Selexipag for Pulmonary Arterial Hypertension (SALTO Trial)

Palo Alto (17 mi)

Age: < 18

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Actelion

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?The purpose of this study is to evaluate whether the addition of selexipag to standard of care treatment delays disease progression in children with Pulmonary Arterial Hypertension (PAH) in comparison to placebo.

Eligibility Criteria

This trial is for children aged 2-18 with Pulmonary Arterial Hypertension (PAH) who are already on stable PAH treatment. They must weigh at least 9 kg and have a confirmed diagnosis through right heart catheterization. It's not for those with certain types of PAH, previous selexipag use, life-threatening diseases, or pregnancy.

Inclusion Criteria

I am between 2 and 18 years old and weigh at least 9 kilograms.

My heart condition allows me to perform light to moderate activities.

My PAH diagnosis was confirmed by a heart catheterization test.

Exclusion Criteria

I have a serious illness and am expected to live less than a year.

I have been treated with Uptravi before.

My PAH is caused by portal hypertension, schistosomiasis, or specific lung diseases.

My PAH is linked to Eisenmenger syndrome.

Treatment Details

The study tests if adding Selexipag to standard treatments can slow down the progression of PAH in children compared to a placebo. Participants will be randomly assigned to receive either Selexipag or a placebo alongside their current treatment.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: SelexipagExperimental Treatment1 Intervention

Participants will receive selexipag based on the body weight on Day 1 and will continue thereafter with twice daily dosing. Selexipag will be uptitrated during the first 12 weeks until the participants reaches the individual maximum tolerated dose (iMTD) or until a maximum dose corresponding to their baseline body-weight category is achieved. Uptitration is followed by a maintenance period after Week 12 until end of treatment (EOT), at the maximum tolerated dose.

Group II: PlaceboPlacebo Group1 Intervention

Participants will receive matching placebo based on the body weight on Day 1 and will continue thereafter with twice daily dosing.