Selexipag for Pulmonary Arterial Hypertension

Recruiting at 38 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

63 Participants Needed

Trial Summary

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.

What data supports the effectiveness of the drug Selexipag for treating pulmonary arterial hypertension?

Selexipag is shown to be effective in delaying disease progression and reducing the risk of hospitalization for pulmonary arterial hypertension (PAH). In the GRIPHON study, it significantly reduced the risk of complications related to PAH and improved exercise capacity compared to a placebo. ^{1 2 3 4 5}

Is selexipag safe for humans?

Selexipag is generally well tolerated in humans, with a safety profile similar to other treatments targeting the prostacyclin pathway, though it may cause some adverse events (unwanted side effects) that need careful management. ^{1 3 5 6 7}

How is the drug Selexipag different from other treatments for pulmonary arterial hypertension?

Selexipag is unique because it is an oral medication that specifically targets the prostacyclin receptor, helping to delay disease progression and reduce hospitalization risk for pulmonary arterial hypertension patients. Unlike traditional prostanoid therapies, it is designed to have fewer side effects while providing similar benefits. ^{4 5 8 9 10}

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.

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.

Timeline

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Selexipag: A Review in Pulmonary Arterial Hypertension. [2018]

2. England 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. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Selexipag, an Oral Prostacyclin-Receptor Agonist for Pulmonary Arterial Hypertension. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

Disease characteristics, treatments, and outcomes of patients with pulmonary arterial hypertension treated with selexipag in real-world settings from the SPHERE registry (SelexiPag: tHe usErs dRug rEgistry). [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Patient and disease characteristics of the first 500 patients with pulmonary arterial hypertension treated with selexipag in real-world settings from SPHERE. [2022]

6. England pubmed.ncbi.nlm.nih.gov

Real-world practice patterns and characteristics of adverse events with selexipag in Korean patients with pulmonary arterial hypertension. [2022]

7. England pubmed.ncbi.nlm.nih.gov

Selexipag for the treatment of pulmonary arterial hypertension. [2019]

8. Switzerland pubmed.ncbi.nlm.nih.gov

Selexipag-based triple combination therapy improves prognosis in Chinese pulmonary arterial hypertension patients. [2022]

9. United States pubmed.ncbi.nlm.nih.gov

Investigation of Potential Pharmacodynamic and Pharmacokinetic Interactions Between Selexipag and Warfarin in Healthy Male Subjects. [2019]

10. England pubmed.ncbi.nlm.nih.gov

An evaluation of selexipag for the treatment of pulmonary hypertension. [2022]