Selexipag for Pulmonary Arterial Hypertension

(SALTO Trial)

Not currently recruiting at 184 trial locations

Overseen By Study Contact

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Actelion

Must be taking: Endothelin antagonists, PDE-5 inhibitors

Disqualifiers: Eisenmenger syndrome, Life expectancy <12 months, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 5 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether adding selexipag to standard care can slow the progression of Pulmonary Arterial Hypertension (PAH) in children. PAH is a condition where high blood pressure in the lungs' arteries makes breathing difficult and causes fatigue. Participants will receive either selexipag or a placebo (a pill with no active medicine) to compare results. Children struggling with PAH who are already on specific treatments might be suitable for this study. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but it requires that you have been on a stable dose of at least one PAH-specific treatment for at least 3 months before starting the study.

Is there any evidence suggesting that selexipag is likely to be safe for children with Pulmonary Arterial Hypertension?

Research has shown that selexipag is generally well-tolerated by people with pulmonary arterial hypertension (PAH). Studies have found that patients can use selexipag safely over a long period. Common side effects include worsening of PAH in about 17% of patients and headaches in about 4%. Some cases also reported right ventricular failure, a more serious condition.

Selexipag has been used alongside other treatments and has improved certain health indicators, suggesting it is generally safe with other therapies. These findings indicate that selexipag can be a safe option for managing PAH, though, like all medications, it carries some risk of side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for PAH?

Researchers are excited about Selexipag for pulmonary arterial hypertension (PAH) because it offers a unique approach compared to current treatments. Unlike standard options like endothelin receptor antagonists or phosphodiesterase-5 inhibitors, Selexipag specifically targets the prostacyclin pathway, which is crucial for widening blood vessels and reducing blood pressure in the lungs. This targeted action could mean better management of PAH symptoms and improved quality of life for patients. Additionally, the oral administration of Selexipag offers a convenient alternative to intravenous prostacyclin therapies, making it easier for patients to adhere to their treatment regimen.

What evidence suggests that selexipag might be an effective treatment for Pulmonary Arterial Hypertension?

Research has shown that selexipag effectively treats pulmonary arterial hypertension (PAH). It improves survival rates, with 60% of patients living for 10 years after starting treatment. Studies indicate that beginning selexipag within a year of diagnosis can reduce hospital visits and medical costs. Additionally, early use of selexipag lowers the risk of hospitalizations related to PAH and other health issues. This medication targets a specific part of the body to relax blood vessels, which can lower blood pressure in the lungs. In this trial, participants will receive either selexipag or a placebo to further evaluate its effectiveness. Overall, selexipag serves as a helpful treatment for managing PAH. ^{2 3 6 7 8}

Who Is on the Research Team?

Actelion Clinical Trial

Principal Investigator

Actelion

Are You a Good Fit for This Trial?

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 have been on a stable dose of treatment for pulmonary arterial hypertension for at least 3 months.

I have pulmonary arterial hypertension (PAH) and may also have Down syndrome.

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

Exclusion Criteria

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

You are allergic or very sensitive to selexipag or its ingredients.

Pregnant, planning to become pregnant, or lactating

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Up to 6 weeks

Treatment

Participants receive either selexipag or placebo with uptitration for 12 weeks followed by a maintenance period until end of treatment

Up to 5 years

Open-label extension

Participants may continue to receive selexipag for an additional 3 years

3 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

30 days

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• Selexipag

Trial Overview 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.

How Is the Trial Designed?

Group I: Selexipag Experimental Treatment 1 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: Placebo Placebo Group 1 Intervention

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

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

A 42-year-old woman with high-risk idiopathic pulmonary arterial hypertension (PAH) showed significant clinical and hemodynamic improvement after being treated with an off-label triple oral combination therapy that included selexipag, a phosphodiesterase-5 inhibitor, and an endothelin receptor antagonist.

This case suggests that oral combination therapies, like the one used in this patient, could be a viable alternative to parenteral prostacyclin analogues for high-risk PAH patients, warranting further investigation in controlled clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Upfront triple oral combination therapy including selexipag in a high-risk patient with idiopathic pulmonary arterial hypertension: a case report. Rossi, S., Pietrangelo, C., Pierdomenico, SD., et al.[2022]

Selexipag, an oral prostacyclin agonist approved for pulmonary arterial hypertension (PAH), has shown significant efficacy in reducing death and hospitalization rates in PAH patients during the phase 3 GRIPHON study, which involved multiple centers.

While selexipag improved patients' 6-minute walk distance, indicating better symptom management, it did not demonstrate a significant impact on overall mortality, highlighting the need for further studies to fully understand its long-term safety and efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Selexipag in Pulmonary Arterial Hypertension: Most Updated Evidence From Recent Preclinical and Clinical Studies. Ghosh, RK., Ball, S., Das, A., et al.[2018]

Selexipag is an effective treatment for pulmonary arterial hypertension (PAH), showing significant improvements in pulmonary artery obstruction and right ventricular function in both animal models and clinical trials, including a large study with 1156 patients.

In clinical trials, selexipag demonstrated good tolerability and a significant reduction in the risk of death or complications related to PAH, indicating its potential to prevent disease progression.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Pharmacological characteristics and clinical study results of Selexipag (Uptravi&#174; tablets), a selective prostacyclin receptor agonist]. Kuwano, K., Kosugi, K., Fuchikami, C., et al.[2021]

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 ... Among patients with pulmonary arterial hypertension treated with selexipag, the 10-year survival rate was 60%, according to findings presented ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37804160/

Impact of selexipag use within 12 months of pulmonary ... Selexipag initiation within 12 months of PAH diagnosis demonstrated reductions in all-cause hospitalization rate and medical costs.

3. sciencedirect.com sciencedirect.com/science/article/pii/S1053249823020272

Disease characteristics, treatments, and outcomes of ... Estimated 18-month overall survival rates were 89.4%, 84.2%, and 94.5% in the overall, newly, and previously initiated patient populations, respectively. CI, ...

4. jamanetwork.com jamanetwork.com/journals/jamanetworkopen/fullarticle/2823882

Novel Approach to Evaluate the Role of Selexipag in ... The authors reported that early exposure to selexipag was associated decreased risk for all-cause hospitalization, PAH-related hospitalization, ...

5. nejm.org nejm.org/doi/full/10.1056/NEJMoa1503184

Selexipag for the Treatment of Pulmonary Arterial ... Selexipag, an oral selective IP prostacyclin-receptor agonist, was shown to be beneficial in the treatment of pulmonary arterial hypertension.

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. jnjmedicalconnect.com jnjmedicalconnect.com/media/attestation/congresses/pulmonary-hypertension/2025/ishlt/longterm-survival-and-safety-with-selexipag-in-pulmonary-arterial-hypertension-pah-10year-results-fr.pdf

10-year data on oral selexipag: long-term survival, safety, ... OLE: open label extension; PAH: pulmonary arterial hypertension. Introduction. These analyses from GRIPHON and its OLE report survival among patients treated ...

8. academic.oup.com academic.oup.com/eurheartj/article/43/Supplement_2/ehac544.1932/6745401

Efficacy and safety of selexipag in real-life in patients with ... Selexipag, added as triple combination therapy in patients with PAH intermediate risk, improved risk variables, was well tolerated and achieved ...