Selexipag for Pulmonary Arterial Hypertension

Not currently recruiting at 49 trial locations

Overseen By Study Contact

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Actelion

Must be taking: Endothelin antagonists, PDE-5 inhibitors

Must not be taking: Prostacyclin analogs, Investigational drugs

Disqualifiers: Portal hypertension, Eisenmenger syndrome, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 16 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine the appropriate dose of selexipag for children with Pulmonary Arterial Hypertension (PAH), a serious condition where lung blood vessels narrow, hindering the heart's ability to pump blood. Researchers seek to assess whether the adult dose is safe and effective for children. The trial includes children diagnosed with PAH, particularly those whose daily activities are affected or who have related health issues such as congenital heart disease or conditions linked to drugs, HIV, or connective tissue disease. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must have a stable dose of endothelin receptor antagonists or phosphodiesterase type 5 inhibitors for at least 3 months before joining. If you are on prostacyclin or its analogs, you must stop them 2 months before enrolling.

Is there any evidence suggesting that selexipag is likely to be safe for humans?

Research has shown that selexipag has been tested for safety in people with pulmonary arterial hypertension (PAH). One study found that the most common side effect was a worsening of PAH, occurring in 17% of patients. Headaches and right-side heart problems were also reported, but less frequently, at 4%. Despite these side effects, selexipag has generally been well-tolerated.

Another study found that some people stopped using the drug due to side effects, with about 22% discontinuing within 18 months for this reason. However, overall, long-term studies support the safety of selexipag, indicating that its side effects are consistent and manageable over time.

These findings suggest that while some side effects exist, selexipag is considered safe enough for continued testing in clinical trials. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Pulmonary Arterial Hypertension?

Selexipag is unique because it works by targeting the prostacyclin pathway, which plays a crucial role in managing pulmonary arterial hypertension (PAH). Most treatments for PAH, like endothelin receptor antagonists or phosphodiesterase-5 inhibitors, focus on different pathways. Selexipag's mechanism helps relax blood vessels and reduce pressure in the lungs, offering a new approach to controlling symptoms and potentially improving patient outcomes. Researchers are excited about its twice-daily oral administration, which provides a convenient option compared to some existing therapies that require injections or inhalation.

What evidence suggests that selexipag might be an effective treatment for pulmonary arterial hypertension?

Research has shown that selexipag, which participants in this trial will receive, effectively treats pulmonary arterial hypertension (PAH). One study found that selexipag reduced the risk of death by 45% compared to other PAH treatments. Long-term data indicates that 60% of patients using selexipag remain alive after 10 years. Real-world studies also demonstrate that most patients tolerate selexipag well. These findings suggest that selexipag can be a helpful option for managing PAH. ^{2 3 6 7 8}

Who Is on the Research Team?

Catherine Boisson

Principal Investigator

Actelion

Are You a Good Fit for This Trial?

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.

Exclusion Criteria

You have certain types of serious heart conditions from birth.

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

Known hypersensitivity to the investigational treatment or to any of the excipients of the drug formulations

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive selexipag, starting with up-titration for 12 weeks followed by a stable maintenance period

16 weeks

Weekly visits during up-titration, then regular visits as per protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue treatment with selexipag as long as it is beneficial

What Are the Treatments Tested in This Trial?

Interventions

• Selexipag

Trial Overview

The 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.

How Is the Trial Designed?

Group I: open label selexipag Experimental Treatment 1 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.

Who Is Running the Clinical Trial?

Actelion

Lead Sponsor

Trials

192

Recruited

35,500+

Jean-Paul Clozel

Actelion

Chief Executive Officer since 1997

MD from University of Basel

Martine Clozel

Actelion

Chief Medical Officer since 1997

MD from University of Geneva

Published Research Related to This Trial

Initiating oral selexipag within 12 months of a pulmonary arterial hypertension (PAH) diagnosis significantly reduced the rate of all-cause hospitalizations by 24% compared to patients who did not start any prostacyclin pathway agent during the same period.

Patients who started selexipag also experienced lower overall medical costs, saving an average of $23,623, although there was no significant difference in PAH-related hospitalizations or disease progression between the two groups.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Impact of selexipag use within 12 months of pulmonary arterial hypertension diagnosis on hospitalizations and medical costs: A retrospective cohort study. Tsang, Y., Stokes, M., Kim, YJ., et al.[2023]

In a study of 113 Korean patients with pulmonary arterial hypertension (PAH) treated with selexipag over 24 weeks, adverse events (AEs) were more common during the dose-titration phase than during the maintenance phase, indicating careful monitoring is crucial during initial treatment.

The most frequent AEs included diarrhea, headache, nausea/vomiting, and myalgia, but importantly, the incidence and severity of these AEs did not increase with higher doses of selexipag, suggesting that higher doses can be safely managed without a corresponding rise in adverse effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real-world practice patterns and characteristics of adverse events with selexipag in Korean patients with pulmonary arterial hypertension. Chang, SA., Lee, SH., Choi, JH., et al.[2022]

Citations

1.

healio.com

healio.com/news/pulmonology/20250530/longterm-data-on-selexipag-for-pah-reveal-60-survival-rate-at-10-years

Long-term data on selexipag for PAH reveal 60% survival ...

The 10-year survival rate was 60%, according to findings presented at the 2025 International Society for Heart and Lung Transplantation Annual Meeting.

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC6381437/

Real-life data on Selexipag for the treatment of pulmonary ...

Furthermore, we recorded tolerability. Twenty-six patients were treated with selexipag, of whom 23 had PAH and three had chronic thromboembolic PH. At baseline, ...

3.

uptravihcp.com

uptravihcp.com/sphere-registry/

Real-World Hospitalization Analysis: UPTRAVI® (selexipag)

Disease characteristics, treatments, and outcomes of patients with pulmonary arterial hypertension treated with UPTRAVI® in real-world settings from the ...

4.

jamanetwork.com

jamanetwork.com/journals/jamanetworkopen/fullarticle/2823878

Early Addition of Selexipag to Double Therapy for ...

Early results from the ongoing EXPOSURE study showed a 45% reduction in risk of death with selexipag compared with other PAH therapies. In a ...

5.

sciencedirect.com

sciencedirect.com/science/article/pii/S2589790X2500647X

Real-world persistence of selexipag for pulmonary arterial ...

Real-world data from the Canadian population suggests low long-term persistence on selexipag though persistence is similar to reported rates for ...

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC8799580/

Long-Term Survival, Safety and Tolerability with Selexipag ...

The GRIPHON open-label extension study provides data on long-term safety, tolerability and survival for patients with pulmonary arterial hypertension (PAH) ...

7.

jhltonline.org

jhltonline.org/article/S1053-2498(23)02027-2/fulltext

tHe usErs dRug rEgistry

In patients with pulmonary arterial hypertension, 18-month discontinuation rates for adverse events were 22.0%, 32.0%, and 11.9%, and 18-month ...

8.

uptravihcp.com

uptravihcp.com/safety/

Safety Profile | UPTRAVI® (selexipag) HCP

The safety of UPTRAVI® was well established in GRIPHON, with adverse events being consistent across post hoc analyses and open-label extension.