study tests the drug selexipag (Uptravi) in children to find the right starting dose that matches adult exposure levels. It will look at how kids' bodies process the drug and its active metabolite, focusing on those between ages 2 and under 18 with PAH.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: open label selexipagExperimental Treatment1 Intervention

The first dose of selexipag (Uptravi) will be administered in the evening of Day 1 and will be based on the body weight. Thereafter selexipag will be administered twice daily (morning and evening). Selexipag will be up-titrated during the first 12 weeks, with weekly increments equal to the starting dose until the participants reach their individual maximum tolerated dose (iMTD) or until a maximum dose corresponding to their baseline weight category is achieved (which will be 8-fold of the corresponding starting dose). Up-titration is followed by a stable maintenance treatment period from Week 12 to Week 16, at the maximum tolerated dose. Thereafter, participants will be treated with selexipag as long as the treatment is beneficial to the participants, as per investigator's decision.