What side effects are associated with this type of intervention?

Common treatments for Pulmonary Arterial Hypertension (PAH), particularly prostacyclin analogues like epoprostenol, treprostinil, and iloprost, can cause side effects such as headache, nausea, vomiting, diarrhea, flushing, jaw pain, and potential site pain or infection with IV or subcutaneous administration. Selexipag, an oral prostacyclin receptor agonist, may have similar side effects but with fewer complications related to administration routes. Endothelin receptor antagonists (ERAs) like bosentan and macitentan can cause hepatotoxicity, anemia, headache, and peripheral edema. Phosphodiesterase-5 inhibitors (PDE5Is) such as sildenafil and tadalafil may lead to headaches, flushing, dyspepsia, and visual disturbances.

What prior FDA approvals exist?

The FDA has approved several treatments for Pulmonary Arterial Hypertension (PAH) that are similar to Selexipag, a Prostacyclin Receptor Agonist. These include other prostacyclin analogs such as Epoprostenol, Treprostinil, and Iloprost. Epoprostenol is administered intravenously, Treprostinil can be administered subcutaneously, intravenously, orally, or by inhalation, and Iloprost is administered by inhalation. These drugs work by mimicking the effects of prostacyclin, leading to vasodilation and inhibition of platelet aggregation, which helps to reduce the symptoms and progression of PAH.

What other types of research exist?

Promising novel alternatives to Selexipag for PAH patients include macitentan (an ERA with a favorable safety profile), riociguat (a soluble guanylate cyclase stimulator), and newer PDE5 inhibitors like tadalafil. Additionally, inhaled prostacyclin analogues such as iloprost and treprostinil have shown efficacy in improving functional status and may be considered based on patient-specific factors.

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Prostacyclin Receptor Agonist

Selexipag for Pulmonary Arterial Hypertension

Phase 2

Waitlist Available

Research Sponsored by Actelion

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Pulmonary arterial hypertension (PAH) diagnosis confirmed by documented historical right heart catheterization (RHC) performed at any time before participant's enrollment

Word Health Organization functional class (WHO FC) II to III

Must not have

Participants with PAH associated with Eisenmenger syndrome

Participants with pulmonary hypertension due to lung disease

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 1,week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. week 2, 4 and 6: pre-dose (up to week 12)

Awards & highlights

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Selexipag may cause side effects like headaches, diarrhea, jaw pain, muscle pain or cramps, nausea or vomiting, redness of skin or rash. The severity of these side effects can vary from child to child.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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My heart condition moderately affects my daily activities.

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have PAH linked to Eisenmenger syndrome.

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I have high blood pressure in my lungs because of lung disease.

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I have a known liver condition.

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My PAH is due to portal hypertension, schistosomiasis, PVOD, or pulmonary capillary hemangiomatosis.

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I have severe kidney problems.

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I haven't taken prostacyclin or similar drugs in the last 2 months and won't during the trial.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 1,week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. week 2, 4 and 6: pre-dose (up to week 12)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 1,week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. week 2, 4 and 6: pre-dose (up to week 12)

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 1,week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. week 2, 4 and 6: pre-dose (up to week 12) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Area Under the Plasma Concentration-time Curve Over a Dose Interval at Steady State of Selexipag and Its Metabolite ACT-333679 Combined (AUCτ, ss, Combined)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Pulmonary Arterial Hypertension (PAH) treatments target various pathways to reduce pulmonary vascular resistance and improve patient outcomes. Prostacyclin Receptor Agonists, like Selexipag, mimic the effects of prostacyclin, leading to vasodilation and inhibition of smooth muscle cell proliferation. Endothelin Receptor Antagonists (ERAs) block endothelin-1, a potent vasoconstrictor, thereby reducing vasoconstriction and vascular remodeling. Phosphodiesterase 5 Inhibitors (PDE5Is) increase cyclic GMP levels, promoting vasodilation and reducing pulmonary pressure. Soluble Guanylate Cyclase Stimulators enhance the nitric oxide pathway, leading to vasodilation and improved blood flow. These mechanisms are crucial for PAH patients as they help alleviate symptoms, improve exercise capacity, and potentially slow disease progression.

[Pharmacological characteristics and clinical study results of Selexipag (Uptravi® tablets), a selective prostacyclin receptor agonist].